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

Brief 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).

Source: 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. Not a U.S. Government endorsement.

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