A Study to Evaluate Pharmacokinetics, Efficacy, Safety, Tolerability, and Pharmacodynamics of Satralizumab in Pediatric Patients With Aquaporin-4 Antibody Positive Neuromyelitis Optica Spectrum Disorder (NMOSD) (SAkuraSun)
A Phase III, Multicenter, Open-Label, Uncontrolled Study to Evaluate Pharmacokinetics, Efficacy, Safety, Tolerability, and Pharmacodynamics of Satralizumab in Pediatric Patients With AQP4 Antibody Positive Neuromyelitis Optica Spectrum Disorder (NMOSD)
This study will primarily evaluate the pharmacokinetics of satralizumab in pediatric patients aged 2-11 years with anti-aquaporin-4 (AQP4) antibody seropositive neuromyelitis optica spectrum disorder (NMOSD). Efficacy, safety, tolerability, and pharmacodynamics will be evaluated in a descriptive manner, given the small number of patients who will be enrolled in this study.
Studienübersicht
Status
Bedingungen
Intervention / Behandlung
Studientyp
Einschreibung (Geschätzt)
Phase
- Phase 3
Kontakte und Standorte
Studienkontakt
- Name: Reference Study ID Number: WN41733 https://forpatients.roche.com/ No attachments to email below.
- Telefonnummer: 888-662-6728 (U.S.)
- E-Mail: global-roche-genentech-trials@gene.com
Studieren Sie die Kontaktsicherung
- Name: Fastest response: use the inquiry form. https://www.gene.com/contact-us/submit-medical-inquiry
- E-Mail: global.medical_information@roche.com
Studienorte
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Ciudad Autonoma Buenos Aires, Argentinien, C1249ABN
- Rekrutierung
- Hospital de Pediatría S.A.M.I.C.- Prof. Dr. Juan P. Garrahan
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Córdoba, Argentinien, X5004FHP
- Rekrutierung
- Clinica Universitaria Reina Fabiola
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Guangdong
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Guangzhou, Guangdong, China, 510120
- Rekrutierung
- Guangzhou Women and Children's Medical Center
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Shanghai Municipality
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Shanghai, Shanghai Municipality, China, 201102
- Rekrutierung
- Children's Hospital of Fudan University
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Val-de-Marne
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Le Kremlin-Bicêtre, Val-de-Marne, Frankreich, 94275
- Rekrutierung
- Centre Hospitalier Universitaire de Bicêtre
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Lazio
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Rome, Lazio, Italien, 00165
- Rekrutierung
- IRCCS Ospedale Pediatrico Bambino Gesù - INCIPIT - PIN
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Lombardy
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Pavia, Lombardy, Italien, 27100
- Rekrutierung
- Fondazione Istituto Neurologico Mondino IRCCS
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Mexico CITY (federal District)
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DF, Mexico CITY (federal District), Mexiko
- Rekrutierung
- Grupo Medico Camino
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Mexico City, Mexico CITY (federal District), Mexiko, 06720
- Rekrutierung
- Hospital Infantil de Mexico Federico Gomez
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Gdansk, Polen, 80-214
- Aktiv, nicht rekrutierend
- Uniwersyteckie Centrum Kliniczne
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Warsaw, Polen, 04-730
- Rekrutierung
- Instytut "Pomnik - Centrum Zdrowia Dziecka"
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Kocaeli, Türkei (türkiye), 6810
- Rekrutierung
- Kocaeli University Research and Application Hospit
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Colorado
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Denver, Colorado, Vereinigte Staaten, 80218-1007
- Rekrutierung
- Children's Hospital Colorado.
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London, Vereinigtes Königreich, WC1N 3JH
- Rekrutierung
- Great Ormond Street Hospital for Children
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Teilnahmekriterien
Zulassungskriterien
Studienberechtigtes Alter
Akzeptiert gesunde Freiwillige
Studienberechtigte Geschlechter
Beschreibung
Inclusion Criteria:
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Age at screening 2-11 years, inclusive
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Body weight at screening >=10 kg
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For female patients of childbearing potential (postmenarchal): agreement to either remain completely abstinent (refrain from heterosexual intercourse) or to use a reliable means of contraception
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Diagnosed as having NMOSD with AQP4 antibody seropositive status as defined by the Wingerchuk 2015 criteria Clinical evidence of at least one documented attack (including first attack) in the last year prior to screening:
- Documented history of a positive anti-AQP4 antibody test or a positive anti-AQP4 antibody test at screening;
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Neurological stability for >=30 days prior to baseline
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Expanded Disability Status Scale (EDSS) 0 to 6.5
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For participants receiving a baseline immunosuppressant treatment and planning to continue on these therapies, treatment must be at stable dose for 4 weeks prior to baseline
Exclusion Criteria:
- Pregnancy or lactation
- Evidence of other demyelinating disease mimicking NMOSD
- Active or presence of recurrent bacterial, viral, fungal, mycobacterial infection, or other infection at baseline
- Evidence of chronic active hepatitis B or C
- Evidence of untreated latent or active tuberculosis (TB)
- Receipt of a live or live-attenuated vaccine within 6 weeks prior to baseline
- History of severe allergic reaction to a biologic agent
- IVIg or subcutaneous Ig (SCIg) within 4 weeks prior to enrollment/Day 1
Studienplan
Wie ist die Studie aufgebaut?
