Clinical trial NCT04951622: A Study of Nipocalimab Administered to Adults With Generalized Myasthenia Gravis
Official title: Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Nipocalimab Administered to Adults With Generalized Myasthenia Gravis
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE3
Conditions: Myasthenia Gravis
Lead sponsor: Janssen Research & Development, LLC
Sponsor class: INDUSTRY
First posted: 2021-07-07
Enrollment: 196
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to evaluate the efficacy and safety of nipocalimab compared to placebo in participants with generalized myasthenia gravis (gMG).


Eligibility criteria: Inclusion Criteria:

* Diagnosis of myasthenia gravis (MG) with generalized muscle weakness meeting the clinical criteria for generalized myasthenia gravis (gMG) as defined by the Myasthenia Gravis Foundation of America (MGFA) Clinical Classification Class II a/b, III a/b, or IVa/b at screening
* Myasthenia Gravis - Activities of Daily Living (MG-ADL) score of greater than or equal to (\>=) 6 at screening and baseline
* Has sufficient venous access to allow drug administration by infusion and blood sampling as per the protocol
* A woman of childbearing potential must have a negative highly sensitive serum (beta-human chorionic gonadotropin \[beta-hCG\]) at screening and a negative urine pregnancy test at Day 1 prior to administration of study intervention
* A male participant must agree not to donate sperm for the purpose of reproduction during the study and for a minimum 90 days after receiving the last administration of study intervention

Exclusion Criteria:

* Has any confirmed or suspected clinical immunodeficiency syndrome not related to treatment of his/her gMG, or has a family history of congenital or hereditary immunodeficiency unless confirmed absent in the participant
* Has MGFA Class I disease or presence of MG crisis (MGFA Class V) at screening, history of MG crisis within 1 month of screening, or fixed weakness (and/or 'burnt out' MG)
* Has had a thymectomy within 12 months prior to screening, or thymectomy is planned during the study
* Has known allergies, hypersensitivity, or intolerance to nipocalimab or its excipients
* Has experienced myocardial infarction, unstable ischemic heart disease, or stroke within 12 weeks of screening

Primary outcomes:
measure: Average Change from Baseline in Myasthenia Gravis - Activities of Daily Living (MG-ADL) Score
timeFrame: Baseline up to Week 24
Secondary outcomes:
measure: Average Change in Quantitative Myasthenia Gravis (QMG) Score Over Weeks 22 and 24 of the Double-blind Placebo-controlled Phase
timeFrame: Up to Weeks 22 and 24
measure: Percentage of Participants whose Average MG-ADL Total Score Over Weeks 22, 23, and 24 is at least a 2-Point Improvement from Baseline of the Double-blind Placebo-controlled Phase
timeFrame: Baseline up to Weeks 22, 23 and 24
measure: Percentage of Participants with Improvement in MG-ADL Total Score Greater Than Or Equal to (>=) 2 Points at Week 1 and/or Week 2 of the Double-blind Placebo-controlled Phase
timeFrame: Weeks 1 and 2
measure: Percentage of Participants with Improvement in MG-ADL Total Score >= 2 Points at Week 4 through Week 24 of the Double-blind Placebo-controlled Phase with No More Than 2 Non-consecutive Excursions Allowed Between Weeks 6 through Week 23
timeFrame: Week 4 up to Week 24
measure: Percentage of Participants whose Average Improvement in MG-ADL Total Score Over Weeks 22, 23, and 24 of the Double-blind, Placebo-controlled Phase is at Least a 50% Improvement from Baseline
timeFrame: Baseline, Weeks 22, 23 and 24
measure: Percentage of Participants with Adverse Events (AEs)
timeFrame: Up to Week 24 of double-blind placebo-controlled phase and up to extension Week 24 of open-label extension phase (up to Week 48)
measure: Percentage of Participants with Serious Adverse Events (SAEs)
timeFrame: Up to Week 24 of double-blind placebo-controlled phase and up to extension Week 24 of open-label extension phase (up to Week 48)
measure: Percentage of Participants with Adverse Events of Special Interest (AESIs)
timeFrame: Up to Week 24 of double-blind placebo-controlled phase and up to extension Week 24 of open-label extension phase (up to Week 48)
measure: Percentage of Participants with Change in Vital Signs
