Clinical Trial: A Retrospective, Observational, Noninterventional Data Collection Study for Patients With Molybdenum Cofactor Deficiency Who Have Been Previously Treated With Cyclic Pyranopterin Monophosphate (cPMP)

Study Status: Completed
Recruit Status: Completed
Study Type: Observational

Official Title: A Retrospective, Observational, Noninterventional Data Collection Study for Patients With Molybdenum Cofactor Deficiency Who Have Been Previously Treated With Cyclic Pyran

Brief Summary: The primary objective is to assess safety and efficacy data of Escherichia coli-derived cPMP in patients with molybdenum cofactor deficiency (MoCD).

Detailed Summary:
Sponsor: Alexion Pharmaceuticals

Current Primary Outcome: 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.


Original Primary Outcome:

Current Secondary Outcome:

Original Secondary Outcome:

Information By: Alexion Pharmaceuticals

Dates:
Date Received: July 12, 2012
Date Started: November 2012
Date Completion:
Last Updated: February 17, 2016
Last Verified: February 2016