Phase 2 N=35 Randomized Treatment

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

Aplastic Anemia

Bottom Line

View on ClinicalTrials.gov: NCT02094417 ↗

Enrolled (actual)

Serious AEs

20.0%

Results posted

Feb 2021

Primary outcome: Primary: Proportion of Subjects Achieving a Platelet Response at Week 9 — 0; 0; 3; 7 Participants

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: AMG531 (Drug)
Age: Adult, Older Adult · 19+ yrs
Sex: All
Sponsor: Kyowa Kirin Korea Co., Ltd.
Primary completion: Apr 2017

Outcome Measures

Outcome	Result	p-value
PRIMARY Proportion of Subjects Achieving a Platelet Response at Week 9	0; 0; 3; 7	—
SECONDARY Proportion of Subjects Achieving a Platelet Response	3; 3; 6; 10; 4; 4	—
SECONDARY Proportion of Subjects Achieving Platelet Transfusion Independency	0; 2; 0; 2; 0; 2	—
SECONDARY Proportion of Subjects Achieving Erythroid Response and/or Neutrophil Response	0; 2; 1; 3; 0; 4	—
SECONDARY Duration of Platelet Response and Time to Platelet Response	24.5; 21.0; 503.5; 131.0; 59.0; 44.0	—
SECONDARY Proportion of Subjects Achieving Tri-lineage Responses	0; 0; 0; 0; 0; 0	—
SECONDARY Duration of Study Drug Discontinuation While Maintaining Stable Response	273.0; 483.0	—

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

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT02094417). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.