Clinical Trial: A Clinical Assessment Study in X-Linked Myotubular Myopathy (XLMTM) Subjects
Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Observational
Official Title: INCEPTUS: A Prospective, Non-Interventional Clinical Assessment Study in X Linked Myotubular Myopathy (XLMTM) Subjects Aged 3 Years and Younger
Brief Summary: This is a pre-Phase 1 prospective, non interventional clinical assessment study to evaluate XLMTM subjects aged 3 years and younger. Many of these clinically relevant measures have not yet been routinely assessed in this population and may provide important insight on the natural history of XLMTM and for future evaluation of potential therapies.
Detailed Summary:
Sponsor: Audentes Therapeutics
Current Primary Outcome:
- Characterize the disease course and natural history of XLMTM using respiratory assessment of strength as measured by PImax [ Time Frame: 3 months to 2 years ]
- Characterize the disease course and natural history of XLMTM using neuromuscular assessments, as measured by CHOP INTEND/MFM-20 [ Time Frame: 3 months to 2 years ]
Original Primary Outcome: Same as current
Current Secondary Outcome: Characterize quality of life as measured by PedsQL [ Time Frame: 3 months to 2 years ]
Original Secondary Outcome: Same as current
Information By: Audentes Therapeutics
Dates:
Date Received: March 1, 2016
Date Started: July 2016
Date Completion: January 2019
Last Updated: February 6, 2017
Last Verified: February 2017
