2 completed, 2 unknown, 1 active not recruiting. 5 Phase 2, 1 Phase 1.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 5 | Completed 2, Unknown 2, Active not recruiting 1 |
| Phase 1 | 1 | Completed 1 |
Follow Wolfram Syndrome: new trials by email Literature Review of Wolfram Syndrome trials Research Report on Wolfram Syndrome
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 |
|---|---|---|---|---|---|---|
| Tirzepatide Monotherapy in Patients With Wolfram Syndrome Type 1 Tirzepatide - Wolfram Syndrome | Phase 2 | Unknown | Ospedale San Raffaele | 10 | 2024-01-01 | 2024-02-22 |
| AMX0035 in Adult Patients With Wolfram Syndrome AMX0035 - Wolfram Syndrome | Phase 2 | Active not recruiting | Amylyx Pharmaceuticals Inc. | 12 | 2023-03-03 | 2026-04-14 |
| Efficacy Study of Daily Administration of VPA in Patients Affected by Wolfram Syndrome Depakine - Wolfram Syndrome | Phase 2 | Unknown | Centre d'Etude des Cellules Souches | 23 | 2021-11-26 | 2023-11-28 |
| Efficacy and Safety Trial of Sodium Valproate, in Paediatric and Adult Patients With Wolfram Syndrome Sodium Valproate 200Mg E/C Tablet, Sodium Valproate matched placebo - Wolfram Syndrome | Phase 2 | Completed | University of Birmingham | 63 | 2018-12-28 | 2026-05-05 |
| A Clinical Trial of Dantrolene Sodium in Pediatric and Adult Patients With Wolfram Syndrome dantrolene sodium - Wolfram Syndrome, Diabetes Mellitus | Phase 1 / Phase 2 | Completed | Washington University School of Medicine | 21 | 2017-01-01 | 2024-03-13 |
Diabetes Mellitus (2) · Ataxia (1) · Optic Atrophy (1)
Abnormalities, Multiple (5) · Blindness (5) · Congenital Abnormalities (5) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (5) · Cranial Nerve Diseases (5) · Deaf-Blind Disorders (5) · Deafness (5) · Diabetes Insipidus (5) · Diabetes Mellitus, Type 1 (5) · Ear Diseases (5) · Endocrine System Diseases (5) · Eye Diseases (5) · Eye Diseases, Hereditary (5) · Female Urogenital Diseases (5) · Female Urogenital Diseases and Pregnancy Complications (5)
Valproic Acid (2) · 4-phenylbutyric acid (1) · Dantrolene (1) · Tirzepatide (1) · sodium phenylbutyrate and taurursodiol (1)
Amylyx Pharmaceuticals Inc. (1) · Centre d'Etude des Cellules Souches (1) · Ospedale San Raffaele (1) · University of Birmingham (1) · Washington University School of Medicine (1)
France (2) · Spain (2) · United States (2) · Italy (1) · Poland (1) · United Kingdom (1)
Camille Bedrosian, MD (1) · Fumihiko Urano, MD (1) · Lorenzo Piemonti, MD (1) · Timothy Barrett, PhD, MB, BS (1)
R21DK113487 (1)