Genetic Diseases, X-Linked clinical trials at UC Health
3 in progress, 2 open to new patients
“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
“Would the study drug (CAP-1002) help improve weakened skeletal and heart muscle caused by DMD?”
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 UCLA UC Davis