| Recruiting | Study of MRM-3379 in Male Participants With Fragile X Syndrome (BLOOM) NCT07209462 | Mirum Pharmaceuticals, Inc. | Phase 2 |
| Not Yet Recruiting | Effect of CANnabidiol on Anxiety and GABAergic Function in Individuals with Fragile-X Syndrome NCT06261502 | Université de Sherbrooke | Phase 2 |
| Not Yet Recruiting | Effect of CBD on the Brain NCT06261450 | Université de Sherbrooke | Phase 2 |
| Recruiting | Physical Activity and Community EmPOWERment Project NCT06740162 | University of North Carolina, Chapel Hill | N/A |
| Recruiting | Development of Non-Invasive Prenatal Diagnosis for Single Gene Disorders NCT06147414 | Assistance Publique - Hôpitaux de Paris | — |
| Recruiting | Sertraline vs. Placebo in the Treatment of Anxiety in Children and AdoLescents With NeurodevelopMental Disorde NCT06081348 | Holland Bloorview Kids Rehabilitation Hospital | Phase 2 |
| Completed | Study of SPG601 in Adult Men With Fragile X Syndrome NCT06413537 | Spinogenix | Phase 2 |
| Completed | Placebo-Controlled, Single-Dose Challenge Study of Gaboxadol in Adult Males With Fragile X Syndrome (FXS) NCT06334419 | Craig Erickson | Phase 2 |
| Recruiting | Probiotic Intervention for Microbiome Modifications and Clinical Improvements in Fragile X Syndrome NCT06279858 | Specila hospital for cerebral palsy and developmental neurology | N/A |
| Recruiting | Speech-in-noise Perception in Autism and Fragile X NCT06088589 | Children's Hospital Medical Center, Cincinnati | N/A |
| Recruiting | Web Intervention for Parents of Youth With Genetic Syndromes (WINGS) NCT06139172 | Rush University Medical Center | N/A |
| Completed | CTH120 First-in-Human Study: Single and Multiple Ascending Doses and Potential Food Interaction NCT06480968 | Connecta Therapeutics, S.L. | Phase 1 |
| Unknown | Optical Imaging as a Tool for Monitoring Brain Function in Fragile X Syndrome NCT06293027 | IRCCS Fondazione Stella Maris | — |
| Recruiting | Alpha Auditory Entrainment for Cognitive Enhancement and Sensory Hypersensitivity in Youth With Developmental NCT06227780 | Children's Hospital Medical Center, Cincinnati | N/A |
| Suspended | An Investigation of Psilocybin on Behavioural and Cognitive Symptoms of Adults With Fragile X Syndrome NCT05832255 | Nova Mentis Life Science Corp | Phase 2 |
| Enrolling By Invitation | Sensitivity of the NIH Toolbox to Stimulant Treatment in Intellectual Disabilities NCT05301361 | University of California, Davis | Phase 1 |
| Completed | A Study of BPN14770 in Male Adults (Aged 18 to 45) With Fragile X Syndrome NCT05358886 | Tetra Discovery Partners | Phase 3 |
| Active Not Recruiting | An Open-Label Extension Study of BPN14770 in Subjects With Fragile X Syndrome NCT05367960 | Tetra Discovery Partners | Phase 3 |
| Recruiting | Tracking Early Emergence of Sound Perception Impairments in FXS With Multimodal fNIRS/EEG- Infant NCT06560242 | Children's Hospital Medical Center, Cincinnati | N/A |
| Recruiting | Tracking Early Emergence of Sound Perception Impairments in FXS With Multimodal fNIRS/EEG-Preschool Age NCT05957549 | Children's Hospital Medical Center, Cincinnati | N/A |
| Active Not Recruiting | Evaluating the Neurophysiologic and Clinical Effects of Single Dose Drug Challenge NCT05418049 | Children's Hospital Medical Center, Cincinnati | Phase 2 |
