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Facioscapulohumeral Muscular Dystrophy clinical trials at UC Health
2 in progress, 1 open to eligible people

  • Clinical Trial Readiness to Solve Barriers to Drug Development in FSHD

    open to eligible people ages 18-75

    The primary cause of facioscapulohumeral muscular dystrophy (FSHD), a common adult-onset dystrophy, was recently discovered identifying targets for therapy. As multiple drug companies pursue treatments for FSHD, there is an urgent need to define the clinical trial strategies which will hasten drug development, including creating disease-relevant outcome measures and optimizing inclusion criteria. This proposal will develop two new outcome measures and optimize eligibility criteria by testing 160 patients in 7 sites over a period of 18 months.

    at UCLA

  • A Study of the Long-Term Effects of Experimental ACE-083 for CMT type 1, CMT type X, or Facioscapulohumeral Muscular Dystrophy

    Sorry, accepting new patients by invitation only

    This is an open-label, multicenter, phase 2 extension study to evaluate the safety, tolerability, PK, PD, and efficacy of ACE-083 in subjects with FSHD previously enrolled in Study A083-02 and subjects with CMT1 and CMTX previously enrolled in Study A083-03. This study will be conducted in two Parts: Part 1, which is a loading phase of 6 months' duration, and Part 2, the maintenance phase, which will last up to 24 months.

    at UC Davis UC Irvine UCLA

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