# Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD)

Clinical trial · Vertex Pharmaceuticals Incorporated

- Archive id: nct:NCT05329649
- Published: 2022-04-15
- Venue: ClinicalTrials.gov
- ClinicalTrials.gov: https://clinicaltrials.gov/study/NCT05329649
- Source URL: https://clinicaltrials.gov/study/NCT05329649
- Provenance: clinicaltrials

## Abstract

This is a single-dose, open-label study in pediatric participants with severe SCD and hydroxyurea (HU) failure or intolerance. The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) (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: ACTIVE_NOT_RECRUITING
- phase: PHASE3
- study type: INTERVENTIONAL
- enrollment: 13
- sponsor: Vertex Pharmaceuticals Incorporated
- conditions: Sickle Cell Disease; Hydroxyurea Failure; Hydroxyurea Intolerance; Hemoglobinopathies; Hematological Diseases
- interventions: BIOLOGICAL: CTX001
- start date: 2022-05-02
- start date type: ACTUAL
- primary completion date: 2027-06-06
- 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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