Aplastic AnemiaPhase 2/3NCT02773290
Study of Romiplostim(AMG531) in Subjects With Aplastic Anemia
A Phase 2/3 study testing Romiplostim in people with Aplastic Anemia, run by Kyowa Kirin Co., Ltd. at 3 sites in 2 countries.
Trial summary
The objective of this study is to evaluate the efficacy of romiplostim administered once weekly to Aplastic Anemia (AA) patients with thrombocytopenia refractory to or ineligible for immunosuppressive therapy in Japan and Korea. Read more about this study
- Who is it for?
- Ages 20 years and older
- Conditions the study is for
- Aplastic Anemia
- What is being tested?
- Romiplostim
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 46 participants
- Where
- 3 sitesin 2 countries
- Study dates
- May 2016to 28 Jul 2020
- Phase
- Phase 2/3
- 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 20 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Diagnosis of Aplastic Anemia (AA) confirmed by peripheral blood and bone-marrow examinations, etc.
- Refractory to at least one course of immunosuppressive therapy including horse or rabbit anti-human thymocyte immunoglobulin (ATG); or ineligible for ATG treatment and refractory to cyclosporin (CyA)
- Thrombocytopenia defined as a platelet count of ≤ 30 × 10^9/L
- Preserving main organ function as a result of screening as follows;
- Total bilirubin: < 1.5 times the upper limit of the laboratory normal range
- Alanine aminotransferase: < 3.0 times the upper limit of the laboratory normal range
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- Aspartate aminotransferase: < 3.0 times the upper limit of the laboratory normal range
- Creatinine value: ≤ 2.0 mg/dL
- An Eastern Cooperative Oncology Group performance status score of 0 to 2 at screening
- ≥ 20 years of age at the time of obtaining informed consent
- Patients who have provided written informed consent of their free will to participate in this study
Who cannot join
- Concurrent active infection not adequately responding to appropriate therapy
- Bone marrow reticulin grade of ≥ 2 based on the grading scale for reticulin indicated in Bone Marrow Pathology (2nd edition)
- Proportion of blasts in bone marrow > 2%
- Previous or concurrent active malignancies, other than localized tumors diagnosed more than one year previously and treated surgically with curative intent (basal cell carcinoma; or surgically resected in situ carcinoma of the cervix with an apparent success of ≥ 12 months prior to enrollment; as well as other cancers which have not been treated and remained disease-free for at least 5 years before enrollment are eligible)
- Clinically significant cardiac disease (class III or IV of the New York Heart Association classification; unstable angina pectoris; myocardial infarction within 6 months before enrollment; cardiac disease accompanied by angioplasty or stenting within 6 months before enrollment; or clinically significant cardiac arrhythmias) or uncontrollable hypertension
- Arterial or venous thrombosis within one year before enrollment
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- Positive for anti-human immunodeficiency virus antibodies, hepatitis B surface antigen, or hepatitis C virus-RNA at screening
- Thrombocytopenia due to any other cause (e.g., myelodysplastic syndrome, idiopathic thrombocytopenic purpura, or liver cirrhosis)
- Patients with acute myeloblastic leukemia or chronic myelomonocytic leukemia
- Concurrent occurrence of hemolytic predominant paroxysmal nocturnal hemoglobinuria (Hemolytic predominant is defined as lactate dehydrogenase > 1.5 times the upper limit of the laboratory normal range)
- Uncontrolled diabetes mellitus
- Receiving other investigational products within 16 weeks before romiplostim treatment initiation
- Receiving any agent to treat AA, including the following agents before romiplostim treatment initiation;
- ATG treatment within 6 months before romiplostim treatment initiation
- CyA or anabolic steroid treatment within 6 weeks before romiplostim treatment initiation:
- However, the patients who are treated with a CyA or anabolic steroid for at least 6 months before romiplostim treatment initiation may be enrolled if their blood cell count are stable at screening, and their dosage and administration will be kept for 6 weeks before romiplostim treatment initiation and during romiplostim dosing period.
