A Clinical Study Evaluating the Safety and Efficacy of CS-101 in Treating Subjects With β-thalassemia

NCT06024876 | Completed Early Phase 1

• 1 other identifier: CS-101-06
• Study Type: interventional
• Target: 5
• Locations: 1 country
• Sites: 1

Brief Summary

The goal of this open label, single-arm clinical study is to learn about the safety and efficacy of CS-101 in treating β-thalassemia.

Conditions

Beta-Thalassemia

Outcome Measures

Primary Outcomes (7)

• Frequency and severity of adverse events(AEs）as assessed by CTCAE v5.0

  From signing informed consent to 12 months post-CS-101 infusion

• Time to neutrophil and platelet engraftment

  Time to neutrophil engraftment is defined as first day of 3 consecutive measurements of absolute neutrophil count≥0.5×10\^9/L on three different days； Time to platelet engraftment is defined as first day of 3 consecutive measurements of absolute platelet count≥20×10\^9/L on three different days and without platelet transfusion in the past 7 days；

  Days post-CS-101 infusion

• Proportion of subjects with engraftment

  Subjects with engraftment is defined as neutrophil engrafted

  within 42 days post-CS-101infusion

• Incidence of transplant-related mortality

  From baseline to 100 days post-CS-101 infusion

• All-cause mortality

  From signing informed consent to 12 months post-CS-101 infusion

• Proportion of subjects achieving transfusion independence for at least 6 consecutive months

  From 3 months up to 12 months post-CS-101 infusion

• Time to last red blood cell(RBC) transfusion

  Days post-CS-101 infusion

Secondary Outcomes (3)

• Change in total hemoglobin(Hb) concentration over time

  up to 12 months post-CS-101 infusion

• Change in fetal hemoglobin(HbF) concentration over time

  up to 12 months post-CS-101 infusion

• Chimerism level in Peripheral blood and bone marrow

  up to 12 months post-CS-101 infusion

Study Arms (1)

CS-101

EXPERIMENTAL

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

Biological: CS-101

Interventions

CS-101 BIOLOGICAL

Autologous CD34+ hematopoietic stem cell suspension modified by ex vivo base editing technique

CS-101

Eligibility Criteria

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

You may qualify if:

• to 35 years old(inclusive) male or female subjects at the time of informed consenting
• Diagnosis of β-thalassemia, genotypes include but are not limited to β+β0，βEβ0，β0β0, etc
• History of at least≥8 units/year of packed RBC transfusions in the prior 12 months prior to the screening period
• Generally in good condition, Karnofsky performance score≥60 points for subjects≥16 years old at the time of autologous hematopoietic stem cell collection, or Lansky Play-Performance score≥60 points for subjects under 16 years old, or equivalent clinical evaluation as the investigator site's common practice

You may not qualify if:

• Treatment with other investigational medications or other experimental interventions 30 days prior to signing informed consent or within 6 half-lives of the drug, whichever is longer.
• Subjects who have received or are receiving thalidomide and/or Luspatercept, when their drug-drug interaction on the efficacy and safety of CS-101 cannot be ruled out, unless at least there are 3 test results showing the total hemoglobin level before transfusion is below 9g/dL in the past 6 months before screening.
• Previously received allogeneic hematopoietic stem cell transplantation or gene(edited) therapy.
• Subjects have available related fully matching donors and are eligible and prepared for allogeneic hematopoietic stem cell transplantation.
• Those with active infections, including but not limited to: HIV, hepatitis B, hepatitis C, cytomegalovirus, Epstein-Barr virus and treponema pallidum test positive, or known tuberculosis, parasitic infection, etc. who are judged by the investigator to be unsuitable to participate in this study.
• Echocardiography results with ejection fraction below 45%.
• Advanced liver disease, defined as：
• Aspartate aminotransferase (AST), alanine aminotransferase (ALT) \>3 × upper limit of normal (ULN) or:
• Baseline International Normalized Ratio (INR) \>1.5 × ULN.
• MRI during the screening period showed heavy iron overload and is judged by the investigator to be unable to participate in the study.

Sponsors & Collaborators

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

Study Sites (1)

The First Affiliated Hospital of Guangxi Medical University

Nanning, China

Location

MeSH Terms

Conditions

beta-Thalassemia

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

Study Officials

• Yongrong Lai, M.D.

  First Affiliated Hospital of Guangxi Medical University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 23, 2023
• First Posted: September 6, 2023
• Study Start: August 26, 2023
• Primary Completion: July 1, 2025
• Study Completion: July 1, 2025
• Last Updated: February 12, 2026

Record last verified: 2026-02

Locations

CN (1)