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 in Hemophilia A Patients with Residual FVIII Levels Receiving Prophylactic FVIII

Study 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 in Hemophilia A Patients with Residual FVIII Levels Receiving Prophylactic FVIII
Long title: 
The dossier concerns a clinical trial involving two protocols, entitled “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 in Hemophilia A Patients with Residual FVIII Levels ≤ 1 IU/dL Receiving Prophylactic FVIII Infusions” (Protocol number: 270-301) and “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 4E13 vg/kg in Hemophilia A Patients with Residual FVIII Levels ≤ 1 IU/dL Receiving Prophylactic FVIII Infusions” (Protocol number: 270-302), respectively.
Date receipt dossier: 
22 feb 2019
EU record number: 
B/BE/19/BVW2
EudraCT number: 
2017-003573-34
Company / Sponsor: 
BioMarin Pharmaceutical Inc.
Phase: 
III
Treated organism: 
Humans
Indication category: 
Genetic disorders
Disease: 
Hemophilia A
Therapeutic approach: 
Gene therapy
Genetic modification: 
Recombinant adeno-associated virus serotype 5 (AAV5) containing a gene cassette expressing the human coagulation factor VIII (FVIII) gene
Method of transfer of nucleic acid of interest: 
Non-replicating, non-integrating recombinant adeno-associated virus serotype 5 (AAV5)
Administered biological material: 
Recombinant virus
Route of administration: 
Intravenous
Locations in Belgium: 
Universitair Ziekenhuis, Leuven - Cliniques Universitaires Saint-Luc, Bruxelles - Universitair Ziekenhuis, Antwerpen
Nr of subjects: 
A maximum of 7 patients will be treated in Belgium in Study 270-301, and up to 3 patients will be treated in Study 270-302
Foreseen duration: 
5 years starting 31/05/2019
Type of procedure: 
Contained use and Deliberate release
Current status: 
Assessed

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