Myasthenia Gravis
Conditions
Interventions
Experimental: Nipocalimab
Participants age 2 to less than (<) 18 years of age (globally) and 8 to <18 years of age (for US sites only) will be divided into 2 cohorts as per their ageadolescents 12 to
Sponsors
Tominaga Yushin
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: - Age: For US sites only: 8 to <18 years - 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 IIa/b, IIIa/b, or IVa/b at screening - Has a positive serologic test for acetylcholine receptor (anti-AChR) antibodies or muscle-specific tyrosine kinase (anti-MuSK) antibodies at screening - A participant using herbal, naturopathic, traditional Chinese remedies, ayurvedic or nutritional supplements, or medical marijuana (with a doctor's prescription) is eligible if the use of these medications is acceptable to the Investigator. These remedies must remain at a stable dose and regimen throughout the study - Has sufficient venous access to allow drug administration by infusion and blood sampling as per the protocol - Participants should have a body weight and body mass index between 5th and 95th percentile for age and sex. Obese participants greater than 95th percentile and underweight participants below 5th percentile may participate following medical clearance - A female 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
Exclusion criteria
Exclusion criteria: - Has a history of severe and/or uncontrolled hepatic (example, viral/alcoholic/ autoimmune hepatitis/ cirrhosis/ and/or metabolic liver disease), gastrointestinal, renal, pulmonary, cardiovascular (including congenital heart diseases), psychiatric, neurological or musculoskeletal disorder or any other medical disorder(s) (example, diabetes mellitus), or clinically significant abnormalities in screening laboratory, that might interfere with participant's full participation in the study, and/or might jeopardize the safety of the participant or the validity of the study results - Has any confirmed or suspected clinical immunodeficiency syndrome not related to treatment of his/her generalized myasthenia gravis (gMG), or has a family history of congenital or hereditary immunodeficiency unless confirmed absent in the participant - Has had a thymectomy within 12 months prior to screening, or thymectomy is planned during the Active treatment Phase of the study - Has shown a previous severe immediate hypersensitivity reaction, such as anaphylaxis to therapeutic proteins (example, monoclonal antibodies) - Has experienced myocardial infarction, unstable ischemic heart disease, or stroke within 12 weeks of screening
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 1. Change from Baseline in Total Serum Immunoglobulin-G (IgG) Levels Change from baseline in total serum IgG levels were reported. [Time Frame: Up to 3 years] 2. Number of Participants with Infectious Adverse Events (AEs) 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. [Time Frame: Up to 3 years] 3. Number of Participants with Serious AEs (SAEs) 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. [Time Frame: Up to 3 years] 4. Number of Participants with Adverse Events of Special Interests (AESIs) 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]) c) opportunistic infections and d) Serious and non-serious deep-vein thrombosis (DVT) and/or pulmonary embolism (PE). Any AE occurring at or after the initial administration of study intervention through end of study is treatment emergent. [Time Frame: Up to 3 years] 5. Number of Participants with Abnormalities in Clinical Laboratory Tests Number of participants with abnormalities in clinical laboratory tests (including chemistry, hematology, coagulation, and urinalysis) will be reported. [Time Frame: Up to 3 years] 6. Number of Participa | — |
Secondary
| Measure | Time frame |
|---|---|
| 15. Change from Baseline in Myasthenia Gravis -Activities of Daily Living (MG-ADL) Score 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. [Time Frame: Up to 3 years] 16. Change in the Quantitative Myasthenia Gravis (QMG) Score 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. [Time Frame: Up to 3 years] 17. European Quality of Life 5-Dimension Youth (EQ-5D-Y) Tool Score 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). [Time Frame: Up to 3 years] 18. Neurological Quality of Life (Neuro-QoL) Pedia | — |
Countries
Japan, Netherlands, Poland, United States Of America
Contacts
Public ContactInformation Center Medical
Janssen Pharmaceutical K.K.
Outcome results
None listed