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.
Studieoversikt
Status
Intervensjon / Behandling
Studietype
Registrering (Antatt)
Fase
- Fase 3
Kontakter og plasseringer
Studiekontakt
- Navn: Reference Study ID Number: WN41733 https://forpatients.roche.com/ No attachments to email below.
- Telefonnummer: 888-662-6728 (U.S.)
- E-post: global-roche-genentech-trials@gene.com
Studer Kontakt Backup
- Navn: Fastest response: use the inquiry form. https://www.gene.com/contact-us/submit-medical-inquiry
- E-post: global.medical_information@roche.com
Studiesteder
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Ciudad Autonoma Buenos Aires, Argentina, C1249ABN
- Rekruttering
- Hospital de Pediatría S.A.M.I.C.- Prof. Dr. Juan P. Garrahan
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Córdoba, Argentina, X5004FHP
- Rekruttering
- Clinica Universitaria Reina Fabiola
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Colorado
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Denver, Colorado, Forente stater, 80218-1007
- Rekruttering
- Children's Hospital Colorado.
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Val-de-Marne
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Le Kremlin-Bicêtre, Val-de-Marne, Frankrike, 94275
- Rekruttering
- Centre Hospitalier Universitaire de Bicêtre
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Lazio
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Rome, Lazio, Italia, 00165
- Rekruttering
- IRCCS Ospedale Pediatrico Bambino Gesù - INCIPIT - PIN
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Lombardy
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Pavia, Lombardy, Italia, 27100
- Rekruttering
- Fondazione Istituto Neurologico Mondino IRCCS
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Guangdong
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Guangzhou, Guangdong, Kina, 510120
- Rekruttering
- Guangzhou Women and Children's Medical Center
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Shanghai Municipality
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Shanghai, Shanghai Municipality, Kina, 201102
- Rekruttering
- Children's Hospital of Fudan University
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Mexico CITY (federal District)
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DF, Mexico CITY (federal District), Mexico
- Rekruttering
- Grupo Medico Camino
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Mexico City, Mexico CITY (federal District), Mexico, 06720
- Rekruttering
- Hospital Infantil de Mexico Federico Gomez
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Gdansk, Polen, 80-214
- Aktiv, ikke rekrutterende
- Uniwersyteckie Centrum Kliniczne
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Warsaw, Polen, 04-730
- Rekruttering
- Instytut "Pomnik - Centrum Zdrowia Dziecka"
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London, Storbritannia, WC1N 3JH
- Rekruttering
- Great Ormond Street Hospital for Children
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Kocaeli, Tyrkia (Türkiye), 6810
- Rekruttering
- Kocaeli University Research and Application Hospit
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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 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
Studieplan
Hvordan er studiet utformet?
Designdetaljer
- Primært formål: Behandling
- Tildeling: Ikke-randomisert
- Intervensjonsmodell: Parallell tildeling
- Masking: Ingen (Open Label)
Våpen og intervensjoner
Deltakergruppe / Arm |
Intervensjon / Behandling |
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Eksperimentell: 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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Eksperimentell: 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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Eksperimentell: 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. |
Hva måler studien?
Primære resultatmål
Resultatmål |
Tidsramme |
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Summary of Observed Serum Concentration of Satralizumab
Tidsramme: Week 48
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Week 48
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Apparent Clearance [CL/F] of Satralizumab
Tidsramme: Week 48
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Week 48
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Apparent Volume of Distribution [V/F] of Satralizumab
Tidsramme: Week 48
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Week 48
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Area Under the Concentration-time Curve [AUC] of Satralizumab
Tidsramme: Week 48
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Week 48
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Sekundære resultatmål
Resultatmål |
Tidsramme |
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Endring fra baseline i Expanded Disability Status Scale (EDSS) i uke 24 og 48
Tidsramme: Baseline, uke 24, uke 48
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Baseline, uke 24, uke 48
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Proportion of Relapse-free Participants by Week 48
Tidsramme: 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
Tidsramme: 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
Tidsramme: Week 48
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Week 48
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Time to Relapse Requiring Rescue Therapy
Tidsramme: Week 48
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Week 48
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Change from Baseline in Visual Acuity at Weeks 24 and 48
Tidsramme: 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
Tidsramme: 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
Tidsramme: Baseline, Week 24, Week 48
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Baseline, Week 24, Week 48
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Incidence and Severity of Adverse Events
Tidsramme: Week 48
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Week 48
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Samarbeidspartnere og etterforskere
Sponsor
Etterforskere
- Studieleder: Clinical Trials, Hoffmann-La Roche
Publikasjoner og nyttige lenker
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
Ytterligere relevante MeSH-vilkår
- Sykdommer i nervesystemet
- Autoimmune sykdommer
- Sykdommer i immunsystemet
- Øyesykdommer
- Demyeliniserende autoimmune sykdommer, CNS
- Autoimmune sykdommer i nervesystemet
- Demyeliniserende sykdommer
- Myelitt, tverrgående
- Optisk nevritt
- Synsnervesykdommer
- Sykdommer i kranienerve
- Neuromyelitt Optica
- Satralizumab
Andre studie-ID-numre
- WN41733
- 2019-004092-39 (EudraCT-nummer)
- 2023-507817-85-00 (Ctis)
- 2023 (U.S. NIH-stipend/kontrakt)
- 2019 (U.S. NIH-stipend/kontrakt)
Plan for individuelle deltakerdata (IPD)
Planlegger du å dele individuelle deltakerdata (IPD)?
IPD-planbeskrivelse
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
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