2 completed, 1 recruiting, 1 terminated, 1 unknown. 2 Phase 2, 2 Phase 3, 1 Phase 1.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 2 | Completed 1, Withdrawn 1 |
| Phase 3 | 2 | Terminated 1, Withdrawn 1 |
| Phase 1 | 1 | Completed 1 |
Follow Lesch-Nyhan Syndrome: new trials by email Literature Review of Lesch-Nyhan Syndrome trials Research Report on Lesch-Nyhan 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 |
|---|---|---|---|---|---|---|
| A Natural History Study Seeks to Understand the Clinical, Genomic, Pharmacological, Laboratory, and Dietary Determinates of Pyrimidine and Purine Metabolism Disorders AMPD3, OMIM*102772, AMP Deaminase Deficiency, AK1, OMIM *103000, Adenylate Kinase Deficiency | Recruiting | National Human Genome Research Institute (NHGRI) | 999 | 2023-12-19 | 2026-08-19 | |
| Physiotherapy Assessment Based on the ICF Model in The Lesch-Nyhan Syndrome: Case Report Lesch-Nyhan Syndrome | Unknown | Uskudar University | 1 | 2022-09-26 | 2022-09-21 | |
| Ecopipam Treatment of Self-Injurious Behavior in Subjects With Lesch-Nyhan Disease Ecopipam, Placebo - Lesch-Nyhan Disease, Self-injurious Behavior | Phase 3 | Terminated | Emalex Biosciences Inc. | 9 | 2012-12-01 | 2024-04-22 |
| Safety and Tolerability of the D1 Dopamine Receptor Antagonist Ecopipam in Patients With Lesch-Nyhan Disease Ecopipam - Lesch-Nyhan Disease | Phase 1 | Completed | Psyadon Pharma | 5 | 2010-02-01 | 2013-04-18 |
| Trial of Kuvan in Lesch-Nyhan Disease sapropterin - Behavioral Manifestations of Lesch-Nyhan Disease | Phase 2 / Phase 3 | Withdrawn | University of California, San Diego | 2009-04-01 | 2019-08-02 | |
| Phase II Pilot Study of Aminoimidazole Carboxamide Riboside (AICAR), a Precursor of Purine Synthesis, for Lesch-Nyhan Disease aminomidazole carboxamide riboside - Lesch-Nyhan Syndrome | Phase 2 | Completed | National Center for Research Resources (NCRR) | 2 | 1996-02-01 | 2005-06-24 |
Self-Injurious Behavior (3) · ADA2 protein, human (1) · Adenine phosphoribosyltransferase deficiency (1) · Adenosine monophosphate deaminase deficiency (1) · Adenylosuccinate lyase deficiency (1) · Anemia (1) · Arts syndrome (1) · Beta-Ureidopropionase Deficiency (1) · Charcot-Marie-Tooth Disease (1) · Dihydropyrimidinase Deficiency (1) · Dihydropyrimidine Dehydrogenase Deficiency (1) · Genee-Wiedemann syndrome (1) · Genetic Diseases, Inborn (1) · Hemolysis (1)
Brain Diseases (6) · Brain Diseases, Metabolic (6) · Brain Diseases, Metabolic, Inborn (6) · Central Nervous System Diseases (6) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (6) · Genetic Diseases, X-Linked (6) · Heredodegenerative Disorders, Nervous System (6) · Intellectual Disability (6) · Nervous System Diseases (6) · Neurobehavioral Manifestations (6) · Nutritional and Metabolic Diseases (6) · Purine-Pyrimidine Metabolism, Inborn Errors (6) · X-Linked Intellectual Disability (6) · Genetic Diseases, Inborn (5) · Metabolic Diseases (5)
ecopipam (2) · sapropterin (1)
Emalex Biosciences Inc. (1) · National Center for Research Resources (NCRR) (1) · National Human Genome Research Institute (NHGRI) (1) · Psyadon Pharma (1) · University of California, San Diego (1) · Uskudar University (1)
United States (4) · Spain (1) · Turkey (Türkiye) (1)
Bruce Barshop (1) · H J Jinnah, MD (1) · Hyder A Jinnah, MD (1) · Oleg A Shchelochkov, M.D. (1) · William L Nyhan, MD (1) · William L Nyhan, MD, PhD (1)