# A Long-term Follow-up Study in Participants Who Received CTX001

> **NCT04208529** · PHASE3 · ENROLLING_BY_INVITATION · sponsor: **Vertex Pharmaceuticals Incorporated** · enrollment: 160 (estimated)

## Conditions studied

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

## Interventions

- **BIOLOGICAL:** CTX001

## Key facts

- **NCT ID:** NCT04208529
- **Lead sponsor:** Vertex Pharmaceuticals Incorporated
- **Sponsor class:** INDUSTRY
- **Phase:** PHASE3
- **Study type:** INTERVENTIONAL
- **Status:** ENROLLING_BY_INVITATION
- **Start date:** 2021-01-20
- **Primary completion:** 2039-09-30
- **Final completion:** 2039-09-30
- **Target enrollment:** 160 (ESTIMATED)
- **Last updated:** 2026-03-25

## Collaborators

- [object Object]

## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT04208529

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT04208529, "A Long-term Follow-up Study in Participants Who Received CTX001". Retrieved via AI Analytics 2026-06-16 from https://api.ai-analytics.org/clinical/NCT04208529. Licensed CC0.

---

*[Clinical trials dataset](/datasets/clinical-trials) · CC0 1.0*