Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.
RECRUITING
PHASE4
20 participants
INTERVENTIONAL
2021-12-29
2025-12-30
Brief Summary
Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.
Related Clinical Trials
Explore similar clinical trials based on study characteristics and research focus.
A Trial of Metformin in Individuals With Fragile X Syndrome (Met)
NCT03862950
Metformin in Patients With Fragile X
NCT04141163
A Trial of Metformin in Individuals With Fragile X Syndrome
NCT03479476
Metformin in Children and Adults With Fragile X Syndrome
NCT03722290
Study of Acamprosate in Fragile x Syndrome
NCT01911455
Detailed Description
Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.
Studies showed that there were pathogenically over activates of mTOR and MAPK/ERK pathways in FXS. Metformin, a guanidine derivative ,has been shown to reduce mTORC1 pathway activity in an AMPK-dependent manner and has also been shown to reduce MAPK pathway activity. Metformin treatments for FXS have been reported in animal experiments, some open label trials of metformin in FXS patients had been reported. Therefore, metformin has potential to rescue symptoms in children with FXS.
In this study, researchers hope to investigate the improvement effect of metformin on FXS symptoms such as behavior problems, cognition, language.
The intervention period is 6 months, follow-up visit at 1 year. The researchers will also assess the side effects of the study medication throughout the trial.
Conditions
See the medical conditions and disease areas that this research is targeting or investigating.
Study Design
Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.
RANDOMIZED
PARALLEL
TREATMENT
DOUBLE
Study Groups
Review each arm or cohort in the study, along with the interventions and objectives associated with them.
Metformin group
The patients will be obtain Metformin starting from 50mg everyday to 1-2g per day for 6 months.
Metformin
The patients in this group will be obtain Metformin starting from 50mg everyday at night, and gradually increase to maximum tolerable dose of 1-2g per day according to weights of patients for 6 months.
Placebo group
The patients will be obtain starch tablets starting from 50mg everyday to 1-2g per day for 6 months.
Placebo
The patients in this group will be obtain starch tablets (which had same appearance and size comparing to Metformin) starting from 50mg everyday to 1-2g per day according to weights of patients for 6 months.
Interventions
Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.
Metformin
The patients in this group will be obtain Metformin starting from 50mg everyday at night, and gradually increase to maximum tolerable dose of 1-2g per day according to weights of patients for 6 months.
Placebo
The patients in this group will be obtain starch tablets (which had same appearance and size comparing to Metformin) starting from 50mg everyday to 1-2g per day according to weights of patients for 6 months.
Eligibility Criteria
Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.
Inclusion Criteria
* Participate in the study with the informed consent of the guardian
* BMI\>the 3rd percentile
* Not taking more than 2 therapeutic drugs
* Able to receive regular follow-up visits
Exclusion Criteria
* Primary heart disease
* Severe infection or acute clinical illness
* Gastrointestinal, renal, or hepatic disease
* Previous history of lactic acidosis
* previous use of metformin intolerant
* Use of angiotensin converting enzyme inhibitors, use of anticoagulants, vitamin B12 deficiency, alcohol consumption
* Unstable systemic diseases other than FXS
* Changes in clinical medication
2 Years
16 Years
ALL
No
Sponsors
Meet the organizations funding or collaborating on the study and learn about their roles.
Children's Hospital of Fudan University
OTHER
Responsible Party
Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.
Locations
Explore where the study is taking place and check the recruitment status at each participating site.
Children's Hospital of Fudan University
Shanghai, Shanghai Municipality, China
Countries
Review the countries where the study has at least one active or historical site.
Central Contacts
Reach out to these primary contacts for questions about participation or study logistics.
Facility Contacts
Find local site contact details for specific facilities participating in the trial.
Other Identifiers
Review additional registry numbers or institutional identifiers associated with this trial.
FXS2021_1.0
Identifier Type: -
Identifier Source: org_study_id
More Related Trials
Additional clinical trials that may be relevant based on similarity analysis.