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

Brief summary (registry, in English)

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).

What you commit to

• Study type: Interventional · Phase 3
• Planned participants: 160
• From 20 Jan 2021 to the primary completion date, 30 Sept 2039
• 20 sites in 6 countries
• Visits and tests: ask the trial team, the registry does not list them

Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.

Who cannot take part

Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.

Who the study is looking for

Age in the registry: 2 years to 64 years · Sex: All · Does not accept healthy volunteers

Full criteria on ClinicalTrials.gov

Sites (20)

Sites in registry order. Pick your city on the disease page to sort them by distance.

Related publications

• Correction of Ineffective Erythropoiesis and Normalization of Iron Homeostasis After Exagamglogene Autotemcel in Transfusion-Dependent β-Thalassemia.Am J Hematol · 2026 · PMID 42252696