This is a multi-site, observational study to evaluate the long-term safety and efficacy of CTX001 in subjects who received CTX001 in Study CTX001-111 (NCT03655678) or VX21-CTX001-141 (transfusion-dependent β-thalassemia [TDT] studies) or Study CTX001-121 (NCT03745287) or VX21-CTX001-151 (severe sickle cell disease [SCD] studies; NCT05329649).
- Subjects (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
- Subjects must have received CTX001 infusion in a parent study
- There are no exclusion criteria
Interested in this trial?
About clinical trials
Volunteers who participate in clinical trials help make new medicines a reality. Clinical trials are carefully designed research studies in humans that evaluate the safety and efficacy of an investigational medicine.
If you’re interested in participating in a Vertex clinical trial, we encourage you to speak with your health care provider first about what’s involved and your eligibility.
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Enrolling by invitation1Columbia University Medical Center (21+ years)New York New York 10032 United States
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Enrolling by invitation1Columbia University Medical CenterNew York New York 10032 United States
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Enrolling by invitation1Atrium Health Levine Children's HospitalCharlotte North Carolina 28203 United States
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Enrolling by invitation1Children's Hospital of PhiladelphiaPhiladelphia Pennsylvania 19104 United States
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Enrolling by invitation1St. Jude Children's Research HospitalMemphis Tennessee 38105 United States
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Enrolling by invitation1The Children's Hospital at TriStar Centennial Medical Center/ Sarah Cannon Center for Blood CancersNashville Tennessee 37203 United States
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Enrolling by invitation1Methodist Healthcare System of San Antonio, Methodist Hospital, Methodist Children's HospitalSan Antonio Texas 78229 United States