Fragile X Syndrome Clinical Trials

18 recruiting

Fragile X Syndrome Trials at a Glance

16 actively recruiting trials for fragile x syndrome are listed on ClinicalTrialsFinder across 6 cities in 12 countries. The largest study group is Not Applicable with 9 trials, with the heaviest enrollment activity in Cincinnati, Chicago, and Baltimore. Lead sponsors running fragile x syndrome studies include Children's Hospital Medical Center, Cincinnati, Bambino Gesù Hospital and Research Institute, and Assistance Publique - Hôpitaux de Paris.

Browse fragile x syndrome trials by phase

Treatments under study

About Fragile X Syndrome Clinical Trials

Looking for clinical trials for Fragile X Syndrome? There are currently 18 studies actively recruiting participants. Clinical trials offer access to new treatments before they are widely available, and every approved therapy in use today was first tested through a clinical trial.

Below you can browse trials, sign up for alerts when new Fragile X Syndrome trials open, and view eligibility criteria for each study. Each listing includes the study phase, locations, and enrollment details.

Frequently Asked Questions

Common questions about Fragile X Syndrome clinical trials

A clinical trial is a carefully designed research study that tests new medical treatments, drugs, devices, or approaches in human volunteers. Every approved medication and treatment available today was proven safe and effective through clinical trials.

All clinical trials are reviewed and approved by Institutional Review Boards (IRBs) — independent committees that evaluate patient safety. Trials follow strict protocols, and your health is monitored closely throughout. You can withdraw at any time.

Not necessarily. Many trials compare the new treatment against the current standard of care, meaning all participants receive active treatment. When placebos are used, they are typically combined with standard treatment, not given alone. The trial description will always specify the design.

Under the Affordable Care Act, most private insurers are required to cover routine patient care costs during a clinical trial. The sponsor typically covers the investigational treatment itself. Medicare also covers routine costs for qualifying trials.

Yes. Participation is completely voluntary. You can withdraw at any time, for any reason, without it affecting your access to standard medical care.

Each trial has specific eligibility criteria — including age, diagnosis, disease stage, prior treatments, and general health. Browse the trials listed above and check their eligibility sections. You can also contact the trial site directly to discuss your situation.

Showing 116 of 16 trials

Recruiting
Phase 2

Study of MRM-3379 in Male Participants With Fragile X Syndrome (BLOOM)

Fragile X Syndrome
Mirum Pharmaceuticals, Inc.60 enrolled15 locationsNCT07209462
Recruiting

Development of Non-Invasive Prenatal Diagnosis for Single Gene Disorders

Cystic FibrosisHuntington DiseaseSickle Cell Disease+12 more
Assistance Publique - Hôpitaux de Paris550 enrolled1 locationNCT06147414
Recruiting
Not Applicable

Optical Imaging in X-linked Disorders.

Fragile X Syndrome (FXS)Creatine Transporter Deficiency
Hospices Civils de Lyon88 enrolled1 locationNCT06868979
Recruiting
Not Applicable

Alpha Auditory Entrainment for Cognitive Enhancement and Sensory Hypersensitivity in Youth With Developmental Disorders

Fragile X SyndromeAutism Spectrum DisorderAsperger Syndrome+1 more
Children's Hospital Medical Center, Cincinnati180 enrolled1 locationNCT06227780
Recruiting
Phase 4

The Psychiatric and Cognitive Phenotypes in Velocardiofacial Syndrome

Williams SyndromeFragile X SyndromeVelocardiofacial Syndrome
The Chaim Sheba Medical Center400 enrolled1 locationNCT00768820
Recruiting
Not Applicable

Speech-in-noise Perception in Autism and Fragile X

Fragile X SyndromeAutism Spectrum Disorder
Children's Hospital Medical Center, Cincinnati60 enrolled1 locationNCT06088589
Recruiting
Not Applicable

Tracking Early Emergence of Sound Perception Impairments in FXS With Multimodal fNIRS/EEG- Infant

Fragile X Syndrome
Children's Hospital Medical Center, Cincinnati30 enrolled1 locationNCT06560242
Recruiting
Not Applicable

Physical Activity and Community EmPOWERment Project

Williams SyndromeWAGR SyndromePrader-Willi Syndrome+10 more
University of North Carolina, Chapel Hill376 enrolled2 locationsNCT06740162
Recruiting
Not Applicable

Web Intervention for Parents of Youth With Genetic Syndromes (WINGS)

Tuberous SclerosisFragile X SyndromeRett Syndrome+5 more
Rush University Medical Center92 enrolled1 locationNCT06139172
Recruiting
Phase 2

Sertraline vs. Placebo in the Treatment of Anxiety in Children and AdoLescents With NeurodevelopMental Disorders

AnxietyTuberous SclerosisFragile X Syndrome+14 more
Holland Bloorview Kids Rehabilitation Hospital130 enrolled8 locationsNCT06081348
Recruiting
Not Applicable

Tracking Early Emergence of Sound Perception Impairments in FXS With Multimodal fNIRS/EEG-Preschool Age

Fragile X Syndrome
Children's Hospital Medical Center, Cincinnati90 enrolled1 locationNCT05957549
Recruiting
Phase 4

Metformin in Children With Fragile X Syndrome

Fragile X SyndromeMetformin
Children's Hospital of Fudan University20 enrolled1 locationNCT05120505
Recruiting
Not Applicable

Group CBT in Adolescents With Fragile X Syndrome and in Adolescents With Autism Spectrum Disorder

Autism Spectrum DisorderFragile X Syndrome (FXS)
Bambino Gesù Hospital and Research Institute20 enrolled1 locationNCT06677866
Recruiting
Not Applicable

Probiotic Intervention for Microbiome Modifications and Clinical Improvements in Fragile X Syndrome

Fragile X Syndrome
Specila hospital for cerebral palsy and developmental neurology15 enrolled1 locationNCT06279858
Recruiting
Phase 3

Clinical study of cannabidiol in children, adolescents and young adults with Fragile X syndrome (RECONNECT)

Fragile X Syndrome
Zynerba Pharmaceuticals Pty Ltd250 enrolled8 locationsACTRN12621000874819
Recruiting
Phase 2

Clinical study Of caNNabidiol in children and adolesCenTs with Fragile X Open-Label Extension (CONNECT-FX OLE)

Fragile X Syndrome
Zynerba Pharmaceuticals Pty. Ltd300 enrolled5 locationsACTRN12618001868279