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

> **NCT05329649** · PHASE3 · ACTIVE_NOT_RECRUITING · sponsor: **Vertex Pharmaceuticals Incorporated** · enrollment: 13 (actual)

## Conditions studied

- Sickle Cell Disease
- Hydroxyurea Failure
- Hydroxyurea Intolerance
- Hemoglobinopathies
- Hematological Diseases

## Interventions

- **BIOLOGICAL:** CTX001

## Key facts

- **NCT ID:** NCT05329649
- **Lead sponsor:** Vertex Pharmaceuticals Incorporated
- **Sponsor class:** INDUSTRY
- **Phase:** PHASE3
- **Study type:** INTERVENTIONAL
- **Status:** ACTIVE_NOT_RECRUITING
- **Start date:** 2022-05-02
- **Primary completion:** 2026-05-31
- **Final completion:** 2026-05-31
- **Target enrollment:** 13 (ACTUAL)
- **Last updated:** 2026-03-09

## Collaborators

- CRISPR Therapeutics

## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT05329649

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT05329649, "Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD)". Retrieved via AI Analytics 2026-07-24 from https://api.ai-analytics.org/clinical/NCT05329649. Licensed CC0.

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