Severe Aplastic Anemia (SAA)Phase 2NCT07523009
CsA+EPAG/HPAG+Romiplostim N01 in the Treatment of Newly-diagnosed SAA/TD-NSAA
A Phase 2 study testing Cyclosporine (CsA), Thrombopoietin Receptor Agonist and Romiplostim N01 in people with Severe Aplastic Anemia (SAA) and Transfusion-dependent Non-severe Aplastic Anemia, run by Peking Union Medical College Hospital.
Trial summary
This study aimed to explore the efficacy and safety of cyclosporine (CsA) combined with eltrombopag (EPAG)/hetrombopag (HPAG) and romiplostim N01 in the treatment of newly-diagnosed transfusion-dependent aplastic anemia (TD-NSAA) and severe aplastic anemia (SAA) Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Severe Aplastic Anemia (SAA), Transfusion-dependent Non-severe Aplastic Anemia
- What is being tested?
- Cyclosporine (CsA), Thrombopoietin Receptor Agonist and Romiplostim N01
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- Jun 2026to Dec 2029
- Phase
- Phase 2
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Age ≥ 18 years old;
- Diagnosed with aplastic anemia (AA) through routine blood tests, bone marrow puncture, bone marrow biopsy, and exclusion tests, and determined as transfusion-dependent non-severe aplastic anemia (TD-NSAA) or severe aplastic anemia (SAA) according to the Camitta criteria; Platelet < 30×10^9/L;
- Had no HLA-matched donors or was not suitable for first-line allogeneic hematopoietic stem cell transplantation (HSCT);
- Not suitable for ATG, due to reasons such as age, complications, and the patient's own wishes;
- With baseline liver and kidney functions <2 ULN;
- ECOG score ≤ 2;
- Signed the informed consent;
Who cannot join
- Had other primary or secondary bone marrow failure (BMF) diseases, such as Fanconi anemia, congenital keratinization disorder, etc.;
- With evidence of clonal hematological bone marrow diseases (MDS, AML) in cytogenetics;
- PNH clone ≥ 50%;
- Received HSCT before enrollment;
- Previously used immunosuppressive treatments such as ATG, CsA, TPO receptor agonists (TPO-RAs);
- Allergic or intolerant to romiplostim N01, eltrombopag, hetrombopag, or CsA;
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- Pregnant or lactating patients;
- Severe bleeding or infection that cannot be controlled by standard treatment;
- History of arterial or venous thrombosis;
- Complicated with malignant tumors;
- Participated in other clinical trials within 3 months;
- Patients considered not suitable to participate in this study by the investigator.
About this study
Official title: Cyclosporine, Eltrombopag or Hetrombopag, and Romiplostim N01 in the Treatment of Newly-diagnosed Transfusion-dependent Non-severe Aplastic Anemia/ Severe Aplastic Anemia
This study aimed to explore the efficacy and safety of cyclosporine (CsA) combined with eltrombopag (EPAG)/hetrombopag (HPAG) and romiplostim N01 in the treatment of newly-diagnosed transfusion-dependent aplastic anemia (TD-NSAA) and severe aplastic anemia (SAA)
Read the detailed description
For SAA/TD-NSAA patients without HLA-matched donors and those over 40 years old, the first choice is immunosuppressive therapy (IST) + cyclosporine A (CsA) combined with thrombopoietin receptor agonists (TPO-RAs).
TPO-RAs could bind to the thrombopoietin (TPO) receptor, causing conformational changes in the TPO receptor and activating the JAK2/STAT5 pathway, thereby increasing the proliferation of megakaryocyte progenitor cells and platelet production. Previous studies have shown that compared with IST alone, the combination of IST and eltrombopag (EPAG)/hetrombopag (HPAG) as the first-line treatment for SAA can increase the overall response rate (ORR) to 60%-70%.
However, ATG treatment requires hospitalization and has significant toxic effects, leading to a high risk of complications in the elderly or patients with poor health. Therefore, in recent years, treatment regimens without ATG have also been gradually explored. The results of the SOAR trial showed that in newly diagnosed SAA patients, the overall hematological response rate after 6 months of CsA + EPAG was 46%.
