NCT06479616

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

A Long-term Follow-up Study Evaluating the Safety and Efficacy of Subjects With β-thalassemia Treated With Base-edited Autologous Hematopoietic Stem Cell (CS-101) Transplantation

• Status: Recruiting
• Phase: N/A
• Study type: Interventional
• Enrollment: 5 (estimated)
• Sponsor: Children's Hospital of Fudan University · Academic / Other
• Sex: All
• Age: 3 Years – 17 Years
• Healthy volunteers: Not accepted

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)

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 -101-03 (NCT06065189) . Subjects in the CS-101-03 study will be entered into long-term follow-up of this study up to 2 years post-infusion at the completion of the last (6-month) follow-up visit after treatment with CS-101 Injection.

Conditions

• Beta-Thalassemia

Interventions

Type	Name	Description
GENETIC	CS-101	Autologous CD34+ hematopoietic stem cell suspension modified by in vitro base editing technique

Timeline

• Start date: 2024-06-18
• Primary completion: 2026-12-01
• Completion: 2027-06-01
• First posted: 2024-06-28
• Last updated: 2025-09-10

Locations

1 site across 1 country: China