Aplastic AnemiaPhase 2/3NCT03957694
Study of AMG531(Romiplostim) in Patients With Aplastic Anemia
A Phase 2/3 study testing Romiplostim in people with Aplastic Anemia, run by Kyowa Kirin Co., Ltd. at 1 site in Japan.
Trial summary
To evaluate the hematological responses based on the response assessment criteria when AMG531 is subcutaneous (SC)-administered with anti-human thymocyte immunoglobulin (ATG) + ciclosporin A (CsA) therapy for 6 months in patients with aplastic anemia (AA) who were previously untreated with immunosuppressive therapy. 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
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 17 participants
- Where
- 1 siteJapan
- Study dates
- 25 Apr 2019to 26 May 2021
- 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
- Voluntary signed informed consent to participate in the study;
- A diagnosis of AA confirmed by blood and bone-marrow examinations, etc.;
- Considered to require new treatment with ATG and CsA provided that NSAA must be platelet or erythrocyte transfusion-dependent.
- An Eastern Cooperative Oncology Group (ECOG) performance status (PS) score of 0 to 1at screening
Who cannot join
- Previously treated with ATG, CsA, or Alemtuzumab;
- Diagnosed as having congenital AA (Fanconi anemia, congenital dyskeratosis, etc.);
- Diagnosed as having AML or chronic myelomonocytic leukemia;
- Concurrent thrombocytopenia of other etiologies (e.g., MDS, ITP, cirrhosis);
- Concurrent active infection not adequately responding to appropriate therapy;
- Concurrent clinically significant illness(es) items which are deemed by the Investigator to be likely to affect the study conduct and assessments.
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- Having active malignancies, or having a history of treatment of malignancies within 5 years prior to informed consent.
- Concurrent PNH
- Having Grade 2 or higher bone marrow reticulin based on Bone Marrow Pathology (2nd edition) ;
- History of chromosome aberrations discovered in bone marrow cells.
- Having blast cells > 2% in bone marrow;
- Positive for anti-human immunodeficiency virus (HIV) antibody;
- Receiving prophylactic or therapeutic treatment for hepatitis type B
- Positive for hepatitis C virus (HCV) antibody, and HCV infection being confirmed
- Planned hematopoietic stem cell transplantation during the study;
- Systemic treatment with any of the following medication for the treatment of AA within 4 weeks before Day 1, however, excluding their use as premedication:
- Anabolic steroids
- Corticosteroids;
- Pregnant or breastfeeding women, or women willing to become pregnant;
- Other conditions unsuitable for participation in the study in the opinion of the Investigator.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Phase 2/3 Study of AMG531 in Patients With Aplastic Anemia PreviouslyUntreated With Immunosuppressive Therapy
To evaluate the hematological responses based on the response assessment criteria when AMG531 is subcutaneous (SC)-administered with anti-human thymocyte immunoglobulin (ATG) + ciclosporin A (CsA) therapy for 6 months in patients with aplastic anemia (AA) who were previously untreated with immunosuppressive therapy.
- Conditions
- Aplastic Anemia
- Being tested
- Romiplostim
- Keywords
- aplastic anemia, romiplostim
- Medical subject headings
- Anemia, Aplastic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RomiplostimDrug
Romiplostim SC. Initial dose is 10 ug/kg/. Maximum dose is 20 ug/kg. Original duration is 6 months from first administration but if investigator decide that the patient is needed more treatment, they move to extention period and take treatment up to 1 year.
| Study group | Role | What they receive |
|---|---|---|
| AMG531 | Experimental | Not described Receives: Drug: Romiplostim |
What is being measured
The primary outcome is the main question the study is designed to answer.