timeFrame: Up to Week 24 of double-blind placebo-controlled phase and up to extension Week 24 of open-label extension phase (up to Week 48)
measure: Percentage of Participants with Change in Clinical Laboratory Values
timeFrame: Up to Week 24 of double-blind placebo-controlled phase and up to extension Week 24 of open-label extension phase (up to Week 48)
measure: Percentage of Participants with Change in Columbia-Suicide Severity Rating Scale (C-SSRS) Score
timeFrame: Up to Week 24 of double-blind placebo-controlled phase and up to extension Week 24 of open-label extension phase (up to Week 48)
measure: Percentage of Participants with Improvement in QMG Score of >= 3 Points from Baseline at Week 2 through Week 24 of the Double-blind Placebo-controlled Phase with No More than 2 Non-consecutive Excursions Allowed at Weeks 4 through 22
timeFrame: Week 2 up to Week 24
measure: Average Change From Baseline in the Fatigue Items of the Quality of Life in Neurological Disorders Scale (Neuro-QoL Fatigue) Total Score Over Weeks 22 and 24 of Double-blind Placebo-controlled Phase
timeFrame: Baseline up to Weeks 22 and 24
measure: Average Change from Baseline in the Revised Myasthenia Gravis Quality of Life - 15 Scale (MG-Qol15r) Score Over Weeks 22 And 24 of the Double-blind Placebo-controlled Phase
timeFrame: Baseline up to Weeks 22 and 24
measure: Change from Baseline in the Visual Analog Scale (VAS) Score of European Quality of Life (EuroQol) 5-Dimension 5-Level (EQ-5D-5L) Scale Over 24 Weeks of the Double-blind Placebo-controlled Phase
timeFrame: Baseline up to 24 weeks
measure: Change from Baseline in the Health Status Index of the EQ-5D-5L Scale Over 24 Weeks of the Double-blind Placebo-controlled Phase
timeFrame: Baseline up to 24 weeks
measure: Serum Nipocalimab Concentrations Over Time
timeFrame: Up to 4 years and 8 months
measure: Number of Participants with Antibodies to Nipocalimab (Anti-Drug Antibodies [ADAs] and Neutralizing Antibodies [NAbs])
timeFrame: Up to 4 years and 8 months
measure: Percentage of Participants with MG-ADL Score of 0 or 1 Over Time in the Double-blind, Placebo-controlled Phase
timeFrame: Up to Week 24
measure: Percentage of Participants with MG-ADL Score of 0 or 1 at Any Time During the Double-blind, Placebo-controlled Phase
timeFrame: Up to Week 24
measure: Percentage of Participants with MG-ADL Score of 0 or 1 at 50% of Timepoints During the Double-blind, Placebo-controlled Phase
timeFrame: Up to Week 24
measure: Percentage of Participants with MG-ADL Score of 0 or 1 at 75% of Timepoints During the Double-blind, Placebo-controlled Phase
timeFrame: Up to Week 24
measure: Change in Total Serum Immunoglobulin G (IgG) Concentrations
timeFrame: Up to 4 years and 8 months
measure: Change in Levels of Autoantibodies Associated with Generalized Myasthenia Gravis (gMG)
timeFrame: Up to 4 years and 8 months
measure: Change from Baseline in MG-ADL Score as a Function of IgG
timeFrame: Baseline up to 4 years and 8 months
measure: Change from Baseline in QMG Score as a Function of IgG
timeFrame: Baseline up to 4 years and 8 months
measure: Change From Baseline in MG-ADL Score as a Response to Percent Change in Autoantibody Levels, in Seropositive Participants Treated with Nipocalimab
timeFrame: Baseline up to 4 years and 8 months
measure: Change From Baseline in QMG Score as a Response to Percent Change in Autoantibody Levels, in Seropositive Participants Treated with Nipocalimab
timeFrame: Baseline up to 4 years and 8 months
Other outcomes:
Locations:
city: Paradise Valley
country: United States
facility: Neuromuscular Research Center and Clinic
state: Arizona
city: Scottsdale
country: United States
facility: HonorHealth Neurology
state: Arizona
city: Los Angeles
country: United States
facility: University of Southern California
state: California
city: Palo Alto
country: United States
facility: Stanford University
state: California
city: Pasadena
country: United States
facility: Care Access Research
state: California
city: Aurora
country: United States
facility: University of Colorado Anschutz Medical Campus
state: Colorado
city: New Haven
country: United States
facility: Yale New Haven Hospital
state: Connecticut
city: Boca Raton
country: United States
facility: FM Clinical Research, LLC South Florida Neurology Associates, P. A.
