Duchenne Muscular Dystrophy (DMD)NCT04254172
A Low Interventional Study to Monitor Activity Using Wearable Sensors in Duchenne Muscular Dystrophy
An observational study of Duchenne Muscular Dystrophy (DMD), run by Pfizer at 1 site in United States.
Trial summary
The purpose of this low interventional study is to collect data on everyday movement in boys with Duchenne muscular dystrophy (DMD) using wearable activity sensors. The activity sensors could provide useful information beyond what is currently collected by functional (movement, strength) assessments in clinic. Read more about this study
- Who is it for?
- Ages 4 to 12 years, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy (DMD)
- What is being tested?
- Activity Monitor
- Type of treatment
- Medical device
- How many people
- 2 participants
- Where
- 1 siteUnited States
- Study dates
- 19 Feb 2020to 19 Aug 2020
- Phase
- Observational
- Are there results?
- None posted in the registry
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 12 years
- Men only
- No healthy volunteers
- Child
Study population: Ambulatory boys with Duchenne muscular dystrophy who receive care at Nationwide Children's Hospital
Who may be able to join
- Diagnosis of Duchenne muscular dystrophy confirmed by medical history and genetic testing
- Body weight between 15 and 50 kg
- Receipt of glucocorticoids for 6 months and a stable daily dose for at least 3 months prior to study entry
- Ability to rise from floor within seven (7) seconds and ability to walk
Who cannot join
- Current exposure to systemic immunosuppressant agents other than glucocorticoids.
- Prior exposure to any gene therapy agent, including exon-skipping and missense agents.
- Exposure to other investigational drugs within 30 days or 5 half-lives, whichever is longer.
- Any injury which may impact functional testing per investigator's judgement. 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 at screening.
- Any planned surgeries which may impact physical activity and performance.
- Presence or history of musculoskeletal or neurological disease in addition to DMD.
Show all 9Show fewer
- Any known allergies or skin reactions to stainless steel, versaflex, and silicon that may cause possible discomfort by wearable sensors.
- Evidence or history of clinically significant hematological, renal, endocrine, pulmonary, gastrointestinal, cardiovascular, hepatic, cancer, autoimmune or allergic disease that may interfere with the study conduct as per investigator's judgment, excluding untreated, asymptomatic, seasonal allergies at time of screening.
- Investigator site staff members directly involved in the conduct of the study and their family members, site staff members otherwise supervised by the investigator, or participants who are Pfizer employees, including their family members, directly involved in the conduct of the study.
Trial locations and contacts
1 site in 1 country.
United States1 site
Nationwide Children's Hospital
Columbus, Ohio, 43205
About this study
Official title: A SINGLE-SITE, PROSPECTIVE, NATURAL HISTORY LOW INTERVENTIONAL STUDY TO ESTABLISH NORMATIVE DATA OF REAL-WORLD ACTIVITY MEASURES USING WEARABLE SENSORS IN AMBULATORY BOYS WITH DUCHENNE MUSCULAR DYSTROPHY (DMD)
The purpose of this low interventional study is to collect data on everyday movement in boys with Duchenne muscular dystrophy (DMD) using wearable activity sensors. The activity sensors could provide useful information beyond what is currently collected by functional (movement, strength) assessments in clinic. This information can help with the understanding of the impact of DMD, and perhaps with how possible treatments can affect this impact.
- Conditions
- Duchenne Muscular Dystrophy (DMD)
- Being tested
- Activity Monitor
- 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.
Activity MonitorDevice
Wrist and ankle sensors to be worn continuously for 2-week intervals.
| Study group | Role | What they receive |
|---|---|---|
| Single cohort | There is no randomization or stratification in this study. All subjects will complete the same study assessments. Receives: Device: Activity Monitor |
What is being measured
The primary outcome is the main question the study is designed to answer.
Mean change from baseline and variability of activity measures
Measured: baseline, 3, 6, 9, and 12 months
Secondary outcomes (2)
Mean change from baseline in functional assessment scores obtained in the clinic
Measured: baseline, 3, 6, 9, and 12 months
Comparison of mean changes from baseline and correlation coefficient between activity monitoring data and functional data obtained in clinic
Measured: baseline, 3, 6, 9, and 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Feb 2020
- Study start19 Feb 2020
- Primary completion19 Aug 2020
- Study completion19 Aug 2020
Why it stopped: This low-interventional study (involving no study drug) has been terminated early due to the COVID-19 pandemic, and not for any safety or efficacy reasons.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Duchenne Muscular Dystrophy (DMD)Studies of the same condition, open and under way.
- More trials run by PfizerThe sponsor's other registered studies.
- 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
- Observational
- Observational model
- Case only
- Time perspective
- Prospective
- Enrolment
- 2 participants (actual)
Sponsor and investigators
- Lead sponsor
- Pfizer
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Pfizer CT.gov Call Center, Study director, Pfizer
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04254172
- Sponsor's protocol ID
- C3391005
- Status last verified
- Oct 2021
- First submitted
- 31 Jan 2020
- Last updated
- 15 Oct 2021
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- Yes
- 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.