Designdetails
- Hauptzweck: Behandlung
- Zuteilung: Nicht randomisiert
- Interventionsmodell: Parallele Zuordnung
- Maskierung: Keine (Offenes Etikett)
Waffen und Interventionen
Teilnehmergruppe / Arm |
Intervention / Behandlung |
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Experimental: Cohort 1: Participants with body weight ≥10kg to <20kg
Satralizumab will be administered SC Q6W in a cohort of at least 2 evaluable patients |
Participants will receive satralizumab treatment for a minimum of 48 weeks and then will have the opportunity to enter an optional satralizumab extension (OSE) period. |
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Experimental: Cohort 2 Participants with body weight ≥20kg to <40kg
Satralizumab will be administered SC at Weeks 0, 2, 4, and Q4W thereafter. |
Participants will receive satralizumab treatment for a minimum of 48 weeks and then will have the opportunity to enter an optional satralizumab extension (OSE) period. |
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Experimental: Cohort 3 Participants with body weight ≥40kg
Satralizumab will be administered SC at Weeks 0, 2, 4, and Q4W thereafter. |
Participants will receive satralizumab treatment for a minimum of 48 weeks and then will have the opportunity to enter an optional satralizumab extension (OSE) period. |
Was misst die Studie?
Primäre Ergebnismessungen
Ergebnis Maßnahme |
Zeitfenster |
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Summary of Observed Serum Concentration of Satralizumab
Zeitfenster: Week 48
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Week 48
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Apparent Clearance [CL/F] of Satralizumab
Zeitfenster: Week 48
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Week 48
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Apparent Volume of Distribution [V/F] of Satralizumab
Zeitfenster: Week 48
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Week 48
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Area Under the Concentration-time Curve [AUC] of Satralizumab
Zeitfenster: Week 48
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Week 48
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Sekundäre Ergebnismessungen
Ergebnis Maßnahme |
Zeitfenster |
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Veränderung gegenüber dem Ausgangswert in der Expanded Disability Status Scale (EDSS) in den Wochen 24 und 48
Zeitfenster: Baseline, Woche 24, Woche 48
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Baseline, Woche 24, Woche 48
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Proportion of Relapse-free Participants by Week 48
Zeitfenster: Week 48
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Week 48
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Annualized Relapse Rate (ARR), defined as the average number of relapses for each year of the study
Zeitfenster: Week 48
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Week 48
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Time to First Relapse (TFR) after enrollment, defined as the time from enrollment until the first occurrence of relapse, as determined by the investigator
Zeitfenster: Week 48
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Week 48
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Time to Relapse Requiring Rescue Therapy
Zeitfenster: Week 48
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Week 48
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Change from Baseline in Visual Acuity at Weeks 24 and 48
Zeitfenster: Baseline, Week 24, Week 48
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Baseline, Week 24, Week 48
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Change from Baseline in FACES Pain Rating Scale at Weeks 24 and 48
Zeitfenster: Baseline, Week 24, Week 48
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Baseline, Week 24, Week 48
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Change from Baseline in EuroQol 5-Dimension, Youth (EQ-5D-Y) Score and its Proxy at Weeks 24 and 48
Zeitfenster: Baseline, Week 24, Week 48
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Baseline, Week 24, Week 48
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Incidence and Severity of Adverse Events
Zeitfenster: Week 48
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Week 48
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Mitarbeiter und Ermittler
Sponsor
Ermittler
- Studienleiter: Clinical Trials, Hoffmann-La Roche
Publikationen und hilfreiche Links
Studienaufzeichnungsdaten
Haupttermine studieren
Studienbeginn (Tatsächlich)
Primärer Abschluss (Geschätzt)
Studienabschluss (Geschätzt)
Studienanmeldedaten
Zuerst eingereicht
Zuerst eingereicht, das die QC-Kriterien erfüllt hat
Zuerst gepostet (Tatsächlich)
Studienaufzeichnungsaktualisierungen
Letztes Update gepostet (Tatsächlich)
Letztes eingereichtes Update, das die QC-Kriterien erfüllt
Zuletzt verifiziert
Mehr Informationen
Begriffe im Zusammenhang mit dieser Studie
Zusätzliche relevante MeSH-Bedingungen
- Erkrankungen des Nervensystems
- Autoimmunerkrankungen
- Erkrankungen des Immunsystems
- Augenkrankheiten
- Demyelinisierende Autoimmunerkrankungen, ZNS
- Autoimmunerkrankungen des Nervensystems
- Demyelinisierende Krankheiten
- Myelitis, quer
- Optikusneuritis
- Sehnervenerkrankungen
- Erkrankungen der Hirnnerven
- Neuromyelitis optica
- satralizumab
Andere Studien-ID-Nummern
- WN41733
- 2019-004092-39 (EudraCT-Nummer)
- 2023-507817-85-00 (Ctis)
- 2023 (US NIH Stipendium/Vertrag)
- 2019 (US NIH Stipendium/Vertrag)
Plan für individuelle Teilnehmerdaten (IPD)
Planen Sie, individuelle Teilnehmerdaten (IPD) zu teilen?
Beschreibung des IPD-Plans
For eligible studies, qualified researchers may request access to individual patient level clinical data. See Roche's commitment to transparency of clinical study information here: https://go.roche.com/data_sharing
Arzneimittel- und Geräteinformationen, Studienunterlagen
Studiert ein von der US-amerikanischen FDA reguliertes Arzneimittelprodukt
Studiert ein von der US-amerikanischen FDA reguliertes Geräteprodukt
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