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

A Long-term Follow-up Study Evaluating the Safety and Efficacy of Subjects With β-thalassemia Treated With CS-101 Transplantation

Status

Enrolling by invitation

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT06717932

Enrollment

Registered

2024-12-05

Start date

2025-05-15

Completion date

2039-11-01

Last updated

2026-02-10

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Beta-Thalassemia

Brief summary

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.

Detailed 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.

Interventions

None listed

Study design

Observational model

OTHER

Time perspective

PROSPECTIVE

Eligibility

Sex/Gender

ALL

Age

12 Years to 35 Years

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

Design outcomes

Primary

Measure	Time frame	Description
Occurrence of achieving transfusion independence for at least 12 consecutive months	Through 15 years post CS-101 infusion	—
Frequency and severity of SAEs and CS-101 related AES as assessed by CTCAE v5.0	Through 15 years post CS-101 infusion	CommonTerminology Criteria for Adverse Events#CTCAE#has 5 levels of AE determination, increasing in severity as the level increases
Occurrence of all-cause death	Through 15 years post CS-101 infusion	—

Secondary

Measure	Time frame
Chimerism level in bone marrow Proportion of alleles with intended genetic modification in	Through 15 years post CS-101 infusion
Change in F-Cell concentration over time	Through 15 years post CS-101 infusion
Chimerism level in Peripheral blood Proportion of alleles with intended genetic modification in	Through 15 years post CS-101 infusion
Change in fetal hemoglobin(HbF) concentration over time	Through 15 years post CS-101 infusion
Change in total hemoglobin(Hb) concentration over time	Through 15 years post CS-101 infusion

Countries

China

Outcome results