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NCT Number: NCT04951622

A Study of Nipocalimab Administered to Adults With Generalized Myasthenia Gravis

The purpose of this study is to evaluate the efficacy and safety of nipocalimab compared to placebo in participants with generalized myasthenia gravis (gMG). The purpose of the subcutaneous substudy is to evaluate how well it works in the body (pharmacodynamic [PD]) when given as an injection under the skin (subcutaneous) compared to when given through a vein (intravenous) in participants with gMG.

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Key information

Conditions

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

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Melbourne Neurology Group, North Melbourne, Australia

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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
  • For the SC Substudy (Cohort 1 and Cohort 2): Has reasonable abdominal skin area for SC administration
  • For the SC Substudy (Cohort 1 and Cohort 2): Participants must be willing to comply with maintaining their stable dose of corticosteroids and/or immunosuppressants for the initial 8 weeks of the SC substudy, that is, through the SC Week 8 visit

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
  • For the SC Substudy (Cohort 1): Participants who have undergone a recent tapering of their concomitant MG medication in the OLE
  • For the SC Substudy (Cohort 1): Participants deteriorating during the OLE in the month prior to SC Dose 1 of the SC substudy such that they meet the criteria for clinical deterioration
  • For the SC Substudy (Cohort 2): History of an unprovoked pulmonary embolism within 1 year prior to screening or history of recurrent deep vein thrombosis (DVT)

Treatment and study plan

Nipocalimab

Drug

Nipocalimab will be administered as an IV infusion.

Other names: JNJ-80202135, M281

Placebo

Drug

Matching placebo will be administered as an IV infusion.

Nipocalimab SC-LIV

Drug

Nipocalimab will be administered subcutaneously.

Primary outcomes

  1. Double-blind (DB) Phase: Average Change From Baseline in Myasthenia Gravis - Activities of Daily Living (MG-ADL) Total Score Over Weeks 22, 23, and 24

    Time frame: Baseline, Weeks 22, 23, and 24

    Average change from baseline over multiple timepoints (Weeks 22, 23, and 24) was reported in this outcome measure. The MG-ADL provided a rapid assessment of the participant's MG symptom severity of eight functions (talking, chewing, swallowing, breathing, impairment of ability to brush teeth or comb hair, impairment of ability to arise from a chair, double vision, eyelid droop) rated on a 4-point scale ranging from 0 (normal) to 3 (severe). MG-ADL total score was sum of 8 individual items, which ranging from 0 to 24. A higher score indicated greater symptom severity. Baseline was defined as the average of the screening and Day 1 total scores.

  2. Sub Study: Percent Change in Anti-AChR Autoantibody Titer From Pre-first Nipocalimab Dose on Day 1 up to Week 8 (Day 57)

    Time frame: From pre-first nipocalimab dose on Day 1 up to Week 8 (Day 57)

  3. Sub Study: Percent Change in Total IgG Levels From Pre-first Nipocalimab Dose on Day 1 up to Week 8 (Day 57)

    Time frame: From pre-first nipocalimab dose on Day 1 up to Week 8 (Day 57)

Secondary outcomes

  1. DB Phase: Average Change From Baseline in Quantitative Myasthenia Gravis (QMG) Score Over Weeks 22 and 24

    Time frame: Baseline, Weeks 22, and 24

  2. DB Phase: Percentage of Participants Who Had Achieved at Least a 2-point Average Improvement From Baseline in Myasthenia Gravis - Activities of Daily Living (MG-ADL) Total Score Over Weeks 22, 23, and 24

    Time frame: Weeks 22, 23, and 24

  3. DB Phase: Percentage of Participants Who Had Achieved an Improvement of Greater Than or Equal to (>=) 2 Points in the Myasthenia Gravis - Activities of Daily Living (MG-ADL) Total Score at Week 1 and/or Week 2

    Time frame: Weeks 1 and 2

  4. DB Phase: Percentage of Participants Who Had an Improvement of >= 2 Points in the MG-ADL Total Score From Week 4 Through Week 24 With no More Than 2 Non-consecutive Excursions Allowed Between Weeks 6 Through 23

    Time frame: From Week 4 up to Week 24

  5. DB Phase: Percentage of Participants Who Had Achieved at Least a 50 Percent (%) Average Improvement From Baseline in the Myasthenia Gravis - Activities of Daily Living (MG-ADL) Total Score Over Weeks 22, 23, and 24

