Valoctocogene roxaparvovec-rvox

(270-205 [GENEr8-INH])

Trial Status Active, Not Recruiting

Condition

Hemophilia A

Technology

• Gene Therapy

Title

Safety, Tolerability, and Efficacy Study of Valoctocogene Roxaparvovec in Hemophilia A With Active or Prior Inhibitors

Study Description

This Phase 1/2 clinical study will evaluate the safety and efficacy of valoctocogene-roxaparvovec-rvox (BMN 270) in patients with severe hemophilia A and inhibitors to FVIII. Part A of the study will include subjects who have active inhibitors to FVIII, and Part B will include subjects with a prior history of inhibitors.

Primary Outcome Measure*

• Treatment-related adverse events over 60 months

Select Inclusion Criteria*

• Males ≥18 years of age with hemophilia A
• Documented prior residual FVIII activity ≤1 IU/dL

Secondary Outcome Measures*

• Change in median FVIII activity
• Change in FVIII inhibiter titer
• Absence of recurrence of FVIII inhibitors

Select Exclusion Criteria*

• Detectable pre-existing antibodies to the AAV5 capsid
• Active infection or immunosuppressive disorder (patients with HIV and undetectable viral load are not excluded)
• Receiving immune tolerance induction therapy or prophylaxis with FVIII (Part A only)

* Additional measures/criteria may apply.
AAV, adeno-associated virus; FVIII, factor VIII; HIV, human immunodeficiency virus.

For reference and comprehensive trial information, visit ClinicalTrials.gov NCT04684940

Pre-IND

Phase 1

Phase 2

Phase 3

Phase 4

Valoctocogene roxaparvovec-rvox

(270-304 [GENEr8-JPN)

Trial Status Active, Not Recruiting

Condition

Hemophilia A

Technology

• Gene Therapy

Title

Phase 3 Study for Efficacy and Safety Outcomes Data in Japanese Patients With Severe Hemophilia A (GENEr8-JPN)

Study Description

This Phase 3 clinical study will evaluate the safety and effectiveness of valoctocogene roxaparvovec in Japanese patients with severe hemophilia A.

Primary Outcome Measure*

• Change from baseline in FVIII activity

Select Inclusion Criteria*

• Japanese males ≥18 years of age with hemophilia A​
• Residual FVIII levels ≤1 IU/d​L

Secondary Outcome Measures*

• Change from baseline in utilization of FVIII replacement therapy​
• Change from baseline in number of bleeding episodes requiring FVIII replacement treatment​

Select Exclusion Criteria*

• Detectable pre-existing antibodies to the AAV5 capsid​
• Active infection or any immunosuppressive disorder, including HIV infection​
• Significant liver dysfunction​

* Additional measures/criteria may apply.
AAV, adeno-associated virus; FVIII, factor VIII; HIV, human immunodeficiency virus.

For reference and comprehensive trial information, visit ClinicalTrials.gov NCT06224907

Pre-IND

Phase 1

Phase 2

Phase 3

Phase 4

Valoctocogene roxaparvovec-rvox

(270-401 [GENEr8-LTE])

Trial Status Enrolling by Invitation

Condition

Hemophilia A

Technology

• Gene Therapy

Title

A Long-Term Follow-Up Study in Severe Hemophilia A Subjects Who Received BMN 270 in a Prior BioMarin Clinical Trial (270-401)

Study Description

The BMN 270 (valoctocogene-roxaparvovec-rvox) clinical development program consists of multiple interventional studies designed to assess the safety and efficacy of a single infusion of valoctocogene-roxaparvovec-rvox for at least 5 years post-infusion. This long-term follow-up study is needed to help further understand the long-term safety of valoctocogene-roxaparvovec-rvox beyond 5 years and to assess the durability of efficacy.

Primary Outcome Measure*

• Evaluate the long-term safety of valoctocogene-roxaparvovec-rvox

Select Inclusion Criteria*

• Subjects must have completed their primary treatment study
• Subjects must be capable of giving signed informed consent

Secondary Outcome Measures*

• Evaluate the long-term effects of valoctocogene-roxaparvovec-rvox in subjects with hemophilia A previously treated in a BioMarin clinical trial
• Evaluate the use of hemostatic agents

Select Exclusion Criteria*

• Subjects must enroll in Study 270-401 within 4 months of the date of that study completion visit
• Subjects must be healthy and not have a condition that would interfere with interpretation of the study

* Additional measures/criteria may apply.

For reference and comprehensive trial information, visit ClinicalTrials.gov NCT05768386

Pre-IND

Phase 1

Phase 2

Phase 3

Phase 4

Valoctocogene roxaparvovec-rvox

(270-601 [GENEr8-COAS])

Trial Status Not started

Condition

Hemophilia A

Technology

• Gene Therapy

Title

GENEr8-COAS: A Non-Interventional, Multi-National, Longitudinal Study of Patients Treated with Roctavian (valoctocogene roxaparvovec)

Study Description

This study is a non-interventional, multi national, longitudinal cohort study will describe the bleeding profile and long-term durability of coagulation factor VIII (FVIII) expression in subjects administered with Roctavian.

Select Inclusion Criteria*

• Diagnosed with HA and treated with Roctavian. Patients will be offered the opportunity to enroll in this study only after a clinical decision has been made that they will receive Roctavian, however, only subjects who are eventually administered Roctavian will be included in the analysis.

* Additional measures/criteria may apply.

For reference and comprehensive trial information, visit EMA.Europa.eu EUPAS49071

Pre-IND

Phase 1

Phase 2

Phase 3

Phase 4

Valoctocogene roxaparvovec-rvox

(270-801 [GENEr8-GTR])

Trial Status Recruiting

Condition

Hemophilia A

Technology

• Gene Therapy

Title

GENEr8-GTR: A Retrospective Cohort Study of Patients Treated with Roctavian(valoctocogene roxaparvovec): An Analysis of Patient Registries

Study Description

This study is being undertaken to better characterize the long-term effectiveness and safety outcomes of patients treated with Roctavian in a real-world setting

* Additional measures/criteria may apply.

For reference and comprehensive trial information, visit EMA.Europa.eu EUPAS49243

Pre-IND

Phase 1

Phase 2

Phase 3

Phase 4