X-linked chromosomal disorder (DBCOND0008737)
Identifiers
- Synonyms
- Genetic Disease, X-Linked / X-linked Genetic Diseases / Genetic Diseases, X-Linked / Genetic Diseases, X-Linkeds / X-linked hereditary disease (disorder)
Associated Data
- Indicated Drugs and Targets
- Not Available
- Clinical Trials
Identifier Title Drug(s) Purpose Phase Status NCT00001405 Recruitment and Apheresis Collection of Peripheral Blood Hematopoietic Stem Cells, Mononuclear Cells and Granulocytes No drug interventions Not Available Not Available recruiting NCT00100230 DHA and X-Linked Retinitis Pigmentosa prevention 2 completed NCT04157595 Mackenzie's Mission: The Australian Reproductive Carrier Screening Project No drug interventions health_services_research Not Available active_not_recruiting NCT01826487 Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) treatment 3 completed NCT00359411 Genetic Studies of X-linked Lymphoproliferative Disease No drug interventions Not Available Not Available completed NCT03734588 Dose-finding Study of SPK-8016 Gene Therapy in Patients With Hemophilia A to Support Evaluation in Individuals With FVIII Inhibitors No drug interventions treatment 1 / 2 completed NCT03876301 Lead-in Study to Collect Prospective Efficacy and Safety Data of Current FVIII Prophylaxis Replacement Therapy in Adult Hemophilia A Participants No drug interventions Not Available Not Available completed NCT02090959 An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy No drug interventions treatment 3 terminated NCT05432349 Rett Syndrome Registry No drug interventions Not Available Not Available recruiting NCT05126758 A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy No drug interventions treatment 3 recruiting NCT03179631 Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy No drug interventions treatment 3 completed NCT03479476 A Trial of Metformin in Individuals With Fragile X Syndrome No drug interventions treatment 2 / 3 completed NCT03406780 A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy No drug interventions treatment 2 completed NCT03321604 Kidney Information Network for Disease Research and Education No drug interventions Not Available Not Available terminated NCT03862950 A Trial of Metformin in Individuals With Fragile X Syndrome (Met) No drug interventions treatment 2 recruiting