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/15/BVW2
A Phase 1b/3, Multicenter, Open-label Trial of Talimogene Laherparepvec in Combination With Pembrolizumab for Treatment of Subjects With Recurrent or Metastatic Squamous Cell Carcinoma of the Head and Neck (20130232) 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/15/BVW1
A Phase 1b/3, Multicenter, Open-label Trial of Talimogene Laherparepvec in Combination With Pembrolizumab (MK-3475) for Treatment of Unresected, Stage IIIB to IVM1c Melanoma Amgen Ltd
Humans
Insertion of a gene expressing granulocyte-macrophage colony-stimulating factor (GM-CSF) - Functional deletion of two genes (ICP34.5 and ICP47) of the HSV-1 genome
Only notified under the "contained use" procedure. Dossier submitted on 14 October 2015
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A phase II, single-arm, multi-center trial to determine the efficacy and safety of CTL019 in pediatric patients with relapsed and refractory B-cell acute lymphoblastic leukemia Novartis Pharma Services AG
Humans
Chimeric antigen receptor against CD19
Only notified under the "contained use" procedure. Dossier submitted on 20 August 2015
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A single arm Phase I/II study of the safety and efficacy of gene-modified WT1 TCR therapy in patients with myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML) Catapult Cell Therapy
Humans
Expression of the Wilms' tumour antigen 1 (WT1)- specific T cell receptor (TCR)
Only notified under the "contained use" procedure. Dossier submitted on 24 October 1996
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Prospective, open-label, parallel-group, randomized, multicenter trial comparing the efficacy of surgery, radiation, and injection of murine cells producing herpes simplex thymidine kinase vector followed by intravenous ganciclovir against the efficacy of Genetic therapy, Inc., Sandoz Pharma, Ltd
Humans
Thymidine Kinase (HSV-TK1), neomycin resistance (NeoR)
Only notified under the "contained use" procedure. Dossier submitted on 21 October 1996
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A phase I study in patients with recurrent or metastatic squamous cell carcinoma of the head and neck using SCH 58500 (rAd/p53) administered by single intratumoral injection Schering Plough NV/SA
Humans
Wild-type p53
Only notified under the "contained use" procedure. Dossier submitted on 2 January 1996
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Gene therapy for the treatment of glioblastoma multiforme with in vivo tumor transduction with the herpes simplex thymidine kinase gene /ganciclovir system Sandoz Pharma, LTD
Thymidine Kinase (HSV-TK1), neomycin resistance (NeoR)
