Duchenne Muscular DystrophyPhase 3FOR-DMDNCT01603407
Finding the Optimum Regimen for Duchenne Muscular Dystrophy
A Phase 3 study testing Prednisone and Deflazacort in people with Duchenne Muscular Dystrophy, run by University of Rochester at 32 sites in 5 countries.
Trial summary
The Finding the Optimum Regimen for Duchenne Muscular Dystrophy (FOR DMD) study will compare three ways of giving corticosteroids to boys with Duchenne muscular dystrophy (DMD) to determine which of the three ways increases muscle strength the most, and which causes the fewest side effects. Read more about this study
- Who is it for?
- Ages 4 to 7 years, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy
- What is being tested?
- Prednisone and Deflazacort
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 196 participants
- Where
- 32 sitesin 5 countries
- Study dates
- Jan 2013to Nov 2019
- Phase
- Phase 3
- 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 4 to 7 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- Evidence of signed and dated informed consent form.
- Confirmed diagnosis of Duchenne muscular dystrophy
- Age greater than or equal to 4 years and less than 8 years old
- Ability to rise independently from floor, from supine to standing
- Willingness and ability to comply with scheduled visits, drug administration plan and study procedures
- Ability to maintain reproducible FVC measurements.
Who cannot join
- History of major renal or hepatic impairment, immunosuppression or other contraindications to corticosteroid therapy.
- History of chronic systemic fungal or viral infections. Acute bacterial infection(including TB) would exclude from enrolment until the infection had been appropriately treated and resolved.
- Diabetes mellitus.
- Idiopathic hypercalcuria.
- Lack of chicken pox immunity and refusal to undergo immunization.
- Evidence of symptomatic cardiomyopathy at screening assessment (one to three months prior to the baseline visit). Asymptomatic cardiac abnormality on investigation would not be an exclusion.
Show all 13Show fewer
- Current or previous treatment (greater than four consecutive weeks of oral therapy) with corticosteroids or other immunosuppressive treatments for DMD or other recurrent indications (e.g., asthma), unless approved by FOR-DMD Team (i.e., concurrent participation in another allowed DMD trial).
- Inability to take tablets, as assessed by the site investigator by the end of the screening period (the screening period ranges from one to three months prior to the baseline visit).
- Allergy/sensitivity to study drugs or their formulations including lactose and/or sucrose intolerance.
- Severe behavioral problems, including severe autism.
- Previous or ongoing medical condition, medical history, physical findings or laboratory abnormalities that could affect safety, make it unlikely that treatment and follow up will be correctly completed or impair the assessment of study results, in the judgment of the site investigator.
- Weight of less than 13 kilograms.
- Exposure to any investigational drug currently or within 3 months prior to start of study treatment.
Trial locations and contacts
32 sites in 5 countries.
32 sites in 5 countries
United States14 sites
University of New Mexico Health Science Center
Albuquerque, New Mexico, 87131
Boston Children's Hospital
Boston, Massachusetts, 02115
University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, 27599
Ann and Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60614
Nationwide Children's Hospital
Columbus, Ohio, 43205
Penn State Hershey Medical Center
Hershey, Pennsylvania, 17033
University of Kansas Medical Center
Kansas City, Kansas, 66160
University of California Los Angeles (UCLA) Medical Center
Los Angeles, California, 90095
Vanderbilt Children's Hospital
Nashville, Tennessee, 37232
Nemours Children's Hospital
Orlando, Florida, 32827
University of Rochester Medical Center
Rochester, New York, 14642
University of California Davis Medical Center
Sacramento, California, 95817
University of Utah Medical Center
Salt Lake City, Utah, 84132
Children's National Medical Center
Washington D.C., District of Columbia, 20010
United Kingdom7 sites
Heart of England NHS Foundation Trust Birmingham Heartland's Hospital
Birmingham, B9 5SS
Greater Glasgow and Clyde NHS Yorkhill Hospital
Glasgow, Scotland, G3 8SJ
