B/BE/24/BVW5 |
Phase 3, open-label, single-arm study to evaluate efficacy and safety of FIX gene transfer with PF-06838435 (rAAV-Spark100-hFIX-R338L) in adult male participants with moderately severe to severe hemophilia B (FIX:C ≤2%) (BeneGene-2) |
Humans |
Recombinant adeno associated viral vector containing the codon-optimised human factor IX (FIX) minigene (R338L variant) |
Only notified under the "contained use" procedure. Dossier submitted on 03/05/2021. |
Administration of genetically engineered T cells (KTE-X19) in Pediatric and Adolescent Subjects with Relapsed/Refractory B-precursor Acute Lymphoblastic Leukemia or Relapsed/Refractory B-cell Non-Hodgkin Lymphoma |
Humans |
CD19-CAR |
B/BE/20/BVW6 |
Dossier withdrawn by the notifier |
Humans |
Non-replicating recombinant vector derived from adeno-associated virus AAV6 serotype, containing a codon-optimised version of the human Factor VIII gene |
B/BE/20/BVW4 |
A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy |
Humans |
Non-replicating recombinant vector derived from adeno-associated virus AAV9, lacking all AAV viral genes and containing a miniaturized version of the human dystrophin 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 25/04/2018. |
A Phase 3, Randomized, Open-Label Study Evaluating the Efficacy of Axicabtagene Ciloleucel versus Standard of Care Therapy in Subjects with Relapsed/Refractory Diffuse Large B Cell Lymphoma (ZUMA-7) |
Humans |
Chimeric antigen receptor against CD19 |
Only notified under the "contained use" procedure. Dossier submitted on 24/04/2018. |
A Phase 2a Randomized, Double-blind, Placebo-controlled rial to Assess the safety, Immunogenicity, and Efficacy of the Recombinant MVA-BN-RSV Vaccine against Respiratory Syncytial Virus Infection in the Virus Challenge Model in Healthy Adult Volunteers |
Humans |
Vaccinia virus Ankara strain expression glycoproteins of different RSV strain (MVA-BN) |
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 14/10/2015. |
A phase II, single-arm, multi-center trial to determine the efficacy and safety of CTL019 in pediatric patients with relapsed and refractory B-cell acute lymphoblastic leukemia |
Humans |
Chimeric antigen receptor against CD19 |
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 |