    Time frame: Weeks 22, 23, and 24

  6. Percentage of Participants With Treatment-emergent Adverse Events (TEAEs)

    Time frame: From start of treatment (DB phase Day 1) up to 4 years 9 months

  7. Percentage of Participants With Treatment-emergent Serious Adverse Events (TESAEs)

    Time frame: From start of treatment (DB phase Day 1) up to 4 years 9 months

  8. Percentage of Participants With AEs of Special Interest (AESIs)

    Time frame: From start of treatment (DB phase Day 1) up to 4 years 9 months

  9. Number of Participants With Change in Vital Signs

    Time frame: From start of treatment (DB phase Day 1) up to 4 years 9 months

  10. Number of Participants With Change in Clinical Laboratory Values

    Time frame: From start of treatment (DB phase Day 1) up to 4 years 9 months

  11. Number of Participants With Change From Baseline in Columbia-Suicide Severity Rating Scale (C-SSRS) Score

    Time frame: From start of treatment (Day 1) up to 4 years 9 months

  12. DB Phase: Percentage of Participants Who Had an Improvement of >= 3 Points in Quantitative Myasthenia Gravis (QMG) Score From Baseline, at Week 2 Through Week 24, With No More Than 2 Non-consecutive Excursions Allowed Between at Weeks 4 Through Week 22

    Time frame: Baseline, Week 2 up to Week 24

  13. DB Phase: Average Change From Baseline in the Fatigue Items of the Quality of Life in Neurological Disorders (Neuro-QoL) Fatigue Scale Total Score Over Weeks 22 and 24

    Time frame: Baseline up to Weeks 22, and 24

  14. DB Phase: Average Change From Baseline in the Revised Myasthenia Gravis Quality of Life (Revised) Instrument (MG-Qol15r) Score Over Weeks 22 And 24

    Time frame: Baseline up to Weeks 22, and 24

  15. DB Phase: 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

    Time frame: Baseline up to Week 24

  16. DB Phase: Change From Baseline in the Health Status Index of the EuroQol 5-Dimension 5-Level (EQ-5D-5L) Scale Over 24 Weeks

    Time frame: Baseline up to Week 24

  17. DB Phase: Percentage of Participants With Myasthenia Gravis - Activities of Daily Living (MG-ADL) Score of 0 or 1 Over Time

    Time frame: Baseline up to Week 24

  18. DB Phase: Percentage of Participants With Myasthenia Gravis - Activities of Daily Living (MG-ADL) Score of 0 or 1 at Any Time

    Time frame: Baseline up to Week 24

  19. DB Phase: Percentage of Participants With Myasthenia Gravis - Activities of Daily Living (MG-ADL) Score of 0 or 1 at 50% of Timepoints

    Time frame: Baseline up to Week 24

  20. DB Phase: Percentage of Participants With Myasthenia Gravis - Activities of Daily Living (MG-ADL) Score of 0 or 1 at 75% of Timepoints

    Time frame: Baseline up to Week 24

  21. Serum Concentrations of Nipocalimab Over Time

    Time frame: DB Phase: Predose and 45 minutes post-dose on Day 1, Weeks 2, 4, 8, 12,16, 20, 24;OL Phase: Predose on Day 1, Weeks 8, 16, 24, 36, 48, 60, 72, 84, and 96

  22. Number of Participants With Antibodies to Nipocalimab (Anti-Drug Antibodies [ADAs] and Neutralizing Antibodies [NAbs])

    Time frame: From start of treatment (Day 1) up to 4 years 9 months

  23. Percent Change From Baseline in Total Serum Immunoglobulin G (IgG) Concentrations

    Time frame: Baseline up to 4 years 9 months

  24. Change From Baseline in Levels of Autoantibodies Associated With Generalized Myasthenia Gravis (gMG)

    Time frame: Baseline up to 4 years 9 months

  25. Change From Baseline in Myasthenia Gravis - Activities of Daily Living (MG-ADL) Score as a Function of IgG

    Time frame: Baseline up to 4 years 9 months

  26. Change From Baseline in Quantitative Myasthenia Gravis (QMG) Score as a Function of Immunoglobulin G (IgG)

    Time frame: Baseline up to 4 years 9 months

  27. Change From Baseline in Myasthenia Gravis - Activities of Daily Living (MG-ADL) Score as a Response to Percent Change in Autoantibody Levels in Seropositive Participants Treated With Nipocalimab

