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

Launched by VERTEX PHARMACEUTICALS INCORPORATED · Dec 20, 2019

Keywords

ClinConnect Summary

This is a long-term follow-up study of CTX001, a patient’s own gene-edited blood stem cell therapy used to treat transfusion-dependent beta-thalassemia and sickle cell disease. The trial will track safety and how well CTX001 works for up to 15 years after the infusion. It is open-label and single-group (no comparison or control group). Eligible participants are both children (age 2 and up) and adults who have already received CTX001 in a prior study, and they join by invitation at many centers around the world.

Researchers will monitor long-term safety (for example, new cancers, new blood or bone marrow problems, serious adverse events) and a range of effectiveness measures, including blood results (hemoglobin and fetal hemoglobin), transfusion needs, iron levels, and quality of life. Outcomes differ for sickle cell disease and beta-thalassemia, with goals like fewer painful crises, fewer hospital visits, and more transfusion independence. The study is sponsored by Vertex Pharmaceuticals with CRISPR Therapeutics and includes numerous international sites; results have not been published yet.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • 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
• Exclusion Criteria:
• • There are no exclusion criteria