Study of cPMP (Precusor Z) to Treat Molybdenum Cofactor Deficiency (MoCD) Type A
|ClinicalTrials.gov Identifier: NCT00957749|
Recruitment Status : Withdrawn (IND application was withdrawn, and therefore study listing is being withdrawn.)
First Posted : August 12, 2009
Last Update Posted : February 1, 2011
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.
|Condition or disease||Intervention/treatment||Phase|
|Molybdenum Cofactor Deficiency Type A||Drug: cPMP||Phase 1 Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||10 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|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|
|Study Start Date :||August 2009|
|Estimated Primary Completion Date :||April 2010|
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.
- Urine biomarkers SSC and sulfite [ Time Frame: Daily collection throughout study; analyzed at 3 months ]
- neurological examination [ Time Frame: collected daily; analyzed at 3 months ]
- Safety measures (vital signs, adverse events) [ Time Frame: collected daily; analyzed at 3 months ]
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00957749
|Monash Medical Centre|
|Melbourne, Victoria, Australia, 3168|
|Principal Investigator:||Alex Veldman, MD||Monash Medical Centre|