state: Florida
city: Jacksonville
country: United States
facility: University of Florida Health Jacksonville
state: Florida
city: Port Charlotte
country: United States
facility: Medsol Clinical Research Center Inc
state: Florida
city: Tampa
country: United States
facility: University of South Florida
state: Florida
city: Augusta
country: United States
facility: Augusta University
state: Georgia
city: Kansas City
country: United States
facility: University of Kansas Medical Center
state: Kansas
city: Boston
country: United States
facility: St. Elizabeth Medical Center
state: Massachusetts
city: Burlington
country: United States
facility: Lahey Hospital & Medical Center
state: Massachusetts
city: Saint Louis
country: United States
facility: Washington University School Of Medicine
state: Missouri
city: Durham
country: United States
facility: Duke University School of Medicine
state: North Carolina
city: Cincinnati
country: United States
facility: University of Cincinnati
state: Ohio
city: Cleveland
country: United States
facility: Cleveland Clinic
state: Ohio
city: Columbus
country: United States
facility: The Ohio State University
state: Ohio
city: Charleston
country: United States
facility: Medical University of South Carolina
state: South Carolina
city: Cordova
country: United States
facility: Wesley Neurology
state: Tennessee
city: Dallas
country: United States
facility: UT Southwestern Medical Center
state: Texas
city: Burlington
country: United States
facility: University of Vermont
state: Vermont
city: North Melbourne
country: Australia
facility: Melbourne Neurology Group
city: Southport
country: Australia
facility: Gold Coast University Hospital
city: Anderlecht
country: Belgium
facility: ULB Hôpital Erasme
city: Brugge
country: Belgium
facility: AZ Sint Jan Brugge Oostende AV
city: Brussels
country: Belgium
facility: Cliniques Universitaires Saint Luc
city: Gent
country: Belgium
facility: AZ Sint-Lucas
city: Leuven
country: Belgium
facility: University Hospitals Leuven
city: Ottawa
country: Canada
facility: The Ottawa Hospital Research Institute
state: Ontario
city: Toronto
country: Canada
facility: Toronto General Hospital
state: Ontario
city: Montreal
country: Canada
facility: McGill University
state: Quebec
city: Beijing
country: China
facility: Beijing Tiantan Hospital, Capital Medical University
city: Beijing
country: China
facility: Xuanwu Hospital ,Capital Medical University
city: Beijing
country: China
facility: Beijing Hospital
city: Changchun
country: China
facility: The First Bethune Hospital of Jilin University
city: Changsha
country: China
facility: Xiangya Hospital Central South University
city: Chengdu
country: China
facility: West China Hospital of Sichuan University
city: Fuzhou
country: China
facility: Fujian Medical University Union Hospital
city: Guangzhou
country: China
facility: The First Affiliated Hospital of Guangzhou University of Traditional Chinese Medicine
city: Hangzhou
country: China
facility: Sir Run Run Shaw Hospital Zhejiang University School of Medicine
city: Jinan
country: China
facility: Qianfoshan hospital of Shandong Province
city: Jinan
country: China
facility: Qilu Hospital of Shandong University
city: Shanghai
country: China
facility: Huashan Hospital Fudan University
city: Tianjin
country: China
facility: Tianjin Medical University General Hospital
city: Xi'An
country: China
facility: The Second Affiliated Hospital of Air Force Medical University - Tangdu Hospital
city: Brno
country: Czechia
facility: Neurologie a rehabilitace Skopalíkova
city: Brno
country: Czechia
facility: Fakultni nemocnice Brno
city: Praha
country: Czechia
facility: Vseobecna Fakultní Nemocnice
city: Aalborg
country: Denmark
facility: Aalborg University Hospital
city: København Ø
country: Denmark
facility: Rigshospitalet
city: Bron
country: France
facility: Hopital Pierre Wertheimer
city: Grenoble
country: France
facility: CHU Grenoble
city: Paris
country: France
facility: Hopital de la Pitie Salpetriere
city: Provence-Alpes-Côte d'Azur
country: France
facility: Hopital PASTEUR
city: Provence-Alpes-Côte d'Azur
country: France
facility: Hopital PASTEUR
city: Berlin
country: Germany
facility: NeuroCure Clinical Research Center
city: Göttingen
