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Clinical trial

An Open-Label Study to Evaluate the Safety and Efficacy of a Single Dose of Autologous CD34+ Human Hematopoietic Stem Cells Modified Using Transformer Base Editor in Participants With Severe Sickle Cell Disease

NCT07000318 is registered in ClinicalTrials.gov with Children's Hospital of Fudan University as lead sponsor.

NCT NCT07000318
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Registry record

As filed in ClinicalTrials.gov.

Title
An Open-Label Study to Evaluate the Safety and Efficacy of a Single Dose of Autologous CD34+ Human Hematopoietic Stem Cells Modified Using Transformer Base Editor in Participants With Severe Sickle Cell Disease
Lead sponsor
Children's Hospital of Fudan University
Phase
Phase 1
Overall status
Not yet recruiting
Start date
September 30, 2026
Completion date
March 31, 2029
Enrollment
5 (estimated)
Sites
1

Conditions

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

sickle cell disease