Thalassemia, BetaNCT06041620
Safety and Efficacy Evaluation of Autologous CRISPR-Cas12b Edited Hematopoietic Stem Cells
A study testing VGB-Ex01 in people with Thalassemia, Beta and Thalassemia Major, run by Institute of Hematology & Blood Diseases Hospital, China at 1 site in China.
Trial summary
This is a single-arm, open, single-injection exploratory clinical study with two transfusion-dependent β thalassemia (β-TDT) participants planned to enroll. Read more about this study
- Who is it for?
- Ages 3 to 35 years
- Conditions the study is for
- Thalassemia, Beta, Thalassemia Major
- What is being tested?
- VGB-Ex01
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 1 participants
- Where
- 1 siteChina
- Study dates
- 31 Aug 2023to 31 Dec 2023
- Phase
- Interventional
- Are there results?
- None posted in the registry
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 3 to 35 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Age 3-35 years old (inclusive), male or female;
- The subject and/or his/her legally recognized representative/parent/guardian fully understands the study and all information related to the study and has signed the informed consent form;
- Clinical diagnosis of transfusion-dependent β-thalassemia (TDT) with a blood transfusion record within 2 years (inclusive) prior to screening showing a history of ≥ 10 units (U)/kg/year (or ≥ 100 mL/kg/year) or ≥ 8 times/year of suspended RBC transfusions in at least 1 consecutive 12-month period;
- Karnofsky score (for subjects aged ≥ 16 years) or Lansky score (for subjects aged < 16 years) of ≥ 80;
- Subjects in stable disease state who are eligible for hematopoietic stem cell transplantation as per investigator's judgment;
- Access to diagnosis and treatment records issued by medical professional institutions within 2 years prior to screening, including the records of blood transfusions, hematology, serum chemistry, and other examinations;
- Willing and able to comply with study procedures, with good compliance, and willing to receive and complete the follow-up study with a duration of at least 2 years;
- Subjects of childbearing potential (including female subjects of childbearing potential and male subjects whose partners are of childbearing potential) must use effective contraception within 12 months of treatment.
Who cannot join
- Diagnosis of associated α-thalassemia: > 1 alpha chain deletion or alpha gene functional defect;
- Have available HLA-fully matched donors and acceptable for allogeneic hematopoietic stem cell transplantation;
- Irregular antibody or platelet antibody positive;
- Prior allogeneic bone marrow transplantation or gene therapy;
- Subjects with clinically significant and active bacterial, viral, fungal, or parasitic infection as determined by the investigator at screening, including but not limited to those with positive etiology of human immunodeficiency virus (HIV-1/2), human cytomegalovirus (HCMV-DNA), Epstein-Barr virus (EBV-DNA), or Treponema pallidum antibody (TP-Ab), or with previous hepatitis B or C infection;
- Subjects with an injury of major organs
Show all 15Show fewer
- Contraindications for hematopoietic stem cell collection and poor collection efficiency judged by the investigator;
- Contraindications to the clinical investigational product and its excipients, G-CSF (hematopoietic stem cell mobilization), plerixafor (hematopoietic stem cell mobilization), busulfan (myeloablation), and other drugs;
- Participation within 3 months prior to screening or current participation in another interventional clinical study;
- History or family history of malignancy or myeloproliferative disorder;
- History of uncontrollable epilepsy, mental disorder, or other psychiatric disorders;
- Abuse of psychoactive substance, drug, or alcohol within 6 months prior to enrollment;
- Pregnant or breastfeeding females;
- Other diseases or reasons that interfere with study procedures;
- Any other conditions that the investigator deems unsuitable for the subject's participation in the study.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Study to Evaluate the Efficacy and Safety of Autologous CRISPR-Cas12b Edited Hematopoietic Stem Cells in Transfusion-dependent β Thalassemia Patients
This is a single-arm, open, single-injection exploratory clinical study with two transfusion-dependent β thalassemia (β-TDT) participants planned to enroll.
