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
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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
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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
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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
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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
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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
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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
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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/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/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 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 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 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
B/BE/20/BVW3
A Phase I/IIa study of intra-tumoral BT-001 (TG6030) administered alone and in combination with pembrolizumab in patients with cutaneous or, subcutaneous lesions or easily injectable lymph nodes of metastatic/advanced solid tumors Transgene
Humans
Replicative oncolytic vaccinia virus derived from the Copenhagen strain, genetically modified by inactivation of its thymidine kinase (TK) and ribonucleotide reductase (RR) genes and by addition of genes encoding for the human granulocyte-macrophage colony-stimulating factor (hGM-CSF) cytokine and for a monoclonal antibody targeting the Cytotoxic T-Lymphocyte-Antigen 4 (CTLA-4).
B/BE/20/BVW2
A first-in human, Phase 1b/2, study to evaluate the safety, tolerability and immunogenicity of HPV viral vectored vaccines in women with low-grade HPV-related cervical lesions Vaccitech Limited
Humans
The study involves two GMOs. ChAdOx1-HPV is a recombinant replication-incompetent chimpanzee-derived adenovirus (ChAdY25) viral vector. MVA-HPV is a modified vaccinia virus Ankara vector (MVA). Both vectors encode a fusion of sequences derived from HPV.
CYAD-211-001
An open-label phase I, multi-center study to determine the recommended dose of the chimeric antigen receptor T-cell treatment CYAD-211 after a non-myeloablative preconditioning chemotherapy in multiple myeloma patients with relapsed or refractory disease. Celyad
Humans
BCMA-CAR and ShRNA CD3ζ
J&JBE-018_004
A randomized, double-blind, placebo-controlled Phase 1/2a study to evaluate the safety, reactogenicity, and immunogenicity of Ad26COVS1 in adults aged 18 to 65 years, inclusive and adults aged 65 years and older. Janssen Vaccines & Prevention B.V.
Humans
Recombinant Adenoviral vector serotype 26 expressing the spike glycoprotein 1 from SARS-CoV2
COVID-19-101
A randomized, placebo-controlled trial, to evaluate the safety and immunogenicity of the COVID-19 vaccine, a measles vector-based vaccine candidate against COVID-19 in healthy volunteers consisting of an unblinded dose escalation and a blinded treatment p Institut Pasteur & Themis Bioscience GmbH
Humans
Recombinant measles virus vaccine strain (Schwarz strain) expressing the Spike glycoprotein 1 from SARS-CoV2
Only notified under the "contained use" procedure. Dossier submitted on 18 February, 2020
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A phase III, randomized observer blinded, placebo-controlled, multi-center study, to evaluate safety and immunogenicity of the Chikungunya vaccine MV-CHIK in healthy adults Themis Bioscience GmbH
Humans
Recombinant measles virus vaccine strain (Schwarz strain) expressing env (E1, E2, E3), capsid C en structural protein 6K from CHIKV
