Transfusion Dependent Beta-ThalassaemiaNCT05752123
A Study to Evaluate the Safety and Efficacy 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 EdiGene (GuangZhou) Inc. at 1 site in China.
Trial summary
This is an open label, single site 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
- 2 participants
- Where
- 1 siteChina
- Study dates
- 18 Feb 2023to 12 Mar 2025
- 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;
- 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
1 site in 1 country.
About this study
Official title: A Single Site, 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, single site study to evaluate the safety and Efficacy of ET-01 Transplantation in subjects with Transfusion Dependent β-Thalassaemia.
- Conditions
- Transfusion Dependent Beta-Thalassaemia
- Being tested
- ET-01
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 in 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 posted2 Mar 2023
- Study start18 Feb 2023
- Primary completion29 Jan 2024
- Study completion12 Mar 2025
Why it stopped: Development plan change
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.
- 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
- 2 participants (actual)
Sponsor and investigators
- Lead sponsor
- EdiGene (GuangZhou) Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- The 923rd Hospital of Joint Logistics Support Force of People's Liberation Army
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05752123
- Sponsor's protocol ID
- EDI-001-002
- Status last verified
- Mar 2025
- First submitted
- 16 Feb 2023
- Last updated
- 30 Mar 2025
- 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 30 Mar 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Single Site, Open Label Study to Evaluate the Safety and Efficacy of ET-01 Transplantation in Subjects with Transfusion Dependent Β-Thalassemia
- ClinicalTrials.gov ID
- NCT05752123
- Acronym / sponsor ID
- EDI-001-002
- Conditions
- Transfusion Dependent Beta-Thalassaemia
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Development plan change
- Enrolment
- 2 participants (actual)
- Lead sponsor
- EdiGene (GuangZhou) Inc.Industry (pharma, biotech, devices)
- Collaborators
- The 923rd Hospital of Joint Logistics Support Force of People's Liberation Army
- Study start
- 18 Feb 2023 (actual)
- Primary completion
- 29 Jan 2024 (actual)
- Study completion
- 12 Mar 2025 (actual)
- First posted
- 2 Mar 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, single site study to evaluate the safety and Efficacy of ET-01 Transplantation in subjects with Transfusion Dependent β-Thalassaemia.
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 (6 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;
- Organs in good function.
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 in 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 was stopped early (reason given: Development plan change). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Mar 2025.
- 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.
The record lists no contact. Use the official record to reach the sponsor.
1 site 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 EdiGene (GuangZhou) Inc. 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 6 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: stopped early, as of the sponsor's last update on 30 Mar 2025. 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: Development plan change). 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?
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 30 Mar 2025.