Aplastic AnemiaPhase 2NCT02094417
A Phase 2 Study to Evaluate the Efficacy and Safety of AMG531 in Aplastic Anemia
A Phase 2 study testing AMG531 in people with Aplastic Anemia, run by Kyowa Kirin Korea Co., Ltd. at 1 site in South Korea.
Trial summary
The present study will be conducted to evaluate the efficacy and safety of AMG531 and to determine the recommended initial dose of AMG531 on the basis of its efficacy and safety when it is administered subcutaneously (SC) to the Aplastic Anemia (AA) patients with immunosuppressive-therapy refractory thrombocytopenia… Read more about this study
- Who is it for?
- Ages 19 years and older
- Conditions the study is for
- Aplastic Anemia
- What is being tested?
- AMG531
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 35 participants
- Where
- 1 siteSouth Korea
- Study dates
- 14 Apr 2014to 7 Nov 2017
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 19 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patient who is diagnosed as AA and refractory to immunosuppressive therapy
- Platelet ≤ 30,000/μL
Who cannot join
- Concurrent active infection not adequately responding to appropriate therapy
- HIV positivity
- Bone marrow reticulin grade of > 1
- Clinically significant cardiac disease
- Arterial or venous thrombosis within the last 1 year before enrollment
- Other cause of thrombocytopenia
Show all 11Show fewer
- AA with hemolytic predominant paroxysmal nocturnal hemoglobinuria (PNH)
- Uncontrolled diabetes
- Receiving any agent used to treat AA, including antithymocyte globulin (ATG) or ATG + cyclosporine within 6 months before starting study treatment and/or cyclosporine or anabolic hormone within 6 weeks before starting the study treatment
- History of PEG-rHuMGDF, recombinant human thrombopoietin, AMG531, and other thrombopoietin (TPO)-receptor agonist
- Who plans to conduct hematopoietic stem cell transplantation within 1 year
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Phase 2, Randomized, Open-Label, Parallel, Comparative, Dose-Finding Study to Evaluate the Efficacy and Safety of AMG531 in Aplastic Anemia Subjects With Thrombocytopenia Refractory to Immunosuppressive Therapy
The present study will be conducted to evaluate the efficacy and safety of AMG531 and to determine the recommended initial dose of AMG531 on the basis of its efficacy and safety when it is administered subcutaneously (SC) to the Aplastic Anemia (AA) patients with immunosuppressive-therapy refractory thrombocytopenia and also to assess the pharmacokinetics of this product. Its efficacy and safety during the extension period beyond one year will also be evaluated.
- Conditions
- Aplastic Anemia
- Being tested
- AMG531
- Keywords
- Anemia, Aplastic, Thrombopoietin, Thrombocytopenia
- Medical subject headings
- Anemia, Aplastic, Jacobs syndrome, Thrombocytopenia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AMG531Drug
Subcutaneous, weekly injection
| Study group | Role | What they receive |
|---|---|---|
| AMG531 (Dose 1) | Experimental | Not described Receives: Drug: AMG531 |
| AMG531 (Dose 2) | Experimental | Not described Receives: Drug: AMG531 |
| AMG531 (Dose 3) | Experimental | Not described Receives: Drug: AMG531 |
| AMG531 (Dose 4) | Experimental | Not described Receives: Drug: AMG531 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Proportion of Subjects Achieving a Platelet Response at Week 9
The proportion of subjects achieving a platelet response at Week 9, and two-sided 95% confidence interval will be calculated. Platelet response is defined as 1) achieving absolute platelet increase of ≥ 20x10^9/L above baseline or 2) increase to ≥ 10x10^9/L and by at least 100% from baseline.
Measured: At week 9
Secondary outcomes (6)
Proportion of Subjects Achieving a Platelet Response
The proportion of subjects with a platelet response in any time during the initial dose evaluation period, Week 1 through Week 12, Week 1 through Week 16, Week 1 through Week 24, Week 1 through Week 52, Week 1 through Week 104 and Week 1 through Week 156.
