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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)

Primary Purpose

Neuromyelitis Optica Spectrum Disorder, NMOSD

Status

Recruiting

Phase

Phase 3

Locations

International

Study Type

Interventional

Intervention

Satralizumab

About this trial

This is an interventional treatment trial for Neuromyelitis Optica Spectrum Disorder

Eligibility Criteria

2 Years - 11 Years (Child)All SexesDoes not accept healthy volunteers

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

Sites / Locations

Children's Hospital Colorado.Recruiting
Hospital de Pediatría S.A.M.I.C.- Prof. Dr. Juan P. GarrahanRecruiting
Clinica Universitaria Reina FabiolaRecruiting
Centre Hospitalier Universitaire de BicêtreRecruiting
IRCCS Ospedale Pediatrico Bambino Gesù - INCIPIT - PINRecruiting
Fondazione Istituto Neurologico Mondino IRCCSRecruiting
Uniwersyteckie Centrum KliniczneRecruiting
Great Ormond Street Hospital for ChildrenRecruiting

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Arm Label

Cohort 1: Participants with body weight ≥10kg to <20kg

Cohort 2 Participants with body weight ≥20kg to <40kg

Cohort 3 Participants with body weight ≥40kg

Arm Description

Satralizumab will be administered SC Q6W in a cohort of at least 2 evaluable patients

Satralizumab will be administered SC at Weeks 0, 2, 4, and Q4W thereafter.

Outcomes

Primary Outcome Measures

Summary of observed serum concentration [Cthrough] of satralizumab

Apparent clearance [CL/F] of satralizumab

Apparent volume of distribution [V/F] of satralizumab

Area under the concentration-time curve [AUC] of satralizumab

Secondary Outcome Measures

Proportion of relapse-free patients by Week 48

Annualized relapse rate (ARR), defined as the average number of relapses for each year of the study

Time to first relapse (TFR) after randomization, defined as the time from randomization until the first occurrence of relapse, as determined by the investigator

Time to relapse requiring rescue therapy

Change from baseline in Expanded Disability Status Scale (EDSS) at Weeks 24 and 48

Change from baseline in visual acuity at Weeks 24 and 48

Change from baseline in FACES Pain Rating Scale at Weeks 24 and 48

Change from baseline in EuroQol 5-Dimension, Youth (EQ-5D-Y) score and its proxy at Weeks 24 and 48

Incidence and severity of adverse events

Full Information

NCT ID

NCT05199688

First Posted

December 3, 2021

Last Updated

October 3, 2023

Sponsor

Hoffmann-La Roche

1. Study Identification

Unique Protocol Identification Number

NCT05199688

Brief Title

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)

Acronym

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)

Study Type

Interventional

2. Study Status

Record Verification Date

October 2023

Overall Recruitment Status

Recruiting

Study Start Date

October 31, 2023 (Anticipated)

Primary Completion Date

September 9, 2024 (Anticipated)

Study Completion Date

February 28, 2027 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Hoffmann-La Roche

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Neuromyelitis Optica Spectrum Disorder, NMOSD

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 3

Interventional Study Model

Parallel Assignment

Masking

None (Open Label)

Allocation

Non-Randomized

Enrollment

8 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

Cohort 1: Participants with body weight ≥10kg to <20kg

Arm Type

Experimental

Arm Description

Satralizumab will be administered SC Q6W in a cohort of at least 2 evaluable patients

Arm Title

Cohort 2 Participants with body weight ≥20kg to <40kg

Arm Type

Experimental

Arm Description

Satralizumab will be administered SC at Weeks 0, 2, 4, and Q4W thereafter.

Arm Title

Cohort 3 Participants with body weight ≥40kg

Arm Type

Experimental

Arm Description

Satralizumab will be administered SC at Weeks 0, 2, 4, and Q4W thereafter.

Intervention Type

Drug

Intervention Name(s)

Satralizumab

Intervention Description

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.

