Clinical trial NCT05199688: 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)
Official title: 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)
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE3
Conditions: Neuromyelitis Optica Spectrum Disorder
NMOSD
Lead sponsor: Hoffmann-La Roche
Sponsor class: INDUSTRY
First posted: 2022-01-20
Enrollment: 8
Sex: ALL
Minimum age (years): 2
Maximum age (years): 11
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: 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.


Eligibility 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

Primary outcomes:
measure: Summary of observed serum concentration [Cthrough] of satralizumab
timeFrame: Week 48
measure: Apparent clearance [CL/F] of satralizumab
timeFrame: Week 48
measure: Apparent volume of distribution [V/F] of satralizumab
timeFrame: Week 48
measure: Area under the concentration-time curve [AUC] of satralizumab
timeFrame: Week 48
Secondary outcomes:
measure: Proportion of relapse-free patients by Week 48
timeFrame: Week 48
measure: Annualized relapse rate (ARR), defined as the average number of relapses for each year of the study
timeFrame: Week 48
measure: Time to first relapse (TFR) after randomization, defined as the time from randomization until the first occurrence of relapse, as determined by the investigator
timeFrame: Week 48
measure: Time to relapse requiring rescue therapy
timeFrame: Week 48
measure: Change from baseline in Expanded Disability Status Scale (EDSS) at Weeks 24 and 48
timeFrame: Baseline, Week 24, Week 48
measure: Change from baseline in visual acuity at Weeks 24 and 48
timeFrame: Baseline, Week 24, Week 48
measure: Change from baseline in FACES Pain Rating Scale at Weeks 24 and 48
timeFrame: Baseline, Week 24, Week 48
measure: Change from baseline in EuroQol 5-Dimension, Youth (EQ-5D-Y) score and its proxy at Weeks 24 and 48
timeFrame: Baseline, Week 24, Week 48
measure: Incidence and severity of adverse events
timeFrame: Week 48
Other outcomes:
Locations:
city: Denver
country: United States
facility: Children's Hospital Colorado.
state: Colorado
city: Ciudad Autonoma Buenos Aires
country: Argentina
facility: Hospital de Pediatría S.A.M.I.C.- Prof. Dr. Juan P. Garrahan
city: Cordoba
country: Argentina
facility: Clinica Universitaria Reina Fabiola
city: Le Kremlin-bicêtre
country: France
facility: Centre Hospitalier Universitaire de Bicêtre
city: Roma
country: Italy
facility: IRCCS Ospedale Pediatrico Bambino Gesù - INCIPIT - PIN
state: Lazio
city: Pavia
country: Italy
facility: Fondazione Istituto Neurologico Mondino IRCCS
state: Lombardia
city: Gda?sk
country: Poland
facility: Uniwersyteckie Centrum Kliniczne
city: London
country: United Kingdom
facility: Great Ormond Street Hospital for Children
Study officials:
affiliation: Hoffmann-La Roche
name: Clinical Trials
role: STUDY_DIRECTOR
References: