B/BE/19/BVW2 - Summary Notification Information Format (SNIF) Public information presented according to the provisions of Directive 2001/18/EC
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
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