Aplastic AnemiasPhase 3NCT07001397
Study on the Short-term Efficacy and Safety of Recombinant Human Thrombopoietin Combined With Immunosuppressant Sequential Eltrombopag Ethanolamine Dry Suspension in the Treatment of SAA/TD-NSAA
A Phase 3 study testing EPAG-pfos in people with Aplastic Anemias, run by Peking Union Medical College Hospital.
Trial summary
The study included adult patients ≥18 years old with SAA and TD-NSAA who had no HLA-matched sibling donors or were not suitable for first-line allogeneic hematopoietic stem cell transplantation due to age, complications, patient wishes, etc. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Aplastic Anemias
- What is being tested?
- EPAG-pfos
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 70 participants
- Where
- No sites listed yet
- Study dates
- 23 Jun 2025to 23 Mar 2027
- Phase
- Phase 3
- 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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Age ≥18 years old, gender not limited;
- Patients diagnosed with aplastic anemia (AA) through blood routine, bone marrow puncture, bone marrow biopsy and excluded examinations, and determined as SAA or TD-NSAA according to the Camitta criteria;
- Those who have not received treatment with immunosuppressants (such as cyclosporine, tacrolimus, ATG, etc.);
- The patient has no HLA-matched sibling donor or is not suitable for first-line allogeneic hematopoietic stem cell transplantation due to reasons such as age, complications, or the patient's wishes.
- Before treatment, the total bilirubin (TBIL) of the patient was less than 1.5 times the upper limit of normal value (ULN), alanine aminotransferase (ALT) and aspartate aminotransferase (AST) were less than 3 times the ULN, and serum creatinine was less than 1.5 times the ULN.
- ECOG score ≤2 points;
- Voluntarily join this study, sign the informed consent form, have good compliance and be willing to cooperate with regular follow-up for therapeutic effect evaluation and side effect monitoring;
Who cannot join
- Pregnant or lactating patients;
- Have a history of any arterial or venous thrombosis (including stroke, transient ischemic attack, myocardial infarction, deep vein thrombosis or pulmonary embolism) within half a year before enrollment and have at least two of the following risk factors: Hormone replacement therapy, oral contraceptives (including estrogen), smoking, diabetes, hypercholesterolemia, drug-controlled hypertension, hereditary coagulation disorders;
- Patients with severe cardiovascular diseases (such as NYHA Cardiac Function Score grade III-IV) within half a year before enrollment, known arrhythmias that increase the risk of thromboembolism such as atrial fibrillation, after coronary stent implantation, angioplasty, and after coronary artery bypass grafting;
- Combined with malignant tumors that seriously affect survival;
- Within 2 weeks after the start of the study until the end of the study, continuous drug treatment (including but not limited to aspirin, clopidogrel and/or non-steroidal anti-inflammatory drugs NSAIDs) or anticoagulant treatment that affects platelet function for more than 3 days;
- Currently, there is a severe or difficult-to-control infection (CTC AE level 2 infection);
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- Bone marrow biopsy within 4 weeks before the administration of the first dose of the study indicates the presence of primary diseases other than SAA that can cause thrombocytopenia (such as primary myelodysplastic syndrome (MDS), congenital bone marrow failure diseases (such as Fanconi anemia, congenital keratosis, etc.) Or accompanied by obvious paroxysmal nocturnal hemoglobinuria (PNH) clones (> 50%) and AA with clinical and biochemical indicators of hemolysis; And the researcher judged that the subject was not suitable to participate in this study.
- All laboratory or clinical evidence of HIV infection, previous clinical history of hepatitis C, previous hepatitis B infection, or active hepatitis at the time of screening. Laboratory tests during the screening period indicated hepatitis C infection or hepatitis B infection. (Defined as a positive HBsAg test. Additionally, if the HBsAg test is negative but HBcAb is positive, regardless of the status of HBsAb, HBV DNA testing is required. If it is positive, the subject should be excluded.)
- Chronic liver disease-related AA (hepatitis disease-related AA, liver cirrhosis, etc.);
- Had received treatment with TPO or TPO receptor agonists (TPO-RAS, including but not limited to eltrombopag, romisetine, avatrombopag, heltrombopag, rutrombopag, etc.) before screening;
- Suffer from severe autoimmune diseases or immunodeficiency diseases;
- Suffer from serious mental disorders;
- Patients who the researchers consider unsuitable to participate in this trial, such as those with any other medical, social or psychological factors that may affect safety or compliance with the research procedures.
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
Hanbing contact
About this study
The study included adult patients ≥18 years old with SAA and TD-NSAA who had no HLA-matched sibling donors or were not suitable for first-line allogeneic hematopoietic stem cell transplantation due to age, complications, patient wishes, etc. After admission, they were treated with standard IST combined with rhTPO for at least 28 days. After discharge, the sequential treatment of EPAG-PFOS (with an initial dose of 150mg/d) was continued for 2 months (8 weeks), and the follow-up was conducted until the 6th month (24 weeks). It was compared with the historical cohort previously treated with IST combined with EPAG to explore the short-term efficacy and safety of this treatment regimen.
