| 
            B/BE/18/BVW6           | 
                  
            Global study of a recombinant adeno-associated virus designed to address the monogenic root cause of Spinal Muscular Atrophy           | 
                  
                  
            Humans           | 
                  
            Recombinant adeno-associated virus serotype 9 (AAV9) containing the cDNA of the human SMN (Survival Motor Neuron) gene           | 
              
          
                  | 
            Only notified under the "contained use" procedure. Dossier submitted on 24/04/2017.           | 
                  
            A Phase 1, Open Label, Non-comparative Study to Evaluate the Safety and the Ability of UCART19 to Induce Molecular Remission in Paediatric Patients With Relapsed/Refractory B Acute Lymphoblastic Leukaemia           | 
                  
                  
            Humans           | 
                  
            chimeric antigen receptor and suicide-ligand (RQR8)           | 
              
          
                  | 
            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            |