En fase II -studie for å evaluere den kliniske effektiviteten, sikkerheten og toleransen av MAS825 hos barn og voksne deltakere med stilles sykdom
En åpen en-etikett fase II-studie for å evaluere den kliniske effektiviteten, sikkerheten og toleransen av MAS825 hos pediatriske og voksne deltakere med stilles sykdom
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
Studieoversikt
Detaljert beskrivelse
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.
Studietype
Registrering (Antatt)
Fase
- Fase 2
Kontakter og plasseringer
Studiekontakt
- Navn: Novartis Pharmaceuticals
- Telefonnummer: 1-888-669-6682
- E-post: novartis.email@novartis.com
Studer Kontakt Backup
- Navn: Novartis Pharmaceuticals
- Telefonnummer: +41613241111
Studiesteder
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Ontario
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Toronto, Ontario, Canada, M5G 1X8
- Rekruttering
- Novartis Investigative Site
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Quebec
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Montreal, Quebec, Canada, H4A 3J1
- Tilbaketrukket
- Novartis Investigative Site
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Montreal, Quebec, Canada, H3T 1C5
- Tilbaketrukket
- Novartis Investigative Site
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Ohio
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Cincinnati, Ohio, Forente stater, 45229
- Aktiv, ikke rekrutterende
- Cincinnati Childrens Hospital
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Oregon
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Portland, Oregon, Forente stater, 97232
- Rekruttering
- Legacy Emanuel Research Hosp Portland
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Hovedetterforsker:
- Daniel Joseph Kingsbury
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Ta kontakt med:
- Brenna Bogle
- Telefonnummer: +1 503 413 5447
- E-post: bbogle@lhs.org
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Utah
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Salt Lake City, Utah, Forente stater, 84113
- Rekruttering
- University of Utah
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Ta kontakt med:
- Tressie Rollins
- Telefonnummer: +1 415 515 8212
- E-post: tressie.rollins@hsc.utah.edu
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Hovedetterforsker:
- Aimee Ogden Hersh
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Bordeaux, Frankrike, 33076
- Rekruttering
- Novartis Investigative Site
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Bron, Frankrike, 69677
- Rekruttering
- Novartis Investigative Site
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Le Kremlin-Bicêtre, Frankrike, 94275
- Rekruttering
- Novartis Investigative Site
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Lille, Frankrike, 59037
- Rekruttering
- Novartis Investigative Site
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Paris, Frankrike, 75015
- Rekruttering
- Novartis Investigative Site
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Paris, Frankrike, 75970
- Rekruttering
- Novartis Investigative Site
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FI
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Florence, FI, Italia, 50139
- Rekruttering
- Novartis Investigative Site
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GE
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Genova, GE, Italia, 16147
- Rekruttering
- Novartis Investigative Site
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RM
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Roma, RM, Italia, 00165
- Rekruttering
- Novartis Investigative Site
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Utrecht, Nederland, 3584 CX
- Aktiv, ikke rekrutterende
- Novartis Investigative Site
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Madrid, Spania, 28046
- Rekruttering
- Novartis Investigative Site
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Valencia, Spania, 46026
- Rekruttering
- Novartis Investigative Site
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Barcelona
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Esplugues, Barcelona, Spania, 08950
- Rekruttering
- Novartis Investigative Site
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Fatih
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Istanbul, Fatih, Tyrkia (Türkiye), 34098
- Rekruttering
- Novartis Investigative Site
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Sihhiye Altindag
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Ankara, Sihhiye Altindag, Tyrkia (Türkiye), 06230
- Rekruttering
- Novartis Investigative Site
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Heidelberg, Tyskland, 69120
- Rekruttering
- Novartis Investigative Site
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Münster, Tyskland, 48149
- Rekruttering
- Novartis Investigative Site
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North Rhine-Westphalia
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Sankt Augustin, North Rhine-Westphalia, Tyskland, 53757
- Rekruttering
- Novartis Investigative Site
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Deltakelseskriterier
Kvalifikasjonskriterier
Alder som er kvalifisert for studier
Tar imot friske frivillige
Kjønn som er kvalifisert for studier
Beskrivelse
Inclusion Criteria:
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Age ≥ 1 with a diagnosis of Still's Disease
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Active diseases defined as:
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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
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Intolerance or inadequate response to available biologic therapy
Exclusion Criteria:
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Patients out of weight range
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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
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Glucocorticoid dose exceeding a set limit
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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
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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
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Live vaccinations within a set time prior to MAS825 treatment. Live vaccines are prohibited up to several months following the last dose
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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
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History of hypersensitivity to any of the study drugs or to drugs of similar chemical classes or to any of the excipients
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Pregnant or breastfeeding women
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Women of child-bearing potential who do not agree to comply with required contraceptive use
Other protocol-defined inclusion/exclusion criteria may apply.
