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

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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04205435
Recruitment Status : Not yet recruiting
First Posted : December 19, 2019
Last Update Posted : December 19, 2019
Sponsor:
Collaborator:
Xiangya Hospital of Central South University
Information provided by (Responsible Party):
Shanghai Bioray Laboratory Inc.

Study Description
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Brief Summary:
This is a non-randomized, open label, single-site, 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.

Condition or disease Intervention/treatment Phase
β-thalassemia Major Biological: γ-globin reactivated autologous hematopoietic stem cells Phase 1 Phase 2

Detailed 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.
Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 12 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: a Single Center, 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
Estimated Study Start Date : March 1, 2020
Estimated Primary Completion Date : March 1, 2021
Estimated Study Completion Date : January 1, 2022

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Thalassemia

Arms and Interventions
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Arm Intervention/treatment
Experimental: γ-globin reactivated autologous hematopoietic stem cells
each subject will accept one dose of γ-globin reactivated autologous hematopoietic stem cells
 Biological: γ-globin reactivated autologous hematopoietic stem cells
gene edited autologous hematopoietic stem cells with γ-globin expression


Outcome Measures
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Primary Outcome Measures :
  1. Incidence and Severity of Adverse Events as a Measure of Safety and Tolerability Adverse events assessed according to NCI-CTCAE v4.03 criteria [ Time Frame: up to 6 months post transplant ]

Secondary Outcome Measures :
  1. efficacy of γ-globin reactivated autologous hematopoietic stem cells [ Time Frame: 12 months post transplant ]
    Independence on blood transfusion after transplantation; Or, The amount of red cell transfusion in a year post transplantation reduced 20% than that of the last year.


Eligibility Criteria
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Ages Eligible for Study:   5 Years to 30 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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 acceptable for autologous hematopoietic stem cell transplantation.

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. 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.

Contacts and Locations
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Information from the National Library of Medicine

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


Contacts
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Contact: Wei Li, PhD +8618621670308 wli@bioraylab.com

Sponsors and Collaborators
Shanghai Bioray Laboratory Inc.
Xiangya Hospital of Central South University
Investigators
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Principal Investigator: Bin Fu, Prof Xiangya Hospital Central University
More Information
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Responsible Party: Shanghai Bioray Laboratory Inc.
ClinicalTrials.gov Identifier: NCT04205435    
Other Study ID Numbers: 2019-BRL-00CH1
First Posted: December 19, 2019    Key Record Dates
Last Update Posted: December 19, 2019
Last Verified: December 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Thalassemia
beta-Thalassemia
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
 Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn