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

Primary Purpose

Juvenile Idiopathic Arthritis

Status

Terminated

Phase

Not Applicable

Locations

Czechia

Study Type

Interventional

Intervention

Givinostat

About this trial

This is an interventional treatment trial for Juvenile Idiopathic Arthritis focused on measuring JIA

Eligibility Criteria

2 Years - 18 Years (Child, Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

1st Faculty of Medicine and General Faculty Hospital

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Givinostat

Arm Description

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

Outcomes

Primary Outcome Measures

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

Secondary Outcome Measures

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)

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)

Full Information

NCT ID

NCT01557452

First Posted

March 14, 2012

Last Updated

March 10, 2021

Sponsor

Italfarmaco

Collaborators

Parexel

1. Study Identification

Unique Protocol Identification Number

NCT01557452

Brief Title

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

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)

Study Type

Interventional

2. Study Status

Record Verification Date

March 2021

Overall Recruitment Status

Terminated

Why Stopped

Previous study DSC/08/2357/36 did not show efficacy for Givinostat in JIA. Sponsor decision to stop development of Givinostat in polyarticular course Juvenile Idiopathic Arthritis wasn't related to any tolerability concerns.

Study Start Date

December 28, 2011 (Actual)

Primary Completion Date

January 27, 2014 (Actual)

Study Completion Date

January 27, 2014 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Italfarmaco

Collaborators

Parexel

4. Oversight

Data Monitoring Committee

5. 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);

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Juvenile Idiopathic Arthritis

Keywords

JIA

7. Study Design

Primary Purpose

Treatment

Study Phase

Not Applicable

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

1 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Givinostat

Arm Type

Experimental

Arm Description

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

Intervention Type

Drug

Intervention Name(s)

Givinostat

Other Intervention Name(s)

ITF2357

Intervention Description

ready-to-use oral suspension, administered in fed condition and on a outpatient basis, especially intended for paediatric patients

Primary Outcome Measure Information:

Title

Number of Participants With Serious Adverse Events (SAE) and Adverse Events (AE) of Interest

Description

Time Frame

Through end of treatment, up to 108 weeks.

Secondary Outcome Measure Information:

Title

Number of Patients Who Maintained PedACR30 Response

Description

Time Frame

At weeks 48, 60 and 108

Title

Number of Patients Who Reached PedACR70 Response

Description

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)

Time Frame

At weeks 48, 60 and 108

10. Eligibility

Sex

All

Minimum Age & Unit of Time

2 Years

Maximum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

Eligibility 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

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Pavla Dolezalova, MD

Organizational Affiliation

General Faculty Hospital Department of Pediatrics and Adolescent Medicine, Praha, Czech Republic

Official's Role

Principal Investigator

Facility Information:

Facility Name

1st Faculty of Medicine and General Faculty Hospital

City

Praha

ZIP/Postal Code

12109

Country

Czechia

12. IPD Sharing Statement

Learn more about this trial

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

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