# Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)

> **NCT05356195** · PHASE3 · ACTIVE_NOT_RECRUITING · sponsor: **Vertex Pharmaceuticals Incorporated** · enrollment: 16 (actual)

## Conditions studied

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

## Interventions

- **BIOLOGICAL:** CTX001

## Key facts

- **NCT ID:** NCT05356195
- **Lead sponsor:** Vertex Pharmaceuticals Incorporated
- **Sponsor class:** INDUSTRY
- **Phase:** PHASE3
- **Study type:** INTERVENTIONAL
- **Status:** ACTIVE_NOT_RECRUITING
- **Start date:** 2022-05-03
- **Primary completion:** 2026-05-31
- **Final completion:** 2026-05-31
- **Target enrollment:** 16 (ACTUAL)
- **Last updated:** 2026-03-03

## Collaborators

- [object Object]

## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT05356195

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT05356195, "Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)". Retrieved via AI Analytics 2026-06-25 from https://api.ai-analytics.org/clinical/NCT05356195. Licensed CC0.

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