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

Bioray Laboratories

Status

Completed

Conditions

β Thalassemia Major

Treatments

Biological: γ-globin reactivated autologous hematopoietic stem cells

Study type

Interventional

Funder types

Other

Industry

Identifiers

NCT04211480

2019-BRL-00CH1

Details and patient eligibility

About

This is a non-randomized, open label, single-dose, phase 1/2 study in up to 12 participants with β-thalassemia major.This study aims to evaluate the safety and efficacy of the treatment with γ-globin reactivated autologous hematopoietic stem cells in subjects with β-thalassemia major.

Full description

γ-globin reactivated autologous hematopoietic stem cells will be manufactured using Crispr/Cas9 gene editing system. Subject participation for this study will be 1 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.

Enrollment

6 patients

Sex

All

Ages

5 to 15 years old

Volunteers

No Healthy Volunteers

Inclusion criteria

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.

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.