Studieplan
Hvordan er studiet utformet?
Designdetaljer
- Primært formål: Behandling
- Tildeling: N/A
- Intervensjonsmodell: Enkeltgruppeoppdrag
- Masking: Ingen (Open Label)
Våpen og intervensjoner
Deltakergruppe / Arm |
Intervensjon / Behandling |
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Eksperimentell: MAS825
Experimental drug |
Experimental drug
Andre navn:
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Hva måler studien?
Primære resultatmål
Resultatmål |
Tiltaksbeskrivelse |
Tidsramme |
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Number of participants with clinical response based on one set of response criteria
Tidsramme: Day 85
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Established measure for efficacy of treatments in the rheumatologic conditions taking into account self-assessment, function and survival. |
Day 85
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Sekundære resultatmål
Resultatmål |
Tiltaksbeskrivelse |
Tidsramme |
|---|---|---|
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Number of participants with clinical response based on another set of response criteria
Tidsramme: Day 85
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Established measure for efficacy of treatments in the rheumatologic conditions taking into account self-assessment, function and survival. |
Day 85
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Response based on a biomarker level
Tidsramme: Baseline, Day 85
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To evaluate the efficacy of MAS825 in reducing clinical disease activity |
Baseline, Day 85
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Change in physician assessment based on laboratory features of MAS825
Tidsramme: Baseline, 15 months
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To assess the effect of MAS825 on macrophage activation syndrome |
Baseline, 15 months
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Change from baseline in Glucocorticoid (GC) dose
Tidsramme: Baseline, Day 85, Month 6, Month 12
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To evaluate the efficacy of MAS825 as a glucocorticoid-sparing agent |
Baseline, Day 85, Month 6, Month 12
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Change from baseline in patient/parent assessment of physical function as assessed by Quality of Life instrument
Tidsramme: Baseline, Day 85
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To evaluate the efficacy of MAS825 in improving participant's quality of life |
Baseline, Day 85
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Clinically inactive disease whilst on non-treatment dose corticosteroid at any time during the study
Tidsramme: 15 months
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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. |
15 months
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Samarbeidspartnere og etterforskere
Sponsor
Studierekorddatoer
Studer hoveddatoer
Studiestart (Faktiske)
Primær fullføring (Antatt)
Studiet fullført (Antatt)
Datoer for studieregistrering
Først innsendt
Først innsendt som oppfylte QC-kriteriene
Først lagt ut (Faktiske)
Oppdateringer av studieposter
Sist oppdatering lagt ut (Faktiske)
Siste oppdatering sendt inn som oppfylte QC-kriteriene
Sist bekreftet
Mer informasjon
Begreper knyttet til denne studien
Nøkkelord
Ytterligere relevante MeSH-vilkår
Andre studie-ID-numre
- CMAS825J12201
- 2024-520208-25-00 (Annen identifikator: EU CT Number)
Plan for individuelle deltakerdata (IPD)
Planlegger du å dele individuelle deltakerdata (IPD)?
IPD-planbeskrivelse
Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com.
Legemiddel- og utstyrsinformasjon, studiedokumenter
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