A Study To Evaluate Efficacy, Safety, Pharmacokinetics, And Pharmacodynamics Of Satralizumab In Patients With Generalized Myasthenia Gravis

NCT04963270 | Terminated Phase 3 Results DMC FDA Drug

• 1 other identifier: WN42636
• Study Type: interventional
• Target: 188
• Locations: 17 countries
• Sites: 76

Brief Summary

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

Conditions

Generalized Myasthenia Gravis

Outcome Measures

Primary Outcomes (1)

• DB Period: Mean Change From Baseline in Total Myasthenia Gravis Activities of Daily Living (MG-ADL) Score in the AChR+ Population

  The MG-ADL scale was used to assess the degree of gMG symptoms (six items: diplopia, ptosis, difficulties with chewing, swallowing, talking, and respiratory problems) and functional limitations in carrying out activities of daily living (two items: ability to brush teeth or comb hair and impairment in the ability to arise from a chair) that are present and clinically relevant in gMG participants. Each of the eight items was ranked on a 0-3 scale, with 3 representing the most severe symptoms or impaired performance and 0 representing no symptoms or impaired performance. The total MG-ADL score was calculated as the sum of each item score, with a maximum score ranging from 0 (least severe symptoms/impairment) to 24 (most severe symptoms/impairment). Higher scores indicate greater disease severity.

  At Week 24

Secondary Outcomes (28)

• DB Period: Mean Change From Baseline in Total MG-ADL Score in the Overall Population (OP) at Week 24

  At Week 24

• DB Period: Percentage of Participants With a ≥ 2-point Reduction From Baseline in Total MG-ADL Score in AChR+ Population at Week 24

  At Week 24

• DB Period: Percentage of Participants With a ≥ 2-point Reduction From Baseline in Total MG-ADL Score in OP at Week 24

  At Week 24

• DB Period: Mean Change From Baseline in Quantitative Myasthenia Gravis (QMG) Score in AChR+ Population at Week 24

  At Week 24

• DB Period: Mean Change From Baseline in QMG Score in OP at Week 24

  At Week 24

Study Arms (2)

Satralizumab

EXPERIMENTAL

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.

Drug: Satralizumab

Placebo

PLACEBO COMPARATOR

Participants will receive placebo at Weeks 0, 2, 4, and Q4W thereafter

Other: Placebo

Interventions

Satralizumab DRUG

Satralizumab will be administered as a subcutaneous injection

Satralizumab

Placebo OTHER

Satralizumab placebo will be administered as a subcutaneous injection

Placebo

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• Hoffmann-La Roche: lead
• Chugai Pharmaceutical: collaborator

Study Sites (76)

Keck School of Medicine of USC

Los Angeles, California, 90033, United States

Location

University of California Irvine - Manchester Pavilion

Orange, California, 92868, United States

Location

SC3 Research Group, Inc

Pasadena, California, 91105, United States

Location

Medical Faculty Associates Inc.

Washington D.C., District of Columbia, 20037, United States

Location

University of Chicago Hospital

Chicago, Illinois, 60637, United States

Location

Prairie Education and Research

O'Fallon, Illinois, 62269, United States

Location

Hospital Italiano

CABA, C1199ABA, Argentina

Location

Hospital Ramos Mejía

CABA, C1221ADC, Argentina

Location

Hospital Britanico

Ciudad Autonoma Bs As, C1280AEB, Argentina

Location

Fundación Scherbovsky

Mendoza, M5500AYB, Argentina

Location

INECO Neurociencias Orono

Rosario, S2000DTC, Argentina

Location

Concord Repatriation General Hospital

Concord, New South Wales, 2139, Australia

Location

Hospital Sao Lucas - PUCRS

Porto Alegre, Rio Grande do Sul, 90610-000, Brazil

Location

Hospital das Clinicas - UNICAMP

Campinas, São Paulo, 13083-887, Brazil

Location

Faculdade de Medicina do ABC - FMABC

Santo André, São Paulo, 09060-650, Brazil

Location

Hospital Sao Paulo

São Paulo, São Paulo, 04037-002, Brazil

Location

MUCH - Montreal Neurological Institute & Hospital

Montreal, Quebec, H3A 2B4, Canada

Location

Beijing Tongren Hospital

Beijing, 100730, China

Location

Beijing Tiantan Hospital,Capital Medical University

Beijing, DUMMY_VALUE, China

Location

The First Hospital of Jilin University

Changchun, 130021, China

Location

West China Hospital - Sichuan University

Chengdu, 610047, China

Location

The Affiliated Hospital of Guizhou Medical University

Guiyang, DUMMY_VALUE, China

Location

Sir Run Run Shaw Hospital

Hangzhou, 310016, China

Location

The First Affiliated Hospital Of Shandong First Medical University

Jinan, DUMMY_VALUE, China

Location

Huashan Hospital, Fudan University

Shanghai, 200040, China

Location

Children's Hospital of Fudan University

Shanghai, 201102, China

Location

Tianjin Medical University General Hospital

Tianjin, 300052, China

Location

Tongji Hospital Tongji Medical College Huazhong University of Science and Technology

Wuhan, 430030, China

Location

Tangdu Hospital

Xi'an, 710038, China

Location

Aarhus Universitetshospital

Aarhus N, 8200, Denmark

Location

Rigshospitalet

København Ø, 2100, Denmark

Location

CHU Bordeaux

Bordeaux, 33076, France

Location

APHP Raymond Poincare

Garches, 92380, France

Location

Hopital Timone Adultes

Marseille, 13385, France

Location

CHU de Nantes - Hotel Dieu

Nantes, 44093, France

Location

CHU Nice - Hôpital Pasteur 2

Nice, 06000, France

Location

St. Josef-Hospital, Klinik für Neurologie

Bochum, 44791, Germany

Location

Universitätsklinikum Essen (AöR)

