AzTrials
AzTrials

Duchenne Muscular DystrophyNCT03760029

A Natural History Study In Chinese Male Patients With Duchenne Muscular Dystrophy

A study testing Visit frequency in people with Duchenne Muscular Dystrophy, run by Pfizer at 8 sites in China.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

This is a multicenter, prospective, single cohort study designed to describe the natural history of DMD in Chinese male patients. A total of approximately 330 subjects will be enrolled with the target number of subjects in each group as below: Group 1, Ambulatory subjects aged <6 years, approximately 100 subjects… Read more about this study

Who is it for?
Ages 0 years and older, men only
Conditions the study is for
Duchenne Muscular Dystrophy
What is being tested?
Visit frequency
Could I receive a placebo?
No, everyone receives an active treatment
How many people
312 participants
Where
8 sitesChina
Study dates
24 Jul 2019to 21 Mar 2023
Phase
Interventional
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 0 years and older
  • Men only
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • Chinese male patients with any age, diagnosed with DMD. Diagnosis must be confirmed in subject's medical history and by genetic testing obtained during routine clinical care for diagnostic purposes as reported from an appropriate regulated laboratory using a clinically validated genetic test (genetic testing is not provided by the sponsor).
  • Subjects who are >=4 years old must be receiving glucocorticosteroids for a minimum of 6 months prior to signing informed consent. There should be no significant change (<0.2 mg/kg) in dosage or dose regimen (not related to body weight change) for at least 3 months immediately prior to signing the informed consent. Subjects who are aged >4 years will be exempt from this requirement; those not taking GC will be eligible if the initiation of GC treatment in these subjects is considered inappropriate in the opinion of Investigators.

Who cannot join

  • Any injury which may impact functional testing. Previous injuries must be fully healed prior to consenting. Prior lower limb fractures must be fully healed and at least 3 months from injury date.
  • Presence or history of other musculoskeletal or neurologic disease or somatic disorder not related to DMD including pulmonary, cardiac, and cognitive diseases.
  • Subjects >=4 years old who have not completed the varicella vaccination.
  • Participation in other studies involving investigational drug(s) for a minimum of 90 days prior to signing the informed consent and/or during study participation.

Trial locations and contacts

8 sites in 1 country.

8 sites in 1 country

China8 sites

  • Peking University First Hospital

    Beijing, 100034

  • Beijing Children's Hospital, Capital Medical University

    Beijing, 100045

  • Children's Hospital of Chongqing Medical University

    Chongqing, Chongqing Municipality, 400014

  • Children's Hospital of Chongqing Medical University (Liangjiang Branch)

    Chongqing, Chongqing Municipality, 401122

  • The First Affiliated Hospital Of Fujian Medical University

    Fuzhou, Fujian, 350005

  • Huashan Hospital, Fudan University

    Shanghai, 200040

  • Affiliated children's hospital of fudan university

    Shanghai, 201102

  • Children's Hospital of Fudan University

    Shanghai, 201102

Other trials in China

About this study

Official title: A NATURAL HISTORY STUDY IN CHINESE MALE PATIENTS WITH DUCHENNE MUSCULAR DYSTROPHY

This is a multicenter, prospective, single cohort study designed to describe the natural history of DMD in Chinese male patients. A total of approximately 330 subjects will be enrolled with the target number of subjects in each group as below:

  • Group 1, Ambulatory subjects aged <6 years, approximately 100 subjects;
  • Group 2, Ambulatory subjects aged >=6 years, approximately 180 subjects;
  • Group 3, Non-ambulatory subjects, approximately 50 subjects. Subjects will visit sites every 6 months. Each subject will be observed for at least 24 months. All subjects will remain enrolled until the study completion date, such that some will have data collected after Month 24. Subjects, who complete Visit 5/Month 24 at least 6 months prior to study completion, will be asked to complete an additional visit at Month 30.
Being tested
Visit frequency
Medical subject headings
Muscular Dystrophy, Duchenne

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Visit frequencyOther

    All subjects need to visit sites more frequently than in routine clinical practice.

Study groups in NCT03760029
Study groupRoleWhat they receive
Study armOtherAll subjects in this study will be observed for 24-30 months.
Receives: Other: Visit frequency

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Age of Participants When They Failed to Walk

    Participant's age at life-altering clinical milestones- failure to walk was calculated based on the birthdate and the date of failure to walk as reported by caregiver during 30 months of this study. Participants who were not reported being failure to walk by their caregivers were censored on the day of their last visit. Kaplan-Meier method was used for analysis.

    Measured: Up to Month 30

  2. Age of Participants When They Failed to Stand

    Participant's age at life-altering clinical milestones- failure to stand was calculated based on the birthdate and the date of failure to stand as reported by caregiver during 30 months of this study. Participants who were not reported being failure to stand by their caregivers were censored on the day of their last visit. Kaplan-Meier method was used for analysis.

    Measured: Up to Month 30

  3. Age of Participants When They Failed to Self-feed

    Participant's age at life-altering clinical milestones- failure to self-feed during 30 months of this study was analyzed using the Kaplan-Meier method. Age was summarized in years.

    Measured: Up to Month 30

  4. Change From Baseline in Northstar Ambulatory Assessment (NSAA) Total Score at Month 6: Ambulatory Participants Aged >=3 Years

    NSAA is a 17-item test that grades performance of various functional skills using the following scale: 0 (unable to achieve goal independently), 1 (modified method but achieves goal with no physical assistance), or 2 ("normal"- no obvious modification of activity). The scale assesses activities required to remain functionally ambulant (e.g. rise from the floor), activities that can be difficult even early in the disease (example [e.g.] standing on heels) and activities that are known to progressively deteriorate over time (stand from a chair, walk). NSAA total score was calculated by adding the responses of all 17 items and ranged from 0 to 34, with higher scores indicating better function. NSAA was only performed in ambulatory participants aged >=3 years old as pre-specified in protocol.

    Measured: Baseline (Day 1) and Month 6

  5. Change From Baseline in NSAA Total Score at Month 12: Ambulatory Participants Aged >=3 Years

    NSAA is a 17-item test that grades performance of various functional skills using the following scale: 0 (unable to achieve goal independently), 1 (modified method but achieves goal with no physical assistance), or 2 ("normal"- no obvious modification of activity). The scale assesses activities required to remain functionally ambulant (e.g. rise from the floor), activities that can be difficult even early in the disease (e.g. standing on heels) and activities that are known to progressively deteriorate over time (stand from a chair, walk). NSAA total score was calculated by adding the responses of all 17 items and ranged from 0 to 34, with higher scores indicating better function. NSAA was only performed in ambulatory participants aged >=3 years old as pre-specified in protocol.

    Measured: Baseline (Day 1) and Month 12

  6. Change From Baseline in NSAA Total Score at Month 18: Ambulatory Participants Aged >=3 Years

    NSAA is a 17-item test that grades performance of various functional skills using the following scale: 0 (unable to achieve goal independently), 1 (modified method but achieves goal with no physical assistance), or 2 ("normal"- no obvious modification of activity). The scale assesses activities required to remain functionally ambulant (e.g. rise from the floor), activities that can be difficult even early in the disease (e.g. standing on heels) and activities that are known to progressively deteriorate over time (stand from a chair, walk). NSAA total score was calculated by adding the responses of all 17 items and ranged from 0 to 34, with higher scores indicating better function. NSAA was only performed in ambulatory participants aged >=3 years old as pre-specified in protocol.

