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

Study 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).

Conditions

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

Interventions / Drugs

CTX001

View on ClinicalTrials.gov ↗

Sponsor

VRTX

Vertex Pharmaceuticals

Gene Therapy

C55/100

Study Details

Enrollment160 participants

Primary Completion—

Lead SponsorVertex Pharmaceuticals Incorporated

Data Retrieved2026-04-11 02:55:58.035356+00:00

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