Long-term follow-up of people treated with CTX001 for thalassemia or sickle cell disease
Follows people with beta-thalassemia or sickle cell disease after treatment with CTX001.
Plain-language title generated from the registry record
Registry title: A Long-term Follow-up Study in Participants Who Received CTX001
Key facts
- Where
- Charlotte, Chicago, Memphis, Nashville, New York, Palo Alto, Philadelphia, and San Antonio, United StatesSee the 9 sites
- Phase
- Phase 3
Could this be for you?
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 2 and 64 years old?
Source: NCT04208529, who can take part
Where in United States
- Levine Children's Hospital - HematologyCharlotte, United StatesNo longer taking part
- Ann & Robert H. Lurie Children's Hospital of Chicago - HematologyChicago, United StatesNo longer taking part
- St. Jude Children's Research HospitalMemphis, United StatesNo longer taking part
- TriStar Medical Group Children's Specialists - Pediatric OncologyNashville, United StatesNo longer taking part
- Herbert Irving Pavilion - HematologyNew York, United StatesNo longer taking part
- New York Presbyterian Hospital - Morgan Stanley Children's HospitalNew York, United StatesNo longer taking part
- Lucile Packard Children's HospitalPalo Alto, United StatesNo longer taking part
- The Children's Hospital of Philadelphia - HematologyPhiladelphia, United StatesNo longer taking part
- Methodist Healthcare System of San Antonio, Methodist Hospital, Methodist Children's HospitalSan Antonio, United StatesNo longer taking part
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Vertex Pharmaceuticals Incorporated
- Trial code
- NCT04208529EU 2024-512654-19-00
- Record updated
- 11 August 2026
- Original description
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).Read full original descriptionShow less
A Long-term Follow-up Study of Subjects With β-thalassemia or Sickle Cell Disease Treated With Autologous CRISPR-Cas9 Modified Hematopoietic Stem Cells (CTX001)
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).
Inclusion: 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: There are no exclusion criteria.