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Anti-diabetic drug

Metformin for Fragile X Syndrome

Phase 2

Recruiting

Led By Francois Bolduc, MD

Research Sponsored by University of Alberta

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Subject has Fragile X syndrome with a molecular genetic confirmation of the full FMR1 mutation (>200 CGG repeats) or the other loss of function mutations of the FMR1 gene (SNVs and deletions of the gene).

Subjects who are capable of becoming pregnant must use an acceptable method of birth control for the duration of the study. Acceptable forms of birth control include abstinence (only for subjects who are not sexually active), intrauterine devices in place for at least 3 months, oral contraceptives, surgical sterilization, or adequate barrier methods.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline, week 8, end of treatment/week 16

Awards & highlights

Study Summary

This trial will study whether metformin is a safe and effective treatment for language deficits, behavior problems, and obesity/excessive appetite in people with fragile X syndrome.

Who is the study for?

This trial is for males and non-pregnant females aged 6-35 with Fragile X syndrome confirmed by genetic testing. Participants must be able to attend clinic visits, have a caregiver involved, speak English or French, and use birth control if applicable. Exclusions include pregnancy, severe vitamin B12 deficiency, certain medical conditions, history of adverse reactions to metformin or recent treatment with it.Check my eligibility

What is being tested?

The study tests the safety and effectiveness of metformin compared to a placebo in treating language deficits, behavioral issues, and obesity/excessive appetite in individuals with Fragile X syndrome. It's a double-blind trial where participants are randomly assigned to receive either metformin or placebo over four months.See study design

What are the potential side effects?

While not specified here, common side effects of metformin can include digestive issues like nausea and diarrhea; however specific side effects will be monitored throughout the trial given the unique population.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have Fragile X syndrome confirmed by genetic testing.

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I am using or willing to use an approved birth control method during the study.

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I or my caregiver can fluently understand and communicate in English or French for study materials.

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I am 13 or younger with an IQ of 85 or less and can speak in 3-word phrases.

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I am a male or a non-pregnant, non-breastfeeding female aged between 6 and 35.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline, week 8, end of treatment/week 16

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline, week 8, end of treatment/week 16

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline, week 8, end of treatment/week 16 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Change from baseline in the Expressive Language Sampling (ELS) mean Number of Different Words (NDW) score

Secondary outcome measures

Change from baseline in Event Related Potentials during the Auditory Oddball Paradigm and Pseudoword Paradigm

Change from baseline in Eye Tracking

Change from baseline in The Memory Game

+19 more

Trial Design

2Treatment groups

Active Control

Placebo Group

Group I: Active Metformin MedicationActive Control1 Intervention

The active metformin medication will be dosed in a weight-dependent manner. For participants under 50kg at baseline, the initial dose will be 250mg once per day, and if this dose is well tolerated, they will increase each week by 250mg until a maximum dose of 1000mg daily is reached. For participants at and above 50kg at baseline, the initial dose will be 500mg once per day, and if this dose is well tolerated, they will increase each week by 500mg until a maximum dose of 2000mg daily is reached. After the 4-week titration period, each participant will continue dosing at his or her maximum tolerated dose daily for the remaining 12 weeks of the study.

Group II: Placebo MedicationPlacebo Group1 Intervention

The placebo will be dosed in a weight-dependent manner. For participants under 50kg at baseline, the initial dose will be 250mg once per day, and if this dose is well tolerated, they will increase each week by 250mg until a maximum dose of 1000mg daily is reached. For participants at and above 50kg at baseline, the initial dose will be 500mg once per day, and if this dose is well tolerated, they will increase each week by 500mg until a maximum dose of 2000mg daily is reached. After the 4-week titration period, each participant will continue dosing at his or her maximum tolerated dose daily for the remaining 12 weeks of the study.

0 / 5Eligibility criteria met

Find a Location

Who is running the clinical trial?

