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β-globin Restored Autologous HSC in β-thalassemia Major Patients | Clinical Trials | Clareo Health

TERMINATEDPHASE1/PHASE2

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

a Safety and Efficacy Study of β-globin Restored Autologous Hematopoietic Stem Cells for β-thalassemia Major Patients With CVS-654 Mutation

NCT04205435•Bioray Laboratories

View on ClinicalTrials.gov

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.

Eligibility

Age

5 - 15 years

Sex

ALL

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.

Locations (1)

Shanghai Bioraylaboratory Inc

Shanghai, Shanghai Municipality, China

Key Dates

Start Date

November 1, 2021

Primary Completion Date

June 1, 2022

Completion Date

July 25, 2022

Last Updated

October 13, 2022

Enrollment

ACTUAL participants

Conditions

β-thalassemia Major

Interventions

β-globin restored autologous HSC

BIOLOGICAL

Comparison of Deferasirox and Desferoxamine in Patients of β-Thalassemia Major With Iron Overload

NCT06468423

Pateints of β-thalassemia Major With Iron Overload

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COMPLETEDPHASE2

A 4-year Extension Study to Core 1-year Study of Iron Chelation Therapy With Deferasirox in β-thalassemia Major Pediatric Patients With Transfusional Iron Overload.

NCT00390858

Transfusional Iron Overloadβ-thalassemia Major+1 more

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: October 13, 2022Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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