Trial Review

The ANZCTR website is back online for trial registration and updates. We apologise for any inconvenience caused while the site was inactive.



Reset your password and enable multi-factor authentication (MFA)


For ANZCTR account holders: to help ensure the cyber safety of your account, you’ll need to reset your password and set-up multi-factor authentication (MFA) as per the instructions below.


  1. Go to the Login page, click ‘reset password’ and follow the instructions.
  2. Check your email for the link to set a new password.
  3. Create a new password that meets requirements. New passwords must include at least one lowercase letter, one uppercase letter, one number and one special character (e.g. !#$%&@).
  4. Return to the Login page and enter your new password. A verification code will be sent to your email.
  5. Check your email for the code and enter it on the Login page. If the code is entered incorrectly, you can re-enter the correct one or request a new one.

Learn more about MFA and its importance on the Australian Signals Directorate website.

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this information for consumers
Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT04963270




Registration number
NCT04963270
Ethics application status
Date submitted
7/07/2021
Date registered
15/07/2021
Date last updated
28/02/2025

Titles & IDs
Public title
A Study To Evaluate Efficacy, Safety, Pharmacokinetics, And Pharmacodynamics Of Satralizumab In Patients With Generalized Myasthenia Gravis
Scientific title
A Phase III, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study To Evaluate Efficacy, Safety, Pharmacokinetics, And Pharmacodynamics Of Satralizumab In Patients With Generalized Myasthenia Gravis
Secondary ID [1] 0 0
WN42636
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Generalized Myasthenia Gravis 0 0
Condition category
Condition code
Inflammatory and Immune System 0 0 0 0
Autoimmune diseases
Neurological 0 0 0 0
Other neurological disorders
Musculoskeletal 0 0 0 0
Other muscular and skeletal disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Satralizumab
Other interventions - Placebo

Experimental: Satralizumab - Participants will receive Satralizumab at Weeks 0, 2, 4, and Q4W thereafter. Adolescent patients who first enter the study in the OLE period will receive satralizumab SC loading doses at Week 0, 2, and 4 in the OLE, followed by maintenance doses Q4W thereafter and will remain on stable background therapy until Week 24 of the OLE.

Placebo comparator: Placebo - Participants will receive placebo at Weeks 0, 2, 4, and Q4W thereafter


Treatment: Drugs: Satralizumab
Satralizumab will be administered as a subcutaneous injection

