Still´s Disease
Conditions
Keywords
Still´s disease, pediatric, adult, JIA, AOSD, Juvenile Idiopathic Arthritis, Adult Onset Still s disease, rheumatology, MAS825
Brief summary
The study is a phase II trial designed to evaluate the clinical efficacy, safety, and tolerability of MAS825 in pediatric and adult participants with Still's disease
Interventions
DRUGMAS825
Experimental drug
Sponsors
Novartis Pharmaceuticals
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
1 Years to 100 Years
Healthy volunteers
No
Inclusion criteria
* Age ≥ 1 with a diagnosis of Still's Disease * Active diseases defined as: * CRP or ferritin levels greater than ULN, and any of: * Fever ≥ 38°C attributed to Still's Disease activity and documented for a number of days prior to Day 1 or * Rash attributed to Still's Disease activity or * Musculoskeletal involvement: arthritis in a number of joints per ACP criteria for active joint or * Serositis or * Macrophage activation syndrome activity as defined by ferritin levels and at least one of: platelet count, a biomarker or fibrinogen levels attributed to Still's Disease activity by the investigator * Need for glucocorticoids (prednisone or equivalent)
Exclusion criteria
* Patients out of weight range * Ongoing or previous treatment with immunomodulatory drugs * A limited number of Still's Disease patients that have previously received MAS825 through a managed access program are permitted on the study * Glucocorticoid dose exceeding a set limit * Any conditions or significant medical problems which places the patient at unacceptable risk for MAS825 therapy * Still's disease patients with evidence of macrophage activation syndrome are permitted in the study * Still's Disease patients with evidence of interstitial lung disease including those requiring supplemental oxygen therapy are permitted in the study * History of ongoing, chronic or possibly recurrent infection (e.g. HIV, TB, HCV, HBV) and/or symptoms and signs of clinically significant active bacterial, fungal or viral infections * Live vaccinations within a set time prior to MAS825 treatment. Live vaccines are prohibited up to several months following the last dose * History of malignancy of any organ system, including post-transplant lymphoproliferative disorder, treated or untreated, within a number of years, regardless of whether there is evidence of local recurrence and metastases * History of hypersensitivity to any of the study drugs or to drugs of similar chemical classes or to any of the excipients * Pregnant or breastfeeding women * Women of child-bearing potential who do not agree to comply with required contraceptive use Other protocol-defined inclusion/
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of participants with clinical response based on one set of response criteria | Day 85 | Established measure for efficacy of treatments in the rheumatologic conditions taking into account self-assessment, function and survival. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Number of participants with clinical response based on another set of response criteria | Day 85 | Established measure for efficacy of treatments in the rheumatologic conditions taking into account self-assessment, function and survival. |
| Response based on a biomarker level | Baseline, Day 85 | To evaluate the efficacy of MAS825 in reducing clinical disease activity |
| Change in physician assessment based on laboratory features of MAS825 | Baseline, 15 months | To assess the effect of MAS825 on macrophage activation syndrome |
| Change from baseline in Glucocorticoid (GC) dose | Baseline, Day 85, Month 6, Month 12 | To evaluate the efficacy of MAS825 as a glucocorticoid-sparing agent |
| Change from baseline in patient/parent assessment of physical function as assessed by Quality of Life instrument | Baseline, Day 85 | To evaluate the efficacy of MAS825 in improving participant's quality of life |
| Clinically inactive disease whilst on non-treatment dose corticosteroid at any time during the study | 15 months | The number of participants with inactive disease will be assessed. Disease activity will be evaluated by the investigator based on physical examination and laboratory parameters. Inactive disease is defined according to protocol-specific criteria, which include clinical signs, laboratory values, and physician assessment. |
Countries
Canada, France, Germany, Italy, Netherlands, Spain, Turkey (Türkiye), United States
Contacts
CONTACTNovartis Pharmaceuticals
Outcome results
None listed