In a phase II/III study for refractory AA, romiplostim monotherapy achieved an ORR of 84% at week 27. Although both romiplostim and eltrombopag/hyrtiopeg activate the TPO receptor (c-Mpl), there are differences and complementarities in their molecular mechanisms. Romiplostim is a peptide mimetic that binds to the extracellular domain of the receptor to mimic endogenous TPO; while eltrombopag and hetrombopag are small molecules that target the transmembrane domain, among which hetrombopag replaces the biphenyl structure to enhance lipophilicity, improve efficacy, and reduce liver toxicity. The binding sites of the two drugs are spatially separated and may produce a synergistic effect through different intensities and dynamics of downstream STAT, PI3K/AKT, and other signaling pathways.
- Being tested
- Cyclosporine (CsA), Thrombopoietin Receptor Agonist, Romiplostim N01
- Medical subject headings
- Anemia, Aplastic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Cyclosporine (CsA)Drug
CsA 3-5mg/kg/d
Thrombopoietin Receptor AgonistDrug
Eltrombopag: initial dose 50mg/d, maximum dose 150mg/d hetrombopag 7.5mg/d, maximum dose 15mg/d
Romiplostim N01Drug
Romiplostim N01: 20 µg/kg, subcutaneously, once a week
| Study group | Role | What they receive |
|---|---|---|
| CsA+EPAG/HPAG+Romiplostim N01 | Experimental | CsA 3-5mg/kg/d, trough concentration 100-200ng/ml Eltrombopag 50mg/d, increased by 25mg every two weeks Hetrombopag 7.5mg/d, increased by 2.5mg every two weeks Romiplostim N01 20µg/kg subcutaneously, once a week Receives: Drug: Cyclosporine (CsA); Drug: Thrombopoietin Receptor Agonist; Drug: Romiplostim N01 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall response rate (ORR)
ORR=CRR+PRR
Measured: 6-month
Secondary outcomes (3)
ORR
ORR=PRR+CRR
Measured: 3-month, 12-month
red blood cell (RBC)/platelet (PLT) transfusion independent rate
Proportion of patients who achieve red blood cell (RBC)/platelet (PLT) transfusion independence for 8 weeks or longer
Measured: 3-month, 6-month, 12-month
AE rate
proportion of patients with adverse events, according to CTCAE
Measured: through study completion, an average of 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Apr 2026
- Study startJun 2026 (estimated)
- Primary completionDec 2028 (estimated)
- Study completionDec 2029 (estimated)
Why it stopped: The registration of this research plan was incorrect. A new registration has been re-applied for.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Severe Aplastic Anemia (SAA)Studies 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
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
- NCT07523009
- Sponsor's protocol ID
- LP-N01
- Status last verified
- Jun 2026
- First submitted
- 27 Mar 2026
- Last updated
- 4 Jun 2026
- Results posted
- 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 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Cyclosporine, Eltrombopag or Hetrombopag, and Romiplostim N01 in the Treatment of Newly-diagnosed Transfusion-dependent Non-severe Aplastic Anemia/ Severe Aplastic Anemia
- ClinicalTrials.gov ID
- NCT07523009
- Acronym / sponsor ID
- LP-N01
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: The registration of this research plan was incorrect. A new registration has been re-applied for.
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Peking Union Medical College HospitalUniversity, hospital or other
- Study start
- Jun 2026 (estimated)
- Primary completion
- Dec 2028 (estimated)
- Study completion
- Dec 2029 (estimated)
- First posted
- 13 Apr 2026 (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 study aimed to explore the efficacy and safety of cyclosporine (CsA) combined with eltrombopag (EPAG)/hetrombopag (HPAG) and romiplostim N01 in the treatment of newly-diagnosed transfusion-dependent aplastic anemia (TD-NSAA) and severe aplastic anemia (SAA)
Detailed description
For SAA/TD-NSAA patients without HLA-matched donors and those over 40 years old, the first choice is immunosuppressive therapy (IST) + cyclosporine A (CsA) combined with thrombopoietin receptor agonists (TPO-RAs).
TPO-RAs could bind to the thrombopoietin (TPO) receptor, causing conformational changes in the TPO receptor and activating the JAK2/STAT5 pathway, thereby increasing the proliferation of megakaryocyte progenitor cells and platelet production. Previous studies have shown that compared with IST alone, the combination of IST and eltrombopag (EPAG)/hetrombopag (HPAG) as the first-line treatment for SAA can increase the overall response rate (ORR) to 60%-70%.
