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)

Neuromyelitis Optica Spectrum Disorder Clinical Trial

— SAkuraSun  

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)

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05199688
• Other study ID #: WN41733
• Secondary ID: 2019-004092-39
• Status: Recruiting
• Phase: Phase 3
• Start date: June 28, 2024
• Est. completion date: September 12, 2029

Study information

• Verified date: April 2024
• Source: Hoffmann-La Roche
• Contact: Reference Study ID Number: WN41733 https://forpatients.roche.com
• Phone: 888-662-6728 (U.S.)
• Email: global-roche-genentech-trials[@]gene.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 8
• Est. completion date: September 12, 2029
• Est. primary completion date: March 31, 2027
• Accepts healthy volunteers: No
• Gender: All
• Age group: 2 Years to 11 Years

Eligibility

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

Related Conditions & MeSH terms

• Neuromyelitis Optica
• Neuromyelitis Optica Spectrum Disorder

Intervention

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.

Locations

Country	Name	City	State
Argentina	Hospital de Pediatría S.A.M.I.C.- Prof. Dr. Juan P. Garrahan	Ciudad Autonoma Buenos Aires
Argentina	Clinica Universitaria Reina Fabiola	Cordoba
France	Centre Hospitalier Universitaire de Bicêtre	Le Kremlin-bicêtre
Italy	Fondazione Istituto Neurologico Mondino IRCCS	Pavia	Lombardia
Italy	IRCCS Ospedale Pediatrico Bambino Gesù - INCIPIT - PIN	Roma	Lazio
Poland	Uniwersyteckie Centrum Kliniczne	Gdansk
United Kingdom	Great Ormond Street Hospital for Children	London
United States	Children's Hospital Colorado.	Denver	Colorado

Sponsors (1)

Lead Sponsor	Collaborator
Hoffmann-La Roche

Countries where clinical trial is conducted

United States,  Argentina,  France,  Italy,  Poland,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Summary of observed serum concentration [Cthrough] of satralizumab		Week 48
Primary	Apparent clearance [CL/F] of satralizumab		Week 48
Primary	Apparent volume of distribution [V/F] of satralizumab		Week 48
Primary	Area under the concentration-time curve [AUC] of satralizumab		Week 48
Secondary	Proportion of relapse-free patients by Week 48		Week 48
Secondary	Annualized relapse rate (ARR), defined as the average number of relapses for each year of the study		Week 48
Secondary	Time to first relapse (TFR) after randomization, defined as the time from randomization until the first occurrence of relapse, as determined by the investigator		Week 48
Secondary	Time to relapse requiring rescue therapy		Week 48
Secondary	Change from baseline in Expanded Disability Status Scale (EDSS) at Weeks 24 and 48		Baseline, Week 24, Week 48
Secondary	Change from baseline in visual acuity at Weeks 24 and 48		Baseline, Week 24, Week 48
Secondary	Change from baseline in FACES Pain Rating Scale at Weeks 24 and 48		Baseline, Week 24, Week 48
Secondary	Change from baseline in EuroQol 5-Dimension, Youth (EQ-5D-Y) score and its proxy at Weeks 24 and 48		Baseline, Week 24, Week 48
Secondary	Incidence and severity of adverse events		Week 48