Metformin in Patients With Fragile X
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|ClinicalTrials.gov Identifier: NCT04141163|
Recruitment Status : Recruiting
First Posted : October 28, 2019
Last Update Posted : October 31, 2019
|Condition or disease||Intervention/treatment||Phase|
|Fragile X Syndrome||Drug: Metformin Drug: Placebo oral tablet||Phase 1 Phase 2|
This trial will be a randomized placebo controlled, double-blind, parallel group design study of treatment with metformin on the primary outcome of safety/tolerance with secondary outcome measurements of the effects on cognition (encompassing social and repetitive behavior), attention, anxiety, and physiological and biochemical biomarkers of patients with FXS. FXS represents a well-defined population of ASD in which to test a specific targeted treatment looking at a well-defined set of cognitive and bioassay measures.
Trial length is designed to have a chance at seeing if the medication can improve cognitive outcome measures. The study duration includes the screening period and a 24-week single-blind drug/placebo phase.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||40 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|Official Title:||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|
|Actual Study Start Date :||October 29, 2019|
|Estimated Primary Completion Date :||March 2021|
|Estimated Study Completion Date :||March 2021|
Subjects randomized to metformin will start at 500mg once a day for 7 days and increase as is tolerated to 500mg twice a day for 7 days, then to 1000mg in the morning and 500mg at dinner for 7 days and then to the target dose of 1,000mg twice a day.
Metformin, 1,1 dimethylbiguanide, or systematic (IUPAC) name N,N-dimethylimidodicarbonimidic diamide, is an oral anti-diabetic medicine approved in the US by the FDA in 1994. It is marketed alone under the names metformin (generic), Glucophage XR, Riomet, Fortamet, Glumetza, Obimet, Gluformin, Dianben, Diabex, and Diaformin and in combination with other drugs under the names Actoplus Met, Metaglip, Glucovance, Janumet, Kombiglyze XR, and PrandiMet
Other Name: glucophage
Placebo Comparator: Placebo
Subjects randomized to placebo will start at 500mg once a day for 7 days and increase as is tolerated to 500mg twice a day for 7 days, then to 1000mg in the morning and 500mg at dinner for 7 days and then to the target dose of 1,000mg twice a day.
Drug: Placebo oral tablet
No therapeutic effect
- 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. [ Time Frame: 1-2 years ]measured by the number of reported adverse events, assessed using the Safety Monitoring Uniform Report Form (SMURF), modified for metformin use
- Patients taking Metforming have improved cognition, sleep, attention or anxiety from baseline to the end of the study [ Time Frame: 1-2 years ]measured by a variety of questionnaires and assessments done throughout the length of the study
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04141163
|Contact: Lauren Fedoremail@example.com|
|United States, New Jersey|
|Rowan University School of Osteopathic Medicine||Recruiting|
|Stratford, New Jersey, United States, 08084|
|Contact: Lauren Fedor 856-566-6003 firstname.lastname@example.org|
|Principal Investigator:||Sean McBride, MD, PhD||Rowan University|