Trial Review

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Trial registered on ANZCTR


Registration number
ACTRN12609000849291
Ethics application status
Approved
Date submitted
12/01/2009
Date registered
30/09/2009
Date last updated
15/02/2011
Type of registration
Prospectively registered

Titles & IDs
Public title
A multi-centre study of the safety, tolerability and effects of intravenously administered Cyclic Pyranopterin Monophosphate (cPMP) in patients with molybdenum cofactor deficiency type A.
Scientific title
A multi-centre, open-label study of the safety, tolerability and pharmacodynamics of intravenously administered Cyclic Pyranopterin Monophosphate (cPMP) (precursor Z) in patients with molybdenum CoFactor deficiency type A
Secondary ID [1] 253609 0
cPMP01-08
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Molybdenum Cofactor Deficiency (MoCD) Type A 4188 0
Condition category
Condition code
Human Genetics and Inherited Disorders 4397 4397 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Cyclic pyranopterin monophosphate (cPMP), also known as precursor Z, which has been extracted from cultures of E. coli and purified.
The first day treatment will be a total dose of 80 micrograms per kilogram of body weight of cPMP and administered as four intravenous (IV) infusions.
On days 2, 3 and 4, the dose will be administered as two infusions.
On days 5 and 6, the dose will be administered as one infusion over three hours and;
on days 7 to 12, the dose will be administered as one infusion over one hour.
On subsequent days, the daily dose will be 160 microgram per kilogram administered as an infusion over one hour.
The dose and regimen will be adjusted according to the urine metabolites, sulphur cysteine and sulphate levels.
The administration of cPMP will continue for a minimum of 90 days provided the patient is deriving a benefit from cPMP treatment.
Intervention code [1] 3904 0
Treatment: Drugs
Comparator / control treatment
No control group as this is a Single Group Study
Control group
Uncontrolled

Outcomes
Primary outcome [1] 5275 0
To assess the safety, tolerability and pharmacodynamics of cPMP treatment by means of a physical examination including head circumference, neurological examinations, electrocardiograph (ECG), vital signs, adverse events, blood gas analysis, blood chemistry, hematology, urinalysis (including creatinine, s-sulfocysteine (SSC), xanthine and uric acid) and dipstick for sulfite.
Timepoint [1] 5275 0
Assessed at multiple intervals, daily, for 90 days.
Secondary outcome [1] 257805 0
Nil.
Timepoint [1] 257805 0
Nil.

Eligibility
Key inclusion criteria
-Neonate or infant, less then 6 weeks at the time of diagnosis, age less than 8 weeks at start of treatment with the study medication. It is important to diagnose the condition and initiate treatment as soon after birth as possible.
-Documented diagnosis of molybdenum cofactor deficiency (MoCD) Type A based on the absence of cPMP and the presence of sulfite and s-sulfocysteine in the urine, absence of urothione in the urine and genetic analysis showing a mutation in the MOCS1 gene
- A parent or legal guardian voluntarily provided written informed consent to participate in the study and comply with study procedures.
- Approval of the study protocol by the local Human Research Ethics Committee (HREC) / Independent Review Board (IRB) and government or regulatory authorities (if applicable).
Minimum age
0 Weeks
Maximum age
6 Weeks
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- MoCD Type B (MOCS2 mutation) or Type C (gephyrin gene mutation)
- Sulfite oxidase deficiency
- Patients older than 6 weeks at the time of diagnosis

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 1
Type of endpoint/s
Pharmacodynamics
Statistical methods / analysis

Recruitment
Recruitment status
Suspended
Date of first participant enrolment
Anticipated
30/09/2009
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
10
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment outside Australia
Country [1] 1522 0
Turkey
State/province [1] 1522 0
Country [2] 1523 0
Germany
State/province [2] 1523 0
Country [3] 1524 0
United States of America
State/province [3] 1524 0
Country [4] 1525 0
United Kingdom
State/province [4] 1525 0

Funding & Sponsors
Funding source category [1] 4368 0
Commercial sector/Industry
Name [1] 4368 0
PMP(Vic) Pty Ltd
Country [1] 4368 0
Australia
Primary sponsor type
Commercial sector/Industry
Name
PMP(Vic) Pty Ltd
Address
Level 1
74 Kingsway
Glen Waverley, Victoria 3150
Country
Australia
Secondary sponsor category [1] 3934 0
Commercial sector/Industry
Name [1] 3934 0
Orphatec Pharmaceuticals
Address [1] 3934 0
Otto-Fischer-Str. 12-14
50674 Koeln
Country [1] 3934 0
Germany

Ethics approval
Ethics application status
Approved

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 35082 0
Address 35082 0
Country 35082 0
Phone 35082 0
Fax 35082 0
Email 35082 0
Contact person for public queries
Name 12429 0
Yelda Ogru
Address 12429 0
Level 1
74 Kingsway
Glen Waverley
VICTORIA 3150
Country 12429 0
Australia
Phone 12429 0
+61447924339
Fax 12429 0
Email 12429 0
yelda.ogru@orphatec.com
Contact person for scientific queries
Name 3357 0
Yelda Ogru
Address 3357 0
Level 1
74 Kingsway
Glen Waverley
VICTORIA 3150
Country 3357 0
Australia
Phone 3357 0
+61447924339
Fax 3357 0
Email 3357 0
yelda.ogru@orphatec.com

No information has been provided regarding IPD availability


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

Documents added manually
No documents have been uploaded by study researchers.

Documents added automatically
No additional documents have been identified.