Observational study on Duchenne Muscular Dystrophy

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

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.

Why it matters

Who can join: the full rules

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 it's running

• 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

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• VM100: a nutritional supplement to see if it improves quality of life for boys with Duchenne

  This Phase 2 study tests VM100, a nutritional drink, in boys aged 8 and up with Duchenne muscular dystrophy to see if it can improve quality of life and reduce fatigue.

  Recruiting Phase 2 University of Florida · Gainesville, Florida · NCT07766980

• KER-065 for Duchenne Muscular Dystrophy: safety and potential benefit in boys and men

  This study tests whether KER-065 is safe and can help with symptoms in males with Duchenne muscular dystrophy, including both ambulatory and nonambulatory participants who are on standard care.

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• GNR-097 gene therapy for Duchenne muscular dystrophy in boys

  This study tests a gene therapy (GNR-097) to see if it is safe, tolerable, and potentially beneficial for ambulatory boys with Duchenne muscular dystrophy.

  Recruiting at 6 sites Phase 1/2 Ao Generium · Minsk and 5 other locations · NCT07673809

• ELEVIDYS safety in Duchenne patients treated after market approval

  This study tests the safety of ELEVIDYS, with sirolimus as a protective treatment, in male children and young adults with Duchenne Muscular Dystrophy who have previously received ELEVIDYS or are eligible to receive it commercially.

  Recruiting Phase 4 Sarepta Therapeutics, Inc. · Houston, Texas · NCT07542314

• SGT-003 gene therapy for ambulant boys and young men with Duchenne muscular dystrophy (IMPACT DUCHENNE)

  This trial will test whether a single intravenous infusion of SGT-003 can help ambulatory boys and young men with Duchenne muscular dystrophy maintain or improve walking and motor function.

  Recruiting at 4 sites Phase 3 Solid Biosciences Inc. · Little Rock, Arkansas and 6 other locations · NCT07160634

• Evaluating Givinostat for Young Children with Duchenne Muscular Dystrophy

  This study is testing a new drug called Givinostat to see if it's safe and how it works in young children aged 2 to 6 with Duchenne Muscular Dystrophy.

  Recruiting at 7 sites Phase 2 Italfarmaco · Brussels and 8 other locations · NCT06769633

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.