Clinical trials: Molybdenum Cofactor Deficiency
- Condition Molybdenum Cofactor Deficiency
- Funder type Industry (pharma, biotech, devices)
- Age group Children (under 18)
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
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