2 active not recruiting, 1 completed, 1 recruiting, 1 terminated. 2 Phase 2, 1 Phase 1, 1 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 2 | Recruiting 1, Terminated 1 |
| Phase 1 | 1 | Active not recruiting 1 |
| Phase 3 | 1 | Active not recruiting 1 |
Follow OTC Deficiency: new trials by email Literature Review of OTC Deficiency trials Research Report on OTC Deficiency
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 Study to Evaluate the Pharmacodynamics and Safety of ARCT-810 in Participants With OTCD ARCT-810 - OTC Deficiency, Ornithine Transcarbamylase Deficiency | Phase 2 | Recruiting | Arcturus Therapeutics, Inc. | 9 | 2024-11-04 | 2025-03-17 |
| Study of CMP-CPS-001 in Healthy Volunteers and Participants With Abnormal Heterozygous OTC Genotype CMP-CPS-001, Placebo - Healthy Volunteers, OTC Deficiency | Phase 1 | Active not recruiting | CAMP4 Therapeutics Corporation | 120 | 2024-02-05 | 2026-04-22 |
| Clinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC) Deficiency DTX301, Placebo, Oral Corticosteroids - OTC Deficiency | Phase 3 | Active not recruiting | Ultragenyx Pharmaceutical Inc | 37 | 2022-10-18 | 2026-09-09 |
| Study for Adolescents and Adults With Ornithine Transcarbamylase Deficiency to Evaluate Safety and Tolerability of ARCT-810 ARCT-810, Placebo - Ornithine Transcarbamylase Deficiency, OTC Deficiency | Phase 2 | Terminated | Arcturus Therapeutics, Inc. | 8 | 2022-10-17 | 2025-09-26 |
| Patient and Observer Reported Outcome Measurements in Inborn Errors of Metabolism Survey - Inborn Errors of Metabolism, Urea Cycle Disorder | Completed | University Children's Hospital, Zurich | 69 | 2019-08-01 | 2020-11-04 |
Ornithine Carbamoyltransferase Deficiency Disease (5) · Urea Cycle Disorders, Inborn (2) · Amino Acid Metabolism, Inborn Errors (1) · Maple Syrup Urine Disease (1) · Metabolism, Inborn Errors (1) · Methylmalonic acidemia (1) · Phenylketonurias (1)
Brain Diseases (5) · Brain Diseases, Metabolic (5) · Brain Diseases, Metabolic, Inborn (5) · Central Nervous System Diseases (5) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (5) · Genetic Diseases, Inborn (5) · Genetic Diseases, X-Linked (5) · Metabolic Diseases (5) · Nervous System Diseases (5) · Nutritional and Metabolic Diseases (5) · Amino Acid Metabolism, Inborn Errors (4) · Metabolism, Inborn Errors (4) · Urea Cycle Disorders, Inborn (3)
Adrenal Cortex Hormones (1) · Prednisolone (1) · Sodium Acetate (1) · Surveys and Questionnaires (1)
Arcturus Therapeutics, Inc. (2) · CAMP4 Therapeutics Corporation (1) · Ultragenyx Pharmaceutical Inc (1) · University Children's Hospital, Zurich (1)
France (2) · Netherlands (2) · Spain (2) · United States (2) · Argentina (1) · Australia (1) · Belgium (1) · Brazil (1) · Canada (1) · Germany (1) · Italy (1) · Japan (1) · Portugal (1) · Sweden (1) · Switzerland (1)
Gloria Wong, MD (1) · Margreet Wagenmakers (1) · Martina Huemer (1)