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

This study has been completed.
Information provided by (Responsible Party):
Alexion Pharmaceuticals Identifier:
First received: July 12, 2012
Last updated: February 17, 2016
Last verified: February 2016
The primary objective is to assess safety and efficacy data of Escherichia coli-derived cPMP in patients with molybdenum cofactor deficiency (MoCD).

Molybdenum Cofactor Deficiency

Study Type: Observational
Study Design: Time Perspective: Retrospective
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)

Resource links provided by NLM:

Further study details as provided by Alexion Pharmaceuticals:

Primary Outcome Measures:
  • Safety and Efficacy [ Time Frame: For up to 60 months from the initial date of treatment with cPMP ] [ Designated as safety issue: No ]
    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.

Enrollment: 15
Study Start Date: November 2012
Study Completion Date: October 2014
Primary Completion Date: October 2014 (Final data collection date for primary outcome measure)

Ages Eligible for Study:   Child, Adult, Senior
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
All patients who received only intravenous cPMP under named-patient use will be eligible

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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT01640717

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

Responsible Party: Alexion Pharmaceuticals Identifier: NCT01640717     History of Changes
Other Study ID Numbers: ALX-MCD-501  ALX-MCD-501 
Study First Received: July 12, 2012
Last Updated: February 17, 2016
Health Authority: United States: Food and Drug Administration

Additional relevant MeSH terms:
Metal Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Metabolic Diseases
Trace Elements
Growth Substances
Physiological Effects of Drugs processed this record on October 21, 2016