| 
            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           | 
              
          
                  | 
            MB-CART2019.1           | 
                  
            A phase I/II safety, dose finding and feasibility trial of MB-CART2019.1 in patients with relapsed or resistant B-NHL           | 
                  
                  
            Humans           | 
                  
            CD19 and CD20 chimeric antigen receptor           | 
              
          
                  | 
            Only notified under the "contained use" procedure. Dossier submitted on 09/11/2001.           | 
                  
            A phase III, multi-center, open-label, randomized study to compare the overall survival and safety of bi-weekly intratumoral administration of INGN 201 versus weekly methotrewate in 240 patients with refractory squamous cell carcinoma of the head and neck           | 
                  
                  
            Humans           | 
                  
            Wild-type p53            | 
              
          
                  | 
            Only notified under the "contained use" procedure. Dossier submitted on 09/11/2001.           | 
                  
            A phase III, multi-center, open-label, randomized study to compare the effectiveness and safety of intratumoral administration of INGN 201 in combination with chemotherapy versus chemotherapy alone in 288 patients with recurrent squamous cell carcinoma of           | 
                  
                  
            Humans           | 
                  
            Wild-type p53            |