Trials Sponsors Conditions Drugs Pricing

Conditions

Beta-Thalassemia Major

Outcome Measures

Primary Outcomes (8)

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
From signing informed consent to 12 months post-CS-101 infusion
Overall survival rate
Up to 12 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
Time to neutrophil engraftment is defined as first day of 3 consecutive measurements of absolute neutrophil count≥0.5×10\^9/L on three different days.
Up to 12 months post-CS-101 infusion
Time to platelet engraftment
Time to platelet engraftment is defined as first day of 3 consecutive measurements of absolute platelet count≥20×10\^9/L on three different days and without platelet transfusion.
Up to 12 months post-CS-101 infusion
Incidence of transplant-related mortality
Incidence of transplant-related mortality(Transplant-related mortality events defined as deaths assessed by the investigator as potentially transplant-related)
From baseline to 100 days post-CS-101 infusion
Change in fetal hemoglobin(HbF) concentration over time
Fetal hemoglobin(HbF) concentration from baseline to 9 months post-CS-101 infusion
Up to 12 months post-CS-101 infusion
Change in total hemoglobin(Hb) concentration over time
Total hemoglobin concentration change from baseline to 9 months post-CS-101 infusion
Up to 12 months post-CS-101 infusion

Secondary Outcomes (4)

Chimerism level in Peripheral blood and bone marrow
Up to 12 months post-CS-101 infusion
Proportion of subjects achieving transfusion independence for at least 6 consecutive months
From CS-101 infusion up to 12 months post-CS-101 infusion
Proportion of subjects achieving fetal hemoglobin(HbF) increase≥2.0 g/dL
Up to 2 months post-CS-101 infusion
Proportion of subjects achieving fetal hemoglobin(HbF) increase≥7.0 g/dL
Up to 6 months post-CS-101 infusion

Study Arms (1)

CS-101 injection

EXPERIMENTAL

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

Genetic: CS-101 injection

Interventions

CS-101 injectionGenetic

Autologous CD34+ hematopoietic stem cell suspension modified by in vitro 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:

to 35 years old(inclusive) male or female subjects at the time of informed consenting. For minors, their legal representative is required to sign the informed consent form, besides, if the subjects aged 8 years or older, they should provide a signed and dated.
Diagnosis of β-thalassemia major.

Sponsors & Collaborators

CorrectSequence Therapeutics Co., Ltd
Industrylead

Study Sites (3)

The First Affiliated Hospital of Guangxi Medical University

Nanning, Guangxi, China

Children's Hospital of Fudan University

Shanghai, Shanghai Municipality, China

Ruijin Hospital Shanghai JiaoTong University School of Medicine

Shanghai, Shanghai Municipality, China

MeSH Terms

Conditions

beta-Thalassemia

Condition Hierarchy (Ancestors)

ThalassemiaAnemia, Hemolytic, CongenitalAnemia, HemolyticAnemiaHematologic DiseasesHemic and Lymphatic DiseasesHemoglobinopathiesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

Yaliang Li
CorrectSequence Therapeutics Co., Ltd
STUDY DIRECTOR

Enrollment

9 participants (actual enrollment reported by the registry).

Study Design

Study Type: interventional
Phase: phase 1
Allocation: NA
Masking: NONE
Purpose: TREATMENT
Intervention Model: SINGLE GROUP
Sponsor Type: INDUSTRY
Responsible Party: SPONSOR

Study Record Dates

First Submitted

February 23, 2024

First Posted

March 4, 2024

Study Start

April 18, 2024

Actual (reported)

Primary Completion

You may not qualify if:

Treatment with other investigational medications or other experimental interventions 30 days prior to signing informed consent or within 6 half-lives of the drug, whichever is longer
Subjects who have received or are receiving thalidomide and/or Luspatercept in the past 6 months before screening
Previously received allogeneic hematopoietic stem cell transplantation or gene(edited) therapy
Subjects have available related fully matching donors and are eligible and prepared for allogeneic hematopoietic stem cell transplantation
Patients with coexisting α-thalassemia and more than 2 deletions or non-deletional mutations in the α-globin chain coding genes
Known to be allergic to drugs used during autologous hematopoietic stem cell transplantation (including but not limited to granulocyte colony-stimulating factor, busulfan, dextran), excipients(such as dimethyl sulfoxide), or instruments(such as intravenous catheters) as determined by the investigator are deemed unsuitable to participate in this study
Those with positive results in HIV, cytomegalovirus, Epstein-Barr virus and treponema pallidum test, active infection of hepatitis B, hepatitis C, or known tuberculosis, parasitic infection, etc. Hepatitis B stabilized on medication(HBV-DNA test negative) and cured hepatitis C(HCV-RNA test negative) can be considered acceptable.
Echocardiography shows ejection fraction below 45%
Laboratory indicators, defined as：Aspartate aminotransferase(AST), alanine aminotransferase(ALT) \>3× upper limit of normal(ULN) or Baseline International Normalized Ratio(INR)\>1.5×ULN.
MRI during the screening period shows severe cardiac iron overload and other conditions, and are judged by the investigator to be intolerable or inappropriate for autologous hematopoietic stem cell transplantation
Patients with past/present history of cancer
Known neurological disorders, psychological problems or mental illness, and is judged by the investigator to be unable to cooperate with the study procedures
Known history of uncontrolled epileptic seizures and is judged by the investigator to be unfit to participate in this study
The investigators determined that a non-hypersplenism-induced white blood cell count of\<3×10\^9/L, and/or a platelet count of\<100×10\^9/L.
Known history of other serious cardiovascular, pulmonary, renal diseases, digestive tract conditions, liver diseases and / or other conditions, etc., and are judged by the investigator to be intolerable or inappropriate for autologous hematopoietic stem cell mobilization, collection, and myeloablative conditioning and infusion
+1 more criteria

A Safety and Efficacy Study Evaluating CS-101 in Subjects 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 (8)

Secondary Outcomes (4)

Study Arms (1)

CS-101 injection

Interventions

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (3)

MeSH Terms

Conditions

Condition Hierarchy (Ancestors)

Study Officials

Enrollment

Study Design

Study Record Dates

You may not qualify if:

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 (8)

Secondary Outcomes (4)

Study Arms (1)

CS-101 injection

Interventions

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (3)

MeSH Terms

Conditions

Condition Hierarchy (Ancestors)

Study Officials

Enrollment

Study Design

Study Record Dates

You may not qualify if:

Source record

Locations