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Study to Evaluate the Efficacy and Safety of Valoctocogene Roxaparvovec, With Prophylactic Steroids in Hemophilia A (GENEr8-3)

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 evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04323098
Recruitment Status : Active, not recruiting
First Posted : March 26, 2020
Last Update Posted : October 14, 2022
Sponsor:
Information provided by (Responsible Party):
BioMarin Pharmaceutical

Study Description
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Brief Summary:
This Phase III clinical study will evaluate the safety and effectiveness of valoctocogene roxaparvovec in combination with prophylactic corticosteroids in patients with severe hemophilia A.

Condition or disease Intervention/treatment Phase
Hemophilia A Biological: valoctocogene roxaparvovec Phase 3

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 22 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official 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
Actual Study Start Date : November 10, 2020
Estimated Primary Completion Date : January 2023
Estimated Study Completion Date : January 2027

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Hemophilia Steroids

Arms and Interventions
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Arm Intervention/treatment
Experimental: valoctocogene roxaparvovec
Single administration of valoctocogene roxaparvovec at a dose of 6E13 vg/kg with prophylactic corticosteroids
 Biological: valoctocogene roxaparvovec
Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Hemophilia A
Other Name: BMN 270


Outcome Measures
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Primary Outcome Measures :
  1. Change in median FVIII activity as measured by chromogenic substrate assay, after administration of BMN 270 with prophylactic corticosteroids [ Time Frame: 52 weeks ]

Secondary Outcome Measures :
  1. Change in the annualized utilization (IU/kg) of exogenous FVIII replacement therapy or emicizumab for subjects receiving FVIII or emicizumab prophylaxis respectively after administration of BMN 270 with prophylactic corticosteroids [ Time Frame: 52 weeks ]
  2. Change in the annualized number of bleeding episodes requiring exogenous FVIII replacement treatment after administration of BMN 270 with prophylactic corticosteroids [ Time Frame: 52 weeks ]
  3. Impact of BMN 270 with prophylactic corticosteroids on quality of life as measured by the Haemo-QoL-A questionnaire [ Time Frame: 52 weeks ]
    Haemo-QoL-A is a hemophilia-specific, health-related quality of life questionnaire for adults, which uses a scale from 0 (none of the time) to 5 (All of the time) to report data.


Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   Male
Gender Based Eligibility:   Yes
Gender Eligibility Description:   Biological males only
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. 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.
  2. Must have been on prophylactic hemophilia 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 (EDs).
  4. No previous documented history of a detectable FVIII inhibitor, <0.6 Bethesda Units (BU).
  5. Sexually active participants must agree to use an acceptable method of effective contraception. Participants must agree to contraception use for at least 12 weeks post-infusion.

Exclusion Criteria:

  1. Detectable pre-existing antibodies to the AAV5 capsid. Up to 25% of subjects may have detectable pre-existing AAV5 capsid antibodies, so long as the titer level is below the minimum required dilution (< 20).
  2. Any evidence of active infection or any immunosuppressive disorder; patients with HIV infection and undetectable viral load are not excluded.
  3. Significant renal dysfunction or liver dysfunction, infection or history of hepatic malignancy.
  4. Evidence of any bleeding disorder not related to hemophilia A.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04323098


Locations
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United States, Missouri
Washington University School of Medicine
Saint Louis, Missouri, United States, 63110
United States, Ohio
Nationwide Children's Hospital
Columbus, Ohio, United States, 43205
Australia
The Royal Adelaide Hospital
Adelaide, Australia
Royal Brisbane and Women's Hospital
Brisbane, Australia
Alfred Hospital
Melbourne, Australia
Fiona Stanley Hospital
Perth, Australia
Royal Prince Alfred Hospital
Sydney, Australia
Brazil
Campinas University Clinical Hospital
Campinas, Brazil
Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo
São Paulo, Brazil
Taiwan
Changhua Christian Medical Foundation Changhua Christian Hospital
Changhua, Taiwan
Taichung Veterans General Hospital
Taichung, Taiwan
Sponsors and Collaborators
BioMarin Pharmaceutical
Investigators
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Study Director: Medical Monitor, MD BioMarin Pharmaceutical
More Information
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Responsible Party: BioMarin Pharmaceutical
ClinicalTrials.gov Identifier: NCT04323098    
Other Study ID Numbers: BMN 270-303
2018-004616-21 ( EudraCT Number )
First Posted: March 26, 2020    Key Record Dates
Last Update Posted: October 14, 2022
Last Verified: October 2022

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by BioMarin Pharmaceutical:
Gene Therapy
Clotting Disorders
Blood Disorder
Blood Coagulation Disorders
Inherited Blood Coagulation Disorders
Hematologic Diseases
 Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases
Factor VIII
Coagulants
Additional relevant MeSH terms:
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Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
 Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn