Severe Aplastic Anemia (SAA)Phase 2NCT07558434
CsA+EPAG/HPAG+Romiplostim N01 in Newly-diagnosed SAA/TD-NSAA
A Phase 2 study testing Cyclosporine (CsA), thrombopoietin receptor agonist (TPO-RA) 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) +eltrombopag (EPAG)/hetrombopag (HPAG)+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 (TPO-RA) and Romiplostim N01
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 43 participants
- Where
- No sites listed yet
- Study dates
- Apr 2026to Dec 2029
- Phase
- Phase 2
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.
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
Official title: Cyclosporine + Eltrombopag/Hetrombopag + 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)
+eltrombopag (EPAG)/hetrombopag (HPAG)+romiplostim N01 in the treatment of newly-diagnosed transfusion-dependent aplastic anemia (TD-NSAA) and severe aplastic anemia (SAA)
- Being tested
- Cyclosporine (CsA), thrombopoietin receptor agonist (TPO-RA), 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, trough concentration 100-200ng/ml
thrombopoietin receptor agonist (TPO-RA)Drug
Also called Eltrombopag (EPAG), Hetrombopag (HPAG)
Eltrombopag 50mg/d, increased by 25mg every two weeks Hetrombopag 7.5mg/d, increased by 2.5mg every two weeks
Romiplostim N01Drug
Romiplostim N01 20µg/kg, subcutaneously, once a week
| Study group | Role | What they receive |
|---|---|---|
| CsA+EPAG/HPAG+Romiplostim N01 | Experimental | CsA 3-5mg/kg/d EPAG 50mg/d or HPAG 7.5mg/d Romiplostim N01 20µg/kg, subcutaneously, once a week Receives: Drug: Cyclosporine (CsA); Drug: thrombopoietin receptor agonist (TPO-RA); 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 posted30 Apr 2026
- Study startApr 2026 (estimated)
- Primary completionDec 2028 (estimated)
- Study completionDec 2029 (estimated)
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
- 43 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
- NCT07558434
- Sponsor's protocol ID
- K10230
- Status last verified
- Apr 2026
- First submitted
- 23 Apr 2026
- Last updated
- 30 Apr 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No