Study Summary
This is a single-dose, open-label study in pediatric participants with TDT. 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 |
|---|---|---|---|
| TriStar Medical Group Children's Specialists - Pediatric Oncology | Nashville | Tennessee | United States |
| Hospital for Sick Children - Hematology | Toronto | Canada | |
| University Hospital Dusseldorf - 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 | |
| Great Ormond Street Hospital for Children | London | United Kingdom | |
| St.Mary's Hospital - Children's Clinical Research Facility | London | United Kingdom |