3 recruiting, 2 terminated, 1 completed. 2 Phase 3, 1 Not applicable, 1 Phase 2, 1 Phase 4.
| Phase | Trials | Status |
|---|---|---|
| Phase 3 | 2 | Completed 1, Terminated 1 |
| Not applicable | 1 | Recruiting 1 |
| Phase 2 | 1 | Terminated 1 |
| Phase 4 | 1 | Recruiting 1 |
Follow Alstrom Syndrome: new trials by email Literature Review of Alstrom Syndrome trials Research Report on Alstrom 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 |
|---|---|---|---|---|---|---|
| Real-World Effects of MC4R Agonist Therapy in BBS and Severe Genetic Obesity Setmelanotide - Bardet Biedl Syndrome (BBS), Bardet Biedl Syndrome | Phase 4 | Recruiting | Tom Hühne | 200 | 2023-01-01 | 2026-06-29 |
| COhort for Bardet-Bield Syndrome and Alström Syndrome for Translational Research Monocentric Interventional Study Skin biopsy - Bardet-Biedl Syndrome, Alström Syndrome | Not applicable | Recruiting | University Hospital, Strasbourg, France | 350 | 2020-06-16 | 2021-09-10 |
| Setmelanotide (RM-493), Melanocortin-4 Receptor (MC4R) Agonist, in Bardet-Biedl Syndrome (BBS) and Alström Syndrome (AS) Participants With Moderate to Severe Obesity Setmelanotide, Placebo - Bardet Biedl Syndrome (BBS), Alström Syndrome (AS) | Phase 3 | Completed | Rhythm Pharmaceuticals, Inc. | 52 | 2018-11-23 | 2023-12-01 |
| Open-Label Rollover Study of PBI 4050 in Subjects With Alström Syndrome PBI-4050 - Alström Syndrome | Phase 2 / Phase 3 | Terminated | Liminal BioSciences Ltd. | 10 | 2017-10-09 | 2020-11-03 |
| Clinical Study of a Single Ciliopathy: Alström Syndrome Alström Syndrome (ALMS) | Terminated | University Hospital, Strasbourg, France | 75 | 2014-04-01 | 2019-06-21 | |
| Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford Rare Disorders, Undiagnosed Disorders | Recruiting | Sanford Health | 20000 | 2010-07-01 | 2025-05-29 |
Bardet-Biedl Syndrome (3) · Retinitis Pigmentosa (2) · 3-Methylglutaconic Aciduria, Type V (1) · Achalasia Addisonianism Alacrimia syndrome (1) · Acrodysostosis (1) · Addison Disease (1) · Alagille Syndrome (1) · Amnesia, Transient Global (1) · Aniridia (1) · Aniridia and Absent Patella (1) · Aniridia, Partial, with Unilateral Renal Agenesis and Psychomotor Retardation (1) · Ataxia (1) · Ataxia Neuropathy Spectrum (1) · Ataxia Telangiectasia (1)
Abnormalities, Multiple (6) · Congenital Abnormalities (6) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (6) · Eye Diseases (6) · Eye Diseases, Hereditary (6) · Genetic Diseases, Inborn (6) · Hereditary Sensory and Motor Neuropathy (6) · Heredodegenerative Disorders, Nervous System (6) · Nervous System Diseases (6) · Nervous System Malformations (6) · Neurodegenerative Diseases (6) · Neuromuscular Diseases (6) · Peripheral Nervous System Diseases (6) · Polyneuropathies (6) · Ciliopathies (5)
setmelanotide (2) · setogepram (1)
University Hospital, Strasbourg, France (2) · Liminal BioSciences Ltd. (1) · Rhythm Pharmaceuticals, Inc. (1) · Sanford Health (1) · Tom Hühne (1)
France (3) · United Kingdom (2) · United States (2) · Australia (1) · Canada (1) · Germany (1) · Puerto Rico (1) · Spain (1)
David Meeker, MD (1) · Metin Cetiner, PD Dr. med. (1)
PMID 34013094, PMID 36356613, PMID 36647077, PMID 36961653, PMID 40903014, PMID 41703984, PMID 42821228
2018 (1)