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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

NA

Total Enrollment

6 participants

Study Classification

INTERVENTIONAL

Study Start Date

2020-10-01

Study Completion Date

2023-11-27

Brief Summary

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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.

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Detailed Description

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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.

Conditions

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β Thalassemia Major

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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γ-globin reactivated autologous hematopoietic stem cells

each subject will accept one dose of γ-globin reactivated autologous hematopoietic stem cells

Group Type EXPERIMENTAL

γ-globin reactivated autologous hematopoietic stem cells

Intervention Type BIOLOGICAL

gene edited autologous hematopoietic stem cells with γ-globin expression

Interventions

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γ-globin reactivated autologous hematopoietic stem cells

gene edited autologous hematopoietic stem cells with γ-globin expression

Intervention Type BIOLOGICAL

Eligibility Criteria

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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.

Minimum Eligible Age

5 Years

Maximum Eligible Age

15 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No