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

ß-thalassemia Major Clinical Trial

Official title:

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

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT04205435
• Other study ID #: 2019-BRL-00CH2
• Status: Terminated
• Phase: Phase 1/Phase 2
• Start date: November 1, 2021
• Est. completion date: July 25, 2022

Study information

• Verified date: September 2021
• Source: Bioray Laboratories
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 2
• Est. completion date: July 25, 2022
• Est. primary completion date: June 1, 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: 5 Years to 15 Years

Eligibility

Inclusion Criteria:

• 5-15 years old. Clinically diagnosed as ß-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.

Related Conditions & MeSH terms

• beta-Thalassemia
• ß-thalassemia Major
• Thalassemia

Intervention

Biological:
• ß-globin restored autologous HSC
gene edited autologous hematopoietic stem cells with ß-globin restoration

Locations

Country	Name	City	State
China	Shanghai Bioraylaboratory Inc	Shanghai	Shanghai

Sponsors (2)

Lead Sponsor	Collaborator
Bioray Laboratories	PLA 923 Hospital

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Proportion of subjects with engraftment;		up to 42 days post transplant
Primary	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	Proportion of subjects achieving transfusion independence;		up to 24 months post transplant
Secondary	Proportion of subjects with a > = 50% reduced annualized volume of packed RBC transfusions.		up to 24 months post transplant