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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT03392974




Registration number
NCT03392974
Ethics application status
Date submitted
22/12/2017
Date registered
8/01/2018
Date last updated
3/10/2023

Titles & IDs
Public title
Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kg
Scientific title
A Phase 3 Open-Label, Single-Arm Study To Evaluate The Efficacy and Safety of BMN 270, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII at a Dose of 4E13vg/kg in Hemophilia A Patients With Residual FVIII Levels =1IU/dL Receiving Prophylactic FVIII Infusions
Secondary ID [1] 0 0
2017-003573-34
Secondary ID [2] 0 0
270-302
Universal Trial Number (UTN)
Trial acronym
GENEr8-2
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Hemophilia A 0 0
Condition category
Condition code
Blood 0 0 0 0
Clotting disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Other interventions - Valoctocogene Roxaparvovec

Experimental: Valoctocogene Roxaparvovec Open Label - Single administration of valoctocogene roxaparvovec at a dose of 4E13 vg/kg


Other interventions: Valoctocogene Roxaparvovec
Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Hemophilia A
Intervention code [1] 0 0
Other interventions
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Change of the Median Factor VIII (FVIII) Activity
Timepoint [1] 0 0
Week 52
Secondary outcome [1] 0 0
Change in the Annualized Utilization (IU/kg) of Exogenous FVIII Replacement Therapy
Timepoint [1] 0 0
Weeks 5 through Week 52
Secondary outcome [2] 0 0
Change in the Annualized Number of Bleeding Episodes Requiring Exogenous FVIII Replacement Treatment
Timepoint [2] 0 0
Weeks 5 though Week 52

Eligibility
Key inclusion criteria
1. Males = 18 years of age with hemophilia A and residual FVIII levels = 1 IU/dL as
evidenced by medical history.

2. Must have been on prophylactic FVIII replacement therapy for at least 12 months prior
to study entry.

3. Treated/exposed to FVIII concentrates or cryoprecipitate for a minimum of 150 exposure
days.

4. No previous documented history of a detectable FVIII inhibitor of less than 0.6
Bethesda Units (BU).
Minimum age
18 Years
Maximum age
No limit
Sex
Males
Can healthy volunteers participate?
No
Key exclusion criteria
1. Detectable pre-existing antibodies to the AAV5 capsid.

2. Any evidence of active infection or any immunosuppressive disorder, including HIV
infection.

3. Significant liver dysfunction, prior liver biopsy showing significant fibrosis, liver
cirrhosis of any etiology or history of hepatic malignancy.

4. Evidence of any bleeding disorder not related to hemophilia A.

5. Active Hepatitis C.

6. Prior treatment with any vector/gene transfer agent.

Study design
Purpose of the study
Treatment
Allocation to intervention
N/A
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 3
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
14/03/2018
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
5/06/2023
Sample size
Target
Accrual to date
Final
1
Recruitment in Australia
Recruitment state(s)
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Pennsylvania

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
BioMarin Pharmaceutical
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
This Phase III clinical study will assess the efficacy of BMN 270 defined as FVIII activity,
during weeks 49-52 following intravenous infusion of BMN 270 and assess the impact of BMN 270
on usage of exogenous FVIII replacement therapy and the number of bleeding episodes from week
5 to week 52.
Trial website
https://clinicaltrials.gov/ct2/show/NCT03392974
Trial related presentations / publications
Public notes
This record is viewable in the ANZCTR as it had previously listed Australia and/or New Zealand as a recruitment site, however these sites have since been removed

Contacts
Principal investigator
Name 0 0
Medical Director, MD
Address 0 0
BioMarin Pharmaceutical
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/ct2/show/NCT03392974