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OpenTrials
Completed

NCT Number: NCT01640717

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

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

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Key information

Conditions

Molybdenum Cofactor Deficiency

Age range

1 day and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Neonatologist, Department of Paediatrics, Mercy Hospital for Women, Heidelberg, Australia

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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.

Exclusion criteria

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

Treatment and study plan

Primary outcomes

  1. 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.

Sponsors and collaborators

Lead sponsor

Origin Biosciences

Industry

Registry information

Important dates

Study start
2012
Primary completion
2014
Study completion
2014
First posted
Jul 16, 2012
Registry last updated
Mar 19, 2019

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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