Clinical trial NCT01640717: A Retrospective, Observational, Noninterventional Data Collection Study for Patients With Molybdenum Cofactor Deficiency Who Have Been Previously Treated With Cyclic Pyranopterin Monophosphate (cPMP)
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)
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Molybdenum Cofactor Deficiency
Lead sponsor: Origin Biosciences
Sponsor class: INDUSTRY
First posted: 2012-07-16
Enrollment: 15
Sex: ALL
Minimum age (years): 1
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: OTHER
Time perspective: RETROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: All patients who received only intravenous cPMP under named-patient use will be eligible

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


Eligibility criteria: 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.

Primary outcomes:
measure: Safety and Efficacy
timeFrame: For up to 60 months from the initial date of treatment with cPMP
Secondary outcomes:
Other outcomes:
Locations:
city: Nashville
country: United States
facility: Vanderbilt Children's Hospital
state: Tennessee
city: Milwaukee
country: United States
facility: Children's Hospital of Wisconsin
state: Wisconsin
city: Heidelberg
country: Australia
facility: Neonatologist, Department of Paediatrics, Mercy Hospital for Women
city: Melbourne
country: Australia
facility: Monash Medical Centre
city: Westmead
country: Australia
facility: Western Sydney Genetics Program & Sydney Medical School
city: Frankfurt
country: Germany
facility: Frankfurt Children's Hospital
city: Koblenz
country: Germany
facility: Akademisches Lehrkrankenhaus der Johannes Gutenberg
city: Koln
country: Germany
facility: University Hospital of Cologne
city: Groningen
country: Netherlands
facility: Beatrix Children's Hospital
city: Gaziantep
country: Turkey
facility: TC Saglik Bakanligi Gaziantep Cocuk Hastaliklari Hastanesi
city: Birmingham
country: United Kingdom
facility: Birmingham Children's Hospital
city: Glasgow
country: United Kingdom
facility: Royal Hospital for Sick Children
city: Manchester
country: United Kingdom
facility: Manchester Academic Health Science Centre
Study officials:
References: