B/BE/24/BVW6
A randomised, double-blind, placebo-controlled, multi-centre, Phase I study to evaluate the safety, reactogenicity and immunogenicity of AstriVax’ investigational therapeutic hepatitis B virus (HBV) vaccine (AVX70371) in adult patients with chronic HBV AstriVax NV
Humans
Full genome of the live attenuated yellow fever virus (YFV) strain 17D (YF17D) containing the sequence of the HBV core antigen (HBc)
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/24/BVW4
A phase 3 multinational, open-label, systemic gene delivery study to evaluate the safety and efficacy of SRP-9003 in subjects with limb girdle muscular dystrophy 2E/R4 Sarepta Therapeutics
Humans
Non-replicating recombinant vector derived from adeno-associated virus AAV, serotype rh74, lacking all AAV viral genes and carrying the full-length sarcoglycan-beta (SGCB) gene
Only notified under the "contained use" procedure. Dossier submitted on 23 October, 2024
.
A Phase 2, randomized, open-label, controlled study to evaluate the efficacy and safety of rapcabtagene autoleucel versus comparator in participants with severe refractory idiopathic inflammatory myopathies. Novartis Pharma Services AG
Humans
CD19 CAR
Only notified under the "contained use" procedure. Dossier submitted on 23 October, 2024
.
Phase II study evaluating rapcabtagene autoleucel in participants with diffuse cutaneous systemic sclerosis (dcSSc) Novartis Pharma Services AG
Humans
CD19 CAR
Only notified under the "contained use" procedure. Dossier submitted on 23 August, 2024
.
A Phase 3, Randomized, Open-Label Study to Compare the Efficacy and Safety of Anitocabtagene Autoleucel Versus Standard of Care Therapy in Participants With Relapsed/Refractory Multiple Myeloma Kite Pharma
Humans
ddBCMA CAR
Only notified under the "contained use" procedure. Dossier submitted on 5 June, 2024
.
A Phase 2, Open-Label, Randomized, Multicenter Study of KYV 101, an Autologous Fully Human Anti-CD19 Chimeric Antigen Receptor T-Cell (CD19 CAR T) Therapy, in Subjects with Refractory Primary and Secondary Progressive Multiple Sclerosis (KYSA-7) Kyverna Therapeutics, Inc.
Humans
CD19 CAR
Only notified under the "contained use" procedure. Dossier submitted on 1 March, 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 Celgene
Humans
CD19 CAR
Only notified under the "contained use" procedure. Dossier submitted on 1 March, 2024
.
A Phase 1b Multicenter, Open-label, Study of JNJ-90009530, an Autologous Anti-CD20 CAR-T Cell Therapy in Adult Participants with Relapsed or Refractory B-cell Non- Janssen-Cilag International NV
Humans
CD20 CAR
Only notified under the "contained use" procedure. Dossier submitted on 18 January, 2024
.
A phase I/II, multicenter study evaluating the feasibility, safety, and efficacy of point-of-care manufactured GLPG5101 (19CP02) in subjects with relapsed/refractory B-cell non-Hodgkin lymphoma (r/r NHL) (CP0201-NHL) Galapagos NV
Humans
BCMA CAR
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
.
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
.
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
.
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/19/BVW4
Importation of doses of V920 for Emergency Use Merck Sharp & Dohme B.V.
Humans
Recombinant Vesicular Stomatitis Virus (rVSV) in which the gene encoding for the VSV glycoprotein G has been deleted and replaced with the gene encoding the Zaire Ebola virus (ZEBOV) glycoprotein (GP).
B/BE/19/BVW3
A Phase 1, Multi-center, Open-label, Dose De-escalation Study to Evaluate the Safety and efficacy of Talimogene Laherparepvec in Pediatric Subjects With Advanced Noncentral Nervous System Tumors That are Amenable to Direct Injection Amgen Ltd
Humans
Insertion of a gene expressing granulocyte-macrophage colony-stimulating factor (hGM-CSF) - Functional deletion of two genes (ICP34.5 and ICP47) of the HSV-1 genome
B/BE/19/BVW2
A Phase 3 Open-Label, Single-Arm Study To Evaluate The Efficacy and Safety of BMN 270, an Adeno-Associated Virus Vector–Mediated Gene Transfer of Human Factor VIII in Hemophilia A Patients with Residual FVIII Levels Receiving Prophylactic FVIII BioMarin Pharmaceutical Inc.
Humans
Recombinant adeno-associated virus serotype 5 (AAV5) containing a gene cassette expressing the human coagulation factor VIII (FVIII) gene
Only notified under the "contained use" procedure. Dossier submitted on 2 December, 2019
.
A randomized Double-blind, Placebo-controlled First-in-Human, Phase 1/2a Study to Evaluate the Safety, Reactogenicity and Immunogenicity of Monovalent HPV16 and HPV18 Ad26-vectored Vaccine Components and an MVA-vectored HPV16/18 Vaccine Component in Oth Janssen-Cilag International NV
Humans
Recombinant Adenovirus serotype 26 and MVA Bavarian Nordic strain
