Beta-ThalassemiaNCT03351829
Gene Therapy of Beta Thalassemia Using a Self-inactivating Lentiviral Vector
A study testing Gene-modified autologous hematopoeitic stem cells in people with Beta-Thalassemia, run by Shenzhen Geno-Immune Medical Institute at 1 site in China.
Trial summary
This is a Phase I/II clinical trial of gene transfer for treating Beta-thalassemia using a self-inactivating lentiviral vector to functionally correct the defective gene(s). The objectives are to evaluate the safety and efficacy of the gene transfer clinical protocol. Read more about this study
- Who is it for?
- Ages 4 to 70 years
- Conditions the study is for
- Beta-Thalassemia
- What is being tested?
- Gene-modified autologous hematopoeitic stem cells
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteChina
- Study dates
- 31 Dec 2026to 31 Dec 2030
- Phase
- Interventional
- 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 4 to 70 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Diagnosis of Beta Thalathemia.
- Age: ≥ 4 years.
- Karnofsky: ≥ 80%.
- Left ventricular ejection fraction (LVEF): > 50%; no obvious heart disease and pulmonary hypertension.
- Pulmonary function is normal; forced expiratory volumein one second (FEV1) and vital capacity greater than 60% and DLCO > 50%.
- Serum creatinine ≤ 2 × upper limit of normal range.
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- MRI showed no super-iron load in the heart and liver, and no severe cirrhosis.
- Normal Coagulation.
- Written, informed consent obtained prior to any study-specific procedures.
Who cannot join
- Diagnosis of active malignant disease (other than Bowen disease or cervical cancer); or has family history of cancer.
- Myelopathy, tumor-related cytogenetic changes or other more severe blood diseases.
- Has alcoholism experience within 6 months prior to enrollment.
- History of epilepsy.
- History of bone marrow transplantation.
- Existence of an available HLA-identical related donor.
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- Pregnant or lactating females.
- Subject infected with HIV (HIV antibody positive), Treponema pallidum antibody positive or TB culture positive.
- Patients, in the opinion of investigators, may not be eligible or not able to comply with the study.
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
Shenzhen Geno-immune Medical Institute
Shenzhen, Guangdong, 518000
Lung-Ji Chang, PhD, +86 0755-86573763, c@mkgimi.com
About this study
This is a Phase I/II clinical trial of gene transfer for treating Beta-thalassemia using a self-inactivating lentiviral vector to functionally correct the defective gene(s). The objectives are to evaluate the safety and efficacy of the gene transfer clinical protocol.
Read the detailed description
Important Regulatory Notice:
This trial record is only for global academic information registration on ClinicalTrials.gov. Neither the sponsor Beijing Meikang Jimian Biotechnology Co., Ltd. nor collaborator Shenzhen Geno-Immune Medical Institute has obtained NMPA clinical trial approval or clinical technology filing permission to carry out interventional cell therapy trials in mainland China.
ClinicalTrials.gov registration alone does not represent legal approval by Chinese health and drug regulatory authorities.
Thalassemia is considered the most common genetic disorder worldwide. Beta-thalassemia is caused by mutations in the beta-globin gene which encodes the beta-globin protein, leading to the ineffective erythropoiesis, hemolysis and anemia. Currently, the only cure for thalassemia is bone marrow transplantation from a related, compatible donor, which has, however, the significant risk of transplant related mortality, graft versus host disease and limited source. Therefore, gene therapy, achieved by transplantation of the patient's own stem cells that have been genetically-modified with the corrected gene, could potentially cure thalassemia.
This study will use a gene transfer procedure performed to insert the beta-globin gene into the participant's autologous stem cells (hematopoeitic stem cells) using a self-inactivating lentiviral vector. The purpose of this study is to evaluate the safety and effectiveness of the gene transfer procedure and to determine the ability of the gene-corrected cells at generating new, healthy blood cells in patients.