The General Infirmary at Leeds
Leeds, England, LS2 9NS
Alder Hey Children's Hospital NHS Trust
Liverpool, L12 2AP
Great Ormond Street Hospital for Children NHS Trust
London, WC1N 3JH
Royal Manchester Children's Hospital
Manchester, M13 9WL
Institute of Human Genetics, International Centre for Life
Newcastle upon Tyne, NE1 3BZ
Germany4 sites
Children's Hospital, Technical University Dresden
Dresden, 01307
University Hospital of Essen
Essen, 45122
University Medical Center Freiburg
Freiburg im Breisgau, 79110
Children's University Hospital, Göttingen
Göttingen, 37075
Italy4 sites
University of Messina AOU Policlinico Gaetano Martino
Messina, 98125
C. Besta Neurological Institute Foundation
Milan, 20133
University of Padova School of Medicine
Padova, 35128
Neuromuscular Center University of Turin
Torino, 10126
Canada3 sites
Alberta Children's Hospital
Calgary, Alberta, T3B 6A8
Children's Hospital London Health Sciences Centre
London, Ontario, N6A 4G5
Children's Hospital of Eastern Ontario (CHEO)
Ottawa, Ontario, K1H 8L1
About this study
Official title: Duchenne Muscular Dystrophy: Double-blind Randomized Trial to Find Optimum Steroid Regimen
The Finding the Optimum Regimen for Duchenne Muscular Dystrophy (FOR DMD) study will compare three ways of giving corticosteroids to boys with Duchenne muscular dystrophy (DMD) to determine which of the three ways increases muscle strength the most, and which causes the fewest side effects. Using the results of this study, the investigators aim to provide patients and families with clearer information about the best way to take these drugs.
Read the detailed description
Boys with Duchenne muscular dystrophy experience progressive muscle weakness as they grow up. Corticosteroids are currently the only medicine that has been shown to increase muscle strength in boys with DMD. Benefits include an increase in the length of time that boys could continue to walk, reduction in the development of curvature of the spine, a longer time of adequate breathing, and possible protection against the development of heart problems.
Doctors have tried different ways of prescribing corticosteroids in order to decrease undesirable side effects of the drug. No controlled, long-term study has ever looked at the effects of different corticosteroids to see which one improves strength the most and which one causes the fewest side effects, over a period of time. Different doctors in different countries prescribe the drugs in different ways, and some do not prescribe corticosteroids at all.
The FOR DMD study will enroll boys with DMD ages 4-7. The study will look at three ways of taking the following corticosteroids by the mouth to determine which increases muscle strength the most, and which causes the fewest side effects:
- Prednisone 0.75mg/kg/day
- Prednisone 0.75mg/kg/day switching between 10 days on and 10 days off treatment
- Deflazacort 0.9mg/kg/day.
The study will take place at 40 academic medical centers in the United States, Canada, United Kingdom, Germany and Italy.
- Conditions
- Duchenne Muscular Dystrophy
- Being tested
- Prednisone, Deflazacort
- Keywords
- prednisone, deflazacort, muscular dystrophy, neuromuscular disease, multi-center
- Medical subject headings
- Muscular Dystrophy, Duchenne, Muscular Dystrophies, Neuromuscular Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PrednisoneDrug
daily prednisone (0.75 mg/kg/day) tablets for 36-60 months
PrednisoneDrug
intermittent prednisone (0.75 mg/kg/day, 10 days on, 10 days off) tablets for 36 to 60 months
DeflazacortDrug
daily deflazacort (0.9 mg/kg/day) tablets for 36-60 months
| Study group | Role | What they receive |
|---|---|---|
| Daily prednisone | Experimental | daily prednisone (0.75 mg/kg/day) Receives: Drug: Prednisone |
| Intermittent prednisone | Experimental | intermittent prednisone (0.75 mg/kg/day, 10 days on, 10 days off) Receives: Drug: Prednisone |
| Daily deflazacort | Experimental | daily deflazacort (0.9 mg/kg/day Receives: Drug: Deflazacort |
What is being measured
The primary outcome is the main question the study is designed to answer.
Forced Vital Capacity
Forced vital capacity was measured during a spirometry test. Forced expiratory volume (FEV) measures how much air a person can exhale during a forced breath. Forced vital capacity (FVC) is the total amount of air exhaled during the FEV test.
