Facioscapulohumeral Muscular Dystrophy clinical trials at University of California Health

6 in progress, 3 open to eligible people

Showing trials for

  • First-in-human Study of EPI-321 in Facioscapulohumeral Muscular Dystrophy

    open to eligible people ages 18-75

    The goal of this clinical trial is to learn how safe and tolerable EPI-321 is and whether there may be early signs it is working in male or female adult (18 to 75 years) participants with facioscapulohumeral muscular dystrophy (FSHD) Type 1 condition. The main questions it aims to answer are: How safe is EPI-321 and how well can people handle it over time? How does EPI-321 interact with its target and does it show early signs of working? Participants will receive a single dose of EPI-321 through a vein while being closely watched in a hospital and visit the clinic regularly for tests and checkups for about 5 years after getting EPI-321.

    at UCLA

  • Del-brax (Also Referred to as AOC 1020) in Participants With FSHD

    open to eligible people ages 16-70

    A Randomized, Double-blind, Placebo-controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Intravenous AOC 1020 for the Treatment of Facioscapulohumeral Muscular Dystrophy (FSHD)

    at UC Irvine

  • Motor Outcomes to Validate Evaluations in FSHD (MOVE FSHD)

    open to all eligible people

    The primary goal of this proposal is to collect motor and functional outcomes specific to FSHD over time. By collecting measures specific to FSHD, this will help ensure the best level of clinical care is being provided. Also, the hope is to speed up drug development by gaining a better understanding of how having FSHD impacts motor function and other health outcomes (i.e. breathing, wheelchair use, etc.) and how big a change in motor function would be clinically meaningful to those with FSHD. Motor Outcomes to Validate Evaluations in FSHD (MOVE FSHD) will have approximately 450 FSHD participants followed for a minimum of 3 years. A subset of MOVE FSHD participants, approximately 200, will participate in the MOVE+ sub-study which includes whole body MRI and reachable workspace, as well as optional muscle biopsy and wearable device (US participants only).

    at UCLA

  • AOC 1020 in Participants with Facioscapulohumeral Muscular Dystrophy (FSHD)

    Sorry, accepting new patients by invitation only

    A Phase 2 Open-label Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of AOC 1020 Administered Intravenously to Participants with Facioscapulohumeral Muscular Dystrophy (FSHD)

    at UCLA UCSD

  • Readiness to Solve Barriers to Drug Development in FSHD

    Sorry, in progress, not accepting new patients

    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 (FSHD-COM and EIM) and optimize eligibility criteria by testing 320 patients across 14 international sites over a period of 24 months.

    at UCLA

  • Motor Outcomes to Validate Evaluations in Pediatric FSHD (MOVE Peds)

    Sorry, not currently recruiting here

    The primary goal of this study is to validate motor and functional outcomes and refine clinical trial strategies for pediatric-onset FSHD

    at UC Irvine

Our lead scientists for Facioscapulohumeral Muscular Dystrophy research studies include .