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NCT Number: NCT06479616

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

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)

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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

3 year–17 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Not applicable

Primary location

Children's Hospital of Fudan University

Shanghai, Shanghai Municipality, 201102, China

Location status: Recruiting

Location contact

Zifeng Li, M.S.

CONTACT

[email protected]

+86-13920704768

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 -101-03 (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.

Who can participate

Healthy volunteers accepted: No

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

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

Treatment and study plan

CS-101

Genetic

Autologous CD34+ hematopoietic stem cell suspension modified by in vitro base editing technique

Primary outcomes

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

    Time frame: 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

  2. Occurrence of all-cause death

    Time frame: Signing of informed consent up to 2 years post CS-101 infusion

  3. New malignancies and hematologic disorders

    Time frame: Signing of informed consent up to 2 years post CS-101 infusion

    Based on ICD-11

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

    Time frame: From 3 months after last RBC transfusion up to 2 years post CS-101 infusion

Secondary outcomes

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

    Time frame: up to 2 years post-CS-101 infusion

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

    Time frame: up to 2 years post-CS-101 infusion

  3. Chimerism level in Peripheral blood and bone marrow Proportion of alleles with intended genetic modification in peripheral blood leukocytes and bone marrow over time

    Time frame: up to 2 years post-CS-101 infusion

    Proportion of alleles with intended genetic modification in peripheral blood leukocytes and bone marrow over time

Study contacts

Contact information is provided by the study sponsor or research team.

Xiaowen Zhai, M.D.

CONTACT

[email protected]

+86-021-64931126

Zifeng Li, M.S.

CONTACT

[email protected]

+86-13920704768

Sponsors and collaborators

Lead sponsor

Children's Hospital of Fudan University

Other

Registry information

Official study title

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

Important dates

Study start
2024
Primary completion
2026
Study completion
2027
First posted
Jun 28, 2024
Registry last updated
Sep 10, 2025

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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