Clinical trial NCT04963270: A Study To Evaluate Efficacy, Safety, Pharmacokinetics, And Pharmacodynamics Of Satralizumab In Patients With Generalized Myasthenia Gravis
Official 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
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE3
Conditions: Generalized Myasthenia Gravis
Lead sponsor: Hoffmann-La Roche
Sponsor class: INDUSTRY
Collaborators: name: Chugai Pharmaceutical
First posted: 2021-07-15
Enrollment: 188
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: This study will evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of satralizumab compared with placebo in participants with generalized myasthenia gravis (gMG).


Eligibility criteria: 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.

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

Primary outcomes:
measure: DB Period: Mean Change From Baseline in Total Myasthenia Gravis Activities of Daily Living (MG-ADL) Score in the AChR+ Population
timeFrame: At Week 24
Secondary outcomes:
measure: DB Period: Mean Change From Baseline in Total MG-ADL Score in the Overall Population (OP) at Week 24
timeFrame: At Week 24
measure: DB Period: Percentage of Participants With a ≥ 2-point Reduction From Baseline in Total MG-ADL Score in AChR+ Population at Week 24
timeFrame: At Week 24
measure: DB Period: Percentage of Participants With a ≥ 2-point Reduction From Baseline in Total MG-ADL Score in OP at Week 24
timeFrame: At Week 24
measure: DB Period: Mean Change From Baseline in Quantitative Myasthenia Gravis (QMG) Score in AChR+ Population at Week 24
timeFrame: At Week 24
measure: DB Period: Mean Change From Baseline in QMG Score in OP at Week 24
timeFrame: At Week 24
measure: 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
timeFrame: At Week 24
measure: DB Period: Mean Change From Baseline in MG-QOL 15r Total Score in OP at Week 24
timeFrame: At Week 24
measure: 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
timeFrame: At Week 24
measure: DB Period: Mean Change From Baseline in Neuro-QoL Fatigue Subscale Total Score in OP at Week 24
timeFrame: At Week 24
measure: DB Period: Percentage of Participants With a ≥ 3-point Reduction From Baseline in QMG Score in AChR+ Population at Week 24
timeFrame: At Week 24
measure: DB Period: Percentage of Participants With a ≥ 3-point Reduction From Baseline in QMG Score in OP at Week 24
timeFrame: At Week 24
measure: DB Period: Mean Change From Baseline in Total Myasthenia Gravis Composite (MGC) Score in AChR+ Population at Week 24
timeFrame: At Week 24
measure: DB Period: Mean Change From Baseline in Total MGC Score in OP at Week 24
timeFrame: At Week 24
measure: DB Period: Percentage of Participants With a ≥ 3-point Reduction From Baseline in Total MGC Score in AChR+ Population at Week 24
timeFrame: At Week 24
measure: DB Period: Percentage of Participants With a ≥ 3-point Reduction From Baseline in Total MGC Score in OP at Week 24
timeFrame: At Week 24
measure: DB Period: Percentage of Participants Who Achieved Minimal Symptom Expression (Total MG-ADL Score of 0 or 1) in AChR+ Population at Week 24
timeFrame: At Week 24
measure: DB Period: Percentage of Participants Who Achieved Minimal Symptom Expression (Total MG-ADL Score of 0 or 1) in OP at Week 24
timeFrame: At Week 24
measure: DB Period: Percentage of Participants With at Least One gMG-Related Exacerbation Between Baseline and Week 24 in AChR+ Population
timeFrame: Baseline up to Week 24
measure: DB Period: Percentage of Participants With at Least One gMG-Related Exacerbation Between Baseline and Week 24 in OP
timeFrame: Baseline up to Week 24
measure: DB Period: Percentage of Participants in AChR+ Population Receiving Rescue Therapy Between Baseline and Week 24
