Efficacy and Safety Dose Finding Study of Givinostat to Treat Polyarticular Course Juvenile Idiopathic Arthritis

• Register: ClinicalTrials.gov
• Last refreshed on: 19 October 2017
• Main ID: NCT01261624
• Date of registration: 15/12/2010
• Prospective Registration: No
• Primary sponsor: Italfarmaco
• Public title: Efficacy and Safety Dose Finding Study of Givinostat to Treat Polyarticular Course Juvenile Idiopathic Arthritis
• Scientific title: A Multicenter, Open Label, Dose Finding Study to Evaluate Efficacy and Safety of Givinostat Administered in Two Different Doses in Patients With Poly JIA Not Adequately Responding to the Standard Treatment.
• Date of first enrolment: October 2010
• Target sample size: 16
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/show/NCT01261624
• Study type: Interventional
• Phase: Phase 2

Countries of recruitment

Belgium, Czech Republic, Italy, Romania, Serbia, Slovenia, Spain

Contacts

• Name: Francesco Zulian, MD
• Affiliation: Azienda Ospedaliera-Università di Padova - Unità di Reumatologia Pediatrica

Key inclusion & exclusion criteria

Inclusion Criteria:

- patients of both genders, aged 2 to 17 years, with established diagnosis of
polyarticular course Juvenile Idiopathic Arthritis (see before for specific subtypes)
according to ILAR (International League Against Rheumatism) criteria (Petty RE et al.,
2004) for at least six months before the study entry

- age at polyarticular JIA diagnosis < 16 years

- active disease for at least 6 months prior to enrolment as defined by the following
criteria:

- presence of at least 5 active joints (those with swelling or, in the absence of
swelling, limited range of motion accompanied by pain/tenderness)

- inadequate response to, or intolerance to, at least one biologic agent such as, but
not limited to, etanercept, in?iximab, and adalimumab.

- maximum allowed steroid dose 0.2 mg/kg/day or 10 mg/day (whichever is lower) of
prednisone or equivalent

- in case of concomitant methotrexate treatment, it has to be on a stable dose =15 mg/m2
weekly for at least 1 month before patient's enrolment

- other disease-modifying anti-rheumatic drugs possibly previously introduced have to be
discontinued for a period of at least five half-lives

- concomitant nonsteroidal anti-inflammatory drugs, if any, on a stable dose for at
least four weeks before patient's enrolment

Exclusion Criteria:

- patient 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 in the last 6 months

- a baseline prolongation of QT/QTc interval, use of concomitant medications that
prolong the QT/QTc interval or history of additional risk factors for TdP (e.g., heart
failure, hypokalemia, family history of Long QT Syndrome) (Appendix C)

- clinically significant cardiovascular disease

- clinically significant illness i.e. any condition (including laboratory abnormalities)
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 situations 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 <100x109/L

- absolute neutrophil count <1.5x109/L

- serum creatinine >2xULN (Upper limit of normal).

- total serum bilirubin >1.5xULN.

- serum AST/ALT > 3xULN.

- congenital heart and/or central nervous system disorders



Age minimum: 2 Years
Age maximum: 17 Years
Gender: All

Health Condition(s) or Problem(s) studied

Polyarticular Course Juvenile Idiopathic Arthritis

Intervention(s)

Drug: Givinostat

Primary Outcome(s)

ACR Pediatric Response Level (ACRPRL) 30 After 12 Weeks of Treatment [Time Frame: 12 weeks of treatment]

Secondary Outcome(s)

ACR Pediatric Response Level (ACR 50, 70, 90 and 100) at Week 12 [Time Frame: at week12]

Secondary ID(s)

DSC/08/2357/36

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 16/04/2014
• URL: https://clinicaltrials.gov/ct2/show/results/NCT01261624