Study of Romiplostim(AMG531) in Subjects With Aplastic Anemia

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Brief Title

Study of Romiplostim(AMG531) in Subjects With Aplastic Anemia

Official Title

Phase 2/3 Study of Romiplostim(AMG531) in Subjects With Aplastic Anemia Refractory to or Ineligible for Immunosuppressive Therapy

Brief Summary

      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 Phase

Phase 2/Phase 3

Study Type

Interventional


Primary Outcome

Proportion of subjects achieving a hematological response (any of the platelet response, erythroid response, and neutrophil response) at Week 27

Secondary Outcome

 Proportion of subjects with a hematological response at the end-of- treatment examination

Condition

Aplastic Anemia

Intervention

Romiplostim

Study Arms / Comparison Groups

 Romiplostim
Description:  Weekly Subcutaneous (SC) administration

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Biological

Estimated Enrollment

46

Start Date

May 2016

Completion Date

December 2020

Primary Completion Date

December 2020

Eligibility Criteria

        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
      

Gender

All

Ages

20 Years - N/A

Accepts Healthy Volunteers

No

Contacts

, , 

Location Countries

Japan

Location Countries

Japan

Administrative Informations


NCT ID

NCT02773290

Organization ID

531-002


Responsible Party

Sponsor

Study Sponsor

Kyowa Kirin Co., Ltd.


Study Sponsor

, , 


Verification Date

February 2020