A Long-term Follow-up Study in Subjects Who Received CTX001

Enrolling by invitation

Not Applicable

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

NCT04208529

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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Enrolling by invitation

Lucile Packard Children's Hospital

Palo Alto California 94304 United States