- A history of polyethylene glycol-conjugated recombinant human megakaryocyte growth and development factor, recombinant human thrombopoietin (TPO), romiplostim, or other TPO-receptor agonists
- Having a plan to undergo hematopoietic stem cell transplantation within 1 year
- Having hypersensitivity to any recombinant protein E. coli derivative protein
- Lactating or pregnant women or women of child-bearing potential who have no intention of using oral contraceptives or birth control
- Having abnormalities by the cytogenetic test in bone marrow cells
- Patients who are considered to be ineligible for the study by the investigator or subinvestigator for reasons other than above
Trial locations and contacts
3 sites in 2 countries.
3 sites in 2 countries
About this study
Official title: Phase 2/3 Study of Romiplostim(AMG531) in Subjects With Aplastic Anemia Refractory to or Ineligible for Immunosuppressive Therapy
The objective of this study is to evaluate the efficacy of romiplostim administered once weekly to Aplastic Anemia (AA) patients with thrombocytopenia refractory to or ineligible for immunosuppressive therapy in Japan and Korea.
Safety and pharmacokinetics of romiplostim after repeated administration will also be assessed.
- Conditions
- Aplastic Anemia
- Being tested
- Romiplostim
- Keywords
- Aplastic Anemia
- Medical subject headings
- Anemia, Aplastic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RomiplostimBiological
Weekly SC administration
| Study group | Role | What they receive |
|---|---|---|
| Romiplostim | Experimental | Weekly Subcutaneous (SC) administration Receives: Biological: Romiplostim |
What is being measured
The primary outcome is the main question the study is designed to answer.
Proportion of subjects achieving a hematological response (any of the platelet response, erythroid response, and neutrophil response) at Week 27
Measured: At 27 weeks after dosing
Secondary outcomes (4)
Proportion of subjects with a hematological response at the end-of- treatment examination
Measured: Up to 52 weeks after dosing
Time from the first romiplostim administration to hematological response
Measured: Up to 52 weeks after dosing
In subjects receiving platelet transfusion as a pretreatment within 8 weeks prior to the first romiplostim administration; proportion of subjects with transfusion independence or decreased platelet transfusion requirement
Measured: Up to 52 weeks after dosing
Proportion of subjects achieving platelet response, erythroid response, or neutrophil response at each of Week 27 and end of treatment.
Measured: At 27 weeks and 52 weeks after dosing
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 May 2016 (estimated)
- Study startMay 2016
- Primary completion14 Nov 2017
- Study completion28 Jul 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Aplastic AnemiaStudies of the same condition, open and under way.
- More trials run by Kyowa Kirin Co., Ltd.The 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 2/3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 46 participants (actual)
Sponsor and investigators
- Lead sponsor
- Kyowa Kirin Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02773290
- Sponsor's protocol ID
- 531-002
- Status last verified
- Jul 2024
- First submitted
- 9 May 2016
- Last updated
- 16 Jul 2024
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- No
Publications
Jang JH, Mitani K, Tomiyama Y, Miyazaki K, Nagafuji K, Usuki K, Uoshima N, Fujisaki T, Kosugi H, Matsumura I, Sasaki K, Kizaki M, Sawa M, Hidaka M, Kobayashi N, Ichikawa S, Yonemura Y, Murotani K, Shimizu M, Matsuda A, Ozawa K, Nakao S, Lee JW. Predictive factors of romiplostim response in patients with refractory aplastic anemia: data from two clinical trials. Ann Hematol. 2025 Aug;104(8):4003-4011. doi: 10.1007/s00277-025-06337-7. Epub 2025 Jul 1.
PubMed 40593138
Study at a glance
From the registry record, last updated by the sponsor on 16 Jul 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 2/3 Study of Romiplostim(AMG531) in Subjects With Aplastic Anemia Refractory to or Ineligible for Immunosuppressive Therapy
- ClinicalTrials.gov ID
- NCT02773290
- Acronym / sponsor ID
- 531-002
- Conditions
- Aplastic Anemia
- Keywords
- Aplastic Anemia
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 46 participants (actual)
- Lead sponsor
- Kyowa Kirin Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- May 2016
- Primary completion
- 14 Nov 2017 (actual)
- Study completion
- 28 Jul 2020 (actual)
- First posted
- 16 May 2016 (estimated)
- 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 objective of this study is to evaluate the efficacy of romiplostim administered once weekly to Aplastic Anemia (AA) patients with thrombocytopenia refractory to or ineligible for immunosuppressive therapy in Japan and Korea.
