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Facioscapulohumeral Muscular Dystrophy clinical trials at UC Health
2 in progress, 2 open to new patients

  • 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

  • Study of ACE-083 in Patients With Facioscapulohumeral Muscular Dystrophy (FSHD)

    open to eligible people ages 18 years and up

    Study A083-02 is a multicenter, Phase 2 study to evaluate the safety, tolerability, pharmacodynamics (PD), efficacy, and pharmacokinetics (PK) of ACE 083 in patients with FSHD to be conducted in two parts. Part 1 is open-label, dose-escalation and Part 2 is randomized, double-blind, and placebo-controlled.

    at UCLAUC Davis