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



Status:Completed
Healthy:No
Age Range:Any
Updated:3/21/2019
Start Date:November 2012
End Date:October 2014

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


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.
We found this trial at
3
sites
9000 W Wisconsin Ave #270
Milwaukee, Wisconsin 53226
(414) 266-2000
Children's Hospital of Wisconsin Nothing matters more than our children. At Children's Hospital of Wisconsin,...
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Milwaukee, WI
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Heidelberg,
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2200 Children's Way
Nashville, Tennessee 37232
(615) 936-1000
Vanderbilt Children's Hospital Monroe Carell Jr. Children's Hospital at Vanderbilt is one of the nation's...
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from
Nashville, TN
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