Observational cohort study on Facioscapulohumeral Muscular Dystrophy Type 1
Official title: A Registered Observational Cohort Study of Facioscapulohumeral Muscular Dystrophy Type 1
Run by First Affiliated Hospital of Fujian Medical University · NCT04369209 · Data updated 2026-11-12
This study is collecting information from people with Facioscapulohumeral Muscular Dystrophy Type 1 in China to better understand how the condition affects them and help doctors make better treatment decisions.
Quick facts
| Study type | Observational |
|---|---|
| Sex | All |
| Enrollment | 1000 (estimated) |
| Sponsor | First Affiliated Hospital of Fujian Medical University Academic / other |
| Locations | 1 site (Fuzhou, Fujian) |
| Status | Recruiting |
| Started | January 2001 |
| Main results expected | December 2031 |
| Study ends | December 2031 |
| Registry record updated | August 26, 2024 |
| Trial ID | NCT04369209 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Ideal candidates include male and female individuals of all ages with genetically-confirmed FSHD1.
✕Probably not a fit
Patients with other neuromuscular diseases or serious systemic illnesses may not benefit from this study.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
This observational cohort study aims to collect data from genetically-confirmed FSHD1 patients across China, including those with and without symptoms. The study will gather retrospective and prospective data to enhance understanding of the prevalence, progression, and natural history of FSHD1. By analyzing this data, the study seeks to inform healthcare providers in making critical medical and financial decisions regarding the management of FSHD1.
Why it matters
Potential benefit: If successful, this study could provide valuable insights that improve the management and treatment of patients with FSHD1.
How similar studies have gone: While similar observational studies have been conducted, this specific cohort study is novel in its nationwide approach to FSHD1 in China.
Who can join: the full rules
Show full inclusion / exclusion criteria
Inclusion Criteria: * Male or female subjects of all ages at baseline * Subjects, with or without symptoms, with FSHD1 genetic confirmation through PFGE-based Southern blotting * Unrelated healthy controls Exclusion Criteria: * Decline to participate * Other neuromuscular disease (such as Limb-girdle muscular dystrophy or Myotonic dystrophy) * Serious systemic illness (such as heart, liver, kidney disease or major mental illness)
Where it's running
-
First Affiliated Hospital of Fujian Medical University
Fuzhou, Fujian, China Recruiting
Study contacts
- Study coordinator: Ning Wang
- Email: ningwang@fjmu.edu.cn
- Phone: 13805015340
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How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.