Clinical trial NCT04095936: 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
Overall status: COMPLETED
Phase: PHASE2
PHASE3
Conditions: Aplastic Anemia
Keywords: aplastic anemia
romiplostim
Lead sponsor: Kyowa Kirin Co., Ltd.
Sponsor class: INDUSTRY
First posted: 2019-09-19
Enrollment: 24
Sex: ALL
Minimum age (years): 20
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: multi-national, open-label, phase 2/3 study
Primary purpose: TREATMENT

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.


Eligibility criteria: Inclusion Criteria:

1. Voluntary signed informed consent to participate in the study;
2. A diagnosis of AA confirmed by blood and bone-marrow examinations, etc.;
3. Considered to require new treatment with immunosuppressive therapy provided that NSAA must be platelet or erythrocyte transfusion-dependent.
4. An Eastern Cooperative Oncology Group (ECOG) performance status (PS) score of 0 to 1at screening;

Exclusion Criteria:

1. Previously treated with Anti-human thymocyte immunoglobulin (ATG), CsA, or Alemtuzumab;
2. Diagnosed as having congenital AA (Fanconi anemia, congenital dyskeratosis, etc.);
3. Diagnosed as having acute myelocytic leukemia (AML) or chronic myelomonocytic leukemia;
4. Concurrent thrombocytopenia of other etiologies (e.g., myelodysplastic syndrome (MDS), idiopathic thrombocytopenic purpura (ITP), cirrhosis);
5. Concurrent active infection not adequately responding to appropriate therapy;
6. Concurrent clinically significant illness(es) items which are deemed by the Investigator to be likely to affect the study conduct and assessments.
7. Having active malignancies, or having a history of treatment of malignancies within 5 years prior to informed consent.
8. Concurrent paroxysmal nocturnal hemoglobinuria (PNH)
9. Having Grade 2 or higher bone marrow reticulin based on Bone Marrow Pathology (2nd edition) ;
10. History of chromosome aberrations discovered in bone marrow cells.
11. Having blast cells \> 2% in bone marrow;
12. Positive for anti-human immunodeficiency virus (HIV) antibody;
13. Receiving prophylactic or therapeutic treatment for hepatitis type B
14. Having hepatitis C virus (HCV) infection confirmed by HCV-RNA or other tests at screening.
15. Planned hematopoietic stem cell transplantation during the study;
16. Systemic treatment with any of the following medication for the treatment of AA within 4 weeks before Day 1:

    * Anabolic steroids
    * Corticosteroids;
17. Pregnant or breastfeeding women, or women willing to become pregnant;
18. Other conditions unsuitable for participation in the study in the opinion of the Investigator.

Primary outcomes:
measure: Rate of achievement of complete response (CR) or partial response (PR)
timeFrame: 27 weeks post-dose
Secondary outcomes:
measure: Rate of achievement of CR or PR
timeFrame: 14 weeks
measure: Rate of achievement of CR
timeFrame: Weeks 14 and 27
measure: The time to CR or PR
timeFrame: Each time point evaluated weekly until Week 27
measure: Reduction or independence of platelet and/or erythrocyte transfusion
timeFrame: Week 27
measure: Change from baseline in platelet count (/µL)
timeFrame: Each time point evaluated weekly until Week 27
measure: Change from baseline in hemoglobin (Hb) concentration (g/dL)
timeFrame: Each time point evaluated weekly until Week 27
measure: Change from baseline in neutrophil count (/µL)
timeFrame: Each time point evaluated weekly until Week 27
measure: Change from baseline in reticulocyte count (/µL)
timeFrame: Each time point evaluated weekly until Week 27
Other outcomes:
Locations:
city: Shinagawa
country: Japan
facility: NTT Medical center Tokyo
state: Tokyo
Study officials:
References: