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

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Observational

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

Brief 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.

Detailed Summary:
Sponsor: Valerion Therapeutics, LLC

Current Primary Outcome: Time to characterize the disease course in MTM patients [ Time Frame: Up to 24 Months ]

Study-specific functional assessments and patient questionnaires will be used and will be based on the age and ambulatory status of the participant


Original Primary Outcome: Time to characterize the disease course in MTM patients [ Time Frame: Up to 24 Months ]

Study-specific functional assessments and patient questonnaires will be used and will be based on the age and ambulatory status of the participant


Current Secondary Outcome: Change in disease severity and disease progression [ Time Frame: Baseline, Month 3 (EU only), Month 6, Month 12 and Month 24 ]

Study-specific functional assessments and patient questionnaires will be used and will be based on the age and ambulatory status of the participant


Original Secondary Outcome: Change in disease severity and disease progression [ Time Frame: Baseline, Month 3 (EU only), Month 6, Month 12 and Month 24 ]

Study-specific functional assessments and patient questonnaires will be used and will be based on the age and ambulatory status of the participant


Information By: Valerion Therapeutics, LLC

Dates:
Date Received: February 4, 2014
Date Started: February 2014
Date Completion: June 2017
Last Updated: January 26, 2017
Last Verified: January 2017