Safety and Efficacy Evaluation of γ-globin Reactivated Autologous Hematopoietic Stem Cells

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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

December 25, 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