A Retrospective, Observational, Noninterventional Data Collection Study for Patients With Molybdenum Cofactor Deficiency Who Have Been Previously Treated With Cyclic Pyranopterin Monophosphate (cPMP)
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government.
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Safety and Efficacy [ Time Frame: For up to 60 months from the initial date of treatment with cPMP ]
This is a noninterventional, observational, retrospective study to collect data on pediatric patients with MoCD who have received E. coli derived cPMP by intravenous only administration. The study will neither provide treatment with cPMP nor alter any ongoing treatment schedules; rather, its objective is to retrospectively collect data on MoCD history and previous treatment with intravenous E. coli derived cPMP, which is documented in the medical records of patients who have received treatment according to a named patient treatment plan.
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Ages Eligible for Study:
Child, Adult, Older Adult
Sexes Eligible for Study:
Accepts Healthy Volunteers:
All patients who received only intravenous cPMP under named-patient use will be eligible
Male or female of any age.
Patient with MoCD type A, suspected type A, or type B.
Patient previously received cPMP only by intravenous route of administration.
Parent(s) or legal guardian(s), depending on local regulations, has voluntarily provided written informed consent for the Investigator, Investigator's designee, or Sponsor designee to review, collect, transmit, and analyze data extracted from the medical record. In the case of a deceased patient for whom the parents or legal guardians could not be located, the appropriate ethical review committee may assign another person as legal representative to provide consent, where applicable per local and country regulations.
Patient's parent(s) or legal guardian(s) are unable to understand the nature and scope of the study.