Nipocalimab: Nipocalimab will be administered as an IV infusion.
Study summary
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.
Eligibility
Sex
ALL
Min age
2 Years
Max age
17 Years
Healthy volunteers
No
Key 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
Key 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 musculoskeletal disorder, any other medical disorder(s) (example, diabetes mellitus), risk factors for thrombosis events (example, a history of venous thromboembolism \[VTE\] or antiphospholipid syndrome, or a personal or family history of heritable coagulation disorder such as factor V leiden, protein S or protein C deficiency, atrial fibrillation/flutter, major orthopedic surgery or significant trauma that may increase the risk of VTE, is expected to be immobilized for prolonged periods of time), or has 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
Primary outcome measure(s)
Change from Baseline in Total Serum Immunoglobulin-G (IgG) Levels — Up to 3 years Change from baseline in total serum IgG levels were reported.
Number of Participants with Infectious Adverse Events (AEs) — Up to 3 years 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 Serious AEs (SAEs) — Up to 3 years 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 Adverse Events of Special Interests (AESIs) — Up to 3 years 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.
Number of Participants with Abnormalities in Clinical Laboratory Tests — Up to 3 years 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 — Up to 3 years 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 Examination — Up to 3 years 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 Concentration of Nipocalimab over Time — Up to 3 years Serum samples will be analyzed to determine concentrations of nipocalimab using a validated, specific, and sensitive immunoassay method.
Clearance (CL) of Nipocalimab — Up to 3 years CL is defined as the volume of serum from which nipocalimab is completely removed per unit time.
Volume of Distribution (V) of Nipocalimab — Up to 3 years V is defined as the representation of nipocalimab's propensity to either remain in the serum or redistribute to other tissue compartments.
Half-life (t1/2) of Nipocalimab — Up to 3 years t1/2 is defined as the time it takes for nipocalimab's active substance in the body to reduce by half.
Steady-state Peak Concentration (Cpeak,ss) of Nipocalimab — Up to 3 years Cpeak,ss is defined as the peak serum concentration of nipocalimab at steady state.
Steady-state Trough concentration (Ctrough,ss) of Nipocalimab — Up to 3 years 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.
Steady-state Area Under the Curve (AUCss) of Nipocalimab — Up to 3 years AUCss is defined as the area under the curve for nipocalimab at steady state.
Trial sites (19)
Facility
City
Region
Status
Phoenix Children's Hospital
Phoenix
Arizona
Recruiting
Childrens Hospital Los Angeles
Los Angeles
California
Completed
Lucile Packard Children's Hospital Stanford
Palo Alto
California
Recruiting
UCSF Benioff Children's Hospital
San Francisco
California
Recruiting
Children's Hospital Colorado
Aurora
Colorado
Recruiting
University of South Florida Morsani Center for Advanced Healthcare
Tampa
Florida
Recruiting
University of Kansas Medical Center
Lawrence
Kansas
Completed
C.S. Mott Children's Hospital
Ann Arbor
Michigan
Recruiting
Penn State Milton S Hershey Medical Ctr
Hershey
Pennsylvania
Recruiting
Childrens Hospital Of Philadelphia
Philadelphia
Pennsylvania
Recruiting
University of Pittsburgh Medical Center
Pittsburgh
Pennsylvania
Terminated
Nagano Children's Hospital
Azumino-shi
Japan
Recruiting
Chiba University Hospital
Chiba
Japan
Completed
University of Miyazaki Hospital
Miyazaki
Japan
Recruiting
Hyogo College of Medicine Hospital
Nishinomiya-Shi
Japan
Recruiting
Saitama Prefecture Children's Medical Center
Saitama Shi
Japan
Recruiting
Tokyo Women's Medical University Hospital
Shinjuku-ku
Japan
Recruiting
Leiden University Medical Center
Leiden
Netherlands
Recruiting
Uniwersyteckie Centrum Kliniczne
Gdansk
Poland
Recruiting
More Janssen Research & Development, LLC trials in the Netherlands
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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