Observational study on Duchenne Muscular Dystrophy
A Registered Cohort Study on Duchenne Muscular Dystrophy
This study looks at how Duchenne Muscular Dystrophy and similar conditions develop over time in patients to help improve their care.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 2000 (estimated) |
| Ages | 2 Years and up |
| Sex | All |
| Sponsor | First Affiliated Hospital of Fujian Medical University Academic / other |
| Locations | 1 site (Fuzhou) |
| Trial ID | NCT04012671 on ClinicalTrials.gov |
What this trial studies
This study aims to investigate the clinical spectrum and natural progression of dystrophinopathy, which includes Duchenne Muscular Dystrophy and related conditions. It is a prospective multicenter natural history study that will assess both clinical and genetic factors in patients diagnosed with dystrophinopathy. The goal is to optimize clinical management based on the findings from this cohort.
Who should consider this trial
Good fit: Ideal candidates include individuals over 2 years old diagnosed with Duchenne Muscular Dystrophy or female carriers with genotypic confirmation.
Not a fit: Patients with other clinically significant illnesses may not receive benefit from this study.
Why it matters
Potential benefit: If successful, this study could enhance understanding of Duchenne Muscular Dystrophy and improve clinical management strategies for affected patients.
How similar studies have performed: Other studies on the natural history of dystrophinopathy have shown promise, indicating that this approach is supported by previous findings.
Eligibility criteria
Show full inclusion / exclusion criteria
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
Where this trial is running
Fuzhou
- First Affiliated Hospital of Fujian Medical University — Fuzhou, China (Recruiting)
Study contacts
- Study coordinator: Ning Wang, MD, PhD
- Email: ningwang@fjmu.edu.cn
- Phone: 13805015340
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.