    Time frame: Baseline up to 4 years 9 months

  28. Change From Baseline in QMG Score as a Response to Percent Change in Autoantibody Levels in Seropositive Participants Treated With Nipocalimab

    Time frame: Baseline up to 4 years 9 months

  29. Sub Study: Number of Participants With Treatment-Emergent AEs

    Time frame: Up to SC Week 8 (Day 57)

  30. Sub Study: Number of Participants With Abnormalities in Vital Signs

    Time frame: Up to SC Week 8 (Day 57)

  31. Sub Study: Number of Participants With Abnormalities in Physical Examinations

    Time frame: Up to SC Week 8 (Day 57)

  32. Sub Study: Number of Participants With Abnormalities in Laboratory Parameters

    Time frame: Up to SC Week 8 (Day 57)

  33. Sub Study: Numeric Pain Rating Scale (NPRS) Assessment With SC Use of Nipocalimab

    Time frame: Up to SC Week 8 (Day 57)

  34. Sub Study: Number of Participants With Injection Site-Reactions

    Time frame: Up to SC Week 8 (Day 57)

  35. Sub Study: Change From Baseline in MG-ADL Clinician-Reported Outcome Measures up to Week 8 (Day 57)

    Time frame: From baseline (pre-first nipocalimab dose on Day 1) up to Week 8 (Day 57)

  36. Sub Study: Change From Baseline in QMG Clinician-Reported Outcome Measures up to Week 8 (Day 57)

    Time frame: From baseline (pre-first nipocalimab dose on Day 1) up to Week 8 (Day 57)

  37. Sub Study: Change From Baseline in Myasthenia Gravis Foundation of America (MGFA) Clinician-Reported Measures up to Week 8 (Day 57)

    Time frame: From baseline (pre-first nipocalimab dose on Day 1) up to Week 8 (Day 57)

  38. Sub Study: Change From Baseline in C-SSRS Clinician-Reported Outcome Measures up to Week 8 (Day 57)

    Time frame: From baseline (pre-first nipocalimab dose on Day 1) up to Week 8 (Day 57)

  39. Sub Study: Change From Baseline in Neuro-QoL Participant-Reported Outcome Measures up to Week 8 (Day 57)

    Time frame: From baseline (pre-first nipocalimab dose on Day 1) up to Week 8 (Day 57)

  40. Sub Study: Change From Baseline in Patient Global Impression of Severity (PGIS) Scale Score up to Week 8 (Day 57)

    Time frame: From baseline (pre-first nipocalimab dose on Day 1) up to Week 8 (Day 57)

  41. Sub Study: Change From Baseline in Patient Global Impression of Change (PGIC) Scale Score up to Week 8 (Day 57)

    Time frame: From baseline (pre-first nipocalimab dose on Day 1) up to Week 8 (Day 57)

  42. Sub Study: Change From Baseline in MG-QoL Participant-Reported Outcome Measures up to Week 8 (Day 57)

    Time frame: From baseline (pre-first nipocalimab dose on Day 1) up to Week 8 (Day 57)

  43. Sub Study: Change From Baseline in EQ-5D-5L Participant-Reported Outcome Measures up to Week 8 (Day 57)

    Time frame: From baseline (pre-first nipocalimab dose on Day 1) up to Week 8 (Day 57)

  44. Sub Study: Percent Change in Total IgG Levels From Pre-first Nipocalimab Dose on Day 1 up to Week 8 (Day 57)

    Time frame: From pre-first nipocalimab dose on Day 1 up to Week 8 (Day 57)

  45. Sub Study: Percent Change in Anti-AChR Autoantibody Titer From Pre-first Nipocalimab Dose on Day 1 up to Week 8 (Day 57)

    Time frame: From pre-first nipocalimab dose on Day 1 up to Week 8 (Day 57)

Study contacts

Contact information is provided by the study sponsor or research team.

Study Contact

CONTACT

[email protected]

844-434-4210

Sponsors and collaborators

Lead sponsor

Janssen Research & Development, LLC

Industry

Registry information

Official study 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

Important dates

Study start
2021
Primary completion
2023
Study completion
2029
First posted
Jul 7, 2021
Registry last updated
Jul 27, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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