Genetic Diseases, X-Linked clinical trials at UC Health
4 in progress, 3 open to new patients
“Would the study drug (CAP-1002) help improve weakened skeletal and heart muscle caused by DMD?”
open to eligible males ages 10 years and up
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
“Learn if Metformin may be helpful for language, behavior problems, and obesity / excessive appetite in patients with Fragile X Syndrome.”
open to eligible people ages 6-25
This study is a controlled trial of metformin in individuals with fragile X syndrome between the ages of 6 and 25 years. Participants will be randomized in a double-blind design to either drug or placebo and will attend three visits to the study site in a 4-month period for a series of tests. The primary objectives are to assess safety, tolerability, and efficacy of metformin in the treatment of language deficits, behavior problems, and obesity/excessive appetite in individuals with fragile X syndrome.
at UC Davis
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 UCSF UC Davis
Sorry, in progress, not accepting new patients
The main goal of this Phase 3 extension study is to obtain long term safety of ataluren in boys with nonsense mutation dystrophinopathy as determined by adverse events and laboratory abnormalities. The study will also assess changes in physical function, pulmonary function and other important clinical and laboratory measures.
at UCLA UC Davis