A Safety and Efficacy Study Evaluating CTX001 in Participants With Transfusion-Dependent β-Thalassemia
Vertex Pharmaceuticals Incorporated
View at source ↗Clinical trial
Source: ClinicalTrials.gov (U.S. National Library of Medicine), data processed 2026-10-29T09:00:05; modifications: selected fields only; whitespace normalized; brief summary truncated to 2,000 characters. Not a U.S. Government endorsement.
Brief summary
This is a single-arm, open-label, multi-site, single-dose Phase 1/2/3 study in participans with transfusion-dependent β-thalassemia (TDT). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) using CTX001.
Source: ClinicalTrials.gov (U.S. National Library of Medicine), data processed 2026-10-29T09:00:05; modifications: selected fields only; whitespace normalized; brief summary truncated to 2,000 characters. Not a U.S. Government endorsement.
Trial details
- status
- COMPLETED
- phase
- PHASE2, PHASE3
- study type
- INTERVENTIONAL
- enrollment
- 59
- sponsor
- Vertex Pharmaceuticals Incorporated
- conditions
- Beta-Thalassemia; Thalassemia; Genetic Diseases, Inborn; Hematologic Diseases; Hemoglobinopathies
- interventions
- BIOLOGICAL: CTX001
- start date
- 2018-09-14
- start date type
- ACTUAL
- primary completion date
- 2025-11-13
- has results
- false
- data source
- ClinicalTrials.gov
- data processed
- 2026-10-29T09:00:05
- modifications
- selected fields only; whitespace normalized; brief summary truncated to 2,000 characters
Record
- Archive ID
- nct:NCT03655678
- Published
- Aug 31, 2018
- Updated
- Dec 17, 2025
- Venue
- ClinicalTrials.gov
- ClinicalTrials.gov
- NCT03655678
- Categories
- Beta-Thalassemia, Thalassemia, Genetic Diseases, Inborn, Hematologic Diseases, Hemoglobinopathies
- License
- ClinicalTrials.gov terms (attribution, processing date, modifications noted)
- Provenance
- clinicaltrials