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
Information for the public
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Public information presented according to the provisions of Directive 2001/18/EC
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Information related to the decision procedure
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24 May 2019 - The Biosafety Advisory Council issues a positive advice (with conditions) for this trial
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10 July 2019: The Federal Ministers give a final decision (positive with conditions) for this trial