Other interventions: Placebo
Satralizumab placebo will be administered as a subcutaneous injection
Intervention code [1] 0 0
Treatment: Drugs
Intervention code [2] 0 0
Other interventions
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
DB Period: Mean Change From Baseline in Total Myasthenia Gravis Activities of Daily Living (MG-ADL) Score in the AChR+ Population
Timepoint [1] 0 0
At Week 24
Secondary outcome [1] 0 0
DB Period: Mean Change From Baseline in Total MG-ADL Score in the Overall Population (OP) at Week 24
Timepoint [1] 0 0
At Week 24
Secondary outcome [2] 0 0
DB Period: Percentage of Participants With a = 2-point Reduction From Baseline in Total MG-ADL Score in AChR+ Population at Week 24
Timepoint [2] 0 0
At Week 24
Secondary outcome [3] 0 0
DB Period: Percentage of Participants With a = 2-point Reduction From Baseline in Total MG-ADL Score in OP at Week 24
Timepoint [3] 0 0
At Week 24
Secondary outcome [4] 0 0
DB Period: Mean Change From Baseline in Quantitative Myasthenia Gravis (QMG) Score in AChR+ Population at Week 24
Timepoint [4] 0 0
At Week 24
Secondary outcome [5] 0 0
DB Period: Mean Change From Baseline in QMG Score in OP at Week 24
Timepoint [5] 0 0
At Week 24
Secondary outcome [6] 0 0
DB Period: Mean Change From Baseline in Myasthenia Gravis Quality of Life 15 Scale (Revised) (MG-QOL 15r) Total Score in AChR+ Population at Week 24
Timepoint [6] 0 0
At Week 24
Secondary outcome [7] 0 0
DB Period: Mean Change From Baseline in MG-QOL 15r Total Score in OP at Week 24
Timepoint [7] 0 0
At Week 24
Secondary outcome [8] 0 0
DB Period: Mean Change From Baseline in Quality of Life in Neurological Disorders (Neuro-QoL) Fatigue Subscale Total Score in AChR+ Population at Week 24
Timepoint [8] 0 0
At Week 24
Secondary outcome [9] 0 0
DB Period: Mean Change From Baseline in Neuro-QoL Fatigue Subscale Total Score in OP at Week 24
Timepoint [9] 0 0
At Week 24
Secondary outcome [10] 0 0
DB Period: Percentage of Participants With a = 3-point Reduction From Baseline in QMG Score in AChR+ Population at Week 24
Timepoint [10] 0 0
At Week 24
Secondary outcome [11] 0 0
DB Period: Percentage of Participants With a = 3-point Reduction From Baseline in QMG Score in OP at Week 24
Timepoint [11] 0 0
At Week 24
Secondary outcome [12] 0 0
DB Period: Mean Change From Baseline in Total Myasthenia Gravis Composite (MGC) Score in AChR+ Population at Week 24
Timepoint [12] 0 0
At Week 24
Secondary outcome [13] 0 0
DB Period: Mean Change From Baseline in Total MGC Score in OP at Week 24
Timepoint [13] 0 0
At Week 24
Secondary outcome [14] 0 0
DB Period: Percentage of Participants With a = 3-point Reduction From Baseline in Total MGC Score in AChR+ Population at Week 24
Timepoint [14] 0 0
At Week 24
Secondary outcome [15] 0 0
DB Period: Percentage of Participants With a = 3-point Reduction From Baseline in Total MGC Score in OP at Week 24
Timepoint [15] 0 0
At Week 24
Secondary outcome [16] 0 0
DB Period: Percentage of Participants Who Achieved Minimal Symptom Expression (Total MG-ADL Score of 0 or 1) in AChR+ Population at Week 24
Timepoint [16] 0 0
At Week 24
Secondary outcome [17] 0 0
DB Period: Percentage of Participants Who Achieved Minimal Symptom Expression (Total MG-ADL Score of 0 or 1) in OP at Week 24
Timepoint [17] 0 0
At Week 24
Secondary outcome [18] 0 0
DB Period: Percentage of Participants With at Least One gMG-Related Exacerbation Between Baseline and Week 24 in AChR+ Population
Timepoint [18] 0 0
Baseline up to Week 24
Secondary outcome [19] 0 0
DB Period: Percentage of Participants With at Least One gMG-Related Exacerbation Between Baseline and Week 24 in OP
Timepoint [19] 0 0
Baseline up to Week 24
Secondary outcome [20] 0 0
DB Period: Percentage of Participants in AChR+ Population Receiving Rescue Therapy Between Baseline and Week 24
Timepoint [20] 0 0
Baseline up to Week 24
Secondary outcome [21] 0 0
DB Period: Percentage of Participants in OP Receiving Rescue Therapy Between Baseline and Week 24
Timepoint [21] 0 0
Baseline up to Week 24
Secondary outcome [22] 0 0
DB Period: Duration of Meaningful Improvement, Defined as = 2-Point Reduction From Baseline in Total MG-ADL Score in AChR+ Population
Timepoint [22] 0 0
Baseline, Week 24
Secondary outcome [23] 0 0
DB Period: Duration of Meaningful Improvement, Defined as = 2-Point Reduction From Baseline in Total MG-ADL Score in OP
Timepoint [23] 0 0
Baseline, Week 24
Secondary outcome [24] 0 0
DB Period: Number of Participants With Adverse Events (AEs)
Timepoint [24] 0 0
Day 1 up to approximately 24 weeks
Secondary outcome [25] 0 0
DB Period: Serum Levels of Interleukin-6 (IL-6)
Timepoint [25] 0 0
Baseline, Weeks 2, 4, 8, 12, 16, 20 and 24
Secondary outcome [26] 0 0
DB Period: Serum Levels of Soluble Interleukin-6 Receptors (sIL-6R)
Timepoint [26] 0 0
Baseline, Weeks 2, 4, 8, 12, 16, 20 and 24
Secondary outcome [27] 0 0
Number of Participants With Anti-drug Antibodies (ADAs) to Satralizumab
Timepoint [27] 0 0
Baseline to Week 24
Secondary outcome [28] 0 0
Serum Concentrations of Satralizumab
Timepoint [28] 0 0
Weeks 0, 2, 4, 8, 12, 16, 20 and 24