Achievement of complete response (CR) or partial response (PR)
Measured: 27 weeks post-dose
Secondary outcomes (8)
Achievement of CR or PR
Measured: Week 14
Achievement of CR
Measured: Weeks 14 and 27
The time to CR or PR
Measured: Each time point evaluated weekly until Week 27
Reduction or independence of platelet and/or erythrocyte transfusion
Measured: Week 27
Change from baseline in platelet count (/µL)
Measured: Each time point evaluated weekly until Week 27
Change from baseline in hemoglobin (Hb) concentration (g/dL)
Measured: Each time point evaluated weekly until Week 27
Change from baseline in neutrophil count (/µL)
Measured: Each time point evaluated weekly until Week 27
Change from baseline in reticulocyte count (/µL)
Measured: Each time point evaluated weekly until Week 27
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 May 2019
- Study start25 Apr 2019
- Primary completion5 Nov 2020
- Study completion26 May 2021
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 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
- Model description
- multi-national, open-label, phase 2/3 study
- Masking
- None (open label)
- Enrolment
- 17 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
- NCT03957694
- Sponsor's protocol ID
- 531-003
- Status last verified
- May 2022
- First submitted
- 9 May 2019
- Last updated
- 26 May 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 26 May 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2/3 Study of AMG531 in Patients With Aplastic Anemia PreviouslyUntreated With Immunosuppressive Therapy
- ClinicalTrials.gov ID
- NCT03957694
- Acronym / sponsor ID
- 531-003
- Conditions
- Aplastic Anemia
- Keywords
- aplastic anemia, romiplostim
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 17 participants (actual)
- Lead sponsor
- Kyowa Kirin Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 25 Apr 2019 (actual)
- Primary completion
- 5 Nov 2020 (actual)
- Study completion
- 26 May 2021 (actual)
- First posted
- 21 May 2019 (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.
To evaluate the hematological responses based on the response assessment criteria when AMG531 is subcutaneous (SC)-administered with anti-human thymocyte immunoglobulin (ATG) + ciclosporin A (CsA) therapy for 6 months in patients with aplastic anemia (AA) who were previously untreated with immunosuppressive therapy.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- multi-national, open-label, phase 2/3 study
| Arm | Type | Receives |
|---|---|---|
| AMG531 | Experimental | Drug: Romiplostim |
Who may qualify
- Ages
- 20 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Voluntary signed informed consent to participate in the study;
- A diagnosis of AA confirmed by blood and bone-marrow examinations, etc.;
- Considered to require new treatment with ATG and CsA provided that NSAA must be platelet or erythrocyte transfusion-dependent.
- An Eastern Cooperative Oncology Group (ECOG) performance status (PS) score of 0 to 1at screening
Key exclusion criteria (20 listed)
- Previously treated with ATG, CsA, or Alemtuzumab;
- Diagnosed as having congenital AA (Fanconi anemia, congenital dyskeratosis, etc.);
- Diagnosed as having AML or chronic myelomonocytic leukemia;
- Concurrent thrombocytopenia of other etiologies (e.g., MDS, ITP, cirrhosis);
- Concurrent active infection not adequately responding to appropriate therapy;
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 |
|---|---|---|
| RomiplostimRomiplostim SC. Initial dose is 10 ug/kg/. Maximum dose is 20 ug/kg. Original duration is 6 months from first administration but if investigator decide that the patient is needed more treatment, they move to extention period and take treatment up to 1 year. | Drug | AMG531 |
Primary outcome
Achievement of complete response (CR) or partial response (PR)
Time frame: 27 weeks post-dose
8 secondary outcomes
Achievement of CR or PR
Time frame: Week 14
Achievement of CR
Time frame: Weeks 14 and 27
The time to CR or PR
Time frame: Each time point evaluated weekly until Week 27
Reduction or independence of platelet and/or erythrocyte transfusion
Time frame: Week 27
Change from baseline in platelet count (/µL)
Time frame: Each time point evaluated weekly until Week 27
Change from baseline in hemoglobin (Hb) concentration (g/dL)
Time frame: Each time point evaluated weekly until Week 27
Change from baseline in neutrophil count (/µL)
Time frame: Each time point evaluated weekly until Week 27
Change from baseline in reticulocyte count (/µL)
Time frame: Each time point evaluated weekly until Week 27
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 26 May 2022.
- Individual participant dataSharing planned: Undecided
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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 2/3 study testing Romiplostim in people with Aplastic Anemia, run by Kyowa Kirin Co., Ltd. at 1 site in Japan.
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 4 inclusion and 20 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 26 May 2022. 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 Japan. 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?
Achievement of complete response (CR) or partial response (PR) (time frame: 27 weeks post-dose). 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 26 May 2022.