B/BE/25/BVW9
Phase 1/2 Investigation Of Novel Experimental Regimen in Amyotrophic Lateral Sclerosis (PIONEER-ALS): An Open-Label, Uncontrolled, Multicenter Study to Assess the Safety and Tolerability of Two Doses of VTx-002 VectorY Therapeutics B.V.
Humans
Non-replicating recombinant AAV vector with a AAV5.2 capsid and carrying the transgene corresponding to the single-chain variable fragment that binds to human TDP-43
B/BE/25/BVW8
A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Investigate the Efficacy of a Single Intravenous Dose of SGT-003 in Ambulant Males With Duchenne Muscular Dystrophy Solid Biosciences Inc.
Humans
Non-replicating recombinant vector derived from adeno-associated virus AAV, lacking all AAV viral genes and carrying the human micro-dystrophin (h-µD5) gene
B/BE/25/BVW7
A Randomized, Controlled, Partially Masked, Phase 3b Study to Assess the Injection Burden, Efficacy, Safety, and Long-Term Preservation of Visual Acuity of Surabgene Lomparvovec (ABBVRGX-314) in a Real-World Context in Subjects with Neovascular Age-Relate AbbVie Deutschland GmbH & Co. KG
Humans
Non-replicating recombinant vector derived from adeno-associated virus AAV8 carrying the human anti-vascular endothelial growth factor (VEGF) antigen-binding fragment.
B/BE/25/BVW6
A phase 1, placebo-controlled, randomised, participant- and assessor-blind, single-centre study to assess the safety and immunogenicity of 2 dosages of Nipah measles vector vaccine (MV-NiV) administered subcutaneously either a single dose or as 2 consecut University of Tokyo
Humans
A live attenuated virus (LAV)-MV-NiV, including the full genome of a live-attenuated measles virus (MV) strain, genetically engineered to express the glycoprotein G of the Nipah virus.
B/BE/25/BVW5
GenePHIT: A study to learn more about how well a new gene therapy (AB-1002) works and its safety in participants with congestive heart failure Asklepios BioPharmaceutical (AskBio)
Humans
Non-replicating recombinant vector derived from adeno-associated virus AAV2i8 carrying the transgene encoding for the human activated Inhibitor-1c ( I-1c)
B/BE/25/BVW4
An Open-Label Dose-Escalation Study to Assess the Safety and Tolerability of a Single Intravitreal Injection of SPVN20 Gene Therapy in Subjects with No Light Perception Due to End-Stage Rod-Cone Dystrophy, and Who Retain Dormant Foveal Cone Photoreceptors SparingVision
Humans
Non-replicating recombinant vector derived from adeno-associated virus AAV2 carrying the G protein-gated inwardly rectifying potassium (GIRK) channel 1, mutated for an F137S amino-acid substitution (GIRK1(F137S)) gene
B/BE/25/BVW3
Phase 3, Open-label, Single-dose Study of CSL222 in Adolescent Male Subjects (≥ 12 to < 18 Years of Age) with Severe or Moderately Severe Hemophilia B CSL Behring LLC
Humans
Recombinant adeno associated viral vector containing the codon-optimised human PADUA factor IX (FIX) gene
B/BE/25/BVW2
A randomised, double-blind, placebo-controlled, single centre, Phase I study to evaluate the safety, reactogenicity and immunogenicity of AstriVax’ investigational therapeutic hepatitis B virus (HBV) vaccine (AVX70371) in healthy adults aged 18 to 40 year 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)
Only notified under the "contained use" procedure. Dossier submitted on 10 September, 2025
.
A phase 2, Multicenter, Open-Label Study Of CC-97540 (BMS-986353), CD-19-Targeted NEXT-T Chimeric Antigen Receptor (CAR) T Cells, in Participants with Active Systemic Lupus Erythematosus (SLE) (Including Lupus Nephritis) with Inadequate Response to Glucoc Celgene
Humans
CD19 CAR
Only notified under the "contained use" procedure. Dossier submitted on 25 March, 2025
.
EXACT 2 is a randomized, multicenter, double-blind, placebo-controlled Phase 2b trial designed to evaluate the safety, feasibility, and efficacy of percutaneous endovascular catheter-facilitated intramyocardial administration of XC001 in patients with ref XyloCor Therapeutics
Humans
VEGF
Only notified under the "contained use" procedure. Dossier submitted on 20 March, 2025
.
A Phase 3, Randomized, Open-Label, Multicenter Study to Compare the efficacy and Safety of BMS-986393, a GPRC5D-directed CAR-T Cell Therapy, Versus Standard Regimens in Adult Participants with Relapsed or Refractory and Lenalidomide-refractory Multiple My Celgene
Humans
GPRC5D 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
.
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
.
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
.
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
Only notified under the "contained use" procedure. Dossier submitted on 5 July, 2005
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A multicenter, randomized, double-blind, placebo-controlled study evaluating the efficacy of Biobypass (ADGVVEGF121.10NH) delivered by NOGATM-guided/MYOSTARTM catheter in "no option" patients with class II-IV stable angina GenVec, Inc.
Humans
human VEGF121
Only notified under the "contained use" procedure. Dossier submitted on 1 February, 2005
.
A Controlled, Randomised, Parallel Group, Multicentre Study of the Efficacy and Safety of Herpes Simplex Virus-Thymidine Kinase Gene Therapy (CereproTM), with Subsequent Ganciclovir, for the Treatment of Patients with Operable High-Grade Glioma Ark Therapeutics Ltd
Humans
Thymidine Kinase (HSV-TK1)
