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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT04951622




Registration number
NCT04951622
Ethics application status
Date submitted
30/06/2021
Date registered
7/07/2021

Titles & IDs
Public title
A Study of Nipocalimab Administered to Adults With Generalized Myasthenia Gravis
Scientific 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
Secondary ID [1] 0 0
2020-005732-29
Secondary ID [2] 0 0
CR109046
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
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(has expanded access)
Description of intervention(s) / exposure
Treatment: Drugs - Nipocalimab
Treatment: Drugs - Placebo

Experimental: Nipocalimab - Double-blind Placebo-controlled Phase: Participants will receive nipocalimab intravenous (IV) infusions once every 2 weeks (q2w) up to 24 weeks during double-blind placebo-controlled phase.

Open-label Extension (OLE) Phase: Participants who complete the double-blind placebo-controlled phase will enter the OLE phase and continue to receive nipocalimab q2w IV infusion till study end.

Placebo comparator: Placebo - Double-blind Placebo-controlled Phase: Participants will receive matching placebo of nipocalimab IV infusion q2w up to 24 weeks during double-blind placebo-controlled phase.


Treatment: Drugs: Nipocalimab
Nipocalimab will be administered as an IV infusion.

Treatment: Drugs: Placebo
Matching placebo will be administered as an IV infusion.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Average Change from Baseline in Myasthenia Gravis - Activities of Daily Living (MG-ADL) Score
Timepoint [1] 0 0
Baseline up to Week 24
Secondary outcome [1] 0 0
Average Change in Quantitative Myasthenia Gravis (QMG) Score Over Weeks 22 and 24 of the Double-blind Placebo-controlled Phase
Timepoint [1] 0 0
Up to Weeks 22 and 24
Secondary outcome [2] 0 0
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
Timepoint [2] 0 0
Baseline up to Weeks 22, 23 and 24
Secondary outcome [3] 0 0
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
Timepoint [3] 0 0
Weeks 1 and 2
Secondary outcome [4] 0 0
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
Timepoint [4] 0 0
Week 4 up to Week 24
Secondary outcome [5] 0 0
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
Timepoint [5] 0 0
Baseline, Weeks 22, 23 and 24
Secondary outcome [6] 0 0
Percentage of Participants with Adverse Events (AEs)
Timepoint [6] 0 0
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)
Secondary outcome [7] 0 0
Percentage of Participants with Serious Adverse Events (SAEs)
Timepoint [7] 0 0
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)
Secondary outcome [8] 0 0
Percentage of Participants with Adverse Events of Special Interest (AESIs)
Timepoint [8] 0 0
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)
Secondary outcome [9] 0 0
Percentage of Participants with Change in Vital Signs
Timepoint [9] 0 0
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)
Secondary outcome [10] 0 0
Percentage of Participants with Change in Clinical Laboratory Values
Timepoint [10] 0 0
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)
Secondary outcome [11] 0 0
Percentage of Participants with Change in Columbia-Suicide Severity Rating Scale (C-SSRS) Score
Timepoint [11] 0 0
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)
Secondary outcome [12] 0 0
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
Timepoint [12] 0 0
Week 2 up to Week 24
Secondary outcome [13] 0 0
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
Timepoint [13] 0 0
Baseline up to Weeks 22 and 24
Secondary outcome [14] 0 0
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
Timepoint [14] 0 0
Baseline up to Weeks 22 and 24
Secondary outcome [15] 0 0
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
Timepoint [15] 0 0
Baseline up to 24 weeks
Secondary outcome [16] 0 0
Change from Baseline in the Health Status Index of the EQ-5D-5L Scale Over 24 Weeks of the Double-blind Placebo-controlled Phase
Timepoint [16] 0 0
Baseline up to 24 weeks
Secondary outcome [17] 0 0
Serum Nipocalimab Concentrations Over Time
Timepoint [17] 0 0
Up to 4 years and 8 months
Secondary outcome [18] 0 0
Number of Participants with Antibodies to Nipocalimab (Anti-Drug Antibodies [ADAs] and Neutralizing Antibodies [NAbs])
Timepoint [18] 0 0
Up to 4 years and 8 months
Secondary outcome [19] 0 0
Percentage of Participants with MG-ADL Score of 0 or 1 Over Time in the Double-blind, Placebo-controlled Phase
Timepoint [19] 0 0
Up to Week 24
Secondary outcome [20] 0 0
Percentage of Participants with MG-ADL Score of 0 or 1 at Any Time During the Double-blind, Placebo-controlled Phase
Timepoint [20] 0 0
Up to Week 24
Secondary outcome [21] 0 0
Percentage of Participants with MG-ADL Score of 0 or 1 at 50% of Timepoints During the Double-blind, Placebo-controlled Phase
Timepoint [21] 0 0
Up to Week 24
Secondary outcome [22] 0 0
Percentage of Participants with MG-ADL Score of 0 or 1 at 75% of Timepoints During the Double-blind, Placebo-controlled Phase
Timepoint [22] 0 0
Up to Week 24
Secondary outcome [23] 0 0
Change in Total Serum Immunoglobulin G (IgG) Concentrations
Timepoint [23] 0 0
Up to 4 years and 8 months
Secondary outcome [24] 0 0
Change in Levels of Autoantibodies Associated with Generalized Myasthenia Gravis (gMG)
Timepoint [24] 0 0
Up to 4 years and 8 months
Secondary outcome [25] 0 0
Change from Baseline in MG-ADL Score as a Function of IgG
Timepoint [25] 0 0
Baseline up to 4 years and 8 months
Secondary outcome [26] 0 0
Change from Baseline in QMG Score as a Function of IgG
Timepoint [26] 0 0
Baseline up to 4 years and 8 months
Secondary outcome [27] 0 0
Change From Baseline in MG-ADL Score as a Response to Percent Change in Autoantibody Levels, in Seropositive Participants Treated with Nipocalimab
Timepoint [27] 0 0
Baseline up to 4 years and 8 months
Secondary outcome [28] 0 0
Change From Baseline in QMG Score as a Response to Percent Change in Autoantibody Levels, in Seropositive Participants Treated with Nipocalimab
Timepoint [28] 0 0
Baseline up to 4 years and 8 months

