Clinical trial NCT02094417: A Phase 2 Study to Evaluate the Efficacy and Safety of AMG531 in Aplastic Anemia
Official 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Aplastic Anemia
Keywords: Anemia, Aplastic
Thrombopoietin
Thrombocytopenia
Lead sponsor: Kyowa Kirin Co., Ltd.
Sponsor class: INDUSTRY
First posted: 2014-03-21
Enrollment: 35
Sex: ALL
Minimum age (years): 19
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The present study will be conducted to evaluate the efficacy and safety of AMG531 and to determine the recommended initial dose of AMG531 on the basis of its efficacy and safety when it is administered subcutaneously (SC) to the Aplastic Anemia (AA) patients with immunosuppressive-therapy refractory thrombocytopenia and also to assess the pharmacokinetics of this product. Its efficacy and safety during the extension period beyond one year will also be evaluated.


Eligibility 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

Primary outcomes:
measure: Proportion of Subjects Achieving a Platelet Response at Week 9
timeFrame: At week 9
Secondary outcomes:
measure: Proportion of Subjects Achieving a Platelet Response
timeFrame: Initial dose evaluation period (Week 9), Week 12, Week 16, Week 24, Week 52, Week 104, and Week 156
measure: Proportion of Subjects Achieving Platelet Transfusion Independency
timeFrame: Initial dose evaluation period (Week 9), Week 12, Week 16, Week 24, Week 52, Week 104, and Week 156
measure: Proportion of Subjects Achieving Erythroid Response and/or Neutrophil Response
timeFrame: Initial dose evaluation period (Week 9), Week 12, Week 16, Week 24, Week 52, Week 104, and Week 156
measure: Duration of Platelet Response and Time to Platelet Response
timeFrame: Initial dose evaluation period (Week 9), Week 12, Week 16, Week 24, Week 52, Week 104, and Week 156
measure: Proportion of Subjects Achieving Tri-lineage Responses
timeFrame: Initial dose evaluation period (Week 9), Week 12, Week 16, Week 24, Week 52, Week 104, and Week 157
measure: Duration of Study Drug Discontinuation While Maintaining Stable Response
timeFrame: Initial dose evaluation period (Week 9), Week 12, Week 16, Week 24, Week 52, Week 104, and Week 156
Other outcomes:
Locations:
city: Seoul
country: Korea, Republic of
facility: Korea, Republic of
Study officials:
References:
citation: Lee JW, Lee SE, Jung CW, Park S, Keta H, Park SK, Kim JA, Oh IH, Jang JH. Romiplostim in patients with refractory aplastic anaemia previously treated with immunosuppressive therapy: a dose-finding and long-term treatment phase 2 trial. Lancet Haematol. 2019 Nov;6(11):e562-e572. doi: 10.1016/S2352-3026(19)30153-X. Epub 2019 Aug 29.