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.
Search for the closest clinical trial from your location
Enrolling by invitation1Lucile Packard Children's HospitalPalo Alto California 94304 United States
Enrolling by invitation1Ann & Robert Lurie Children's Hospital of ChicagoChicago Illinois 60611 United States
Enrolling by invitation1Columbia University Medical Center (21+ years)New York New York 10032 United States
Enrolling by invitation1Columbia University Medical CenterNew York New York 10032 United States
Enrolling by invitation1Atrium Health Levine Children's HospitalCharlotte North Carolina 28203 United States
Enrolling by invitation1Children's Hospital of PhiladelphiaPhiladelphia Pennsylvania 19104 United States
Enrolling by invitation1St. Jude Children's Research HospitalMemphis Tennessee 38105 United States
Enrolling by invitation1The Children's Hospital at TriStar Centennial Medical Center/ Sarah Cannon Center for Blood CancersNashville Tennessee 37203 United States
Enrolling by invitation1Methodist Healthcare System of San Antonio, Methodist Hospital, Methodist Children's HospitalSan Antonio Texas 78229 United States