# Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease

Clinical trial · Vertex Pharmaceuticals Incorporated

- Archive id: nct:NCT05477563
- Published: 2022-07-28
- Venue: ClinicalTrials.gov
- ClinicalTrials.gov: https://clinicaltrials.gov/study/NCT05477563
- Source URL: https://clinicaltrials.gov/study/NCT05477563
- Provenance: clinicaltrials

## Abstract

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.

_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: RECRUITING
- phase: PHASE3
- study type: INTERVENTIONAL
- enrollment: 26
- sponsor: Vertex Pharmaceuticals Incorporated
- conditions: Beta-Thalassemia; Thalassemia; Hematologic Diseases; Genetic Diseases, Inborn; Hemoglobinopathies; Sickle Cell Disease; Sickle Cell Anemia
- interventions: BIOLOGICAL: CTX001
- start date: 2022-08-02
- start date type: ACTUAL
- primary completion date: 2027-06-09
- 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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