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Facioscapulohumeral Dystrophy in Children

NCT02625662 · Status: COMPLETED · Type: OBSERVATIONAL · Enrollment: 32

Last updated 2019-09-26

No results posted yet for this study

Summary

This study will focus on the symptoms, natural history and clinical impact of facioscapulohumeral muscular dystrophy (FSHD) in children.

Symptoms of classical FSHD start in adulthood. However, a small subgroup of FSHD patients have an early, childhood onset. This early onset is associated with faster progression and other symptoms like hearing loss and epilepsy.

The symptoms, natural history and clinical impact of FSHD in children are largely unknown.

The results of this study will be vital for adequate symptomatic management and trial-readiness.

Conditions

Sponsors & Collaborators

  • Leiden University Medical Center

    collaborator OTHER

  • Princess Beatrix Muscle Foundation

    collaborator OTHER

  • University Medical Center Nijmegen

    lead OTHER

Principal Investigators

  • Baziel van Engelen, MD, PhD · Nijmegen University Medical Center

Eligibility

Max Age
17 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2015-11-30
Primary Completion
2017-08-31
Completion
2019-09-10

Countries

  • Netherlands

Study Locations

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Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02625662 on ClinicalTrials.gov