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NCT05265273PHASE2, PHASE3Recruiting

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

Janssen Research & Development, LLC

Start Date

7/20/2022

Completion Date

7/2/2029

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 Criteria

Age Range: 2 years to 17 years

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

Interventions

DRUG

Nipocalimab

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Conditions

Myasthenia Gravis

Locations

Phoenix Children's Hospital

Phoenix, Arizona 85016

United States

Childrens Hospital Los Angeles

Los Angeles, California 90027

United States

Lucile Packard Children's Hospital Stanford

Palo Alto, California 94304

United States

UCSF Benioff Children's Hospital

San Francisco, California 94158

United States

Children's Hospital Colorado

Aurora, Colorado 80045

United States

University of South Florida Morsani Center for Advanced Healthcare

Tampa, Florida 33613

United States

University of Kansas Medical Center

Lawrence, Kansas 66045

United States

C.S. Mott Children's Hospital

Ann Arbor, Michigan 48109

United States

Penn State Milton S Hershey Medical Ctr

Hershey, Pennsylvania 17033

United States

Childrens Hospital Of Philadelphia

Philadelphia, Pennsylvania 19106

United States

University of Pittsburgh Medical Center

Pittsburgh, Pennsylvania 15224

United States

Nagano Children's Hospital

Azumino-shi, 399-8288

Japan

Chiba University Hospital

Chiba, 260 8677

Japan

University of Miyazaki Hospital

Miyazaki, 889-1692

Japan

Hyogo College of Medicine Hospital

Nishinomiya-Shi, 663-8501

Japan

Saitama Prefecture Children's Medical Center

Saitama Shi, 330-8777

Japan

Tokyo Women's Medical University Hospital

Shinjuku-ku, 162-8666

Japan

Leiden University Medical Center

Leiden, 2333 ZA

Netherlands

Uniwersyteckie Centrum Kliniczne

Gdansk, 80 211

Poland