Enrolling by invitation
Not Applicable
A Long-term Follow-up Study in Subjects Who Received CTX001

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).

ClinicalTrials.gov Identifier

Eligibility criteria
Sex
Age
2 Years and older
Phase
Not Applicable
Key inclusion criteria
  • 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
Key exclusion criteria
  • There are no exclusion criteria
Trial details
Condition
Beta-Thalassemia, Thalassemia, Sickle Cell Disease, Hematologic Diseases, Hemoglobinopathies, Genetic Diseases, Inborn, Sickle Cell Anemia
Study duration
January 2021 - September 2039
Study type
Observational
Intervention/Treatment
Biological : CTX001
Estimated enrollment
114

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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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