Study to Evaluate the Efficacy and Safety of Valoctocogene Roxaparvovec, With Prophylactic Steroids in Hemophilia A

NCTID	NCT04323098 (View at clinicaltrials.gov)
Description	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. (Show More)
Development Status	Approved
Indication	Hemophilia A
Disease Ontology Term	DOID:12134
Compound Name	ROCTAVIAN
Compound Alias	valoctogene roxaparvovec
Sponsor	BioMarin Pharmaceutical
Funder Type	Industry
Recruitment Status	Active not recruiting
Enrollment Count	22
Results Posted	View Results

Therapy Information

Target Gene/Variant	F8
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intravenous
Drug Product Type	Viral vector
Target Tissue/Cell	Hepatocyte
Delivery System	Viral transduction
Vector Type	AAV5
Editor Type	none
Dose 1	6E13 vg/kg
Dose 2
Dose 3
Dose 4
Dose 5

Study Record Dates

Current Stage	Phase3
Submit Date	2020-03-24
Completion Date	2027-01
Last Update	2024-04-04

Participation Criteria

Eligible Age	>=18 Years
Standard Ages	Adult, Older adult
Sexes Eligible for Study	MALE

Locations

No.of Trial Sites	12
Locations	United States,Taiwan,Brazil,Australia

Regulatory Information

Has US IND	True
FDA Designations	Breakthrough Therapy, Fast Track, Orphan Drug Designation, Regenerative Medicine Advanced Therapy
Recent Updates	FDA approved 6/29/23, price/treatment $2.9M

Resources/Links

Clinical Publications

Matching-adjusted indirect comparison of bleeding outcomes in severe haemophilia A: Comparing valoctocogene roxaparvovec gene therapy, emicizumab prophylaxis, and FVIII replacement prophylaxis
Safety and efficacy of valoctocogene roxaparvovec with prophylactic glucocorticoids: 1-year results from the phase 3b, single-arm, open-label GENEr8-3 study
Two-Year Outcomes of Valoctocogene Roxaparvovec Therapy for Hemophilia A
Outcomes and management of invasive procedures in participants with hemophilia A post gene therapy: a post hoc analysis of the GENEr8-1 phase III trial
Interindividual variability in transgene mRNA and protein production following adeno-associated virus gene therapy for hemophilia A
Valoctocogene Roxaparvovec Gene Therapy for Hemophilia A
Persistence of haemostatic response following gene therapy with valoctocogene roxaparvovec in severe haemophilia A
Early Phase Clinical Immunogenicity of Valoctocogene Roxaparvovec, an AAV5-Mediated Gene Therapy for Hemophilia A
Evaluation of one-stage and chromogenic assays for the laboratory measurement of factor VIII activity following valoctocogene roxaparvovec infusion
Blood biodistribution and vector shedding of valoctocogene roxaparvovec in people with severe hemophilia A
Valoctocogene roxaparvovec gene therapy provides durable haemostatic control for up to 7 years for haemophilia A
Activity of transgene-produced B-domain-deleted factor VIII in human plasma following AAV5 gene therapy
Clinical immunogenicity outcomes from GENEr8-1, a phase 3 study of valoctocogene roxaparvovec, an AAV5-vectored gene therapy for hemophilia A
Efficacy, safety, and quality of life 4 years after valoctocogene roxaparvovec gene transfer for severe hemophilia A in the phase 3 GENEr8-1 trial
Three-year outcomes of valoctocogene roxaparvovec gene therapy for hemophilia A
Comparative Effectiveness of Valoctocogene Roxaparvovec and Prophylactic Factor VIII Replacement in Severe Hemophilia A
Long-term safety and efficacy outcomes of valoctocogene roxaparvovec gene transfer up to 6 years post-treatment
Health-related quality of life following valoctocogene roxaparvovec gene therapy for severe hemophilia A in the phase 3 trial GENEr8-1