The purpose of this trial is to investigate the use of metformin in the treatment of Fragile
X syndrome (FXS) patients. Metformin is an FDA approved compound with an established safety
profile and minimal side effects that specifically targets and normalizes multiple aspects of
the pathophysiology in FXS. This is a randomized double-blind placebo-controlled 2-arm
parallel group design study of the drug metformin and placebo in FXS subjects with a primary
outcome measure of safety/tolerability and secondary outcome measures on cognition,
attention, anxiety, sleep, and physiologic and biochemical biomarkers.
Phase:
Phase 1/Phase 2
Details
Lead Sponsor:
Rowan University
Collaborators:
FRAXA Research Foundation University of Pennsylvania