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

Completed

Conditions studied: Myotubular Myopathy

In brief

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.

Key facts

Study ID

NCT02057705

Run by

Valerion Therapeutics, LLC

People needed

48

Starts

2014-02-01

Expected to finish

2017-06-26

Last updated by the study team

2018-06-07

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Patients of any age (newborns included) may participate.
• Patients over 18 years of age and parent(s)/legal guardian(s) of patients <18 years of age must provide written informed consent prior to participating in the study and informed assent will be obtained from minors at least 7 years of age when required by regulation.
• MTM resulting from a mutation in the MTM1 gene.
• Male or symptomatic female. A symptomatic female will be defined by the motor function assessment by Motor Function Measure (MFM) or North Star Ambulatory Assessment (NSAA) below 80% of the total score.
• Willing and able to comply with all protocol requirements and procedures.

You may not qualify if…

• Other disease which may significantly interfere with the assessment of MTM and is clearly not related to the disease.
• Currently enrolled in a treatment study; or treatment with an experimental therapy other than pyridostigmine.

Where it is running

• Boston Children's Hospital, 300 Longwood Avenue — Boston, Massachusetts, United States
• Centre Hospitalier Regional de la Citadelle — Liège, Belgium
• Hospital for Sick Children, 555 University Avenue — Toronto, Ontario, Canada
• Hôpital Femme Mère Enfant, CHU Lyon Escale — Bron, France
• Roger Salengro Hospital, CHU, Lille — Lille, France
• Croix Rousse Hospital — Lyon, France
• Hôpital Armand Trousseau — Paris, France
• Institut I-Motion, Hôpital A. Trousseau — Paris, France
• Institut de Myologie, GH Pitié Salpêtrière, Bâtiment Babinski — Paris, France
• Hôpital Sainte Musse — Toulon, France
• University Hospital of Essen — Essen, Germany
• Bambino Gesù Children's Hospital — Rome, Italy
• Hôpital Puertas de Mar — Cadiz, Spain

Full record on ClinicalTrials.gov

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