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

NCT06685536 | Enrolling By Invitation

• 1 other identifier: CS-101-13
• Study Type: observational
• Target: 5
• Locations: 1 country
• Sites: 1

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 -101-06 (NCT06024876),No investigational drug product will be administered in the study.

Conditions

Beta-Thalassemia

Outcome Measures

Primary Outcomes (4)

• Frequency and severity of SAEs and CS-101 related AES as assessed by CTCAE v5.0

  CommonTerminology Criteria for Adverse Events#CTCAE#has 5 levels of AE determination, increasing in severity as the level increases

  Through 15 years post CS-101 infusion

• New malignancies and hematologic disorders

  Based on ICD-11

  Through 15 years post CS-101 infusion

• Occurrence of all-cause death

  Through 15 years post CS-101 infusion

• Occurrence of achieving transfusion independence for at least 12 consecutive months

  Through 15 years post CS-101 infusion

Secondary Outcomes (5)

• 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

• Chimerism level in Peripheral blood Proportion of alleles with intended genetic modification in

  Through 15 years post CS-101 infusion

• 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

Interventions

Safety and efficacy assessments OTHER

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

Eligibility Criteria

• Age: 6 Years - 35 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)
• Sampling Method: Non-Probability Sample

Study Population

Subject with β-thalassemia treated with CS-101 infusion in last study

You may qualify if:

• 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 study

Sponsors & Collaborators

• First Affiliated Hospital of Guangxi Medical University: collaborator
• CorrectSequence Therapeutics Co., Ltd: lead

Study Sites (1)

The First Affiliated Hospital of Guangxi Medical University

Nanning, Guangxi, China

Location

MeSH Terms

Conditions

beta-Thalassemia

Interventions

Safety

Condition Hierarchy (Ancestors)

Thalassemia; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Accident Prevention; Accidents; Public Health; Environment and Public Health

Study Officials

• Yongrong Lai, M.D.

  First Affiliated Hospital of Guangxi Medical University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: OTHER
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 7, 2024
• First Posted: November 12, 2024
• Study Start: January 18, 2025
• Primary Completion (Estimated): July 1, 2039
• Study Completion (Estimated): July 1, 2039
• Last Updated: February 10, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)