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

Completed

• Conditions: Molybdenum Cofactor Deficiency

• Registration Number: NCT01640717
• Lead Sponsor: Origin Biosciences

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 Description

Not available

Study Timeline

• Study First Submitted: 2012-07-12
• Study First Submitted QC: 2012-07-13
• Study First Posted: 2012-07-16
• Study Start: 2012-11
• Primary Completion: 2014-10
• Study Completion: 2014-10
• Status Verified: 2019-03
• Last Update Submitted: 2019-03-15
• Last Update Posted: 2019-03-19

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 15

Inclusion Criteria

1. Male or female of any age.
2. Patient with MoCD type A, suspected type A, or type B.
3. Patient previously received cPMP only by intravenous route of administration.
4. 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.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Safety and Efficacy	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.

Trial Locations

Locations (13)

Vanderbilt Children's Hospital; 🇺🇸 Nashville, Tennessee, United States

Neonatologist, Department of Paediatrics, Mercy Hospital for Women; 🇦🇺 Heidelberg, Australia

Birmingham Children's Hospital; 🇬🇧 Birmingham, United Kingdom

Monash Medical Centre; 🇦🇺 Melbourne, Australia

Western Sydney Genetics Program & Sydney Medical School; 🇦🇺 Westmead, Australia

Royal Hospital for Sick Children; 🇬🇧 Glasgow, United Kingdom

Manchester Academic Health Science Centre; 🇬🇧 Manchester, United Kingdom

Akademisches Lehrkrankenhaus der Johannes Gutenberg; 🇩🇪 Koblenz, Germany

TC Saglik Bakanligi Gaziantep Cocuk Hastaliklari Hastanesi; 🇹🇷 Gaziantep, Turkey

Frankfurt Children's Hospital; 🇩🇪 Frankfurt, Germany

University Hospital of Cologne; 🇩🇪 Koln, Germany

Beatrix Children's Hospital; 🇳🇱 Groningen, Netherlands

Children's Hospital of Wisconsin; 🇺🇸 Milwaukee, Wisconsin, United States