X-linked disease
X-linked disease
Explore linked clinical trials. Standard reference: MONDO:0000425418 results · Page 1 of 17
Clinical trials
Swipe the table sideways for more columns.
| Trial | Conditions | Status | Phase | Updated | More info | NCT ID |
|---|---|---|---|---|---|---|
| A Study to Investigate the Effects and Safety of Codabakalner for the Treatment of FXS in Male Participants and Adult Participants With ADS | Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Neurobehavioral Manifestations, Neurologic Manifestations, Sex Chromosome Disorders, X-linked disease, autism, autism spectrum disorder, chromosomal disorder, fragile X syndrome, hereditary disease, inherited neurodegenerative disorder, intellectual disability, nervous system disorder, neurodevelopmental disorder, pervasive developmental disorder, psychiatric disorder | Not yet recruiting | Phase 2 / Phase 3 | 2026-10-09 | NCT07439510 | |
| Self-Efficacy Enhancement Among Online Support Group of Primary Caregivers of Children With Neuro Muscular Dystrophy | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Duchenne muscular dystrophy, Muscular Disorders, Atrophic, X-linked disease, central nervous system disorder, hereditary disease, motor neuron disorder, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, spinal cord disorder, spinal muscular atrophy | Active, not recruiting | Not applicable | 2026-10-09 | NCT07356063 | |
| Allogeneic Hematopoietic Stem Cell Transplantation for Chronic Granulomatous Disease (CGD) With an Alemtuzumab, Busulfan and TBI-based Conditioning Regimen Combined With Cytokine (IL-6, +/- IFN-gamma) Antagonists | Chronic Disease, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Disease Attributes, Hemic and Lymphatic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, X-linked disease, chronic granulomatous disease, hematologic disorder, hereditary disease, immune system disorder, inborn error of immunity, leukocyte disorder, phagocyte bactericidal dysfunction | Recruiting | Phase 1 / Phase 2 | 2026-10-09 | NCT05463133 | |
| Phase 1/2 Study of KRRO-121 in Healthy Volunteers and Patients With UCD | Adult-onset citrullinemia type 2, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Nutritional and Metabolic Diseases, X-linked disease, arginase deficiency, argininosuccinic aciduria, brain disorder, central nervous system disorder, hereditary disease, hyperammonemia due to N-acetylglutamate synthase deficiency, inborn disorder of amino acid metabolism, inborn errors of metabolism, metabolic disease, nervous system disorder, ornithine carbamoyltransferase deficiency, urea cycle disorder | Recruiting | Phase 1 / Phase 2 | 2026-10-08 | NCT07773246 | |
| Evaluating VM100 Nutritional Supplement for Improving Quality of Life in Duchenne Muscular Dystrophy Patients | Bacterial Infections and Mycoses, Central Nervous System Bacterial Infections, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Duchenne muscular dystrophy, Fatigue, Infections, Muscular Disorders, Atrophic, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, Treponema infectious disease, X-linked disease, bacterial infectious disease, central nervous system disorder, central nervous system infectious disorder, gram-negative bacterial infections, hereditary disease, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, nervous system disorder, neuromuscular disease, neurosyphilis, spinal cord disorder, spirochaetales infections, syphilis, tabes dorsalis | Recruiting | Phase 2 | 2026-10-08 | NCT07766980 | |
| Registry for Duchenne and Becker Muscular Dystrophy | Becker muscular dystrophy, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Duchenne muscular dystrophy, Muscular Disorders, Atrophic, X-linked disease, hereditary disease, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, nervous system disorder, neuromuscular disease | Recruiting | Not stated | 2026-10-08 | NCT07402122 | |
| ATTUNE: A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intrathecally-Administered ION440 in Participants With Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome (MDS) | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Neurobehavioral Manifestations, Neurologic Manifestations, Rett syndrome, X-linked disease, hereditary disease, inherited neurodegenerative disorder, intellectual disability, nervous system disorder | Recruiting | Phase 1 / Phase 2 | 2026-10-08 | NCT06430385 | |
| A Study to Investigate the Safety and Effectiveness of a Coagulation Factor IX Gene Insertion Therapy (REGV131-LNP1265) in Pediatric, Adolescent and Adult Participants With Hemophilia B | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, X-linked disease, blood coagulation disease, coagulation protein disease, hematologic disorder, hemophilia B, hemorrhagic disease, hereditary disease, inherited blood coagulation disorder | Recruiting | Phase 1 / Phase 2 | 2026-10-08 | NCT06379789 | |
