Muscular Dystrophies clinical trials at UC Health
5 in progress, 3 open to eligible people
open to eligible people ages 6-65
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
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.
open to eligible males ages 5 years and up
This study is a long-term study of ataluren in participants with nonsense mutation Duchenne muscular dystrophy.
at UC Davis UCSF
Sorry, in progress, not accepting new patients
HOPE-2 is a double-blind clinical trial evaluating the safety and efficacy of a cell therapy called CAP-1002 in study participants with Duchenne muscular dystrophy (DMD). 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.
at UC Davis
Sorry, accepting new patients by invitation only
The overall goal of this natural history study is to define the key LGMD2i phenotypes as measured by standard clinical outcome assessments (COAs), and to validate a muscle biomarker for LGMD 2i to support therapeutic development.
at UC Irvine