country: Germany
facility: Universitatsmedizin Gottingen
city: Leipzig
country: Germany
facility: Universitaetsklinikum Leipzig
city: Lübeck
country: Germany
facility: Universitatsklinikum Schleswig Holstein Campus Lubeck
city: Ulm
country: Germany
facility: Universitatsklinikum Ulm
city: Wiesbaden
country: Germany
facility: DKD HELIOS Klinik Wiesbaden, Fachbereich Neurologie
city: Catania
country: Italy
facility: U.O.P.I. di Psichiatria
city: Cefalu
country: Italy
facility: Fondazione Istituto G. Giglio
city: Milano
country: Italy
facility: Istituto Neurologico Carlo Besta
city: Napoli
country: Italy
facility: Azienda Ospedaliera Univ.- Università Degli studi della Campania - Luigi Vanvitelli
city: Pavia
country: Italy
facility: IRCCS C. Mondino, Istituto Neurologico Nazionale, Fondazione
city: Roma
country: Italy
facility: Azienda ospedaliera Sant'Andrea di Roma- Università di Roma La Sapienza
city: Roma
country: Italy
facility: Policlinico Universitario Agostino Gemelli
city: Chiba
country: Japan
facility: Chiba University Hospital
city: Hanamaki
country: Japan
facility: General Hanamaki Hospital
city: Hiroshima shi
country: Japan
facility: Hiroshima University Hospital
city: Itabashi Ku
country: Japan
facility: Teikyo University Hospital
city: Kawasaki Shi
country: Japan
facility: St Marianna University Hospital
city: Kita Gun
country: Japan
facility: Kagawa University Hospital
city: Kumamoto
country: Japan
facility: Kumamoto University Hospital
city: Morioka-shi
country: Japan
facility: Iwate Medical University Hospital
city: Nagoya-shi
country: Japan
facility: National Hospital Organization Nagoya Medical Center
city: Niigata
country: Japan
facility: Niigata City General Hospital
city: Nishinomiya-Shi
country: Japan
facility: Hyogo College of Medicine Hospital
city: Sapporo
country: Japan
facility: Sapporo Medical University Hospital
city: Sapporo
country: Japan
facility: Hokkaido Medical Center
city: Sendai-City
country: Japan
facility: National Hospital Organization Sendai Medical Center
city: Tokushima
country: Japan
facility: Tokushima University Hospital
city: Tokyo
country: Japan
facility: Tokyo Medical University Hospital
city: Busan
country: Korea, Republic of
facility: Pusan National University Hospital
city: Daegu
country: Korea, Republic of
facility: Kyungpook National University Chilgok Hospital
city: Daegu
country: Korea, Republic of
facility: Kyungpook National University Hospital
city: Seoul
country: Korea, Republic of
facility: Severance Hospital Yonsei University Health System
city: Aguascalientes
country: Mexico
facility: iBiomed Research Unit
city: Cuernavaca
country: Mexico
facility: Consultorio Dr. Miguel Cortes
city: Guadalajara
country: Mexico
facility: Hospital Civil de Guadalajara Fray Antonio Alcalde
city: Bydgoszcz
country: Poland
facility: Neurocentrum Bydgoszcz Sp Z O O
city: Katowice
country: Poland
facility: NZOZ Wielospecjalistyczna Poradnia Lekarska 'Synapsis'
city: Krakow
country: Poland
facility: Centrum Neurologii Klinicznej Krakowska Akademia Neurologii
city: Lublin
country: Poland
facility: Prywatny Gabinet Lekarski
city: Warsaw
country: Poland
facility: Centrum Medyczne NeuroProtect
city: Alicante
country: Spain
facility: Hosp. Gral. Univ. de Alicante
city: Barcelona
country: Spain
facility: Hosp. de La Santa Creu I Sant Pau
city: Barcelona
country: Spain
facility: Hosp Univ Vall D Hebron
city: Barcelona
country: Spain
facility: Hosp Clinic de Barcelona
city: Bilbao
country: Spain
facility: Hosp. Univ. de Basurto
city: Sevilla
country: Spain
facility: Hosp. Virgen Macarena
city: Sevilla
country: Spain
facility: Hosp. Virgen Del Rocio
city: Valencia
country: Spain
facility: Hosp. Univ. I Politecni La Fe
city: Karlstad
country: Sweden
facility: Karlstad Central Hospital
city: Stockholm
country: Sweden
facility: Karolinska Universitetssjukhuset Solna
city: Taichung
country: Taiwan
facility: China Medical University Hospital
city: Taipei
country: Taiwan
facility: Shin Kong Wu Ho Su Memorial Hospital
city: Taipei
country: Taiwan
facility: Taipei Veterans General Hospital
Study officials:
affiliation: Janssen Research & Development, LLC
name: Janssen Research & Development, LLC Clinical Trial
role: STUDY_DIRECTOR
References: