Uno studio di fase II per valutare l'efficacia clinica, la sicurezza e la tollerabilità di MAS825 nei partecipanti pediatrici e adulti con malattia di Still
Uno studio di fase II in aperto per valutare l'efficacia clinica, la sicurezza e la tollerabilità di MAS825 in partecipanti pediatrici e adulti con malattia di Still
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
Panoramica dello studio
Descrizione dettagliata
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.
Tipo di studio
Iscrizione (Stimato)
Fase
- Fase 2
Contatti e Sedi
Contatto studio
- Nome: Novartis Pharmaceuticals
- Numero di telefono: 1-888-669-6682
- Email: novartis.email@novartis.com
Backup dei contatti dello studio
- Nome: Novartis Pharmaceuticals
- Numero di telefono: +41613241111
Luoghi di studio
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Ontario
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Toronto, Ontario, Canada, M5G 1X8
- Reclutamento
- Novartis Investigative Site
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Quebec
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Montreal, Quebec, Canada, H4A 3J1
- Ritirato
- Novartis Investigative Site
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Montreal, Quebec, Canada, H3T 1C5
- Ritirato
- Novartis Investigative Site
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Bordeaux, Francia, 33076
- Reclutamento
- Novartis Investigative Site
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Bron, Francia, 69677
- Reclutamento
- Novartis Investigative Site
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Le Kremlin-Bicêtre, Francia, 94275
- Reclutamento
- Novartis Investigative Site
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Lille, Francia, 59037
- Reclutamento
- Novartis Investigative Site
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Paris, Francia, 75015
- Reclutamento
- Novartis Investigative Site
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Paris, Francia, 75970
- Reclutamento
- Novartis Investigative Site
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Heidelberg, Germania, 69120
- Reclutamento
- Novartis Investigative Site
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Münster, Germania, 48149
- Reclutamento
- Novartis Investigative Site
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North Rhine-Westphalia
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Sankt Augustin, North Rhine-Westphalia, Germania, 53757
- Reclutamento
- Novartis Investigative Site
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FI
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Florence, FI, Italia, 50139
- Reclutamento
- Novartis Investigative Site
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GE
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Genova, GE, Italia, 16147
- Reclutamento
- Novartis Investigative Site
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RM
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Roma, RM, Italia, 00165
- Reclutamento
- Novartis Investigative Site
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Utrecht, Olanda, 3584 CX
- Attivo, non reclutante
- Novartis Investigative Site
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Madrid, Spagna, 28046
- Reclutamento
- Novartis Investigative Site
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Valencia, Spagna, 46026
- Reclutamento
- Novartis Investigative Site
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Barcelona
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Esplugues, Barcelona, Spagna, 08950
- Reclutamento
- Novartis Investigative Site
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Ohio
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Cincinnati, Ohio, Stati Uniti, 45229
- Attivo, non reclutante
- Cincinnati Childrens Hospital
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Oregon
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Portland, Oregon, Stati Uniti, 97232
- Reclutamento
- Legacy Emanuel Research Hosp Portland
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Investigatore principale:
- Daniel Joseph Kingsbury
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Contatto:
- Brenna Bogle
- Numero di telefono: +1 503 413 5447
- Email: bbogle@lhs.org
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Utah
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Salt Lake City, Utah, Stati Uniti, 84113
- Reclutamento
- University of Utah
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Contatto:
- Tressie Rollins
- Numero di telefono: +1 415 515 8212
- Email: tressie.rollins@hsc.utah.edu
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Investigatore principale:
- Aimee Ogden Hersh
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Fatih
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Istanbul, Fatih, Turchia (Türkiye), 34098
- Reclutamento
- Novartis Investigative Site
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Sihhiye Altindag
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Ankara, Sihhiye Altindag, Turchia (Türkiye), 06230
- Reclutamento
- Novartis Investigative Site
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Criteri di partecipazione
Criteri di ammissibilità
Età idonea allo studio
Accetta volontari sani
Sessi ammissibili allo studio
Descrizione
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.
Piano di studio
Come è strutturato lo studio?
Dettagli di progettazione
- Scopo principale: Trattamento
- Assegnazione: N / A
- Modello interventistico: Assegnazione di gruppo singolo
- Mascheramento: Nessuno (etichetta aperta)
Armi e interventi
Gruppo di partecipanti / Arm |
Intervento / Trattamento |
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Sperimentale: MAS825
Experimental drug |
Experimental drug
Altri nomi:
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Cosa sta misurando lo studio?
Misure di risultato primarie
Misura del risultato |
Misura Descrizione |
Lasso di tempo |
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Number of participants with clinical response based on one set of response criteria
Lasso di tempo: 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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Misure di risultato secondarie
Misura del risultato |
Misura Descrizione |
Lasso di tempo |
|---|---|---|
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Number of participants with clinical response based on another set of response criteria
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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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Collaboratori e investigatori
Sponsor
Studiare le date dei record
Studia le date principali
Inizio studio (Effettivo)
Completamento primario (Stimato)
Completamento dello studio (Stimato)
Date di iscrizione allo studio
Primo inviato
Primo inviato che soddisfa i criteri di controllo qualità
Primo Inserito (Effettivo)
Aggiornamenti dei record di studio
Ultimo aggiornamento pubblicato (Effettivo)
Ultimo aggiornamento inviato che soddisfa i criteri QC
Ultimo verificato
Maggiori informazioni
Termini relativi a questo studio
Parole chiave
Termini MeSH pertinenti aggiuntivi
Altri numeri di identificazione dello studio
- CMAS825J12201
- 2024-520208-25-00 (Altro identificatore: EU CT Number)
Piano per i dati dei singoli partecipanti (IPD)
Hai intenzione di condividere i dati dei singoli partecipanti (IPD)?
Descrizione del piano IPD
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.
Informazioni su farmaci e dispositivi, documenti di studio
Studia un prodotto farmaceutico regolamentato dalla FDA degli Stati Uniti
Studia un dispositivo regolamentato dalla FDA degli Stati Uniti
Queste informazioni sono state recuperate direttamente dal sito web clinicaltrials.gov senza alcuna modifica. In caso di richieste di modifica, rimozione o aggiornamento dei dettagli dello studio, contattare register@clinicaltrials.gov. Non appena verrà implementata una modifica su clinicaltrials.gov, questa verrà aggiornata automaticamente anche sul nostro sito web .