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Prospective, Longitudinal Study of the Natural History and Functional Status of Patients With Myotubular Myopathy (MTM)

NCT02057705 · Status: COMPLETED · Type: OBSERVATIONAL · Enrollment: 48

Last updated 2018-06-07

No results posted yet for this study

Summary

This is a prospective, non-interventional, longitudinal study of the natural history and function of approximately 60 patients with MTM from the United States, Canada and Europe. The duration of the study, including the enrollment period, will be 36 months. Data from the study will be used to characterize the disease course of MTM and determine which outcome measures will be the best to assess the efficacy of potential therapies.

Conditions

  • Myotubular Myopathy

Sponsors & Collaborators

  • Institut de Myologie, France

    collaborator OTHER

  • Genethon

    collaborator OTHER

  • Valerion Therapeutics, LLC

    lead INDUSTRY

Principal Investigators

  • Hal Landy, MD · Valerion Therapeutics, LLC

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2014-02-28
Primary Completion
2017-06-26
Completion
2017-06-26

Countries

  • United States
  • Belgium
  • Canada
  • France
  • Germany
  • Italy
  • Spain

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 NCT02057705 on ClinicalTrials.gov