β-thalassemiaEarly Phase 1NCT05745532
Safety and Efficacy Evaluation of β-globin Restored Autologous Hematopoietic Stem Cells in β-thalassemia Major Patients
A Early Phase 1 study testing β-globin restored autologous hematopoietic stem cells in people with β-thalassemia, run by Shenzhen Hemogen at 1 site in China.
Trial 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 Read more about this study
- Who is it for?
- Ages 8 to 16 years
- Conditions the study is for
- β-thalassemia
- What is being tested?
- β-globin restored autologous hematopoietic stem cells
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteChina
- Study dates
- 1 Dec 2020to 30 Dec 2025
- Phase
- Early Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 8 to 16 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
Shenzhen Children's Hospital
Shenzhen, Guangdong, 518083
Haigang Sun, 13823168465, sunhaigang@genomics.cn
About this study
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
Read the 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.
- Conditions
- β-thalassemia
- Being tested
- β-globin restored autologous hematopoietic stem cells
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
β-globin restored autologous hematopoietic stem cellsBiological
β-globin-restored autologous hematopoietic stem cells modified with LentiHBBT87Q
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Biological: β-globin restored autologous hematopoietic stem cells |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 0-100 days
Overall survival
Number of patients alive through the whole trial will be record
Measured: 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
Measured: 0-24 months
Replication competent lentivirus (RCL)
The percentage of RCL should be negative in the 24 months after transplant
Measured: 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.
Measured: 0-24 months
Secondary outcomes (3)
The average Insertion copy number (VCN) in peripheral blood mononuclear cells
The average insertion copy number (VCN) should be ≥0.1 in peripheral blood mononuclear cells
Measured: 18-24 Months
The expression level of exogenous adult hemoglobin
Exogenous adult hemoglobin will be evaluated by globin chains and hemoglobin synthesis on peripheral blood by HPLC and the exogenous adult hemoglobin level is ≥2.0g/dL
Measured: 18-24 Months
Change from baseline in annualized frequency of packed RBC transfusions
Compare the annualized number of pRBC transfusions before gene therapy with the Month 18 and Month 24 period after transplant, the percentage change will be recorded
Measured: 18-24 Months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Feb 2023
- Study start1 Dec 2020
- Primary completion30 May 2025 (estimated)
- Study completion30 Dec 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for β-thalassemiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Shenzhen Hemogen
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Chao Liu, PHD, Principal investigator, Shenzhen HemogenSixi Liu, Professor, Principal investigator, Shenzhen Children's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05745532
- Sponsor's protocol ID
- SZ-101
- Status last verified
- Nov 2024
- First submitted
- 16 Feb 2023
- Last updated
- 29 Nov 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Publications
Liu S, Chen Y, Li Y, Qian L, Dong G, Zhang Q, Zhou X, Guo H, Sun H, Lin Q, Yu J, Zeng X, Zou H, He J, Yao W, Ouyang W, Liu C. Safety and efficacy of hemobeglogene autotemcel(hemo-cel) gene therapy in five patients with transfusion-dependent beta-thalassemia. Mol Ther. 2026 May 6;34(5):2611-2620. doi: 10.1016/j.ymthe.2026.02.017. Epub 2026 Feb 14.
PubMed 41693207
Study at a glance
From the registry record, last updated by the sponsor on 29 Nov 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Safety and Efficacy Evaluation of β-globin Restored Autologous Hematopoietic Stem Cells in β-thalassemia Major Patients
- ClinicalTrials.gov ID
- NCT05745532
- Acronym / sponsor ID
- SZ-101
- Conditions
- β-thalassemia
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- Shenzhen HemogenIndustry (pharma, biotech, devices)
- Study start
- 1 Dec 2020 (actual)
- Primary completion
- 30 May 2025 (estimated)
- Study completion
- 30 Dec 2025 (estimated)
- First posted
- 27 Feb 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Experimental | Experimental | Biological: β-globin restored autologous hematopoietic stem cellsTen transfusion-dependent β-thalassaemia subjects aged 8-16 years will be reinfused with β-globin-restored autologous hematopoietic stem cells modified with LentiHBBT87Q. |
Who may qualify
- Ages
- 8 to 16 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (4 listed)
- 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
Key exclusion criteria (5 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| β-globin restored autologous hematopoietic stem cellsβ-globin-restored autologous hematopoietic stem cells modified with LentiHBBT87Q | Biological | Experimental |
Primary outcome
Frequency and severity of adverse events (AEs) and serious adverse events (SAEs)
Time frame: 0-100 days
The number and the percentage of adverse events related to transplantation in 100 days will be summarized according to NCI CTCAE 5.0
Overall survival
Time frame: 0-24 months
Number of patients alive through the whole trial will be record
Proportion of engraftment
Time frame: 0-24 months
Neutrophil count [ANC] >=500 /mm3 for 3 consecutive days and platelet count [PLT] >20,000/mm3 for7 consecutive days
Replication competent lentivirus (RCL)
Time frame: 0-24 months
The percentage of RCL should be negative in the 24 months after transplant
Dynamics of viral integration sites (VIS)
Time frame: 0-24 months
Evaluation of the percentage of participants without abnormal clonal proliferation and polyclonal engraftment . More than 1000 VIS retrieved from peripheral blood should be checked.
3 secondary outcomes
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
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Nov 2024.
- Derived from this study: PubMed 41693207Liu S, Chen Y, Li Y, Qian L, Dong G, Zhang Q, Zhou X, Guo H, Sun H, Lin Q, Yu J, Zeng X, Zou H, He J, Yao W, Ouyang W, Liu C. Safety and efficacy of hemobeglogene autotemcel(hemo-cel) gene therapy in five patients with transfusion-dependent beta-thalassemia. Mol Ther. 2026 May 6;34(5):2611-2620. doi: 10.1016/j.ymthe.2026.02.017. Epub 2026 Feb 14.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Chao Liu, PHD principal investigator
Shenzhen Hemogen
Sixi Liu, Professor principal investigator
Shenzhen Children's Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing β-globin restored autologous hematopoietic stem cells in people with β-thalassemia, run by Shenzhen Hemogen at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 29 Nov 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Haigang Sun (13823168465, sunhaigang@genomics.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Frequency and severity of adverse events (AEs) and serious adverse events (SAEs) (time frame: 0-100 days). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05745532, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Nov 2024.