MONICA-SC: A Study to Evaluate the Efficacy, Safety and Tolerability of Blisibimod (A-623) Administration in Subjects With ITP

Study Overview
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Brief Summary
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The purpose of this study is to evaluate efficacy, safety and tolerability of blisibimod when administered on top of standard-of-care to subjects with Immune Thrombocytopenic Purpura (ITP).

Official Title
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MONICA-SC: A Randomized, Double-Blind, Placebo-Controlled Phase 2/3 Study to Evaluate the Efficacy, Safety and Tolerability of Blisibimod (A-623) Administration in Subjects With Immune Thrombocytopenic Purpura (ITP)

Conditions
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Immune Thrombocytopenic Purpura, Idiopathic Thrombocytopenic Purpura

Intervention / Treatment
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* Biological: Blisibimod
* Other: Placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: 18 to 75 years of age(male or female). Diagnosis of ITP according to the guidelines of the American Society of Hematology (ASH) and British Committee for Standards in Hematology. Platelet counts at Screening of 30 billion/L or less for subjects not on ITP medication, or 50 billion/L or less for subjects receiving stable background ITP medication. Exclusion Criteria: Subjects who have had a splenectomy for any reason. Currently receiving high-dose ITP medications, eltrombopag, romiplostim, rituximab, or investigational therapeutic agents. Nursing or pregnant. Active infection requiring hospitalization or treatment with parenteral antibiotics within the past 60 days. Any known history of bone marrow stem cell disorder. Active hepatitis B, active hepatitis C or a documented history of HIV, hepatitis B, or hepatitis C. Liver disease. Malignancy within the past 5 years. History of active tuberculosis (TB) or history of TB infection. Subject has not yet completed at least 3 months or 5 half-lives (whichever is longer) since ending other investigational study. History of congenital immunodeficiency.

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 75 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Blisibimod<br> | Biological: Blisibimod<br> <br> * Other names: A-623;|
| Placebo Comparator: Placebo<br> | Other: Placebo<br> <br> |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Achievement of a durable platelet response of 50 billion platelets per liter or higher over the last weeks of treatment. |  | 24 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Achievement of a durable platelet count of 50 billion platelets per liter or higher over the last weeks of treatment under conditions of decreased concomitant steroid medication. |  | 24 weeks | 
| Achievement of a transient improvement in platelet count of 50 billion platelets per liter or higher at any 4 weeks of the treatment period. |  | 24 weeks | 
| Change in background corticosteroid dose. |  | baseline to 24 weeks | 
| Percentage of subjects requiring rescue therapy. |  | 24 weeks | 
| Time to treatment failure. |  | 24 weeks | 
| Change in bleeding risk. |  | baseline to 24 weeks | 
| Safety profile (AEs, vitals signs, labs) |  | 24 weeks | 
| Biomarker changes from baseline. |  | baseline to 24 weeks | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Immune Thrombocytopenic Purpura, Idiopathic Thrombocytopenic Purpura, Chronic ITP