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Clinical trial

A Long-term Follow-up Study of Subjects With β-thalassemia or Sickle Cell Disease Treated With Autologous CRISPR-Cas9 Modified Hematopoietic Stem Cells (CTX001)

NCT04208529 is registered in ClinicalTrials.gov with Vertex Pharmaceuticals Incorporated as lead sponsor.

NCT NCT04208529
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Registry record

As filed in ClinicalTrials.gov.

Title
A Long-term Follow-up Study of Subjects With β-thalassemia or Sickle Cell Disease Treated With Autologous CRISPR-Cas9 Modified Hematopoietic Stem Cells (CTX001)
Lead sponsor
Vertex Pharmaceuticals Incorporated
Phase
Phase 3
Overall status
Enrolling by invitation
Start date
January 20, 2021
Completion date
September 30, 2039
Enrollment
160 (estimated)
Sites
20

Conditions

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

beta-thalassemia, genetic diseases, inborn, hematologic diseases, hemoglobinopathies, sickle cell anemia, sickle cell disease, thalassemia