- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT02773290
Study of Romiplostim(AMG531) in Subjects With Aplastic Anemia
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.
Study Overview
Study Type
Enrollment (Actual)
Phase
- Phase 2
- Phase 3
Contacts and Locations
Study Locations
-
-
-
Kanazawa, Japan
-
Tokyo, Japan
-
-
-
-
-
Seoul, Korea, Republic of
-
-
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
Inclusion Criteria:
- 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
- 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
Exclusion Criteria:
- 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
- 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
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: Romiplostim
Weekly Subcutaneous (SC) administration
|
Weekly SC administration
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
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
|
At 27 weeks after dosing
|
Secondary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
Proportion of subjects with a hematological response at the end-of- treatment examination
Time Frame: Up to 52 weeks after dosing
|
Up to 52 weeks after dosing
|
Time from the first romiplostim administration to hematological response
Time Frame: Up to 52 weeks after dosing
|
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
|
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
|
At 27 weeks and 52 weeks after dosing
|
Collaborators and Investigators
Sponsor
Study record dates
Study Major Dates
Study Start
Primary Completion (Actual)
Study Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimate)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- 531-002
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Aplastic Anemia
-
Assistance Publique - Hôpitaux de ParisNot yet recruitingSevere Aplastic Anemia | Idiopathic Aplastic Anemia | Moderate Aplastic Anemia Requiring Transfusions
-
University of UtahNovartisCompletedSevere Aplastic Anemia | Moderate Aplastic Anemia | Very Severe Aplastic AnemiaUnited States
-
City of Hope Medical CenterNational Cancer Institute (NCI)RecruitingRecurrent Severe Aplastic Anemia | Refractory Severe Aplastic AnemiaUnited States
-
Peking University People's HospitalRecruiting
-
Boston Children's HospitalNational Heart, Lung, and Blood Institute (NHLBI); National Institutes of Health... and other collaboratorsRecruitingSevere Aplastic AnemiaUnited States
-
Federal Research Institute of Pediatric Hematology...RecruitingAcquired Aplastic AnemiaRussian Federation
-
Shanghai General Hospital, Shanghai Jiao Tong University...Ruijin Hospital; Xinhua Hospital, Shanghai Jiao Tong University School of Medicine and other collaboratorsCompleted
-
Nagoya UniversityUnknownAcquired Aplastic Anemia.Japan
-
Navy General Hospital, BeijingPeking Union Medical College Hospital; Cancer Institute and Hospital, Chinese... and other collaboratorsUnknownSevere Aplastic AnemiaChina
-
Jiangsu HengRui Medicine Co., Ltd.Recruiting
Clinical Trials on Romiplostim
-
Assistance Publique - Hôpitaux de ParisCompletedPersistent Thrombocytopenia Following Allogeneic Hematopoietic Stem Cell Transplantation (HSCT)France
-
Memorial Sloan Kettering Cancer CenterTel-Aviv Sourasky Medical Center; AmgenActive, not recruitingThrombocytopenia | Lymphoma PatientsUnited States
-
AmgenCompletedMyelodysplastic Syndromes | Thrombocytopenia | MDS
-
AmgenCompletedImmune ThrombocytopeniaUnited States, Canada, Belgium, Czechia, Mexico, Turkey, Australia, Spain, France, Israel, Hungary, South Africa, Poland, Russian Federation, United Kingdom, Brazil, Switzerland
-
Federal Research Institute of Pediatric Hematology...CompletedWiskott-Aldrich SyndromeRussian Federation
-
Kyowa Hakko Kirin China Pharmaceutical Co., LTD.CompletedImmune ThrombocytopeniaChina
-
Samsung Medical CenterUnknown
-
Kyowa Hakko Kirin China Pharmaceutical Co., LTD.CompletedImmune Thrombocytopenia (ITP)China
-
Memorial Sloan Kettering Cancer CenterAmgenCompletedMultiple Myeloma | Hodgkin Lymphoma | Non-Hodgkin Lymphoma | HDT-AHCTUnited States
-
Memorial Sloan Kettering Cancer CenterTerminatedSolid Tumor | Solid Carcinoma | Solid Tumor, ChildhoodUnited States