    Measured: Baseline (Day 1) and Month 18

  7. Change From Baseline in NSAA Total Score at Month 24: Ambulatory Participants Aged >=3 Years

    NSAA is a 17-item test that grades performance of various functional skills using the following scale: 0 (unable to achieve goal independently), 1 (modified method but achieves goal with no physical assistance), or 2 ("normal"- no obvious modification of activity). The scale assesses activities required to remain functionally ambulant (e.g. rise from the floor), activities that can be difficult even early in the disease (e.g. standing on heels) and activities that are known to progressively deteriorate over time (stand from a chair, walk). NSAA total score was calculated by adding the responses of all 17 items and ranged from 0 to 34, with higher scores indicating better function. NSAA was only performed in ambulatory participants aged >=3 years old as pre-specified in protocol.

    Measured: Baseline (Day 1) and Month 24

  8. Change From Baseline in NSAA Total Score at Month 30: Ambulatory Participants Aged >=3 Years

    NSAA is a 17-item test that grades performance of various functional skills using the following scale: 0 (unable to achieve goal independently), 1 (modified method but achieves goal with no physical assistance), or 2 ("normal"- no obvious modification of activity). The scale assesses activities required to remain functionally ambulant (e.g. rise from the floor), activities that can be difficult even early in the disease (e.g. standing on heels) and activities that are known to progressively deteriorate over time (stand from a chair, walk). NSAA total score was calculated by adding the responses of all 17 items and ranged from 0 to 34, with higher scores indicating better function. NSAA was only performed in ambulatory participants aged >=3 years old as pre-specified in protocol.

    Measured: Baseline (Day 1) and Month 30

  9. Change From Baseline in Performance of Upper Limb (PUL) 2.0 Total Score at Month 6: Participants Aged >=10 Years

    PUL 2.0 scale is a 22-item scale used to assess the change that occurs in motor performance of the upper limb overtime from when a participant is still ambulant to the time participant loses all arm function when non-ambulant. PUL 2.0 includes an entry item to define broad starting functional level and 22 items subdivided into shoulder level (six items), mid-level (nine items), and distal level (seven items). Each dimension (shoulder, mid, distal) can be scored separately. There is maximum score of 12 for shoulder level, 17 for mid-level, and 13 for distal level. The total score was calculated by adding three level scores and ranged from 0-42. Higher score indicates better upper limb function. PUL 2.0 total score was assessed in participants aged >=10 years only as pre-specified in protocol.

    Measured: Baseline (Day 1) and Month 6

  10. Change From Baseline in PUL 2.0 Total Score at Month 12: Participants Aged >=10 Years

    PUL 2.0 scale is a 22-item scale used to assess the change that occurs in motor performance of the upper limb overtime from when a participant is still ambulant to the time participant loses all arm function when non-ambulant. PUL 2.0 includes an entry item to define broad starting functional level and 22 items subdivided into shoulder level (six items), mid-level (nine items), and distal level (seven items). Each dimension (shoulder, mid, distal) can be scored separately. There is maximum score of 12 for shoulder level, 17 for mid-level, and 13 for distal level. The total score was calculated by adding three level scores and ranged from 0-42. Higher score indicates better upper limb function. PUL 2.0 total score was assessed in participants aged >=10 years only as pre-specified in protocol.

    Measured: Baseline (Day 1) and Month 12

  11. Change From Baseline in PUL 2.0 Total Score at Month 18: Participants Aged >=10 Years

    PUL 2.0 scale is a 22-item scale used to assess the change that occurs in motor performance of the upper limb overtime from when a participant is still ambulant to the time participant loses all arm function when non-ambulant. PUL 2.0 includes an entry item to define broad starting functional level and 22 items subdivided into shoulder level (six items), mid-level (nine items), and distal level (seven items). Each dimension (shoulder, mid, distal) can be scored separately. There is maximum score of 12 for shoulder level, 17 for mid-level, and 13 for distal level. The total score was calculated by adding three level scores and ranged from 0-42. Higher score indicates better upper limb function. PUL 2.0 total score was assessed in participants aged >=10 years only as pre-specified in protocol.

    Measured: Baseline (Day 1) and Month 18

  12. Change From Baseline in PUL 2.0 Total Score at Month 24: Participants Aged >=10 Years

    PUL 2.0 scale is a 22-item scale used to assess the change that occurs in motor performance of the upper limb overtime from when a participant is still ambulant to the time participant loses all arm function when non-ambulant. PUL 2.0 includes an entry item to define broad starting functional level and 22 items subdivided into shoulder level (six items), mid-level (nine items), and distal level (seven items). Each dimension (shoulder, mid, distal) can be scored separately. There is maximum score of 12 for shoulder level, 17 for mid-level, and 13 for distal level. The total score was calculated by adding three level scores and ranged from 0-42. Higher score indicates better upper limb function. PUL 2.0 total score was assessed in participants aged >=10 years only as pre-specified in protocol.

    Measured: Baseline (Day 1) and Month 24

  13. Change From Baseline in PUL 2.0 Total Score at Month 30: Participants Aged >=10 Years

    PUL 2.0 scale is a 22-item scale used to assess the change that occurs in motor performance of the upper limb overtime from when a participant is still ambulant to the time participant loses all arm function when non-ambulant. PUL 2.0 includes an entry item to define broad starting functional level and 22 items subdivided into shoulder level (six items), mid-level (nine items), and distal level (seven items). Each dimension (shoulder, mid, distal) can be scored separately. There is maximum score of 12 for shoulder level, 17 for mid-level, and 13 for distal level. The total score was calculated by adding three level scores and ranged from 0-42. Higher score indicates better upper limb function. PUL 2.0 total score was assessed in participants aged >=10 years only as pre-specified in protocol.

    Measured: Baseline (Day 1) and Month 30

  14. Change From Baseline in Rise From Floor Velocity at Month 6: Ambulatory Participants Aged >=3 Years Only

    The rise from floor velocity was defined as the reciprocal of the time (in seconds) to rise from floor. The rise from floor test was performed only in ambulatory participants aged >=3 years old as pre-specified in the protocol.

    Measured: Baseline (Day 1) and Month 6

  15. Change From Baseline in Rise From Floor Velocity at Month 12: Ambulatory Participants Aged >=3 Years Only

    The rise from floor velocity was defined as the reciprocal of the time (in seconds) to rise from floor. The rise from floor test was performed only in ambulatory participants aged >=3 years old as pre-specified in the protocol.

    Measured: Baseline (Day 1) and Month 12

  16. Change From Baseline in Rise From Floor Velocity at Month 18: Ambulatory Participants Aged >=3 Years Only

    The rise from floor velocity was defined as the reciprocal of the time (in seconds) to rise from floor. The rise from floor test was performed only in ambulatory participants aged >=3 years old as pre-specified in the protocol.

