COVID-19 studies are our top priority. For all other trials, there is a 4-week delay in processing a trial submitted/resubmitted to the ANZCTR and additional delays for updates of registered trials. We appreciate your patience.

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this information for consumers
Trial details imported from

For full trial details, please see the original record at

Registration number
Ethics application status
Date submitted
Date registered
Date last updated

Titles & IDs
Public title
Study to Evaluate the Efficacy and Safety of Valoctocogene Roxaparvovec, With Prophylactic Steroids in Hemophilia A
Scientific title
A Phase 3b, Single Arm, Open-Label Study to Evaluate the Efficacy and Safety of BMN 270, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII, With Prophylactic Corticosteroids in Hemophilia A Patients
Secondary ID [1] 0 0
Secondary ID [2] 0 0
BMN 270-303
Universal Trial Number (UTN)
Trial acronym
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

Study type
Description of intervention(s) / exposure
Other interventions - valoctocogene roxaparvovec

Experimental: valoctocogene roxaparvovec - Single administration of valoctocogene roxaparvovec at a dose of 6E13 vg/kg with prophylactic corticosteroids

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

Primary outcome [1] 0 0
Change of the median Factor VIII (FVIII) activity - Changes in the median Factor VIII activity (IU/mL) will be measured using the chromogenic FVIII assay
Timepoint [1] 0 0
52 weeks
Secondary outcome [1] 0 0
Change in the annualized utilization (IU/kg) of exogenous FVIII replacement therapy or emicizumab
Timepoint [1] 0 0
52 weeks
Secondary outcome [2] 0 0
Change in the annualized number of bleeding episodes requiring exogenous FVIII replacement treatment or emicizumab
Timepoint [2] 0 0
52 weeks
Secondary outcome [3] 0 0
Assess the impact of valoctocogene roxaparvovec following Patient Reported Outcome (PRO) Haemo-QoL-A - Haemo-QoL-A is a hemophilia-specific, health-related quality of life questionnaire for adults on a scale of 0-5 with the lower value representing a better outcome
Timepoint [3] 0 0
52 weeks

Key inclusion criteria
- Males = 18 years of age with hemophilia A and residual FVIII levels = 1 IU/dL as
evidenced by medical history, at the time of signing the informed consent.

- Must have been on prophylactic hemophilia therapy for at least 12 months prior to
study entry. High-quality, well-documented historical data concerning bleeding
episodes and hemophilia therapy over the previous 12 months must be available.

- Treated/exposed to FVIII concentrates or cryoprecipitate for a minimum of 150 exposure
days (EDs).

- No previous documented history of a detectable FVIII inhibitor, and results from a
Bethesda assay or Bethesda assay with Nijmegen modification of less than 0.6 Bethesda
Units (BU) (or less than 1.0 BU for laboratories with a historical lower sensitivity
cutoff for inhibitor detection of 1.0 BU) on 2 consecutive occasions at least one week
apart within the past 12 months (at least one of which should be tested at the central
Minimum age
18 Years
Maximum age
No limit
Can healthy volunteers participate?
Key exclusion criteria
- Detectable pre-existing antibodies to the AAV5 capsid.

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

- Significant liver dysfunction with any of the following abnormal laboratory results:

- ALT (alanine aminotransferase) > 1.25x ULN;

- AST (aspartate aminotransferase) > 1.25x ULN;

- GGT (gamma-glutamyltransferase) > 1.25x ULN;

- Total bilirubin > 1.25x ULN;

- Alkaline phosphatase > 1.25x ULN

- INR (international normalized ratio) = 1.4. Subjects whose liver laboratory
assessments fall outside of these ranges may undergo repeat testing of the entire
liver test panel within the same Screening window and, if eligibility criteria
are met on retest, may be enrolled after confirmation by the Medical Monitor.

- Most recent, prior FibroScan or liver biopsy showing significant fibrosis of 3 or 4 as
rated on a scale of 0 4 on the Batts Ludwig or METAVIR scoring systems, or an
equivalent grade of fibrosis if an alternative scale is used.

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

- Platelet count of < 100 x 10^9/L.

- Creatinine = 1.5 mg/dL.

- Liver cirrhosis of any etiology as assessed by liver ultrasound.

- Chronic or active hepatitis B as evidenced by positive serology testing (hepatitis B
surface antigen [HBsAg], hepatitis B surface antibody [HBsAb], and hepatitis B core
antibody [HBcAb]) and confirmatory HBV DNA testing. Refer to the Centers for Disease
Control (CDC) table for the interpretation of serological test results.

- Active Hepatitis C as evidenced by detectable HCV RNA or currently on antiviral

- Active malignancy, except non-melanoma skin cancer.

- History of hepatic malignancy.

- History of arterial or venous thromboembolic events (eg, deep vein thrombosis,
nonhemorrhagic stroke, pulmonary embolism, myocardial infarction, arterial embolus),
with the exception of catheter-associated thrombosis for which anti-thrombotic
treatment is not currently ongoing.

- Known inherited or acquired thrombophilia, including conditions associated with
increased thromboembolic risk, such as atrial fibrillation.

Study design
Purpose of the study
Allocation to intervention
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 3
Type of endpoint(s)
Statistical methods / analysis

Recruitment status
Not yet recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Date of last participant enrolment
Date of last data collection
Sample size
Accrual to date
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
The Royal Adelaide Hospital (RAH) - Adelaide
Recruitment hospital [2] 0 0
Royal Brisbane and Women's Hospital - Brisbane
Recruitment hospital [3] 0 0
Alfred Hospital - Melbourne
Recruitment hospital [4] 0 0
Fiona Stanley Hospital - Perth
Recruitment hospital [5] 0 0
Royal Prince Alfred Hospital - Sydney
Recruitment postcode(s) [1] 0 0
- Adelaide
Recruitment postcode(s) [2] 0 0
- Brisbane
Recruitment postcode(s) [3] 0 0
- Melbourne
Recruitment postcode(s) [4] 0 0
- Perth
Recruitment postcode(s) [5] 0 0
- Sydney
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Country [2] 0 0
United States of America
State/province [2] 0 0
Country [3] 0 0
United States of America
State/province [3] 0 0
Country [4] 0 0
United States of America
State/province [4] 0 0
Country [5] 0 0
State/province [5] 0 0
Country [6] 0 0
State/province [6] 0 0
São Paulo
Country [7] 0 0
State/province [7] 0 0

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

Ethics approval
Ethics application status

Brief summary
This research study will test and confirm the safety and effectiveness of the study drug
(valoctocogene roxaparvovec) that contains the correct gene to make Factor VIII so that the
body can make its own Factor VIII that functions properly. Only one dose of valoctocogene
roxaparvovec is being given in this study, and this dose has been previously studied in
another clinical trial in patients with hemophilia A. This is a phase 3 study that is meant
to show that the study drug with prophylactic corticosteroids is safe and works to help treat
hemophilia A. The study will see if liver cells are able to make Factor VIII that functions
properly after receiving this study drug. The study will also examine the effects that the
study drug has on how much Factor VIII concentrates patients have to inject into their veins
and on their bleeding episodes after the study drug has been administered. Finally, the study
will see if and how the body responds to the study drug - for example, whether liver cells
become inflamed or whether the body makes antibodies (something the immune system makes to
protect itself against things like bacteria and viruses) against the vector or the new Factor
VIII gene.
Trial website
Trial related presentations / publications
Public notes

Principal investigator
Name 0 0
Medical Monitor, 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
Trial Specialist
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