Transfusion Dependent Beta-ThalassaemiaNCT04390971
Safety and Efficacy Evaluation of ET-01 Transplantation in Subjects With Transfusion Dependent β-Thalassaemia
A study testing ET-01 in people with Transfusion Dependent Beta-Thalassaemia, run by Institute of Hematology & Blood Diseases Hospital, China at 3 sites in China.
Trial summary
This is an open label, multi-center study to evaluate the safety and Efficacy of ET-01 Transplantation in subjects with Transfusion Dependent β-Thalassaemia. Read more about this study
- Who is it for?
- Ages 6 to 35 years
- Conditions the study is for
- Transfusion Dependent Beta-Thalassaemia
- What is being tested?
- ET-01
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 3 participants
- Where
- 3 sitesChina
- Study dates
- 10 Feb 2023to 5 Mar 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 6 to 35 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Subject and/or subject's legal personal representative fully understand and voluntarily sign informed consent forms;
- 6~35 years old, all gender;
- Diagnosis of transfusion dependent β-thalassemia (β-TDT) as defined by protocol;
- Eligible for autologous stem cell transplant;
- Eligible for autologous stem cell transplant;
- Organs in good function.
- Other protocol defined inclusion criteria may apply.
Who cannot join
- Subjects with associated α-thalassemia;
- Active bacterial, virus, fungal or parasitic infection, including HIV infection, HbsAg and HBV DNA positive, HCV DNA positive, or Treponema Pallidum infection;
- HLA identical sibling or unrelated donors are available;
- Prior allo-HSCT or gene therapy.
- Other protocol defined exclusion criteria may apply.
Trial locations and contacts
3 sites in 1 country.
China3 sites
The Affiliated Hospital of Guizhou Medical University
Guiyang, Guizhou, 550004
Institute of Hematology & Blood Diseases Hospital
Tianjin, Tianjin Municipality, 300020
Affiliated Hospital of Zunyi Medical University
Zunyi, Guizhou, 563000
About this study
Official title: A Multi-center, Open Label Study to Evaluate the Safety and Efficacy of ET-01 Transplantation in Subjects With Transfusion Dependent β-Thalassemia
This is an open label, multi-center study to evaluate the safety and Efficacy of ET-01 Transplantation in subjects with Transfusion Dependent β-Thalassaemia.
Read the detailed description
After the proper subject is recruited, subject will go through steps generally as stem cell mobilization, apheresis, conditioning and ET-01 infusion to complete therapy. Two years follow-up will be carried out post-transplantation and related data will be collected. Subjects who have been treated by ET-01 will be asked to participate in a long-term follow up study. Monitoring the long-term efficacy and safety up to 15 years after ET-01 transplant.
- Conditions
- Transfusion Dependent Beta-Thalassaemia
- Being tested
- ET-01
- Keywords
- Beta-Thalassaemia, Hematopoietic Stem-Cell Transplantation
- Medical subject headings
- beta-Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ET-01Biological
Recruited participants will receive ET-01 IV infusion after conditioning.
| Study group | Role | What they receive |
|---|---|---|
| ET-01 | Experimental | BCL11A Enhancer modified Autologous Hematopoietic Stem Cells. Receives: Biological: ET-01 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Frequency and severity of AEs & SAEs identified according to NCI CTCAE 5.0.
