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Enrolling by invitation NCT06717932

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

— Observational Beta-Thalassemia

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
This is an observational study: the protocol does not assign a study treatment.
Who it may be relevant to
Registry conditions: Beta-Thalassemia. Basic parameters: 12 years — 35 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
China
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

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Official title

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

Overview

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.

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 -101-01 (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.

Primary outcome measures

  • 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]
  • Occurrence of all-cause death [Time frame: Through 15 years post CS-101 infusion]
  • Occurrence of achieving transfusion independence for at least 12 consecutive months [Time frame: Through 15 years post CS-101 infusion]
Secondary outcome measures (5)
  • Change in fetal hemoglobin(HbF) concentration over time [Time frame: Through 15 years post CS-101 infusion]
  • Change in total hemoglobin(Hb) concentration over time [Time frame: Through 15 years post CS-101 infusion]
  • Chimerism level in Peripheral blood Proportion of alleles with intended genetic modification in [Time frame: Through 15 years post CS-101 infusion]
  • Chimerism level in bone marrow Proportion of alleles with intended genetic modification in [Time frame: Through 15 years post CS-101 infusion]
  • Change in F-Cell concentration over time [Time frame: Through 15 years post CS-101 infusion]

Eligibility criteria

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

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Observational model
Other

Study locations

China · 1 center
  • The First Affiliated Hospital of Guangxi Medical University — Nanning

Identifiers

NCT: NCT06717932 · CS-101-05

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