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 01/03/2024. |
A Phase 1, Multicenter, Single-arm, Dose--escalation Study of CC 97540 (BMS-986353), CD19-Targeted NEX-T Chimeric Antigen Receptor (CAR) T Cells, Evaluating Safety and Tolerability in Participants with Relapsing Forms of Multiple Sclerosis (RMS) or Progre |
Humans |
CD19 CAR |
Only notified under the "contained use" procedure. Dossier submitted on 22/09/2023. |
A Randomized, Open-Label, Phase 3 Trial to Compare the Efficacy and Safety of Idecabtagene Vicleucel (Ide-Cel) with Lenalidomide (LEN) Maintenance Versus Lenalidomide Maintenance Therapy Alone in Adult Participants with Newly Diagnosed Multiple Myeloma (N |
Humans |
BCMA CAR |
Only notified under the "contained use" procedure. Dossier submitted on 18/09/2023. |
A Phase 1, Multicenter, Open-Label Study Of CC-97540 (BMS 986353), CD19-Targeted NEX-T Chimeric Antigen Receptor (CAR) T Cells, in Participants with Severe, Refractory Systemic Lupus Erythematosus (SLE) |
Humans |
CD19 CAR |
B/BE/21/BVW4 |
An Open-Label, Multicenter, Non-Randomized, Dose-Confirmation and Cohort-Expansion Phase 1b Study to Evaluate the Safety, Tolerability, and Anti-Tumor Activity of ATP128, VSV-GP128 and BI 754091, in Patients with Stage IV Colorectal Cancer |
Humans |
VSV-GP128 is a recombinant live-attenuated Vesicular Stomatitis Virus (VSV), modified to minimize the potential of neurotoxicity for humans and skin disease in animals, and engineered to contain cancer antigens, which help it induce an immune response against colorectal tumour cells. |
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Dossier withdrawn by the notifier |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 |
B/BE/18/BVW7 |
Phase III trial investigating a AAV vector containing a variant of human factor IX gene administered to adult subjects with severe or moderately-severe hemophilia B |
Humans |
Recombinant adeno associated viral vector containing the wild type modified to express the Padua derivative of human coagulation factor IX cDNA |
bb2121-MM-001 |
A phase II, multicenter study to determine the efficacy and safety of bb2121 in subjects with relapsed and refractory multiple myeloma |
Humans |
BCMA02 (human B cell maturation antigen)-CAR |
Only notified under the "contained use" procedure. Dossier submitted on 24/04/2018. |
A phase 2, single-arm, multi-cohort, multi-center trial to determine the efficacy and safety of jcar017 in adult subjects with aggressive b-cell non-hodgkin lymphoma |
Humans |
EGFRt en chimeric antigen receptor against CD19 |
68284528MMY2001 |
A phase III, multicenter, randomized, open-label study to compare the efficacy and safety of bb2121 versus standard triplet regimens in subjects with relapsed and refractory multiple myeloma (RRMM) (KarMMa-3) |
Humans |
BCMA02 (human B cell maturation antigen) -Chimeric antigen receptor |
Only notified under the "contained use" procedure. Dossier submitted on 23/11/2016. |
A phase 2, open-label, multi-cohort, single-arm, multi-center trial to determine the safety, feasibility, and efficacy of JCAR015 in adult subjects with B-cell acute lymphoblastic leukemia |
Humans |
Chimeric antigen receptor against CD19 |
Only notified under the "contained use" procedure. Dossier submitted on 13/10/2000. |
A phase III open-label, comparative, multicentre trials to test the concept of durable virologic suppression in subjects with primary HIV-1 infection after intensive induction of quadruple HAART followed by double-blind randomization to HIV vaccination wi |
Humans |
vCP-1452 |
Only notified under the "contained use" procedure. Dossier submitted on 26/03/1998. |
Pilot study of immunization with recombinant canarypox virus vCP1469A expressing the MAGE-1.A1 and MAGE-3.A1 cytolytic T lymphocytes epitopes in patients with malignant melanoma, non-small cell lung carcinoma, head-and-neck squamous cell carcinoma, esopha |
Humans |
HLA-A1 restricted CTL epitope of MAGE-1 and
MAGE-3 genes |