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Charcot-Marie-Tooth Disease clinical trials at UC Health
2 in progress, 1 open to eligible people

  • Study of ACE-083 in Patients With Charcot-Marie-Tooth Disease

    open to eligible people ages 18 years and up

    This is a multicenter, phase 2 study to evaluate the safety, tolerability, pharmacodynamics (PD), efficacy, and pharmacokinetics (PK) of ACE-083 in patients with CMT1 and CMTX, to be conducted in two parts. Part 1 is non-randomized, open-label, dose-escalation and Part 2 is randomized, double-blind, and placebo-controlled.

    at UC Irvine

  • 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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