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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT02047461




Registration number
NCT02047461
Ethics application status
Date submitted
9/01/2014
Date registered
28/01/2014
Date last updated
17/10/2023

Titles & IDs
Public title
Safety & Efficacy Study of ORGN001 (Formerly ALXN1101) in Pediatric Patients With MoCD Type A Currently Treated With rcPMP
Scientific title
A Phase 2, Multicenter, Multinational, Open-Label, Dose-Escalation Study to Evaluate the Safety and Efficacy of ORGN001 (Formerly ALXN1101) in Pediatric Patients With Molybdenum Cofactor Deficiency (MoCD) Type A Currently Treated With Recombinant Escherichia Coli-derived Cyclic Pyranopterin Monophosphate (rcPMP)
Secondary ID [1] 0 0
2013-002701-56
Secondary ID [2] 0 0
ALXN1101-MCD-201
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Molybdenum Cofactor Deficiency, Type A 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
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - ORGN001 (formerly ALXN1101)

Experimental: ORGN001 (formerly ALXN1101) - daily IV infusions


Treatment: Drugs: ORGN001 (formerly ALXN1101)
IV infusion
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Safety of ORGN001 (Formerly ALXN1101)
Timepoint [1] 0 0
Baseline to Month 24 for all patients plus additional follow-up up to Month 90
Secondary outcome [1] 0 0
Pharmacokinetics (Actual Plasma Concentration) of ORGN001 (Formerly ALXN1101)
Timepoint [1] 0 0
First 6 months at each dose level, where available
Secondary outcome [2] 0 0
S-sulfocysteine (Umol/L) Normalized to Urine Creatinine (mmol/L) - Change From Baseline Over Time
Timepoint [2] 0 0
Baseline to Month 24 for all patients plus additional follow-up to Month 90
Secondary outcome [3] 0 0
Effect of ORGN001 (Formerly ALXN1101) on Neurologic Function Including Motor Examination
Timepoint [3] 0 0
Baseline to Month 24 for all patients plus additional follow-up until Month 30
Secondary outcome [4] 0 0
Long-term Safety of ORGN001 (Formerly ALXN1101)
Timepoint [4] 0 0
Baseline to Month 24 for all patients plus additional follow up until Month 72

Eligibility
Key inclusion criteria
* Male or female patients with a genetically confirmed diagnosis of MoCD Type A (MOCS1 mutation)
* Currently treated with rcPMP infusions
Minimum age
No limit
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- Current or planned treatment with another investigational drug or device, with the exception rcPMP treatment through Day -1.

Study design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
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 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/04/2014
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
1/10/2022
Sample size
Target
Accrual to date
Final
8
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Monash Medical Centre - Melbourne
Recruitment postcode(s) [1] 0 0
- Melbourne
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Wisconsin
Country [2] 0 0
Netherlands
State/province [2] 0 0
Groningen
Country [3] 0 0
Tunisia
State/province [3] 0 0
Tunis
Country [4] 0 0
United Kingdom
State/province [4] 0 0
Glasgow
Country [5] 0 0
United Kingdom
State/province [5] 0 0
Manchester

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

Ethics approval
Ethics application status

Summary
Brief summary
This study will include a screening period, a 6-month treatment period, followed by long-term extension period expected to last approximately 72 months.
Trial website
https://clinicaltrials.gov/study/NCT02047461
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/NCT02047461