History of Changes for Study: NCT04211480

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

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Study Record Versions

Version	Submitted Date	Changes
1	December 24, 2019	None (earliest Version on record)
2	May 27, 2020	Recruitment Status, Study Status, Arms and Interventions, Outcome Measures, Contacts/Locations, Study Description, Study Identification, Eligibility, Oversight and Sponsor/Collaborators
3	December 25, 2020	IPDSharing, Outcome Measures, Study Status, Contacts/Locations and Sponsor/Collaborators
4	January 6, 2021	Study Status and Study Identification
5	January 6, 2021	Contacts/Locations, Eligibility and Study Status
6	August 26, 2021	Study Status, Study Description, Sponsor/Collaborators and Study Identification
7	January 5, 2022	IPDSharing, Study Status and Conditions
8	March 26, 2022	Outcome Measures and Study Status
9	May 9, 2022	Study Status and IPDSharing
10	June 5, 2022	Study Status, IPDSharing and Eligibility
11	October 9, 2022	Recruitment Status, Study Status, Contacts/Locations and Study Design
12	January 4, 2024	Recruitment Status and Study Status

Comparison Format:

Merged
Side-by-Side

Study Identification


Unique Protocol ID:	2019-BRL-00CH2
Brief Title:	β-globin Restored Autologous HSC in β-thalassemia Major Patients
Official Title:	a Safety and Efficacy Study of β-globin Restored Autologous Hematopoietic Stem Cells for β-thalassemia Major Patients With CVS-654 Mutation
Secondary IDs:

Study Status


Record Verification:	December 2019
Overall Status:	Not yet recruiting
Study Start:	March 1, 2020
Primary Completion:	February 28, 2021 [Anticipated]
Study Completion:	March 1, 2022 [Anticipated]

First Submitted:	December 23, 2019
First Submitted that Met QC Criteria:	December 24, 2019
First Posted:	December 26, 2019 [Actual]

Last Update Submitted that Met QC Criteria:	December 24, 2019
Last Update Posted:	December 26, 2019 [Actual]

Sponsor/Collaborators


Sponsor:	Bioray Laboratories
Responsible Party:	Sponsor
Collaborators:	Xiangya Hospital of Central South University

Oversight


U.S. FDA-regulated Drug:	No
U.S. FDA-regulated Device:	No
Data Monitoring:	No

Study Description


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.
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.

Conditions


Conditions:	Thalassemia Major
Keywords:

Study Design


Study Type:	Interventional
Primary Purpose:	Treatment
Study Phase:	Not Applicable
Interventional Study Model:	Single Group Assignment
Number of Arms:	1
Masking:	None (Open Label)
Allocation:	N/A
Enrollment:	12 [Anticipated]

Arms and Interventions

Arms	Assigned Interventions
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

Outcome Measures


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 β-globin restored autologous hematopoietic stem cells [ Time Frame: 12 months post transplant ] Independence on blood transfusion post transplant; Or, The amount of red cell transfusion in a year post transplant reduced 50% than that of the last year.

Eligibility


Minimum Age:	5 Years
Maximum Age:	15 Years
Sex:	All
Gender Based:
Accepts Healthy Volunteers:	No
Criteria:	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 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/Locations


Central Contact Person:	Wei Li, PhD Telephone: +8618621670308 Email: wli@bioraylab.com
Study Officials:	Bin Fu, Prof. Principal Investigator Xiangya Hospital Central University
Locations:

IPDSharing


Plan to Share IPD:	No

References


Links:
Available IPD/Information: