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/23/BVW3
A Phase I, randomized, double-blind, multi-centre, placebo-controlled, dose-escalation study to evaluate the safety, reactogenicity and immunogenicity of AstriVax’ investigational vaccine for the prevention of yellow fever (AVX70120), and of AstriVax’ inv AstriVax NV
Humans
Full genome of the live attenuated yellow fever virus (YFV) strain 17D (YF17D) containing the sequence of the surface glycoprotein from the rabies virus (RabG)
B/BE/23/BVW2
A Phase 2b, Randomized, Double-masked, Multicenter, Dose-ranging, Sham-controlled Clinical Trial to Evaluate Intravitreal JNJ-81201887(AAVCAGsCD59) Compared to Sham Procedure for the Treatment of Geographic Atrophy (GA) Secondary to Age-related Macular De Janssen-Cilag International NV
Humans
Non-replicating recombinant vector derived from adeno-associated virus AAV2 carrying the soluble human CD59 gene
Only notified under the "contained use" procedure. Dossier submitted on 22 September 2023
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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 Celgene
Humans
BCMA CAR
Only notified under the "contained use" procedure. Dossier submitted on 18 September 2023
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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) Celgene
Humans
CD19 CAR
Only notified under the "contained use" procedure. Dossier submitted on 22 May 2023
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A randomized, open-label, Phase 2 study evaluating lymphodepletion with fludarabine (F), cyclophosphamide (C), and ALLO-647 (A) vs FC alone, in subjects with R/R LBCL receiving ALLO-501A allogeneic CAR T cell therapy Allogene therapeutics
Humans
CD19 CAR + knockouts CD52 and TRAC via TALEN technology
B/BE/18/BVW9
A study to evaluate safety, reactogenicity and immunogenicity of GSK Biologicals’ RSV investigational vaccine based on viral proteins encoded by chimpanzee-derived adenovector (ChAd155-RSV) (GSK3389245A) in infants. GlaxoSmithKline Biologicals SA
Humans
Recombinant replication-defective simian (chimpanzee-derived) group C adenovirus viral vector construct engineered to express three proteins from the Respiratory Syncytial Virus (RSV)
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 uniQure biopharma B.V.
Humans
Recombinant adeno associated viral vector containing the wild type modified to express the Padua derivative of human coagulation factor IX cDNA
B/BE/18/BVW6
Global study of a recombinant adeno-associated virus designed to address the monogenic root cause of Spinal Muscular Atrophy AveXis
Humans
Recombinant adeno-associated virus serotype 9 (AAV9) containing the cDNA of the human SMN (Survival Motor Neuron) gene
B/BE/18/BVW5
A Phase 1b/2a study to assess the safety and tolerability of GM Lactococcus lactis in subjects with Type 1 Diabetes Mellitus Intrexon T1D Partners, LLC
Humans
Biologically contained strain of Lactococcus lactis, genetically modified to secrete human interleukin-10 and proinsulin
B/BE/18/BVW4
A first-time-in human, Phase I, study to evaluate the reactogenicity, safety immunogenicity and efficacy of HBV viral vectored vaccines given in a prime-boost schedule in chronic Hepatitis B patients GlaxoSmithKline Biologicals SA
Humans
The study involves two GMOs. The GMO ChAd155-hIi-HBV is a viral suspension of a recombinant replication-defective simian (chimpanzee-derived) group C adenovirus serotype 155 (ChAd155) viral vector encoding a fusion of sequences derived from two hepatitis B virus (HBV) protein antigens. The GMO MVA-HBV is a modified vaccinia virus Ankara vector (MVA) encoding a fusion of sequences derived from two hepatitis B virus (HBV) protein antigens.
