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 is currently recruiting participants. (see Contacts and Locations)
Verified December 2013 by Alexion Pharmaceuticals
Information provided by (Responsible Party):
Alexion Pharmaceuticals
ClinicalTrials.gov Identifier:
First received: July 12, 2012
Last updated: December 2, 2013
Last verified: December 2013

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: 5 years ] [ 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.

Estimated Enrollment: 11
Study Start Date: November 2012
Estimated Study Completion Date: March 2014
Estimated Primary Completion Date: March 2014 (Final data collection date for primary outcome measure)

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 ClinicalTrials.gov identifier: NCT01640717

Contact: Alexion Pharmaceuticals (Sponsor) clinicaltrials@alxn.com

United States, Tennessee
Vanderbilt Children's Hospital Not yet recruiting
Nashville, Tennessee, United States
Contact: Alexion Pharmaceuticals       clinicaltrials@alxn.com   
United States, Wisconsin
Children's Hospital of Wisconsin Completed
Milwaukee, Wisconsin, United States
Neonatologist, Department of Paediatrics, Mercy Hospital for Women Completed
Heidelberg, Australia
Monash Medical Centre Active, not recruiting
Melbourne, Australia
Western Sydney Genetics Program & Sydney Medical School Completed
Westmead, Australia
Charite Universitatsmedizin Berlin Not yet recruiting
Berlin, Germany
Contact: Alexion Pharmaceuticals       clinicaltrials@alxn.com   
Akademisches Lehrkrankenhaus der Johannes Gutenberg Recruiting
Koblenz, Germany
Contact: Alexion Pharmaceuticals       clinicaltrials@alxn.com   
University Hospital of Cologne Not yet recruiting
Koln, Germany
Contact: Alexion pharmaceuticals       clinicaltrials@alxn.com   
Beatrix Children's Hospital Division of Metabolic Diseases Recruiting
Groningen, Netherlands
Contact: Alexion Pharmaceuticals       clinicaltrials@alxn.com   
United Kingdom
Royal Hospital for Sick Children Recruiting
Glasgow, United Kingdom
Contact: Alexion Pharmaceuticals       clinicaltrials@alxn.com   
Genetic Medicine Manchester Academic Health Science Centre, Wilink Biochemical Genetics Unit Completed
Manchester, United Kingdom
Sponsors and Collaborators
Alexion Pharmaceuticals
  More Information

No publications provided

Responsible Party: Alexion Pharmaceuticals
ClinicalTrials.gov Identifier: NCT01640717     History of Changes
Other Study ID Numbers: ALX-MCD-501, ALX-MCD-501
Study First Received: July 12, 2012
Last Updated: December 2, 2013
Health Authority: United States: Food and Drug Administration

Keywords provided by Alexion Pharmaceuticals:

Additional relevant MeSH terms:
Metal Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Metabolic Diseases

ClinicalTrials.gov processed this record on September 18, 2014