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Prospective Natural History Study of Patients With Myotubular Myopathy and Other CentroNuclear Myopathies

NCT03351270 · Status: COMPLETED · Phase: NA · Type: INTERVENTIONAL · Enrollment: 60

Last updated 2023-03-02

No results posted yet for this study

Summary

This is the 2 years extension of the prospective and longitudinal study of the natural history and functional status of patients with myotubular myopathy and other centronuclear (CNM) sponsored by Dynacure including ten additional pediatric patients with mutation in MTM1 or DNM2 genes). the patients are planned to be enrolled in one year leading to an expected total number of 70 patients followed at least over 1 year period. Data from the study will be used to characterize the disease course of CNM and determine which outcome measures will be the best to assess the efficacy of potential therapies.

Conditions

  • Centronuclear Myopathy

Interventions

OTHER

Standardized strength, respiratory and motor function assessments

Standardized assessments will be adjusted by the age, the ambulant status and the respiratory status of the patient. The visit frequency will also be adjusted according to the age and to the time spent in the study varying between quarterly to yearly.

Sponsors & Collaborators

  • Dynacure

    collaborator INDUSTRY

  • Institut de Myologie, France

    lead OTHER

Principal Investigators

  • Laurent Servais, MD · Centre de référence neuromusculaire, CHR La Citadelle; 4000 Liege Belgium

Study Design

Allocation
NA
Purpose
OTHER
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2017-05-28
Primary Completion
2021-09-30
Completion
2021-09-30

Countries

  • Belgium
  • 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 NCT03351270 on ClinicalTrials.gov