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

NACompletedINTERVENTIONAL
Enrollment

60

Participants

Timeline

Start Date

May 28, 2017

Primary Completion Date

September 30, 2021

Study Completion Date

September 30, 2021

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.

Trial Locations (11)

4000

Centre de référence neuromusculaire, CHR La Citadelle, Liège

59000

Hôpital Roger Salengro, CHU Lille, Lille

69004

Hôpital de la Croix Rousse, Lyon

69500

Hôpital Femme Mère Enfant, CHU Lyon L'Escale, Bron

75012

Hôpital Armand Trousseau, Paris

I-Motion Institute - Trousseau Hospital, Paris

75013

Institute of Myology, Paris

83056

Hôpital Sainte Musse, Toulon

D-45147

Universitätsklinikum Essen (AöR), Essen

00146

Bambino Gesu Children's Hospital, Roma

21-11009

Hospital Puerta del Mar, Cadiz

Sponsors

Collaborators (1)

All Listed Sponsors
collaborator

Dynacure

INDUSTRY

lead

Institut de Myologie, France

OTHER