Trial Review

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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT01640717




Registration number
NCT01640717
Ethics application status
Date submitted
12/07/2012
Date registered
16/07/2012
Date last updated
19/03/2019

Titles & IDs
Public title
A Retrospective, Observational, Noninterventional Data Collection Study for Patients With Molybdenum Cofactor Deficiency Who Have Been Previously Treated With Cyclic Pyranopterin Monophosphate (cPMP)
Scientific title
A Retrospective, Observational, Noninterventional Data Collection Study for Patients With Molybdenum Cofactor Deficiency Who Have Been Previously Treated With Cyclic Pyranopterin Monophosphate (cPMP)
Secondary ID [1] 0 0
ALX-MCD-501
Secondary ID [2] 0 0
ALX-MCD-501
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Molybdenum Cofactor Deficiency 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Metabolic and Endocrine 0 0 0 0
Metabolic disorders
Metabolic and Endocrine 0 0 0 0
Other metabolic disorders

Intervention/exposure
Study type
Observational
Patient registry
Target follow-up duration
Target follow-up type
Description of intervention(s) / exposure
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Safety and Efficacy
Timepoint [1] 0 0
For up to 60 months from the initial date of treatment with cPMP

Eligibility
Key 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.
Minimum age
1 Day
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Patient's parent(s) or legal guardian(s) are unable to understand the nature and scope of the study.

Study design
Purpose
Duration
Selection
Timing
Retrospective
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/11/2012
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
1/10/2014
Sample size
Target
Accrual to date
Final
15
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Neonatologist, Department of Paediatrics, Mercy Hospital for Women - Heidelberg
Recruitment hospital [2] 0 0
Monash Medical Centre - Melbourne
Recruitment hospital [3] 0 0
Western Sydney Genetics Program & Sydney Medical School - Westmead
Recruitment postcode(s) [1] 0 0
- Heidelberg
Recruitment postcode(s) [2] 0 0
- Melbourne
Recruitment postcode(s) [3] 0 0
- Westmead
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Tennessee
Country [2] 0 0
United States of America
State/province [2] 0 0
Wisconsin
Country [3] 0 0
Germany
State/province [3] 0 0
Frankfurt
Country [4] 0 0
Germany
State/province [4] 0 0
Koblenz
Country [5] 0 0
Germany
State/province [5] 0 0
Koln
Country [6] 0 0
Netherlands
State/province [6] 0 0
Groningen
Country [7] 0 0
Turkey
State/province [7] 0 0
Gaziantep
Country [8] 0 0
United Kingdom
State/province [8] 0 0
Birmingham
Country [9] 0 0
United Kingdom
State/province [9] 0 0
Glasgow
Country [10] 0 0
United Kingdom
State/province [10] 0 0
Manchester

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Origin Biosciences
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
The primary objective is to assess safety and efficacy data of Escherichia coli-derived cPMP in patients with molybdenum cofactor deficiency (MoCD).
Trial website
https://clinicaltrials.gov/study/NCT01640717
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT01640717