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

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT01640717

Enrollment

Registered

2012-07-16

Start date

2012-11-30

Completion date

2014-10-31

Last updated

2019-03-19

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Molybdenum Cofactor Deficiency

Brief summary

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

Interventions

None listed

Study design

Observational model

OTHER

Time perspective

RETROSPECTIVE

Eligibility

Sex/Gender

ALL

Age

1 Days to No maximum

Healthy volunteers

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.

Design outcomes

Primary

Measure	Time frame	Description
Safety and Efficacy	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.

Countries

Australia, Germany, Netherlands, Turkey (Türkiye), United Kingdom, United States

Outcome results