A Registered Cohort Study on FSHD1

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Total Enrollment

1000 participants

Study Classification

OBSERVATIONAL

Study Start Date

2001-01-31

Study Completion Date

2031-12-31

Brief Summary

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The data to be collected is intended to help healthcare providers make important medical and financial decisions concerning FSHD1, through an enhanced understanding of the prevalence, progression and natural history of FSHD1.

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Detailed Description

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The China FSHD1 patient registry is a nationwide, population-based, non-interventional, observational cohort clinical study of all age groups of genetically-confirmed FSHD1 patients from families (with at least 1 affected member), collecting data retrospectively at study entry and prospectively during follow up. The data to be collected is intended to help healthcare providers make important medical and financial decisions concerning FSHD1, through an enhanced understanding of the prevalence, progression and natural history of FSHD1.

Conditions

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Facioscapulohumeral Muscular Dystrophy Type 1 (FSHD1)

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Eligibility Criteria

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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)

Eligible Sex

ALL

Accepts Healthy Volunteers

Yes