2 completed, 1 active not recruiting. 2 Phase 2, 1 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 2 | Completed 2 |
| Phase 3 | 1 | Completed 1 |
Follow Molybdenum Cofactor Deficiency, Type A: new trials by email Literature Review of Molybdenum Cofactor Deficiency, Type A trials Research Report on Molybdenum Cofactor Deficiency, Type A
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 |
|---|---|---|---|---|---|---|
| Early Check: Expanded Screening in Newborns Confirmatory Testing - Spinal Muscular Atrophy, Fragile X Syndrome | Active not recruiting | RTI International | 30000 | 2018-10-15 | 2026-07-01 | |
| 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 |
Molybdenum Cofactor Deficiency, Complementation Group A (3) · Molybdenum cofactor deficiency (2) · 3-Hydroxy-3-Methylglutaryl-CoA Lyase Deficiency (1) · 3-Hydroxyacyl-CoA Dehydrogenase Deficiency (1) · 3-methylcrotonyl CoA carboxylase 1 deficiency (1) · 3-methylcrotonyl CoA carboxylase 2 deficiency (1) · Acidemia, isovaleric (1) · Acrodermatitis enteropathica (1) · Adenine phosphoribosyltransferase deficiency (1) · Adrenal hyperplasia, congenital, type 5 (1) · Adrenocorticotropic hormone deficiency (1) · Amaurosis congenita of Leber, type 2 (1) · Anemia, Sickle Cell (1) · Angelman Syndrome (1) · Aortic Stenosis, Supravalvular (1)
Abnormalities, Multiple (1) · Albinism (1) · Albinism, Oculocutaneous (1) · Amino Acid Metabolism, Inborn Errors (1) · Anemia (1) · Anemia, Hemolytic (1) · Anemia, Hemolytic, Congenital (1) · Aortic Valve Disease (1) · Aortic Valve Stenosis (1) · Arrhythmias, Cardiac (1) · Avitaminosis (1) · Basal Ganglia Diseases (1) · Blood Coagulation Disorders (1) · Blood Coagulation Disorders, Inherited (1) · Blood Platelet Disorders (1)
Origin Biosciences (2) · RTI International (1)
United States (3) · United Kingdom (2) · Australia (1) · Israel (1) · Netherlands (1) · Norway (1) · Spain (1) · Tunisia (1) · Turkey (Türkiye) (1)
Curt Scharfe, MD, PhD (1) · Liza Squires, M.D. (1)