Clinical Trial: Study of cPMP (Precusor Z) to Treat Molybdenum Cofactor Deficiency (MoCD) Type A
Study Status: Withdrawn
Recruit Status: Withdrawn
Study Type: Interventional
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
Brief Summary:
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.
Detailed Summary:
Sponsor: Orphatech Pharmaceuticals, GmbH
Current Primary Outcome: Urine biomarkers SSC and sulfite [ Time Frame: Daily collection throughout study; analyzed at 3 months ]
Original Primary Outcome: Same as current
Current Secondary Outcome:
- neurological examination [ Time Frame: collected daily; analyzed at 3 months ]
- Safety measures (vital signs, adverse events) [ Time Frame: collected daily; analyzed at 3 months ]
Original Secondary Outcome: Same as current
Information By: Orphatech Pharmaceuticals, GmbH
Dates:
Date Received: July 10, 2009
Date Started: August 2009
Date Completion:
Last Updated: January 29, 2011
Last Verified: January 2011
