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.
Key facts
- Who runs it
- Vertex Pharmaceuticals Incorporated
- Where
- Charlotte, Chicago, Memphis, Nashville, New York, Palo Alto, Philadelphia, and San Antonio, United StatesSee the 9 sitesAlso in 5 other countries, 11 sites
- Registry title
- A Long-term Follow-up Study in Participants Who Received CTX001
- Status
- Enrolling by invitation
- Phase
- Phase 3
- People taking part
- 160 people, planned
- Started
- 20 Jan 2021
- Expected to end
- 30 Sept 2039
- Type of study
- Interventional
- Treatments tested
- CTX001
- Record updated
- 11 August 2026
Who can take part
AI summary
From the trial's eligibility criteria. Machine-read from the registry text on this page, so it can be wrong. The registry text below and the trial team decide who can take part.
The trial is looking for people who already received a CTX001 infusion in an earlier study for transfusion-dependent β-thalassemia or severe sickle cell disease. It follows them over the long term.
- Age
- 2 Years to 64 Years
- Sex
- All sexes can join.
- Disease stage
- Transfusion-dependent β-thalassemia or severe sickle cell disease, as treated in the earlier (parent) studies.
- Earlier treatment
- Must have received a CTX001 infusion in a parent study.
Main reasons for not taking part
- There are no exclusion criteria.
The registry criteria, word for word
Show all 3 criteriaShow less
Who can take part
- 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
Who cannot take part
- There are no exclusion criteria
Check if you might fit
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?
Where in United States
Also in 5 other countries, 11 sites
- Levine Children's Hospital - HematologyCharlotte, United StatesEnrolling by invitation
- Ann & Robert H. Lurie Children's Hospital of Chicago - HematologyChicago, United StatesEnrolling by invitation
- St. Jude Children's Research HospitalMemphis, United StatesEnrolling by invitation
- TriStar Medical Group Children's Specialists - Pediatric OncologyNashville, United StatesEnrolling by invitation
- Herbert Irving Pavilion - HematologyNew York, United StatesEnrolling by invitation
- New York Presbyterian Hospital - Morgan Stanley Children's HospitalNew York, United StatesEnrolling by invitation
- Lucile Packard Children's HospitalPalo Alto, United StatesEnrolling by invitation
- The Children's Hospital of Philadelphia - HematologyPhiladelphia, United StatesEnrolling by invitation
- Methodist Healthcare System of San Antonio, Methodist Hospital, Methodist Children's HospitalSan Antonio, United StatesEnrolling by invitation
About the trial
What the trial wants to find out
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).
The group in the trial
- CTX001Treatment being tested
All participants who complete or discontinue one of the multiple parent studies (CTX001-111, CTX001-121, CTX001-141, CTX001-151 and CTX001-161) after CTX001 infusion will be asked to participate in this long-term follow-up study.
Results explained
A paper that names this trial, explained in plain words.
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?
Contact the study team: see the registry record → Contacts and Locations
Print a page for my doctorData and sources
- Registry record
- Listed on ClinicalTrials.gov and on the EU's official list of trials (CTIS).
- Official title
- 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)
- Plain-language title
- Long-term follow-up of people treated with CTX001 for thalassemia or sickle cell diseaseGenerated from the registry record. The registry title is the official one.
- AI summary
- From the trial's eligibility criteria. Read by machine on 12 November 2026.
- Fit questions
- Built from NCT04208529, who can take part
- Publications
- Correction of Ineffective Erythropoiesis and Normalization of Iron Homeostasis After Exagamglogene Autotemcel in Transfusion-Dependent β-Thalassemia.American journal of hematology, 2026
- Improvements in health-related quality of life in patients with severe sickle cell disease after exagamglogene autotemcel.Blood advances, 2025
- Improvements in health-related quality of life in patients with transfusion-dependent β-thalassemia after exagamglogene autotemcel.Blood advances, 2025
- Record updated
- 11 August 2026