This multi-site, open-label rollover study evaluates the long-term safety and efficacy of CTX001 (Exagamglogene autotemcel, Exa-cel) in pediatric and adult participants who received it in a parent study. The parent studies are 111 (NCT03655678), 141 (NCT05356195) and 161 (NCT05477563) for transfusion-dependent beta-thalassemia (TDT), and 121 (NCT03745287), 151 (NCT05329649) and 161 (NCT05477563) for severe sickle cell disease (SCD). Participants, or their legally authorized representative or guardian, must sign the informed consent form and must have received a CTX001 infusion in a parent study; enrollment is by invitation.
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.
Inclusion Criteria: * Participants (or his or her legally appointed and authorized representative or guardian) must sign and date informed consent form (ICF) and, where applicable, an assent form * Participants must have received CTX001 infusion in a parent study Exclusion Criteria: * There are no exclusion criteria