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

Molybdenum Cofactor Deficiency Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01640717
• Other study ID #: ALX-MCD-501
• Secondary ID: ALX-MCD-501
• Status: Completed
• Start date: November 2012
• Est. completion date: October 2014

Study information

• Verified date: March 2019
• Source: Origin Biosciences
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

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

Recruitment information / eligibility

• Status: Completed
• Enrollment: 15
• Est. completion date: October 2014
• Est. primary completion date: October 2014
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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.

Related Conditions & MeSH terms

• Molybdenum Cofactor Deficiency

Locations

Country	Name	City	State
Australia	Neonatologist, Department of Paediatrics, Mercy Hospital for Women	Heidelberg
Australia	Monash Medical Centre	Melbourne
Australia	Western Sydney Genetics Program & Sydney Medical School	Westmead
Germany	Frankfurt Children's Hospital	Frankfurt
Germany	Akademisches Lehrkrankenhaus der Johannes Gutenberg	Koblenz
Germany	University Hospital of Cologne	Koln
Netherlands	Beatrix Children's Hospital	Groningen
Turkey	TC Saglik Bakanligi Gaziantep Cocuk Hastaliklari Hastanesi	Gaziantep
United Kingdom	Birmingham Children's Hospital	Birmingham
United Kingdom	Royal Hospital for Sick Children	Glasgow
United Kingdom	Manchester Academic Health Science Centre	Manchester
United States	Children's Hospital of Wisconsin	Milwaukee	Wisconsin
United States	Vanderbilt Children's Hospital	Nashville	Tennessee

Sponsors (1)

Lead Sponsor	Collaborator
Origin Biosciences

Countries where clinical trial is conducted

United States,  Australia,  Germany,  Netherlands,  Turkey,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Safety and Efficacy	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.	For up to 60 months from the initial date of treatment with cPMP