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Clinical trial

A Phase 1/2 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 Cells Transduced Ex Vivo With a Lentiviral β-A-T87Q Globin Vector (LentiGlobin BB305 Drug Product)

NCT02151526 is registered in ClinicalTrials.gov with Genetix Biotherapeutics Inc. as lead sponsor.

NCT NCT02151526
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Registry record

As filed in ClinicalTrials.gov.

Title
A Phase 1/2 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 Cells Transduced Ex Vivo With a Lentiviral β-A-T87Q Globin Vector (LentiGlobin BB305 Drug Product)
Lead sponsor
Genetix Biotherapeutics Inc.
Phase
Phase 1/2
Overall status
Completed
Start date
June 7, 2013
Completion date
February 26, 2019
Enrollment
7 (actual)
Sites
1

Conditions

As the registry lists them; a condition with its own page is linked.

beta-thalassemia major, sickle cell disease