Measured: Average of Months 3, 6, 12, 18, 24, 30 and 36 visits
Rise From the Floor Velocity
Reciprocal of time to rise from the floor
Measured: Average of Months 3, 6, 12, 18, 24, 30 and 36 visits
Treatment Satisfaction Questionnaire for Medication (TSQM) Global Satisfaction With Treatment Score
The TSQM Global Satisfaction with Treatment is a 14-item questionnaire that ranges from 0 - 100 with higher scores indicating better outcomes.
Measured: Average of Months 3, 6, 12, 18, 24, 30 and 36 visits
Secondary outcomes (14)
North Star Ambulatory Assessment (NSAA) Score
The North Star Ambulatory Assessment (NSAA) is a 17-item rating scale that is used to measure functional motor abilities in ambulant children with Duchenne Muscular Dystrophy (DMD). It is usually used to monitor the progression of the disease and treatment effects.
The activities are graded as follows:
2 - "Normal" - no obvious modification of activity
1 - Modified method but achieves goal independent of physical assistance from another 0 - Unable to achieve independently This scale is ordinal with 34 as the maximum score indicating fully-independent function.
Measured: Average of Months 3, 6, 12, 18, 24, 30 and 36 visits
6 Minute Walk Test
Measures the total distance walked in 6 minutes averaged over all post-baseline follow-up visits through Month 36.
Measured: Average of Months 3, 6, 12, 18, 24, 30 and 36 visits
Range of Motion (Goniometry) of Left Ankle
Range of motion at the ankle joint in dorsiflexion measured in degrees from plantigrade averaged over all post-baseline visits.
Measured: Average of Months 3, 6, 12, 18, 24, 30 and 36 visits
Range of Motion (Goniometry) of Right Ankle
Range of motion at the ankle joint in dorsiflexion measured in degrees from plantigrade averaged over all post-baseline visits.
Measured: Average of Months 3, 6, 12, 18, 24, 30 and 36 visits
Number of Participants Who Tolerated the Regimen
The number of participants who completed 36 months of follow-up on the originally assigned dosage (for weight) of study medication.
Measured: 3 years
Heart Rate
Measured by trans-thoracic echocardiogram and 12-lead ECG.
Measured: 36 months
Quality of Life - Parent
Quality of life was measured by parent/guardian self-report for all children utilizing the PEDSQL measurement tool. This is a 23-question tool. Scores can range from 0 to 100, with higher scores indicating better quality of life for the child.
Measured: Average of Months 12, 24, and 36 visits
Quality of Life- Child
Quality of life was measured by child self-report in children age 5 and older utilizing the PEDSQL measurement tool. This is a 23-question tool. Scores can range from 0 to 100, with higher scores indicating better quality of life.
Measured: Average of Months 12, 24, and 36 visits
Left Ventricular Ejection Fraction Percent
Measured by trans-thoracic echocardiogram and 12-lead ECG.
Measured: 36 months
Fractional Shortening Percent
Measured by trans-thoracic echocardiogram and 12-lead ECG.
Measured: 36 months
PR Interval
Measured by trans-thoracic echocardiogram and 12-lead ECG.
Measured: 36 months
Participant Weight
Measured: 36 months
Participant Height
Measured: 36 months
Participant Body Mass Index
Measured: 36 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 May 2012 (estimated)
- Study startJan 2013
- Primary completionNov 2019
- Study completionNov 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (62)Papers the sponsor has linked to this study.
- Other trials for Duchenne Muscular DystrophyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 196 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Rochester
- Sponsor type
- University, hospital or other
- Collaborators
- Newcastle University, University Medical Center Freiburg, National Institute of Neurological Disorders and Stroke (NINDS)
- Responsible party
- Principal investigator, Robert Griggs, MD, University of Rochester
- Investigators
- Robert C. Griggs, MD, Principal investigator, University of RochesterKate Bushby, MD, Principal investigator, Newcastle University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01603407
- Sponsor's protocol ID
- U01NS061799
- Other IDs
- 2010-023744-33, U01NS061799, 46102316
- Acronym
- FOR-DMD
- Status last verified
- Aug 2022
- First submitted
- 3 Apr 2012
- Last updated
- 12 Aug 2022
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- Individual data sharing
- No
Publications
Angelini C, Pegoraro E, Turella E, Intino MT, Pini A, Costa C. Deflazacort in Duchenne dystrophy: study of long-term effect. Muscle Nerve. 1994 Apr;17(4):386-91. doi: 10.1002/mus.880170405.
