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).
Age range
2 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
New malignancies
Timeframe: Signing of informed consent up to 15 years post CTX001 infusion
New or worsening hematologic disorders
Timeframe: Signing of informed consent up to 15 years post CTX001 infusion
All-cause mortality
Timeframe: Signing of informed consent up to 15 years post CTX001 infusion
Serious adverse events (SAEs)
Timeframe: Signing of informed consent up to 15 years post CTX001 infusion
CTX001-related adverse events (AEs)
Timeframe: Signing of informed consent up to 15 years post CTX001 infusion