    Measured: Baseline (Day 1) and Month 18

  17. Change From Baseline in Rise From Floor Velocity at Month 24: Ambulatory Participants Aged >=3 Years Only

    The rise from floor velocity was defined as the reciprocal of the time (in seconds) to rise from floor. The rise from floor test was performed only in ambulatory participants aged >=3 years old as pre-specified in the protocol.

    Measured: Baseline (Day 1) and Month 24

  18. Change From Baseline in Rise From Floor Velocity at Month 30: Ambulatory Participants Aged >=3 Years Only

    The rise from floor velocity was defined as the reciprocal of the time (in seconds) to rise from floor. The rise from floor test was performed only in ambulatory participants aged >=3 years old as pre-specified in the protocol.

    Measured: Baseline (Day 1) and Month 30

  19. Change From Baseline in 10 Meter Walk or Run Velocity at Month 6: Ambulatory Participants Aged >=3 Years

    The 10 meter walk or run test was performed as part of NSAA. The 10 meter walk or run velocity was defined as the reciprocal of the time (in seconds) to complete the 10 meter run or walk test. The 10 meter walk or run test was performed in ambulatory children >=3 years old only as pre-specified in the protocol.

    Measured: Baseline (Day 1) and Month 6

  20. Change From Baseline in 10 Meter Walk or Run Velocity at Month 12: Ambulatory Participants Aged >=3 Years

    The 10 meter walk or run test was performed as part of NSAA. The 10 meter walk or run velocity was defined as the reciprocal of the time (in seconds) to complete the 10 meter run or walk test. The 10 meter walk or run test was performed in ambulatory children >=3 years old only as pre-specified in the protocol.

    Measured: Baseline (Day 1) and Month 12

  21. Change From Baseline in 10 Meter Walk or Run Velocity at Month 18: Ambulatory Participants Aged >=3 Years

    The 10 meter walk or run test was performed as part of NSAA. The 10 meter walk or run velocity was defined as the reciprocal of the time (in seconds) to complete the 10 meter run or walk test. The 10 meter walk or run test was performed in ambulatory children >=3 years old only as pre-specified in the protocol.

    Measured: Baseline (Day 1) and Month 18

  22. Change From Baseline in 10 Meter Walk or Run Velocity at Month 24: Ambulatory Participants Aged >=3 Years

    The 10 meter walk or run test was performed as part of NSAA. The 10 meter walk or run velocity was defined as the reciprocal of the time (in seconds) to complete the 10 meter run or walk test. The 10 meter walk or run test was performed in ambulatory children >=3 years old only as pre-specified in the protocol.

    Measured: Baseline (Day 1) and Month 24

  23. Change From Baseline in 10 Meter Walk or Run Velocity at Month 30: Ambulatory Participants Aged >=3 Years

    The 10 meter walk or run test was performed as part of NSAA. The 10 meter walk or run velocity was defined as the reciprocal of the time (in seconds) to complete the 10 meter run or walk test. The 10 meter walk or run test was performed in ambulatory children >=3 years old only as pre-specified in the protocol.

    Measured: Baseline (Day 1) and Month 30

  24. Change From Baseline in Knee Extension of Muscle Strength at Month 6: Participants Aged >=5 Years

    Muscle strength was recorded by handheld myometry. Left and right knee extension was analyzed. The muscle strength test was only performed in participants >=5 years old as pre-specified in protocol.

    Measured: Baseline (Day 1) and Month 6

  25. Change From Baseline in Knee Extension of Muscle Strength at Month 12: Participants Aged >=5 Years

    Muscle strength was recorded by handheld myometry. Left and right knee extension was analyzed. The muscle strength test was only performed in participants >=5 years old as pre-specified in protocol.

    Measured: Baseline (Day 1) and Month 12

  26. Change From Baseline in Knee Extension of Muscle Strength at Month 18: Participants Aged >=5 Years

    Muscle strength was recorded by handheld myometry. Left and right knee extension was analyzed. The muscle strength test was only performed in participants >=5 years old as pre-specified in protocol.

    Measured: Baseline (Day 1) and Month 18

  27. Change From Baseline in Knee Extension of Muscle Strength at Month 24: Participants Aged >=5 Years

    Muscle strength was recorded by handheld myometry. Left and right knee extension was analyzed. The muscle strength test was only performed in participants >=5 years old as pre-specified in protocol.

    Measured: Baseline (Day 1) and Month 24

  28. Change From Baseline in Knee Extension of Muscle Strength at Month 30: Participants Aged >=5 Years

    Muscle strength was recorded by handheld myometry. Left and right knee extension was analyzed. The muscle strength test was only performed in participants >=5 years old as pre-specified in protocol.

    Measured: Baseline (Day 1) and Month 30

  29. Change From Baseline in Elbow Flexion of Muscle Strength at Month 6: Participants Aged >=5 Years

    Muscle strength was recorded by handheld myometry. Left and right elbow flexion were analyzed. The muscle strength test was only performed in participants >=5 years old as pre-specified in the protocol.

    Measured: Baseline (Day 1) and Month 6

  30. Change From Baseline in Elbow Flexion of Muscle Strength at Month 12: Participants Aged >=5 Years

    Muscle strength was recorded by handheld myometry. Left and right elbow flexion were analyzed. The muscle strength test was only performed in participants >=5 years old as pre-specified in the protocol.

    Measured: Baseline (Day 1) and Month 12

  31. Change From Baseline in Elbow Flexion of Muscle Strength at Month 18: Participants Aged >=5 Years

    Muscle strength was recorded by handheld myometry. Left and right elbow flexion were analyzed. The muscle strength test was only performed in participants >=5 years old as pre-specified in the protocol.

    Measured: Baseline (Day 1) and Month 18

  32. Change From Baseline in Elbow Flexion of Muscle Strength at Month 24: Participants Aged >=5 Years

    Muscle strength was recorded by handheld myometry. Left and right elbow flexion were analyzed. The muscle strength test was only performed in participants >=5 years old as pre-specified in the protocol.

    Measured: Baseline (Day 1) and Month 24

  33. Change From Baseline in Elbow Flexion of Muscle Strength at Month 30: Participants Aged >=5 Years

    Muscle strength was recorded by handheld myometry. Left and right elbow flexion were analyzed. The muscle strength test was only performed in participants >=5 years old as pre-specified in the protocol.

    Measured: Baseline (Day 1) and Month 30

  34. Change From Baseline in Elbow Extension of Muscle Strength at Month 6: Participants Aged >=5 Years

    Muscle strength was recorded by handheld myometry. Left and right elbow extension were analyzed. The muscle strength test was only performed in participants >=5 years old as pre-specified in protocol.

    Measured: Baseline (Day 1) and Month 6

  35. Change From Baseline in Elbow Extension Muscle Strength at Month 12: Participants Aged >=5 Years

    Muscle strength was recorded by handheld myometry. Left and right elbow extension were analyzed. The muscle strength test was only performed in participants >=5 years old as pre-specified in protocol.

    Measured: Baseline (Day 1) and Month 12

  36. Change From Baseline in Elbow Extension Muscle Strength at Month 18: Participants Aged >=5 Years

    Muscle strength was recorded by handheld myometry. Left and right elbow extension were analyzed. The muscle strength test was only performed in participants >=5 years old as pre-specified in protocol.

