Study of Romiplostim(AMG531) in Subjects With Aplastic Anemia

• Register: ClinicalTrials.gov
• Last refreshed on: 22 July 2024
• Main ID: NCT02773290
• Date of registration: 09/05/2016
• Prospective Registration: No
• Primary sponsor: Kyowa Kirin Co., Ltd.
• Public title: Study of Romiplostim(AMG531) in Subjects With Aplastic Anemia
• Scientific title: Phase 2/3 Study of Romiplostim(AMG531) in Subjects With Aplastic Anemia Refractory to or Ineligible for Immunosuppressive Therapy
• Date of first enrolment: May 2016
• Target sample size: 46
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/ct2/show/NCT02773290
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2/Phase 3

Countries of recruitment

Japan, Korea, Republic of

Key inclusion & exclusion 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


Age minimum: 20 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Aplastic Anemia

Intervention(s)

Biological: Romiplostim

Primary Outcome(s)

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]

Secondary Outcome(s)

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]

Time from the first romiplostim administration to hematological response [Time Frame: 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]

Proportion of subjects with a hematological response at the end-of- treatment examination [Time Frame: Up to 52 weeks after dosing]

Secondary ID(s)

531-002

Source(s) of Monetary Support

Please refer to primary and secondary sponsors