A Retrospective, Observational, Noninterventional Data Collection Study for Patients With Molybdenum Cofactor Deficiency Who Have Been Previously Treated With Cyclic Pyranopterin Monophosphate (cPMP)
Trial Snapshot
- Phase
- Not Applicable
- Status
- Completed
- Sponsor
- Enrollment
- 15
- Locations
- 13
- Primary Endpoint
- Safety and Efficacy
Study Overview
Brief Summary
The primary objective is to assess safety and efficacy data of Escherichia coli-derived cPMP in patients with molybdenum cofactor deficiency (MoCD).
Study Design
- Study Type
- Observational
- Observational Model
- Other
- Time Perspective
- Retrospective
Eligibility Criteria
- Ages
- 1 Day to — (Child, Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •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.
Exclusion Criteria
- •Patient's parent(s) or legal guardian(s) are unable to understand the nature and scope of the study.
Outcomes
Primary Outcomes
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.
Secondary Outcomes
No secondary outcomes reported