    Measured: Baseline (Day 1) and Month 18

  37. Change From Baseline in Elbow Extension Muscle Strength at Month 24: Participants Aged >=5 Years

    Muscle strength was recorded by handheld myometry. Left and right elbow extension were analyzed. The muscle strength test was only performed in participants >=5 years old as pre-specified in protocol.

    Measured: Baseline (Day 1) and Month 24

  38. Change From Baseline in Elbow Extension Muscle Strength at Month 30: Participants Aged >=5 Years

    Muscle strength was recorded by handheld myometry. Left and right elbow extension were analyzed. The muscle strength test was only performed in participants >=5 years old as pre-specified in protocol.

    Measured: Baseline (Day 1) and Month 30

  39. Change From Baseline in Shoulder Abduction of Muscle Strength at Month 6: Participants Aged >=5 Years

    Muscle strength was recorded by handheld myometry. Left and right shoulder abduction were analyzed. The muscle strength test was only performed in participants >=5 years old as pre-specified in the protocol.

    Measured: Baseline (Day 1) and Month 6

  40. Change From Baseline in Shoulder Abduction of Muscle Strength at Month 12: Participants Aged >=5 Years

    Muscle strength was recorded by handheld myometry. Left and right shoulder abduction were analyzed. The muscle strength test was only performed in participants >=5 years old as pre-specified in the protocol.

    Measured: Baseline (Day 1) and Month 12

  41. Change From Baseline in Shoulder Abduction of Muscle Strength at Month 18: Participants Aged >=5 Years

    Muscle strength was recorded by handheld myometry. Left and right shoulder abduction were analyzed. The muscle strength test was only performed in participants >=5 years old as pre-specified in the protocol.

    Measured: Baseline (Day 1) and Month 18

  42. Change From Baseline in Shoulder Abduction of Muscle Strength at Month 24: Participants Aged >=5 Years

    Muscle strength was recorded by handheld myometry. Left and right shoulder abduction were analyzed. The muscle strength test was only performed in participants >=5 years old as pre-specified in the protocol.

    Measured: Baseline (Day 1) and Month 24

  43. Change From Baseline in Shoulder Abduction of Muscle Strength at Month 30: Participants Aged >=5 Years

    Muscle strength was recorded by handheld myometry. Left and right shoulder abduction were analyzed. The muscle strength test was only performed in participants >=5 years old as pre-specified in the protocol.

    Measured: Baseline (Day 1) and Month 30

  44. Change From Baseline in Range of Motion (ROM) at Bilateral Ankles at Month 6

    Range of motion was evaluated by using goniometry to record any occurrences of ankle contractures. The ROM at left and right ankles were measured in degrees of passive dorsiflexion.

    Measured: Baseline (Day 1) and Month 6

  45. Change From Baseline in ROM at Bilateral Ankles at Month 12

    Range of motion was evaluated by using goniometry to record any occurrences of ankle contractures. The ROM at left and right ankles were measured in degrees of passive dorsiflexion.

    Measured: Baseline (Day 1) and Month 12

  46. Change From Baseline in ROM at Bilateral Ankles at Month 18

    Range of motion was evaluated by using goniometry to record any occurrences of ankle contractures. The ROM at left and right ankles were measured in degrees of passive dorsiflexion.

    Measured: Baseline (Day 1) and Month 18

  47. Change From Baseline in ROM at Bilateral Ankles at Month 24

    Range of motion was evaluated by using goniometry to record any occurrences of ankle contractures. The ROM at left and right ankles were measured in degrees of passive dorsiflexion.

    Measured: Baseline (Day 1) and Month 24

  48. Change From Baseline in ROM at Bilateral Ankles at Month 30

    Range of motion was evaluated by using goniometry to record any occurrences of ankle contractures. The ROM at left and right ankles were measured in degrees of passive dorsiflexion.

    Measured: Baseline (Day 1) and Month 30

  49. Change From Baseline in ROM at Bilateral Elbows at Month 6

    Range of motion was evaluated by using goniometry to record any occurrences of elbow contractures. The ROM at left and right elbows were measured in degrees of passive extension.

    Measured: Baseline (Day 1) and Month 6

  50. Change From Baseline in ROM at Bilateral Elbows at Month 12

    Range of motion was evaluated by using goniometry to record any occurrences of elbow contractures. The ROM at left and right elbows were measured in degrees of passive extension.

    Measured: Baseline (Day 1) and Month 12

  51. Change From Baseline in ROM at Bilateral Elbows at Month 18

    Range of motion was evaluated by using goniometry to record any occurrences of elbow contractures. The ROM at left and right elbows were measured in degrees of passive extension.

    Measured: Baseline (Day 1) and Month 18

  52. Change From Baseline in ROM at Bilateral Elbows at Month 24

    Range of motion was evaluated by using goniometry to record any occurrences of elbow contractures. The ROM at left and right elbows were measured in degrees of passive extension.

    Measured: Baseline (Day 1) and Month 24

  53. Change From Baseline in ROM at Bilateral Elbows at Month 30

    Range of motion was evaluated by using goniometry to record any occurrences of elbow contractures. The ROM at left and right elbows were measured in degrees of passive extension.

    Measured: Baseline (Day 1) and Month 30

  54. Change From Baseline in Percent Predicted Forced Vital Capacity (%pFVC) at Month 12: Participants Aged >=6 Years

    Forced vital capacity (FVC) is the volume of air that can be maximally forcefully exhaled after taking the deepest breath possible and was measured using spirometry. The percent predicted FVC was calculated from FVC (measured in liter) according to age, height (estimated height as derived from the ulna length for non-ambulatory participants), ethnicity, and gender using multi-ethnic reference values for spirometry. The pulmonary function assessments were performed in participants aged >=6 years as pre-specified in the protocol.

    Measured: Baseline (Day 1) and Month 12

  55. Change From Baseline in %pFVC at Month 24: Participants Aged >=6 Years

    FVC is the volume of air that can be maximally forcefully exhaled after taking the deepest breath possible and was measured using spirometry. The percent predicted FVC was calculated from FVC (measured in liter) according to age, height (estimated height as derived from the ulna length for non-ambulatory participants), ethnicity, and gender using multi-ethnic reference values for spirometry. The pulmonary function assessments were performed in participants aged >=6 years as pre-specified in the protocol.

    Measured: Baseline (Day 1) and Month 24

  56. Change From Baseline in %pFVC at Month 30: Participants Aged >=6 Years

    FVC is the volume of air that can be maximally forcefully exhaled after taking the deepest breath possible and was measured using spirometry. The percent predicted FVC was calculated from FVC (measured in liter) according to age, height (estimated height as derived from the ulna length for non-ambulatory participants), ethnicity, and gender using multi-ethnic reference values for spirometry. The pulmonary function assessments were performed in participants aged >=6 years as pre-specified in the protocol.

