Aplastic AnemiaPhase 2NCT07532408

Romiplostim N01 in the Treatment of Refractory Chemoradiotherapy-induced AA

A Phase 2 study testing Romiplostim N01 in people with Aplastic Anemia, run by Peking Union Medical College Hospital.

Withdrawn

Withdrawn. The study stopped before anyone enrolled.

Trial summary

This study aimed to explore the efficacy and safety of romiplostim N01 in the treatment of relapsed/refractory chemoradiotherapy-induced aplastic anemia 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: 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 relapsed/refractory chemoradiotherapy-induced aplastic anemia (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 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;
PNH clone ≥ 50%;
Received HSCT before enrollment;
Received ATG treatment before enrollment;
Severe bleeding or infection that cannot be controlled by standard treatment;

Show all 12Show fewer

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.

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 relapsed/refractory chemoradiotherapy-induced aplastic anemia

Read the detailed description

Currently, there are few studies on the use of TPO-RAs after tumor radiotherapy and chemotherapy. Our center has published a study on the efficacy and safety of avatrombopag (AVA) in AA secondary to radiotherapy and chemotherapy. The study demonstrated that the ORR at 1, 3, and 6 months were 32.4%, 55.9%, and 58.8%, respectively. In a phase II/III study for refractory AA, romiplostim monotherapy achieved an ORR of 84% at week 27, which showed that romiplostim seemed to be effective and safe in patients with refractory aplastic anemia, with a recommended starting dose of 20 μg/kg once a week.

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 groups in NCT07532408
Study group	Role	What they receive
Romiplostim N01	Experimental	Romiplostim N01 20 µg/kg subcutaneously once a week for at least 3 months 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+CRR
Measured: 3-month, 6-month

Secondary outcomes (4)

CRR
Complete response rate
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
Predictive factors of efficacy
Predictive factors of ORR or CRR
Measured: 3-month
Clonal evolution rate
Proportion of patients progressed to MDS/ 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.

Jun 2026Dec 2029

First posted15 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 done.

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: 0 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT07532408
Sponsor's protocol ID: LP-N01-CR
Status last verified: Jun 2026
First submitted: 8 Apr 2026
Last updated: 4 Jun 2026
Results posted: No
Data monitoring committee: No
FDA-regulated drug: No
FDA-regulated device: No
Individual data sharing: No

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

Official record

RecruitingPhase 1/2
The Safety and Efficacy of Daratumumab in Patients With Refractory Aplastic Anemia
Aplastic Anemia, Refractory
RecruitingPhase 1/2
Pacritinib, a Kinase Inhibitor of CSF1R, IRAK1, JAK2, and FLT3, in Adults and Pediatric Participants 12 Years of Age or Older With Myelodysplastic Syndromes or Myelodysplastic/Myeloproliferative Neoplasms
Myelodysplastic Syndromes
RecruitingObservational study
Defining the Natural History of Squamous Cell Carcinoma in Fanconi Anemia
Fanconi Anemia, Inherited Bone Marrow Failure Syndrome
Not yet recruitingObservational study
Clinical Genetics Branch Eligibility Screening Survey
Melanoma, Li-Fraumeni Syndrome
RecruitingObservational study
HPC Offered for PRESERVE Expansion
Bone Marrow Transplant (BMT)

All Aplastic Anemia trials

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: Romiplostim N01 in the Treatment of Refractory Chemoradiotherapy-induced Aplastic Anemia: a Single-center, Prospective, Open-label Study
ClinicalTrials.gov ID: NCT07532408
Acronym / sponsor ID: LP-N01-CR
Conditions: Aplastic Anemia
Phase: Phase 2
Study type: Interventional
Recruitment status: WithdrawnReason stopped: The registration of this research plan was incorrect. A new registration has been done.
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: 15 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 romiplostim N01 in the treatment of relapsed/refractory chemoradiotherapy-induced aplastic anemia

Detailed description

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 subcutaneously once a week for at least 3 months

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 aplastic anemia (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 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;
PNH clone ≥ 50%;
Received HSCT 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
Time frame: 3-month, 6-month
Overall response rate (ORR) = PRR+CRR

4 secondary outcomes

CRR
Time frame: 3-month, 6-month, 12-month
AE rate
Time frame: through study completion, an average of 1 year
Predictive factors of efficacy
Time frame: 3-month
Clonal evolution 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

Related trials for Aplastic Anemia

RecruitingPhase 1/2
The Safety and Efficacy of Daratumumab in Patients With Refractory Aplastic Anemia
Aplastic Anemia, Refractory
RecruitingPhase 1/2
Pacritinib, a Kinase Inhibitor of CSF1R, IRAK1, JAK2, and FLT3, in Adults and Pediatric Participants 12 Years of Age or Older With Myelodysplastic Syndromes or Myelodysplastic/Myeloproliferative Neoplasms
Myelodysplastic Syndromes
RecruitingObservational study
Defining the Natural History of Squamous Cell Carcinoma in Fanconi Anemia
Fanconi Anemia, Inherited Bone Marrow Failure Syndrome
Not yet recruitingObservational study
Clinical Genetics Branch Eligibility Screening Survey
Melanoma, Li-Fraumeni Syndrome
RecruitingObservational study
HPC Offered for PRESERVE Expansion
Bone Marrow Transplant (BMT)

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 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: 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?

ORR (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?

On the ClinicalTrials.gov record for NCT07532408.

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.