33 terminated. 33 Phase 3, 11 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 3 | 33 | Terminated 33 |
| Phase 2 | 11 | Terminated 11 |
Follow Abnormalities, Multiple: new trials by email Literature Review of Abnormalities, Multiple trials Research Report on Abnormalities, Multiple
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 |
|---|---|---|---|---|---|---|
| ARD-101 for Treatment of PWS: The Hunger Elimination or Reduction Objective Trial Placebo, ARD-101 - Hyperphagia, Prader-Willi Syndrome | Phase 3 | Terminated | Aardvark Therapeutics, Inc. | 68 | 2024-12-20 | 2026-06-30 |
| Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome (AS) GTX-102, Sham-LP - Angelman Syndrome | Phase 3 | Terminated | Ultragenyx Pharmaceutical Inc | 129 | 2024-12-03 | 2026-11-03 |
| Long-term Extension of GTX-102 in Angelman Syndrome GTX-102 - Angelman Syndrome | Phase 3 | Terminated | Ultragenyx Pharmaceutical Inc | 66 | 2024-07-31 | 2026-11-03 |
| The Hunger Elimination or Reduction Objective (HERO ) Open -Label Extension (OLE) Trial ARD-101 - Hyperphagia, Prader-Willi Syndrome | Phase 3 | Terminated | Aardvark Therapeutics, Inc. | 19 | 2024-07-23 | 2026-06-30 |
| LIFE-DSR-Biomarker Sub-study of Biomarkers in Down Syndrome Related Alzheimer's Disease (DS-AD) [18F]MK-6240 - Down Syndrome | Phase 3 | Terminated | LuMind IDSC Foundation | 5 | 2023-06-27 | 2025-03-06 |
| A Study to Test Whether Spesolimab Helps People With a Skin Disease Called Netherton Syndrome Spesolimab - solution for infusion, Placebo matching to spesolimab - solution for infusion, Spesolimab - solution for injection - Netherton Syndrome | Phase 2 / Phase 3 | Terminated | Boehringer Ingelheim | 43 | 2023-06-12 | 2026-03-12 |
| A Study to Assess RAD011 (Cannabidiol Oral Solution) for the Treatment of Participants With Prader-Willi Syndrome RAD011, Placebo - Prader-Willi Syndrome | Phase 2 / Phase 3 | Terminated | Radius Pharmaceuticals, Inc. | 4 | 2022-04-13 | 2023-10-19 |
| Roll-over Study to Assess Safety of Lixivaptan in Participants With ADPKD Who Completed Study PA-ADPKD-303 Lixivaptan - Polycystic Kidney Disease, Adult | Phase 3 | Terminated | Palladio Biosciences | 1 | 2022-02-10 | 2023-05-12 |
| Study to Evaluate the Efficacy Safety and Tolerability of QR-421a in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene With Early to Moderate Vision Loss (Celeste) QR-421a, Sham-procedure - Retinitis Pigmentosa, Usher Syndrome Type 2 | Phase 2 / Phase 3 | Terminated | Laboratoires Thea | 5 | 2021-12-15 | 2024-08-07 |
| Study to Evaluate the Efficacy Safety and Tolerability of Ultevursen in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene (Sirius) Ultevursen, Sham-procedure - Retinitis Pigmentosa, Usher Syndrome Type 2 | Phase 2 / Phase 3 | Terminated | Laboratoires Thea | 7 | 2021-12-08 | 2024-07-12 |
| Efficacy and Safety of Lixivaptan in the Treatment of Autosomal Dominant Polycystic Kidney Disease Lixivaptan, Placebo - Autosomal Dominant Polycystic Kidney, ADPKD | Phase 3 | Terminated | Palladio Biosciences | 12 | 2021-10-28 | 2023-02-06 |
| Safety of Lixivaptan in Subjects Previously Treated With Tolvaptan for Autosomal Dominant Polycystic Kidney Disease Lixivaptan - Polycystic Kidney Disease, Adult, ADPKD | Phase 3 | Terminated | Palladio Biosciences | 7 | 2020-09-02 | 2023-04-10 |
| A Pilot Study of the Efficacy and Safety of Dupilumab Versus Placebo in Patients With Netherton Syndrome Dupilumab Prefilled Syringe, Placebo Prefilled Syringe - Netherton Syndrome | Phase 2 / Phase 3 | Terminated | University Hospital, Toulouse | 10 | 2020-07-23 | 2026-09-21 |
