These trials completed more than 12 months ago and should have reported results.

These trials are not yet due to report results: some are ongoing, some completed within the past 12 months.

These trials have problematic data on the registry. Details why »

Status Trial ID Title Completion date Category
Trial is partly outside EEC 2011-001953-10 A phase 2/3 study of the efficacy and safety of hematopoietic stem cells transduced with Lenti D lentiviral vector for the treatment of cerebral adrenoleukodystrophy (CALD) bad-data
Exempt, with results 2012-000695-42 A Phase I/II Open Label Study Evaluating the Safety and Efficacy of Gene Therapy of the β-Hemoglobinopathies (Sickle Cell Anemia and β-Thalassemia Major) by Transplantation of Autologous CD34+ Stem Ce... 2019-02-26 not-yet-due
Ongoing 2013-002245-11 Long-term Follow-up of Subjects with Hemoglobinopathies Treated with Ex Vivo Gene Therapy using Autologous Hematopoietic Stem Cells Transduced with a Lentiviral Vector not-yet-due
Other 2015-002805-13 Longterm Follow-up of Subjects With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product not-yet-due
Ongoing 2015-004122-33 A Phase 3 Single Arm Study Evaluating the Efficacy and Safety of Gene Therapy in Subjects with Transfusion-dependent β-Thalassemia, who do not have a β0/β0 Genotype, by Transplantation of Autologous C... not-yet-due
Other 2016-003611-35 A Phase 3 Single Arm Study Evaluating the Efficacy and Safety of Gene Therapy in Subjects with Transfusion-dependent β-Thalassemia by Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo ... not-yet-due
Other 2018-001145-14 A Phase 3 Study of Lenti-D Drug Product After Myeloablative Conditioning Using Busulfan and Fludarabine in Subjects ≤17 Years of Age With Cerebral Adrenoleukodystrophy (CALD) not-yet-due