Condition: Beta-Thalassemia · Thalassemia · Hematologic Diseases · Sponsor: Vertex Pharmaceuticals Incorporated
This is a single-dose, open-label study in participants with transfusion-dependent β-thalassemia (TDT) or severe sickle cell disease (SCD). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) using CTX001.
This description comes directly from the study's public registry record.
Medical Information · 6173416777 · medicalinfo@vrtx.com
Always discuss trial participation with your own doctor first.
| New York Presbyterian Hospital - Morgan Stanley Children's Hospital | New York, New York, United States | Recruiting |
| Levine Children's Hospital - Hematology | Charlotte, North Carolina, United States | Recruiting |
| TriStar Medical Group Children's Specialists - Pediatric Oncology | Nashville, Tennessee, United States | Recruiting |
| University Hospital Dusseldorf - Department of Pediatric Oncology, Hematology and Clinical Immunology | Düsseldorf, Germany | Recruiting |
| IRCSS Ospedale Pediatrico Bambino Gesu - Dipartimento di Onco-Ematologia e Terapia Cellulare e Genica | Rome, Italy | Recruiting |
| King Faisal Specialist Hospital & Research Centre - Riyadh - Hematology | Al Mathar Ash Shamali, Saudi Arabia | Recruiting |
Get one email when the public record changes — results posted, or the study's status changes. Nothing else, ever.
We email about this public record only. Unsubscribe anytime with one click. Never medical advice.
This page is independently generated by Eichor from the public ClinicalTrials.gov record and re-synced daily. It is not the sponsor's official website unless claimed. Nothing here is medical advice; eligibility is always determined by the study team — talk to your own doctor first.
Source record: clinicaltrials.gov/study/NCT05477563