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) |
B/BE/22/BVW4 |
A Phase I/II, Multicenter, Open-Label Study of Nous-209 Genetic Vaccine for the Treatment of Microsatellite Unstable Solid Tumors |
Humans |
The study involves two GMOs: (i) A replication-incompetent adenovirus (GAd20 with deletions of the viral E1, E3 and E4 coding regions) isolated from a gorilla and encoding the FSP neoantigens; (ii) an attenuated, replication-defective orthopoxvirus (Modified Vaccinia virus Ankara), encoding for the same neoantigens. |
Only notified under the "contained use" procedure. Dossier submitted on 25/07/2022. |
A Phase I/II study to evaluate the feasibility, safety and preliminary efficacy of point-of-care manufactured anti-CD19 CAR T in subjects with relapsed or refractory Chronic Lymphocytic Leukemia or Small Lymphocytic Lymphoma (SLL) |
Humans |
CD19 CAR |
Only notified under the "contained use" procedure. Dossier submitted on 07/06/2022. |
A phase I/II open label, multicenter study evaluating the feasibility, safety and efficacy of point-of-care manufactured anti-BCMA CAR T cells (BCMACP03) in subjects with relapsed/refractory Multiple Myeloma (r/r MM) (Papilio-1) |
Humans |
BCMA CAR |
CP0201-NHL |
A Phase I/II, multicenter study, evaluating the feasibility, safety, and efficacy of point-of-care manufactured 19CP02 in subjects with relapsed/refractory B-cell non-Hodglin lymphoma |
Humans |
CD19-CAR |
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Dossier withdrawn by the notifier |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/BVW5 |
Phase 1b Study to Evaluate the Safety, Tolerability, and Anti-Tumor Activity of Nous-PEV, with pembrolizumab, in Patients with Unresectable Stage III / IV Cutaneous Melanoma and with Stage IV NSCLC |
Humans |
The study involves two GMOs: (i) A replication-incompetent adenovirus (GAd20 with deletions of the viral E1, E3 and E4 coding regions) isolated from a gorilla and encoding the PEV neoantigens gene; (ii) an attenuated, replication-defective orthopoxvirus (Modified Vaccinia virus Ankara), encoding for the neoantigens 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 |
ADP-0055-001 |
A phase I, ascending dose, to evaluate safety and efficacy of ADP A2M4CD8 in HLA-A2+ patients with MAGE-A4 psoitives tumors |
Humans |
MAGE-A4 specific T cell receptor (TCR) with a CD8α co-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 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 |
B/BE/07/BVW3 |
A multi-centre phase I study to evaluate the safety and tolerability of a heterologous prime-boost vaccination with INX102-3697 HBV pDNA/INX102-0557 HBV MVA in healthy volunteers and HBeAg+ chronic hepatitis patients |
Humans |
Hepatitis B virus polyepitope gene |
Only notified under the "contained use" procedure. Dossier submitted on 02/01/1996. |
Gene therapy for the treatment of glioblastoma multiforme with in vivo tumor transduction with the herpes simplex thymidine kinase gene /ganciclovir system |
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Thymidine Kinase (HSV-TK1), neomycin resistance (NeoR) |