A Phase II Trial to Evaluate the Clinical Efficacy, Safety and Tolerability of MAS825 in Pediatric and Adult Participants With Still's Disease

Official title

An Open-label Phase II Trial to Evaluate the Clinical Efficacy, Safety and Tolerability of MAS825 in Pediatric and Adult Participants With Still's Disease

Overview

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

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.

Interventions

• Drug MAS825
  Experimental drug

Primary outcome measures

• Number of participants with clinical response based on one set of response criteria [Time frame: Day 85]

Secondary outcome measures (6)

• Number of participants with clinical response based on another set of response criteria [Time frame: Day 85]
• Response based on a biomarker level [Time frame: Baseline, Day 85]
• Change in physician assessment based on laboratory features of MAS825 [Time frame: Baseline, 15 months]
• Change from baseline in Glucocorticoid (GC) dose [Time frame: Baseline, Day 85, Month 6, Month 12]
• Change from baseline in patient/parent assessment of physical function as assessed by Quality of Life instrument [Time frame: Baseline, Day 85]
• Clinically inactive disease whilst on non-treatment dose corticosteroid at any time during the study [Time frame: 15 months]

Eligibility criteria

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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation

N/A

Model

Single group

Masking

Open label

Primary purpose

Treatment

Study locations

France · 6 centers

• Novartis Investigative Site — Bordeaux
• Novartis Investigative Site — Bron
• Novartis Investigative Site — Le Kremlin-Bicêtre
• Novartis Investigative Site — Lille
• Novartis Investigative Site — Paris
• Novartis Investigative Site — Paris

Germany · 3 centers

• Novartis Investigative Site — Sankt Augustin
• Novartis Investigative Site — Heidelberg
• Novartis Investigative Site — Münster

Italy · 3 centers

• Novartis Investigative Site — Florence
• Novartis Investigative Site — Genova
• Novartis Investigative Site — Roma

Spain · 3 centers

• Novartis Investigative Site — Esplugues
• Novartis Investigative Site — Madrid
• Novartis Investigative Site — Valencia

United States · 2 centers

• Cincinnati Childrens Hospital — Cincinnati
• Legacy Emanuel Research Hosp Portland — Portland

Canada · 2 centers

• Novartis Investigative Site — Montreal
• Novartis Investigative Site — Montreal

Turkey (Türkiye) · 2 centers

• Novartis Investigative Site — Istanbul
• Novartis Investigative Site — Ankara

Netherlands · 1 center

• Novartis Investigative Site — Utrecht

Identifiers

NCT: NCT07203001 · CMAS825J12201 · 2024-520208-25

Primary sources (government registries)

• ClinicalTrials.gov (US NIH)

View this study on ClinicalTrials.gov ↗