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. It is processed to molybdopterin, which is further processed to molybdenum cofactor. Molybdenum cofactor is essential for the function of important enzymes.
There is currently no treatment for MoCD, and affected infants develop severe neurological damage which often results in infant death.
This study is the first clinical trial to investigate the potential of replacement of cPMP to infants with MoCD Type A. The safety, tolerability, and pharmacodynamics of daily intravenous administration of cPMP over 3 months will be determined.
|Study Design:||Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||A Multicenter, Open-Label Study of the Safety, Tolerability, and Pharmacodynamics of Intravenously Administered cPMP (Precursor Z) in Patients With Molybdenum Cofactor Deficiency Type A|
- Urine biomarkers SSC and sulfite [ Time Frame: Daily collection throughout study; analyzed at 3 months ] [ Designated as safety issue: No ]
- neurological examination [ Time Frame: collected daily; analyzed at 3 months ] [ Designated as safety issue: No ]
- Safety measures (vital signs, adverse events) [ Time Frame: collected daily; analyzed at 3 months ] [ Designated as safety issue: Yes ]
|Study Start Date:||August 2009|
|Estimated Primary Completion Date:||April 2010 (Final data collection date for primary outcome measure)|
Intravenous solution administered daily. Dose titrated from 80 μg/kg on Days 1-12 to 120 μg/kg on Days 13-34 to 160 μg/kg for days 35-90.
|Monash Medical Centre|
|Melbourne, Victoria, Australia, 3168|
|Principal Investigator:||Alex Veldman, MD||Monash Medical Centre|