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NCT02057705 · ClinicalTrials.gov registry record
Prospective, Longitudinal Study of the Natural History and Functional Status of Patients With Myotubular Myopathy (MTM)
A clinical trial of Myotubular Myopathy, sponsored by Valerion Therapeutics.
- Completed
- Registry status
- 48
- Enrollment target
- 13
- Study locations
NCT02057705: Completed study of Myotubular Myopathy, sponsored by Valerion Therapeutics.
NCT02057705 is a study of Myotubular Myopathy that has completed, run by Valerion Therapeutics. The registered enrollment target is 48 participants. The trial reports 13 study locations across 3 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT02057705, a study of Myotubular Myopathy, has completed, sponsored by Valerion Therapeutics.
- COMPLETED
- Registry status
- 48 participants
- Enrollment target
- 13
- Study locations
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.
Primary Outcome
Study-specific functional assessments and patient questionnaires will be used and will be based on the age and ambulatory status of the participant
Conditions Studied
Study Locations (13)
Other
- Centre Hospitalier Regional de la Citadelle - Liège
- Hôpital Femme Mère Enfant, CHU Lyon Escale - Bron
- Roger Salengro Hospital, CHU, Lille - Lille
- Croix Rousse Hospital - Lyon
- Hôpital Armand Trousseau - Paris
- Institut I-Motion, Hôpital A. Trousseau - Paris
- Institut de Myologie, GH Pitié Salpêtrière, Bâtiment Babinski - Paris
- Hôpital Sainte Musse - Toulon
- University Hospital of Essen - Essen
- Bambino Gesù Children's Hospital - Rome
- Hôpital Puertas de Mar - Cadiz
Massachusetts
- Boston Children's Hospital, 300 Longwood Avenue - Boston
Ontario
- Hospital for Sick Children, 555 University Avenue - Toronto
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 48 participants |
| Start Date | 2014-02 |
| Est. Completion | 2017-06-26 |
What the finished NCT02057705 record still lists
NCT02057705 is an observational study that tracks outcomes without assigning an intervention. The registry caps enrollment at 48 participants, a relatively small participant target.
The record links to 1 condition, with Myotubular Myopathy appearing as the primary indexed condition, and to 0 interventions.
NCT02057705 lists 13 locations in 3 states (Other, Massachusetts, Ontario).
Frequently Asked Questions
What is clinical trial NCT02057705 about?
NCT02057705 is a clinical study titled "Prospective, Longitudinal Study of the Natural History and Functional Status of Patients With Myotubular Myopathy (MTM)". 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 charac...
What is the current status of trial NCT02057705?
This trial is currently completed. The enrollment target is 48 participants. The study started on 2014-02. Estimated completion is 2017-06-26.
What conditions does trial NCT02057705 study?
This clinical trial studies the following conditions: Myotubular Myopathy.
Who is sponsoring clinical trial NCT02057705?
This trial is sponsored by Valerion Therapeutics, which has 2 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT02057705 being conducted?
This trial has 13 study locations across Massachusetts, Ontario. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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