Status:
ACTIVE_NOT_RECRUITING
Base-edited Autologous Hematopoietic Stem Cell Transplantation in Treating Patients With β-thalassemia Major
Lead Sponsor:
Children's Hospital of Fudan University
Collaborating Sponsors:
CorrectSequence Therapeutics Co., Ltd
Conditions:
Beta-Thalassemia
Eligibility:
All Genders
3-17 years
Phase:
EARLY_PHASE1
Brief Summary
The goal of this open label, single-arm clinical study is to learn about the safety and efficacy of base-edited autologous hematopoietic stem cell transplantation(CS-101) in treating patients with β-t...
Detailed Description
CS-101 is an autologous CD34+ cell suspension modified by ex vivo base editing technology, removing the inhibitory effect of BCL11A on the γ-globin coding gene, inducing the production of γ-globin cha...
Eligibility Criteria
Inclusion
- 3 to 17 years old(inclusive) male or female subjects at the time of informed consenting
- Diagnosis of β-thalassemia, genotypes include but are not limited to β+β0, βEβ0, β0β0, etc
- Generally in good condition, Karnofsky performance score≥60 points for subjects≥16 years old at the time of autologous hematopoietic stem cell collection, or Lansky Play-Performance score≥60 points for subjects under 16 years old, or equivalent clinical evaluation as the investigator site's common practice
- For female subjects of childbearing potential: use effective contraceptive measures for at least 1 month prior to 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
- 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, when their drug-drug interaction on the efficacy and safety of CS-101 cannot be ruled out, unless at least there are 3 test results showing the total hemoglobin level before transfusion is below 9g/dL 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.
- Subjects 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 active infections, including but not limited to: HIV, hepatitis B, hepatitis C, cytomegalovirus, Epstein-Barr virus and treponema pallidum test positive, or known tuberculosis, parasitic infection, etc. who are judged by the investigator to be unsuitable to participate in this study
- Echocardiography results with ejection fraction below 45%
- Subjects who are febrile (temperature over 37.3° C) should be held back from enrolment.
- Advanced liver disease, 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 showed heavy iron overload and is judged by the investigator to be unable to participate in the study.
- 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
- 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
Key Trial Info
Start Date :
November 7 2023
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
September 30 2025
Estimated Enrollment :
5 Patients enrolled
Trial Details
Trial ID
NCT06065189
Start Date
November 7 2023
End Date
September 30 2025
Last Update
February 10 2025
Active Locations (1)
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1
Children's Hospital of Fudan University
Shanghai, Shanghai Municipality, China, 201102