Read the detailed description
Through CRISPR-Cas 12b editing tool with independent intellectual property rights of Chinese Academy of Sciences, HBG1/2 promoter was edited to reactivate gamma-globin and induce fetal hemoglobin (HbF) expression. This leads to a subsequent reduction in ineffective red blood cell production (due to a reduction in the uncompounded alpha-globin chain) and improved red blood cell survival (due to reduced hemolysis), ultimately improving the sequelae of anemia and reducing the need for transfusion. Safety and efficacy will be evaluated continuously throughout the study, follow-up was up to 24 months. After the end of this trial, participants who received the infusion of autologous CRISPR-Cas12b edited hematopoietic stem cells (VGB-Ex01) will be invited to participate in the long-term follow-up study to complete the 15-year follow-up plan.
- Conditions
- Thalassemia, Beta, Thalassemia Major
- Being tested
- VGB-Ex01
- Medical subject headings
- beta-Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VGB-Ex01Biological
CRISPR-Cas12b editing hematopoietic stem cells
| Study group | Role | What they receive |
|---|---|---|
| VGB-Ex01 | Experimental | Each subject will accept one dose of VGB-Ex01. Receives: Biological: VGB-Ex01 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of adverse events and serious adverse events
An adverse event is any untoward medical occurrence in a clinical investigation/participant administered a product; the event will not need to have a causal relationship with the treatment. A serious adverse event is any untoward medical occurrence at any dose that resulted in death; life threatening;require inpatient hospitalization or prolongation of existing hospitalization; result in persistent or significant disability/incapacity; result in congenital anomaly/birth defect.
Measured: Baseline up to 24 months
Number of subjects with neutrophil implantation ≤ 42 days
Neutrophil implantation was defined as 3 consecutive days with 3 tests for ANC≥500/μL.
Measured: Baseline up to 42 days
Secondary outcomes (5)
Number of subjects transfusion independence (TI) for at least 6 months after transfusion
TI defined as Hb≥9g/dL without red blood cell infusion
Measured: Baseline up to 6 months
Number of subjects transfusion independence (TI) for at least 12 months after transfusion
TI defined as Hb≥9g/dL without red blood cell infusion
Measured: Baseline up to 12 months
Fetal hemoglobin (HbF) concentration
Changes in HbF concentration from baseline after transfusion
Measured: Baseline up to 24 months
Total hemoglobin (Hb) concentration
Changes in Hb concentration compared with baseline after transfusion
Measured: Baseline up to 24 months
The proportion of circulating red blood cells
Changes in the proportion of circulating red blood cells expressing fetal hemoglobin compared to baseline
Measured: Baseline up to 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Sep 2023
- Study start31 Aug 2023
- Primary completion31 Dec 2023
- Study completion31 Dec 2023
Why it stopped: Suboptimal treatment response
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Thalassemia, BetaStudies of the same condition, open and under way.
- More trials run by Institute of Hematology & Blood Diseases Hospital, ChinaThe 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
- 1 participants (actual)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Collaborators
- Shanghai Vitalgen BioPharma Co., Ltd.
- Responsible party
- Principal investigator, Jun Shi, Institute of Hematology & Blood Diseases Hospital, China
- Investigators
- Jun Shi, PhD, Principal investigator, Institute of Hematology & Blood Diseases Hospital, China
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06041620
- Sponsor's protocol ID
- IIT2023048
- Status last verified
- Nov 2026
- First submitted
- 11 Sep 2023
- Last updated
- 4 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 4 Nov 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Study to Evaluate the Efficacy and Safety of Autologous CRISPR-Cas12b Edited Hematopoietic Stem Cells in Transfusion-dependent β Thalassemia Patients
- ClinicalTrials.gov ID
- NCT06041620
- Acronym / sponsor ID
- IIT2023048
- Conditions
- Thalassemia, Beta, Thalassemia Major
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Suboptimal treatment response
- Enrolment
- 1 participants (actual)
- Lead sponsor
- Institute of Hematology & Blood Diseases Hospital, ChinaUniversity, hospital or other
- Collaborators
- Shanghai Vitalgen BioPharma Co., Ltd.
- Study start
- 31 Aug 2023 (actual)
- Primary completion
- 31 Dec 2023 (actual)
- Study completion
- 31 Dec 2023 (actual)
- First posted
- 18 Sep 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 a single-arm, open, single-injection exploratory clinical study with two transfusion-dependent β thalassemia (β-TDT) participants planned to enroll.
