Muscular Dystrophies clinical trials at University of California Health
9 in progress, 6 open to eligible people
A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy
open to eligible males ages 10 years and up
HOPE-3 is a multi-center, randomized, double-blind, placebo-controlled clinical trial evaluating the safety and efficacy of a cell therapy called 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 CAP-1002 or placebo every 3 months for a total of 4 doses during a 12-month period. All participants will be eligible to receive CAP-1002 for an additional 12 months as part of an open label extended assessment period.
at UC Davis
A Study to Evaluate the Safety of 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 LION-101 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: 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.
Defining Clinical Endpoints in Limb Girdle Muscular Dystrophy (LGMD)
open to eligible people ages 4-65
Limb Girdle Muscular Dystrophy comprise a group of disorders made up of over 30 mutations which share a common phenotype of progressive weakness of the shoulder and hip girdle muscles. While the individual genetic mutations are rare, as a cohort, LGMDs are one of the four most common muscular dystrophies. The overall goal of project 1 is to define the key phenotypes as measured by standard clinical outcome assessments (COAs) for limb girdle muscular dystrophies (LGMD) to hasten therapeutic development.
at UC Irvine
Defining Endpoints in Becker Muscular Dystrophy
open to eligible males ages 8 years and up
This is a 24-month, observational study of 150 participants with Becker muscular dystrophy (BMD)
at UC Irvine
Extension of AOC 1001-CS1 (MARINA) Study in Adult Myotonic Dystrophy Type 1 (DM1) Patients
Sorry, accepting new patients by invitation only
AOC 1001-CS2 (MARINA-OLE) is a Phase 2 extension of the AOC 1001-CS1 (MARINA) study to evaluate the safety, tolerability, efficacy, pharmacokinetics and pharmacodynamics of multiple-doses of AOC 1001 Administered Intravenously to Adult Myotonic Dystrophy Type 1 (DM1) patients
Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy
Sorry, in progress, not accepting new patients
This study is a long-term study of ataluren in participants with nonsense mutation Duchenne muscular dystrophy.
at UC Davis UCSF
Phase 1/2 Study of AOC 1020 in Adults With Facioscapulohumeral Muscular Dystrophy (FSHD)
Sorry, not currently recruiting here
A Randomized, Double-blind, Placebo-controlled, Phase 1/2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of AOC 1020 Administered Intravenously to Adult Participants with Facioscapulohumeral Muscular Dystrophy (FSHD)
at UC Irvine UCSD
Our lead scientists for Muscular Dystrophies research studies include Karunesh Ganguly, MD, PhD Tahseen Mozaffar, MD.