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

NCT04205435 | Terminated Phase 1

• 1 other identifier: 2019-BRL-00CH2
• Study Type: interventional
• Target: 2
• Locations: 1 country
• Sites: 1

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.

Conditions

β-thalassemia Major

Outcome Measures

Primary Outcomes (2)

• Proportion of subjects with engraftment;

  up to 42 days post transplant

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

  up to 60 days post transplant

Secondary Outcomes (2)

• Proportion of subjects achieving transfusion independence;

  up to 24 months post transplant

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

  up to 24 months post transplant

Study Arms (1)

β-globin restored autologous HSC

EXPERIMENTAL

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

Biological: β-globin restored autologous HSC

Interventions

β-globin restored autologous HSC BIOLOGICAL

gene edited autologous hematopoietic stem cells with β-globin restoration

β-globin restored autologous HSC

Eligibility Criteria

• Age: 5 Years - 15 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

• \- 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.

Sponsors & Collaborators

• Bioray Laboratories: lead
• The 923rd Hospital of Joint Logistics Support Force of People's Liberation Army: collaborator

Study Sites (1)

Shanghai Bioraylaboratory Inc

Shanghai, Shanghai Municipality, 200241, China

Location

Related Publications (1)

• 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.

  PMID: 34175041 DERIVED

Study Officials

• Xinhua Zhang, Prof

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

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 18, 2019
• First Posted: December 19, 2019
• Study Start: November 1, 2021
• Primary Completion: June 1, 2022
• Study Completion: July 25, 2022
• Last Updated: October 13, 2022

Record last verified: 2021-09

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)