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

Official title

An Open-Label Uncontrolled Multicenter Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Safety and Activity of Nipocalimab in Children Aged 2 to Less Than 18 Years With Generalized Myasthenia Gravis

Overview

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.

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]
• 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]

Secondary outcome measures (9)

• Change from Baseline in Myasthenia Gravis -Activities of Daily Living (MG-ADL) Score [Time frame: Up to 3 years]
• Change in the Quantitative Myasthenia Gravis (QMG) Score [Time frame: Up to 3 years]
• European Quality of Life 5-Dimension Youth (EQ-5D-Y) Tool Score [Time frame: Up to 3 years]
• Neurological Quality of Life (Neuro-QoL) Pediatric Fatigue Score [Time frame: Up to 3 years]
• Patient Global Impression of Severity (PGI-S) Score [Time frame: Up to 3 years]
• Patient Global Impression of Change (PGI-C) Score [Time frame: Up to 3 years]
• Number of Participants with Anti-Drug Antibodies [ADAs] to Nipocalimab [Time frame: Up to 3 years]
• Number of Participants with Neutralizing Antibodies (NAbs) to Nipocalimab [Time frame: Up to 3 years]
• Number of Participants with Vaccine Antibody Titers to Diphtheria or Tetanus [Time frame: Up to 3 years]

Eligibility criteria

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

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

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation

N/A

Model

Single group

Masking

Open label

Primary purpose

Treatment

Study locations

United States · 11 centers

• Phoenix Children's Hospital — Phoenix
• Childrens Hospital Los Angeles — Los Angeles
• Lucile Packard Children's Hospital Stanford — Palo Alto
• UCSF Benioff Children's Hospital — San Francisco
• Children's Hospital Colorado — Aurora
• University of South Florida Morsani Center for Advanced Healthcare — Tampa
• University of Kansas Medical Center — Lawrence
• C.S. Mott Children's Hospital — Ann Arbor
• … and 3 more centers

Japan · 6 centers

• Nagano Children's Hospital — Azumino-shi
• Chiba University Hospital — Chiba
• University of Miyazaki Hospital — Miyazaki
• Hyogo College of Medicine Hospital — Nishinomiya-Shi
• Saitama Prefecture Children's Medical Center — Saitama Shi
• Tokyo Women's Medical University Hospital — Shinjuku-ku

Netherlands · 1 center

• Leiden University Medical Center — Leiden

Poland · 1 center

• Uniwersyteckie Centrum Kliniczne — Gdansk

Identifiers

NCT: NCT05265273 · CR109137 · 80202135MYG2001 · 2021-002479-20 · 2022-502539-21-00

Primary sources (government registries)

• ClinicalTrials.gov (US NIH)

View this study on ClinicalTrials.gov ↗