183 completed, 53 recruiting, 34 unknown, 33 terminated. 128 Phase 2, 62 Phase 3, 58 Phase 1, 52 Not applicable, 9 Phase 4.
Follow X-Linked Intellectual Disability: new trials by email Literature Review of X-Linked Intellectual Disability trials Research Report on X-Linked Intellectual Disability
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| Efficacy and Safety of NTI164 in Children and Young Adults With Rett Syndrome NTI164, Placebo - RETT Syndrome With Proven MECP2 Mutation, Rett Syndrome | Phase 2 / Phase 3 | Not yet recruiting | Fenix Innovation Group | 40 | 2027-07-01 | 2026-09-09 |
| IT and IV Lentiviral Gene Therapy for X-ALD Intrathecal and intravenous LV gene therapy - X-linked Adrenoleukodystrophy | Not applicable | Not yet recruiting | Shenzhen Geno-Immune Medical Institute | 30 | 2027-06-01 | 2026-09-01 |
| A Study to Investigate the Effects and Safety of Codabakalner for the Treatment of FXS in Male Participants and Adult Participants With ADS Codabakalner, Placebo - Fragile X Syndrome (FXS), Autism Spectrum Disorder (ASD) | Phase 2 / Phase 3 | Not yet recruiting | Spinogenix | 120 | 2026-12-01 | 2026-10-09 |
| To Investigate the Efficacy of Treatment With Oral NA-921 (Bionetide) Versus Placebo in Females With Rett Syndrome Bionetide, Placebo - Rett Syndrome | Phase 3 | Recruiting | Biomed Industries, Inc. | 210 | 2026-09-30 | 2026-03-27 |
| A Pilot Study of an Antioxidant Cocktail vs. Placebo in the Treatment of Children and Adolescents With Rett Syndrome Rett-T, Placebo - Rett Syndrome | Phase 2 | Recruiting | Holland Bloorview Kids Rehabilitation Hospital | 35 | 2026-08-24 | 2026-09-02 |
| NORTHERA (DROXIDOPA) for Dysautonomia in Pediatric Survivors of Menkes Disease Droxidopa Oral Product, Placebo Control - Menkes Disease | Phase 1 / Phase 2 | Recruiting | Stephen G. Kaler | 6 | 2026-08-10 | 2026-10-05 |
| Adapting a Psychosocial Support Intervention for the Rett Syndrome Community Those who have patients prescribed Daybue, Caregiver Speaks + Daybue - Rett Syndrome, Family Caregivers | Not applicable | Not yet recruiting | Colorado State University | 60 | 2026-08-01 | 2026-07-28 |
| The Neurocognitive Bases of Trust in Intellectual Disability Confidence adjustment assessment, clinical assessment, Cognitive assessment - Down Syndrome (Trisomy 21), Fragile X Syndrome (FXS) | Recruiting | Hospices Civils de Lyon | 112 | 2026-07-17 | 2026-08-05 | |
| A Phase 3 Study of Fenfluramine Hydrochloride in Rett Syndrome fenfluramine hydrochloride, Placebo - Rett Syndrome | Phase 3 | Recruiting | UCB BIOSCIENCES, Inc. | 200 | 2026-07-14 | 2026-10-15 |
| Safety, Tolerability, and Preliminary Effectiveness of CTH120 in Fragile X Syndrome CTH120, CTH120 placebo hard capsules - Fragile X Syndrome (FXS), Fragile X Syndrome | Phase 2 | Recruiting | Connecta Therapeutics, S.L. | 30 | 2026-05-21 | 2026-07-17 |
| Safety and Preliminary Efficacy of TSHA-102 Gene Therapy in Pediatric Females Aged >2 to <4 Years With Rett Syndrome TSHA-102 - Rett Syndrome | Phase 3 | Active not recruiting | Taysha Gene Therapies, Inc. | 4 | 2026-05-08 | 2026-08-19 |
| Optical Imaging in X-linked Disorders. Clinical assessment, Parental questionnaires, Cognitive assessment - Fragile X Syndrome (FXS), Creatine Transporter Deficiency | Not applicable | Recruiting | Hospices Civils de Lyon | 118 | 2026-03-30 | 2026-05-15 |
| Rett REVOLUTION Trial: An Exploratory Evaluation of the Safety and Efficacy of Vorinostat in Rett Syndrome Vorinostat (SAHA), Placebo - Rett Syndrome | Phase 1 | Recruiting | Unravel Biosciences, Inc. | 15 | 2026-03-15 | 2026-09-17 |
