Safety and Efficacy Evaluation of β-globin Restored Autologous Hematopoietic Stem Cells in β-thalassemia Major Patients

NCT05745532 | Recruiting Early Phase 1

• 1 other identifier: SZ-101
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

Brief Summary

This is an open label study to evaluate the safety and efficacy of β-globin Restored Autologous Hematopoietic Stem Cells in ß-Thalassemia Major Patients

Conditions

β-thalassemia

Outcome Measures

Primary Outcomes (5)

• Frequency and severity of adverse events (AEs) and serious adverse events (SAEs)

  The number and the percentage of adverse events related to transplantation in 100 days will be summarized according to NCI CTCAE 5.0

  0-100 days

• Overall survival

  Number of patients alive through the whole trial will be record

  0-24 months

• Proportion of engraftment

  Neutrophil count \[ANC\] \>=500 /mm3 for 3 consecutive days and platelet count \[PLT\] \>20,000/mm3 for7 consecutive days

  0-24 months

• Replication competent lentivirus (RCL)

  The percentage of RCL should be negative in the 24 months after transplant

  0-24 months

• Dynamics of viral integration sites (VIS)

  Evaluation of the percentage of participants without abnormal clonal proliferation and polyclonal engraftment . More than 1000 VIS retrieved from peripheral blood should be checked.

  0-24 months

Secondary Outcomes (3)

• The average Insertion copy number (VCN) in peripheral blood mononuclear cells

  18-24 Months

• The expression level of exogenous adult hemoglobin

  18-24 Months

• Change from baseline in annualized frequency of packed RBC transfusions

  18-24 Months

Study Arms (1)

Experimental

EXPERIMENTAL

Ten transfusion-dependent β-thalassaemia subjects aged 8-16 years will be reinfused with β-globin-restored autologous hematopoietic stem cells modified with LentiHBBT87Q.

Biological: β-globin restored autologous hematopoietic stem cells

Interventions

β-globin restored autologous hematopoietic stem cells BIOLOGICAL

β-globin-restored autologous hematopoietic stem cells modified with LentiHBBT87Q

Experimental

Eligibility Criteria

• Age: 8 Years - 16 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• years old. Subject and/or subject's legal guardian fully understand and voluntarily sign informed consent;
• Clinically diagnosed as transfusion-dependent β-thalassemia major;
• With sufficient RBC infusion, subjects must maintain hemoglobin ≥9g/dL, serum ferritin threshold ≤ 3000 ng/mL and the liver iron overload mild or absent for at least 3 months before mobilization of hematopoietic stem cell;
• Follow the arrangements for treatment and regular medical checks within two years post-transplantation

You may not qualify if:

• The physical condition does not meet the requirements for hematopoietic stem cell mobilization and transplantation myeloablation;
• Received gene therapy and allogeneic HSCT in the past.
• Have an available HLA matched donor.
• Enrolling in another clinical trial.
• Other unsuitable conditions identified by doctors.

Sponsors & Collaborators

• Shenzhen Hemogen: lead

Study Sites (1)

Shenzhen Children's Hospital

Shenzhen, Guangdong, 518083, China

RECRUITING

Study Officials

• Chao Liu, PHD

  Shenzhen Hemogen

  PRINCIPAL INVESTIGATOR

• Sixi Liu, Professor

  Shenzhen Children's Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Haigang Sun

CONTACT

13823168465; sunhaigang@genomics.cn

Study Design

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

Study Record Dates

• First Submitted: February 16, 2023
• First Posted: February 27, 2023
• Study Start: December 1, 2020
• Primary Completion: May 30, 2025
• Study Completion: December 30, 2025
• Last Updated: November 29, 2024

Record last verified: 2024-11

Locations

CN (1)