Aplastic AnemiaPhase 2NCT07572084
Romiplostim N01 in Chemoradiotherapy-induced Aplastic Anemia
A Phase 2 study testing Romiplostim N01 in people with Aplastic Anemia, run by Peking Union Medical College Hospital.
Trial summary
This study aimed to explore the efficacy and safety of romiplostim N01 in the treatment of chemoradiotherapy-induced aplastic anemia (AA) Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Aplastic Anemia
- What is being tested?
- 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
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Age ≥ 18 years old;
- Diagnosed with relapsed/refractory chemoradiotherapy-induced AA. Relapsed/ refractory was defined as patients who had no response to at least 3 months of cyclosporine or thrombopoietin receptor agonists (TPO-RAs) such as eltrombopag;
- At least one of the following conditions must be met: hemoglobin < 90 g/ L, platelet count < 30×109/L, neutrophil count < 1.0×109/L;
- With baseline liver and kidney functions <2 upper limit of normal (ULN);
- Without active infection that cannot be controlled by standard treatment;
- Signed the informed consent;
- ECOG score ≤ 2;
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;
- Paroxysmal nocturnal hemoglobinuria (PNH) clone ≥ 50%;
- Received hematopoietic stem cell transplantation before enrollment;
- Received ATG treatment before enrollment;
- Severe bleeding or infection that cannot be controlled by standard treatment;
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- Allergic or intolerant to romiplostim N01;
- Active infections of HIV, HCV, or HBV, liver cirrhosis, or portal hypertension
- History of malignant tumors within 5 years;
- History of arterial or venous thrombosis;
- Pregnant or lactating patients;
- Participated in other clinical trials within 3 months.
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: Romiplostim N01 in the Treatment of Refractory Chemoradiotherapy-induced Aplastic Anemia: a Single-center, Prospective, Open-label Study
This study aimed to explore the efficacy and safety of romiplostim N01 in the treatment of chemoradiotherapy-induced aplastic anemia (AA)
- Conditions
- Aplastic Anemia
- Being tested
- 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.
Romiplostim N01Drug
Romiplostim N01 20 µg/kg subcutaneously once a week for at least 3 months
| Study group | Role | What they receive |
|---|---|---|
| Romiplostim N01 | Experimental | Romiplostim N01 20 µg/kg Receives: Drug: Romiplostim N01 |
What is being measured
The primary outcome is the main question the study is designed to answer.
ORR (Overall Response Rate)
ORR=PRR (Partial Response Rate)+CRR (Complete Response Rate)
Measured: 3-month, 6-month
Secondary outcomes (4)
CRR
Proportion of patients who achieved CR
Measured: 3-month, 6-month
Adverse event (AE) rate
Proportion of patients with AEs, according to CTCAE
Measured: through study completion, an average of 1 year
Predictive factors for ORR or CRR
Calculated by single-/multivariate regression
Measured: 3-month
Clonal evolution rate
Proportion of patients progressed to myelodysplastic syndromes (MDS)/ acute myeloid leukemia (AML)
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 posted7 May 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 Aplastic AnemiaStudies 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
- NCT07572084
- Sponsor's protocol ID
- K10498
- Status last verified
- Apr 2026
- First submitted
- 23 Apr 2026
- Last updated
- 7 May 2026
- 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 7 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Romiplostim N01 in the Treatment of Refractory Chemoradiotherapy-induced Aplastic Anemia: a Single-center, Prospective, Open-label Study
- ClinicalTrials.gov ID
- NCT07572084
- Acronym / sponsor ID
- K10498
- Conditions
- Aplastic Anemia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 43 participants (estimated)
- Lead sponsor
- Peking Union Medical College HospitalUniversity, hospital or other
- Study start
- Apr 2026 (estimated)
- Primary completion
- Dec 2028 (estimated)
- Study completion
- Dec 2029 (estimated)
- First posted
- 7 May 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 romiplostim N01 in the treatment of chemoradiotherapy-induced aplastic anemia (AA)
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Romiplostim N01 | Experimental | Drug: Romiplostim N01Romiplostim N01 20 µg/kg |
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 relapsed/refractory chemoradiotherapy-induced AA. Relapsed/ refractory was defined as patients who had no response to at least 3 months of cyclosporine or thrombopoietin receptor agonists (TPO-RAs) such as eltrombopag;
- At least one of the following conditions must be met: hemoglobin < 90 g/ L, platelet count < 30×109/L, neutrophil count < 1.0×109/L;
- With baseline liver and kidney functions <2 upper limit of normal (ULN);
- Without active infection that cannot be controlled by standard treatment;
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;
- Paroxysmal nocturnal hemoglobinuria (PNH) clone ≥ 50%;
- Received hematopoietic stem cell transplantation before enrollment;
- Received ATG treatment before enrollment;
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 |
|---|---|---|
| Romiplostim N01Romiplostim N01 20 µg/kg subcutaneously once a week for at least 3 months | Drug | Romiplostim N01 |
Primary outcome
ORR (Overall Response Rate)
Time frame: 3-month, 6-month
ORR=PRR (Partial Response Rate)+CRR (Complete Response Rate)
4 secondary outcomes
CRR
Time frame: 3-month, 6-month
Adverse event (AE) rate
Time frame: through study completion, an average of 1 year
Predictive factors for ORR or CRR
Time frame: 3-month
Clonal evolution rate
Time frame: through study completion, an average of 1 year
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 7 May 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.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Romiplostim N01 in people with 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: opening soon, as of the sponsor's last update on 7 May 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?
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 Bing Han (+86 13601059938, hanbing_li@sina.com.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
ORR (Overall Response Rate) (time frame: 3-month, 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 7 May 2026.