# A Safety and Efficacy Study Evaluating CTX001 in Participants With Transfusion-Dependent β-Thalassemia

Clinical trial · Vertex Pharmaceuticals Incorporated

- Archive id: nct:NCT03655678
- Published: 2018-08-31
- Venue: ClinicalTrials.gov
- ClinicalTrials.gov: https://clinicaltrials.gov/study/NCT03655678
- Source URL: https://clinicaltrials.gov/study/NCT03655678
- Provenance: clinicaltrials

## Abstract

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.

_Abstract reproduced under 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._

## 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

Topics: [CRISPR & Gene-Editing Therapies](https://space-kitty.com/topics/gene-editing-therapies)

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