A multi-centre study of the safety, tolerability and effects of intravenously administered Cyclic Pyranopterin Monophosphate (cPMP) in patients with molybdenum cofactor deficiency type A.

Cyclic pyranopterin monophosphate (cPMP), also known as precursor Z, which has been extracted from cultures of E. coli and purified. The first day treatment will be a total dose of 80 micrograms per kilogram of body weight of cPMP and administered as four intravenous (IV) infusions. On days 2, 3 and 4, the dose will be administered as two infusions. On days 5 and 6, the dose will be administered as one infusion over three hours and; on days 7 to 12, the dose will be administered as one infusion over one hour. On subsequent days, the daily dose will be 160 microgram per kilogram administered as an infusion over one hour. The dose and regimen will be adjusted according to the urine metabolites, sulphur cysteine and sulphate levels. The administration of cPMP will continue for a minimum of 90 days provided the patient is deriving a benefit from cPMP treatment.