PubMed 8170484Atkinson MJ, Sinha A, Hass SL, Colman SS, Kumar RN, Brod M, Rowland CR. Validation of a general measure of treatment satisfaction, the Treatment Satisfaction Questionnaire for Medication (TSQM), using a national panel study of chronic disease. Health Qual Life Outcomes. 2004 Feb 26;2:12. doi: 10.1186/1477-7525-2-12.
PubMed 14987333Backman E, Henriksson KG. Low-dose prednisolone treatment in Duchenne and Becker muscular dystrophy. Neuromuscul Disord. 1995 May;5(3):233-41. doi: 10.1016/0960-8966(94)00048-e.
PubMed 7633189Bianchi ML. How to manage osteoporosis in children. Best Pract Res Clin Rheumatol. 2005 Dec;19(6):991-1005. doi: 10.1016/j.berh.2005.06.006.
PubMed 16301193Biggar WD, Bachrach LK, Henderson RC, Kalkwarf H, Plotkin H, Wong BL. Bone health in Duchenne muscular dystrophy: a workshop report from the meeting in Cincinnati, Ohio, July 8, 2004. Neuromuscul Disord. 2005 Jan;15(1):80-5. doi: 10.1016/j.nmd.2004.09.010. No abstract available.
PubMed 15639125Biggar WD, Gingras M, Fehlings DL, Harris VA, Steele CA. Deflazacort treatment of Duchenne muscular dystrophy. J Pediatr. 2001 Jan;138(1):45-50. doi: 10.1067/mpd.2001.109601.
PubMed 11148511Biggar WD, Harris VA, Eliasoph L, Alman B. Long-term benefits of deflazacort treatment for boys with Duchenne muscular dystrophy in their second decade. Neuromuscul Disord. 2006 Apr;16(4):249-55. doi: 10.1016/j.nmd.2006.01.010. Epub 2006 Mar 20.
PubMed 16545568Biggar WD, Politano L, Harris VA, Passamano L, Vajsar J, Alman B, Palladino A, Comi LI, Nigro G. Deflazacort in Duchenne muscular dystrophy: a comparison of two different protocols. Neuromuscul Disord. 2004 Sep;14(8-9):476-82. doi: 10.1016/j.nmd.2004.05.001.
PubMed 15336688Brooke MH, Fenichel GM, Griggs RC, Mendell JR, Moxley R, Miller JP, Province MA. Clinical investigation in Duchenne dystrophy: 2. Determination of the "power" of therapeutic trials based on the natural history. Muscle Nerve. 1983 Feb;6(2):91-103. doi: 10.1002/mus.880060204.
PubMed 6343858Brooke MH, Fenichel GM, Griggs RC, Mendell JR, Moxley RT 3rd, Miller JP, Kaiser KK, Florence JM, Pandya S, Signore L, et al. Clinical investigation of Duchenne muscular dystrophy. Interesting results in a trial of prednisone. Arch Neurol. 1987 Aug;44(8):812-7. doi: 10.1001/archneur.1987.00520200016010.
PubMed 3632393Brooke MH, Fenichel GM, Griggs RC, Mendell JR, Moxley R, Florence J, King WM, Pandya S, Robison J, Schierbecker J, et al. Duchenne muscular dystrophy: patterns of clinical progression and effects of supportive therapy. Neurology. 1989 Apr;39(4):475-81. doi: 10.1212/wnl.39.4.475.
PubMed 2927672Bushby K, Muntoni F, Urtizberea A, Hughes R, Griggs R. Report on the 124th ENMC International Workshop. Treatment of Duchenne muscular dystrophy; defining the gold standards of management in the use of corticosteroids. 2-4 April 2004, Naarden, The Netherlands. Neuromuscul Disord. 2004 Sep;14(8-9):526-34. doi: 10.1016/j.nmd.2004.05.006. No abstract available.