St. Justine's HospitalOTHER

196 Previous Clinical Trials

78,909 Total Patients Enrolled

University of AlbertaLead Sponsor

889 Previous Clinical Trials

385,014 Total Patients Enrolled

Francois Bolduc, MDPrincipal InvestigatorUniversity of Alberta

Media Library

Metformin (Anti-diabetic drug) Clinical Trial Eligibility Overview. Trial Name: NCT03862950 — Phase 2

Genetic Diseases Research Study Groups: Active Metformin Medication, Placebo Medication

Genetic Diseases Clinical Trial 2023: Metformin Highlights & Side Effects. Trial Name: NCT03862950 — Phase 2

Metformin (Anti-diabetic drug) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03862950 — Phase 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Does this test have an age cap, and if so, is participation over 45 years of age still possible?

"According to the enrollment rules of this medical research, only individuals between 6 and 35 years old can join. There are 446 trials for minors and 1168 studies which specifically target people over 65."

Answered by AI

How many individuals are being granted access to this clinical research?

"Affirmative, according to the data present at clinicaltrials.gov this medical research is currently recruiting subjects. It was first advertised on May 24th 2019 and its parameters were amended as recently as November 25th 2022. This investigation seeks 120 participants from two distinct sites."

Answered by AI

Is the door of opportunity currently open for participants to join this medical experiment?

"Affirmative. The clinicaltrials.gov website reveals that this medical trial began on May 24th 2019 and continues to actively recruit participants with a current goal of 120 across two sites."

Answered by AI

To what ailments is Active Metformin Medication typically prescribed?

"Exercise is commonly treated with Active Metformin Medication, which has also been found to be a successful course of treatment for other illnesses such as type 1 diabetes mellitus, diabetic ketoacidosis and polycystic ovary syndrome."

Answered by AI

Could you provide information on additional clinical trials involving Active Metformin Medication?

"Currently, there are 170 Active Metformin Medication studies underway with a total of 43 in the third phase. The majority of these trials take place in Pittsburgh, PA., but 1903 other sites across the country have active research as well."

Answered by AI

Who meets the criteria for inclusion in this medical research?

"This clinical trial is recruiting 120 participants aged between 6 and 35 with trinucleotide repeat expansion. Qualifying subjects need to meet a number of additional criteria: being male or non-pregnant, female; practising an effective form of birth control (abstinence, intrauterine device use for at least 3 months, oral contraceptives, surgical sterilization or barrier methods); having Fragile X Syndrome confirmed via molecular genetic testing (>200 CGG repeats) or other loss of function mutations on the FMR1 gene (SNVs and deletions); accompanied by a willing caregiver capable of understanding study materials in English/French;"

Answered by AI

Has Active Metformin Medication been granted the stamp of approval by the FDA?

"Our team's assessment of the safety profile for Active Metformin Medication is a 2, seeing as there are preliminary results which show it to be safe but few studies that affirm its effectiveness."

Answered by AI

Recent research and studies

Nature MedicineJournal

Metformin Ameliorates Core Deficits in a Mouse Model of Fragile X Syndrome

Gantois, Ilse, Arkady Khoutorsky, Jelena Popic, Argel Aguilar-Valles, Erika Freemantle, Ruifeng Cao, Vijendra Sharma, et al.. 2017. “Metformin Ameliorates Core Deficits in a Mouse Model of Fragile X Syndrome”. Nature Medicine. Springer Science and Business Media LLC. doi:10.1038/nm.4335.

Molecular PsychiatryJournal

Insulin Signaling Misregulation Underlies Circadian and Cognitive Deficits in a Drosophila Fragile X Model

Monyak, R E, D Emerson, B P Schoenfeld, X Zheng, D B Chambers, C Rosenfelt, S Langer, et al.. 2016. “Insulin Signaling Misregulation Underlies Circadian and Cognitive Deficits in a Drosophila Fragile X Model”. Molecular Psychiatry. Springer Science and Business Media LLC. doi:10.1038/mp.2016.51.

Clinical GeneticsJournal

Metformin as Targeted Treatment in Fragile X Syndrome

Dy, A.B.C., F. Tassone, M. Eldeeb, M.J. Salcedo-Arellano, N. Tartaglia, and R. Hagerman. 2017. “Metformin as Targeted Treatment in Fragile X Syndrome”. Clinical Genetics. Wiley. doi:10.1111/cge.13039.

clinicaltrials.govStudy