Study of cPMP (Precusor Z) to Treat Molybdenum Cofactor Deficiency (MoCD) Type A
Status:
Withdrawn
Trial end date:
1969-12-31
Target enrollment:
Participant gender:
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.