PubMed 15336694Bushby K, Muntoni F, Bourke JP. 107th ENMC international workshop: the management of cardiac involvement in muscular dystrophy and myotonic dystrophy. 7th-9th June 2002, Naarden, the Netherlands. Neuromuscul Disord. 2003 Feb;13(2):166-72. doi: 10.1016/s0960-8966(02)00213-4. No abstract available.
PubMed 12565916Bushby K, Finkel R, Birnkrant DJ, Case LE, Clemens PR, Cripe L, Kaul A, Kinnett K, McDonald C, Pandya S, Poysky J, Shapiro F, Tomezsko J, Constantin C; DMD Care Considerations Working Group. Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management. Lancet Neurol. 2010 Jan;9(1):77-93. doi: 10.1016/S1474-4422(09)70271-6. Epub 2009 Nov 27.
PubMed 19945913Bushby K, Finkel R, Birnkrant DJ, Case LE, Clemens PR, Cripe L, Kaul A, Kinnett K, McDonald C, Pandya S, Poysky J, Shapiro F, Tomezsko J, Constantin C; DMD Care Considerations Working Group. Diagnosis and management of Duchenne muscular dystrophy, part 2: implementation of multidisciplinary care. Lancet Neurol. 2010 Feb;9(2):177-89. doi: 10.1016/S1474-4422(09)70272-8. Epub 2009 Nov 27.
PubMed 19945914
Study at a glance
From the registry record, last updated by the sponsor on 12 Aug 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Duchenne Muscular Dystrophy: Double-blind Randomized Trial to Find Optimum Steroid Regimen
- ClinicalTrials.gov ID
- NCT01603407
- Acronym / sponsor ID
- FOR-DMD / U01NS061799
- Conditions
- Duchenne Muscular Dystrophy
- Keywords
- prednisone, deflazacort, muscular dystrophy, neuromuscular disease, multi-center
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 196 participants (actual)
- Lead sponsor
- University of RochesterUniversity, hospital or other
- Collaborators
- Newcastle University, University Medical Center Freiburg, National Institute of Neurological Disorders and Stroke (NINDS)
- Study start
- Jan 2013
- Primary completion
- Nov 2019 (actual)
- Study completion
- Nov 2019 (actual)
- First posted
- 23 May 2012 (estimated)
- Results
- Posted 12 Aug 2022
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The Finding the Optimum Regimen for Duchenne Muscular Dystrophy (FOR DMD) study will compare three ways of giving corticosteroids to boys with Duchenne muscular dystrophy (DMD) to determine which of the three ways increases muscle strength the most, and which causes the fewest side effects. Using the results of this study, the investigators aim to provide patients and families with clearer information about the best way to take these drugs.
Detailed description
Boys with Duchenne muscular dystrophy experience progressive muscle weakness as they grow up. Corticosteroids are currently the only medicine that has been shown to increase muscle strength in boys with DMD. Benefits include an increase in the length of time that boys could continue to walk, reduction in the development of curvature of the spine, a longer time of adequate breathing, and possible protection against the development of heart problems.
Doctors have tried different ways of prescribing corticosteroids in order to decrease undesirable side effects of the drug. No controlled, long-term study has ever looked at the effects of different corticosteroids to see which one improves strength the most and which one causes the fewest side effects, over a period of time. Different doctors in different countries prescribe the drugs in different ways, and some do not prescribe corticosteroids at all.
The FOR DMD study will enroll boys with DMD ages 4-7. The study will look at three ways of taking the following corticosteroids by the mouth to determine which increases muscle strength the most, and which causes the fewest side effects:
- Prednisone 0.75mg/kg/day
- Prednisone 0.75mg/kg/day switching between 10 days on and 10 days off treatment
- Deflazacort 0.9mg/kg/day.
The study will take place at 40 academic medical centers in the United States, Canada, United Kingdom, Germany and Italy.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Daily prednisone | Experimental | Drug: Prednisonedaily prednisone (0.75 mg/kg/day) |
| Intermittent prednisone | Experimental | Drug: Prednisoneintermittent prednisone (0.75 mg/kg/day, 10 days on, 10 days off) |
| Daily deflazacort | Experimental | Drug: Deflazacortdaily deflazacort (0.9 mg/kg/day |
Who may qualify
- Ages
- 4 to 7 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (6 listed)
- Evidence of signed and dated informed consent form.