| Intensive Multimodal Neurorehabilitation Targeting Neuroplasticity in Pediatric Neurodevelopmental and Chromosomal Disorders Neurodevelopmental Disorders, Neurodevelopmental Disorders (NDD) | Recruiting | Healing Hope International | 100 | 2026-03-01 | 2026-03-25 | |
| Inhibition of Aggressive Behavior in Participants With Fragile X Syndrome SRX246, Placebo - Aggressive Behavior in Fragile X Syndome | Phase 2 | Not yet recruiting | Azevan Pharmaceuticals | 80 | 2026-03-01 | 2026-01-09 |
| RGX-121-3102 Gene Therapy in Participants With MPS II (Hunter Syndrome) RGX-121-3102 - MPS II, Hunter Syndrome (MPS II) | Phase 3 | Active not recruiting | REGENXBIO Inc. | 2 | 2025-11-25 | 2026-06-04 |
| Study of MRM-3379 in Male Participants With Fragile X Syndrome (BLOOM) Low dose of MRM-3379, Middle Dose of MRM-3379, High dose of MRM-3379 - Fragile X Syndrome | Phase 2 | Recruiting | Mirum Pharmaceuticals, Inc. | 60 | 2025-11-22 | 2026-09-29 |
| Lamivudine as a Novel Clinical Effort for Rett Syndrome Lamivudine - Rett Syndrome | Phase 2 | Enrolling by invitation | Maria Denise Fernandes Carvalho de Andrade | 10 | 2025-11-05 | 2026-06-10 |
| Efficacy and Safety of the Treatment of Pyruvate Dehydrogenase Deficiency Patients With Glycerol Phenylbutyrate (RAVICTI) Glycerol Phenylbutyrate 1100 MG/ML [Ravicti] - Pyruvate Dehydrogenase Complex Deficiency Disease | Phase 2 | Recruiting | Assistance Publique - Hôpitaux de Paris | 15 | 2025-10-01 | 2026-03-31 |
| Effect of CANnabidiol on Anxiety and GABAergic Function in Individuals With Fragile-X Syndrome CBD Oral Solution, Placebo - Fragile X Syndrome | Phase 2 | Recruiting | Université de Sherbrooke | 40 | 2025-09-01 | 2026-08-28 |
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Fragile X Syndrome (113) · Rett Syndrome (95) · Mucopolysaccharidosis II (77) · Adrenoleukodystrophy (62) · Sudden Infant Death (34) · Mucopolysaccharidosis I (24) · Intellectual Disability (21) · Leukodystrophy, Metachromatic (21) · Autism Spectrum Disorder (19) · Autistic Disorder (18) · Mucopolysaccharidoses (18) · Genetic Diseases, Inborn (17) · Genetic Diseases, X-Linked (17) · Leukodystrophy, Globoid Cell (17) · Lysosomal Storage Diseases (17)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (368) · Heredodegenerative Disorders, Nervous System (360) · Nervous System Diseases (359) · Neurologic Manifestations (358) · Neurobehavioral Manifestations (357) · Genetic Diseases, Inborn (353) · Genetic Diseases, X-Linked (353) · Intellectual Disability (348) · Nutritional and Metabolic Diseases (172) · Metabolic Diseases (158) · Metabolism, Inborn Errors (149) · Congenital Abnormalities (126) · Chromosome Disorders (114) · Sex Chromosome Disorders (112)
idursulfase (14) · trofinetide (8) · mavoglurant (6) · Alemtuzumab (5) · Metformin (5) · arbaclofen placarbil (5) · BPN14770 (4) · Dextromethorphan (4) · Donepezil (4) · Injections (4) · Lovastatin (4) · Melphalan (4) · Minocycline (4) · Stem Cell Transplantation (4) · gaboxadol (4)
Shire (15) · Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) (9) · Seaside Therapeutics, Inc. (9) · Children's Hospital Medical Center, Cincinnati (8) · Stanford University (8) · JCR Pharmaceuticals Co., Ltd. (7) · Masonic Cancer Center, University of Minnesota (7) · Novartis Pharmaceuticals (6) · REGENXBIO Inc. (6) · University of California, Davis (6) · Assistance Publique - Hôpitaux de Paris (5) · Genetix Biotherapeutics Inc. (5) · Green Cross Corporation (5) · Hugo W. Moser Research Institute at Kennedy Krieger, Inc. (5) · Rett Syndrome Research Trust (5)
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