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The Natural History and Muscle Fatigability of Patients With Congenital Myopathies.

NCT06157268 · Status: RECRUITING · Type: OBSERVATIONAL · Enrollment: 100

Last updated 2024-08-19

No results posted yet for this study

Summary

Core myopathies (CCD/MmD), nemaline myopathies (NEM) and centronuclear myopathies (CNM) are three types of rare congenital myopathies. Not much is known about the natural history and no curative treatment is available for these groups. Also patients report fatigability as one of their symptoms. The goal of this observational study is to study the natural history during 24 months to achieve trial readiness and to study the muscle fatigability in CCD/MmD, NEM and CNM.

Conditions

  • Central Core Disease
  • Multi-Minicore Disease
  • Nemaline Myopathy
  • Centronuclear Myopathy

Interventions

OTHER

Natural history and non therapeutical therapy

This study concerns a natural history study part and a muscle fatigability part. For the natural history study no interventions will be used. For the muscle fatigability part non therapeutical therapies will be used. This includes endurance tests, isokinetic dynamometry and repetitive nerve stimulation.

Sponsors & Collaborators

  • UMC Utrecht

    collaborator OTHER

  • Radboud University Medical Center

    lead OTHER

Principal Investigators

  • Nicol Voermans, MD PhD · Radboud University Medical Center

Eligibility

Min Age
2 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-03-28
Primary Completion
2026-11-30
Completion
2026-11-30

Countries

  • Netherlands

Study Locations

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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 NCT06157268 on ClinicalTrials.gov