3 completed, 2 recruiting, 1 not yet recruiting, 1 unknown. 1 Not applicable, 1 Phase 1, 1 Phase 2, 1 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Not applicable | 1 | Not yet recruiting 1 |
| Phase 1 | 1 | Completed 1 |
| Phase 2 | 1 | Completed 1 |
| Phase 3 | 1 | Recruiting 1 |
Follow X-Linked: new trials by email Literature Review of X-Linked trials Research Report on X-Linked
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 |
|---|---|---|---|---|---|---|
| A Phase 3 Study to Evaluate the Safety and Efficacy of AOC 1044 (Also Referred to as Delpacibart Zotadirsen) in Participants With DMD With Gene Mutations Amenable to Exon 44 Skipping AOC 1044, Placebo - Muscular Dystrophies, Muscular Dystrophies (Duchenne, Becker, Myotonic Dystrophy) | Phase 3 | Recruiting | Avidity Biosciences, Inc. | 70 | 2026-09-01 | 2026-10-13 |
| Morphology in Oral Rare Syndromes & Artificial Intelligence for Clinical Diagnosis intra-oral 3D optical impression - Osteogenesis Imperfecta, Rare Bone Disorders | Not applicable | Not yet recruiting | University Hospital, Bordeaux | 240 | 2026-09-01 | 2026-06-24 |
| Hemophilia A Research Program Hemophilia A, Hemophilia A, Severe | Recruiting | University of Washington | 500 | 2024-07-31 | 2026-02-17 | |
| Better Delineation of DDX3X Related Phenotype and Epigenetic Signature. Epigenetic signatures - Intellectual Developmental Disorder, X-LINKED | Unknown | University Hospital, Montpellier | 10 | 2019-11-01 | 2020-06-18 | |
| Mothers Experiences With X-linked Retinoschisis Compared to Fathers Experiences Retinoschisis, X-Linked | Completed | National Eye Institute (NEI) | 12 | 2017-12-01 | 2019-11-01 | |
| Study of RS1 Ocular Gene Transfer for X-linked Retinoschisis RS1 AAV Vector - Retinoschisis, X-Linked | Phase 1 / Phase 2 | Completed | VegaVect, Inc. | 12 | 2015-02-11 | 2025-02-14 |
| People s Expectations When Enrolling in a Phase I/II RS1 Ocular Gene Transfer Clinical Trial Retinoschisis, X-Linked | Completed | National Eye Institute (NEI) | 13 | 2014-12-13 | 2019-12-09 |
Retinoschisis (3) · Congenital Abnormalities (1) · Familial Hypophosphatemic Rickets (1) · Genetic Diseases, Inborn (1) · Hemophilia A (1) · Hemorrhage (1) · Hemostatic Disorders (1) · Hypophosphatemia (1) · Infant, Newborn, Diseases (1) · Intellectual Disability (1) · Mucopolysaccharidoses (1) · Muscular Diseases (1) · Muscular Disorders, Atrophic (1) · Muscular Dystrophies (1) · Muscular Dystrophy, Duchenne (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (3) · Eye Diseases (3) · Retinal Degeneration (3) · Retinal Diseases (3) · Female Urogenital Diseases and Pregnancy Complications (2) · Genetic Diseases, Inborn (2) · Pathological Conditions, Signs and Symptoms (2) · Urogenital Diseases (2) · Avitaminosis (1) · Blood Coagulation Disorders (1) · Blood Coagulation Disorders, Inherited (1) · Bone Diseases (1) · Bone Diseases, Developmental (1) · Bone Diseases, Metabolic (1) · Calcium Metabolism Disorders (1)
National Eye Institute (NEI) (2) · Avidity Biosciences, Inc. (1) · University Hospital, Bordeaux (1) · University Hospital, Montpellier (1) · University of Washington (1) · VegaVect, Inc. (1)
United States (4) · France (3) · Australia (1) · Belgium (1) · Germany (1) · Italy (1) · Netherlands (1) · Spain (1) · United Kingdom (1)
Amy E Turriff (2) · Grier Page, PhD (1) · Jill M Johnsen, MD (1) · Laryssa A Huryn, M.D. (1) · Shannon Meeks, MD (1)
PMID 30196853, PMID 33601057, PMID 34390869, PMID 36227606
1UG3HL165064-01 (1)