Duchenne Muscular Dystrophy Clinical Trial
Official title:
A Registered Cohort Study on Duchenne Muscular Dystrophy
Dystrophinopathy is a term of X-linked recessive genetic disease, including Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, and the X-linked dilated cardiomyopathy. The aim of this study is to determine the clinical spectrum and natural progression of dystrophinopathy in a prospective multicenter natural history study, to assess the clinical, genetic of patients with dystrophinopathy to optimize clinical management.
Status | Recruiting |
Enrollment | 2000 |
Est. completion date | December 31, 2049 |
Est. primary completion date | December 31, 2039 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 2 Years and older |
Eligibility | Inclusion Criteria: - Beyond 2 years old - Diagnosis with Duchenne Muscular Dystrophy, and female carriers, genotypically confirmed - Diagnosis should be supported by muscle biopsy, if no genetic confirmation. Exclusion Criteria: - Presence of other clinically significant illness |
Country | Name | City | State |
---|---|---|---|
China | First Affiliated Hospital of Fujian Medical University | Fuzhou |
Lead Sponsor | Collaborator |
---|---|
Ning Wang, MD., PhD. |
China,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Age at death | the time when patient die | 20 years |
Status | Clinical Trial | Phase | |
---|---|---|---|
Completed |
NCT05575648 -
Dual Task in Duchenne Muscular Dystrophy
|
N/A | |
Terminated |
NCT03907072 -
Efficacy and Safety Study of WVE-210201 (Suvodirsen) With Open-label Extension in Ambulatory Patients With Duchenne Muscular Dystrophy
|
Phase 2/Phase 3 | |
Not yet recruiting |
NCT06450639 -
An Open-label Study to Assess the Efficacy and Safety of Satralizumab in Duchenne Muscular Dystrophy
|
Phase 2 | |
Completed |
NCT04335942 -
Characterization of the Postural Habits of Wheelchair Users Analysis of the Acceptability of International Recommendations in the Prevention of Pressure Sores Risk by Using a Connected Textile Sensor
|
N/A | |
Active, not recruiting |
NCT04906460 -
Open-label Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy (FORWARD-53)
|
Phase 1/Phase 2 | |
Active, not recruiting |
NCT02500381 -
Study of SRP-4045 (Casimersen) and SRP-4053 (Golodirsen) in Participants With Duchenne Muscular Dystrophy (DMD)
|
Phase 3 | |
Enrolling by invitation |
NCT05967351 -
A Long-term Follow-up Study of Participants Who Received Delandistrogene Moxeparvovec (SRP-9001) in a Previous Clinical Study
|
Phase 3 | |
Recruiting |
NCT03067831 -
Bone Marrow-Derived Autologous Stem Cells for the Treatment of Duchenne Muscular Dystrophy
|
Phase 1/Phase 2 | |
Recruiting |
NCT01834040 -
Study Safety and Efficacy of BMMNC for the Patient With Duchenne Muscular Dystrophy
|
Phase 1/Phase 2 | |
Completed |
NCT02246478 -
A Study of TAS-205 for Duchenne Muscular Dystrophy
|
Phase 1 | |
Active, not recruiting |
NCT01772043 -
Duchenne Muscular Dystrophy Tissue Bank for Exon Skipping
|
N/A | |
Terminated |
NCT01168908 -
Revatio for Heart Disease in Duchenne Muscular Dystrophy and Becker Muscular Dystrophy
|
Phase 2 | |
Completed |
NCT00758225 -
Long-term Safety, Tolerability and Efficacy of Idebenone in Duchenne Muscular Dystrophy (DELPHI Extension)
|
Phase 2 | |
Completed |
NCT03680365 -
Your Voice; Impact of Duchenne Muscular Dystrophy (DMD) on the Lives of Families
|
||
Recruiting |
NCT03513367 -
The Validation Process for Confirmation of the French Version of the Pediatric Quality of Life Inventory :PedsQLTM.
|
||
Recruiting |
NCT05712447 -
Duchenne Muscular Dystrophy Video Assessment Registry
|
||
Recruiting |
NCT01484678 -
Magnetic Resonance Imaging and Biomarkers for Muscular Dystrophy
|
||
Completed |
NCT03319030 -
Aerobic Exercise in Boys With Duchenne Muscular Dystrophy (DMD)
|
||
Terminated |
NCT01753804 -
A Prospective Natural History Study of Progression of Subjects With Duchenne Muscular Dystrophy.
|
N/A | |
Completed |
NCT02530905 -
Dose-Titration and Open-label Extension Study of SRP-4045 in Advanced Stage Duchenne Muscular Dystrophy (DMD) Patients
|
Phase 1 |