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

Not Applicable

Terminated

Conditions: Juvenile Idiopathic Arthritis

Interventions: Drug: Givinostat

Registration Number: NCT01557452

Lead Sponsor: Italfarmaco

Brief Summary: 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: 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.

Recruitment & Eligibility

Status: TERMINATED

Sex: All

Target Recruitment: 1

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

Study & Design

Study Type: INTERVENTIONAL

Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Givinostat	Givinostat	Patient received the dose of 0.75 mg/kg BID from December 28th, 2011 to January 27th, 2014

Primary Outcome Measures

Name	Time	Method
Number of Participants With Serious Adverse Events (SAE) and Adverse Events (AE) of Interest	Through end of treatment, up to 108 weeks.	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

Secondary Outcome Measures

Name	Time	Method
Number of Patients Who Reached PedACR70 Response	At weeks 48, 60 and 108	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)
Number of Patients Who Maintained PedACR30 Response	At weeks 48, 60 and 108	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)

Trial Locations

Locations (1): 1st Faculty of Medicine and General Faculty Hospital
🇨🇿
Praha, Czechia
1st Faculty of Medicine and General Faculty Hospital
🇨🇿Praha, Czechia