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Recruiting NCT06479616

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

No phase Interventional 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
The protocol lists: CS-101.
Who it may be relevant to
Registry conditions: Beta-Thalassemia. Basic parameters: 3 years — 17 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 Base-edited Autologous Hematopoietic Stem Cell (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 -101-03 (NCT06065189)

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 -101-03 (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.

Interventions

  • Genetic CS-101
    Autologous CD34+ hematopoietic stem cell suspension modified by in vitro base editing technique

Primary outcome measures

  • 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]
  • Occurrence of all-cause death [Time frame: Signing of informed consent up to 2 years post CS-101 infusion]
  • New malignancies and hematologic disorders [Time frame: Signing of informed consent up to 2 years post CS-101 infusion]
  • 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 outcome measures (3)
  • Change in fetal hemoglobin(HbF) concentration over time [Time frame: up to 2 years post-CS-101 infusion]
  • Change in total hemoglobin(Hb) concentration over time [Time frame: up to 2 years post-CS-101 infusion]
  • 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]

Eligibility criteria

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

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

Healthy volunteers: No

Study design

Allocation
N/A
Model
Single group
Masking
Open label
Primary purpose
Other

Study locations

China · 1 center
  • Children's Hospital of Fudan University — Shanghai

Identifiers

NCT: NCT06479616 · CS-101-04

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