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Recruiting NCT05745532

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

Early Phase I Interventional β-thalassemia

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: β-globin restored autologous hematopoietic stem cells.
Who it may be relevant to
Registry conditions: β-thalassemia. Basic parameters: 8 years — 16 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
China
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Overview

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

Detailed description

We will recruit ß-thalassaemia major patients and collect their autologous hematopoietic stem cells, which will be modified with the LentiHBBT87Q system to restore β-globin expression. After conditioning, the autologous hematopoietic stem cells with restored β-globin will be reinfused to the patients and followed up for two years to collect data.

Interventions

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

Primary outcome measures

  • Frequency and severity of adverse events (AEs) and serious adverse events (SAEs) [Time frame: 0-100 days]
  • Overall survival [Time frame: 0-24 months]
  • Proportion of engraftment [Time frame: 0-24 months]
  • Replication competent lentivirus (RCL) [Time frame: 0-24 months]
  • Dynamics of viral integration sites (VIS) [Time frame: 0-24 months]
Secondary outcome measures (3)
  • The average Insertion copy number (VCN) in peripheral blood mononuclear cells [Time frame: 18-24 Months]
  • The expression level of exogenous adult hemoglobin [Time frame: 18-24 Months]
  • Change from baseline in annualized frequency of packed RBC transfusions [Time frame: 18-24 Months]

Eligibility criteria

Inclusion criteria

  • 8-16 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

Exclusion criteria

  • 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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
N/A
Model
Single group
Masking
Open label
Primary purpose
Treatment

Study locations

China · 1 center
  • Shenzhen Children's Hospital — Shenzhen

Identifiers

NCT: NCT05745532 · SZ-101

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