Study Summary
This is a single-dose, open-label study in pediatric participants with severe SCD and hydroxyurea (HU) failure or intolerance. The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) (CTX001).
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CTX001BIOLOGICAL
Administered by intravenous infusion following myeloablative conditioning with busulfan.
Study Locations
| Facility | City | State | Country |
|---|---|---|---|
| Levine Children's Hospital - Hematology | Charlotte | North Carolina | United States |
| The Children's Hospital of Philadelphia - Hematology | Philadelphia | Pennsylvania | United States |
| St. Jude Children's Research Hospital | Memphis | Tennessee | United States |
| TriStar Medical Group Children's Specialists - Pediatric Oncology | Nashville | Tennessee | United States |
| University Hospital Duesseldorf - Department of Pediatric Oncology, Hematology and Clinical Immunology | Düsseldorf | Germany | |
| IRCSS Ospedale Pediatrico Bambino Gesu - Dipartimento di Onco-Ematologia e Terapia Cellulare e Genica | Rome | Italy | |
| St.Mary's Hospital - Haematology Dept | London | United Kingdom |