Eligibility
Key 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
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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

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 assessing the outcomes
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
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
15/07/2021
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
17/04/2026
Actual
Sample size
Target
198
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Melbourne Neurology Group - North Melbourne
Recruitment hospital [2] 0 0
Gold Coast University Hospital - Southport
Recruitment postcode(s) [1] 0 0
3051 - North Melbourne
Recruitment postcode(s) [2] 0 0
4215 - Southport
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arizona
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Colorado
Country [4] 0 0
United States of America
State/province [4] 0 0
Connecticut
Country [5] 0 0
United States of America
State/province [5] 0 0
Florida
Country [6] 0 0
United States of America
State/province [6] 0 0
Georgia
Country [7] 0 0
United States of America
State/province [7] 0 0
Kansas
Country [8] 0 0
United States of America
State/province [8] 0 0
Massachusetts
Country [9] 0 0
United States of America
State/province [9] 0 0
Missouri
Country [10] 0 0
United States of America
State/province [10] 0 0
North Carolina
Country [11] 0 0
United States of America
State/province [11] 0 0
Ohio
Country [12] 0 0
United States of America
State/province [12] 0 0
South Carolina
Country [13] 0 0
United States of America
State/province [13] 0 0
Tennessee
Country [14] 0 0
United States of America
State/province [14] 0 0
Texas
Country [15] 0 0
United States of America
State/province [15] 0 0
Vermont
Country [16] 0 0
Belgium
State/province [16] 0 0
Anderlecht
Country [17] 0 0
Belgium
State/province [17] 0 0
Brugge
Country [18] 0 0
Belgium
State/province [18] 0 0
Brussels
Country [19] 0 0
Belgium
State/province [19] 0 0
Gent
Country [20] 0 0
Belgium
State/province [20] 0 0
Leuven
Country [21] 0 0
Canada
State/province [21] 0 0
Ontario
Country [22] 0 0
Canada
State/province [22] 0 0
Quebec
Country [23] 0 0
China
State/province [23] 0 0
Beijing
Country [24] 0 0
China
State/province [24] 0 0
Changchun
Country [25] 0 0
China
State/province [25] 0 0
Changsha
Country [26] 0 0
China
State/province [26] 0 0
Chengdu
Country [27] 0 0
China
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Fuzhou
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China
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Guangzhou
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China
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Hangzhou
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China
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Jinan
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China
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Shanghai
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China
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Tianjin
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China
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Xi'An
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Czechia
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Brno
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Czechia
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Praha
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Denmark
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Aalborg
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Denmark
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København Ø
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France
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Bron
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France
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Grenoble
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France
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Paris
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France
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Provence-Alpes-Côte d'Azur
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Germany
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Berlin
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Germany
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Göttingen
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Germany
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Leipzig
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Germany
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Lübeck
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Germany
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Ulm
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Germany
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Wiesbaden
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Italy
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Catania
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Italy
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Cefalu
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Italy
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Milano
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Italy
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Napoli
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Italy
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Pavia
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Italy
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Roma
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Japan
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Chiba
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Japan
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Hanamaki
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Japan
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Hiroshima shi
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Japan
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Itabashi Ku
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Japan
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Kawasaki Shi
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Japan
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Kita-Gun
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Kumamoto
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Morioka-shi
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Nagoya-shi
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Niigata
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Nishinomiya-Shi
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Japan
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Sapporo
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Japan
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Sendai-City
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Japan
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Tokushima
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Japan
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Tokyo
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Korea, Republic of
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Busan
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Korea, Republic of
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Daegu
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Korea, Republic of
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Seoul
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Mexico
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Aguascalientes
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Mexico
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Cuernavaca
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Mexico
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Guadalajara
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Poland
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Bydgoszcz
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Poland
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Katowice
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Poland
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Krakow
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Poland
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Lublin
Country [79] 0 0
Poland
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Warsaw
Country [80] 0 0
Spain
State/province [80] 0 0
Alicante
Country [81] 0 0
Spain
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Barcelona
Country [82] 0 0
Spain
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Bilbao
Country [83] 0 0
Spain
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Sevilla
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Spain
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Valencia
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Sweden
State/province [85] 0 0
Karlstad
Country [86] 0 0
Sweden
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Stockholm
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Taiwan
State/province [87] 0 0
Taichung
Country [88] 0 0
Taiwan
State/province [88] 0 0
Taipei

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Janssen Research & Development, LLC
Address
Country

Ethics approval
Ethics application status

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

Contacts
Principal investigator
Name 0 0
Janssen Research & Development, LLC Clinical Trial
Address 0 0
Janssen Research & Development, LLC
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Study Contact
Address 0 0
Country 0 0
Phone 0 0
844-434-4210
Fax 0 0
Email 0 0
Participate-In-This-Study@its.jnj.com
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
What data in particular will be shared?
The data sharing policy of the Janssen Pharmaceutical Companies of Johnson \& Johnson is available at www.janssen.com/clinical-trials/transparency. As noted on this site, requests for access to the study data can be submitted through Yale Open Data Access (YODA) Project site at yoda.yale.edu
When will data be available (start and end dates)?
Available to whom?
Available for what types of analyses?
How or where can data be obtained?
IPD available at link: https://www.janssen.com/clinical-trials/transparency


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT04951622