Transfusion Dependent Beta-ThalassaemiaPhase 1ET-01NCT04925206
A Safety and Efficacy Study Evaluating ET-01 in Subjects with Transfusion Dependent Β-Thalassaemia
A Phase 1 study testing ET-01 in people with Transfusion Dependent Beta-Thalassaemia, run by EdiGene (GuangZhou) Inc. at 4 sites in China.
Trial summary
This is a single-arm, open label, multi-center, single-dose phase 1 study in subjects with transfusion dependent β-thalassaemia. The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) using ET-01. Read more about this study
- Who is it for?
- Ages 12 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
- 8 participants
- Where
- 4 sitesChina
- Study dates
- 17 Aug 2021to 12 Mar 2025
- Phase
- Phase 1
- 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 12 to 35 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Subjects and/or legal representative fully understand and voluntarily sign informed consent forms.
- Diagnosis of transfusion dependent β-thalassemia (β-TDT) as defined by protocol.
- Be not appropriate to receive conventional allogeneic hematopoietic stem cell transplantation.
- Lansky/Karnofsky score ≥ 70%.
- Eligible for hematopoietic stem cell transplantation and conditioning with busulfan as per investigator's judgement.
- Other protocol defined inclusion criteria may apply.
Who cannot join
- Subjects with associated α-thalassemia.
- Subjects with any clinically significant acute or uncontrolled infections.
- History of uncontrolled epilepsy or other mental disorders.
- Previous treatment with allogeneic bone marrow transplantation or gene therapy.
- Other protocol defined exclusion criteria may apply.
Trial locations and contacts
4 sites in 1 country.
China4 sites
Nanfang Hospital of Southern Medical University
Guangzhou, Guangdong, 510515
Guangzhou Women and Children's Medical Center
Guangzhou, Guangdong, 510623
Shenzhen Children's Hospital
Shenzhen, Guangdong, 518035
Institute of Hematology & Blood Diseases Hospital
Tianjin, Tianjin Municipality, 300020
About this study
Official title: A Multicenter, Open Label Phase 1 Study to Evaluate the Safety and Efficacy of a Single Dose of Autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) in Subjects with Transfusion Dependent Β-Thalassaemia
This is a single-arm, open label, multi-center, single-dose phase 1 study in subjects with transfusion dependent β-thalassaemia. The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) using ET-01.
- Conditions
- Transfusion Dependent Beta-Thalassaemia
- Being tested
- ET-01
- Keywords
- Beta-Thalassaemia, CRISPR-Cas9, Human Hematopoietic Stem and Progenitor Cells
- 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 following myeloablative conditioning with busulfan.
| Study group | Role | What they receive |
|---|---|---|
| ET-01 | Experimental | Not described 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 collected AEs & SAEs
Measured: Within 24 months after ET-01 infusion
All-cause mortality
Measured: From signing of informed consent up to 24 months post-ET-01 infusion
Incidence of transplant-related mortality
Measured: From baseline (pre-transfusion) up to 12 months post-ET-01 infusion
Total lymphocyte count
Measured: Within 24 months after ET-01 infusion
Proportion of subjects with abnormal proliferation of blood cells
Measured: Within 24 months after ET-01 infusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Jun 2021
- Study start17 Aug 2021
- Primary completion30 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 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 8 participants (actual)
Sponsor and investigators
- Lead sponsor
- EdiGene (GuangZhou) Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04925206
- Sponsor's protocol ID
- EDG-001
- Acronym
- ET-01
- Status last verified
- Mar 2025
- First submitted
- 4 Jun 2021
- Last updated
- 30 Mar 2025
- Results posted
- No
- Data monitoring committee
- Yes
- 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 Multicenter, Open Label Phase 1 Study to Evaluate the Safety and Efficacy of a Single Dose of Autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) in Subjects with Transfusion Dependent Β-Thalassaemia
- ClinicalTrials.gov ID
- NCT04925206
- Acronym / sponsor ID
- ET-01 / EDG-001
- Conditions
- Transfusion Dependent Beta-Thalassaemia
- Keywords
- Beta-Thalassaemia, CRISPR-Cas9, Human Hematopoietic Stem and Progenitor Cells
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Development plan change
- Enrolment
- 8 participants (actual)
- Lead sponsor
- EdiGene (GuangZhou) Inc.Industry (pharma, biotech, devices)
- Study start
- 17 Aug 2021 (actual)
- Primary completion
- 30 Jan 2024 (actual)
- Study completion
- 12 Mar 2025 (actual)
- First posted
- 14 Jun 2021 (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 label, multi-center, single-dose phase 1 study in subjects with transfusion dependent β-thalassaemia. The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) using ET-01.
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-01 |
Who may qualify
- Ages
- 12 to 35 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (6 listed)
- Subjects and/or legal representative fully understand and voluntarily sign informed consent forms.
- Diagnosis of transfusion dependent β-thalassemia (β-TDT) as defined by protocol.
- Be not appropriate to receive conventional allogeneic hematopoietic stem cell transplantation.
- Lansky/Karnofsky score ≥ 70%.
- Eligible for hematopoietic stem cell transplantation and conditioning with busulfan as per investigator's judgement.
Key exclusion criteria (5 listed)
- Subjects with associated α-thalassemia.
- Subjects with any clinically significant acute or uncontrolled infections.
- History of uncontrolled epilepsy or other mental disorders.
- Previous treatment with allogeneic bone marrow transplantation 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 following myeloablative conditioning with busulfan. | Biological | ET-01 |
Primary outcome
Frequency and severity of collected AEs & SAEs
Time frame: Within 24 months after ET-01 infusion
All-cause mortality
Time frame: From signing of informed consent up to 24 months post-ET-01 infusion
Incidence of transplant-related mortality
Time frame: From baseline (pre-transfusion) up to 12 months post-ET-01 infusion
Total lymphocyte count
Time frame: Within 24 months after ET-01 infusion
Proportion of subjects with abnormal proliferation of blood cells
Time frame: Within 24 months after ET-01 infusion
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.
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing ET-01 in people with Transfusion Dependent Beta-Thalassaemia, run by EdiGene (GuangZhou) Inc. at 4 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 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 4 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 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.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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 collected AEs & SAEs (time frame: Within 24 months after ET-01 infusion). The study lists 5 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.