Muscular Dystrophies clinical trials at University of California Health

9 in progress, 5 open to eligible people

Showing trials for

  • AB-1003 (Previously LION-101) in Subjects With Genetic Confirmation of LGMD2I/R9 (Part1)

    open to eligible people ages 18-65

    The purpose of this study is to evaluate the safety and tolerability of a single intravenous infusion of AB-1003 in adults diagnosed with limb girdle muscular dystrophy type 2I/R9 (LGMD2I/R9). Participants will be treated in sequential, dose-level cohorts. (Part 1)

    at UC Irvine

  • BrainGate2 Neural Interface System in Persons With Tetraplegia (BG-Speech-02)

    open to eligible people ages 18-80

    The goal of this study is to improve our understanding of speech production, and to translate this into medical devices called intracortical brain-computer interfaces (iBCIs) that will enable people who have lost the ability to speak fluently to communicate via a computer just by trying to speak.

    at UC Davis

  • BrainGate2: Feasibility Study of an Intracortical Neural Interface System for Persons With Tetraplegia

    ““Turn thoughts into action” for people living with paralysis”

    open to eligible people ages 18-80

    The purpose of this study is to obtain preliminary device safety information and demonstrate proof of principle (feasibility) of the ability of people with tetraplegia to control a computer cursor and other assistive devices with their thoughts.

    at UC Davis

  • ECoG BMI for Motor and Speech Control

    open to eligible people ages 21 years and up

    Test the feasibility of using electrocorticography (ECoG) signals to control complex devices for motor and speech control in adults severely affected by neurological disorders.

    at UCSF

  • SAT-3247 in Pediatric Ambulatory Patients

    open to eligible males ages 7-9

    Phase 2a trial of SAT-3247 in ambulatory DMD patients aged ≥ 7 and < 10 years. The trial will study two doses of SAT-3247 in a randomized, double-blind, placebo-controlled weekday regimen for 12 weeks to determine the optimal dose, safety, tolerability, and preliminary efficacy.

    at UCLA

  • Managed Access Program for Del-zota in Participants With DMD Mutations Amenable to Exon 44 Skipping

    Sorry, not accepting new patients

    The purpose of this Managed Access Program is to allow access to delpacibart zotadirsen (AOC 1044) for eligible patients diagnosed with DMD mutations amenable to exon 44 skipping. The patient's Administering Physician should follow the suggested treatment guidelines and comply with all local health authority regulations.

    at UC Irvine

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

    Sorry, in progress, not accepting new patients

    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

  • Deramiocel (CAP-1002) in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy

    Sorry, in progress, not accepting new patients

    HOPE-3 is a two cohort, Phase 3, multi-center, randomized, double-blind, placebo-controlled clinical trial evaluating the efficacy and safety of a cell therapy called deramiocel (CAP-1002) in study participants with Duchenne muscular dystrophy (DMD) and impaired skeletal muscle function. Non-ambulatory and ambulatory boys and young men who meet eligibility criteria will be randomly assigned to receive either deramiocel or placebo every 3 months for a total of 4 doses during the first 12 months of the study. All participants will be eligible to receive 4 doses of deramiocel for an additional 12 months as part of an open-label extended assessment period. After completion of the first open-label extension (Months 12-24), subjects who have completed Month 24 are eligible to continue onto a Long-Term Open-Label Extension period that will provide treatment with deramiocel until commercial availability, or until sponsor's decision to terminate the trial, or the participant withdraws consent.

    at UC Davis UCSD

  • Defining Endpoints in Becker Muscular Dystrophy

    Sorry, in progress, not accepting new patients

    This is a 24-month, observational study of 50 participants with Becker muscular dystrophy (BMD)

    at UC Irvine

Our lead scientists for Muscular Dystrophies research studies include .

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