- Confirmed diagnosis of Duchenne muscular dystrophy
- Age greater than or equal to 4 years and less than 8 years old
- Ability to rise independently from floor, from supine to standing
- Willingness and ability to comply with scheduled visits, drug administration plan and study procedures
Key exclusion criteria (13 listed)
- History of major renal or hepatic impairment, immunosuppression or other contraindications to corticosteroid therapy.
- History of chronic systemic fungal or viral infections. Acute bacterial infection(including TB) would exclude from enrolment until the infection had been appropriately treated and resolved.
- Diabetes mellitus.
- Idiopathic hypercalcuria.
- Lack of chicken pox immunity and refusal to undergo immunization.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Prednisonedaily prednisone (0.75 mg/kg/day) tablets for 36-60 months | Drug | Daily prednisone |
| Prednisoneintermittent prednisone (0.75 mg/kg/day, 10 days on, 10 days off) tablets for 36 to 60 months | Drug | Intermittent prednisone |
| Deflazacortdaily deflazacort (0.9 mg/kg/day) tablets for 36-60 months | Drug | Daily deflazacort |
Primary outcome
Forced Vital Capacity
Time frame: Average of Months 3, 6, 12, 18, 24, 30 and 36 visits
Forced vital capacity was measured during a spirometry test. Forced expiratory volume (FEV) measures how much air a person can exhale during a forced breath. Forced vital capacity (FVC) is the total amount of air exhaled during the FEV test.
Rise From the Floor Velocity
Time frame: Average of Months 3, 6, 12, 18, 24, 30 and 36 visits
Reciprocal of time to rise from the floor
Treatment Satisfaction Questionnaire for Medication (TSQM) Global Satisfaction With Treatment Score
Time frame: Average of Months 3, 6, 12, 18, 24, 30 and 36 visits
The TSQM Global Satisfaction with Treatment is a 14-item questionnaire that ranges from 0 - 100 with higher scores indicating better outcomes.
14 secondary outcomes
North Star Ambulatory Assessment (NSAA) Score
Time frame: Average of Months 3, 6, 12, 18, 24, 30 and 36 visits
6 Minute Walk Test
Time frame: Average of Months 3, 6, 12, 18, 24, 30 and 36 visits
Range of Motion (Goniometry) of Left Ankle
Time frame: Average of Months 3, 6, 12, 18, 24, 30 and 36 visits
Range of Motion (Goniometry) of Right Ankle
Time frame: Average of Months 3, 6, 12, 18, 24, 30 and 36 visits
Number of Participants Who Tolerated the Regimen
Time frame: 3 years
Heart Rate
Time frame: 36 months
Quality of Life - Parent
Time frame: Average of Months 12, 24, and 36 visits
Quality of Life- Child
Time frame: Average of Months 12, 24, and 36 visits
Left Ventricular Ejection Fraction Percent
Time frame: 36 months
Fractional Shortening Percent
Time frame: 36 months
PR Interval
Time frame: 36 months
Participant Weight
Time frame: 36 months
Participant Height
Time frame: 36 months
Participant Body Mass Index
Time frame: 36 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Aug 2022.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 8170484Angelini C, Pegoraro E, Turella E, Intino MT, Pini A, Costa C. Deflazacort in Duchenne dystrophy: study of long-term effect. Muscle Nerve. 1994 Apr;17(4):386-91. doi: 10.1002/mus.880170405.
- Background: PubMed 14987333Atkinson MJ, Sinha A, Hass SL, Colman SS, Kumar RN, Brod M, Rowland CR. Validation of a general measure of treatment satisfaction, the Treatment Satisfaction Questionnaire for Medication (TSQM), using a national panel study of chronic disease. Health Qual Life Outcomes. 2004 Feb 26;2:12. doi: 10.1186/1477-7525-2-12.
