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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
4/10/2024

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
Mean change from baseline in Myasthenia Gravis Activities of Daily Living (MG-ADL) score in AChR-antibody seropositive population
Timepoint [1] 0 0
Week 24
Secondary outcome [1] 0 0
Mean change from baseline in Quantitative Myasthenia Gravis (QMG) score
Timepoint [1] 0 0
Week 24
Secondary outcome [2] 0 0
Mean change from baseline in Myasthenia Gravis Quality of Life 15 Scale (MG-QOL 15r) score
Timepoint [2] 0 0
Week 24
Secondary outcome [3] 0 0
Mean change from baseline in Quality of Life in Neurological Disorders (Neuro-QoL) Fatigue Subscale score
Timepoint [3] 0 0
Week 24
Secondary outcome [4] 0 0
Mean change from baseline in total Myasthenia Gravis Composite (MGC) score
Timepoint [4] 0 0
Week 24
Secondary outcome [5] 0 0
Proportion of MG-ADL responders
Timepoint [5] 0 0
Week 24
Secondary outcome [6] 0 0
Proportion of QMG responder
Timepoint [6] 0 0
Week 24
Secondary outcome [7] 0 0
Proportion of MGC responders
Timepoint [7] 0 0
Week 24
Secondary outcome [8] 0 0
Proportion of participants who have achieved minimal disease manifestation (total MG-ADL score of 0 or 1)
Timepoint [8] 0 0
Week 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
Completed
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
Argentina
State/province [8] 0 0
San Miguel
Country [9] 0 0
Brazil
State/province [9] 0 0
PR
Country [10] 0 0
Brazil
State/province [10] 0 0
RS
Country [11] 0 0
Brazil
State/province [11] 0 0
SP
Country [12] 0 0
Canada
State/province [12] 0 0
Quebec
Country [13] 0 0
China
State/province [13] 0 0
Beijing City
Country [14] 0 0
China
State/province [14] 0 0
Beijing
Country [15] 0 0
China
State/province [15] 0 0
Changchun City
Country [16] 0 0
China
State/province [16] 0 0
Changsha City
Country [17] 0 0
China
State/province [17] 0 0
Chengdu City
Country [18] 0 0
China
State/province [18] 0 0
Guiyang City
Country [19] 0 0
China
State/province [19] 0 0
Hangzhou City
Country [20] 0 0
China
State/province [20] 0 0
Jinan City
Country [21] 0 0
China
State/province [21] 0 0
Shanghai City
Country [22] 0 0
China
State/province [22] 0 0
Shanghai
Country [23] 0 0
China
State/province [23] 0 0
Tianjin
Country [24] 0 0
China
State/province [24] 0 0
Wenzhou City
Country [25] 0 0
China
State/province [25] 0 0
Wuhan City
Country [26] 0 0
China
State/province [26] 0 0
Xi'an City
Country [27] 0 0
Denmark
State/province [27] 0 0
Aarhus N
Country [28] 0 0
Denmark
State/province [28] 0 0
København Ø
Country [29] 0 0
France
State/province [29] 0 0
Garches
Country [30] 0 0
France
State/province [30] 0 0
Marseille
Country [31] 0 0
France
State/province [31] 0 0
Nice
Country [32] 0 0
France
State/province [32] 0 0
Pessac
Country [33] 0 0
Germany
State/province [33] 0 0
Bochum
Country [34] 0 0
Germany
State/province [34] 0 0
Essen
Country [35] 0 0
Germany
State/province [35] 0 0
Münster
Country [36] 0 0
Italy
State/province [36] 0 0
Campania
Country [37] 0 0
Italy
State/province [37] 0 0
Lombardia
Country [38] 0 0
Italy
State/province [38] 0 0
Sicilia
Country [39] 0 0
Italy
State/province [39] 0 0
Veneto
Country [40] 0 0
Japan
State/province [40] 0 0
Bunkyo-ku
Country [41] 0 0
Japan
State/province [41] 0 0
Chiba-shi, Chiba
Country [42] 0 0
Japan
State/province [42] 0 0
Hanamaki, Iwate
Country [43] 0 0
Japan
State/province [43] 0 0
Hiroshima
Country [44] 0 0
Japan
State/province [44] 0 0
Hokkaido
Country [45] 0 0
Japan
State/province [45] 0 0
Kanagawa
Country [46] 0 0
Japan
State/province [46] 0 0
Kawagoe-shi, Saitama
Country [47] 0 0
Japan
State/province [47] 0 0
Nagasaki
Country [48] 0 0
Japan
State/province [48] 0 0
Narita, Chiba
Country [49] 0 0
Japan
State/province [49] 0 0
Osaka
Country [50] 0 0
Japan
State/province [50] 0 0
Shizuoka
Country [51] 0 0
Japan
State/province [51] 0 0
Suita
Country [52] 0 0
Japan
State/province [52] 0 0
Tokyo
Country [53] 0 0
Korea, Republic of
State/province [53] 0 0
Daegu
Country [54] 0 0
Korea, Republic of
State/province [54] 0 0
Gyeongsangnam-do
Country [55] 0 0
Korea, Republic of
State/province [55] 0 0
Seoul
Country [56] 0 0
Netherlands
State/province [56] 0 0
Leiden
Country [57] 0 0
Poland
State/province [57] 0 0
Gda?sk
Country [58] 0 0
Poland
State/province [58] 0 0
Krakow
Country [59] 0 0
Poland
State/province [59] 0 0
Kraków
Country [60] 0 0
Poland
State/province [60] 0 0
Lublin
Country [61] 0 0
Russian Federation
State/province [61] 0 0
Krasnojarsk
Country [62] 0 0
Russian Federation
State/province [62] 0 0
Novosibirsk
Country [63] 0 0
Spain
State/province [63] 0 0
Barcelona
Country [64] 0 0
Spain
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Navarra
Country [65] 0 0
Spain
State/province [65] 0 0
Madrid
Country [66] 0 0
Spain
State/province [66] 0 0
Valencia
Country [67] 0 0
Taiwan
State/province [67] 0 0
Taipei
Country [68] 0 0
Taiwan
State/province [68] 0 0
Taoyuan City
Country [69] 0 0
Turkey
State/province [69] 0 0
Ankara
Country [70] 0 0
Turkey
State/province [70] 0 0
Izmir
Country [71] 0 0
Turkey
State/province [71] 0 0
Kocaeli
Country [72] 0 0
Turkey
State/province [72] 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
This study will evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of satralizumab compared with placebo in participants with generalized myasthenia gravis (gMG).
Trial website
https://clinicaltrials.gov/study/NCT04963270
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



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT04963270