A Long-term Follow-up Study in Participants Who Received CS-101

NCT06479616 · tracked via the Priya Life Science China tracker

Sponsor

Children's Hospital of Fudan University

Phase

Not applicable

Started

2024-06-18

Last updated

2025-09-10

Condition(s) studied

Beta-Thalassemia

Investigational drug(s) / intervention(s)

CS-101

CS-101: Autologous CD34+ hematopoietic stem cell suspension modified by in vitro base editing technique

Study 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 -101-03 (NCT06065189)

Eligibility

Sex

ALL

Min age

3 Years

Max age

17 Years

Healthy volunteers

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 CS-101 infusion in last IIT study Exclusion Criteria: * There are no exclusion criteria

Primary outcome measure(s)

Frequency and severity of SAEs and CS-101 related AES as assessed by CTCAE v5.0 — Signing of informed consent up to 2 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 — Signing of informed consent up to 2 years post CS-101 infusion
New malignancies and hematologic disorders — Signing of informed consent up to 2 years post CS-101 infusion
Based on ICD-11
Occurrence of achieving transfusion independence for at least 12 consecutive months — From 3 months after last RBC transfusion up to 2 years post CS-101 infusion

Trial sites (1)

Facility	City	Region	Status
Children's Hospital of Fudan University	Shanghai	Shanghai Municipality	Recruiting

More Children's Hospital of Fudan University trials in China

Clinical Cohort Study of DHA in Neurodevelopmental Disorders Active, not recruiting

Effects of Intermittent Dietary Restriction on Cardiometabolic Risk in School-aged Children Recruiting

Preoperative Serum FGF19 in the Prognosis of Biliary Atresia Recruiting

Differences in Prosocial Behaviors and Related Brain Networks in Children With Autism and Typical Development Recruiting

Close Loop Smart Weaning for INO With PPHN Recruiting

Other trials for the same condition

A Study to Evaluate Luspatercept (ACE-536) in Chinese Participants Who Require Regular Red Blood Cell Transfusions Due to Beta (β)-Thalassemia. Active, not recruiting

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

An Open-label Study of a Gene Therapy Product (Vebeglogene Autotemcel) in Transfusion Dependent Beta-Thalassemia Active, not recruiting

A Multiple Ascending Dose Study of 9MW3011 in Patients With Non-transfusion-dependent β-thalassemia Recruiting

CS-101 in Patients With β-thalassemia Enrolling by invitation

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06479616 on ClinicalTrials.gov ↗ ← All trials in China