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

Launched by JANSSEN RESEARCH & DEVELOPMENT, LLC · Feb 22, 2022

Keywords

ClinConnect Summary

This clinical trial is studying a medication called nipocalimab for children and teenagers aged 2 to less than 18 years who have a condition known as generalized myasthenia gravis (gMG). gMG causes weakness in the muscles, which can affect everyday activities. The main goals of the study are to see how nipocalimab affects certain antibodies (called immunoglobulin G) in the blood, as well as to evaluate how safe it is for young patients and how the body processes the medication. The study is currently recruiting participants and aims to help those who are not getting enough benefit from their current treatments.

To be eligible for the trial, children must be diagnosed with gMG and show specific signs of muscle weakness. For sites in the United States, participants need to be between 8 and less than 18 years old. They should have a positive test for certain antibodies related to gMG and be in stable health overall. Participants can expect to receive the nipocalimab treatment and will have regular check-ups to monitor their health and response to the medication. This study is an important step in finding better treatments for children with this condition, and parents should discuss any questions or concerns with their healthcare provider.

Gender

ALL

Eligibility criteria

• 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