β-globin Restored Autologous HSC in β-thalassemia Major Patients
|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. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT04205435|
Recruitment Status : Terminated (Sponsor decision)
First Posted : December 19, 2019
Last Update Posted : October 13, 2022
- Study Details
- Tabular View
- No Results Posted
- How to Read a Study Record
|Condition or disease||Intervention/treatment||Phase|
|β-thalassemia Major||Biological: β-globin restored autologous HSC||Phase 1 Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||2 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||a Safety and Efficacy Study of β-globin Restored Autologous Hematopoietic Stem Cells for β-thalassemia Major Patients With CVS-654 Mutation|
|Actual Study Start Date :||November 1, 2021|
|Actual Primary Completion Date :||June 1, 2022|
|Actual Study Completion Date :||July 25, 2022|
Experimental: β-globin restored autologous HSC
each subject will accept one dose of β-globin restored autologous hematopoietic stem cells
Biological: β-globin restored autologous HSC
gene edited autologous hematopoietic stem cells with β-globin restoration
- Proportion of subjects with engraftment; [ Time Frame: up to 42 days post transplant ]
- 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 60 days post transplant ]
- Proportion of subjects achieving transfusion independence; [ Time Frame: up to 24 months post transplant ]
- Proportion of subjects with a > = 50% reduced annualized volume of packed RBC transfusions. [ Time Frame: up to 24 months post transplant ]
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
|Ages Eligible for Study:||5 Years to 15 Years (Child)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
- 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.
- 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
|Shanghai Bioraylaboratory Inc|
|Shanghai, Shanghai, China, 200241|
|Principal Investigator:||Xinhua Zhang, Prof||PLA 923 Hospital|
|Responsible Party:||Bioray Laboratories|
|Other Study ID Numbers:||
|First Posted:||December 19, 2019 Key Record Dates|
|Last Update Posted:||October 13, 2022|
|Last Verified:||September 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|
Anemia, Hemolytic, Congenital
Genetic Diseases, Inborn