β-globin Restored Autologous HSC in β-thalassemia Major Patients
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The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. |
ClinicalTrials.gov Identifier: NCT04205435 |
Recruitment Status :
Not yet recruiting
First Posted : December 19, 2019
Last Update Posted : January 8, 2021
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
β-thalassemia Major | Biological: β-globin restored autologous HSC | Phase 1 Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 12 participants |
Allocation: | N/A |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | a Safety and Efficacy Study of β-globin Restored Autologous Hematopoietic Stem Cells for β-thalassemia Major Patients With CVS-654 Mutation |
Estimated Study Start Date : | March 1, 2021 |
Estimated Primary Completion Date : | December 1, 2022 |
Estimated Study Completion Date : | December 1, 2023 |

Arm | Intervention/treatment |
---|---|
Experimental: β-globin restored autologous HSC
each subject will accept one dose of β-globin restored autologous hematopoietic stem cells
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Biological: β-globin restored autologous HSC
gene edited autologous hematopoietic stem cells with β-globin restoration |
- safety evaluation of β-globin Restored HSC [ Time Frame: up to 6 months post transplant ]Proportion of engrafments; Overall survival; Incidence and Severity of Adverse Events as a Measure of Safety and Tolerability Adverse events assessed according to NCI-CTCAE v5.0 criteria;
- efficacy evaluation of β-globin restored autologous hematopoietic stem cells [ Time Frame: up to 24 months post transplant ]Proportion of subjects achieving transfusion independence for at least 6 months (TI6); Proportion of subjects achieving TI12; Proportion of alleles with intended genetic modification in bone marrow cells; Change in total hemoglobin and hemoglobin--beta concentration; Change from baselien in annualized frequency and volume of packed RBC transfusions.

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Ages Eligible for Study: | 5 Years to 15 Years (Child) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- 5-15 years old. Clinically diagnosed as β-thalassemia major with IVS-654 gene mutation phenotype;
- Subjects or at least one legal guardian/agent understand and voluntarily sign informed consent.
- Subjects with no affection with EBV, HIV, CMV, TP, HAV, HBV and HCV.
- Subjects body condition eligible for autologous stem cell transplant.
Exclusion Criteria:
- Subjects acceptable for allogeneic hematopoietic stem cell transplantation and have an available fully matched related donor.
Active bacterial, viral, or fungal infection. Treated with erythropoietin prior 3 months. Immediate family member with any known hematological tumor. Subjects with severe psychiatric disorders to be unable to cooperate. Recently diagnosed as malaria. History of complex autoimmune disease. Persistent aspartate transaminase (AST), alanine transaminase (ALT), or total bilirubin value >3 x the upper limit of normal (ULN).
Subjects with severe heart, lung and kidney diseases. With serious iron overload. Any other condition that would render the subject ineligible for HSCT, as determined by the attending transplant physician or Investigator.
Subjects who are receiving treatment from another clinical study, or have received another gene therapy.
Subjects or guardians had resisted the guidance of the attending doctor. Subjects whom the investigators do not consider appropriate for participating in this clinical study.

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04205435
Contact: Wei Li, PhD | +8618621670308 | wli@bioraylab.com |
China, Shanghai | |
Shanghai Bioraylaboratory Inc | |
Shanghai, Shanghai, China, 200241 | |
Contact: Wei Li, PhD wli@bioraylab.com | |
Principal Investigator: Xinhua Zhang, Prof |
Principal Investigator: | Xinhua Zhang, Prof | PLA 923 Hospital |
Responsible Party: | Bioray Laboratories |
ClinicalTrials.gov Identifier: | NCT04205435 |
Other Study ID Numbers: |
2019-BRL-00CH2 |
First Posted: | December 19, 2019 Key Record Dates |
Last Update Posted: | January 8, 2021 |
Last Verified: | January 2021 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | No |
Studies a U.S. FDA-regulated Drug Product: | No |
Studies a U.S. FDA-regulated Device Product: | No |
Thalassemia beta-Thalassemia Anemia, Hemolytic, Congenital Anemia, Hemolytic |
Anemia Hematologic Diseases Hemoglobinopathies Genetic Diseases, Inborn |