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

March 15, 2019 updated by: Origin Biosciences
The primary objective is to assess safety and efficacy data of Escherichia coli-derived cPMP in patients with molybdenum cofactor deficiency (MoCD).

Study Overview

Status

Completed

Conditions

Study Type

Observational

Enrollment (Actual)

15

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Australia
      • Heidelberg, Australia
        • Neonatologist, Department of Paediatrics, Mercy Hospital for Women
      • Melbourne, Australia
        • Monash Medical Centre
      • Westmead, Australia
        • Western Sydney Genetics Program & Sydney Medical School
  • Germany
      • Frankfurt, Germany
        • Frankfurt Children's Hospital
      • Koblenz, Germany
        • Akademisches Lehrkrankenhaus der Johannes Gutenberg
      • Koln, Germany
        • University Hospital of Cologne
  • Netherlands
      • Groningen, Netherlands
        • Beatrix Children's Hospital
  • Turkey
      • Gaziantep, Turkey
        • TC Saglik Bakanligi Gaziantep Cocuk Hastaliklari Hastanesi
  • United Kingdom
      • Birmingham, United Kingdom
        • Birmingham Children's Hospital
      • Glasgow, United Kingdom
        • Royal Hospital for Sick Children
      • Manchester, United Kingdom
        • Manchester Academic Health Science Centre
  • United States
    • Tennessee
      • Nashville, Tennessee, United States, 37232
        • Vanderbilt Children's Hospital
    • Wisconsin
      • Milwaukee, Wisconsin, United States, 53226
        • Children's Hospital of Wisconsin

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

1 day and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

Study Population

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

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Other
  • Time Perspectives: Retrospective

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
For up to 60 months from the initial date of treatment with cPMP

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Origin Biosciences

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

November 1, 2012

Primary Completion (Actual)

October 1, 2014

Study Completion (Actual)

October 1, 2014

Study Registration Dates

First Submitted

July 12, 2012

First Submitted That Met QC Criteria

July 13, 2012

First Posted (Estimate)

July 16, 2012

Study Record Updates

Last Update Posted (Actual)

March 19, 2019

Last Update Submitted That Met QC Criteria

March 15, 2019

Last Verified

March 1, 2019

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ALX-MCD-501 (Registry Identifier: ALX-MCD-501)

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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