| Withdrawn | Investigation of Sulindac (HLX-0201) and Gaboxadol (HLX-0206) in Male Fragile X Syndrome Patients Aged 13-40 NCT04823052 | Healx Limited | Phase 2 |
| Completed | A Randomized Study of BPN14770 in Male Adolescents (Aged 9 to < 18 Years) With Fragile X Syndrome NCT05163808 | Tetra Discovery Partners | Phase 2 / Phase 3 |
| Recruiting | Metformin in Children With Fragile X Syndrome NCT05120505 | Children's Hospital of Fudan University | Phase 4 |
| Completed | ERG/5-HTP in Fragile X Syndrome (FXS) NCT05030129 | Elizabeth Berry-Kravis | Phase 2 |
| Completed | Clinical Study of Cannabidiol in Children, Adolescents, and Young Adults With Fragile X Syndrome NCT04977986 | Zynerba Pharmaceuticals, Inc. | Phase 3 |
| Unknown | Validation of Optical Genome Mapping for the Identification of Constitutional Genomic Variants in a Postnatal NCT05295277 | Bionano Genomics | — |
| Unknown | NIPD on cffDNA for Triplet Repeat Diseases NCT04698551 | University Hospital, Montpellier | — |
| Unknown | Metformin in Patients With Fragile X NCT04141163 | Rowan University | Phase 1 / Phase 2 |
| Completed | A Trial of Metformin in Individuals With Fragile X Syndrome (Met) NCT03862950 | University of Alberta | Phase 2 |
| Enrolling By Invitation | Parent and Infant Inter(X)Action Intervention (PIXI) NCT03836300 | RTI International | N/A |
| Enrolling By Invitation | Open Label Extension to Assess the Long-Term Safety and Tolerability of ZYN002 in Children and Adolescents Wit NCT03802799 | Zynerba Pharmaceuticals, Inc. | Phase 2 / Phase 3 |
| Enrolling By Invitation | Early Check: Expanded Screening in Newborns NCT03655223 | RTI International | — |
| Completed | Metformin in Children and Adults With Fragile X Syndrome NCT03722290 | Université de Sherbrooke | Phase 2 |
| Completed | A 2-Period Crossover Study of BPN14770 in Adults Males With Fragile X Syndrome NCT03569631 | Tetra Discovery Partners | Phase 2 |
| Completed | Clinical Study Of caNNabidiol in childrEn and adolesCenTs With Fragile X (CONNECT-FX) NCT03614663 | Zynerba Pharmaceuticals, Inc. | Phase 2 / Phase 3 |
| Completed | A Trial of Metformin in Individuals With Fragile X Syndrome NCT03479476 | University of California, Davis | Phase 2 / Phase 3 |
| Completed | Pediatric Exploratory Research Study of EGCG Use and Safety (PERSEUS) NCT03624556 | Parc de Salut Mar | N/A |
| Completed | An Initial Study of AZD7325 in Adults With Fragile X Syndrome NCT03140813 | Children's Hospital Medical Center, Cincinnati | Phase 1 |
| Completed | AFQ056 for Language Learning in Children With FXS NCT02920892 | Elizabeth Berry-Kravis | Phase 2 |
| Unknown | Cooperative Parent Mediated Therapy in Children With Fragile X Syndrome and Williams Syndrome NCT04610424 | Bambino Gesù Hospital and Research Institute | N/A |
| Completed | Single Dose Pharmacokinetic (PK) Study NCT03109756 | Healx AI | Phase 1 |
| Completed | Treatment of Disruptive Behaviors in Fragile X Syndrome NCT03510156 | Stanford University | N/A |
| Completed | Pediatric Patients With Metabolic or Other Genetic Disorders NCT02769949 | Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | — |
| Completed | Combined Treatment of Minocycline and Lovastatin to Treat Individuals With Fragile X Syndrome NCT02680379 | Université de Sherbrooke | Phase 2 |
| Completed | Combining Lovastatin and a Parent-Implemented Language Intervention for Fragile X Syndrome NCT02642653 | University of California, Davis | Phase 4 |
