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

> **NCT05477563** · PHASE3 · RECRUITING · sponsor: **Vertex Pharmaceuticals Incorporated** · enrollment: 26 (estimated)

## Conditions studied

- Beta-Thalassemia
- Thalassemia
- Hematologic Diseases
- Genetic Diseases, Inborn
- Hemoglobinopathies
- Sickle Cell Disease
- Sickle Cell Anemia

## Interventions

- **BIOLOGICAL:** CTX001

## Key facts

- **NCT ID:** NCT05477563
- **Lead sponsor:** Vertex Pharmaceuticals Incorporated
- **Sponsor class:** INDUSTRY
- **Phase:** PHASE3
- **Study type:** INTERVENTIONAL
- **Status:** RECRUITING
- **Start date:** 2022-08-02
- **Primary completion:** 2027-06-09
- **Final completion:** 2027-06-09
- **Target enrollment:** 26 (ESTIMATED)
- **Last updated:** 2026-03-23


## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT05477563

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT05477563, "Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease". Retrieved via AI Analytics 2026-06-16 from https://api.ai-analytics.org/clinical/NCT05477563. Licensed CC0.

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