NCT00957749

Study of cPMP (Precusor Z) to Treat Molybdenum Cofactor Deficiency (MoCD) Type A

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

• Status: Withdrawn
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 10 (estimated)
• Sponsor: Orphatech Pharmaceuticals, GmbH · Industry
• Sex: All
• Age: 6 Weeks
• Healthy volunteers: Not accepted

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.

Conditions

• Molybdenum Cofactor Deficiency Type A

Interventions

Type	Name	Description
DRUG	cPMP	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.

Timeline

• Start date: 2009-08-01
• Primary completion: 2010-04-01
• First posted: 2009-08-12
• Last updated: 2011-02-01

Locations

1 site across 1 country: Australia