Molybdenum Cofactor Deficiency Type A Clinical Trial
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
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.
n/a
Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment