Clinical Trial

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

Enrolling by Invitation Phase 3
View on ClinicalTrials.gov →
Summary
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).
Protocol Amendment History 30 amendments
This ClinicalTrials.gov record has been amended 30 times since 2019-12-20; most recent amendment 2026-03-20.
Status change: Not Yet Recruiting → Enrolling by Invitation 2020-02-28
Trial Details
NCT Number NCT04208529
Lead Sponsor Vertex Pharmaceuticals Incorporated
Collaborators: CRISPR Therapeutics
Conditions Beta-Thalassemia, Thalassemia, Sickle Cell Disease, Hematologic Diseases, Hemoglobinopathies, Genetic Diseases, Inborn, Sickle Cell Anemia
Enrollment 160 participants
Start Date 2021-01-20
Primary Completion 2039-09-30 (estimated)
Study Completion 2039-09-30 (estimated)
Updated on ClinicalTrials.gov 2026-08-11