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

March 10, 2021 updated by: Italfarmaco

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

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);

Study Overview

Status

Terminated

Intervention / Treatment

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 Type

Interventional

Enrollment (Actual)

1

Phase

  • Not Applicable

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

      • Praha, Czechia, 12109
        • 1st Faculty of Medicine and General Faculty Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

2 years to 18 years (Child, Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

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

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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

Through end of treatment, up to 108 weeks.

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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)

At weeks 48, 60 and 108
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)

At weeks 48, 60 and 108

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Collaborators

Investigators

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

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

December 28, 2011

Primary Completion (Actual)

January 27, 2014

Study Completion (Actual)

January 27, 2014

Study Registration Dates

First Submitted

March 14, 2012

First Submitted That Met QC Criteria

March 16, 2012

First Posted (Estimate)

March 19, 2012

Study Record Updates

Last Update Posted (Actual)

April 6, 2021

Last Update Submitted That Met QC Criteria

March 10, 2021

Last Verified

March 1, 2021

More Information

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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