| Completed | Neurophysiological and Acute Pharmacological Studies in FXS Patients NCT02998151 | Children's Hospital Medical Center, Cincinnati | EARLY_Phase 1 |
| Completed | Effects of Social Gaze Training on Brain and Behavior in Fragile X Syndrome NCT02616796 | Stanford University | Phase 1 / Phase 2 |
| Completed | A 6-week, Study of MG01CI Low Dose and High Dose Compared With Placebo in Adults and Adolescents With Fragile NCT02126995 | Alcobra Ltd. | Phase 2 |
| Completed | A Safety Study of NNZ-2566 in Patients With Fragile X Syndrome NCT01894958 | Neuren Pharmaceuticals Limited | Phase 2 |
| Completed | Study of Acamprosate in Fragile x Syndrome NCT01911455 | Children's Hospital Medical Center, Cincinnati | Phase 1 |
| Completed | "Using Epigallocatechin Gallate (EGCG) and Cognitive Training to Modulate Cognitive Performance in Patients Wi NCT01855971 | Parc de Salut Mar | Phase 2 |
| Terminated | EXPLAIN -FragilE X Registry: An exPlorative Longitudinal Study for chAracterIzation, Treatment Pathways and pa NCT01711606 | Novartis Pharmaceuticals | — |
| Completed | A Study of RO4917523 in Pediatric Patients With Fragile X Syndrome NCT01750957 | Hoffmann-La Roche | Phase 2 |
| Completed | Cognitive Training for Fragile X Syndrome NCT02747394 | University of California, Davis | N/A |
| Completed | Ganaxolone Treatment in Children With Fragile X Syndrome NCT01725152 | Marinus Pharmaceuticals | Phase 2 |
| Unknown | Validation of Saliva in the Esoterix Genetic Laboratories Fragile X Assay NCT01616589 | Esoterix Genetic Laboratories, LLC | — |
| Completed | A Study of RO4917523 in Patients With Fragile X Syndrome NCT01517698 | Hoffmann-La Roche | Phase 2 |
| Completed | Clinical Study to Assess the Pharmacokinetics, Safety and Tolerability of Single and Multiple Oral Doses of AF NCT01482143 | Novartis Pharmaceuticals | Phase 1 |
| Completed | Trial of Sertraline to Treat Children With Fragile X Syndrome NCT01474746 | Randi J. Hagerman, MD | Phase 2 |
| Terminated | Long-term, Safety and Tolerability Study of AFQ056 in Adolescent Patients With Fragile X Syndrome (Open-label) NCT01433354 | Novartis Pharmaceuticals | Phase 2 / Phase 3 |
| Terminated | An Open Label Extension Study in Subjects With Fragile X Syndrome NCT01555333 | Seaside Therapeutics, Inc. | Phase 3 |
| Completed | Brain Connectivity in Neurodevelopmental Disorders in Response to Treatment NCT01364818 | University of North Carolina, Chapel Hill | — |
| Terminated | Long-term, Safety, Tolerability and Efficacy Study of AFQ056 in Adult Patients With Fragile X Syndrome NCT01348087 | Novartis Pharmaceuticals | Phase 2 |
| Completed | Efficacy and Safety Study of STX209 (Arbaclofen) for the Treatment of Social Withdrawal in Children With Fragi NCT01325220 | Seaside Therapeutics, Inc. | Phase 3 |
| Completed | Efficacy and Safety Study of STX209 (Arbaclofen) for Social Withdrawal in Adolescents and Adults With Fragile NCT01282268 | Seaside Therapeutics, Inc. | Phase 3 |
| Suspended | A Study to Assess the Tolerability of a Single Dose of STX107 in Adults With Fragile X Syndrome NCT01325740 | Seaside Therapeutics, Inc. | Phase 2 |
| Completed | Safety and Efficacy of AFQ056 in Adolescent Patients With Fragile X Syndrome NCT01357239 | Novartis Pharmaceuticals | Phase 2 |