    Measured: Baseline (Day 1) and Month 30

  57. Change From Baseline in %pFVC at Month 12: Participants Aged >=6 Years (Unplanned Analysis)

    FVC is the volume of air that can be maximally forcefully exhaled after taking the deepest breath possible and was measured using spirometry. The percent predicted FVC was calculated from FVC (measured in liter) according to age, height (estimated height as derived from the ulna length for non-ambulatory participants), ethnicity, and gender using multi-ethnic reference values for spirometry. The pulmonary function assessments were performed in participants aged >=6 years as pre-specified in the protocol. Incorrect data were addressed by unplanned analysis.

    Measured: Baseline (Day 1) and Month 12

  58. Change From Baseline in %pFVC at Month 24: Participants Aged >=6 Years (Unplanned Analysis)

    FVC is the volume of air that can be maximally forcefully exhaled after taking the deepest breath possible and was measured using spirometry. The percent predicted FVC was calculated from FVC (measured in liter) according to age, height (estimated height as derived from the ulna length for non-ambulatory participants), ethnicity, and gender using multi-ethnic reference values for spirometry. The pulmonary function assessments were performed in participants aged >=6 years as pre-specified in the protocol. Incorrect data were addressed by unplanned analysis.

    Measured: Baseline (Day 1) and Month 24

  59. Change From Baseline in %pFVC at Month 30: Participants Aged >=6 Years (Unplanned Analysis)

    FVC is the volume of air that can be maximally forcefully exhaled after taking the deepest breath possible and was measured using spirometry. The percent predicted FVC was calculated from FVC (measured in liter) according to age, height (estimated height as derived from the ulna length for non-ambulatory participants), ethnicity, and gender using multi-ethnic reference values for spirometry. The pulmonary function assessments were performed in participants aged >=6 years as pre-specified in the protocol. Incorrect data were addressed by unplanned analysis.

    Measured: Baseline (Day 1) and Month 30

  60. Change From Baseline in Percent Predicted Forced Expiratory Volume in One Second (%pFEV1) at Month 12: Participants Aged >=6 Years

    Forced expiratory volume in one second (FEV1) is the volume of air forcefully exhaled in 1 second and was measured using spirometry. The %pFEV1 was calculated from FEV1 (measured in liter) according to age, height (estimated height as derived from the ulna length for non-ambulatory participants), ethnicity, and gender using multi-ethnic reference values for Spirometry. The pulmonary function assessments were performed only in participants >=6 years old as pre-specified in protocol.

    Measured: Baseline (Day 1) and Month 12

  61. Change From Baseline in %pFEV1 at Month 24: Participants Aged >=6 Years

    FEV1 is the volume of air forcefully exhaled in 1 second and was measured using spirometry. The %pFEV1 was calculated from FEV1 (measured in liter) according to age, height (estimated height as derived from the ulna length for non-ambulatory participants), ethnicity, and gender using multi-ethnic reference values for Spirometry. The pulmonary function assessments were performed only in participants >=6 years old as pre-specified in protocol.

    Measured: Baseline (Day 1) and Month 24

  62. Change From Baseline in %pFEV1 at Month 30: Participants Aged >=6 Years

    FEV1 is the volume of air forcefully exhaled in 1 second and was measured using spirometry. The %pFEV1 was calculated from FEV1 (measured in liter) according to age, height (estimated height as derived from the ulna length for non-ambulatory participants), ethnicity, and gender using multi-ethnic reference values for Spirometry. The pulmonary function assessments were performed only in participants >=6 years old as pre-specified in protocol.

    Measured: Baseline (Day 1) and Month 30

  63. Change From Baseline in %pFEV1 at Month 12: Participants Aged >=6 Years (Unplanned Analysis)

    FEV1 is the volume of air forcefully exhaled in 1 second and was measured using spirometry. The %pFEV1 was calculated from FEV1 (measured in liter) according to age, height (estimated height as derived from the ulna length for non-ambulatory participants), ethnicity, and gender using multi-ethnic reference values for Spirometry. The pulmonary function assessments were performed only in participants >=6 years old as pre-specified in protocol. Incorrect data were addressed by unplanned analysis.

    Measured: Baseline (Day 1) and Month 12

  64. Change From Baseline in %pFEV1 at Month 24: Participants Aged >=6 Years (Unplanned Analysis)

    FEV1 is the volume of air forcefully exhaled in 1 second and was measured using spirometry. The %pFEV1 was calculated from FEV1 (measured in liter) according to age, height (estimated height as derived from the ulna length for non-ambulatory participants), ethnicity, and gender using multi-ethnic reference values for Spirometry. The pulmonary function assessments were performed only in participants >=6 years old as pre-specified in protocol. Incorrect data were addressed by unplanned analysis.

    Measured: Baseline (Day 1) and Month 24

  65. Change From Baseline in %pFEV1 at Month 30: Participants Aged >=6 Years (Unplanned Analysis)

    FEV1 is the volume of air forcefully exhaled in 1 second and was measured using spirometry. The %pFEV1 was calculated from FEV1 (measured in liter) according to age, height (estimated height as derived from the ulna length for non-ambulatory participants), ethnicity, and gender using multi-ethnic reference values for Spirometry. The pulmonary function assessments were performed only in participants >=6 years old as pre-specified in protocol. Incorrect data were addressed by unplanned analysis.

    Measured: Baseline (Day 1) and Month 30

  66. Change From Baseline in Maximum Inspiratory Pressure at Month 12: Participants Aged >=6 Years

    The pulmonary function assessments were performed only in participants >=6 years old as pre-specified in protocol.

    Measured: Baseline (Day 1) and Month 12

  67. Change From Baseline in Maximum Inspiratory Pressure at Month 24: Participants Aged >=6 Years

    The pulmonary function assessments were performed only in participants >=6 years old as pre-specified in protocol.

    Measured: Baseline (Day 1) and Month 24

  68. Change From Baseline in Maximum Inspiratory Pressure at Month 30: Participants Aged >=6 Years

    The pulmonary function assessments were performed only in participants >=6 years old as pre-specified in protocol.

    Measured: Baseline (Day 1) and Month 30

  69. Change From Baseline in Maximum Expiratory Pressure at Month 12: Participants Aged >=6 Years

    The pulmonary function assessments were performed only in participants >=6 years old as pre-specified in SAP.

    Measured: Baseline (Day 1) and Month 12

  70. Change From Baseline in Maximum Expiratory Pressure at Month 24: Participants Aged >=6 Years

    The pulmonary function assessments were performed only in participants >=6 years old as pre-specified in SAP.

    Measured: Baseline (Day 1) and Month 24

  71. Change From Baseline in Maximum Expiratory Pressure at Month 30: Participants Aged >=6 Years

    The pulmonary function assessments were performed only in participants >=6 years old as pre-specified in SAP.

    Measured: Baseline (Day 1) and Month 30

  72. Change From Baseline in Peak Cough Flow at Month 12: Participants Aged >=6 Years

    The pulmonary function assessments were performed only in participants >=6 years old as pre-specified in protocol.

    Measured: Baseline (Day 1) and Month 12

  73. Change From Baseline in Peak Cough Flow at Month 24: Participants Aged >=6 Years

    The pulmonary function assessments were performed only in participants >=6 years old as pre-specified in protocol.

