B/BE/22/BVW6
A phase 3, Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of SRP-9001 in Non-Ambulatory and Ambulatory Subjects With Duchenne Muscular Dystrophy (ENVISION) Sarepta Therapeutics
Humans
Non-replicating recombinant vector derived from adeno-associated virus AAV, serotype rh74, lacking all AAV viral genes and carrying the human micro-dystrophin (hMicro-Dys) gene
B/BE/22/BVW5
A two-part, open-label systemic gene delivery study to evaluate the safety and expression of RO7494222 (SRP-9001) in subjects under the age of four with Duchennne Muscular Dystrophy (ENVOL) F. Hoffmann-La Roche
Humans
Non-replicating recombinant vector derived from adeno-associated virus AAV, serotype rh74, lacking all AAV viral genes and carrying the human micro-dystrophin (hMicro-Dys) gene
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 Nouscom Srl
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 2 September, 2022
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A single-arm, open-label, multi-centre, phase I/II study evaluating the safety and clinical activity of QEL-001, an autologous CAR T regulatory cell treatment targeting HLA-A2, in HLA-A2/ A28neg patients that have received an HLA-A2pos liver transplant Quell Therapeutics Limited
Humans
BCMA CAR; shRNA CD3ζ; tCD34
Only notified under the "contained use" procedure. Dossier submitted on 25 July, 2022
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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 (CLL) or Small Lymphocytic Lymphoma (SLL) (CP0101-CLL) – Eup CellPoint B.V. (a Galapagos company)
Humans
CD19 CAR
Only notified under the "contained use" procedure. Dossier submitted on 7 June, 2022
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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) CellPoint B.V. (a Galapagos company)
Humans
BCMA CAR
B/BE/12/BVW2
Clinical trial evaluating the safety and efficacy of intracoronary administration of MYDICAR (AAV1/SERCA2a) in subjects with heart failure Celladon
Humans
Human SERCA2a:CA++ pump involved in the regulation of heart contraction/relaxation cycle
B/BE/12/BVW1
A multi-center, randomized, double-blind, placebo-controlled Phase I/II trial to compare the safety, tolerability and immunogenicity of the therapeutic THV01 vaccination of HIV-1 clade B infected patients under highly antiretroviral therapy TheraVectys
Humans
genes encoding the epitopes of the HIV-1 Gag, Pol and Nef proteins
Only notified under the "contained use" procedure. Dossier submitted on 8 May, 2012
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A single-center, open-label Phase 1 study to assess the effect of food/beverage and to characterisze the pharmacokinetics of single and multiple oral doses of AG013 in Healthy Subjects ActoGenix
Humans
Human Trefoil Factor 1
Only notified under the "contained use" procedure. Dossier submitted on 23 April, 2012
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Randomized Phase III of haploidentical HCT with or without an add back strategy of HSV-Tk donor lymphocytes in patients with high risk acute leukemia Molmed S.p.A.
Humans
-Thymidine Kinase (HSV-Tk)
- selection marker
B/BE/04/BV1
Evaluation of the safety of Feline Herpes Virus, bivalent deleted live vaccine, administered as intranasal vaccine to cats Pfizer, Animal Health Group
cats
gene for the env glycoprotein or gene for the gag protein of the Feline immunodeficiency virus (FIV)
