Neoplasms

Nervous System Diseases

Immune System Diseases

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Musculoskeletal Diseases

Symptoms and General Pathology

Rare Diseases

Myasthenia Gravis

Muscle Weakness

Paresis

Asthenia

Muscular Diseases

Neuromuscular Manifestations

Neurologic Manifestations

Syndrome

Paraneoplastic Syndromes

Paraneoplastic Syndromes, Nervous System

Nervous System Neoplasms

Autoimmune Diseases

Autoimmune Diseases of the Nervous System

Neurodegenerative Diseases

Neuromuscular Junction Diseases

Neuromuscular Diseases

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.

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.

Nipocalimab will be administered as an IV infusion.

Nipocalimab

Matching placebo will be administered as an IV infusion.

Placebo

Study Contact

Janssen Research & Development, LLC Clinical Trial

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

Myasthenia gravis (MG) is a rare, heterogeneous, neuromuscular disease characterized by fluctuating, fatigable muscle weakness. MG is caused by pathogenic autoantibodies that impair cholinergic transmission in the postsynaptic membrane at the neuromuscular junction and impair or prevent muscle contraction. Nipocalimab (also referred to as JNJ-80202135 or M281) is a fully human, aglycosylated immunoglobulin (Ig)G1 monoclonal antibody (mAb) designed to selectively bind, saturate, and block the IgG binding site on the endogenous neonatal Fc receptor (FcRn). This study will consist of a screening phase (up to 4 weeks), treatment phase (a 24-week double-blind placebo-controlled phase, and an open-label extension \[OLE\] phase \[up to 2 years\]) and a follow-up safety visit (up to 8 weeks after last infusion of study intervention). Efficacy evaluations will include assessments such as Myasthenia Gravis - Activities of Daily Living (MG-ADL) score. Safety evaluations (such as adverse events, physical examination, vital signs, electrocardiogram \[ECG\], and clinical laboratory tests) will be performed. The overall duration of study will be up to 4 years and 8 months.

A Study of Nipocalimab Administered to Adults With Generalized Myasthenia Gravis

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

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

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.

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 will be reported.

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 (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 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.

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.

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 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 (temperature, blood pressure and heart rate) will be reported.

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 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 (excursions defined as loss of improvement in QMG score of \>= 3 points from baseline) will be reported.

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 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.

Change from baseline in the VAS score of EQ-5D-5L scale over 24 weeks of the double-blind placebo-controlled phase will be reported. EQ-5D-5L is a 2-part instrument for use as a measure of health outcome, designed for self-completion by respondents. It consists of EQ-5D-5L descriptive system and EQ VAS. The EQ VAS self-rating records the respondent's own assessment of his or her overall health status at the time of completion, on a scale of 0 (worst imaginable health state) to 100 (best imaginable health state). Positive change in score indicates improvement.

Change from baseline in health status index of EQ-5D-5L scale over 24 weeks of double-blind placebo-controlled phase will be reported. EQ-5D-5L is standardized instrument for use as measure of health outcome, primarily designed for self-completion by respondents. It consists of EQ-5D-5L descriptive system and EQ-VAS. EQ-5D-5L descriptive system comprises following 5 dimensions: Mobility, self-care, usual activities, pain/discomfort and anxiety/depression. Each of 5 dimensions is divided into 5 levels of perceived problems (1 indicating no problem, 2 indicating slight problems, 3 indicating moderate problems, 4 indicating severe problems, 5 indicating extreme problems). Participant selects an answer for each of 5 dimensions considering response that best matches his or her health "today". The responses to 5 dimensions are used to compute a single score ranging from zero (0.0- worst health state) to 1 (1.0- better health state) representing the general health status of the individual.

Serum nipocalimab concentrations over time will be reported.

Number of participants with antibodies to nipocalimab (ADAs and NAbs) will be reported.

Percentage of participants with MG-ADL score of 0 or 1 over time in the double-blind, placebo-controlled phase will be reported.

