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

Clinical trial · Vertex Pharmaceuticals Incorporated

- Archive id: nct:NCT04208529
- Published: 2019-12-23
- Venue: ClinicalTrials.gov
- ClinicalTrials.gov: https://clinicaltrials.gov/study/NCT04208529
- Source URL: https://clinicaltrials.gov/study/NCT04208529
- Provenance: clinicaltrials

## Abstract

This is a multi-site, open- label rollover study to evaluate the long-term safety and efficacy of CTX001 in pediatric and adult participants who received CTX001 in parent studies 111 (NCT03655678) 141 (NCT05356195) or 161 (NCT05477563) (transfusion-dependent β-thalassemia \\\[TDT\\\] studies) or Study 121 (NCT03745287) or 151 (NCT05329649) or 161(NCT05477563) (severe sickle cell disease \\\[SCD\\\] studies).

_Abstract reproduced under ClinicalTrials.gov (U.S. National Library of Medicine), data processed 2026-10-29T09:00:05; modifications: selected fields only; whitespace normalized; brief summary truncated to 2,000 characters._

## Trial details

- status: ENROLLING_BY_INVITATION
- phase: PHASE3
- study type: INTERVENTIONAL
- enrollment: 160
- sponsor: Vertex Pharmaceuticals Incorporated
- conditions: Beta-Thalassemia; Thalassemia; Sickle Cell Disease; Hematologic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Sickle Cell Anemia
- interventions: BIOLOGICAL: CTX001
- start date: 2021-01-20
- start date type: ACTUAL
- primary completion date: 2039-09-30
- has results: false
- data source: ClinicalTrials.gov
- data processed: 2026-10-29T09:00:05
- modifications: selected fields only; whitespace normalized; brief summary truncated to 2,000 characters

Topics: [CRISPR & Gene-Editing Therapies](https://space-kitty.com/topics/gene-editing-therapies)

Cited in dossiers:
- [CRISPR & Gene-Editing Therapies: State of the Field (October 2026)](https://space-kitty.com/dossiers/gene-editing-therapies)

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