NIPH Clinical Trials Search

JAPANESE
国立保健医療科学院
JRCT ID: jRCT2021210027

Registered date:13/08/2021

A Study of Nipocalimab Administered to Adults with Generalized Myasthenia Gravis

Basic Information

Recruitment status Recruiting
Health condition(s) or Problem(s) studiedMyasthenia Gravis
Date of first enrollment22/12/2021
Target sample size180
Countries of recruitmentBelgium,Japan,Canada,Japan,China,Japan,Colombia,Japan,Czechia,Japan,Germany,Japan,Denmark,Japan,Spain,Japan,France,Japan,Kingdom Of Great Britain And Northern Ireland,Japan,Italy,Japan,Mexico,Japan,Poland,Japan,Sweden,Japan,United States Of America,Japan,Australia,Japan,Korea Republic Of,Japan,Taiwan,Japan
Study typeInterventional
Intervention(s)Nipocalimab (JNJ-80202135, M281) Double-blind Placebocontrolled 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 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.

Outcome(s)

Primary OutcomeAverage Change from Baseline in Myasthenia Gravis -Activities of Daily Living (MG-ADL) Score Baseline up to Week 24 Average change from baseline in MG-ADL score over Weeks 22, 23 and 24 of the double-blind placebo-controlled phase will be reported. Averaging over multiple time points (Weeks 22, 23 and 24) will be done to get a single measure. The MG-ADL provides a rapid assessment of the participant's MG symptom severity. 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) are rated on a 4-point scale: 0 (no impairment) to 3 (severe impairment). The total score will be sum of eight function scores and can range from 0 to 24. A higher score indicates greater symptom severity.
Secondary OutcomeAverage Change in Quantitative Myasthenia Gravis (QMG) Score Over Weeks 22 and 24 of the Double-blind Placebo-controlled Phase Up to Weeks 22 and 24 Average change in QMG score over Weeks 22 and 24 of the double-blind placebo-controlled phase will be reported. The QMG test is a standardized quantitative strength assessment comprising 13 components. The quantitative results of each strength component are mapped to the following 4-point scale: 0 equals to (=) none, 1 = mild, 2 = moderate and 3 = severe. The total score will be sum of 13 components scores and can range from 0 to 39. A higher score indicates greater weakness. 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 Baseline up to Weeks 22, 23 and 24 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 will be reported. 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 Weeks 1 and 2 Percentage of participants with improvement in MG-ADL total score >= 2 points at Week 1 and/or Week 2 of the double-blind placebo-controlled phase will be reported. 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 Week 4 up to Week 24 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 (excursion is defined as loss of improvement in MG-ADL score >= 2 points from baseline) will be reported. Percentage of Participants whose Average Improvement in MGADL Total Score Over Weeks 22, 23, and 24 of the Doubleblind, Placebo-controlled Phase is at Least a 50% Improvement from Baseline Baseline, Weeks 22, 23 and 24 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 will be reported. Percentage of Participants with Adverse Events (AEs) 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) An AE is any untoward medical occurrence in a participant participating in a clinical study that does not necessarily have a causal relationship with the pharmaceutical/biological agent under study. Percentage of Participants with Serious Adverse Events (SAEs) 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) A SAE is any untoward medical occurrence that at any dose: results in death; is life-threatening; requires inpatient hospitalization or prolongation of existing hospitalization; results in persistent or significant disability/incapacity; is a congenital anomaly/birth defect; is a suspected transmission of any infectious agent via a medicinal product; is medically important. Percentage of Participants with Adverse Events of Special Interest (AESIs) 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) Percentage of participants with AESIs will be reported. Treatment-emergent AEs associated with the following situations are considered as AESI: 1) severe or medically significant or immediately life-threatening infections requiring intravenous (IV) anti-infective or operative/invasive intervention or requiring hospitalization or prolongation of existing hospitalization; 2) hypoalbuminemia with albumin less than (<) 20 grams per liter (g/L). Treatment-emergent AEs are defined as AEs with onset or worsening on or after date of first dose of study treatment. Percentage of Participants with Change in Vital Signs 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) Percentage of participants with change in vital signs (temperature, blood pressure and heart rate) will be reported. Percentage of Participants with Change in Clinical Laboratory Values 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) Percentage of participants with change in clinical laboratory (serum chemistry, hematology, lipid profiles and urinalysis) values will be reported. Percentage of Participants with Change in Columbia-Suicide Severity Rating Scale (C-SSRS) Score 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) Percentage of participants with change in C-SSRS scale score will be reported. C-SSRS is semi structured clinician-administered questionnaire designed to solicit the occurrence, severity, and frequency of suicide-related ideation and behaviors. Total score ranges from 1 to 10. Higher scores indicate greater severity. 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 Week 2 up to Week 24 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 (excursions defined as loss of improvement in QMG score of >= 3 points from baseline) will be reported. 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 Baseline up to Weeks 22 and 24 Average change from baseline in the Neuro-QoL Fatigue total score over Weeks 22 and 24 of double-blind placebo-controlled phase will be reported. Neuro-QoL fatigue is a 19-item questionnaire developed and validated for use in common neurological conditions which assesses patient-reported fatigue and associated impact on physical, mental, and social activities during the past 7 days. Each item included in the Neuro-QoL Fatigue questionnaire is graded on a 5-point Likert-type scale (1=Never; 2=Rarely; 3=Sometimes; 4=Often; 5=Always). The total scores are calculated by summing 19 items score and can range from 19-95. Higher score reflects more fatigue. 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 Baseline up to Weeks 22 and 24 Average change from baseline in the MG-QoL15r score over Weeks 22 and 24 of the double-blind placebo-controlled phase will be reported. The MG-QoL15r is a participant-reported outcome instrument that measures MG-specific health-related quality of life. The MG-QoL15r contains 15 items that evaluate patients' experience related to Myasthenia Gravis over the "past few weeks" on a 3-point Likert-type scale (0=Not at all; 1=Somewhat; 2=Very much). The total score of the MG-QoL15r can be calculated by summing 15 items score and can range from 0 to 30. A higher score indicates poorer health related quality of life. See attached file for other secondary outcomes.

Key inclusion & exclusion criteria

Age minimum>= 18age old
Age maximumNot applicable
GenderBoth
Include 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
Exclude 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

Related Information

Contact

Public contact
Name Medical Information Center
Address 3-5-2 Nishikanda Chiyoda-ku, Tokyo Tokyo Japan 101-0065
Telephone +81-120-183-275
E-mail DL-JANJP-JCO_TL_TSG_EMP@its.jnj.com
Affiliation Janssen Pharmaceutical K.K.
Scientific contact
Name Masayoshi Nakano
Address 3-5-2 Nishikanda Chiyoda-ku, Tokyo Tokyo Japan 101-0065
Telephone +81-120-183-275
E-mail DL-JANJP-JCO_TL_TSG_EMP@its.jnj.com
Affiliation Janssen Pharmaceutical K.K.