Phase I Safety and Tolerability Study of Staphylococcal Protein A in Adult Patients With Chronic ITP

Study Overview
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Brief Summary
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The primary purpose of this study is to evaluate the safety of multiple doses of Staphylococcal protein A (PRTX-100) in adult patients with Idiopathic Thrombocytopenia Purpura (ITP). The pharmacokinetics, immunogenicity and pharmacodynamics will also be studied. Patients will be enrolled into 1 of 3 dose groups and receive 4 weekly IV doses of PRTX-100. A Safety Monitoring Committee will review safety data through Day 28 for the first 5 patients in a dose group before escalation to the next higher dose level. Patients will be followed for 8 weeks after dosing for safety, PK, immunogenicity and effect on platelet count(pharmacodynamics).

Official Title
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An Open Label, Sequential, Dose Escalation, Repeat-dose Study of the Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of PRTX-100 in Adult Patients With Chronic Idiopathic Thrombocytopenic Purpura (ITP)

Conditions
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Idiopathic Thrombocytopenic Purpura (ITP)

Intervention / Treatment
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* Drug: PRTX-100 (Staphylococcal protein A)


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Diagnosis of chronic ITP > 4 months Mean platelet count <50 x 10^9/L for patients not receiving corticosteroids; or mean platelet count >=50 x 10^9/L for patients receiving stable dose of corticosteroids Exclusion Criteria: Splenectomy within 45 days of screening Rituximab within 6 months prior to screening Cyclophosphamide, vincristine, or any other non-monoclonal antibody treatment for ITP within 3 months prior to screening IVIG, WinRho or other anti-RhD within 30 days prior to screening

Ages Eligible for Study
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Minimum Age: 18 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
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: PRTX-100 (Staphylococcal protein A)<br>Cohort 1: 0.075 mcg/kg Cohort 2: 0.15 mcg/kg Cohort 3: 0.30 mcg/kg | Drug: PRTX-100 (Staphylococcal protein A)<br>* 4 weekly IV (in the vein) doses of 1 of the 3 following PRTX-100 dose levels: Cohort 1: 0.075 mcg/kg Cohort 2: 0.15 mcg/kg Cohort 3: 0.30 mcg/kg<br>* Other names: PRTX-100;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Evaluate the overall safety of PRTX-100 during the 3 month study duration |  | 3 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Characterize the pharmacokinetics of multiple doses of PRTX-100 |  | over the first 35 days | 
| Explore immunogenicity of multiple doses of PRTX-10 |  | 3 months | 
| Evaluate treatment effect on platelet count |  | 3 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Autoimmune Diseases, Bleeding Disorders, Blood and Blood Disorders