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A Clinical Assessment Study in X-Linked Myotubular Myopathy (XLMTM) Subjects

NCT02704273 · Status: COMPLETED · Type: OBSERVATIONAL · Enrollment: 34

Last updated 2022-07-29

No results posted yet for this study

Summary

This is a pre-Phase 1 prospective, non interventional clinical assessment study to evaluate XLMTM subjects aged 3 years and younger. Many of these clinically relevant measures have not yet been routinely assessed in this population and may provide important insight on the natural history of XLMTM and for future evaluation of potential therapies.

Conditions

  • X-linked Myotubular Myopathy

Sponsors & Collaborators

  • Astellas Gene Therapies

    lead INDUSTRY

Principal Investigators

  • Salvador Rico, MD, PhD · Audentes Therapeutics

Eligibility

Max Age
3 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2016-07-31
Primary Completion
2019-09-30
Completion
2019-09-30

Countries

  • United States
  • Canada
  • France
  • Germany
  • United Kingdom

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 NCT02704273 on ClinicalTrials.gov