timeFrame: Baseline up to Week 24
measure: DB Period: Percentage of Participants in OP Receiving Rescue Therapy Between Baseline and Week 24
timeFrame: Baseline up to Week 24
measure: DB Period: Duration of Meaningful Improvement, Defined as ≥ 2-Point Reduction From Baseline in Total MG-ADL Score in AChR+ Population
timeFrame: Baseline, Week 24
measure: DB Period: Duration of Meaningful Improvement, Defined as ≥ 2-Point Reduction From Baseline in Total MG-ADL Score in OP
timeFrame: Baseline, Week 24
measure: DB Period: Number of Participants With Adverse Events (AEs)
timeFrame: Day 1 up to approximately 24 weeks
measure: DB Period: Serum Levels of Interleukin-6 (IL-6)
timeFrame: Baseline, Weeks 2, 4, 8, 12, 16, 20 and 24
measure: DB Period: Serum Levels of Soluble Interleukin-6 Receptors (sIL-6R)
timeFrame: Baseline, Weeks 2, 4, 8, 12, 16, 20 and 24
measure: Number of Participants With Anti-drug Antibodies (ADAs) to Satralizumab
timeFrame: Baseline to Week 24
measure: Serum Concentrations of Satralizumab
timeFrame: Weeks 0, 2, 4, 8, 12, 16, 20 and 24
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: Keck School of Medicine of USC
state: California
city: Orange
country: United States
facility: University of California Irvine - Manchester Pavilion
state: California
city: Pasadena
country: United States
facility: SC3 Research Group, Inc
state: California
city: Washington
country: United States
facility: Medical Faculty Associates Inc.
state: District of Columbia
city: Chicago
country: United States
facility: University of Chicago Hospital
state: Illinois
city: O'Fallon
country: United States
facility: Prairie Education and Research
state: Illinois
city: Caba
country: Argentina
facility: Hospital Italiano
city: Caba
country: Argentina
facility: Hospital Ramos Mejía
city: Ciudad Autonoma Bs As
country: Argentina
facility: Hospital Britanico
city: Mendoza
country: Argentina
facility: Fundación Scherbovsky
city: Rosario
country: Argentina
facility: INECO Neurociencias Orono
city: Concord
country: Australia
facility: Concord Repatriation General Hospital
state: New South Wales
city: Porto Alegre
country: Brazil
facility: Hospital Sao Lucas - PUCRS
state: Rio Grande Do Sul
city: Campinas
country: Brazil
facility: Hospital das Clinicas - UNICAMP
state: São Paulo
city: Santo Andre
country: Brazil
facility: Faculdade de Medicina do ABC - FMABC
state: São Paulo
city: Sao Paulo
country: Brazil
facility: Hospital Sao Paulo
state: São Paulo
city: Montreal
country: Canada
facility: MUCH - Montreal Neurological Institute & Hospital
state: Quebec
city: Beijing
country: China
facility: Beijing Tongren Hospital
city: Beijing
country: China
facility: Beijing Tiantan Hospital,Capital Medical University
city: Changchun City
country: China
facility: The First Hospital of Jilin University
city: Chengdu City
country: China
facility: West China Hospital - Sichuan University
city: Guiyang
country: China
facility: The Affiliated Hospital of Guizhou Medical University
city: Hangzhou
country: China
facility: Sir Run Run Shaw Hospital
city: Jinan
country: China
facility: The First Affiliated Hospital Of Shandong First Medical University
city: Shanghai City
country: China
facility: Huashan Hospital, Fudan University
city: Shanghai
country: China
facility: Children's Hospital of Fudan University
city: Tianjin
country: China
facility: Tianjin Medical University General Hospital
city: Wuhan City
country: China
facility: Tongji Hospital Tongji Medical College Huazhong University of Science and Technology
city: Xi'an City
country: China
facility: Tangdu Hospital
city: Aarhus N
country: Denmark
facility: Aarhus Universitetshospital
city: København Ø
country: Denmark
facility: Rigshospitalet
city: Bordeaux cedex
