A Parallel Group Design Randomized Double-Blind Trial of Metformin Treatment in Patients With Fragile X Syndrome on Safety and Effects on Cognition, Anxiety, Attention and Biomarkers

NCT04141163 Phase 1/Phase 2 UNKNOWN

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.

Details

Lead sponsor	Rowan University
Phase	Phase 1/Phase 2
Status	UNKNOWN
Enrolment	40
Start date	2019-10-29
Completion	2021-03

Conditions

• Fragile X Syndrome

Interventions

• Metformin
• Placebo oral tablet

Primary outcomes

• The safety and tolerability of metformin in patients with Fragile X Syndrome as assessed by the number of adverse events reported during the course of the study. — 1-2 years
  measured by the number of reported adverse events, assessed using the Safety Monitoring Uniform Report Form (SMURF), modified for metformin use

Countries

United States