Safety and Efficacy Evaluation of β-globin Restored Autologous Hematopoietic Stem Cells in β-thalassemia Major Patients
NCT ID: NCT05745532
Last Updated: 2024-11-29
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.
RECRUITING
EARLY_PHASE1
10 participants
INTERVENTIONAL
2020-12-01
2025-12-30
Brief Summary
Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.
Detailed Description
Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.
Conditions
See the medical conditions and disease areas that this research is targeting or investigating.
Study Design
Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.
NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
Review each arm or cohort in the study, along with the interventions and objectives associated with them.
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.
β-globin restored autologous hematopoietic stem cells
β-globin-restored autologous hematopoietic stem cells modified with LentiHBBT87Q
Interventions
Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.
β-globin restored autologous hematopoietic stem cells
β-globin-restored autologous hematopoietic stem cells modified with LentiHBBT87Q
Eligibility Criteria
Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.
Inclusion Criteria
* 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
* 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.
8 Years
16 Years
ALL
No
Sponsors
Meet the organizations funding or collaborating on the study and learn about their roles.
Shenzhen Hemogen
INDUSTRY
Responsible Party
Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.
Principal Investigators
Learn about the lead researchers overseeing the trial and their institutional affiliations.
Chao Liu, PHD
Role: PRINCIPAL_INVESTIGATOR
Shenzhen Hemogen
Sixi Liu, Professor
Role: PRINCIPAL_INVESTIGATOR
Shenzhen Children's Hospital
Locations
Explore where the study is taking place and check the recruitment status at each participating site.
Shenzhen Children's Hospital
Shenzhen, Guangdong, China
Countries
Review the countries where the study has at least one active or historical site.
Central Contacts
Reach out to these primary contacts for questions about participation or study logistics.
Facility Contacts
Find local site contact details for specific facilities participating in the trial.
Other Identifiers
Review additional registry numbers or institutional identifiers associated with this trial.
SZ-101
Identifier Type: -
Identifier Source: org_study_id
NCT04592458
Identifier Type: -
Identifier Source: nct_alias