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 |
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 |
B/BE/22/BVW6 |
A phase 3, Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of SRP-9001 in Non-Ambulatory and Ambulatory Subjects With Duchenne Muscular Dystrophy (ENVISION) |
Humans |
Non-replicating recombinant vector derived from adeno-associated virus AAV, serotype rh74, lacking all AAV viral genes and carrying the human micro-dystrophin (hMicro-Dys) gene |
B/BE/21/BVW5 |
A Phase 3 Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Delivery Study to Evaluate the Safety and Efficacy of SRP-9001 in Subjects With Duchenne Muscular Dystrophy (EMBARK) |
Humans |
Non-replicating recombinant vector derived from adeno-associated virus AAV, serotype rh74, lacking all AAV viral genes and carrying the human micro-dystrophin (hMicro-Dys) gene |
B/BE/21/BVW3 |
Phase I/II study to evaluate the safety and effects on Progranulin levels of PR006 in patients with Fronto-Temporal Dementia with Progranulin mutations (FTD-GRN). |
Humans |
The study involves a replication-incompetent adeno-associated virus, AAV9, lacking all AAV viral genes and encoding for the human Progranulin (GRN) gene |
Only notified under the "contained use" procedure. Dossier submitted on 28/10/2021. |
A Phase I/II, First-in-human, open-label, accelerated-titration, two-part clinical trial of TK-8001 in patients with HLA-A*02:01 genotype and advanced-stage/metastatic, MAGE-A1 + solid tumors that either have no further approved therapeutic alternative(s) |
Humans |
chimere MAGE-A1 TCRs |
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 |
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/BVW3 |
A phase 2 study to assess the safety and efficacy of GM Lactococcus lactis for the attenuation of oral mucositis in subjects with cancers of the head and neck |
Humans |
Biologically contained strain of Lactococcus lactis, genetically modified to secrete human Trefoil Factor 1 (hTFF1). |
Only notified under the "contained use" procedure. Dossier submitted on 13/10/2000. |
A phase III open-label, comparative, multicentre trials to test the concept of durable virologic suppression in subjects with primary HIV-1 infection after intensive induction of quadruple HAART followed by double-blind randomization to HIV vaccination wi |
Humans |
vCP-1452 |
B/BE/98/B6 |
Clinical research program : Gene-therapy by the Use of a Recombinant Adenovirus in the Treatment of p53 Deficient Cancers |
Humans |
wild-type p53 tumor suppressor gene |
Only notified under the "contained use" procedure. Dossier submitted on 18/08/1997. |
A phase II gene therapy study in patients with non-small cell lung cancer using SCH58500 (rAd/p53) in combination with chemotherapy for multiple cycles |
Humans |
Wild-type p53 |
Only notified under the "contained use" procedure. Dossier submitted on 21/10/1996. |
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 |
Humans |
Wild-type p53 |