Clinical trial

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

Name

CS-101-04

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)

Trial arms

Trial start

2024-06-18

Estimated PCD

2026-12-01

Trial end

2027-06-01

Status

Recruiting

Treatment

CS-101

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

Arms:

long term follow up

Size

Primary endpoint

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

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

Occurrence of all-cause death

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

New malignancies and hematologic disorders

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

Occurrence of achieving transfusion independence for at least 12 consecutive months

From 3 months after last RBC transfusion 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

Protocol

{'studyType': 'INTERVENTIONAL', 'phases': ['NA'], 'designInfo': {'allocation': 'NA', 'interventionModel': 'SINGLE_GROUP', 'primaryPurpose': 'OTHER', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 5, 'type': 'ESTIMATED'}}

Updated at

2024-06-28

1 organization

1 product

1 indication

Organization

Children's Hospital of Fudan University

Product

CS-101

Indication

Beta Thalassemia