Eligibility
Key inclusion criteria
* Signed Informed Consent Form
* For adolescent patients: Informed Consent Form for study participation signed by the parents or a legal guardian, and patient assent obtained, as per local requirements
* Ability to comply with the study protocol procedures
* Confirmed diagnosis of gMG (anti-AChR, anti-MuSK or anti-LRP4 present at screening)
* A total MG-ADL score of = 5 points at screening with more than 50% of this score attributed to non-ocular items
* MGFA severity Class II-IV
* Ongoing gMG treatment at a stable dose
* For female patients of childbearing potential: agreement to remain abstinent (refrain from heterosexual intercourse) or use adequate contraception during the treatment period and for at least 3 months after the final dose of satralizumab.
Minimum age
12 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* History of thymectomy within 12 months prior to screening
* Ocular MG (MGFA Class I) and myasthenic crisis (MGFA Class V) within the last 3 months prior to screening
* Known disease other than gMG that would interfere with the course and conduct of the study
* Positive screening tests for hepatitis B virus (HBV) and hepatitis C virus (HCV)
* Evidence of latent or active tuberculosis (excluding patients receiving chemoprophylaxis for latent tuberculosis infection)
* Receipt of live or live attenuated vaccine within 6 weeks prior to baseline
* Pregnant or breastfeeding, or intending to become pregnant during the study or within 3 months after the last dose

