Back to resultsStudy of AMG531 (Romiplostim) in Patients With Aplastic Anemia
Primary Purpose
Aplastic Anemia
Status
Completed
Phase
Phase 2
Locations
Japan
Study Type
Interventional
Intervention
Romiplostim
Sponsored by
About this trial
This is an interventional treatment trial for Aplastic Anemia focused on measuring aplastic anemia, romiplostim
Eligibility Criteria
Inclusion Criteria:
- 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 immunosuppressive therapy 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;
Exclusion Criteria:
- Previously treated with Anti-human thymocyte immunoglobulin (ATG), CsA, or Alemtuzumab;
- Diagnosed as having congenital AA (Fanconi anemia, congenital dyskeratosis, etc.);
- Diagnosed as having acute myelocytic leukemia (AML) or chronic myelomonocytic leukemia;
- Concurrent thrombocytopenia of other etiologies (e.g., myelodysplastic syndrome (MDS), idiopathic thrombocytopenic purpura (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.
- Having active malignancies, or having a history of treatment of malignancies within 5 years prior to informed consent.
- Concurrent paroxysmal nocturnal hemoglobinuria (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
- Having hepatitis C virus (HCV) infection confirmed by HCV-RNA or other tests at screening.
- 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:
- 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.
Sites / Locations
- NTT Medical center Tokyo
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
AMG531
Arm Description
Outcomes
Primary Outcome Measures
Rate of achievement of complete response (CR) or partial response (PR)
Secondary Outcome Measures
Rate of achievement of CR or PR
Rate of achievement of CR
The time to CR or PR
Reduction or independence of platelet and/or erythrocyte transfusion
Change from baseline in platelet count (/µL)
Change from baseline in hemoglobin (Hb) concentration (g/dL)
Change from baseline in neutrophil count (/µL)
Change from baseline in reticulocyte count (/µL)
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT04095936
Brief Title
Study of AMG531 (Romiplostim) in Patients With Aplastic Anemia
Official Title
A Phase 2/3 Study of AMG531 Combined With Ciclosporin A in Patients With Aplastic Anemia Previously Untreated With Immunosuppressive Therapy
Study Type
Interventional
2. Study Status
Record Verification Date
May 2022
Overall Recruitment Status
Completed
Study Start Date
December 3, 2019 (Actual)
Primary Completion Date
October 7, 2021 (Actual)
Study Completion Date
October 29, 2021 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Kyowa Kirin Co., Ltd.
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
5. Study Description
Brief Summary
To evaluate the hematological responses based on the response assessment criteria defined in this study (the 531-004 response assessment criteria) when AMG531 is subcutaneously (SC)-administered with ciclosporin A (CsA) therapy for 6 months in patients with aplastic anemia (AA) who were previously untreated with immunosuppressive therapy.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Aplastic Anemia
Keywords
aplastic anemia, romiplostim
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2, Phase 3
Interventional Study Model
Single Group Assignment
Model Description
multi-national, open-label, phase 2/3 study
Masking
None (Open Label)
Allocation
N/A
Enrollment
24 (Actual)
8. Arms, Groups, and Interventions
Arm Title
AMG531
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
Romiplostim
Intervention Description
Subcutaneous administration of 0 to 20ug/kg for 6 months
Primary Outcome Measure Information:
Title
Rate of achievement of complete response (CR) or partial response (PR)
Time Frame
27 weeks post-dose
Secondary Outcome Measure Information:
Title
Rate of achievement of CR or PR
Time Frame
14 weeks
Title
Rate of achievement of CR
Time Frame
Weeks 14 and 27
Title
The time to CR or PR
Time Frame
Each time point evaluated weekly until Week 27
Title
Reduction or independence of platelet and/or erythrocyte transfusion
Time Frame
Week 27
Title
Change from baseline in platelet count (/µL)
Time Frame
Each time point evaluated weekly until Week 27
Title
Change from baseline in hemoglobin (Hb) concentration (g/dL)
Time Frame
Each time point evaluated weekly until Week 27
Title
Change from baseline in neutrophil count (/µL)
Time Frame
Each time point evaluated weekly until Week 27
Title
Change from baseline in reticulocyte count (/µL)
Time Frame
Each time point evaluated weekly until Week 27
10. Eligibility
Sex
All
Minimum Age & Unit of Time
20 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
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 immunosuppressive therapy 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;
Exclusion Criteria:
Previously treated with Anti-human thymocyte immunoglobulin (ATG), CsA, or Alemtuzumab;
Diagnosed as having congenital AA (Fanconi anemia, congenital dyskeratosis, etc.);
Diagnosed as having acute myelocytic leukemia (AML) or chronic myelomonocytic leukemia;
Concurrent thrombocytopenia of other etiologies (e.g., myelodysplastic syndrome (MDS), idiopathic thrombocytopenic purpura (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.
Having active malignancies, or having a history of treatment of malignancies within 5 years prior to informed consent.
Concurrent paroxysmal nocturnal hemoglobinuria (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
Having hepatitis C virus (HCV) infection confirmed by HCV-RNA or other tests at screening.
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:
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.
Facility Information:
Facility Name
NTT Medical center Tokyo
City
Shinagawa
State/Province
Tokyo
Country
Japan
12. IPD Sharing Statement
Plan to Share IPD
Undecided
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Study of AMG531 (Romiplostim) in Patients With Aplastic Anemia
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