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

> **NCT03655678** · PHASE2,PHASE3 · COMPLETED · sponsor: **Vertex Pharmaceuticals Incorporated** · enrollment: 59 (actual)

## Conditions studied

- Beta-Thalassemia
- Thalassemia
- Genetic Diseases, Inborn
- Hematologic Diseases
- Hemoglobinopathies

## Interventions

- **BIOLOGICAL:** CTX001

## Key facts

- **NCT ID:** NCT03655678
- **Lead sponsor:** Vertex Pharmaceuticals Incorporated
- **Sponsor class:** INDUSTRY
- **Phase:** PHASE2,PHASE3
- **Study type:** INTERVENTIONAL
- **Status:** COMPLETED
- **Start date:** 2018-09-14
- **Primary completion:** 2025-11-13
- **Final completion:** 2025-11-13
- **Target enrollment:** 59 (ACTUAL)
- **Last updated:** 2025-12-17

## Collaborators

- [object Object]

## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT03655678

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT03655678, "A Safety and Efficacy Study Evaluating CTX001 in Participants With Transfusion-Dependent β-Thalassemia". Retrieved via AI Analytics 2026-07-19 from https://api.ai-analytics.org/clinical/NCT03655678. Licensed CC0.

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