Study at a glance
From the registry record, last updated by the sponsor on 15 Oct 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A SINGLE-SITE, PROSPECTIVE, NATURAL HISTORY LOW INTERVENTIONAL STUDY TO ESTABLISH NORMATIVE DATA OF REAL-WORLD ACTIVITY MEASURES USING WEARABLE SENSORS IN AMBULATORY BOYS WITH DUCHENNE MUSCULAR DYSTROPHY (DMD)
- ClinicalTrials.gov ID
- NCT04254172
- Acronym / sponsor ID
- C3391005
- Conditions
- Duchenne Muscular Dystrophy (DMD)
- Study type
- Observational
- Recruitment status
- TerminatedReason stopped: This low-interventional study (involving no study drug) has been terminated early due to the COVID-19 pandemic, and not for any safety or efficacy reasons.
- Enrolment
- 2 participants (actual)
- Lead sponsor
- PfizerIndustry (pharma, biotech, devices)
- Study start
- 19 Feb 2020 (actual)
- Primary completion
- 19 Aug 2020 (actual)
- Study completion
- 19 Aug 2020 (actual)
- First posted
- 5 Feb 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this low interventional study is to collect data on everyday movement in boys with Duchenne muscular dystrophy (DMD) using wearable activity sensors. The activity sensors could provide useful information beyond what is currently collected by functional (movement, strength) assessments in clinic. This information can help with the understanding of the impact of DMD, and perhaps with how possible treatments can affect this impact.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Single cohort | – | Device: Activity MonitorThere is no randomization or stratification in this study. All subjects will complete the same study assessments. |
Who may qualify
- Ages
- 4 to 12 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
- Study population
- Ambulatory boys with Duchenne muscular dystrophy who receive care at Nationwide Children's Hospital
Key inclusion criteria (4 listed)
- Diagnosis of Duchenne muscular dystrophy confirmed by medical history and genetic testing
- Body weight between 15 and 50 kg
- Receipt of glucocorticoids for 6 months and a stable daily dose for at least 3 months prior to study entry
- Ability to rise from floor within seven (7) seconds and ability to walk
Key exclusion criteria (9 listed)
- Current exposure to systemic immunosuppressant agents other than glucocorticoids.
- Prior exposure to any gene therapy agent, including exon-skipping and missense agents.
- Exposure to other investigational drugs within 30 days or 5 half-lives, whichever is longer.
- Any injury which may impact functional testing per investigator's judgement. 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 at screening.
- Any planned surgeries which may impact physical activity and performance.
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 |
|---|---|---|
| Activity MonitorWrist and ankle sensors to be worn continuously for 2-week intervals. | Device | Single cohort |
Primary outcome
Mean change from baseline and variability of activity measures
Time frame: baseline, 3, 6, 9, and 12 months
2 secondary outcomes
Mean change from baseline in functional assessment scores obtained in the clinic
Time frame: baseline, 3, 6, 9, and 12 months
Comparison of mean changes from baseline and correlation coefficient between activity monitoring data and functional data obtained in clinic
Time frame: baseline, 3, 6, 9, and 12 months
Results and safety
The study was stopped early (reason given: This low-interventional study (involving no study drug) has been terminated early due to the COVID-19 pandemic, and not for any safety or efficacy reasons.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Oct 2021.
- To obtain contact information for a study center near you, click here.Link provided by the sponsor.
- Individual participant dataSharing planned: 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.
Related trials for Duchenne Muscular Dystrophy (DMD)
RecruitingEarly Phase 1
Safety and Dystrophin Expression of SPOT-03 in Duchenne Muscular Dystrophy (DMD) Patients
Duchenne Muscular Dystrophy (DMD)
RecruitingPhase 2
Safety and Efficacy of KER-065 in Participants With Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy
Not yet recruitingPhase 2
A Phase 2 Trial to Investigate the Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Tegacorat in Male Participants 4 to <7 Years of Age With Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy (DMD)
Not yet recruitingObservational study
Multimodal Study About Absence of Dp140 in Becker Muscular Dystrophy: Brain-muscle Imaging, Cognition and Muscle Function
Becker Muscular Dystrophy
Enrolling by invitationObservational study
A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical Practice
Duchenne Muscular Dystrophy
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.
Pfizer CT.gov Call Center study director
Pfizer
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Duchenne Muscular Dystrophy (DMD), run by Pfizer at 1 site in United States.
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 4 inclusion and 9 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: stopped early, as of the sponsor's last update on 15 Oct 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. 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?
The study was stopped early (reason given: This low-interventional study (involving no study drug) has been terminated early due to the COVID-19 pandemic, and not for any safety or efficacy reasons.). Results are not available in the source record.
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?
Mean change from baseline and variability of activity measures (time frame: baseline, 3, 6, 9, and 12 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 15 Oct 2021.