Trials Sponsors Conditions Drugs Pricing

Conditions

β-thalassemia Major

Outcome Measures

Primary Outcomes (7)

AEs(Adverse Events) and SAEs(Serious Adverse Events) after CS-101 infusion
Frequency and severity of adverse events(AEs）as assessed by CTCAE(Common Terminology Criteria for Adverse Events)v5.0
Up to 16 months post-CS-101 infusion
Overall survival
Up to 16 months post-CS-101 infusion
Proportion of Subjects with engraftment
Subjects with engraftment is defined as neutrophil engrafted
Within 42 days post-CS-101 infusion
Time to neutrophil engraftment
Up to 16 months post-CS-101 infusion
Time to platelet engraftment
Up to 16 months post-CS-101 infusion
Incidence of transplant-related mortality
Up to 100 days post-CS-101 infusion
Proportion of subjects achieving transfusion independence for at least 12 consecutive months
Maintaining transfusion independence for at least 12 consecutive months while maintaining a weighted mean hemoglobin≥90g/L
Up to 16 months post-CS-101 infusion

Secondary Outcomes (5)

Proportion of subjects achieving transfusion independence for at least 6 consecutive months
Up to 16 months post CS-101 Infusion
Changes in targeted editing efficiency in peripheral blood nucleated cells over time
Up to 16 months post CS-101 infusion
Changes in targeted editing efficiency in bone marrow nucleated cells over time
Up to 16 months post CS-101 infusion
Change in fetal hemoglobin(HbF) concentration over time
Up to 16 months post CS-101 infusion
Change in total hemoglobin(Hb) concentration over time
Up to 16 months post CS-101 infusion

Study Arms (1)

CS-101 injection

EXPERIMENTAL

Autologous CD34+ hematopoietic stem cell suspension modified by ex vivo base editing technique

Genetic: CS-101 injection

Interventions

CS-101 injectionGenetic

Autologous CD34+ hematopoietic stem cell suspension modified by ex vivo base editing technique

CS-101 injection

Registry-reported names are shown in full. Names without a link have not yet been matched to a Syfrah profile.

Eligibility Criteria

Age12 Years - 35 Years

Sexall

Healthy VolunteersNo

Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

Voluntarily signed informed consent. Male or female participants aged 12 to 35 years (inclusive). The participant or their legally authorized representative must sign the informed consent. If the participant is under 18 years of age, their legally authorized representative must also sign the informed consent.

Sponsors & Collaborators

CorrectSequence Therapeutics Co., Ltd
Industrylead

Study Sites (3)

The First Affiliated Hospital of Guangxi Medical University

Nanning, Guangxi, China

Yongrong LaiCONTACT

Yongrong LaiPRINCIPAL INVESTIGATOR

Children's Hospital of Fudan University

Shanghai, Shanghai Municipality, China

Xiaowen ZhaiCONTACT

Xiaowen ZhaiPRINCIPAL INVESTIGATOR

Ruijin Hospital Shanghai JiaoTong University School of Medicine

Shanghai, Shanghai Municipality, China

Saijuan ChenPRINCIPAL INVESTIGATOR

Weili ZhaoPRINCIPAL INVESTIGATOR

Central Study Contacts

Xiaokai Li

CONTACT

+86 18686610731 xiaokai.li@correctsequence.com

Yaliang Li

CONTACT

+8618621046122

Study Design

Study Type: interventional
Phase: phase 2
Allocation: NA
Masking: NONE
Purpose: TREATMENT
Intervention Model: SINGLE GROUP
Sponsor Type: INDUSTRY
Responsible Party: SPONSOR

Study Record Dates

First Submitted

March 19, 2026

First Posted

March 24, 2026

Study Start

April 5, 2026

Estimated (date passed)

This estimate has passed. The registry has not confirmed an actual date for this milestone.

Primary Completion

January 31, 2028

Estimated

You may not qualify if:

Received other investigational products or other experimental interventions within 30 days prior to signing informed consent or within 6 elimination half-lives of the drug (whichever is longer).
Received or is receiving thalidomide, hydroxyurea, and/or luspatercept within 3 months prior to screening.
Previous received allogeneic hematopoietic stem cell transplantation, gene therapy, or gene-editing therapy; or participants who can be maintained with standard therapy.
Participants with a matched sibling donor, or with a matched unrelated / haploidentical related donor and judged by the investigator to have no high-risk factors for allogeneic hematopoietic stem cell transplantation.
Participants with coexisting α-thalassemia with more than 2 α-globin chain gene deletions or non-deletional mutations.
Known hypersensitivity to drugs used during autologous hematopoietic stem cell transplantation, excipients, or devices, judged by the investigator to be ineligible for this study.
Infection with HIV, cytomegalovirus, Epstein-Barr virus, or Treponema pallidum during screening; active HBV or HCV infection (participants with stable hepatitis B after treatment (HBV-DNA negative) and cured hepatitis C (HCV-RNA negative) may be included). Known active bacterial, viral, fungal, or parasitic infection.
Echocardiographic ejection fraction \< 50%.
Laboratory abnormalities: AST or ALT \> 3 × upper limit of normal (ULN); or International normalized ratio (INR) \> 1.5 × ULN.
Cardiac severe iron overload detected by MRI during screening, judged by the investigator to be unsuitable for hematopoietic stem cell transplantation.
Current or history of malignancy.
Participants with known neurological consciousness disorders, psychological problems, or psychiatric diseases judged by the investigator to be unable to comply with study procedures.
Participants with known history of uncontrolled seizures judged by the investigator to be ineligible for this study.
Uncontrolled bleeding disorders.
Leukocyte count \< 3 × 10⁹/L and/or platelet count \< 100 × 10⁹/L not due to hypersplenism.
+4 more criteria

A Phase 2 Safety and Efficacy Study Evaluating CS-101 in Participants With β-Thalassemia Major

Brief Summary

Registry Overview

Trial Relationships

Study Timeline

First Submitted

First Posted

Study Start

Primary Completion

Study Completion

Conditions

Outcome Measures

Primary Outcomes (7)

Secondary Outcomes (5)

Study Arms (1)

CS-101 injection

Interventions

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (3)

Central Study Contacts

Study Design

Study Record Dates

You may not qualify if:

Data Sharing

Source record

Locations

Brief Summary

Registry Overview

Trial Relationships

Related Scientific Literature

Study Timeline

First Submitted

First Posted

Study Start

Primary Completion

Study Completion

Conditions

Outcome Measures

Primary Outcomes (7)

Secondary Outcomes (5)

Study Arms (1)

CS-101 injection

Interventions

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (3)

Central Study Contacts

Study Design

Study Record Dates

You may not qualify if:

Data Sharing

Source record

Locations