Actively Recruiting
A Registered Cohort Study on Duchenne Muscular Dystrophy
Led by Ning Wang, MD., PhD. · Updated on 2021-03-22
2000
Participants Needed
1
Research Sites
521 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Dystrophinopathy refers to a group of X-linked recessive genetic disorders, including Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, and X-linked dilated cardiomyopathy. This research aims to understand the clinical characteristics and natural progression of dystrophinopathy by studying patients over time in multiple centers. The goal is to assess clinical and genetic aspects to improve patient care and management. This observational study follows a registered cohort of individuals diagnosed with Duchenne Muscular Dystrophy, including female carriers confirmed by genetic testing or muscle biopsy if genetic confirmation is unavailable. The study does not involve experimental treatments but focuses on collecting detailed clinical and genetic information to observe disease progression. Participants will be monitored over an extended period, with researchers tracking outcomes such as age at death by 20 years. The study involves regular clinical evaluations to gather data on the clinical spectrum and progression of the disease. Long-term follow-up helps optimize management strategies and provides valuable insights into dystrophinopathy.
CONDITIONS
Brief Title
A Registered Cohort Study on Duchenne Muscular Dystrophy
Who Can Participate
Eligibility Criteria
You may qualify if you...
- 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.
You will not qualify if you...
- Presence of other clinically significant illness
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
Duration - Up to 29 years
Participants with Duchenne Muscular Dystrophy or female carriers are observed over time to track health outcomes including age at death.
Trial Site Locations
Total: 1 location
1
First Affiliated Hospital of Fujian Medical University
Fuzhou, China
Actively Recruiting
Research Team
N
Ning Wang, MD, PhD
M
Ming Jin, MD
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
0
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