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

• ClinicalTrials.gov Identifier: NCT01557452
• Recruitment Status: Terminated
• First Posted: March 19, 2012
• Results First Posted: April 6, 2021
• Last Update Posted: April 6, 2021
• Sponsor: Italfarmaco
• Collaborator: Parexel
• Information provided by (Responsible Party): Italfarmaco

Study Description

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);

Condition or disease	Intervention/treatment	Phase
Juvenile Idiopathic Arthritis	Drug: Givinostat	Not Applicable

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.

Study Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 1 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: An Open-Label Extension of the Dose Finding Study (DSC/08/2357/36) in Patients With Polyarticular Course Juvenile Idiopathic Arthritis (Poly JIA)
• Actual Study Start Date: December 28, 2011
• Actual Primary Completion Date: January 27, 2014
• Actual Study Completion Date: January 27, 2014

Arms and Interventions

Arm	Intervention/treatment
Experimental: Givinostat; Patient received the dose of 0.75 mg/kg BID from December 28th, 2011 to January 27th, 2014	Drug: Givinostat; ready-to-use oral suspension, administered in fed condition and on a outpatient basis, especially intended for paediatric patients; Other Name: ITF2357

Outcome Measures

Primary Outcome Measures :

1. Number of Participants With Serious Adverse Events (SAE) and Adverse Events (AE) of Interest [ Time Frame: 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 :

1. Number of Patients Who Maintained PedACR30 Response [ Time Frame: 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)

2. Number of Patients Who Reached PedACR70 Response [ Time Frame: 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)

Eligibility Criteria

• Ages Eligible for Study: 2 Years to 18 Years (Child, Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Criteria

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

Contacts and Locations

Locations

Czechia

1st Faculty of Medicine and General Faculty Hospital

Praha, Czechia, 12109

Sponsors and Collaborators

Italfarmaco

Parexel

Investigators

• Principal Investigator: Pavla Dolezalova, MD; General Faculty Hospital Department of Pediatrics and Adolescent Medicine, Praha, Czech Republic

More Information

• Responsible Party: Italfarmaco
• ClinicalTrials.gov Identifier: NCT01557452
• Other Study ID Numbers: DSC/11/2357/42; 2011-003341-18 ( EudraCT Number )
• First Posted: March 19, 2012
• Results First Posted: April 6, 2021
• Last Update Posted: April 6, 2021
• Last Verified: March 2021

Keywords provided by Italfarmaco:

JIA

Additional relevant MeSH terms:

Arthritis; Arthritis, Juvenile; Joint Diseases; Musculoskeletal Diseases; Rheumatic Diseases; Connective Tissue Diseases; Autoimmune Diseases; Immune System Diseases; Givinostat hydrochloride; Histone Deacetylase Inhibitors; Enzyme Inhibitors; Molecular Mechanisms of Pharmacological Action