Fragile X Syndrome clinical trials
The registry lists 115 studies for Fragile X Syndrome across more than 10 countries. 25 are recruiting or about to start. 90 of them are mainly about Fragile X Syndrome (how this is decided). Per ClinicalTrials.gov, last updated 2026-11-12.
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· Enrollment: 300 → 450 participants
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Who runs them
Seaside Therapeutics, Inc. (9) · Children's Hospital Medical Center, Cincinnati (8) · Stanford University (7) · University of California, Davis (6) · Novartis Pharmaceuticals (6) · Université de Sherbrooke (4) · Tetra Discovery Partners (4) · Connecta Therapeutics, S.L. (3)
What is being tested here
Drugs under test in Fragile X Syndrome studies, those with the most open studies first. Medicines only given beside them are not counted. How drugs are counted
Coming up
Studies recruiting or running, from their records. “Main data collection” ends at the primary completion date, the sponsor’s own estimate, which often moves.
The 10 nearest, phase 3 first
- Sep 2027An Open-Label Extension Study of BPN14770 in Subjects With Fragile X Syndrome · Phase 3 · Tetra Discovery Partners
- Nov 2026Early Check: Expanded Screening in Newborns · RTI International
- Dec 2026Web Intervention for Parents of Youth With Genetic Syndromes (WINGS) · Rush University Medical Center
- Mar 2027Tracking Early Emergence of Sound Perception Impairments in FXS With Multimodal fNIRS/EEG- Infant · Children's Hospital Medical Center, Cincinnati
- Mar 2027Tracking Early Emergence of Sound Perception Impairments in FXS With Multimodal fNIRS/EEG-Preschool Age · Children's Hospital Medical Center, Cincinnati
- May 2027Development of Non-Invasive Prenatal Diagnosis for Single Gene Disorders · Assistance Publique - Hôpitaux de Paris
- May 2027Safety, Tolerability, and Preliminary Effectiveness of CTH120 in Fragile X Syndrome · Connecta Therapeutics, S.L.
- May 2027Alpha Auditory Entrainment for Cognitive Enhancement and Sensory Hypersensitivity in Youth With Developmental Disorders · Children's Hospital Medical Center, Cincinnati
- Aug 2027The Psychiatric and Cognitive Phenotypes in Velocardiofacial Syndrome · The Chaim Sheba Medical Center
- Sep 2027Sertraline vs. Placebo in the Treatment of Anxiety in Children and AdoLescents With NeurodevelopMental Disorders · Holland Bloorview Kids Rehabilitation Hospital
Who these studies take
Of the 18 Fragile X Syndrome studies recruiting now, 5 (28%) set no upper age limit and 13 (72%) stop at 75 or younger. 15 (83%) take children under 18. 1 (6%) are for women only and 3 (17%) for men only; 7 (39%) take healthy volunteers.
| Age | Women | Men |
|---|---|---|
| 10 | 10 (56%) | 11 (61%) |
| 18 | 10 (56%) | 12 (67%) |
| 30 | 7 (39%) | 9 (50%) |
| 40 | 7 (39%) | 9 (50%) |
| 50 | 6 (33%) | 6 (33%) |
| 65 | 4 (22%) | 4 (22%) |
| 75 | 4 (22%) | 4 (22%) |
| 85 | 4 (22%) | 4 (22%) |
Across all 115 studies, recruiting or not: 84% stop at 75 or younger and 84% at 65; 70% take children; 3% are for women only and 23% for men only; 21% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.
How long Fragile X Syndrome studies take
Of 15 phase 2 interventional studies under Fragile X Syndrome, half reached primary completion within 24 months of starting, counting those still running.
| Start to primary completion | Every sponsor | Industry | Non-industry |
|---|---|---|---|
| Phase 2 | 24 mo15–37 · 15 studies | — | — |
Each figure is the month by which half the studies had reached primary completion, with the range in which the middle half did. Studies still running count until their last registry update; studies that stopped early are left out. Phase 1 includes early phase 1; observational means prospective or not stated, as retrospective and cross-sectional studies date a window of data rather than a study. How these figures are worked out
What changed recently
From the registry's own updates since Oct 15, 2026.
Newly registered2
Studies
Recruiting first, then most recently updated. Showing 50 of 115. Browse all 115 → or search them.
