NCT03392974

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 Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
1

participants targeted

Target at below P25 for phase_3

Timeline
Completed

Started Mar 2018

Longer than P75 for phase_3

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

December 22, 2017

Completed
17 days until next milestone

First Posted

Study publicly available on registry

January 8, 2018

Completed
2 months until next milestone

Study Start

First participant enrolled

March 14, 2018

Completed
1.2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

May 22, 2019

Completed
2.4 years until next milestone

Results Posted

Study results publicly available

October 8, 2021

Completed
1.7 years until next milestone

Study Completion

Last participant's last visit for all outcomes

June 5, 2023

Completed
Last Updated

October 3, 2023

Status Verified

September 1, 2023

Enrollment Period

1.2 years

First QC Date

December 22, 2017

Results QC Date

August 9, 2021

Last Update Submit

September 26, 2023

Conditions

Hemophilia A

Keywords

Hemophilia AGene TherapyClotting DisordersBlood DisorderBlood Coagulation DisordersInherited Blood Coagulation DisordersHematologic DiseasesCoagulation Protein DisordersHemorrhagic DisordersGenetic DiseasesInbornFactor VIIICoagulants

Outcome Measures

Primary Outcomes (1)

  • Change of the Median Factor VIII (FVIII) Activity

    Change of the FVIII activity, as measured by chromogenic substrate assay, at Week 52 post-BMN 270 infusion.

    Week 52

Secondary Outcomes (2)

  • Change in the Annualized Utilization (IU/kg) of Exogenous FVIII Replacement Therapy

    Weeks 5 through Week 52

  • Change in the Annualized Number of Bleeding Episodes Requiring Exogenous FVIII Replacement Treatment

    Weeks 5 though Week 52

Study Arms (1)

Valoctocogene Roxaparvovec Open Label

EXPERIMENTAL

Single administration of valoctocogene roxaparvovec at a dose of 4E13 vg/kg

Biological: Valoctocogene Roxaparvovec

Interventions

Valoctocogene RoxaparvovecBIOLOGICAL

Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Hemophilia A

Also known as: BMN 270
Valoctocogene Roxaparvovec Open Label

Eligibility Criteria

Age18 Years+
Sexmale(Gender-based eligibility)
Gender Eligibility DetailsBiological males only
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Males ≥ 18 years of age with hemophilia A and residual FVIII levels ≤ 1 IU/dL as evidenced by medical history.
  • Must have been on prophylactic FVIII replacement therapy for at least 12 months prior to study entry.
  • Treated/exposed to FVIII concentrates or cryoprecipitate for a minimum of 150 exposure days.
  • No previous documented history of a detectable FVIII inhibitor of less than 0.6 Bethesda Units (BU).

You may not qualify if:

  • Detectable pre-existing antibodies to the AAV5 capsid.
  • Any evidence of active infection or any immunosuppressive disorder, including HIV infection.
  • Significant liver dysfunction, prior liver biopsy showing significant fibrosis, liver cirrhosis of any etiology or history of hepatic malignancy.
  • Evidence of any bleeding disorder not related to hemophilia A.
  • Active Hepatitis C.
  • Prior treatment with any vector/gene transfer agent.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Hemophilia Center of Western Pennsylvania

Pittsburgh, Pennsylvania, 15213-4306, United States

Location

MeSH Terms

Conditions

Hemophilia AHemostatic DisordersHematologic DiseasesBlood Coagulation DisordersBlood Coagulation Disorders, InheritedCoagulation Protein DisordersHemorrhagic DisordersGenetic Diseases, Inborn

Interventions

Valoctocogene Roxaparvovec

Condition Hierarchy (Ancestors)

Hemic and Lymphatic DiseasesCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesVascular DiseasesCardiovascular Diseases

Results Point of Contact

Title
Medical Manager, Clinical Science
Organization
BioMarin Pharmaceutical Inc.
divya.reddy@bmrn.com
415-996-2917

Study Officials

  • Medical Director, MD

    BioMarin Pharmaceutical

    STUDY DIRECTOR

Publication Agreements

PI is Sponsor Employee
No
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 3
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

December 22, 2017

First Posted

January 8, 2018

Study Start

March 14, 2018

Primary Completion

May 22, 2019

Study Completion

June 5, 2023

Last Updated

October 3, 2023

Results First Posted

October 8, 2021

Record last verified: 2023-09

Locations

US(1)