country: France
facility: CHU Bordeaux
city: Garches
country: France
facility: APHP Raymond Poincare
city: Marseille
country: France
facility: Hopital Timone Adultes
city: Nantes
country: France
facility: CHU de Nantes - Hotel Dieu
city: Nice
country: France
facility: CHU Nice - Hôpital Pasteur 2
city: Bochum
country: Germany
facility: St. Josef-Hospital, Klinik für Neurologie
city: Essen
country: Germany
facility: Universitätsklinikum Essen (AöR)
city: Münster
country: Germany
facility: Universitätsklinikum Münster
city: Napoli
country: Italy
facility: Azienda Ospedaliera A. Cardarelli
state: Campania
city: Milano
country: Italy
facility: Fond. Istituto Neurologico C.Besta
state: Lombardia
city: Palermo
country: Italy
facility: A.R.N.A.S. Civico Di Cristina Benfratelli
state: Sicilia
city: Treviso
country: Italy
facility: Ospedale Cà Foncello
state: Veneto
city: Bunkyo-ku
country: Japan
facility: Juntendo University Hospital
city: Chiba-shi, Chiba
country: Japan
facility: Chiba University Hospital
city: Hanamaki, Iwate
country: Japan
facility: General Hanamaki Hospital
city: Hokkaido
country: Japan
facility: Sapporo Medical University Hospital
city: Hokkaido
country: Japan
facility: NHO Hokkaido Medical Center
city: Kanagawa
country: Japan
facility: St. Marianna University Hospital
city: Narita, Chiba
country: Japan
facility: International University of Health and Welfare Narita Hospital
city: Osaka
country: Japan
facility: Kindai University Hospital
city: Shizuoka
country: Japan
facility: Seirei Hamamatsu General Hospital
city: Suita
country: Japan
facility: Osaka University Hospital
city: Tokyo
country: Japan
facility: Tokyo Medical And Dental University, Medical Hospital
city: Tokyo
country: Japan
facility: Tokyo Medical University Hospital
city: Daegu
country: Korea, Republic of
facility: Kyungpook National University Chilgok Hospital
city: Gyeongsangnam-do
country: Korea, Republic of
facility: Pusan National University Yangsan Hospital
city: Seoul
country: Korea, Republic of
facility: Samsung Medical Center
city: Seoul
country: Korea, Republic of
facility: Korea University Anam Hospital
city: Seoul
country: Korea, Republic of
facility: Severance Hospital, Yonsei University Health System
city: Gda?sk
country: Poland
facility: Klinika Neurologii Doros?ych, Uniwersyteckie Centrum Kliniczne
city: Krakow
country: Poland
facility: Krakowska Akademia Neurologii Sp z o.o. Centrum Neurologii K
city: Kraków
country: Poland
facility: Zespol Poradni Specjalistycznych - Poradnia Neurologiczna
city: Lublin
country: Poland
facility: Indywidualna Praktyka Lekarska Prof. Dr Hab. N. Med. Konrad Rejdak.
city: Krasnoyarsk
country: Russian Federation
facility: Krasnoyarsk State Medical Academy
state: Krasnojarsk
city: Novosibirsk
country: Russian Federation
facility: Novosibirsk State Regional Clinical Hospital
city: L'Hospitalet de Llobregat
country: Spain
facility: Hospital Universitari de Bellvitge
state: Barcelona
city: Pamplona
country: Spain
facility: Clinica Universitaria de Navarra
state: Navarra
city: Barcelona
country: Spain
facility: Hospital de la Santa Creu i Sant Pau
city: Valencia
country: Spain
facility: Hospital Universitario la Fe
city: Taipei
country: Taiwan
facility: Taipei Veterans General Hospital-Neurology
city: Taoyuan City
country: Taiwan
facility: Chang Gung Medical Foundation Linkou Branch
city: Ankara
country: Turkey
facility: Hacettepe University Medical Faculty
city: Izmir
country: Turkey
facility: Ege University Medical Faculty
city: Kocaeli
country: Turkey
facility: Kocaeli University Hospital
city: Samsun
country: Turkey
facility: Ondokuz Mayis Univ. Med. Fac.
Study officials:
affiliation: Hoffmann-La Roche
name: Clinical Trials
role: STUDY_DIRECTOR
References: