Trial Outcomes & Findings for Open-Label Extension of the Dose Finding Study (DSC/08/2357/36) in Patients With Poly Juvenile Idiopathic Arthritis (NCT NCT01557452)

Results Overview

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

Recruitment status

TERMINATED

Study phase

NA

Target enrollment

1 participants

Primary outcome timeframe

Through end of treatment, up to 108 weeks.

Results posted on

2021-04-06

Participant Flow

Participant milestones

Participant milestones
Measure	Givinostat Givinostat: oral suspension, 0,75 mg/Kg b.i.d. in fed conditions
Overall Study STARTED	1
Overall Study COMPLETED	1
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Givinostat n=1 Participants Givinostat: oral suspension, 0,75 mg/Kg b.i.d. in fed conditions
Age, Categorical <=18 years	1 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Sex: Female, Male Female	1 Participants n=5 Participants
Sex: Female, Male Male	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	1 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment Czechia	1 participants n=5 Participants

PRIMARY outcome

Timeframe: 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

Outcome measures

Outcome measures
Measure	Givinostat n=1 Participants Givinostat: oral suspension, 0,75 mg/Kg b.i.d. in fed conditions
Number of Participants With Serious Adverse Events (SAE) and Adverse Events (AE) of Interest non serious AE	1 participants
Number of Participants With Serious Adverse Events (SAE) and Adverse Events (AE) of Interest serious AE	0 participants

SECONDARY outcome

Timeframe: 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)

Outcome measures

Outcome measures
Measure	Givinostat n=1 Participants Givinostat: oral suspension, 0,75 mg/Kg b.i.d. in fed conditions
Number of Patients Who Maintained PedACR30 Response week 48	1 participants
Number of Patients Who Maintained PedACR30 Response week 60	0 participants
Number of Patients Who Maintained PedACR30 Response week 108	1 participants

SECONDARY outcome

Timeframe: 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)

Outcome measures

Outcome measures
Measure	Givinostat n=1 Participants Givinostat: oral suspension, 0,75 mg/Kg b.i.d. in fed conditions
Number of Patients Who Reached PedACR70 Response week 48	0 participants
Number of Patients Who Reached PedACR70 Response week 60	1 participants
Number of Patients Who Reached PedACR70 Response week 108	0 participants

Adverse Events

Givinostat

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Givinostat n=1 participants at risk Givinostat: oral suspension, 0,75 mg/kg b.i.d. in fed conditions
Infections and infestations Influenza	100.0% 1/1 • Number of events 1 • Through end of treatment, up to 108 weeks.

Additional Information

Paolo Bettica, MD

Italfarmaco SpA

Phone: +39 02 64431

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place