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Clinical trial

A Phase 1/2/3 Study to Evaluate the Safety and Efficacy of a Single Dose of Autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (CTX001) in Subjects With Severe Sickle Cell Disease

NCT03745287 is registered in ClinicalTrials.gov with Vertex Pharmaceuticals Incorporated as lead sponsor.

NCT NCT03745287
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Registry record

As filed in ClinicalTrials.gov.

Title
A Phase 1/2/3 Study to Evaluate the Safety and Efficacy of a Single Dose of Autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (CTX001) in Subjects With Severe Sickle Cell Disease
Lead sponsor
Vertex Pharmaceuticals Incorporated
Phase
Phase 2/3
Overall status
Completed
Start date
November 27, 2018
Completion date
July 7, 2025
Enrollment
63 (actual)
Sites
17

Conditions

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

hematological diseases, hemoglobinopathies, sickle cell disease