| Agalsidase Beta Long-Term Treatment Outcome for Fabry Disease Patients With IVS4 Mutation in Taiwan | Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Cerebral Small Vessel Diseases, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Fabry disease, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Nutritional and Metabolic Diseases, X-linked disease, brain disorder, cardiovascular disorder, central nervous system disorder, cerebrovascular disorder, hereditary disease, inborn errors of metabolism, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, nervous system disorder, sphingolipidosis, vascular disorder | Active, not recruiting | Not stated | 2026-10-08 | NCT06052800 | |
| Haploidentical Transplant for People With Chronic Granulomatous Disease (CGD) Using Alemtuzumab, Busulfan and TBI With Post-Transplant Cyclophosphamide | Chronic Disease, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Disease Attributes, Hemic and Lymphatic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, X-linked disease, chronic granulomatous disease, graft versus host disease, hematologic disorder, hereditary disease, immune system disorder, inborn error of immunity, leukocyte disorder, phagocyte bactericidal dysfunction | Active, not recruiting | Early phase 1 | 2026-10-08 | NCT03910452 | |
| Adverse Event Data Collection From External Registries on Nonacog Beta Pegol | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, X-linked disease, blood coagulation disease, coagulation protein disease, hematologic disorder, hemophilia B, hemorrhagic disease, hereditary disease, inherited blood coagulation disorder | Enrolling by invitation | Not stated | 2026-10-08 | NCT03690336 | |
| Cancer in Inherited Bone Marrow Failure Syndromes | Bone Marrow Failure Disorders, Congenital Abnormalities, Congenital Bone Marrow Failure Syndromes, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, DNA repair disease, Diamond-Blackfan anemia, Digestive System Diseases, Fanconi anemia, Hemic and Lymphatic Diseases, Infant, Newborn, Diseases, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Nutritional and Metabolic Diseases, Shwachman-Diamond syndrome, Skin Abnormalities, Skin and Connective Tissue Diseases, X-linked disease, anemia, aplastic anemia, bone marrow disorder, dyskeratosis congenita, exocrine pancreatic insufficiency, hematologic disorder, hereditary disease, hereditary skin disorder, inherited aplastic anemia, lipomatosis, metabolic disease, pancreas disorder, pure red-cell aplasia, skin disorder | Recruiting | Not stated | 2026-10-08 | NCT00027274 | |
| Recruitment and Apheresis Collection of Peripheral Blood Hematopoietic Stem Cells, Mononuclear Cells and Granulocytes | Chronic Disease, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Disease Attributes, Granuloma, Hemic and Lymphatic Diseases, Infections, Pathologic Processes, Pathological Conditions, Signs and Symptoms, X-linked disease, chronic granulomatous disease, hematologic disorder, hereditary disease, immune system disorder, inborn error of immunity, leukocyte disorder, lymphatic system disorder, lymphoproliferative syndrome, phagocyte bactericidal dysfunction | Recruiting | Not stated | 2026-10-08 | NCT00001405 | |
| Safety and Effectiveness of Giroctocogene Fitelparvovec or Fidanacogene Elaparvovec in Patients With Hemophilia A or B Respectively | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, Hemorrhage, Pathologic Processes, Pathological Conditions, Signs and Symptoms, X-linked disease, blood coagulation disease, coagulation protein disease, hematologic disorder, hemophilia A, hemophilia B, hemorrhagic disease, hereditary disease, inherited blood coagulation disorder | Recruiting | Phase 3 | 2026-10-07 | NCT05568719 | |
| Multicomponent Pain Self-Management in Adults With Haemophilic Arthropathy | Chronic Pain, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, Kinesiophobia, Neurologic Manifestations, Pain, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, X-linked disease, anxiety disorder, blood coagulation disease, coagulation protein disease, hematologic disorder, hemophilia A, hemophilia B, hemophilic arthropathy, hemorrhagic disease, hereditary disease, inherited blood coagulation disorder, phobic disorder, psychiatric disorder | Not yet recruiting | Not applicable | 2026-10-06 | NCT07860632 | |
| Study to Evaluate the Safety and Effectiveness of ELEVIDYS in Participants With Duchenne Muscular Dystrophy Treated in a Post-Marketing Setting | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Duchenne muscular dystrophy, Muscular Disorders, Atrophic, X-linked disease, hereditary disease, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, nervous system disorder, neuromuscular disease | Recruiting | Phase 4 | 2026-10-06 | NCT07542314 | |
| Phase 3, Open-label, Single-dose Study of CSL222 in Adolescent Male Subjects (≥ 12 to < 18 Years of Age) With Severe or Moderately Severe Hemophilia B | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, X-linked disease, blood coagulation disease, coagulation protein disease, hematologic disorder, hemophilia B, hemorrhagic disease, hereditary disease, inherited blood coagulation disorder | Recruiting | Phase 3 | 2026-10-06 | NCT07080905 | |