    Measured: Baseline (Day 1) and Month 24

  74. Change From Baseline in Peak Cough Flow at Month 30: Participants Aged >=6 Years

    The pulmonary function assessments were performed only in participants >=6 years old as pre-specified in protocol.

    Measured: Baseline (Day 1) and Month 30

  75. Change From Baseline in Peak Cough Flow at Month 12: Participants Aged >=6 Years (Unplanned Analysis)

    The pulmonary function assessments were performed only in participants >=6 years old as pre-specified in protocol. Incorrect data were addressed by unplanned analysis.

    Measured: Baseline (Day 1) and Month 12

  76. Change From Baseline in Peak Cough Flow at Month 24: Participants Aged >=6 Years (Unplanned Analysis)

    The pulmonary function assessments were performed only in participants >=6 years old as pre-specified in protocol. Incorrect data were addressed by unplanned analysis.

    Measured: Baseline (Day 1) and Month 24

  77. Change From Baseline in Peak Cough Flow at Month 30: Participants Aged >=6 Years (Unplanned Analysis)

    The pulmonary function assessments were performed only in participants >=6 years old as pre-specified in protocol. Incorrect data were addressed by unplanned analysis.

    Measured: Baseline (Day 1) and Month 30

  78. Change From Baseline in Left Ventricular Ejection Fraction (LVEF) at Month 12: Participants Aged >=6 Years

    LVEF was the percentage of blood that was ejected out of left ventricle with each contraction, estimated by echocardiography. The LVEF was only performed in participants >=6 years old as pre-specified in protocol.

    Measured: Baseline (Day 1) and Month 12

  79. Change From Baseline in LVEF at Month 24: Participants Aged >=6 Years

    LVEF was the percentage of blood that was ejected out of left ventricle with each contraction, estimated by echocardiography. The LVEF was only performed in participants >=6 years old as pre-specified in protocol.

    Measured: Baseline (Day 1) and Month 24

  80. Change From Baseline in LVEF at Month 30: Participants Aged >=6 Years

    LVEF was the percentage of blood that was ejected out of left ventricle with each contraction, estimated by echocardiography. The LVEF was only performed in participants >=6 years old as pre-specified in protocol.

    Measured: Baseline (Day 1) and Month 30

  81. Change From Baseline in LVEF at Month 12: Participants Aged >=6 Years (Unplanned Analysis)

    LVEF was the percentage of blood that was ejected out of left ventricle with each contraction, estimated by echocardiography. The LVEF was only performed in participants >=6 years old as pre-specified in protocol. Incorrect data were addressed by unplanned analysis.

    Measured: Baseline (Day 1) and Month 12

  82. Change From Baseline in LVEF at Month 24: Participants Aged >=6 Years (Unplanned Analysis)

    LVEF was the percentage of blood that was ejected out of left ventricle with each contraction, estimated by echocardiography. The LVEF was only performed in participants >=6 years old as pre-specified in protocol. Incorrect data were addressed by unplanned analysis.

    Measured: Baseline (Day 1) and Month 24

  83. Change From Baseline in LVEF at Month 30: Participants Aged >=6 Years (Unplanned Analysis)

    LVEF was the percentage of blood that was ejected out of left ventricle with each contraction, estimated by echocardiography. The LVEF was only performed in participants >=6 years old as pre-specified in protocol. Incorrect data were addressed by unplanned analysis.

    Measured: Baseline (Day 1) and Month 30

  84. Change From Baseline in Wechsler Intelligence Scale for Children (WISC)-IV Score at Month 24: Ambulatory Participants >= 6 to <=16 Years

    WISC-IV is an individually administered intelligence test for children between the ages of 6 and 16. The WISC-IV Composites are: Verbal Comprehension, Perceptual Reasoning, Working Memory, and Processing Speed. Scores from the Composites constitute the WISC-IV Full Scale IQ score which ranges from 40 (Exceptionally Low) to 160 (Exceptionally Superior), higher scores indicated more intelligence. The WISC was only performed in ambulatory participants >= 6 to <=16 years old as pre-specified in the protocol.

    Measured: Baseline (Day 1) and Month 24

Secondary outcomes (29)

  1. Number of Participants With Type of DMD Mutation

    Number of participants as per type of mutation: exon deletion, exon duplication, point mutation, small insertion, small deletion and others is presented in this outcome measure. One participant could have more than 1 mutation type.

    Measured: Up to Month 30

  2. Number of Participants With Each Affected Exon by Mutation Types

    Number of participants with each affected exon by mutation types is presented in this outcome measure. Only those categories with non-zero values have been reported.

    Measured: Up to Month 30

  3. Number of Participants With DMD Mutations Affecting Any Exon Between Exon 9 and Exon 13 or Deletion That Affects Both Exon 29 and Exon 30

    Number of participants with DMD mutations affecting any exon between exon 9 and exon 13 or deletion that affects both exon 29 and exon 30 is presented in this outcome measure.

    Measured: Up to Month 30

  4. Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Global Functioning Scale and Each Core Scale Score (Pediatric Parent Report) at Months 6, 12, 18, 24 and 30

    The PODCI is a participant-reported assessment of musculoskeletal health intended for use in children and adolescents. The pediatric version was intended for completion by parents or caregivers of children <=10 years old. It included a Global Function Scale along with core scales: Upper Extremity and Physical Function Core Scale (8 items); Transfer and Basic Mobility Core Scale (11 items); Sports and Physical Functioning Core Scale (21 items); Pain/Comfort Core Scale (3 items); Happiness Core Scale (5 items). The scores of each PODCI global functioning scale and core scales are averaged over the number of items answered (omitted no entry items). Mean of the rescaled values is multiplied by a constant so that each global functioning scale and core scales has a final range of values between 0-100. The higher scores represent less disability and better functioning.

    Measured: Baseline (Day 1) and Months 6, 12, 18, 24 and 30

  5. Change From Baseline in PODCI Global Functioning Scale and Each Core Scale Score (Adolescent Parent Report) at Months 6, 12, 18, 24 and 30

    The PODCI is a participant-reported assessment of musculoskeletal health intended for use in children and adolescents. The pediatric version was intended for completion by parents or caregivers of children <=10 years old. It included a Global Function Scale along with core scales: Upper Extremity and Physical Function Core Scale (8 items); Transfer and Basic Mobility Core Scale (11 items); Sports and Physical Functioning Core Scale (21 items); Pain/Comfort Core Scale (3 items); Happiness Core Scale (5 items). The scores of each PODCI global functioning scale and core scales are averaged over the number of items answered (omitted no entry items). Mean of the rescaled values is multiplied by a constant so that each global functioning scale and core scales has a final range of values between 0-100. The higher scores represent less disability and better functioning.

    Measured: Baseline (Day 1) and Months 6, 12, 18, 24 and 30

  6. Change From Baseline in PODCI Global Functioning Scale and Each Core Scale Score (Adolescent Self Report) at Months 6, 12, 18, 24 and 30

    The PODCI is a participant-reported assessment of musculoskeletal health intended for use in children and adolescents. The pediatric version was intended for completion by parents or caregivers of children <=10 years old. It included a Global Function Scale along with core scales: Upper Extremity and Physical Function Core Scale (8 items); Transfer and Basic Mobility Core Scale (11 items); Sports and Physical Functioning Core Scale (21 items); Pain/Comfort Core Scale (3 items); Happiness Core Scale (5 items). The scores of each PODCI global functioning scale and core scales are averaged over the number of items answered (omitted no entry items). Mean of the rescaled values is multiplied by a constant so that each global functioning scale and core scales has a final range of values between 0-100. The higher scores represent less disability and better functioning.

