Clinical trials: Molybdenum Cofactor Deficiency
Molybdenum Cofactor Deficiency Type A
Study of cPMP (Precusor Z) to Treat Molybdenum Cofactor Deficiency (MoCD) Type A
Molybdenum Cofactor Deficiency Type A (MoCD) is a very rare autosomal recessive disorder that is essentially fatal early in life. Naturally occurring cPMP is present in the body of all healthy normal individuals.
- Trial name
- NCT00957749
- Testing
- cPMP
- Where
- Australia
- Volunteers
- 10 people
- Sponsor
- Orphatech Pharmaceuticals, GmbH
Molybdenum Cofactor Deficiency, Type A
Study of ORGN001 (Formerly ALXN1101) in Neonates, Infants and Children With Molybdenum Cofactor Deficiency (MOCD) Type A
To evaluate the safety and efficacy of ORGN001(formerly ALXN1101) in neonate patients with MoCD Type A
- Trial name
- NCT02629393
- Testing
- ORGN001 (formerly ALXN1101)
- Where
- 11 sites in United States, Israel and 4 more
- Volunteers
- 5 people
- Sponsor
- Origin Biosciences
Molybdenum Cofactor Deficiency, Isolated Sulfite Oxidase Deficiency
A Natural History Study of Molybdenum Cofactor and Isolated Sulfite Oxidase Deficiencies
Primary objective: Characterize the natural history of MoCD type A in terms of survival Secondary objectives: 1. Evaluate blood and urine for biochemical markers 2.
- Trial name
- NCT01735188
- Where
- 32 sites in United States, Canada and 12 more
- Volunteers
- 65 people
- Sponsor
- Origin Biosciences
Molybdenum Cofactor Deficiency, Type A
Safety & Efficacy Study of ORGN001 (Formerly ALXN1101) in Pediatric Patients With MoCD Type A Currently Treated With rcPMP
This study will include a screening period, a 6-month treatment period, followed by long-term extension period expected to last approximately 72 months.
- Trial name
- NCT02047461
- Testing
- ORGN001 (formerly ALXN1101)
- Where
- 6 sites in United States, Australia and 3 more
- Volunteers
- 8 people
- Sponsor
- Origin Biosciences
Spinal Muscular Atrophy, Fragile X Syndrome
Early Check: Expanded Screening in Newborns
Early Check provides voluntary screening of newborns for a selected panel of conditions. The study has three main objectives: 1) develop and implement an approach to identify affected infants, 2) address the impact on infants and…
- Conditions
- Spinal Muscular Atrophy, Fragile X Syndrome, Fragile X - Premutation, Duchenne Muscular Dystrophy
- Trial name
- NCT03655223
- Testing
- Confirmatory Testing
- Where
- United States
- Volunteers
- 30,000 people, healthy volunteers welcome
- Sponsor
- RTI International
Molybdenum Cofactor Deficiency
A Retrospective, Observational, Noninterventional Data Collection Study for Patients With Molybdenum Cofactor Deficiency Who Have Been Previously Treated With Cyclic Pyranopterin Monophosphate (cPMP)
The primary objective is to assess safety and efficacy data of Escherichia coli-derived cPMP in patients with molybdenum cofactor deficiency (MoCD).
- Trial name
- NCT01640717
- Where
- 13 sites in United States, Australia and 4 more
- Volunteers
- 15 people
- Sponsor
- Origin Biosciences
Molybdenum Cofactor Deficiency (MoCD), Rare Autosomal Recessive Disorder
Phase 1 Single Dose Study of ALXN1101 in Healthy Volunteers
Phase 1 single dose study of ALXN1101 in healthy volunteers.
- Conditions
- Molybdenum Cofactor Deficiency (MoCD), Rare Autosomal Recessive Disorder, Deficiency of Activity of Molybdenum-dependent Enzymes (Sulfite Oxidase [SOX], Xanthine Dehydrogenase, and Aldehyde Oxidase)
- Trial name
- NCT01894165
- Testing
- ALXN1101
- Where
- United States
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Origin Biosciences