B/BE/26/BVW4
A Phase 1/2/3, Open-Label, Dose Escalation, Dose Expansion, and Randomized, Controlled Study to Evaluate the Safety and Efficacy of ATSN-201 Gene Therapy in Subjects with RS1-Associated X-linked Retinoschisis (LIGHTHOUSE) ASTENA Therapeutics
Humans
Non-replicating recombinant vector derived from adeno-associated virus AAV44.9 carrying functional copies of the human retinoschisin (hRS1) gene.
B/BE/26/BVW3
A phase I/II/III study with a dose determination part followed by an efficacy and safety evaluation, quadruple blind placebo-controlled part and then by a long-term safety follow up part, in ambulant boys with Duchenne Muscular Dystrophy GENETHON
Humans
Non-replicating recombinant vector derived from adeno-associated virus AAV, lacking all AAV viral genes and carrying the human dystrophin (hMD1) gene
B/BE/26/BVW2
An Open-label, Multicenter, Two Part, Ascending Dose Followed by a Controlled Trial to Assess the Safety and Efficacy of a Subretinal Administration of AAVB-039 in Participants with Stargardt Disease (STGD1) (CELESTE) AAVantgarde Bio UK Ltd.
Humans
Non-replicating recombinant dual vector derived from adeno-associated virus AAV8 carrying functional copies of the ABCA4 gene.
Only notified under the "contained use" procedure. Dossier submitted on 30 March, 2026
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A Phase 3, Randomized, Open-label, Multicenter Study to Compare the Efficacy and Safety of BMS-986353, CD-19-targeted NEXT-T CAR-T Cells, Versus Standard of Care in Participants with Active Systemic Sclerosis (Breakfree-SSc) Celgene
Humans
CD19 CAR
B/BE/17/BVW2
A Phase 1b Study of Talimogene Laherparepvec in Combination With Atezolizumab in Subjects With Triple Negative Breast Cancer and Colorectal Cancer With Liver Metastases 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/17/BVW1
A phase II study evaluating the efficacy and the safety of first-line chemotherapy combined with TG4010 and nivolumab in patients with advanced non-squamous Non-Small-Cell Lung Cancer (NSCLC) Transgene
Humans
Sequences coding for human mucine 1 (MUC1) antigen and interleukin-2 (IL2)
68284528MMY2001
A phase III, multicenter, randomized, open-label study to compare the efficacy and safety of bb2121 versus standard triplet regimens in subjects with relapsed and refractory multiple myeloma (RRMM) (KarMMa-3) Celgene
Humans
BCMA02 (human B cell maturation antigen) -Chimeric antigen receptor
Only notified under the "contained use" procedure. Dossier submitted on 24 April, 2017
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A Phase 1, Open Label, Non-comparative Study to Evaluate the Safety and the Ability of UCART19 to Induce Molecular Remission in Paediatric Patients With Relapsed/Refractory B Acute Lymphoblastic Leukaemia Institut de Recherches Internationales Servier
Humans
chimeric antigen receptor and suicide-ligand (RQR8)
Only notified under the "contained use" procedure. Dossier submitted on 7 April, 2017
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Phase 1 clinical trial evaluation of two genetically modified vaccines against poliovirus UZ Antwerpen
Humans
Genetic modifications of the nOPV2 vaccine candidates aimed at stabilizing the genetic sequence against reversion compared to the Sabin-2 strain, nOPV2 candidate 1 (S2/cre5/S15domV/rec1/hifi3), nOPV2 candidate 2 (S2/S15domV/CpG40)
B/BE/01/B7
Specific immunotherapy against MUC-1 antigen - Study TG4010.04 : "Phase II study with TG4010(MVA-MUC-1-IL-2) in patients with metastatic breast cancer", Study TG4010.05 : "Phase II study with TG 4010 in patients with non small cell lung cancer" Transgene S.A.
Humans
sequences coding for the human MUC-1 antigen and IL-2
B/BE/01/V6
Evaluation of efficacy of Salmonella Dublin-Typhimurium vaccine, double gene deleted avirulent live culture in calves. Pharmacia Animal health
calves
genetic modification of Salmonella enterica by deletion of 2 genes (ssaC and ssaT)
Only notified under the "contained use" procedure. Dossier submitted on 9 November, 2001
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A phase III, multi-center, open-label, randomized study to compare the effectiveness and safety of intratumoral administration of INGN 201 in combination with chemotherapy versus chemotherapy alone in 288 patients with recurrent squamous cell carcinoma of Introgen Therapeutics, Inc.
Humans
Wild-type p53
Only notified under the "contained use" procedure. Dossier submitted on 9 November, 2001
.
A phase III, multi-center, open-label, randomized study to compare the overall survival and safety of bi-weekly intratumoral administration of INGN 201 versus weekly methotrewate in 240 patients with refractory squamous cell carcinoma of the head and neck Introgen Therapeutics, Inc.
Humans
Wild-type p53
