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

Primary Purpose

β-thalassemia Major

Status

Terminated

Phase

Phase 1

Locations

China

Study Type

Interventional

Intervention

β-globin restored autologous HSC

About this trial

This is an interventional treatment trial for β-thalassemia Major

Eligibility Criteria

5 Years - 15 Years (Child)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

Shanghai Bioraylaboratory Inc

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

β-globin restored autologous HSC

Arm Description

each subject will accept one dose of β-globin restored autologous hematopoietic stem cells

Outcomes

Primary Outcome Measures

Proportion of subjects with engraftment;

Incidence and severity of adverse events as a measure of safety and tolerability. Adverse events assessed according to NCI-CTCAE v5.0 criteria

Secondary Outcome Measures

Proportion of subjects achieving transfusion independence;

Proportion of subjects with a > = 50% reduced annualized volume of packed RBC transfusions.

Full Information

NCT ID

NCT04205435

First Posted

December 18, 2019

Last Updated

October 9, 2022

Sponsor

Bioray Laboratories

Collaborators

The 923rd Hospital of Joint Logistics Support Force of People's Liberation Army

1. Study Identification

Unique Protocol Identification Number

NCT04205435

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

Study Type

Interventional

2. Study Status

Record Verification Date

September 2021

Overall Recruitment Status

Terminated

Why Stopped

Sponsor decision

Study Start Date

November 1, 2021 (Actual)

Primary Completion Date

June 1, 2022 (Actual)

Study Completion Date

July 25, 2022 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Bioray Laboratories

Collaborators

The 923rd Hospital of Joint Logistics Support Force of People's Liberation Army

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

5. 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

β-thalassemia Major

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1, Phase 2

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

2 (Actual)

8. Arms, Groups, and Interventions

Arm Title

β-globin restored autologous HSC

Arm Type

Experimental

Arm Description

each subject will accept one dose of β-globin restored autologous hematopoietic stem cells

Intervention Type

Biological

Intervention Name(s)

β-globin restored autologous HSC

Intervention Description

gene edited autologous hematopoietic stem cells with β-globin restoration

Primary Outcome Measure Information:

Title

Proportion of subjects with engraftment;

Time Frame

up to 42 days post transplant

Title

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 Measure Information:

Title

Proportion of subjects achieving transfusion independence;

Time Frame

up to 24 months post transplant

Title

Proportion of subjects with a > = 50% reduced annualized volume of packed RBC transfusions.

Time Frame

up to 24 months post transplant

10. Eligibility

Sex

All

Minimum Age & Unit of Time

5 Years

Maximum Age & Unit of Time

15 Years

Accepts Healthy Volunteers

Eligibility 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 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.

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Xinhua Zhang, Prof

Organizational Affiliation

The 923rd Hospital of Joint Logistics Support Force of People's Liberation Army

Official's Role

Principal Investigator

Facility Information:

Facility Name

Shanghai Bioraylaboratory Inc

City

Shanghai

State/Province

Shanghai

ZIP/Postal Code

200241

Country

China

12. IPD Sharing Statement

Plan to Share IPD

Citations:

PubMed Identifier

34175041

Citation

Brusson M, Miccio A. Genome editing approaches to beta-hemoglobinopathies. Prog Mol Biol Transl Sci. 2021;182:153-183. doi: 10.1016/bs.pmbts.2021.01.025. Epub 2021 Mar 1.

Results Reference

derived

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

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