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 studied: Molybdenum Cofactor Deficiency

In brief

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

Key facts

Study ID

NCT01640717

Run by

Origin Biosciences

People needed

15

Starts

2012-11-01

Expected to finish

2014-10-01

Last updated by the study team

2019-03-19

Who can join

Age: 0 and older. Sex: any. Healthy volunteers: not accepted.

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.

Where it is running

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

Full record on ClinicalTrials.gov

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