    Measured: Baseline (Day 1) and Months 6, 12, 18, 24 and 30

  7. Number of Participants According to Response to Each European Quality of Life (EuroQoL) 5 Dimension 3 Level (EQ-5D-3L) 5 Dimensions at Month 12

    EQ-5D-3L is a participant completed questionnaire designed to assess impact on health related quality of life. It is comprised of five dimensions: mobility, self-care, usual activities, pain/discomfort and anxiety/depression. The responses record three levels of severity (no problems/some or moderate problems/extreme problems) within a particular EQ-5D dimension. EQ-5D-3L assessment was only performed in participants >= 16 years old as pre-specified in protocol.

    Measured: Months 12

  8. Number of Participants According to Response to Each EQ-5D-3L 5 Dimensions at Month 24

    EQ-5D-3L is a participant completed questionnaire designed to assess impact on health related quality of life. It is comprised of five dimensions: mobility, self-care, usual activities, pain/discomfort and anxiety/depression. The responses record three levels of severity (no problems/some or moderate problems/extreme problems) within a particular EQ-5D dimension. EQ-5D-3L assessment was only performed in participants >= 16 years old as pre-specified in protocol.

    Measured: Month 24

  9. Number of Participants According to Response to Each EQ-5D-3L 5 Dimensions at Month 30

    EQ-5D-3L is a participant completed questionnaire designed to assess impact on health related quality of life. It is comprised of five dimensions: mobility, self-care, usual activities, pain/discomfort and anxiety/depression. The responses record three levels of severity (no problems/some or moderate problems/extreme problems) within a particular EQ-5D dimension. EQ-5D-3L assessment was only performed in participants >= 16 years old as pre-specified in protocol.

    Measured: Month 30

  10. Change From Baseline in EQ-5D-3L Visual Analogue Score (VAS) Assessment at Months 12, 24 and 30

    EQ-5D-3L is a participant completed questionnaire designed to assess impact on health related quality of life. EQ-VAS recorded the participant's self-rated health on a vertical scale ranging from 0 (worst imaginable health state) to 100 (best imaginable health state), where higher scores indicated better quality of life. EQ-VAS assessment was only performed in the participants >= 16 years old.

    Measured: Baseline (Day 1) and Months 12, 24 and 30

  11. Change From Baseline in EQ-5D-3L Index Score at Months 12, 24 and 30

    EQ-5D-3L index score, participants rated their current health state on 5 single-item dimensions: mobility, self-care, usual activities, pain/discomfort, and anxiety/depression with each dimension having three levels of severity: 1=no problems, 2=some problems and 3=extreme problems. Scoring formula developed by EuroQol Group assigns a utility value for each domain in the profile. Score was transformed and results in a total index score range of 0 to 1.00. Higher scores indicating a better quality of life. EQ-5D-3L was only performed in participants >= 16 years old.

    Measured: Baseline (Day 1) and Months 12, 24, and 30

  12. Number of Participants According to Response to Each EuroQoL 5 Dimension Youth (EQ-5D-Y) 5 Dimensions at Month 12

    EQ-5D-Y is participant completed questionnaire designed to assess impact on health related quality of life in children and adolescents <16 years old. It is comprised of five dimensions: mobility, self-care, usual activities, pain/discomfort and anxiety/depression. Each dimension has 3 levels: 1=no problems, 2=some problems, and 3=extreme problems.

    Measured: Month 12

  13. Number of Participants According to Response to Each EQ-5D-Y 5 Dimensions at Month 24

    EQ-5D-Y is participant completed questionnaire designed to assess impact on health related quality of life in children and adolescents <16 years old. It is comprised of five dimensions: mobility, self-care, usual activities, pain/discomfort and anxiety/depression. Each dimension has 3 levels: 1=no problems, 2=some problems, and 3=extreme problems.

    Measured: Month 24

  14. Number of Participants According to Response to EQ-5D-Y 5 Dimensions at Month 30

    EQ-5D-Y is participant completed questionnaire designed to assess impact on health related quality of life in children and adolescents <16 years old. It is comprised of five dimensions: mobility, self-care, usual activities, pain/discomfort and anxiety/depression. Each dimension has 3 levels: 1=no problems, 2=some problems, and 3=extreme problems.

    Measured: Month 30

  15. Change From Baseline in EQ-5D-Y VAS Assessment at Months 12, 24, and 30

    EQ-5D-Y is participant completed questionnaire designed to assess impact on health related quality of life in children and adolescents <16 years old. EQ-VAS recorded the participant's self-rated health on a vertical scale ranging from 0 (worst imaginable health state) to 100 (best imaginable health state).

    Measured: Baseline (Day 1) and Months 12, 24, and 30

  16. Change From Baseline in EQ-5D-Y Index Score at Months 12, 24, and 30

    EQ-5D-Y is participant completed questionnaire designed to assess impact on health related quality of life in children and adolescents <=16 years old. It is comprised of five dimensions: mobility, self-care, usual activities, pain/discomfort and anxiety/depression. Each dimension has 3 levels: 1=no problems, 2=some problems, and 3=extreme problems. Participants indicated their health state by choosing the appropriate level from each dimension. The 5 digit health states thus obtained for each dimension were then converted into a single median index value. A health state index score was calculated from individual health profiles using a USA scoring algorithm. Health state index scores generally ranged from -0.109 to 1, where, -0.109= the worst health status, 1= full health. Higher the score the better the better quality of life.

    Measured: Baseline (Day 1) and Months 12, 24, and 30

  17. Change From Baseline in Healthcare Resource Utilization (HRU) Survey Responses: Visit to Primary Care Physician, Emergency Room and Office Visits at Months 12, 24, and 30

    HRU questionnaire is completed by the caregiver and had questions about healthcare resources utilization related to their child's use of healthcare professionals, emergency room visits, and hospitalizations in past 3 months. Change from baseline in mean number of visits to primary care physician, emergency room and office visits is presented in this outcome measure.

    Measured: Baseline (Day 1) and Months 12, 24, and 30

  18. Change From Baseline in HRU Survey Responses: Number of Nights in Hospital Due to Disease/Medication for Disease at Months 12, 24, and 30

    HRU questionnaire is completed by the caregiver and had questions about healthcare resources utilization related to number of nights in hospital due to disease/dedication for disease in past 3 months. Change from baseline in mean number of nights in hospital due to disease or medication for disease is presented in this outcome measure.

    Measured: Baseline (Day 1) and Months 12, 24, and 30

  19. Change From Baseline in Out-of-Pocket Money of HRU Survey at Months 12, 24 and 30

    Caregivers were asked to estimate out-of-pocket costs related to healthcare resource utilization. The number of out of pocket money was defined as the total spent of the past three months in managing child's Duchenne muscular dystrophy.