Measured: Initial dose evaluation period (Week 9), Week 12, Week 16, Week 24, Week 52, Week 104, and Week 156
Proportion of Subjects Achieving Platelet Transfusion Independency
The proportion of subjects achieving platelet transfusion independency in any time during the initial dose evaluation period , Week 1 through Week 12, Week 1 through Week 16, Week 1 through Week 24, Week 1 through Week 52, Week 1 through Week 104, and Week1 through Week 156 will be calculated in the same manner as the primary variables (The number of subjects discontinued or subjects who have missing data will not be calculated). Platelet transfusion independence is defined as achieving transfusion free period of at least 8 consecutive weeks (56 days).
Measured: Initial dose evaluation period (Week 9), Week 12, Week 16, Week 24, Week 52, Week 104, and Week 156
Proportion of Subjects Achieving Erythroid Response and/or Neutrophil Response
The proportion of subjects with erythroid response, neutrophil response and erythroid and/or neutrophil response in any time during the initial dose evaluation period, Week 1 through Week 12, Week 1 through Week 16, Week 1 through Week 24, Week 1 through Week 52, Week 1 through Week 104, and Week1 through Week 156 will be calculated in the same manner as the primary variables (The number of subjects discontinued or subjects who have missing data will not be calculated).
Measured: Initial dose evaluation period (Week 9), Week 12, Week 16, Week 24, Week 52, Week 104, and Week 156
Duration of Platelet Response and Time to Platelet Response
For subjects with a platelet response in any time, time to the initial platelet response and duration of platelet response will be summarized Time to the initial platelet response will be calculated as sampling date (response achieved) minus first dose date of study drug plus 1.
Duration of platelet response will be calculated as the maximum of the duration of platelet response for each subject, each duration of response calculated as the date when response is disappeared minus the date when response is achieved. The censoring date is defined as the date of the last Platelet examination or EOS whichever archives first.
Measured: Initial dose evaluation period (Week 9), Week 12, Week 16, Week 24, Week 52, Week 104, and Week 156
Proportion of Subjects Achieving Tri-lineage Responses
Tri-lineage response is defined in those achieving platelet response, erythroid response, and neutrophil response all together. The proportion of subjects with tri-lineage response will be calculated in the same manner as the primary variables. Time to tri-lineage response will be summarized in the same manner as time to platelet response. Time to tri-lineage response is defined as duration of the time from Day 1 to the date of platelet response, erythroid response or neutrophil response whichever achieves last in Visit.
Measured: Initial dose evaluation period (Week 9), Week 12, Week 16, Week 24, Week 52, Week 104, and Week 157
Duration of Study Drug Discontinuation While Maintaining Stable Response
The longest duration of study drug discontinuation in subjects while maintaining a stable response is summarized
Measured: Initial dose evaluation period (Week 9), Week 12, Week 16, Week 24, Week 52, Week 104, and Week 156
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Mar 2014 (estimated)
- Study start14 Apr 2014
- Primary completion24 Apr 2017
- Study completion7 Nov 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Aplastic AnemiaStudies of the same condition, open and under way.
- 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
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 35 participants (actual)
Sponsor and investigators
- Lead sponsor
- Kyowa Kirin Korea Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02094417
- Sponsor's protocol ID
- 531-KR001
- Status last verified
- Apr 2024
- First submitted
- 18 Mar 2014
- Last updated
- 30 Apr 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- 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 40593138Lee JW, Lee SE, Jung CW, Park S, Keta H, Park SK, Kim JA, Oh IH, Jang JH. Romiplostim in patients with refractory aplastic anaemia previously treated with immunosuppressive therapy: a dose-finding and long-term treatment phase 2 trial. Lancet Haematol. 2019 Nov;6(11):e562-e572. doi: 10.1016/S2352-3026(19)30153-X. Epub 2019 Aug 29.