Percentage of participants with MG-ADL score of 0 or 1 at any time during the double-blind, placebo-controlled phase will be reported.

Percentage of participants with MG-ADL score of 0 or 1 at 50% of timepoints during the double-blind, placebo-controlled phase will be reported.

Percentage of participants with MG-ADL score of 0 or 1 at 75% of timepoints during the double-blind, placebo-controlled phase will be reported.

Change in total serum IgG concentrations will be reported.

Change in levels of autoantibodies associated with gMG will be reported.

Change from baseline in MG-ADL score as a function of IgG will be reported.

Change from baseline in QMG score as a function of IgG will be reported.

Change from baseline in MG-ADL as a response to percent change in autoantibody levels, in seropositive participants (anti-acetylcholine receptor \[anti-AChR\], anti-muscle-specific kinase \[anti-MuSK\], anti-lipoprotein-related protein receptor 4 \[anti-LRP4\]) treated with nipocalimab will be reported.

Change from baseline in QMG score as a response to percent change in autoantibody levels, in seropositive participants (anti-AChR, anti-MuSK, anti-LRP4) treated with nipocalimab will be reported.

Janssen Research & Development, LLC

Participants age 2 to less than (\<) 18 years of age (globally) and 8 to \<18 years of age (for US sites only) will receive nipocalimab once every two weeks for 24 weeks. After Week 24, all participants will have the option to enroll in long term extension (LTE).

The purpose of this study is to determine the effect of nipocalimab on total serum immunoglobulin G (IgG) in pediatric participants 2 to less than (\<) 18 years of age (globally) and 8 to \<18 years of age (for Unites Stated (US) sites only), the safety and tolerability of treatment with nipocalimab in children and adolescents and to evaluate the pharmacokinetics (PK) of nipocalimab in children and adolescents with generalized myasthenia gravis (gMG) who have an insufficient clinical response to ongoing, stable standard-of-care therapy.

A Study of Nipocalimab in Children Aged 2 to Less Than 18 Years With Generalized Myasthenia Gravis

An Open-Label Uncontrolled Multicenter Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Safety and Activity of Nipocalimab in Children Aged 2 to Less Than 18 Years With Generalized Myasthenia Gravis

The data sharing policy of the Janssen Pharmaceutical Companies of Johnson and 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

Change from baseline in total serum IgG antibodies levels were reported.

Number of participants with infectious AEs will be reported. 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.

Number of participants with SAEs will be reported. A SAE is any AE that results in: death, persistent or significant disability/incapacity, requires inpatient hospitalization or prolongation of existing hospitalization, is life-threatening experience, is a congenital anomaly/birth defect, is suspected transmission of any infectious agent via a medicinal product, is medically important to prevent one of the outcomes listed above.

Number of participants with AESIs will be reported. Treatment-emergent AEs associated with the following situations are considered an AESI: a) infections that are severe or require intravenous (IV) anti-infective or operative/invasive intervention; b) hypoalbuminemia with albumin less than (\<)20 grams per liter (g/L) \[\<\] 2.0 grams per deciliter \[g/dL\]) and c) opportunistic infections. Any AE occurring at or after the initial administration of study intervention through end of study is treatment emergent.

Number of participants with abnormalities in clinical laboratory tests (including chemistry, hematology, coagulation, and urinalysis) will be reported.

Number of participants with abnormalities in vital signs including sitting pulse/heart rate, sitting systolic and diastolic blood pressure, and oral temperature (degrees Celsius) will be reported.

Number of participants with abnormalities in physical examinations including height, weight, assessments of the skin, head, eyes, ears, nose, throat, neck, thyroid, lungs, heart, abdomen, lymph nodes and extremities will be reported.

Serum samples will be analyzed to determine concentrations of nipocalimab using a validated, specific, and sensitive immunoassay method.

CL is defined as the volume of serum from which nipocalimab is completely removed per unit time.

