A Phase 2 Study to Evaluate the Efficacy and Safety of AMG531 in Aplastic Anemia

• Register: ClinicalTrials.gov
• Last refreshed on: 6 May 2024
• Main ID: NCT02094417
• Date of registration: 18/03/2014
• Prospective Registration: Yes
• Primary sponsor: Kyowa Kirin Korea Co., Ltd.
• Public title: A Phase 2 Study to Evaluate the Efficacy and Safety of AMG531 in Aplastic Anemia
• Scientific title: A Phase 2, Randomized, Open-Label, Parallel, Comparative, Dose-Finding Study to Evaluate the Efficacy and Safety of AMG531 in Aplastic Anemia Subjects With Thrombocytopenia Refractory to Immunosuppressive Therapy
• Date of first enrolment: April 14, 2014
• Target sample size: 35
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/ct2/show/NCT02094417
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

Korea, Republic of

Key inclusion & exclusion criteria

Inclusion Criteria:

- Patient who is diagnosed as AA and refractory to immunosuppressive therapy

- Platelet = 30,000/µL

Exclusion Criteria:

- Concurrent active infection not adequately responding to appropriate therapy

- HIV positivity

- Bone marrow reticulin grade of > 1

- Clinically significant cardiac disease

- Arterial or venous thrombosis within the last 1 year before enrollment

- Other cause of thrombocytopenia

- AA with hemolytic predominant paroxysmal nocturnal hemoglobinuria (PNH)

- Uncontrolled diabetes

- Receiving any agent used to treat AA, including antithymocyte globulin (ATG) or ATG +
cyclosporine within 6 months before starting study treatment and/or cyclosporine or
anabolic hormone within 6 weeks before starting the study treatment

- History of PEG-rHuMGDF, recombinant human thrombopoietin, AMG531, and other
thrombopoietin (TPO)-receptor agonist

- Who plans to conduct hematopoietic stem cell transplantation within 1 year



Age minimum: 19 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Aplastic Anemia

Intervention(s)

Drug: AMG531

Primary Outcome(s)

Proportion of Subjects Achieving a Platelet Response at Week 9 [Time Frame: At week 9]

Secondary Outcome(s)

Duration of Study Drug Discontinuation While Maintaining Stable Response [Time Frame: Initial dose evaluation period (Week 9), Week 12, Week 16, Week 24, Week 52, Week 104, and Week 156]

Proportion of Subjects Achieving Tri-lineage Responses [Time Frame: Initial dose evaluation period (Week 9), Week 12, Week 16, Week 24, Week 52, Week 104, and Week 157]

Duration of Platelet Response and Time to Platelet Response [Time Frame: Initial dose evaluation period (Week 9), Week 12, Week 16, Week 24, Week 52, Week 104, and Week 156]

Proportion of Subjects Achieving a Platelet Response [Time Frame: Initial dose evaluation period (Week 9), Week 12, Week 16, Week 24, Week 52, Week 104, and Week 156]

Proportion of Subjects Achieving Platelet Transfusion Independency [Time Frame: Initial dose evaluation period (Week 9), Week 12, Week 16, Week 24, Week 52, Week 104, and Week 156]

Proportion of Subjects Achieving Erythroid Response and/or Neutrophil Response [Time Frame: Initial dose evaluation period (Week 9), Week 12, Week 16, Week 24, Week 52, Week 104, and Week 156]

Secondary ID(s)

531-KR001

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 15/02/2021
• URL: https://clinicaltrials.gov/ct2/show/results/NCT02094417