- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT06328764
CS-101 in Patients With β-thalassemia
A Clinical Study Evaluating the Safety and Efficacy of In-vitro tBE Edited Autologous Hematopoietic Stem Progenitor Cells(CS-101) in Treating Subjects With β-thalassemia
Study Overview
Detailed Description
Study Type
Enrollment (Estimated)
Phase
- Early Phase 1
Contacts and Locations
Study Locations
-
-
Guangxi
-
Nanning, Guangxi, China
- The First Affiliated Hospital of Guangxi Medical University
-
-
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
- Child
- Adult
Accepts Healthy Volunteers
Description
Inclusion Criteria:
- 6 to 35 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 History of at least≥8 units/year of packed RBC transfusions in the prior 12 months prior to the screening period 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
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, 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.
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%. 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.
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: CS-101
Autologous CD34+ hematopoietic stem cell suspension modified by in vitro base editing technique
|
Autologous CD34+ hematopoietic stem cell suspension modified by in vitro base editing technique
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Frequency and severity of adverse events(AEs)as assessed by CTCAE v5.0
Time Frame: From signing informed consent to 12 months post-CS-101 infusion
|
From signing informed consent to 12 months post-CS-101 infusion
|
|
Time to neutrophil and platelet engraftment
Time Frame: Days post-CS-101 infusion
|
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; 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;
|
Days post-CS-101 infusion
|
Proportion of subjects with engraftment
Time Frame: within 42 days post-CS-101infusion
|
Subjects with engraftment is defined as neutrophil engrafted
|
within 42 days post-CS-101infusion
|
Incidence of transplant-related mortality
Time Frame: From baseline to 100 days post-CS-101 infusion
|
From baseline to 100 days post-CS-101 infusion
|
|
All-cause mortality
Time Frame: From signing informed consent to 12 months post-CS-101 infusion
|
From signing informed consent to 12 months post-CS-101 infusion
|
|
Proportion of subjects achieving transfusion independence for at least 6 consecutive months
Time Frame: From 3 months up to 12 months post-CS-101 infusion
|
From 3 months up to 12 months post-CS-101 infusion
|
|
Time to last red blood cell(RBC) transfusion
Time Frame: Days post-CS-101 infusion
|
Days post-CS-101 infusion
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Change in total hemoglobin(Hb) concentration over time
Time Frame: up to 12 months post-CS-101 infusion
|
Total hemoglobin concentration change from baseline to 12 months post-CS-101 infusion
|
up to 12 months post-CS-101 infusion
|
Change in fetal hemoglobin(HbF) concentration over time
Time Frame: up to 12 months post-CS-101 infusion
|
γ-globin concentration change from baseline to 12 months post-CS-101 infusion
|
up to 12 months post-CS-101 infusion
|
Chimerism level in Peripheral blood and bone marrow
Time Frame: up to 12 months post-CS-101 infusion
|
Proportion of alleles with intended genetic modification in peripheral blood leukocytes and bone marrow over time
|
up to 12 months post-CS-101 infusion
|
Collaborators and Investigators
Collaborators
Investigators
- Principal Investigator: Yongrong Lai, M.D., First Affiliated Hospital of Guangxi Medical University
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Estimated)
Study Completion (Estimated)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- CS-101-11
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Beta-Thalassemia
-
M.D. Anderson Cancer CenterWithdrawnSickle Cell Disease | Sickle Beta Thalassemia | Beta Thalassemia Major | Sickle Cell-SS Disease | Sickle Beta 0 Thalassemia | Sickle Beta Plus ThalassemiaUnited States
-
CelgeneTerminatedBeta Thalassemia Intermedia | Beta Thalassemia MajorFrance, United Kingdom, Italy, Greece
-
University of British ColumbiaCompletedSickle Cell Disease | Beta-Thalassemia | Sickle Cell Trait | Sickle Cell-Beta Thalassemia | Sickle Cell-SS DiseaseCanada, Nepal
-
Editas Medicine, Inc.RecruitingHemoglobinopathies | Thalassemia Major | Thalassemia Intermedia | Transfusion Dependent Beta ThalassemiaUnited States, Canada
-
Agios Pharmaceuticals, Inc.Active, not recruitingNon-Transfusion-dependent Alpha-Thalassemia | Non-Transfusion-dependent Beta-ThalassemiaSpain, Taiwan, Thailand, United Kingdom, Malaysia, United States, Netherlands, Bulgaria, Turkey, Italy, Canada, Brazil, France, United Arab Emirates, Denmark, Greece, Lebanon, Saudi Arabia
-
Ionis Pharmaceuticals, Inc.TerminatedBeta Thalassemia IntermediaAustralia, Thailand, Greece, Lebanon, Turkey
-
Lantu BiopharmaNot yet recruitingBeta-Thalassemia
-
bluebird bioCenter for International Blood and Marrow Transplant ResearchRecruitingBeta-ThalassemiaUnited States
-
Children's Hospital of Fudan UniversityCorrectSequence Therapeutics Co., LtdRecruiting
-
Assiut UniversityNot yet recruiting
Clinical Trials on CS-101
-
CorrectSequence Therapeutics Co., LtdFirst Affiliated Hospital of Guangxi Medical UniversityActive, not recruiting
-
CorrectSequence Therapeutics Co., LtdRecruitingBeta-Thalassemia MajorChina
-
Children's Hospital of Fudan UniversityCorrectSequence Therapeutics Co., LtdRecruiting
-
Sunovion Respiratory Development Inc.CompletedChronic Obstructive Pulmonary Disease | COPDUnited States
-
Sunovion Respiratory Development Inc.Completed
-
University of OxfordCompleted
-
Virginia Polytechnic Institute and State UniversityRecruiting
-
London School of Hygiene and Tropical MedicineMedical Research Council; University of OxfordCompleted
-
Eximis SurgicalTerminatedLaparoscopic Gynecological SurgeryUnited States
-
Yan JinXijing Hospital; Tang-Du HospitalUnknownJoint Diseases | Osteochondritis | Osteochondritis DissecansChina