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Gene Therapy Trial Report

Summary

A Safety and Efficacy Study Evaluating CS-101 in Subjects With β-Thalassemia Major


NCTID NCT06291961 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Beta-Thalassemia Major
Disease Ontology Term DOID:0080771
Compound Name CS-101
Sponsor CorrectSequence Therapeutics Co., Ltd
Funder Type Industry
Recruitment Status
Completed
Enrollment Count 9 (ACTUAL)
Results Posted Not Available

Therapy Information


Target Gene/Variant BCL11A
Therapy Type Gene editing
Therapy Route Ex-vivo
Mechanism of Action Overexpression of protective allele/gene
Route of Administration Intravenous
Drug Product Type Autologous cells
Target Tissue/Cell CD34+ cells
Delivery System Electroporation
Vector Type none
Editor Type transformer BE RNP
Dose 1 Transduced CD34+ cells

Study Record Dates


Current Stage Phase1
Submit Date 2024-02-23
Completion Date 2025-11-17
Last Update 2026-02-10

Participation Criteria


Eligible Age 12 Years - 35 Years
Standard Ages Child, Adult
Sexes Eligible for Study ALL
Eligibility Criteria
Inclusion Criteria: * 12 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. * Generally in good condition, Karnofsky performance score≥60 points for subjects≥16 years old, or Lansky Play-Performance score≥60 points for subjects under 16 years old. * For female subjects of childbearing potential: use effective contraceptive measures from the start of screening and agree to continue using such measures for contraception throughout the study * For male subjects who have a potential ability to father a child: use condoms or other methods continuously from the start of mobilization to ensure effective contraception for sexual partners during the study period Exclusion Criteria: * 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 * Pregnant or lactating women
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 3
Locations China

Regulatory Information


Has US IND False
FDA Designations
Recent Updates

Resources/Links