1 active not recruiting, 1 completed, 1 recruiting. 1 Phase 1, 1 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 1 | Recruiting 1 |
| Phase 2 | 1 | Recruiting 1 |
Follow Usher syndrome, type 1B: new trials by email Literature Review of Usher syndrome, type 1B trials Research Report on Usher syndrome, type 1B
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 |
|---|---|---|---|---|---|---|
| Study of Subretinally Injected AAVB-081 in Patients With Usher Syndrome Type IB (USH1B) Retinitis Pigmentosa AAVB-081 - Usher Syndrome, Type 1B | Phase 1 / Phase 2 | Recruiting | AAVantgarde Bio Srl | 15 | 2024-07-02 | 2025-05-02 |
| Early Check: Expanded Screening in Newborns Confirmatory Testing - Spinal Muscular Atrophy, Fragile X Syndrome | Active not recruiting | RTI International | 30000 | 2018-10-15 | 2026-07-01 | |
| Natural History Study in Subjects With Usher Syndrome Usher Syndrome, Type 1B | Completed | Fondazione Telethon | 56 | 2018-06-01 | 2024-12-09 |
3-Hydroxy-3-Methylglutaryl-CoA Lyase Deficiency (1) · 3-Hydroxyacyl-CoA Dehydrogenase Deficiency (1) · 3-methylcrotonyl CoA carboxylase 1 deficiency (1) · 3-methylcrotonyl CoA carboxylase 2 deficiency (1) · Acidemia, isovaleric (1) · Acrodermatitis enteropathica (1) · Adenine phosphoribosyltransferase deficiency (1) · Adrenal hyperplasia, congenital, type 5 (1) · Adrenocorticotropic hormone deficiency (1) · Amaurosis congenita of Leber, type 2 (1) · Anemia, Sickle Cell (1) · Angelman Syndrome (1) · Aortic Stenosis, Supravalvular (1) · Arginine-Glycine Amidinotransferase Deficiency (1)
Abnormalities, Multiple (2) · Congenital Abnormalities (2) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (2) · Eye Diseases (2) · Eye Diseases, Hereditary (2) · Genetic Diseases, Inborn (2) · Nervous System Diseases (2) · Neurologic Manifestations (2) · Pathological Conditions, Signs and Symptoms (2) · Retinal Diseases (2) · Albinism (1) · Albinism, Oculocutaneous (1) · Amino Acid Metabolism, Inborn Errors (1) · Anemia (1) · Anemia, Hemolytic (1)
AAVantgarde Bio Srl (1) · Fondazione Telethon (1) · RTI International (1)
Italy (2) · Netherlands (1) · Spain (1) · United Kingdom (1) · United States (1)
Carmen Ayuso (1) · Curt Scharfe, MD, PhD (1) · Ingeborgh van den Born (1)