Open-Label Extension of the Dose Finding Study (DSC/08/2357/36) in Patients With Poly Juvenile Idiopathic Arthritis

Study Overview
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Brief Summary
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Primary Objective of the study: the purpose of this extension study was to determine the safety of Givinostat in a long term treatment of patients who participated in DSC/08/2357/36 study with good results (clinical benefit at least pediACR30 response);

Detailed Description
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Givinostat is expected to exert a clinically relevant therapeutic effect on polyarticular JIA through the inhibition of the production/release of pro-inflammatory cytokines, such as IL-1β, IL-6 and TNFα, which are involved in the pathogenesis of the arthritic process.

Official Title
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An Open-Label Extension of the Dose Finding Study (DSC/08/2357/36) in Patients With Polyarticular Course Juvenile Idiopathic Arthritis (Poly JIA)

Conditions
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Juvenile Idiopathic Arthritis

Intervention / Treatment
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* Drug: Givinostat


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: subjects who had successfully completed the previous Dose Finding Study and were fully compliant to the inclusion/exclusion criteria described in the previous DSC/08/2357/36 Exclusion Criteria: patients with fever related to JIA or other systemic features of JIA during 12 months before entering the study active bacterial or mycotic infection requiring antimicrobial treatment episode of macrophage activation syndrome over the last 6 months baseline prolongation of QT/QTc interval, use of concomitant medications that prolong the QT/QTc interval or history of additional risk factors for TdP. clinically significant cardiovascular disease clinically significant illness i.e. any condition that in the opinion of the Investigator places the patient to unacceptable risk for adverse outcome if he/she were to participate in the study psychiatric illness/social situation that would limit compliance with study medication and protocol requirements inherited metabolic diseases presence of malignancy pregnancy or lactation positive blood test for HIV active EBV infection, active B and/or C hepatitis platelet count <100x10(9)/L

Ages Eligible for Study
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Minimum Age: 2 Years
Maximum 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: Givinostat<br>Patient received the dose of 0.75 mg/kg BID from December 28th, 2011 to January 27th, 2014 | Drug: Givinostat<br>* ready-to-use oral suspension, administered in fed condition and on a outpatient basis, especially intended for paediatric patients<br>* Other names: ITF2357;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of Participants With Serious Adverse Events (SAE) and Adverse Events (AE) of Interest | During the entire study period it was reported only one adverse event considered not drug related by the investigator (Mild flu at week 107 of study treatment). No action was taken and the patient recovered spontaneously | Through end of treatment, up to 108 weeks. | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of Patients Who Maintained PedACR30 Response | This is an open-label treatment extension of the 2010-019094-15 study, an antecedent dose-ranging trial of Givinostat ready-to-use oral suspension formulation. Eligible patients were those who had completed the previous study achieving a clinical benefit, i.e. patients achieving at least an ACR Paediatric 30 (PedACR30). PedACR30 is defined as at least a 30% improvement from baseline in any three of the following six variables in juvenile idiopathic arthritis (JIA) patients, with no more than one variable worsening by more than 30%: physician's global assessment of disease activity; parent/guardian's or patient's global assessment of overall wellbeing; functional ability; number of joints with active arthritis; number of joints with limited range of motion; ESR. (ACR stands for American College of Rheumatology) | At weeks 48, 60 and 108 | 
| Number of Patients Who Reached PedACR70 Response | This is an open-label treatment extension of the 2010-019094-15 study, an antecedent dose-ranging trial of Givinostat ready-to-use oral suspension formulation. Eligible patients were those who had completed the previous study achieving a clinical benefit, i.e. patients achieving at least an ACR Paediatric 30 (PedACR30). PedACR70 is defined as at least a 70% improvement from baseline in any three of the following six variables in juvenile idiopathic arthritis (JIA) patients, with no more than one variable worsening by more than 30%: physician's global assessment of disease activity; parent/guardian's or patient's global assessment of overall wellbeing; functional ability; number of joints with active arthritis; number of joints with limited range of motion; ESR. (ACR stands for American College of Rheumatology) | At weeks 48, 60 and 108 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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JIA