Evaluating satralizumab for children with neuromyelitis optica spectrum disorder
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)
PHASE3 · Hoffmann-La Roche · NCT05199688
This study is testing a new treatment called satralizumab to see if it helps children aged 2 to 11 with neuromyelitis optica spectrum disorder feel better and stay safe.
Quick facts
| Phase | PHASE3 |
|---|---|
| Study type | Interventional |
| Enrollment | 8 (estimated) |
| Ages | 2 Years to 11 Years |
| Sex | All |
| Sponsor | Hoffmann-La Roche (industry) |
| Drugs / interventions | satralizumab |
| Locations | 13 sites (Denver, Colorado and 12 other locations) |
| Trial ID | NCT05199688 on ClinicalTrials.gov |
What this trial studies
This study evaluates the pharmacokinetics, efficacy, safety, tolerability, and pharmacodynamics of satralizumab in pediatric patients aged 2 to 11 years who are positive for anti-aquaporin-4 antibodies and diagnosed with neuromyelitis optica spectrum disorder (NMOSD). The study aims to gather descriptive data on these parameters, given the limited number of eligible patients. Participants must have experienced at least one documented attack of NMOSD in the past year and demonstrate neurological stability prior to the study. The treatment will be administered while monitoring the patients closely for safety and efficacy outcomes.
Who should consider this trial
Good fit: Ideal candidates are children aged 2-11 years who are diagnosed with AQP4 antibody positive NMOSD and have experienced at least one attack in the past year.
Not a fit: Patients with other demyelinating diseases or those currently pregnant or lactating may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could provide a new treatment option for pediatric patients suffering from NMOSD, potentially improving their quality of life.
How similar studies have performed: While this approach is focused on a specific pediatric population, similar studies in adult populations have shown promise, indicating potential for success in this novel application.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Age at screening 2-11 years, inclusive * Body weight at screening \>=10 kg * 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 * 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 * Neurological stability for \>=30 days prior to both screening and baseline * Expanded Disability Status Scale (EDSS) 0 to 6.5 * For patients 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
Where this trial is running
Denver, Colorado and 12 other locations
- Children's Hospital Colorado. — Denver, Colorado, United States (RECRUITING)
- Hospital de Pediatría S.A.M.I.C.- Prof. Dr. Juan P. Garrahan — Ciudad Autonoma Buenos Aires, Argentina (RECRUITING)
- Clinica Universitaria Reina Fabiola — Córdoba, Argentina (RECRUITING)
- Guangzhou Women and Children's Medical Center — Guangzhou, Guangdong, China (RECRUITING)
- Children's Hospital of Fudan University — Shanghai, Shanghai Municipality, China (RECRUITING)
- Centre Hospitalier Universitaire de Bicêtre — Le Kremlin-Bicêtre, France (RECRUITING)
- IRCCS Ospedale Pediatrico Bambino Gesù - INCIPIT - PIN — Rome, Lazio, Italy (RECRUITING)
- Fondazione Istituto Neurologico Mondino IRCCS — Pavia, Lombardy, Italy (RECRUITING)
- Grupo Medico Camino — DF, Mexico CITY (federal District), Mexico (RECRUITING)
- Uniwersyteckie Centrum Kliniczne — Gda?sk, Poland (ACTIVE_NOT_RECRUITING)
- Instytut "Pomnik - Centrum Zdrowia Dziecka" — Warsaw, Poland (RECRUITING)
- Kocaeli University Research and Application Hospit — Kocaeli, Turkey (Türkiye) (RECRUITING)
- Great Ormond Street Hospital for Children — London, United Kingdom (RECRUITING)
Study contacts
- Study coordinator: Reference Study ID Number: WN41733 https://forpatients.roche.com/
- Email: global-roche-genentech-trials@gene.com
- Phone: 888-662-6728 (U.S.)
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions: Neuromyelitis Optica Spectrum Disorder, NMOSD