5 completed. 2 Phase 2, 1 Phase 1, 1 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 2 | Completed 2 |
| Phase 1 | 1 | Completed 1 |
| Phase 3 | 1 | Completed 1 |
Follow Molybdenum cofactor deficiency: new trials by email Literature Review of Molybdenum cofactor deficiency trials Research Report on Molybdenum cofactor 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 |
|---|---|---|---|---|---|---|
| Study of ORGN001 (Formerly ALXN1101) in Neonates, Infants and Children With Molybdenum Cofactor Deficiency (MOCD) Type A ORGN001 (formerly ALXN1101) - Molybdenum Cofactor Deficiency, Type A | Phase 2 / Phase 3 | Completed | Origin Biosciences | 5 | 2016-05-01 | 2023-10-17 |
| Safety & Efficacy Study of ORGN001 (Formerly ALXN1101) in Pediatric Patients With MoCD Type A Currently Treated With rcPMP ORGN001 (formerly ALXN1101) - Molybdenum Cofactor Deficiency, Type A | Phase 2 | Completed | Origin Biosciences | 8 | 2014-04-01 | 2023-10-17 |
| A Natural History Study of Molybdenum Cofactor and Isolated Sulfite Oxidase Deficiencies Molybdenum Cofactor Deficiency, Isolated Sulfite Oxidase Deficiency | Completed | Origin Biosciences | 65 | 2013-08-01 | 2019-03-19 | |
| Phase 1 Single Dose Study of ALXN1101 in Healthy Volunteers ALXN1101, Placebo - Molybdenum Cofactor Deficiency (MoCD), Rare Autosomal Recessive Disorder | Phase 1 | Completed | Origin Biosciences | 24 | 2013-06-01 | 2019-03-19 |
| A Retrospective, Observational, Noninterventional Data Collection Study for Patients With Molybdenum Cofactor Deficiency Who Have Been Previously Treated With Cyclic Pyranopterin Monophosphate (cPMP) Molybdenum Cofactor Deficiency | Completed | Origin Biosciences | 15 | 2012-11-01 | 2019-03-19 |
Molybdenum Cofactor Deficiency, Complementation Group A (2)
nulibry (1)
United States (5) · United Kingdom (4) · Netherlands (3) · Turkey (Türkiye) (3) · Australia (2) · Germany (2) · Israel (2) · Spain (2) · Tunisia (2) · Canada (1) · Italy (1) · Japan (1) · Malaysia (1) · Norway (1) · Poland (1)
Liza Squires, M.D. (1) · Ronald Goldwater, MD (1)