| An Open-label Long-term Follow-up Study of SAT-3247 for Participants With Duchenne Muscular Dystrophy Including Those Who Participated in SAT-3247-CL-101 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Duchenne muscular dystrophy, Muscular Disorders, Atrophic, X-linked disease, hereditary disease, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, nervous system disorder, neuromuscular disease | Enrolling by invitation | Phase 2 | 2026-10-06 | NCT06867107 | |
| A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease | Cardiomegaly, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Danon disease, Hypertrophy, Hypertrophy, Left Ventricular, Neurobehavioral Manifestations, Neurologic Manifestations, Nutritional and Metabolic Diseases, Pathological Conditions, Anatomical, Pathological Conditions, Signs and Symptoms, X-linked disease, aortic valve disorder, aortic valve stenosis, cardiomyopathy, cardiovascular disorder, disorder of glycogen metabolism, heart disorder, heart valve disorder, hereditary disease, hypertrophic cardiomyopathy, inborn carbohydrate metabolic disorder, inborn errors of metabolism, intellectual disability, lysosomal storage disease, metabolic disease, nervous system disorder, subvalvular aortic stenosis | Recruiting | Phase 2 | 2026-10-06 | NCT06092034 | |
| Long-term, Extension Study of DS-5141b in Patients With Duchenne Muscular Dystrophy | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Duchenne muscular dystrophy, Muscular Disorders, Atrophic, X-linked disease, hereditary disease, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, nervous system disorder, neuromuscular disease | Active, not recruiting | Phase 2 | 2026-10-06 | NCT04433234 | |
| Lentiviral Gene Transfer for Treatment of Children Older Than Two Years of Age With X-Linked Severe Combined Immunodeficiency (XSCID) | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Infant, Newborn, Diseases, Primary Immunodeficiency Diseases, X-Linked Combined Immunodeficiency Diseases, X-linked disease, hereditary disease, immune system disorder, inborn error of immunity, severe combined immunodeficiency | Recruiting | Phase 1 / Phase 2 | 2026-10-06 | NCT01306019 | |
| A Study to Learn About the Study Medicine (Called Marstacimab) in People With Hemophilia A or B | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, X-linked disease, blood coagulation disease, coagulation protein disease, hematologic disorder, hemophilia A, hemophilia B, hemorrhagic disease, hereditary disease, inherited blood coagulation disorder | Not yet recruiting | Not stated | 2026-10-05 | NCT07859527 | |
| A Study to Learn About the Investigational Drug Rinzimetostat (ORIC-944) in Patients With mCRPC Who Were Previously Treated With Abiraterone Acetate (Himalayas-1) | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Disease Attributes, Genital Diseases, Kennedy disease, Male Urogenital Diseases, Neoplasms by Site, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Recurrence, Urogenital Diseases, Urogenital Neoplasms, X-linked disease, central nervous system disorder, hereditary disease, inherited neurodegenerative disorder, male reproductive organ cancer, male reproductive system disorder, motor neuron disorder, neoplasm, nervous system disorder, neurodegenerative disease, neuromuscular disease, prostate cancer, prostate disorder, spinal cord disorder, spinal muscular atrophy | Recruiting | Phase 3 | 2026-10-05 | NCT07723248 | |
| NORTHERA (DROXIDOPA) for Dysautonomia in Pediatric Survivors of Menkes Disease | Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hypotension, Orthostatic, Menkes disease, Neurobehavioral Manifestations, Neurologic Manifestations, Nutritional and Metabolic Diseases, Skin and Connective Tissue Diseases, X-linked disease, autonomic nervous system disorder, brain disorder, cardiovascular disorder, central nervous system disorder, disorder of pilosebaceous unit, hereditary disease, hypotensive disorder, inborn errors of metabolism, inborn metal metabolism disorder, inherited neurodegenerative disorder, intellectual disability, metabolic disease, nervous system disorder, orthostatic intolerance, primary dysautonomia, skin disorder, vascular disorder | Recruiting | Phase 1 / Phase 2 | 2026-10-05 | NCT07398508 | |
| Natural History and Advanced Genetic Study of Pyruvate Dehydrogenase Complex Deficiencies | Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Neurobehavioral Manifestations, Neurologic Manifestations, Nutritional and Metabolic Diseases, Pyruvate Dehydrogenase Complex Deficiency Disease, Pyruvate Metabolism, Inborn Errors, X-linked disease, brain disorder, central nervous system disorder, hereditary disease, inborn carbohydrate metabolic disorder, inborn errors of metabolism, inborn mitochondrial metabolism disorder, inherited neurodegenerative disorder, intellectual disability, metabolic disease, nervous system disorder | Recruiting | Not stated | 2026-10-05 | NCT03056794 |