Safety and Efficacy Evaluation of γ-globin Reactivated Autologous Hematopoietic Stem Cells
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|ClinicalTrials.gov Identifier: NCT04211480|
Recruitment Status : Active, not recruiting
First Posted : December 26, 2019
Last Update Posted : October 12, 2022
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|Condition or disease||Intervention/treatment||Phase|
|β Thalassemia Major||Biological: γ-globin reactivated autologous hematopoietic stem cells||Not Applicable|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||6 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||an Open Label Trial of Evaluation of the Safety and Efficacy of Treatment With γ-globin Reactivated Autologous Hematopoietic Stem Cells in Subjects With β-thalassemia Major|
|Actual Study Start Date :||April 1, 2020|
|Estimated Primary Completion Date :||October 15, 2023|
|Estimated Study Completion Date :||December 1, 2023|
Experimental: γ-globin reactivated autologous hematopoietic stem cells
each subject will accept one dose of γ-globin reactivated autologous hematopoietic stem cells
Biological: γ-globin reactivated autologous hematopoietic stem cells
gene edited autologous hematopoietic stem cells with γ-globin expression
- Safety evaluation of γ-globin reactivated autologous hematopoietic stem cells [ Time Frame: up to 24 months post transplant ]Proportion of subjects with engraftment; 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 [ Time Frame: up to 24 months post transplant ]Incidence of AEs and SAEs post transplant
- Efficacy evaluation of γ-globin reactivated 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 concentration; Change from baseline 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|
- Fully understand and voluntarily sign informed consent. 5-15years old. At least one legal guardian and/or Subjects to sign informed consent.
- Clinically diagnosed as β-thalassemia major, phenotypes including β0β0, β+β0,βEβ0 genotype.
- Subjects with no affection with EBV, HIV, CMV, TP, HAV, HBV and HCV.
- Subjects body condition eligible for autologous stem cell transplant.
- 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, serum ferritin>5000mg/ml.
- 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): NCT04211480
|Shanghai Bioray Laboratories Inc.|
|Shanghai, Shanghai, China, 200241|
|Principal Investigator:||Bin Fu, Prof.||Xiangya Hospital Central University|
|Responsible Party:||Bioray Laboratories|
|Other Study ID Numbers:||
|First Posted:||December 26, 2019 Key Record Dates|
|Last Update Posted:||October 12, 2022|
|Last Verified:||October 2022|
|Individual Participant Data (IPD) Sharing Statement:|
|Plan to Share IPD:||Yes|
|Plan Description:||Individual participant data (IPD) that underlie the results reported in published article will be shared, after deidentification (text, tables,figures and appendices). Other available documents include study protocol.|
|Time Frame:||IPD sharing will begin at 6 months and end at 36 months following article publication.|
|Access Criteria:||IPD will be shared with investigators for individual data meta-analysis, after their proposed use of the data has been approved by an independent review committee. Proposals should be directed to firstname.lastname@example.org and email@example.com. To gain access, data requestors will need to sign a data access agreement.|
|Studies a U.S. FDA-regulated Drug Product:||No|
|Studies a U.S. FDA-regulated Device Product:||No|
Anemia, Hemolytic, Congenital
Genetic Diseases, Inborn