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

NCT01640717 | Completed

• 1 other identifier: ALX-MCD-501
• Study Type: observational
• Target: 15
• Locations: 6 countries
• Sites: 13

Brief Summary

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

Conditions

Molybdenum Cofactor Deficiency

Outcome Measures

Primary Outcomes (1)

• 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

Eligibility Criteria

• Age: 1 Day+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

All patients who received only intravenous cPMP under named-patient use will be eligible

You may qualify if:

• 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.

You may not qualify if:

• Patient's parent(s) or legal guardian(s) are unable to understand the nature and scope of the study.

Sponsors & Collaborators

• Origin Biosciences: lead

Study Sites (13)

Vanderbilt Children's Hospital

Nashville, Tennessee, 37232, United States

Location

Children's Hospital of Wisconsin

Milwaukee, Wisconsin, 53226, United States

Location

Neonatologist, Department of Paediatrics, Mercy Hospital for Women

Heidelberg, Australia

Location

Monash Medical Centre

Melbourne, Australia

Location

Western Sydney Genetics Program & Sydney Medical School

Westmead, Australia

Location

University Hospital of Cologne

Cologne, Germany

Location

Frankfurt Children's Hospital

Frankfurt, Germany

Location

Akademisches Lehrkrankenhaus der Johannes Gutenberg

Koblenz, Germany

Location

Beatrix Children's Hospital

Groningen, Netherlands

Location

TC Saglik Bakanligi Gaziantep Cocuk Hastaliklari Hastanesi

Gaziantep, Turkey (Türkiye)

Location

Birmingham Children's Hospital

Birmingham, United Kingdom

Location

Royal Hospital for Sick Children

Glasgow, United Kingdom

Location

Manchester Academic Health Science Centre

Manchester, United Kingdom

Location

MeSH Terms

Conditions

Molybdenum cofactor deficiency

Study Design

• Study Type: observational
• Observational Model: OTHER
• Time Perspective: RETROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 12, 2012
• First Posted: July 16, 2012
• Study Start: November 1, 2012
• Primary Completion: October 1, 2014
• Study Completion: October 1, 2014
• Last Updated: March 19, 2019

Record last verified: 2019-03

Locations

US (2); AU (3); NL (1); TU (1); GB (3); DE (3)