However, ATG treatment requires hospitalization and has significant toxic effects, leading to a high risk of complications in the elderly or patients with poor health. Therefore, in recent years, treatment regimens without ATG have also been gradually explored. The results of the SOAR trial showed that in newly diagnosed SAA patients, the overall hematological response rate after 6 months of CsA + EPAG was 46%.
In a phase II/III study for refractory AA, romiplostim monotherapy achieved an ORR of 84% at week 27. Although both romiplostim and eltrombopag/hyrtiopeg activate the TPO receptor (c-Mpl), there are differences and complementarities in their molecular mechanisms. Romiplostim is a peptide mimetic that binds to the extracellular domain of the receptor to mimic endogenous TPO; while eltrombopag and hetrombopag are small molecules that target the transmembrane domain, among which hetrombopag replaces the biphenyl structure to enhance lipophilicity, improve efficacy, and reduce liver toxicity. The binding sites of the two drugs are spatially separated and may produce a synergistic effect through different intensities and dynamics of downstream STAT, PI3K/AKT, and other signaling pathways.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| CsA+EPAG/HPAG+Romiplostim N01 | Experimental | Drug: Cyclosporine (CsA), Drug: Thrombopoietin Receptor Agonist, Drug: Romiplostim N01CsA 3-5mg/kg/d, trough concentration 100-200ng/ml Eltrombopag 50mg/d, increased by 25mg every two weeks Hetrombopag 7.5mg/d, increased by 2.5mg every two weeks Romiplostim N01 20µg/kg subcutaneously, once a week |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Age ≥ 18 years old;
- Diagnosed with aplastic anemia (AA) through routine blood tests, bone marrow puncture, bone marrow biopsy, and exclusion tests, and determined as transfusion-dependent non-severe aplastic anemia (TD-NSAA) or severe aplastic anemia (SAA) according to the Camitta criteria; Platelet < 30×10^9/L;
- Had no HLA-matched donors or was not suitable for first-line allogeneic hematopoietic stem cell transplantation (HSCT);
- Not suitable for ATG, due to reasons such as age, complications, and the patient's own wishes;
- With baseline liver and kidney functions <2 ULN;
Key exclusion criteria (12 listed)
- Had other primary or secondary bone marrow failure (BMF) diseases, such as Fanconi anemia, congenital keratinization disorder, etc.;
- With evidence of clonal hematological bone marrow diseases (MDS, AML) in cytogenetics;
- PNH clone ≥ 50%;
- Received HSCT before enrollment;
- Previously used immunosuppressive treatments such as ATG, CsA, TPO receptor agonists (TPO-RAs);
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 |
|---|---|---|
| Cyclosporine (CsA)CsA 3-5mg/kg/d | Drug | CsA+EPAG/HPAG+Romiplostim N01 |
| Thrombopoietin Receptor AgonistEltrombopag: initial dose 50mg/d, maximum dose 150mg/d hetrombopag 7.5mg/d, maximum dose 15mg/d | Drug | CsA+EPAG/HPAG+Romiplostim N01 |
| Romiplostim N01Romiplostim N01: 20 µg/kg, subcutaneously, once a week | Drug | CsA+EPAG/HPAG+Romiplostim N01 |
Primary outcome
Overall response rate (ORR)
Time frame: 6-month
ORR=CRR+PRR
3 secondary outcomes
ORR
Time frame: 3-month, 12-month
red blood cell (RBC)/platelet (PLT) transfusion independent rate
Time frame: 3-month, 6-month, 12-month
AE rate
Time frame: through study completion, an average of 1 year
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Jun 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.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Cyclosporine (CsA), Thrombopoietin Receptor Agonist and Romiplostim N01 in people with Severe Aplastic Anemia (SAA) and Transfusion-dependent Non-severe Aplastic Anemia, 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 12 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: withdrawn, as of the sponsor's last update on 4 Jun 2026. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 withdrawn before enrolling participants, so there are no results.
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?
Overall response rate (ORR) (time frame: 6-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 4 Jun 2026.