- Background: PubMed 7633189Backman E, Henriksson KG. Low-dose prednisolone treatment in Duchenne and Becker muscular dystrophy. Neuromuscul Disord. 1995 May;5(3):233-41. doi: 10.1016/0960-8966(94)00048-e.
- Background: PubMed 16301193Bianchi ML. How to manage osteoporosis in children. Best Pract Res Clin Rheumatol. 2005 Dec;19(6):991-1005. doi: 10.1016/j.berh.2005.06.006.
- Background: PubMed 15639125Biggar WD, Bachrach LK, Henderson RC, Kalkwarf H, Plotkin H, Wong BL. Bone health in Duchenne muscular dystrophy: a workshop report from the meeting in Cincinnati, Ohio, July 8, 2004. Neuromuscul Disord. 2005 Jan;15(1):80-5. doi: 10.1016/j.nmd.2004.09.010. No abstract available.
- Background: PubMed 11148511Biggar WD, Gingras M, Fehlings DL, Harris VA, Steele CA. Deflazacort treatment of Duchenne muscular dystrophy. J Pediatr. 2001 Jan;138(1):45-50. doi: 10.1067/mpd.2001.109601.
- Background: PubMed 16545568Biggar WD, Harris VA, Eliasoph L, Alman B. Long-term benefits of deflazacort treatment for boys with Duchenne muscular dystrophy in their second decade. Neuromuscul Disord. 2006 Apr;16(4):249-55. doi: 10.1016/j.nmd.2006.01.010. Epub 2006 Mar 20.
- Background: PubMed 15336688Biggar WD, Politano L, Harris VA, Passamano L, Vajsar J, Alman B, Palladino A, Comi LI, Nigro G. Deflazacort in Duchenne muscular dystrophy: a comparison of two different protocols. Neuromuscul Disord. 2004 Sep;14(8-9):476-82. doi: 10.1016/j.nmd.2004.05.001.
- Background: PubMed 6343858Brooke MH, Fenichel GM, Griggs RC, Mendell JR, Moxley R, Miller JP, Province MA. Clinical investigation in Duchenne dystrophy: 2. Determination of the "power" of therapeutic trials based on the natural history. Muscle Nerve. 1983 Feb;6(2):91-103. doi: 10.1002/mus.880060204.
- Background: PubMed 3632393Brooke MH, Fenichel GM, Griggs RC, Mendell JR, Moxley RT 3rd, Miller JP, Kaiser KK, Florence JM, Pandya S, Signore L, et al. Clinical investigation of Duchenne muscular dystrophy. Interesting results in a trial of prednisone. Arch Neurol. 1987 Aug;44(8):812-7. doi: 10.1001/archneur.1987.00520200016010.
- Background: PubMed 2927672Brooke MH, Fenichel GM, Griggs RC, Mendell JR, Moxley R, Florence J, King WM, Pandya S, Robison J, Schierbecker J, et al. Duchenne muscular dystrophy: patterns of clinical progression and effects of supportive therapy. Neurology. 1989 Apr;39(4):475-81. doi: 10.1212/wnl.39.4.475.
- Background: PubMed 15336694Bushby K, Muntoni F, Urtizberea A, Hughes R, Griggs R. Report on the 124th ENMC International Workshop. Treatment of Duchenne muscular dystrophy; defining the gold standards of management in the use of corticosteroids. 2-4 April 2004, Naarden, The Netherlands. Neuromuscul Disord. 2004 Sep;14(8-9):526-34. doi: 10.1016/j.nmd.2004.05.006. No abstract available.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Robert C. Griggs, MD principal investigator
University of Rochester
Kate Bushby, MD principal investigator
Newcastle University
32 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Prednisone and Deflazacort in people with Duchenne Muscular Dystrophy, run by University of Rochester at 32 sites in 5 countries.
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 6 inclusion and 13 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 12 Aug 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 32 sites in United States, United Kingdom, Germany, Italy, Canada. 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.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 12 Aug 2022). 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?
Forced Vital Capacity (time frame: Average of Months 3, 6, 12, 18, 24, 30 and 36 visits). The study lists 3 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 NCT01603407, which also lists 62 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 12 Aug 2022; AzTrials last refreshed its copy on 31 Oct 2026.