Measured: From ET-01 infusion to 104 weeks post-transplant
All-cause mortality
Measured: From signing of informed consent to 104 weeks post-transplant
Incidence of transplant-related mortality
Measured: Within 100 days post-transplant
Proportion of subjects with engraftment
Measured: Up to 42 days post-transplant
Secondary outcomes (5)
Change of total hemoglobin from baseline
Measured: Within 104 weeks post-transplant
Change of HbF from baseline
Measured: Within 104 weeks post-transplant
Change of proportion of HbF/Hb
Measured: Within 104 weeks post-transplant
Change of frequency of packed RBC transfusions
Measured: From 6 months before recruitment to 104 weeks post-transplant
Change of volume of packed RBC transfusions
Measured: From 6 months before recruitment to 104 weeks post-transplant
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 May 2020
- Study start10 Feb 2023
- Primary completion5 Mar 2023
- Study completion5 Mar 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Transfusion Dependent Beta-ThalassaemiaStudies 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
- 3 participants (actual)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Collaborators
- EdiGene Inc., The Affiliated Hospital Of Guizhou Medical University, Zunyi Medical College
- Responsible party
- Sponsor
- Investigators
- Jun Shi, PhD, Principal investigator, Institute of Hematology & Blood Diseases Hospital, China
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04390971
- Sponsor's protocol ID
- SC2019004
- Status last verified
- Nov 2023
- First submitted
- 12 May 2020
- Last updated
- 11 Feb 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 11 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multi-center, Open Label Study to Evaluate the Safety and Efficacy of ET-01 Transplantation in Subjects With Transfusion Dependent β-Thalassemia
- ClinicalTrials.gov ID
- NCT04390971
- Acronym / sponsor ID
- SC2019004
- Conditions
- Transfusion Dependent Beta-Thalassaemia
- Keywords
- Beta-Thalassaemia, Hematopoietic Stem-Cell Transplantation
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 3 participants (actual)
- Lead sponsor
- Institute of Hematology & Blood Diseases Hospital, ChinaUniversity, hospital or other
- Collaborators
- EdiGene Inc., The Affiliated Hospital Of Guizhou Medical University, Zunyi Medical College
- Study start
- 10 Feb 2023 (actual)
- Primary completion
- 5 Mar 2023 (actual)
- Study completion
- 5 Mar 2023 (actual)
- First posted
- 18 May 2020 (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, multi-center study to evaluate the safety and Efficacy of ET-01 Transplantation in subjects with Transfusion Dependent β-Thalassaemia.
Detailed description
After the proper subject is recruited, subject will go through steps generally as stem cell mobilization, apheresis, conditioning and ET-01 infusion to complete therapy. Two years follow-up will be carried out post-transplantation and related data will be collected. Subjects who have been treated by ET-01 will be asked to participate in a long-term follow up study. Monitoring the long-term efficacy and safety up to 15 years after ET-01 transplant.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| ET-01 | Experimental | Biological: ET-01BCL11A Enhancer modified Autologous Hematopoietic Stem Cells. |
Who may qualify
- Ages
- 6 to 35 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (7 listed)
- Subject and/or subject's legal personal representative fully understand and voluntarily sign informed consent forms;
- 6~35 years old, all gender;
- Diagnosis of transfusion dependent β-thalassemia (β-TDT) as defined by protocol;
- Eligible for autologous stem cell transplant;
- Eligible for autologous stem cell transplant;
Key exclusion criteria (5 listed)
- Subjects with associated α-thalassemia;
- Active bacterial, virus, fungal or parasitic infection, including HIV infection, HbsAg and HBV DNA positive, HCV DNA positive, or Treponema Pallidum infection;
- HLA identical sibling or unrelated donors are available;
- Prior allo-HSCT or gene therapy.
- Other protocol defined exclusion criteria may apply.
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 |
|---|---|---|
| ET-01Recruited participants will receive ET-01 IV infusion after conditioning. | Biological | ET-01 |
Primary outcome
Frequency and severity of AEs & SAEs identified according to NCI CTCAE 5.0.
Time frame: From ET-01 infusion to 104 weeks post-transplant
All-cause mortality
Time frame: From signing of informed consent to 104 weeks post-transplant
Incidence of transplant-related mortality
Time frame: Within 100 days post-transplant
Proportion of subjects with engraftment
Time frame: Up to 42 days post-transplant
5 secondary outcomes
Change of total hemoglobin from baseline
Time frame: Within 104 weeks post-transplant
Change of HbF from baseline
Time frame: Within 104 weeks post-transplant
Change of proportion of HbF/Hb
Time frame: Within 104 weeks post-transplant
Change of frequency of packed RBC transfusions
Time frame: From 6 months before recruitment to 104 weeks post-transplant
Change of volume of packed RBC transfusions
Time frame: From 6 months before recruitment to 104 weeks post-transplant
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Feb 2026.
- Individual participant dataSharing planned: No
Related trials for Transfusion Dependent Beta-Thalassaemia
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Growth and Development-related Outcomes in Children With Transfusion-dependent Beta-thalassemia After 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
RecruitingPhase 3
A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT)
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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
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing ET-01 in people with Transfusion Dependent Beta-Thalassaemia, run by Institute of Hematology & Blood Diseases Hospital, China at 3 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 7 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: finished, as of the sponsor's last update on 11 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 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.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Frequency and severity of AEs & SAEs identified according to NCI CTCAE 5.0. (time frame: From ET-01 infusion to 104 weeks post-transplant). The study lists 4 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 11 Feb 2026.