B/BE/21/BVW8 |
Phase 3 Randomized, Controlled Study of AAV5-hRKp.RPGR for the Treatment of X-linked Retinitis Pigmentosa Associated with Variants in the RPGR gene |
Humans |
Non-replicating recombinant vector derived from adeno-associated virus AAV 5 carrying the human RPGR gene |
Only notified under the "contained use" procedure. Dossier submitted on 03/05/2021. |
Administration of genetically engineered T cells (KTE-X19) in Pediatric and Adolescent Subjects with Relapsed/Refractory B-precursor Acute Lymphoblastic Leukemia or Relapsed/Refractory B-cell Non-Hodgkin Lymphoma |
Humans |
CD19-CAR |
B/BE/18/BVW6 |
Global study of a recombinant adeno-associated virus designed to address the monogenic root cause of Spinal Muscular Atrophy |
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 |
Humans |
Biologically contained strain of Lactococcus lactis, genetically modified to secrete human interleukin-10 and proinsulin |
Only notified under the "contained use" procedure. Dossier submitted on 25/04/2018. |
A Phase 3, Randomized, Open-Label Study Evaluating the Efficacy of Axicabtagene Ciloleucel versus Standard of Care Therapy in Subjects with Relapsed/Refractory Diffuse Large B Cell Lymphoma (ZUMA-7) |
Humans |
Chimeric antigen receptor against CD19 |
Only notified under the "contained use" procedure. Dossier submitted on 24/04/2017. |
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 |
Humans |
chimeric antigen receptor and suicide-ligand (RQR8) |
Only notified under the "contained use" procedure. Dossier submitted on 20/08/2015. |
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) |
Humans |
Expression of the Wilms' tumour antigen 1 (WT1)- specific T cell receptor (TCR) |
B/BE/11/BVW2 |
Clinical Study BNIT-PRV-301 - A Randomized, Double-blind, Phase 3 Efficacy Trial of PROSTVAC +/- GM-CSF in Men With Asymptomatic or Minimally Symptomatic Metastatic, Castrate-Resistant Prostate Cancer |
Humans |
Human gene coding for the prostate-specific antigen (PSA) and genes encoding 3 human immunological costimulatory molecules |