| 
            B/BE/19/BVW2           | 
                  
            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            | 
                  
                  
            Humans           | 
                  
            Recombinant adeno-associated virus serotype 5 (AAV5) containing a gene cassette expressing the human coagulation factor VIII (FVIII) gene           | 
              
          
                  | 
            Only notified under the "contained use" procedure. Dossier submitted on 21/02/2018.           | 
                  
            Efficacy and safety of bilateral intravitreal injection of GS010: a randomized, double-masked, placebo-controlled trial in subjects affected with G11778A ND4 leber hereditary optic neuropathy for up to one year.           | 
                  
                  
            Humans           | 
                  
            human wild-type MT-NDA (mitochondrial NADH Dehydrogenase 4)           | 
              
          
                  | 
            Only notified under the "contained use" procedure. Dossier submitted on 11/07/2002.           | 
                  
            A European and Canadian multicenter, randomized, double-blind, placebo controlled study to evaluate the efficacy and safety of Ad5FGF-4 in patients with stable angina           | 
                  
                  
            Humans           | 
                  
            human FGF-4            |