Clinical trial
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 CD34+ Stem Cells Transduced Ex Vivo With a Lentiviral βA-T87Q-Globin Vector in Subjects ≤50 Years of Age
NCT02906202 is registered in ClinicalTrials.gov with Genetix Biotherapeutics Inc. as lead sponsor.
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Registry record
As filed in ClinicalTrials.gov.
- Title
- 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 CD34+ Stem Cells Transduced Ex Vivo With a Lentiviral βA-T87Q-Globin Vector in Subjects ≤50 Years of Age
- Lead sponsor
- Genetix Biotherapeutics Inc.
- Phase
- Phase 3
- Overall status
- Completed
- Start date
- August 8, 2016
- Completion date
- March 31, 2022
- Enrollment
- 24 (actual)
- Sites
- 8
Conditions
As the registry lists them; a condition with its own page is linked.