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β-globin Restored Autologous HSC in β-thalassemia Major Patients

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: NCT04205435
Recruitment Status : Terminated (Sponsor decision)
First Posted : December 19, 2019
Last Update Posted : October 13, 2022
PLA 923 Hospital
Information provided by (Responsible Party):
Bioray Laboratories

Brief Summary:
This is a single center, single arm, open-label study to determine the safety and efficacy of β-globin restored autologous hematopoietic stem cells in β- thalassemia major patients with CVS-654 mutation.

Condition or disease Intervention/treatment Phase
β-thalassemia Major Biological: β-globin restored autologous HSC Phase 1 Phase 2

Detailed Description:
β-globin restored 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.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 2 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: a Safety and Efficacy Study of β-globin Restored Autologous Hematopoietic Stem Cells for β-thalassemia Major Patients With CVS-654 Mutation
Actual Study Start Date : November 1, 2021
Actual Primary Completion Date : June 1, 2022
Actual Study Completion Date : July 25, 2022

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Thalassemia

Arm Intervention/treatment
Experimental: β-globin restored autologous HSC
each subject will accept one dose of β-globin restored autologous hematopoietic stem cells
Biological: β-globin restored autologous HSC
gene edited autologous hematopoietic stem cells with β-globin restoration

Primary Outcome Measures :
  1. Proportion of subjects with engraftment; [ Time Frame: up to 42 days post transplant ]
  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 60 days post transplant ]

Secondary Outcome Measures :
  1. Proportion of subjects achieving transfusion independence; [ Time Frame: up to 24 months post transplant ]
  2. Proportion of subjects with a > = 50% reduced annualized volume of packed RBC transfusions. [ Time Frame: up to 24 months post transplant ]

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   5 Years to 15 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • 5-15 years old. Clinically diagnosed as β-thalassemia major with IVS-654 gene mutation phenotype;
  • Subjects or at least one legal guardian/agent understand and voluntarily sign informed consent.
  • 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. 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.

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

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China, Shanghai
Shanghai Bioraylaboratory Inc
Shanghai, Shanghai, China, 200241
Sponsors and Collaborators
Bioray Laboratories
PLA 923 Hospital
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Principal Investigator: Xinhua Zhang, Prof PLA 923 Hospital
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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Responsible Party: Bioray Laboratories
ClinicalTrials.gov Identifier: NCT04205435    
Other Study ID Numbers: 2019-BRL-00CH2
First Posted: December 19, 2019    Key Record Dates
Last Update Posted: October 13, 2022
Last Verified: September 2021
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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Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Hematologic Diseases
Genetic Diseases, Inborn