Recruiting

Phase 2

Phase 3

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

Sponsor:

Janssen Research & Development, LLC

Code:

NCT05265273

Conditions

Myasthenia Gravis

Eligibility Criteria

Sex: All

Age: 2 - 17

Healthy Volunteers: Not accepted

Interventions

Nipocalimab

Study Details

Brief 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.

Conditions

Myasthenia Gravis

Study ID

NCT05265273

Start date

Jul 20, 2022

Status verified date

Jul, 2026

Completion date

Jul 2, 2029

Anticipated

Primary completion date

Jun 30, 2027

Anticipated

Eligibility Criteria

Sex: All

Age: 2 - 17

Healthy Volunteers: Not accepted

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

Study Design

Enrollment

12 participants

Anticipated

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

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 age-adolescents 12 to <18 years and children 2 to <12 years and 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).

Interventions

Nipocalimab

Nipocalimab will be administered as an IV infusion.

Primary outcome measure

Change from Baseline in Total Serum Immunoglobulin-G (IgG) Levels [ Time Frame: Up to 3 years ]
Number of Participants with Infectious Adverse Events (AEs) [ Time Frame: Up to 3 years ]
Number of Participants with Serious AEs (SAEs) [ Time Frame: Up to 3 years ]
Number of Participants with Adverse Events of Special Interests (AESIs) [ Time Frame: Up to 3 years ]
Number of Participants with Abnormalities in Clinical Laboratory Tests [ Time Frame: Up to 3 years ]
Number of Participants with Abnormalities in Vital Signs [ Time Frame: Up to 3 years ]
Number of Participants with Abnormalities in Physical Examination [ Time Frame: Up to 3 years ]
Serum Concentration of Nipocalimab over Time [ Time Frame: Up to 3 years ]
Clearance (CL) of Nipocalimab [ Time Frame: Up to 3 years ]
Volume of Distribution (V) of Nipocalimab [ Time Frame: Up to 3 years ]
Half-life (t1/2) of Nipocalimab [ Time Frame: Up to 3 years ]
Steady-state Peak Concentration (Cpeak,ss) of Nipocalimab [ Time Frame: Up to 3 years ]
Steady-state Trough concentration (Ctrough,ss) of Nipocalimab [ Time Frame: Up to 3 years ]
Steady-state Area Under the Curve (AUCss) of Nipocalimab [ Time Frame: Up to 3 years ]

Central Contacts and Locations

Central contacts

Study Contact

844-434-4210 Participate-In-This-Study1@its.jnj.com

Locations

Phoenix Children's Hospital

Recruiting

Phoenix, Arizona, United States, 85016

Lucile Packard Children's Hospital Stanford

Recruiting

Palo Alto, California, United States, 94304

UCSF Benioff Children's Hospital

Recruiting

San Francisco, California, United States, 94158

Children's Hospital Colorado

Recruiting

Aurora, Colorado, United States, 80045

University of South Florida Morsani Center for Advanced Healthcare

Recruiting

Tampa, Florida, United States, 33613

C.S. Mott Children's Hospital

Recruiting

Ann Arbor, Michigan, United States, 48109

Penn State Milton S Hershey Medical Ctr

Recruiting

Hershey, Pennsylvania, United States, 17033

Childrens Hospital Of Philadelphia

Recruiting

Philadelphia, Pennsylvania, United States, 19106

More Information

Sponsor

Janssen Research & Development, LLC

Last update posted

Jul 31, 2026

Last verified

Jul, 2026

Keywords

Pediatric

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-24. This information was provided to ClinicalTrials.gov by Janssen Research & Development, LLC on 2026-07-31. Recruitment status is synced daily from ClinicalTrials.gov and may not reflect the sponsor's current status. Confirm during your call.