A Phase II Trial to Evaluate the Clinical Efficacy, Safety and Tolerability of MAS825 in Pediatric and Adult Participants With Still's Disease
Purpose
The study is a phase II trial designed to evaluate the clinical efficacy, safety, and tolerability of MAS825 (arumakimig) in pediatric and adult participants with Still's disease
Condition
- Still´s Disease
Eligibility
- Eligible Ages
- Between 1 Year and 100 Years
- Eligible Sex
- All
- Accepts 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 - Intolerance or inadequate response to available biologic therapy
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/exclusion criteria may apply.
Study Design
- Phase
- Phase 2
- Study Type
- Interventional
- Allocation
- N/A
- Intervention Model
- Single Group Assignment
- Primary Purpose
- Treatment
- Masking
- None (Open Label)
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
|
Experimental MAS825 |
Experimental drug |
|
Recruiting Locations
Legacy Emanuel Research Hosp Portland
Portland, Oregon 97232
Portland, Oregon 97232
More Details
- Status
- Recruiting
- Sponsor
- Novartis Pharmaceuticals
Detailed Description
This is a multicenter, open-label, single-arm study to assess clinical efficacy, safety and tolerability of MAS825 in participants (both pediatric and adult) with Still's disease who have had inadequate response to IL-1 and/or IL-6 or other available therapies. Participants with associated lung disease and/or macrophage activation syndrome may also be included. This study has three periods (Screening, Treatment Period 1, and Treatment Period 2) and a total duration of approximately 16 months.