    Measured: Baseline (Day 1) and Months 12, 24, and 30

  20. Change From Baseline in Percent Activity Impairment as Per Work Productivity and Activity Impairment Questionnaire Adapted for Caregiving (WPAI:CG) Caregiver Unchanged at Months 12, 24, and 30

    WPAI:CG is a self-reported measure of work productivity and impairment, was completed by caregiver, which had four scores: absenteeism (work time missed); presenteeism (impairment at work/reduced on the job effectiveness); work productivity loss (overall work impairment/absenteeism plus presenteeism); and activity impairment. Each score was expressed as a percentage (0-100%) with higher score indicating greater impairment and less productivity.

    Measured: Baseline (Day 1) and Months 12, 24, and 30

  21. Change From Baseline in Percent Work Time Missed as Per WPAI: CG Caregiver Unchanged at Month 12

    WPAI:CG is a self-reported measure of work productivity and impairment, was completed by caregiver, which had four scores: absenteeism (work time missed); presenteeism (impairment at work/reduced on the job effectiveness); work productivity loss (overall work impairment/absenteeism plus presenteeism); and activity impairment. Each score was expressed as a percentage (0-100%) with higher score indicating greater impairment and less productivity.

    Measured: Baseline (Day 1) and Month 12

  22. Change From Baseline in Percent Work Time Missed as Per WPAI: CG Caregiver Unchanged at Month 24

    WPAI:CG is a self-reported measure of work productivity and impairment, was completed by caregiver, which had four scores: absenteeism (work time missed); presenteeism (impairment at work/reduced on the job effectiveness); work productivity loss (overall work impairment/absenteeism plus presenteeism); and activity impairment. Each score was expressed as a percentage (0-100%) with higher score indicating greater impairment and less productivity.

    Measured: Baseline (Day 1) and Month 24

  23. Change From Baseline in Percent Work Time Missed as Per WPAI: CG Caregiver Unchanged at Month 30

    WPAI:CG is a self-reported measure of work productivity and impairment, was completed by caregiver, which had four scores: absenteeism (work time missed); presenteeism (impairment at work/reduced on the job effectiveness); work productivity loss (overall work impairment/absenteeism plus presenteeism); and activity impairment. Each score was expressed as a percentage (0-100%) with higher score indicating greater impairment and less productivity.

    Measured: Baseline (Day 1) and Month 30

  24. Change From Baseline in Percent Overall Work Impairment WPAI: CG Caregiver Unchanged at Months 12, 24, and 30

    WPAI:CG is a self-reported measure of work productivity and impairment, was completed by caregiver, which had four scores: absenteeism (work time missed); presenteeism (impairment at work/reduced on the job effectiveness); work productivity loss (overall work impairment/absenteeism plus presenteeism); and activity impairment. Each score was expressed as a percentage (0-100%) with higher score indicating greater impairment and less productivity.

    Measured: Baseline (Day 1) and Months 12, 24, and 30

  25. Percent Activity Impairment Score as Per WPAI: CG at Months 12, 24, and 30

    WPAI:CG is a self-reported measure of work productivity and impairment, was completed by caregiver, which had four scores: absenteeism (work time missed); presenteeism (impairment at work/reduced on the job effectiveness); work productivity loss (overall work impairment/absenteeism plus presenteeism); and activity impairment. Each score was expressed as a percentage (0-100%) with higher score indicating greater impairment and less productivity.

    Measured: Months 12, 24, and 30

  26. Percent Work Time Missed Score as Per WPAI: CG at Month 12

    WPAI:CG is a self-reported measure of work productivity and impairment, was completed by caregiver, which had four scores: absenteeism (work time missed); presenteeism (impairment at work/reduced on the job effectiveness); work productivity loss (overall work impairment/absenteeism plus presenteeism); and activity impairment. Each score was expressed as a percentage (0-100%) with higher score indicating greater impairment and less productivity.

    Measured: Month 12

  27. Percent Work Time Missed Score as Per WPAI: CG at Month 24

    WPAI:CG is a self-reported measure of work productivity and impairment, was completed by caregiver, which had four scores: absenteeism (work time missed); presenteeism (impairment at work/reduced on the job effectiveness); work productivity loss (overall work impairment/absenteeism plus presenteeism); and activity impairment. Each score was expressed as a percentage (0-100%) with higher score indicating greater impairment and less productivity.

    Measured: Month 24

  28. Percent Work Time Missed Score as Per WPAI: CG at Month 30

    WPAI:CG is a self-reported measure of work productivity and impairment, was completed by caregiver, which had four scores: absenteeism (work time missed); presenteeism (impairment at work/reduced on the job effectiveness); work productivity loss (overall work impairment/absenteeism plus presenteeism); and activity impairment. Each score was expressed as a percentage (0-100%) with higher score indicating greater impairment and less productivity.

    Measured: Month 30

  29. Percent Overall Work Impairment Scores as Per WPAI: CG at Months 12, 24, and 30

    WPAI:CG is a self-reported measure of work productivity and impairment, was completed by caregiver, which had four scores: absenteeism (work time missed); presenteeism (impairment at work/reduced on the job effectiveness); work productivity loss (overall work impairment/absenteeism plus presenteeism); and activity impairment. Each score was expressed as a percentage (0-100%) with higher score indicating greater impairment and less productivity.

    Measured: Months 12, 24, and 30

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted30 Nov 2018
  2. Study start24 Jul 2019
  3. Primary completion21 Mar 2023
  4. Study completion21 Mar 2023

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase N/A
Primary purpose
Other
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
312 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03760029
Sponsor's protocol ID
C3391004
Status last verified
Sep 2024
First submitted
15 Nov 2018
Last updated
19 Sep 2024
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No: Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical\_trials/trial\_data\_and\_results/data\_requests.

Publications

  • Li X, Yang X, Chang X, Wang Z, Hong S, Zhu W, Shen Q, Zhou Y, Zhu S, Gao YX, Jia C. A natural history study of Chinese individuals with Duchenne muscular dystrophy: Results from 2 years of follow-up and beyond. PLoS One. 2026 Apr 2;21(4):e0345023. doi: 10.1371/journal.pone.0345023. eCollection 2026.

    PubMed 41926354

  • Li X, Lv J, Zhu W, Hong S, Wang Z, Chang X, Gao YX, Zhou Y, Jia C, Fang J, Patterson TA. A 1-year analysis from a natural history study in Chinese individuals with Duchenne muscular dystrophy. Lancet Reg Health West Pac. 2023 Nov 28;42:100944. doi: 10.1016/j.lanwpc.2023.100944. eCollection 2024 Jan.

    PubMed 38089167

Questions about this trial

What is this clinical trial studying?

A study testing Visit frequency in people with Duchenne Muscular Dystrophy, run by Pfizer at 8 sites in China.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 2 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 19 Sep 2024. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 8 sites in China. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

Are results available for this study?

Yes. Results are posted in the registry (first posted 19 Sep 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Age of Participants When They Failed to Walk (time frame: Up to Month 30). The study lists 84 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03760029, which also lists 2 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Sep 2024.

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