PubMed 31474546
Study at a glance
From the registry record, last updated by the sponsor on 30 Apr 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2, Randomized, Open-Label, Parallel, Comparative, Dose-Finding Study to Evaluate the Efficacy and Safety of AMG531 in Aplastic Anemia Subjects With Thrombocytopenia Refractory to Immunosuppressive Therapy
- ClinicalTrials.gov ID
- NCT02094417
- Acronym / sponsor ID
- 531-KR001
- Conditions
- Aplastic Anemia
- Keywords
- Anemia, Aplastic, Thrombopoietin, Thrombocytopenia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 35 participants (actual)
- Lead sponsor
- Kyowa Kirin Korea Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 14 Apr 2014 (actual)
- Primary completion
- 24 Apr 2017 (actual)
- Study completion
- 7 Nov 2017 (actual)
- First posted
- 21 Mar 2014 (estimated)
- Results
- Posted 15 Feb 2021
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The present study will be conducted to evaluate the efficacy and safety of AMG531 and to determine the recommended initial dose of AMG531 on the basis of its efficacy and safety when it is administered subcutaneously (SC) to the Aplastic Anemia (AA) patients with immunosuppressive-therapy refractory thrombocytopenia and also to assess the pharmacokinetics of this product. Its efficacy and safety during the extension period beyond one year will also be evaluated.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| AMG531 (Dose 1) | Experimental | Drug: AMG531 |
| AMG531 (Dose 2) | Experimental | Drug: AMG531 |
| AMG531 (Dose 3) | Experimental | Drug: AMG531 |
| AMG531 (Dose 4) | Experimental | Drug: AMG531 |
Who may qualify
- Ages
- 19 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- Patient who is diagnosed as AA and refractory to immunosuppressive therapy
- Platelet ≤ 30,000/μL
Key exclusion criteria (11 listed)
- Concurrent active infection not adequately responding to appropriate therapy
- HIV positivity
- Bone marrow reticulin grade of > 1
- Clinically significant cardiac disease
- Arterial or venous thrombosis within the last 1 year 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 |
|---|---|---|
| AMG531Subcutaneous, weekly injection | Drug | AMG531 (Dose 1), AMG531 (Dose 2), AMG531 (Dose 3), AMG531 (Dose 4) |
Primary outcome
Proportion of Subjects Achieving a Platelet Response at Week 9
Time frame: At week 9
The proportion of subjects achieving a platelet response at Week 9, and two-sided 95% confidence interval will be calculated. Platelet response is defined as 1) achieving absolute platelet increase of ≥ 20x10^9/L above baseline or 2) increase to ≥ 10x10^9/L and by at least 100% from baseline.
6 secondary outcomes
Proportion of Subjects Achieving a Platelet Response
Time frame: Initial dose evaluation period (Week 9), Week 12, Week 16, Week 24, Week 52, Week 104, and Week 156
Proportion of Subjects Achieving Platelet Transfusion Independency
Time frame: Initial dose evaluation period (Week 9), Week 12, Week 16, Week 24, Week 52, Week 104, and Week 156
Proportion of Subjects Achieving Erythroid Response and/or Neutrophil Response
Time frame: Initial dose evaluation period (Week 9), Week 12, Week 16, Week 24, Week 52, Week 104, and Week 156
Duration of Platelet Response and Time to Platelet Response
Time frame: Initial dose evaluation period (Week 9), Week 12, Week 16, Week 24, Week 52, Week 104, and Week 156
Proportion of Subjects Achieving Tri-lineage Responses
Time frame: Initial dose evaluation period (Week 9), Week 12, Week 16, Week 24, Week 52, Week 104, and Week 157
Duration of Study Drug Discontinuation While Maintaining Stable Response
Time frame: Initial dose evaluation period (Week 9), Week 12, Week 16, Week 24, Week 52, Week 104, and Week 156
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Apr 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- 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.
- Derived from this study: PubMed 31474546Lee JW, Lee SE, Jung CW, Park S, Keta H, Park SK, Kim JA, Oh IH, Jang JH. Romiplostim in patients with refractory aplastic anaemia previously treated with immunosuppressive therapy: a dose-finding and long-term treatment phase 2 trial. Lancet Haematol. 2019 Nov;6(11):e562-e572. doi: 10.1016/S2352-3026(19)30153-X. Epub 2019 Aug 29.
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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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing AMG531 in people with Aplastic Anemia, run by Kyowa Kirin Korea Co., Ltd. at 1 site in South Korea.
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 2 inclusion and 11 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 30 Apr 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in 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 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?
Yes. Results are posted in the registry (first posted 15 Feb 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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 Platelet Response at Week 9 (time frame: At week 9). 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 NCT02094417, which also lists 2 linked publications.
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 30 Apr 2024; AzTrials last refreshed its copy on 29 Oct 2026.