| Extension Study of Patidegib Topical Gel, 2% in Subjects With Gorlin Syndrome (Basal Cell Nevus Syndrome) Patidegib Topical Gel, 2% - Basal Cell Nevus Syndrome | Phase 3 | Terminated | Sol-Gel Technologies, Ltd. | 108 | 2020-06-03 | 2024-09-24 |
| A Trial of Bardoxolone Methyl in Patients With ADPKD - FALCON Bardoxolone methyl oral capsule, Placebo oral capsule - Autosomal Dominant Polycystic Kidney, ADPKD | Phase 3 | Terminated | Biogen | 667 | 2019-05-29 | 2025-06-03 |
| Effects of Livoletide (AZP-531) on Food-related Behaviors in Patients With Prader-Willi Syndrome Livoletide, Placebo - Prader-Willi Syndrome, Hyperphagia | Phase 2 / Phase 3 | Terminated | Millendo Therapeutics SAS | 158 | 2019-03-25 | 2021-02-17 |
| An Extended Access Program for Bardoxolone Methyl in Patients With CKD (EAGLE) Bardoxolone methyl - Chronic Kidney Diseases, Alport Syndrome | Phase 3 | Terminated | Biogen | 270 | 2019-03-08 | 2025-06-03 |
| An Open-Label Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of OV101 in Individuals With Angelman Syndrome OV101 - Angelman Syndrome | Phase 3 | Terminated | Healx AI | 141 | 2019-01-31 | 2025-07-18 |
| A Medical Research Study Designed to Determine if Venglustat Can be a Future Treatment for ADPKD Patients Venglustat, Placebo - Polycystic Kidney, Autosomal Dominant | Phase 2 / Phase 3 | Terminated | Genzyme, a Sanofi Company | 478 | 2018-10-04 | 2023-02-03 |
| 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 |
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Polycystic Kidney, Autosomal Dominant (9) · Prader-Willi Syndrome (8) · Hyperphagia (5) · Down Syndrome (4) · Angelman Syndrome (3) · Basal Cell Nevus Syndrome (2) · Carcinoma, Basal Cell (2) · Cognitive Dysfunction (2) · Eye Abnormalities (2) · Eye Diseases (2) · Eye Diseases, Hereditary (2) · Netherton Syndrome (2) · Obesity (2) · Retinal Diseases (2) · Retinitis Pigmentosa (2)
Congenital Abnormalities (33) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (33) · Genetic Diseases, Inborn (33) · Nervous System Diseases (18) · Chromosome Disorders (16) · Neurologic Manifestations (14) · Intellectual Disability (12) · Neurobehavioral Manifestations (12) · Imprinting Disorders (11) · Ciliopathies (10) · Female Urogenital Diseases and Pregnancy Complications (10) · Pathological Conditions, Signs and Symptoms (10) · Urogenital Diseases (10) · Female Urogenital Diseases (9)
lixivaptan (3) · Donepezil (2) · Intravitreal Injections (2) · Sirolimus (2) · bardoxolone methyl (2) · CKD732 (1) · Everolimus (1) · Injections (1) · Losartan (1) · MTOR Inhibitors (1) · Parathyroid Hormone (1) · Rimonabant (1) · Topiramate (1) · cyclic des-acyl ghrelin (6-13) (1) · gaboxadol (1)
Palladio Biosciences (3) · Aardvark Therapeutics, Inc. (2) · Assistance Publique - Hôpitaux de Paris (2) · Biogen (2) · Eisai Inc. (2) · Laboratoires Thea (2) · Ultragenyx Pharmaceutical Inc (2) · A. Manzoni Hospital (1) · Boehringer Ingelheim (1) · Genzyme, a Sanofi Company (1) · Healx AI (1) · Liminal BioSciences Ltd. (1) · LuMind IDSC Foundation (1) · Mario Negri Institute for Pharmacological Research (1) · Millendo Therapeutics SAS (1)
United States (24) · United Kingdom (12) · France (10) · Australia (9) · Spain (8) · Germany (7) · Italy (7) · Canada (6) · Japan (5) · Netherlands (5) · Belgium (4) · Israel (4) · Poland (3) · South Korea (3) · Bulgaria (2)
Thomas McRae, MD (2) · Arlene Chapman, MD (1) · Dennis Kim, MD (1) · Francesco Locatelli, MD (1) · Herma Neyndorff (1) · Jean-Pierre Chanoine, MD (1) · Moris Angulo, MD (1) · Neera K Dahl, MD, PhD (1) · Nelson Kopyt, DO (1)
PMID 39356039, PMID 20619242, PMID 20965292, PMID 25935877, PMID 26052700, PMID 26912555, PMID 28556449, PMID 33395098, PMID 36204243, PMID 36535535
2017 (1), 2018 (1)