V is defined as the representation of nipocalimab's propensity to either remain in the serum or redistribute to other tissue compartments.

t1/2 is defined as the time it takes for nipocalimab's active substance in the body to reduce by half.

Cpeak,ss is defined as the peak serum concentration of nipocalimab at steady state.

Ctrough,ss will be reported. It is defined as the observed serum concentration of nipocalimab just prior to the beginning of a dosing interval at steady state.

AUCss is defined as the area under the curve for nipocalimab at steady state.

Change from baseline in MG-ADL score will be reported. The MG-ADL score provides a rapid assessment of the participant's myasthenia gravis (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.

The QMG score is a standardized quantitative strength assessment comprising 13 components (and is administered by a trained qualified healthcare professional \[HCP\] eg, physician, physician assistant, nurse practitioner, nurse). 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.

The EQ-5D-Y is a standardized child friendly instrument for use as a measure of health status, primarily designed for self-completion by children and adolescents, or via a proxy version to be completed by the child's caregiver. The EQ-5D-Y descriptive system comprises the following 5 dimensions: Mobility, looking after myself (washing and dressing), usual activities, pain or discomfort and feeling worried or unhappy. Each of the 5 dimensions is divided into 3 levels of perceived problems (Level 1 indicating no problem, Level 2 indicating some problems, Level 3 a lot of problems).

The Neuro-QoL pediatric fatigue score will be used to assess the impact of fatigue in participants aged 10 to less than (\<) 18 years. The participant will rate each of the 11 items on a 5-point scale. Higher scores indicate greater fatigue.

The PGI-S score will be used to assess the severity of fatigue due to generalized myasthenia gravis (gMG) in participants aged 10 to \< 18 years. Participants will be asked to rate their fatigue over the past 7 days using the following 5-point scale: 1 = None, 2 = Mild, 3 = Moderate, 4 = Severe, and 5 = Very severe. Higher scores indicate greater severity of fatigue.

The PGI-C score will be used to assess if there has been an improvement or decline in patient-reported fatigue since the beginning of the treatment in participants aged 10 to \<18 years. Participants will be asked to rate their current fatigue as compared to when they started the study, using the following 7-point scale: 1 = Much better, 2 = Moderately better, 3 = A little better, 4 = No change, 5 = A little worse, 6 = Moderately worse, and 7 = Much worse. Higher scores indicate greater change in overall fatigue.

Number of participants with ADAs to nipocalimab will be reported.

Number of participants with NAbs to nipocalimab will be reported.

Number of participants with vaccine antibody titers to diphtheria or tetanus will be reported.

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
A Study of Nipocalimab Administered to Adults With Generalized Myasthenia Gravis	NCT04951622	Myasthenia Grav...	Nipocalimab Placebo	18 Years -	Janssen Research & Development, LLC
A Study of Nipocalimab Administered to Adults With Generalized Myasthenia Gravis	NCT04951622	Myasthenia Grav...	Nipocalimab Placebo	18 Years -	Janssen Research & Development, LLC
A Study of Nipocalimab in Children Aged 2 to Less Than 18 Years With Generalized Myasthenia Gravis	NCT05265273	Myasthenia Grav...	Nipocalimab	2 Years - 17 Years	Janssen Research & Development, LLC
A Study of Nipocalimab Administered to Adults With Generalized Myasthenia Gravis	NCT04951622	Myasthenia Grav...	Nipocalimab Placebo	18 Years -	Janssen Research & Development, LLC

Spots Global Cancer Trial Database for nipocalimab

Every month we try and update this database with for nipocalimab cancer trials from around the world to help patients and their families find trials that might be right for them.
We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.

Spots Global Cancer Trial Database for nipocalimab

Every month we try and update this database with for nipocalimab cancer trials from around the world to help patients and their families find trials that might be right for them. We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.

Every month we try and update this database with for nipocalimab cancer trials from around the world to help patients and their families find trials that might be right for them.
We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.