- A Study to Assess Safety, Tolerability, Pharmacokinetics, and Efficacy of S247240 in Adult Male Participants With Fragile X SyndromeNot yet recruitingNCT07930520Phase 2
- Inhibition of Aggressive Behavior in Participants With Fragile X SyndromeNot yet recruitingNCT07328529Phase 21 country
- Single Session Support for Parents of Children With Neurodevelopmental Disabilities Struggling With Anxious AvoidanceNot yet recruitingNCT07898592Not applicable1 country
- Development of Non-Invasive Prenatal Diagnosis for Single Gene DisordersRecruitingNCT061474141 country
- A Study to Investigate the Effects and Safety of Codabakalner for the Treatment of FXS in Male Participants and Adult Participants With ADSNot yet recruitingNCT07439510Phase 2/3
- Study of MRM-3379 in Male Participants With Fragile X Syndrome (BLOOM)RecruitingNCT07209462Phase 22 countries
- Effect of CANnabidiol on Anxiety and GABAergic Function in Individuals With Fragile-X SyndromeRecruitingNCT06261502Phase 21 country
- The Neurocognitive Bases of Trust in Intellectual DisabilityRecruitingNCT074340371 country
- Sertraline vs. Placebo in the Treatment of Anxiety in Children and AdoLescents With NeurodevelopMental DisordersRecruitingNCT06081348Phase 21 country
- Safety, Tolerability, and Preliminary Effectiveness of CTH120 in Fragile X SyndromeRecruitingNCT07654114Phase 21 country
- Parent and Infant Inter(X)Action Intervention (PIXI)Enrolling by invitationNCT03836300Not applicable1 country
- Tracking Early Emergence of Sound Perception Impairments in FXS With Multimodal fNIRS/EEG- InfantRecruitingNCT06560242Not applicable1 country
- Optical Imaging in X-linked Disorders.RecruitingNCT06868979Not applicable1 country
- Alpha Auditory Entrainment for Cognitive Enhancement and Sensory Hypersensitivity in Youth With Developmental DisordersRecruitingNCT06227780Not applicable1 country
- Intensive Multimodal Neurorehabilitation Targeting Neuroplasticity in Pediatric Neurodevelopmental and Chromosomal DisordersRecruitingNCT074930961 country
- The Psychiatric and Cognitive Phenotypes in Velocardiofacial SyndromeRecruitingNCT00768820Phase 41 country
- Physical Activity and Community EmPOWERment ProjectRecruitingNCT06740162Not applicable1 country
- Sensitivity of the NIH Toolbox to Stimulant Treatment in Intellectual DisabilitiesEnrolling by invitationNCT05301361Phase 11 country
- Tracking Early Emergence of Sound Perception Impairments in FXS With Multimodal fNIRS/EEG-Preschool AgeRecruitingNCT05957549Not applicable1 country
- Effect of CBD on the BrainNot yet recruitingNCT06261450Phase 2
- Group CBT in Adolescents With Fragile X Syndrome and in Adolescents With Autism Spectrum DisorderRecruitingNCT06677866Not applicable1 country
- Probiotic Intervention for Microbiome Modifications and Clinical Improvements in Fragile X SyndromeRecruitingNCT06279858Not applicable1 country
- Optical Imaging as a Tool for Monitoring Brain Function in Fragile X SyndromeRecruitingNCT062930271 country
- Ghrelin in Patients With a Rare Disease Associated With Intellectual Disability, and Hyperphagia, and/or Overweight, and/or ObesityRecruitingNCT047688031 country
- Validation of Optical Genome Mapping for the Identification of Constitutional Genomic Variants in a Postnatal CohortRecruitingNCT052952771 country
- Speech-in-noise Perception in Autism and Fragile XCompletedNCT06088589Not applicable1 country
- Pediatric Patients With Metabolic or Other Genetic DisordersCompletedNCT027699491 country
- Placebo-Controlled, Single-Dose Challenge Study of Gaboxadol in Adult Males With Fragile X Syndrome (FXS)CompletedNCT06334419Phase 21 country
- Web Intervention for Parents of Youth With Genetic Syndromes (WINGS)Active, not recruitingNCT06139172Not applicable1 country
- Clinical Study of Cannabidiol in Children, Adolescents, and Young Adults With Fragile X SyndromeCompletedNCT04977986Phase 35 countries
- A Randomized Study of BPN14770 in Male Adolescents (Aged 9 to < 18 Years) With Fragile X SyndromeCompletedNCT05163808Phase 2/31 country
- Metformin in Children With Fragile X SyndromeCompletedNCT05120505Phase 41 country
- A Study of BPN14770 in Male Adults (Aged 18 to 45) With Fragile X SyndromeCompletedNCT05358886Phase 31 country
- Early Check: Expanded Screening in NewbornsActive, not recruitingNCT036552231 country
- Open Label Extension to Assess the Long-Term Safety and Tolerability of ZYN002 in Children and Adolescents With FXSActive, not recruitingNCT03802799Phase 2/34 countries
- Study of SPG601 in Adult Men With Fragile X SyndromeCompletedNCT06413537Phase 21 country
- A Trial of Metformin in Individuals With Fragile X Syndrome (Met)CompletedNCT03862950Phase 21 country
- Glutaminergic Transmission in Autism : Molecular Imaging ExplorationCompletedNCT02719951Phase 11 country
- Evaluating the Neurophysiologic and Clinical Effects of Single Dose Drug ChallengeActive, not recruitingNCT05418049Phase 21 country
- An Open-Label Extension Study of BPN14770 in Subjects With Fragile X SyndromeActive, not recruitingNCT05367960Phase 31 country
- NIPD on CFTC for Triplet Repeat DiseasesCompletedNCT03087526Not applicable1 country
- A Non-Interventional Pilot Study to Explore the Role of Gut Flora in AutismWithdrawnNCT041008631 country
- Neural Biomarkers in Fragile X SyndromeCompletedNCT069570541 country
- CTH120 First-in-Human Study: Single and Multiple Ascending Doses and Potential Food InteractionCompletedNCT06480968Phase 11 country
- A Trial of Metformin in Individuals With Fragile X SyndromeCompletedNCT03479476Phase 2/32 countries
- A 2-Period Crossover Study of BPN14770 in Adults Males With Fragile X SyndromeCompletedNCT03569631Phase 21 country
- Protein Synthesis in the Brain of Patients With Fragile X SyndromeCompletedNCT003628431 country
- ERG/5-HTP in Fragile X Syndrome (FXS)CompletedNCT05030129Phase 21 country
- An Investigation of Psilocybin on Behavioural and Cognitive Symptoms of Adults With Fragile X SyndromeSuspendedNCT05832255Phase 21 country
- A Study of OV101 in Individuals With Fragile X SyndromeCompletedNCT03697161Phase 21 country