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

NCT04208529 · Status: ENROLLING_BY_INVITATION · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 160

Last updated 2026-03-25

No results posted yet for this 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

Interventions

BIOLOGICAL

CTX001

CTX001 infusion.

Sponsors & Collaborators

Study Design

Allocation
NA
Purpose
OTHER
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
2 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-01-20
Primary Completion
2039-09-30
Completion
2039-09-30
FDA Drug
Yes

Countries

  • United States
  • Belgium
  • Canada
  • Germany
  • Italy
  • United Kingdom

Study Locations

More Related Trials

Entities

Diseases
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04208529 on ClinicalTrials.gov