Essen, 45147, Germany

Location

Universitätsklinikum Münster

Münster, 48149, Germany

Location

Azienda Ospedaliera A. Cardarelli

Napoli, Campania, 80131, Italy

Location

Fond. Istituto Neurologico C.Besta

Milan, Lombardy, 20133, Italy

Location

A.R.N.A.S. Civico Di Cristina Benfratelli

Palermo, Sicily, 90127, Italy

Location

Ospedale Cà Foncello

Treviso, Veneto, 31100, Italy

Location

Juntendo University Hospital

Bunkyō City, 113-8431, Japan

Location

Chiba University Hospital

Chiba-shi, Chiba, 260-8677, Japan

Location

General Hanamaki Hospital

Hanamaki, Iwate, 025-0082, Japan

Location

Sapporo Medical University Hospital

Hokkaido, 060-8543, Japan

Location

NHO Hokkaido Medical Center

Hokkaido, 063-0005, Japan

Location

St. Marianna University Hospital

Kanagawa, 216-8511, Japan

Location

International University of Health and Welfare Narita Hospital

Narita, Chiba, 286-0124, Japan

Location

Kindai University Hospital

Osaka, 589-8511, Japan

Location

Seirei Hamamatsu General Hospital

Shizuoka, 430-8558, Japan

Location

Osaka University Hospital

Suita, 565-0871, Japan

Location

Tokyo Medical And Dental University, Medical Hospital

Tokyo, 113-8519, Japan

Location

Tokyo Medical University Hospital

Tokyo, 160-0023, Japan

Location

Klinika Neurologii Doros?ych, Uniwersyteckie Centrum Kliniczne

Gda?sk, 80-952, Poland

Location

Zespol Poradni Specjalistycznych - Poradnia Neurologiczna

Krakow, 31-503, Poland

Location

Krakowska Akademia Neurologii Sp z o.o. Centrum Neurologii K

Krakow, 31-505, Poland

Location

Indywidualna Praktyka Lekarska Prof. Dr Hab. N. Med. Konrad Rejdak.

Lublin, 20-016, Poland

Location

Krasnoyarsk State Medical Academy

Krasnoyarsk, Krasnoyarsk Krai, 660022, Russia

Location

Novosibirsk State Regional Clinical Hospital

Novosibirsk, 630087, Russia

Location

Kyungpook National University Chilgok Hospital

Daegu, 41404, South Korea

Location

Pusan National University Yangsan Hospital

Gyeongsangnam-do, 50612, South Korea

Location

Samsung Medical Center

Seoul, (0)6351, South Korea

Location

Korea University Anam Hospital

Seoul, 02841, South Korea

Location

Severance Hospital, Yonsei University Health System

Seoul, 03722, South Korea

Location

Hospital Universitari de Bellvitge

L'Hospitalet de Llobregat, Barcelona, 08907, Spain

Location

Clinica Universitaria de Navarra

Pamplona, Navarre, 31008, Spain

Location

Hospital de la Santa Creu i Sant Pau

Barcelona, 08025, Spain

Location

Hospital Universitario la Fe

Valencia, 46026, Spain

Location

Taipei Veterans General Hospital-Neurology

Taipei, 11217, Taiwan

Location

Chang Gung Medical Foundation Linkou Branch

Taoyuan, 333, Taiwan

Location

Hacettepe University Medical Faculty

Ankara, 06100, Turkey (Türkiye)

Location

Ege University Medical Faculty

Izmir, 35100, Turkey (Türkiye)

Location

Kocaeli University Hospital

Kocaeli, 41380, Turkey (Türkiye)

Location

Ondokuz Mayis Univ. Med. Fac.

Samsun, 55139, Turkey (Türkiye)

Location

Related Publications (1)

• Habib AA, Zhao C, Aban I, Franca MC Jr, Jose JG, Zu Horste GM, Klimiec-Moskal E, Pulley MT, Tavolini D, Krumova P, Lennon-Chrimes S, Smith J, Thanei GA, Blondeau K, Vodopivec I, Wolfe GI, Murai H. Safety and efficacy of satralizumab in patients with generalised myasthenia gravis (LUMINESCE): a randomised, double-blind, multicentre, placebo-controlled phase 3 trial. Lancet Neurol. 2025 Feb;24(2):117-127. doi: 10.1016/S1474-4422(24)00514-3.

  PMID: 39862880 DERIVED

MeSH Terms

Conditions

Myasthenia Gravis

Interventions

satralizumab

Condition Hierarchy (Ancestors)

Paraneoplastic Syndromes, Nervous System; Nervous System Neoplasms; Neoplasms by Site; Neoplasms; Paraneoplastic Syndromes; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Neurodegenerative Diseases; Neuromuscular Junction Diseases; Neuromuscular Diseases; Autoimmune Diseases; Immune System Diseases

Results Point of Contact

• Title: Medical Communications
• Organization: Hoffmann-La Roche

genentech@druginfo.com

800 821-8590

Study Officials

• Clinical Trials

  Hoffmann-La Roche

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 7, 2021
• First Posted: July 15, 2021
• Study Start: October 19, 2021
• Primary Completion: January 29, 2024
• Study Completion: September 2, 2024
• Last Updated: February 28, 2025
• Results First Posted: February 28, 2025

Record last verified: 2025-02

Locations

DK (2); AU (1); RU (2); ES (4); KR (5); CA (1); BR (4); DE (3); FR (5); JP (12); AR (5); PL (4); CN (12); IT (4); US (6); TU (4); TW (2)