Beta-ThalassemiaPhase 1/2NCT06308159
An Open-label Study of a Gene Therapy Product (Vebeglogene Autotemcel) in Transfusion Dependent Beta-Thalassemia
A Phase 1/2 study testing Vebeglogene autotemcel in people with Beta-Thalassemia, run by Lantu Biopharma at 2 sites in China.
Trial summary
This is an interventional study to evaluate the safety and efficacy of autologous Hematopoietic Stem and Progenitor Cells (HSPCs) transduced with lentiviral vector encoding functional hemoglobin subunit beta (HBB) gene in patients with transfusion-dependent beta-thalassemia. Read more about this study
- Who is it for?
- Ages Up to 35 years
- Conditions the study is for
- Beta-Thalassemia
- What is being tested?
- Vebeglogene autotemcel
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 2 sitesChina
- Study dates
- 11 May 2024to 1 Aug 2027
- Phase
- Phase 1/2
- 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 Up to 35 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Patients or parent(s)/legal guardian(s) willing and able to complete the informed consent process and comply with study procedures and visit schedules.
- Diagnosis of beta-thalassemia and a history of RBCs transfusions.
- Documented baseline, or pretransfusion, Hb≤7 g/dL.
- Availability of an adequate and well-documented transfusion history.
Who cannot join
- Active bacterial, viral, fungal, or parasitic infection.
- A white blood cell (WBC) counts<3×10^9/L, and/or platelet counts<100×10^9/L not related to hypersplenism.
- Uncorrected bleeding disorder.
- Presence of severe diseases that judged not compatible with the study procedures, such as severe hepatic disease, kidney disease, lung disease, and/or cardiovascular disease.
- Uncontrolled seizure disorder.
- Any evidence of severe iron overload that, in the investigator's opinion, warrants exclusion.
- Prior autologous hematopoietic stem cell transplantation.
- Prior receipt of gene therapy.
Trial locations and contacts
2 sites in 1 country.
China2 sites
920th Hospital of Joint Logistics Support Force of People's Liberation Army of China
Kunming, Yunnan, 650100
Kunming Hope of Health Hospital
Kunming, Yunnan, 650200
About this study
Official title: An Open-label Clinical Trial of Ex Vivo Beta-globin Lentiviral Vector Transduction of Autologous CD34+HSPCs (Vebeglogene Autotemcel) for the Treatment of Transfusion Dependent Beta-thalassemia Patients
This is an interventional study to evaluate the safety and efficacy of autologous Hematopoietic Stem and Progenitor Cells (HSPCs) transduced with lentiviral vector encoding functional hemoglobin subunit beta (HBB) gene in patients with transfusion-dependent beta-thalassemia.
Read the detailed description
The participant's autologous HSPCs will be transduced with the self-inactivating lentiviral vector, carrying the functional HBB gene.
Study duration per participant is approximately 27 months including an approximately 30-day screening/baseline period, an approximately 60-day mobilization and product manufacture, an approximately 10-day myeloablative conditioning, 1 treatment day, and an approximately 24-month study observation period.
The endpoints will be used to assess the safety and efficacy profiles in patients with transfusion-dependent beta-thalassemia.
- Conditions
- Beta-Thalassemia
- Being tested
- Vebeglogene autotemcel
- Medical subject headings
- beta-Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Vebeglogene autotemcelDrug
Also called Beta-globin lentiviral vector transduced autologous HSPCs
Autologous HSPCs transduced with self-inactivating lentiviral vector encoding functional HBB gene and resuspended in cryopreservative solution in the final immediate container for the intended medical use.
| Study group | Role | What they receive |
|---|---|---|
| Vebeglogene autotemcel | Experimental | One-time infusion of≥5×10^6/kg beta-globin lentiviral vector transduced HSPCs Receives: Drug: Vebeglogene autotemcel |
What is being measured
The primary outcome is the main question the study is designed to answer.
Time and duration of the subject's hemoglobin (Hb)≥9.0 g/dL without receiving red blood cell infusion
Measured: From baseline to Month 24
Secondary outcomes (4)
The prevalence and severity of adverse events (AEs) and serious adverse events (SAEs)
Participants are monitored for safety from baseline up to the end of the follow-up period.
Measured: From baseline to Month 24
The reduction of red blood cells (RBCs) transfusion requirement after product infusion compared to previous transfusion records
The annual number of RBCs transfusions prior to product infusion will be compared to the annual number of RBCs transfusions post-infusion, and the requirement reduction duration should be reported.
Measured: From infusion to Month 24
Number of days required to achieve successful neutrophil and platelet engraftment
Neutrophil engraftment is defined as the time to the first of 3 consecutive days of absolute neutrophil counts (ANC)≥0.5×10^9/L post-infusion without transfusion. Platelet engraftment is defined as the time to the first of 3 consecutive days of platelet values≥20×10^9/L post-infusion without transfusion.
