Juvenile Idiopathic Arthritis
Conditions
Keywords
JIA
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.
Interventions
DRUGGivinostat
ready-to-use oral suspension, administered in fed condition and on a outpatient basis, especially intended for paediatric patients
Sponsors
Parexel
Italfarmaco
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
2 Years to 18 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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
| Measure | Time frame | Description |
|---|---|---|
| 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) |
| 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) |
Countries
Czechia
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Givinostat Givinostat: oral suspension, 0,75 mg/Kg b.i.d. in fed conditions | 1 |
| Total | 1 |
Baseline characteristics
| Characteristic | Givinostat |
|---|---|
| Age, Categorical <=18 years | 1 Participants |
| Age, Categorical >=65 years | 0 Participants |
| Age, Categorical Between 18 and 65 years | 0 Participants |
| Ethnicity (NIH/OMB) Hispanic or Latino | 0 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 1 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Region of Enrollment Czechia | 1 participants |
| Sex: Female, Male Female | 1 Participants |
| Sex: Female, Male Male | 0 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 1 |
| other Total, other adverse events | 1 / 1 |
| serious Total, serious adverse events | 0 / 1 |
Outcome results
Primary
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
Time frame: Through end of treatment, up to 108 weeks.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Givinostat | Number of Participants With Serious Adverse Events (SAE) and Adverse Events (AE) of Interest | non serious AE | 1 participants |
| Givinostat | Number of Participants With Serious Adverse Events (SAE) and Adverse Events (AE) of Interest | serious AE | 0 participants |
Secondary
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)
Time frame: At weeks 48, 60 and 108
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Givinostat | Number of Patients Who Maintained PedACR30 Response | week 48 | 1 participants |
| Givinostat | Number of Patients Who Maintained PedACR30 Response | week 60 | 0 participants |
| Givinostat | Number of Patients Who Maintained PedACR30 Response | week 108 | 1 participants |
Secondary
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)
Time frame: At weeks 48, 60 and 108
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Givinostat | Number of Patients Who Reached PedACR70 Response | week 48 | 0 participants |
| Givinostat | Number of Patients Who Reached PedACR70 Response | week 108 | 0 participants |
| Givinostat | Number of Patients Who Reached PedACR70 Response | week 60 | 1 participants |