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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. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04211480
Recruitment Status : Active, not recruiting
First Posted : December 26, 2019
Last Update Posted : October 12, 2022
Sponsor:
Collaborators:
Xiangya Hospital of Central South University
PLA 923 Hospital
Information provided by (Responsible Party):
Bioray Laboratories

Study Description
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Brief Summary:
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.

Condition or disease Intervention/treatment Phase
β Thalassemia Major Biological: γ-globin reactivated autologous hematopoietic stem cells Not Applicable

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)
Actual Enrollment : 6 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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
Actual Study Start Date : April 1, 2020
Estimated Primary Completion Date : October 15, 2023
Estimated Study Completion Date : December 1, 2023

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. Safety evaluation of γ-globin reactivated autologous hematopoietic stem cells [ Time Frame: up to 24 months post transplant ]
    Proportion of subjects with engraftment; Overall survival.

  2. Incidence and severity of adverse events as a measure of safety and tolerability. Adverse events assessed according to NCI-CTCAE v5.0 criteria [ Time Frame: up to 24 months post transplant ]
    Incidence of AEs and SAEs post transplant


Secondary Outcome Measures :
  1. Efficacy evaluation of γ-globin reactivated autologous hematopoietic stem cells [ Time Frame: up to 24 months post transplant ]
    Proportion of subjects achieving transfusion independence for at least 6 months (TI6); Proportion of subjects achieving TI12; Proportion of alleles with intended genetic modification in bone marrow cells; Change in total hemoglobin concentration; Change from baseline in annualized frequency and volume of packed RBC transfusions.


Eligibility Criteria
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Information from the National Library of Medicine

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Ages Eligible for Study:   5 Years to 15 Years   (Child)
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 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.
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): NCT04211480


Locations
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China, Shanghai
Shanghai Bioray Laboratories Inc.
Shanghai, Shanghai, China, 200241
Sponsors and Collaborators
Bioray Laboratories
Xiangya Hospital of Central South University
PLA 923 Hospital
Investigators
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Principal Investigator: Bin Fu, Prof. Xiangya Hospital Central University
More Information
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Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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Responsible Party: Bioray Laboratories
ClinicalTrials.gov Identifier: NCT04211480    
Other Study ID Numbers: 2019-BRL-00CH1
First Posted: December 26, 2019    Key Record Dates
Last Update Posted: October 12, 2022
Last Verified: October 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: Individual participant data (IPD) that underlie the results reported in published article will be shared, after deidentification (text, tables,figures and appendices). Other available documents include study protocol.
Supporting Materials: Study Protocol
Time Frame: IPD sharing will begin at 6 months and end at 36 months following article publication.
Access Criteria: IPD will be shared with investigators for individual data meta-analysis, after their proposed use of the data has been approved by an independent review committee. Proposals should be directed to yxwu@bio.ecnu.edu.cn and fu.bin@csu.edu.cn. To gain access, data requestors will need to sign a data access agreement.

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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