Print

Purpose

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.

Conditions

Eligibility

Eligible Ages
Between 2 Years and 11 Years
Eligible Sex
All
Accepts Healthy Volunteers
No

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

Study Design

Phase
Phase 3
Study Type
Interventional
Allocation
Non-Randomized
Intervention Model
Parallel Assignment
Primary Purpose
Treatment
Masking
None (Open Label)

Arm Groups

ArmDescriptionAssigned Intervention
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
  • Drug: Satralizumab
    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.
Experimental
Cohort 2 Participants with body weight ≥20kg to <40kg 		Satralizumab will be administered SC at Weeks 0, 2, 4, and Q4W thereafter.
  • Drug: Satralizumab
    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.
Experimental
Cohort 3 Participants with body weight ≥40kg 		Satralizumab will be administered SC at Weeks 0, 2, 4, and Q4W thereafter.
  • Drug: Satralizumab
    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.

Recruiting Locations

Children's Hospital Colorado.
Denver, Colorado 80218-1007

More Details

Status
Recruiting
Sponsor
Hoffmann-La Roche

Study Contact

Reference Study ID Number: WN41733 https://forpatients.roche.com/
888-662-6728 (U.S.)
global-roche-genentech-trials@gene.com

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.