4 completed, 2 not yet recruiting, 2 recruiting, 2 unknown. 5 Phase 2, 1 Not applicable, 1 Phase 1.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 5 | Completed 2, Unknown 2, Active not recruiting 1 |
| Not applicable | 1 | Withdrawn 1 |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 |
|---|---|---|---|---|---|---|
| Does Recessive Optic Atrophy Due to WFS1 Exist? analyse study - Wolfram Syndrome 1, Optic Atrophies, Hereditary | Not yet recruiting | Hôpital Necker-Enfants Malades | 45 | 2026-02-01 | 2026-01-13 | |
| Mechanisms of Fecal Incontinence in Wolfram Syndrome Retrospective case-control analysis - Wolfram Syndrome, Fecal Incontinence | Not yet recruiting | Hôpital Necker-Enfants Malades | 20 | 2025-12-20 | 2025-12-31 | |
| 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 |
| Monogenic Diabetes Misdiagnosed as Type 1 None AHT - Diabetes Mellitus, Type 1, Monogenic Diabetes | Recruiting | McGill University Health Centre/Research Institute of the McGill University Health Centre | 5000 | 2019-09-24 | 2024-12-10 | |
| 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 |
| I-Tracking Neurodegeneration in Early Wolfram Syndrome Wolfram Syndrome | Completed | Washington University School of Medicine | 128 | 2018-08-10 | 2026-09-23 | |
| 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 |
| Tracking Neurodegeneration in Early Wolfram Syndrome Type 1 Diabetes, Diabetes Insipidus | Completed | Washington University School of Medicine | 101 | 2012-04-01 | 2018-07-11 | |
| Wolfram Syndrome and WFS1-related Disorders International Registry and Clinical Study Wolfram Syndrome, Diabetes Mellitus | Recruiting | Washington University School of Medicine | 5000 | 2011-07-01 | 2025-12-23 | |
| GLP Analogs for Diabetes in Wolfram Syndrome Patients Exenatide - Diabetes Mellitus Associated With Genetic Syndrome, Wolfram Syndrome | Not applicable | Withdrawn | Hadassah Medical Organization | 2011-03-01 | 2022-03-29 |
Diabetes Mellitus (6) · Diabetes Insipidus (3) · Optic Atrophy (3) · Ataxia (2) · Deafness (2) · Diabetes Mellitus, Type 1 (2) · Fecal Incontinence (1) · Optic Atrophies, Hereditary (1) · Wolcott-Rallison syndrome (1)
Abnormalities, Multiple (12) · Blindness (12) · Congenital Abnormalities (12) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (12) · Cranial Nerve Diseases (12) · Deaf-Blind Disorders (12) · Ear Diseases (12) · Endocrine System Diseases (12) · Eye Diseases (12) · Eye Diseases, Hereditary (12) · Female Urogenital Diseases (12) · Female Urogenital Diseases and Pregnancy Complications (12) · Genetic Diseases, Inborn (12) · Glucose Metabolism Disorders (12) · Hearing Disorders (12)
Valproic Acid (2) · 4-phenylbutyric acid (1) · Dantrolene (1) · Exenatide (1) · Tirzepatide (1) · sodium phenylbutyrate and taurursodiol (1)
Washington University School of Medicine (4) · Hôpital Necker-Enfants Malades (2) · Amylyx Pharmaceuticals Inc. (1) · Centre d'Etude des Cellules Souches (1) · Hadassah Medical Organization (1) · McGill University Health Centre/Research Institute of the McGill University Health Centre (1) · Ospedale San Raffaele (1) · University of Birmingham (1)
United States (5) · France (3) · Spain (2) · Canada (1) · Italy (1) · Poland (1) · United Kingdom (1)
Camille Bedrosian, MD (1) · Fumihiko Urano, MD (1) · Gil Leibowitz, MD (1) · Lorenzo Piemonti, MD (1) · Tamara G Hershey, PhD (1) · Tamara Hershey, PhD (1) · Timothy Barrett, PhD, MB, BS (1)
PMID 26025012, PMID 26742931, PMID 27459537, PMID 34185708, PMID 40010822
R01DK132090 (1), R01HD070855 (1), R21DK113487 (1)