Measured: From infusion to Month 24
Vector copy number (VCN) in peripheral blood over time
Quantification of the lentiviral vector copy number in individual peripheral blood cells will be conducted to measure the transduction of HSPCs.
Measured: From baseline to Month 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Mar 2024
- Study start11 May 2024
- Primary completion1 Aug 2027 (estimated)
- Study completion1 Aug 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Beta-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
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 6 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Lantu Biopharma
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06308159
- Sponsor's protocol ID
- LT02-101
- Status last verified
- Jun 2026
- First submitted
- 6 Mar 2024
- Last updated
- 15 Jun 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 15 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-label Clinical Trial of Ex Vivo Beta-globin Lentiviral Vector Transduction of Autologous CD34+HSPCs (Vebeglogene Autotemcel) for the Treatment of Transfusion Dependent Beta-thalassemia Patients
- ClinicalTrials.gov ID
- NCT06308159
- Acronym / sponsor ID
- LT02-101
- Conditions
- Beta-Thalassemia
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 6 participants (estimated)
- Lead sponsor
- Lantu BiopharmaIndustry (pharma, biotech, devices)
- Study start
- 11 May 2024 (actual)
- Primary completion
- 1 Aug 2027 (estimated)
- Study completion
- 1 Aug 2027 (estimated)
- First posted
- 13 Mar 2024 (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 interventional study to evaluate the safety and efficacy of autologous Hematopoietic Stem and Progenitor Cells (HSPCs) transduced with lentiviral vector encoding functional hemoglobin subunit beta (HBB) gene in patients with transfusion-dependent beta-thalassemia.
Detailed description
The participant's autologous HSPCs will be transduced with the self-inactivating lentiviral vector, carrying the functional HBB gene.
Study duration per participant is approximately 27 months including an approximately 30-day screening/baseline period, an approximately 60-day mobilization and product manufacture, an approximately 10-day myeloablative conditioning, 1 treatment day, and an approximately 24-month study observation period.
The endpoints will be used to assess the safety and efficacy profiles in patients with transfusion-dependent beta-thalassemia.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Vebeglogene autotemcel | Experimental | Drug: Vebeglogene autotemcelOne-time infusion of≥5×10^6/kg beta-globin lentiviral vector transduced HSPCs |
Who may qualify
- Ages
- Up to 35 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (4 listed)
- Patients or parent(s)/legal guardian(s) willing and able to complete the informed consent process and comply with study procedures and visit schedules.
- Diagnosis of beta-thalassemia and a history of RBCs transfusions.
- Documented baseline, or pretransfusion, Hb≤7 g/dL.
- Availability of an adequate and well-documented transfusion history.
Key exclusion criteria (8 listed)
- Active bacterial, viral, fungal, or parasitic infection.
- A white blood cell (WBC) counts<3×10^9/L, and/or platelet counts<100×10^9/L not related to hypersplenism.
- Uncorrected bleeding disorder.
- Presence of severe diseases that judged not compatible with the study procedures, such as severe hepatic disease, kidney disease, lung disease, and/or cardiovascular disease.
- Uncontrolled seizure disorder.
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 |
|---|---|---|
| Vebeglogene autotemcelAlso: Beta-globin lentiviral vector transduced autologous HSPCsAutologous HSPCs transduced with self-inactivating lentiviral vector encoding functional HBB gene and resuspended in cryopreservative solution in the final immediate container for the intended medical use. | Drug | Vebeglogene autotemcel |
Primary outcome
Time and duration of the subject's hemoglobin (Hb)≥9.0 g/dL without receiving red blood cell infusion
Time frame: From baseline to Month 24
4 secondary outcomes
The prevalence and severity of adverse events (AEs) and serious adverse events (SAEs)
Time frame: From baseline to Month 24
The reduction of red blood cells (RBCs) transfusion requirement after product infusion compared to previous transfusion records
Time frame: From infusion to Month 24
Number of days required to achieve successful neutrophil and platelet engraftment
Time frame: From infusion to Month 24
Vector copy number (VCN) in peripheral blood over time
Time frame: From baseline to Month 24
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 15 Jun 2026.
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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. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Vebeglogene autotemcel in people with Beta-Thalassemia, run by Lantu Biopharma at 2 sites 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 8 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: under way, not looking for participants, as of the sponsor's last update on 15 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites 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 combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Time and duration of the subject's hemoglobin (Hb)≥9.0 g/dL without receiving red blood cell infusion (time frame: From baseline to Month 24). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 15 Jun 2026.