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Enrolling by Invitation

NCT Number: NCT06717932

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

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.

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Key information

Conditions

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

Age range

12 year–35 year

Sex eligibility

All sexes

Study type

Observational

Primary location

The First Affiliated Hospital of Guangxi Medical University

Nanning, China

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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 exclusion criteria

Treatment and study plan

Primary outcomes

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

    Time frame: 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

  2. Occurrence of all-cause death

    Time frame: Through 15 years post CS-101 infusion

  3. Occurrence of achieving transfusion independence for at least 12 consecutive months

    Time frame: Through 15 years post CS-101 infusion

Secondary outcomes

  1. Change in fetal hemoglobin(HbF) concentration over time

    Time frame: Through 15 years post CS-101 infusion

  2. Change in total hemoglobin(Hb) concentration over time

    Time frame: Through 15 years post CS-101 infusion

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

    Time frame: Through 15 years post CS-101 infusion

  4. Chimerism level in bone marrow Proportion of alleles with intended genetic modification in

    Time frame: Through 15 years post CS-101 infusion

  5. Change in F-Cell concentration over time

    Time frame: Through 15 years post CS-101 infusion

Sponsors and collaborators

Lead sponsor

CorrectSequence Therapeutics Co., Ltd

Industry

Registry information

Official study title

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

Important dates

Study start
2025
Primary completion
2039
Study completion
2039
First posted
Dec 5, 2024
Registry last updated
Feb 10, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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