Detailed description
Through CRISPR-Cas 12b editing tool with independent intellectual property rights of Chinese Academy of Sciences, HBG1/2 promoter was edited to reactivate gamma-globin and induce fetal hemoglobin (HbF) expression. This leads to a subsequent reduction in ineffective red blood cell production (due to a reduction in the uncompounded alpha-globin chain) and improved red blood cell survival (due to reduced hemolysis), ultimately improving the sequelae of anemia and reducing the need for transfusion. Safety and efficacy will be evaluated continuously throughout the study, follow-up was up to 24 months. After the end of this trial, participants who received the infusion of autologous CRISPR-Cas12b edited hematopoietic stem cells (VGB-Ex01) will be invited to participate in the long-term follow-up study to complete the 15-year follow-up plan.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| VGB-Ex01 | Experimental | Biological: VGB-Ex01Each subject will accept one dose of VGB-Ex01. |
Who may qualify
- Ages
- 3 to 35 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (8 listed)
- Age 3-35 years old (inclusive), male or female;
- The subject and/or his/her legally recognized representative/parent/guardian fully understands the study and all information related to the study and has signed the informed consent form;
- Clinical diagnosis of transfusion-dependent β-thalassemia (TDT) with a blood transfusion record within 2 years (inclusive) prior to screening showing a history of ≥ 10 units (U)/kg/year (or ≥ 100 mL/kg/year) or ≥ 8 times/year of suspended RBC transfusions in at least 1 consecutive 12-month period;
- Karnofsky score (for subjects aged ≥ 16 years) or Lansky score (for subjects aged < 16 years) of ≥ 80;
- Subjects in stable disease state who are eligible for hematopoietic stem cell transplantation as per investigator's judgment;
Key exclusion criteria (15 listed)
- Diagnosis of associated α-thalassemia: > 1 alpha chain deletion or alpha gene functional defect;
- Have available HLA-fully matched donors and acceptable for allogeneic hematopoietic stem cell transplantation;
- Irregular antibody or platelet antibody positive;
- Prior allogeneic bone marrow transplantation or gene therapy;
- Subjects with clinically significant and active bacterial, viral, fungal, or parasitic infection as determined by the investigator at screening, including but not limited to those with positive etiology of human immunodeficiency virus (HIV-1/2), human cytomegalovirus (HCMV-DNA), Epstein-Barr virus (EBV-DNA), or Treponema pallidum antibody (TP-Ab), or with previous hepatitis B or C infection;
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 |
|---|---|---|
| VGB-Ex01CRISPR-Cas12b editing hematopoietic stem cells | Biological | VGB-Ex01 |
Primary outcome
Incidence of adverse events and serious adverse events
Time frame: Baseline up to 24 months
An adverse event is any untoward medical occurrence in a clinical investigation/participant administered a product; the event will not need to have a causal relationship with the treatment. A serious adverse event is any untoward medical occurrence at any dose that resulted in death; life threatening;require inpatient hospitalization or prolongation of existing hospitalization; result in persistent or significant disability/incapacity; result in congenital anomaly/birth defect.
Number of subjects with neutrophil implantation ≤ 42 days
Time frame: Baseline up to 42 days
Neutrophil implantation was defined as 3 consecutive days with 3 tests for ANC≥500/μL.
5 secondary outcomes
Number of subjects transfusion independence (TI) for at least 6 months after transfusion
Time frame: Baseline up to 6 months
Number of subjects transfusion independence (TI) for at least 12 months after transfusion
Time frame: Baseline up to 12 months
Fetal hemoglobin (HbF) concentration
Time frame: Baseline up to 24 months
Total hemoglobin (Hb) concentration
Time frame: Baseline up to 24 months
The proportion of circulating red blood cells
Time frame: Baseline up to 24 months
Results and safety
The study was stopped early (reason given: Suboptimal treatment response). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Nov 2026.
- Individual participant dataSharing planned: No
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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.
Jun Shi, PhD principal investigator
Institute of Hematology & Blood Diseases Hospital, China
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing VGB-Ex01 in people with Thalassemia, Beta and Thalassemia Major, run by Institute of Hematology & Blood Diseases Hospital, China 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 8 inclusion and 15 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: stopped early, as of the sponsor's last update on 4 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?
The study was stopped early (reason given: Suboptimal treatment response). Results are not available in the source record.
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?
Incidence of adverse events and serious adverse events (time frame: Baseline up to 24 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 4 Nov 2026; AzTrials last refreshed its copy on 8 Nov 2026.