- Conditions
- Aplastic Anemias
- Being tested
- EPAG-pfos
- Keywords
- Aplastic anemia, rhTPO, EPAG-pfos
- Medical subject headings
- Anemia, Aplastic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EPAG-pfosDrug
The initial dose of EPAG-pfos is 150mg/d orally. The dose is adjusted according to hematological indicators, with a maximum dose of 150mg/d until hematological remission is achieved
| Study group | Role | What they receive |
|---|---|---|
| rhTPO+IST+EPAG-pfos | Active comparator | Not described Receives: Drug: EPAG-pfos |
What is being measured
The primary outcome is the main question the study is designed to answer.
overall response rate
Measured: 1 month, 3 month
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Jun 2025
- Study start23 Jun 2025 (estimated)
- Primary completion23 Mar 2026 (estimated)
- Study completion23 Mar 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Aplastic AnemiasStudies of the same condition, open and under way.
- More trials run by Peking Union Medical College HospitalThe 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 70 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Peking Union Medical College Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Bing Han, Peking Union Medical College Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07001397
- Sponsor's protocol ID
- TPO-2024-016
- Status last verified
- May 2025
- First submitted
- 23 May 2025
- Last updated
- 3 Jun 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 3 Jun 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Study on the Short-term Efficacy and Safety of Recombinant Human Thrombopoietin Combined With Immunosuppressant Sequential Eltrombopag Ethanolamine Dry Suspension in the Treatment of SAA/TD-NSAA
- ClinicalTrials.gov ID
- NCT07001397
- Acronym / sponsor ID
- TPO-2024-016
- Conditions
- Aplastic Anemias
- Keywords
- Aplastic anemia, rhTPO, EPAG-pfos
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 70 participants (estimated)
- Lead sponsor
- Peking Union Medical College HospitalUniversity, hospital or other
- Study start
- 23 Jun 2025 (estimated)
- Primary completion
- 23 Mar 2026 (estimated)
- Study completion
- 23 Mar 2027 (estimated)
- First posted
- 3 Jun 2025 (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.
The study included adult patients ≥18 years old with SAA and TD-NSAA who had no HLA-matched sibling donors or were not suitable for first-line allogeneic hematopoietic stem cell transplantation due to age, complications, patient wishes, etc. After admission, they were treated with standard IST combined with rhTPO for at least 28 days. After discharge, the sequential treatment of EPAG-PFOS (with an initial dose of 150mg/d) was continued for 2 months (8 weeks), and the follow-up was conducted until the 6th month (24 weeks). It was compared with the historical cohort previously treated with IST combined with EPAG to explore the short-term efficacy and safety of this treatment regimen.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| rhTPO+IST+EPAG-pfos | Active comparator | Drug: EPAG-pfos |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (7 listed)
- Age ≥18 years old, gender not limited;
- Patients diagnosed with aplastic anemia (AA) through blood routine, bone marrow puncture, bone marrow biopsy and excluded examinations, and determined as SAA or TD-NSAA according to the Camitta criteria;
- Those who have not received treatment with immunosuppressants (such as cyclosporine, tacrolimus, ATG, etc.);
- The patient has no HLA-matched sibling donor or is not suitable for first-line allogeneic hematopoietic stem cell transplantation due to reasons such as age, complications, or the patient's wishes.
- Before treatment, the total bilirubin (TBIL) of the patient was less than 1.5 times the upper limit of normal value (ULN), alanine aminotransferase (ALT) and aspartate aminotransferase (AST) were less than 3 times the ULN, and serum creatinine was less than 1.5 times the ULN.
Key exclusion criteria (13 listed)
- Pregnant or lactating patients;
- Have a history of any arterial or venous thrombosis (including stroke, transient ischemic attack, myocardial infarction, deep vein thrombosis or pulmonary embolism) within half a year before enrollment and have at least two of the following risk factors: Hormone replacement therapy, oral contraceptives (including estrogen), smoking, diabetes, hypercholesterolemia, drug-controlled hypertension, hereditary coagulation disorders;
- Patients with severe cardiovascular diseases (such as NYHA Cardiac Function Score grade III-IV) within half a year before enrollment, known arrhythmias that increase the risk of thromboembolism such as atrial fibrillation, after coronary stent implantation, angioplasty, and after coronary artery bypass grafting;
- Combined with malignant tumors that seriously affect survival;
- Within 2 weeks after the start of the study until the end of the study, continuous drug treatment (including but not limited to aspirin, clopidogrel and/or non-steroidal anti-inflammatory drugs NSAIDs) or anticoagulant treatment that affects platelet function for more than 3 days;
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 |
|---|---|---|
| EPAG-pfosThe initial dose of EPAG-pfos is 150mg/d orally. The dose is adjusted according to hematological indicators, with a maximum dose of 150mg/d until hematological remission is achieved | Drug | rhTPO+IST+EPAG-pfos |
Primary outcome
overall response rate
Time frame: 1 month, 3 month
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 3 Jun 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.
Hanbing contact
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing EPAG-pfos in people with Aplastic Anemias, run by Peking Union Medical College Hospital.
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 13 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 3 Jun 2025. Status can change between updates, so confirm with the study contact before making plans.
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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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 Hanbing (01069155097), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
overall response rate (time frame: 1 month, 3 month). 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 3 Jun 2025.