Brief Title
Safety and Efficacy Evaluation of γ-globin Reactivated Autologous Hematopoietic Stem Cells
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
Brief Summary
This is a single arm, open label, single-dose, phase 1/2 study in up to 5 participants with
β-thalassemia major.The study will evaluate the safety and efficacy of the treatment with
γ-globin reactivated autologous hematopoietic stem cells in subjects with β-thalassemia
major.
Detailed Description
γ-globin reactivated autologous hematopoietic stem cells will be manufactured using
Glycosylase Base Editors. Subject participation for this study will be 2 year. Subjects who
enroll in this study will be asked to participate in a subsequent long-term follow up study
that will monitor the safety and efficacy of the treatment they receive for up to 15 years
post-transplant.
Study Type
Interventional
Primary Outcome
Proportion of subjects achieving successful neutrophil engraftment within 42 days after BRL-103 infusion
Secondary Outcome
Proportion of subjects achieving sustained transfusion independence for at least 3 months (TI3)
Condition
Thalassemia Major
Intervention
γ-globin reactivated autologous hematopoietic stem cells
Study Arms / Comparison Groups
γ-globin reactivated autologous hematopoietic stem cells
Description: each subject will accept one dose of γ-globin reactivated autologous hematopoietic stem cells
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Biological
Estimated Enrollment
5
Start Date
September 8, 2022
Completion Date
November 30, 2024
Primary Completion Date
September 8, 2024
Eligibility Criteria
Key inclusion criteria:
- Fully understand and voluntarily sign informed consent. 3-35years old. At least one
legal guardian and/or Subjects to sign informed consent.
- Clinically diagnosed as β-thalassemia major, phenotypes including β0β0, β+β+、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.
Key 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, 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
Gender
All
Ages
3 Years - 35 Years
Accepts Healthy Volunteers
No
Contacts
lai yongrong, PhD, +8618621670308, [email protected]
Location Countries
China
Location Countries
China
Administrative Informations
NCT ID
NCT05442346
Organization ID
2021-BRL-103
Responsible Party
Sponsor
Study Sponsor
Bioray Laboratories
Collaborators
First Affiliated Hospital of Guangxi Medical University
Study Sponsor
lai yongrong, PhD, Principal Investigator, First Affiliated Hospital of Guangxi Medical University
Verification Date
May 2022