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s


The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Stopped early
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
19/10/2021
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
2/09/2024
Sample size
Target
Accrual to date
Final
188
Recruitment in Australia
Recruitment state(s)
NSW
Recruitment hospital [1] 0 0
Concord Repatriation General Hospital - Concord
Recruitment postcode(s) [1] 0 0
2139 - Concord
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
District of Columbia
Country [3] 0 0
United States of America
State/province [3] 0 0
Illinois
Country [4] 0 0
Argentina
State/province [4] 0 0
Caba
Country [5] 0 0
Argentina
State/province [5] 0 0
Ciudad Autonoma Bs As
Country [6] 0 0
Argentina
State/province [6] 0 0
Mendoza
Country [7] 0 0
Argentina
State/province [7] 0 0
Rosario
Country [8] 0 0
Brazil
State/province [8] 0 0
Rio Grande Do Sul
Country [9] 0 0
Brazil
State/province [9] 0 0
São Paulo
Country [10] 0 0
Canada
State/province [10] 0 0
Quebec
Country [11] 0 0
China
State/province [11] 0 0
Beijing
Country [12] 0 0
China
State/province [12] 0 0
Changchun City
Country [13] 0 0
China
State/province [13] 0 0
Chengdu City
Country [14] 0 0
China
State/province [14] 0 0
Guiyang
Country [15] 0 0
China
State/province [15] 0 0
Hangzhou
Country [16] 0 0
China
State/province [16] 0 0
Jinan
Country [17] 0 0
China
State/province [17] 0 0
Shanghai City
Country [18] 0 0
China
State/province [18] 0 0
Shanghai
Country [19] 0 0
China
State/province [19] 0 0
Tianjin
Country [20] 0 0
China
State/province [20] 0 0
Wuhan City
Country [21] 0 0
China
State/province [21] 0 0
Xi'an City
Country [22] 0 0
Denmark
State/province [22] 0 0
Aarhus N
Country [23] 0 0
Denmark
State/province [23] 0 0
København Ø
Country [24] 0 0
France
State/province [24] 0 0
Bordeaux cedex
Country [25] 0 0
France
State/province [25] 0 0
Garches
Country [26] 0 0
France
State/province [26] 0 0
Marseille
Country [27] 0 0
France
State/province [27] 0 0
Nantes
Country [28] 0 0
France
State/province [28] 0 0
Nice
Country [29] 0 0
Germany
State/province [29] 0 0
Bochum
Country [30] 0 0
Germany
State/province [30] 0 0
Essen
Country [31] 0 0
Germany
State/province [31] 0 0
Münster
Country [32] 0 0
Italy
State/province [32] 0 0
Campania
Country [33] 0 0
Italy
State/province [33] 0 0
Lombardia
Country [34] 0 0
Italy
State/province [34] 0 0
Sicilia
Country [35] 0 0
Italy
State/province [35] 0 0
Veneto
Country [36] 0 0
Japan
State/province [36] 0 0
Bunkyo-ku
Country [37] 0 0
Japan
State/province [37] 0 0
Chiba-shi, Chiba
Country [38] 0 0
Japan
State/province [38] 0 0
Hanamaki, Iwate
Country [39] 0 0
Japan
State/province [39] 0 0
Hokkaido
Country [40] 0 0
Japan
State/province [40] 0 0
Kanagawa
Country [41] 0 0
Japan
State/province [41] 0 0
Narita, Chiba
Country [42] 0 0
Japan
State/province [42] 0 0
Osaka
Country [43] 0 0
Japan
State/province [43] 0 0
Shizuoka
Country [44] 0 0
Japan
State/province [44] 0 0
Suita
Country [45] 0 0
Japan
State/province [45] 0 0
Tokyo
Country [46] 0 0
Korea, Republic of
State/province [46] 0 0
Daegu
Country [47] 0 0
Korea, Republic of
State/province [47] 0 0
Gyeongsangnam-do
Country [48] 0 0
Korea, Republic of
State/province [48] 0 0
Seoul
Country [49] 0 0
Poland
State/province [49] 0 0
Gda?sk
Country [50] 0 0
Poland
State/province [50] 0 0
Krakow
Country [51] 0 0
Poland
State/province [51] 0 0
Kraków
Country [52] 0 0
Poland
State/province [52] 0 0
Lublin
Country [53] 0 0
Russian Federation
State/province [53] 0 0
Krasnojarsk
Country [54] 0 0
Russian Federation
State/province [54] 0 0
Novosibirsk
Country [55] 0 0
Spain
State/province [55] 0 0
Barcelona
Country [56] 0 0
Spain
State/province [56] 0 0
Navarra
Country [57] 0 0
Spain
State/province [57] 0 0
Valencia
Country [58] 0 0
Taiwan
State/province [58] 0 0
Taipei
Country [59] 0 0
Taiwan
State/province [59] 0 0
Taoyuan City
Country [60] 0 0
Turkey
State/province [60] 0 0
Ankara
Country [61] 0 0
Turkey
State/province [61] 0 0
Izmir
Country [62] 0 0
Turkey
State/province [62] 0 0
Kocaeli
Country [63] 0 0
Turkey
State/province [63] 0 0
Samsun

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Hoffmann-La Roche
Address
Country
Other collaborator category [1] 0 0
Commercial sector/industry
Name [1] 0 0
Chugai Pharmaceutical
Address [1] 0 0
Country [1] 0 0

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Clinical Trials
Address 0 0
Hoffmann-La Roche
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Reference Study ID Number: GE42063 https://forpatients.roche.com/
Address 0 0
Country 0 0
Phone 0 0
888-662-6728 (U.S.)
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries

No information has been provided regarding IPD availability


What supporting documents are/will be available?




Results publications and other study-related documents

No documents have been uploaded by study researchers.

Results are available at https://clinicaltrials.gov/study/NCT04963270