NCT05265273 · Janssen Research & Development, LLC
A Study of Nipocalimab in Children Aged 2 to Less Than 18 Years With Generalized Myasthenia Gravis
What this study is about
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 how well patients handle the treatment of treatment with nipocalimab in children and adolescents and to evaluate the how the drug moves through the body (PK) of nipocalimab in children and adolescents with generalized myasthenia gravis (gMG) who have an insufficient clinical response to
View original scientific description
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
Interventions
DRUG
Nipocalimab
Nipocalimab will be administered as an IV infusion.
Primary outcome measures
Change from Baseline in Total Serum Immunoglobulin-G (IgG) Levels
Time frame: Up to 3 years
Change from baseline in total serum IgG levels were reported.
Number of Participants with Infectious Adverse Events (AEs)
Time frame: 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)
Time frame: 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)
Time frame: 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
Time frame: 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
Time frame: 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
Time frame: 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
Time frame: 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
Time frame: 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
Time frame: 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
Time frame: 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
Time frame: 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
Time frame: 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
Time frame: Up to 3 years
AUCss is defined as the area under the curve for nipocalimab at steady state.
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
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 an
Where
- Phoenix, Arizona
- Los Angeles, California
- Palo Alto, California
- San Francisco, California
- Aurora, Colorado
- Tampa, Florida
- Lawrence, Kansas
- Ann Arbor, Michigan
- Hershey, Pennsylvania
- Philadelphia, Pennsylvania
- Pittsburgh, Pennsylvania
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Jul 6, 2026 · Source of record for eligibility and locations