- Conditions
- Beta-Thalassemia
- Being tested
- Gene-modified autologous hematopoeitic stem cells
- Keywords
- Beta Thalassemia, Lentiviral vector, Gene
- Medical subject headings
- beta-Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Gene-modified autologous hematopoeitic stem cellsGenetic
1 infusion of 5x10^6~1x10^7 per kilogram body weight gene-modified cells; or more infusions depending on the circumstances
| Study group | Role | What they receive |
|---|---|---|
| Gene-modified autologous hematopoeitic stem cells | Experimental | Gene-modified autologous hematopoeitic stem cells transduced with lentiviral vector carrying the beta-globin gene ex vivo Receives: Genetic: Gene-modified autologous hematopoeitic stem cells |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety in patients using CTCAE version 4.0 standard to evaluate the level of adverse events
Physiological parameter (measuring cytokine response, fever, symptoms)
Measured: 6 months
Tolerability of transplanted cells that are transduced ex vivo & transplanted in subjects with ß-thalassemia major conditioned with a reduced-intensity non-myeloablative preparative regimen.
Monitoring the following: The occurrence of insertional oncogenesis, which will be investigated by monitoring peripheral blood cell counts \& leukocyte clonality using PCR and sequencing analysis, and qPCR for vector copy number.
Measured: 1 year
Secondary outcomes (1)
Treatment responses
Blood routine indexes will be recorded before and after treatment. Objective response, such as complete response (CR), partial response (PR), stable disease (SD), or progressive disease (PD) will be assessed.
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Nov 2017
- Study start31 Dec 2026 (estimated)
- Primary completion31 Dec 2029 (estimated)
- Study completion31 Dec 2030 (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.
- More trials run by Shenzhen Geno-Immune Medical InstituteThe sponsor's other registered studies.
- 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 N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Shenzhen Geno-Immune Medical Institute
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Lung-Ji Chang, Shenzhen Geno-Immune Medical Institute
- Investigators
- Lung-Ji Chang, PhD, Principal investigator, Shenzhen Geno-Immune Medical Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03351829
- Sponsor's protocol ID
- GIMI-IRB-17008
- Status last verified
- Oct 2026
- First submitted
- 20 Nov 2017
- Last updated
- 2 Nov 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 2 Nov 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Gene Therapy of Beta Thalassemia Using a Self-inactivating Lentiviral Vector
- ClinicalTrials.gov ID
- NCT03351829
- Acronym / sponsor ID
- GIMI-IRB-17008
- Conditions
- Beta-Thalassemia
- Keywords
- Beta Thalassemia, Lentiviral vector, Gene
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Shenzhen Geno-Immune Medical InstituteUniversity, hospital or other
- Study start
- 31 Dec 2026 (estimated)
- Primary completion
- 31 Dec 2029 (estimated)
- Study completion
- 31 Dec 2030 (estimated)
- First posted
- 24 Nov 2017 (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 a Phase I/II clinical trial of gene transfer for treating Beta-thalassemia using a self-inactivating lentiviral vector to functionally correct the defective gene(s). The objectives are to evaluate the safety and efficacy of the gene transfer clinical protocol.
Detailed description
Important Regulatory Notice:
This trial record is only for global academic information registration on ClinicalTrials.gov. Neither the sponsor Beijing Meikang Jimian Biotechnology Co., Ltd. nor collaborator Shenzhen Geno-Immune Medical Institute has obtained NMPA clinical trial approval or clinical technology filing permission to carry out interventional cell therapy trials in mainland China.
ClinicalTrials.gov registration alone does not represent legal approval by Chinese health and drug regulatory authorities.
Thalassemia is considered the most common genetic disorder worldwide. Beta-thalassemia is caused by mutations in the beta-globin gene which encodes the beta-globin protein, leading to the ineffective erythropoiesis, hemolysis and anemia. Currently, the only cure for thalassemia is bone marrow transplantation from a related, compatible donor, which has, however, the significant risk of transplant related mortality, graft versus host disease and limited source. Therefore, gene therapy, achieved by transplantation of the patient's own stem cells that have been genetically-modified with the corrected gene, could potentially cure thalassemia.
