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Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kg (BMN270-302)

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: NCT03392974
Recruitment Status : Active, not recruiting
First Posted : January 8, 2018
Results First Posted : October 8, 2021
Last Update Posted : August 3, 2022
Sponsor:
Information provided by (Responsible Party):
BioMarin Pharmaceutical

Study Description
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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.

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 : 1 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official 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
Actual Study Start Date : March 14, 2018
Actual Primary Completion Date : May 22, 2019
Estimated Study Completion Date : May 2023

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Hemophilia

Arms and Interventions
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Arm Intervention/treatment
Experimental: Valoctocogene Roxaparvovec Open Label
Single administration of valoctocogene roxaparvovec at a dose of 4E13 vg/kg
 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 of the Median Factor VIII (FVIII) Activity [ Time Frame: Week 52 ]
    Change of the FVIII activity, as measured by chromogenic substrate assay, at Week 52 post-BMN 270 infusion.


Secondary Outcome Measures :
  1. Change in the Annualized Utilization (IU/kg) of Exogenous FVIII Replacement Therapy [ Time Frame: Weeks 5 through Week 52 ]
    Change in the annualized utilization (IU/kg) of exogenous FVIII replacement therapy during Week 5 to Week 52 post-BMN 270 infusion from the baseline utilization of exogenous FVIII replacement therapy

  2. Change in the Annualized Number of Bleeding Episodes Requiring Exogenous FVIII Replacement Treatment [ Time Frame: Weeks 5 though Week 52 ]
    Change in the annualized number of bleeding episodes requiring exogenous FVIII replacement treatment (annualized bleeding rate, ABR) during Week 5 to Week 52 of the study post-BMN 270 infusion from the baseline ABR


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.
  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).

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.
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): NCT03392974


Locations
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United States, Pennsylvania
Hemophilia Center of Western Pennsylvania
Pittsburgh, Pennsylvania, United States, 15213-4306
Sponsors and Collaborators
BioMarin Pharmaceutical
Investigators
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Study Director: Medical Director, MD BioMarin Pharmaceutical
  Study Documents (Full-Text)

Documents provided by BioMarin Pharmaceutical:
Study Protocol  [PDF] November 9, 2018
Statistical Analysis Plan  [PDF] February 17, 2021

More Information
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Responsible Party: BioMarin Pharmaceutical
ClinicalTrials.gov Identifier: NCT03392974    
Other Study ID Numbers: BMN 270-302
2017-003573-34 ( EudraCT Number )
First Posted: January 8, 2018    Key Record Dates
Results First Posted: October 8, 2021
Last Update Posted: August 3, 2022
Last Verified: August 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:
Hemophilia A
Gene Therapy
Clotting Disorders
Blood Disorder
Blood Coagulation Disorders
Inherited Blood Coagulation Disorders
Hematologic Diseases
 Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases
Inborn
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