| Completed | Safety and Efficacy Study of Antioxidants for the Treatment of the Fragile X Syndrome NCT01329770 | Yolanda de Diego Otero | Phase 2 |
| Completed | Safety and Efficacy of AFQ056 in Adult Patients With Fragile X Syndrome NCT01253629 | Novartis Pharmaceuticals | Phase 2 |
| Terminated | The Natural History of Reproductive and Overall Health in Girls and Women With a Pre-Mutation in the FMR1 Gene NCT01187524 | Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | — |
| Completed | Acamprosate in Youth With Fragile X Syndrome NCT01300923 | Indiana University | Phase 2 |
| Completed | Trial of Minocycline to Treat Children With Fragile X Syndrome NCT01053156 | University of California, Davis | N/A |
| Completed | A Study With RO4917523 in Patients With Fragile X Syndrome NCT01015430 | Hoffmann-La Roche | Phase 2 |
| Terminated | An Open Label Extension Study of STX209 in Subjects With Fragile X Syndrome NCT01013480 | Seaside Therapeutics, Inc. | Phase 2 |
| Completed | Randomized Controlled Study of Donepezil in Fragile X Syndrome NCT01120626 | Stanford University | Phase 2 |
| Completed | A Single-Dose Study in Normal Volunteers to Assess the Safety, Tolerability and Pharmacokinetics of STX107 NCT00965432 | Seaside Therapeutics, Inc. | Phase 1 |
| Completed | Biomarker and DNA Collection in Subjects Participating in Protocol 22003 NCT00892580 | Seaside Therapeutics, Inc. | — |
| Completed | Riluzole in Fragile X Syndrome NCT00895752 | Indiana University | Phase 4 |
| Completed | A PET Brain Imaging Study of mGluR5 in Subjects With Neuropsychiatric Conditions NCT00870974 | Institute for Neurodegenerative Disorders | Phase 1 |
| Completed | Biomarker Testing and DNA Collection in Subjects Participating in Protocol 22001 NCT00823368 | Seaside Therapeutics, Inc. | — |
| Completed | Safety, Tolerability and Efficacy Study of STX209 in Subjects With Fragile X Syndrome NCT00788073 | Seaside Therapeutics, Inc. | Phase 2 |
| Completed | Teaching Math Skills to Individuals With Fragile X Syndrome NCT01204151 | Stanford University | N/A |
| Completed | Efficacy, Safety and Tolerability of AFQ056 in Fragile X Patients NCT00718341 | Novartis | Phase 2 |
| Completed | Open Label Study Investigating Safety and Efficacy of NPL2009 50 mg - 150 mg on Prepulse Inhibition Tests and NCT00637221 | Neuropharm | Phase 1 / Phase 2 |
| Completed | Add-on Pilot Trial of Minocycline to Treat Fragile X Syndrome NCT00858689 | FRAXA Research Foundation | N/A |
| Completed | A Prospective Open-label Study of Aripiprazole in Fragile X Syndrome NCT00420459 | Indiana University School of Medicine | Phase 2 |
| Completed | Double-blind Placebo Controlled Study of Oxytocin in Fragile X Syndrome NCT01254045 | Stanford University | Phase 2 |
| Completed | Protein Synthesis in the Brain of Patients With Fragile X Syndrome NCT00362843 | National Institute of Mental Health (NIMH) | — |
| Completed | An Open-Label Trial of Donepezil in Fragile X Syndrome NCT00220584 | Stanford University | Phase 1 |
| Unknown | Aromatase Activity and Ovarian Growth Factors in African-American Versus Caucasian Women NCT00334971 | Massachusetts General Hospital | N/A |
| Completed | Effects of CX516 on Functioning in Fragile X Syndrome and Autism NCT00054730 | RespireRx | Phase 2 |
| Recruiting | The Psychiatric and Cognitive Phenotypes in Velocardiofacial Syndrome NCT00768820 | The Chaim Sheba Medical Center | Phase 4 |