This study will use a gene transfer procedure performed to insert the beta-globin gene into the participant's autologous stem cells (hematopoeitic stem cells) using a self-inactivating lentiviral vector. The purpose of this study is to evaluate the safety and effectiveness of the gene transfer procedure and to determine the ability of the gene-corrected cells at generating new, healthy blood cells in patients.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Gene-modified autologous hematopoeitic stem cells | Experimental | Genetic: Gene-modified autologous hematopoeitic stem cellsGene-modified autologous hematopoeitic stem cells transduced with lentiviral vector carrying the beta-globin gene ex vivo |
Who may qualify
- Ages
- 4 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (9 listed)
- Diagnosis of Beta Thalathemia.
- Age: ≥ 4 years.
- Karnofsky: ≥ 80%.
- Left ventricular ejection fraction (LVEF): > 50%; no obvious heart disease and pulmonary hypertension.
- Pulmonary function is normal; forced expiratory volumein one second (FEV1) and vital capacity greater than 60% and DLCO > 50%.
Key exclusion criteria (9 listed)
- Diagnosis of active malignant disease (other than Bowen disease or cervical cancer); or has family history of cancer.
- Myelopathy, tumor-related cytogenetic changes or other more severe blood diseases.
- Has alcoholism experience within 6 months prior to enrollment.
- History of epilepsy.
- History of bone marrow transplantation.
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 |
|---|---|---|
| Gene-modified autologous hematopoeitic stem cells1 infusion of 5x10^6~1x10^7 per kilogram body weight gene-modified cells; or more infusions depending on the circumstances | Genetic | Gene-modified autologous hematopoeitic stem cells |
Primary outcome
Safety in patients using CTCAE version 4.0 standard to evaluate the level of adverse events
Time frame: 6 months
Physiological parameter (measuring cytokine response, fever, symptoms)
Tolerability of transplanted cells that are transduced ex vivo & transplanted in subjects with ß-thalassemia major conditioned with a reduced-intensity non-myeloablative preparative regimen.
Time frame: 1 year
Monitoring the following: The occurrence of insertional oncogenesis, which will be investigated by monitoring peripheral blood cell counts \& leukocyte clonality using PCR and sequencing analysis, and qPCR for vector copy number.
1 secondary outcome
Treatment responses
Time frame: 1 year
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 2 Nov 2026.
- Individual participant dataSharing planned: No
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Evaluation the Safety and Efficacy of KL003 Cell Injection in the Treatment of Transfusion-dependent β-thalassemia.
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Growth and Development-related Outcomes in Children With Transfusion-dependent Beta-thalassemia After Gene Therapy
Transfusion-dependent Beta-Thalassemia, Gene Therapy
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β-Thalassemia Treatment With KL003 Cell Injection
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A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-NTDT)
Non-Transfusion-dependent Alpha-Thalassemia, Non-Transfusion-dependent Beta-Thalassemia
Active, not recruitingObservational study
Investigating the Mechanistic Effects of Mitapivat in Subjects With Sickle Cell Disease
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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.
Lung-Ji Chang, PhD principal investigator
Shenzhen Geno-Immune Medical Institute
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Gene-modified autologous hematopoeitic stem cells in people with Beta-Thalassemia, run by Shenzhen Geno-Immune Medical Institute 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 9 inclusion and 9 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: opening soon, as of the sponsor's last update on 2 Nov 2026. 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.
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 Lung-Ji Chang, PhD (+86 0755-86573763, c@mkgimi.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety in patients using CTCAE version 4.0 standard to evaluate the level of adverse events (time frame: 6 months). The study lists 2 primary outcomes in all. 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 2 Nov 2026.