Primary Outcome Measure Information:

Title

Summary of observed serum concentration [Cthrough] of satralizumab

Time Frame

Week 48

Title

Apparent clearance [CL/F] of satralizumab

Time Frame

Week 48

Title

Apparent volume of distribution [V/F] of satralizumab

Time Frame

Week 48

Title

Area under the concentration-time curve [AUC] of satralizumab

Time Frame

Week 48

Secondary Outcome Measure Information:

Title

Proportion of relapse-free patients by Week 48

Time Frame

Week 48

Title

Annualized relapse rate (ARR), defined as the average number of relapses for each year of the study

Time Frame

Week 48

Title

Time to first relapse (TFR) after randomization, defined as the time from randomization until the first occurrence of relapse, as determined by the investigator

Time Frame

Week 48

Title

Time to relapse requiring rescue therapy

Time Frame

Week 48

Title

Change from baseline in Expanded Disability Status Scale (EDSS) at Weeks 24 and 48

Time Frame

Baseline, Week 24, Week 48

Title

Change from baseline in visual acuity at Weeks 24 and 48

Time Frame

Baseline, Week 24, Week 48

Title

Change from baseline in FACES Pain Rating Scale at Weeks 24 and 48

Time Frame

Baseline, Week 24, Week 48

Title

Change from baseline in EuroQol 5-Dimension, Youth (EQ-5D-Y) score and its proxy at Weeks 24 and 48

Time Frame

Baseline, Week 24, Week 48

Title

Incidence and severity of adverse events

Time Frame

Week 48

10. Eligibility

Sex

All

Minimum Age & Unit of Time

2 Years

Maximum Age & Unit of Time

11 Years

Accepts Healthy Volunteers

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

Central Contact Person:

First Name & Middle Initial & Last Name or Official Title & Degree

Reference Study ID Number: WN41733 https://forpatients.roche.com/

Phone

888-662-6728 (U.S.)

global-roche-genentech-trials@gene.com

First Name & Middle Initial & Last Name or Official Title & Degree

Global Medical Information

global.medical_information@roche.com

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Clinical Trials

Organizational Affiliation

Hoffmann-La Roche

Official's Role

Study Director

Facility Information:

Facility Name

Children's Hospital Colorado.

City

Denver

State/Province

Colorado

ZIP/Postal Code

80218-1007

Country

United States

Individual Site Status

Recruiting

Facility Name

Hospital de Pediatría S.A.M.I.C.- Prof. Dr. Juan P. Garrahan

City

Ciudad Autonoma Buenos Aires

ZIP/Postal Code

C1249ABN

Country

Argentina

Individual Site Status

Recruiting

Facility Name

Clinica Universitaria Reina Fabiola

City

Cordoba

ZIP/Postal Code

X5004FHP

Country

Argentina

Individual Site Status

Recruiting

Facility Name

Centre Hospitalier Universitaire de Bicêtre

City

Le Kremlin-bicêtre

ZIP/Postal Code

94275

Country

France

Individual Site Status

Recruiting

Facility Name

IRCCS Ospedale Pediatrico Bambino Gesù - INCIPIT - PIN

City

Roma

State/Province

Lazio

ZIP/Postal Code

00165

Country

Italy

Individual Site Status

Recruiting

Facility Name

Fondazione Istituto Neurologico Mondino IRCCS

City

Pavia

State/Province

Lombardia

ZIP/Postal Code

27100

Country

Italy

Individual Site Status

Recruiting

Facility Name

Uniwersyteckie Centrum Kliniczne

City

Gdansk

Country

Poland

Individual Site Status

Recruiting

Facility Name

Great Ormond Street Hospital for Children

City

London

ZIP/Postal Code

WC1N 3JH

Country

United Kingdom

Individual Site Status

Recruiting

12. IPD Sharing Statement

Plan to Share IPD

Yes

IPD Sharing Plan Description

Qualified researchers may request access to individual patient level data through the clinical study data request platform (www.vivli.org). Further details on Roche's criteria for eligible studies are available here (https://vivli.org/ourmember/roche/). For further details on Roche's Global Policy on the Sharing of Clinical Information and how to request access to related clinical study documents, see here (https://www.roche.com/research_and_development/who_we_are_how_we_work/clinical_trials/our_commitment_to_data_sharing.htm

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