A Follow-up Study to Evaluate the Long-term Safety and Efficacy in Participants Who Received CS-101 Injection

CorrectSequence Therapeutics

Status: Invitation-only

Conditions: Beta-Thalassemia

Study type: Observational

Funder types: Industry

Identifiers

NCT06717932

CS-101-05

Details and patient eligibility

About

This is a study to evaluate the long-term safety and efficacy of CS-101 in participants who received CS-101 in study CS -101-01 (NCT06291961),No investigational drug product will be administered in the study.

Full description

This is a study to evaluate the long-term safety and efficacy of CS-101 in participants who received CS-101 in study CS -101-01 (NCT06291961). Subjects in the CS-101-01 study will be entered into long-term follow-up of this study up to 15 years post-infusion at the completion of the last (12-month) follow up visit after treatment with CS-101 Injection.

Enrollment: 8 estimated patients

Sex: All

Ages: 12 to 35 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Participants must have received CS-101 infusion in CS-101-01 study
• 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

Exclusion criteria

• There are no exclusion criteria

Trial design

8 participants in 1 patient group

Safety and efficacy assessments

Description:

Safety evaluations,disease-specific assessments,and assessments to monitor for long-term complications of autologous transplant