A Single-arm, Open-label Phase I Clinical Trial to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics and Pharmacodynamic Profile of a Single Dose of CS-101 Injection in Subjects With β-thalassemia Major

CS-101-01

The goal of this open label, single-arm clinical study is to learn about the safety and efficacy of CS-101 in treating patients with β-thalassemia major anemia.

2024-04-18

2025-05-01

2025-07-01

Recruiting

Early phase I

8

Inclusion Criteria: * 7 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

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE1'], 'designInfo': {'allocation': 'NA', 'interventionModel': 'SINGLE_GROUP', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 8, 'type': 'ESTIMATED'}}

2024-05-01