Search the database for deliberate release of GM medicinal products

Displaying 1 - 11 of 11

EU record number	Title	Company / Sponsor	Treated organism	Genetic modification
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)	Pfizer	Humans	Recombinant adeno associated viral vector containing the codon-optimised human factor IX (FIX) minigene (R338L variant)
B/BE/21/BVW2	Phase I study to assess safety, tolerability and immunogenicity of the hRVFV-4s vaccine in healthy subjects	Wageningen Bioveterinary Research (Larissa Consortium)	Humans	hRVFV-4s is a four segments human Rift Valley fever virus vaccine, which derives from a natural clone (Clone 13) that lacks 69% of the gene NSs, a major virulence determinant of the virus. The hRVFV-4s vaccine is further attenuated by splitting the M genome segment into two M-type segments.
B/BE/20/BVW6	Dossier withdrawn by the notifier	Pfizer	Humans	Non-replicating recombinant vector derived from adeno-associated virus AAV6 serotype, containing a codon-optimised version of the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	Pfizer	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
bb2121-MM-001	A phase II, multicenter study to determine the efficacy and safety of bb2121 in subjects with relapsed and refractory multiple myeloma	Celgene	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	Celgene	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)	Celgene	Humans	BCMA02 (human B cell maturation antigen) -Chimeric antigen receptor
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	Institut de Recherches Internationales Servier	Humans	chimeric antigen receptor and suicide-ligand (RQR8)
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	Celgene	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	Novartis Pharma Services AG	Humans	Chimeric antigen receptor against CD19
Only notified under the "contained use" procedure. Dossier submitted on 20/08/2015.	A single arm Phase I/II study of the safety and efficacy of gene-modified WT1 TCR therapy in patients with myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML)	Catapult Cell Therapy	Humans	Expression of the Wilms' tumour antigen 1 (WT1)- specific T cell receptor (TCR)