Safety and pharmacokinetics of romiplostim after repeated administration will also be assessed.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Romiplostim | Experimental | Biological: RomiplostimWeekly Subcutaneous (SC) administration |
Who may qualify
- Ages
- 20 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (11 listed)
- Diagnosis of Aplastic Anemia (AA) confirmed by peripheral blood and bone-marrow examinations, etc.
- Refractory to at least one course of immunosuppressive therapy including horse or rabbit anti-human thymocyte immunoglobulin (ATG); or ineligible for ATG treatment and refractory to cyclosporin (CyA)
- Thrombocytopenia defined as a platelet count of ≤ 30 × 10^9/L
- Preserving main organ function as a result of screening as follows;
- An Eastern Cooperative Oncology Group performance status score of 0 to 2 at screening
Key exclusion criteria (22 listed)
- Concurrent active infection not adequately responding to appropriate therapy
- Bone marrow reticulin grade of ≥ 2 based on the grading scale for reticulin indicated in Bone Marrow Pathology (2nd edition)
- Proportion of blasts in bone marrow > 2%
- Previous or concurrent active malignancies, other than localized tumors diagnosed more than one year previously and treated surgically with curative intent (basal cell carcinoma; or surgically resected in situ carcinoma of the cervix with an apparent success of ≥ 12 months prior to enrollment; as well as other cancers which have not been treated and remained disease-free for at least 5 years before enrollment are eligible)
- Clinically significant cardiac disease (class III or IV of the New York Heart Association classification; unstable angina pectoris; myocardial infarction within 6 months before enrollment; cardiac disease accompanied by angioplasty or stenting within 6 months before enrollment; or clinically significant cardiac arrhythmias) or uncontrollable hypertension
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 |
|---|---|---|
| RomiplostimWeekly SC administration | Biological | Romiplostim |
Primary outcome
Proportion of subjects achieving a hematological response (any of the platelet response, erythroid response, and neutrophil response) at Week 27
Time frame: At 27 weeks after dosing
4 secondary outcomes
Proportion of subjects with a hematological response at the end-of- treatment examination
Time frame: Up to 52 weeks after dosing
Time from the first romiplostim administration to hematological response
Time frame: Up to 52 weeks after dosing
In subjects receiving platelet transfusion as a pretreatment within 8 weeks prior to the first romiplostim administration; proportion of subjects with transfusion independence or decreased platelet transfusion requirement
Time frame: Up to 52 weeks after dosing
Proportion of subjects achieving platelet response, erythroid response, or neutrophil response at each of Week 27 and end of treatment.
Time frame: At 27 weeks and 52 weeks after dosing
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Jul 2024.
- Derived from this study: PubMed 40593138Jang JH, Mitani K, Tomiyama Y, Miyazaki K, Nagafuji K, Usuki K, Uoshima N, Fujisaki T, Kosugi H, Matsumura I, Sasaki K, Kizaki M, Sawa M, Hidaka M, Kobayashi N, Ichikawa S, Yonemura Y, Murotani K, Shimizu M, Matsuda A, Ozawa K, Nakao S, Lee JW. Predictive factors of romiplostim response in patients with refractory aplastic anemia: data from two clinical trials. Ann Hematol. 2025 Aug;104(8):4003-4011. doi: 10.1007/s00277-025-06337-7. Epub 2025 Jul 1.
- 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.
3 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Romiplostim in people with Aplastic Anemia, run by Kyowa Kirin Co., Ltd. at 3 sites in 2 countries.
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 11 inclusion and 22 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: finished, as of the sponsor's last update on 16 Jul 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in Japan, South Korea. Whether a site is taking part right now should be confirmed with that site. See every site.
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 combined 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.
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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Proportion of subjects achieving a hematological response (any of the platelet response, erythroid response, and neutrophil response) at Week 27 (time frame: At 27 weeks after dosing). 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 NCT02773290, which also lists 1 linked publication.
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 16 Jul 2024.