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

• Register: ClinicalTrials.gov
• Last refreshed on: 21 April 2025
• Main ID: NCT05265273
• Date of registration: 22/02/2022
• Prospective Registration: Yes
• Primary sponsor: Janssen Research & Development, LLC
• Public title: A Study of Nipocalimab in Children Aged 2 to Less Than 18 Years With Generalized Myasthenia Gravis
• Scientific 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
• Date of first enrolment: July 20, 2022
• Target sample size: 12
• Recruitment status: Recruiting
• URL: https://clinicaltrials.gov/ct2/show/NCT05265273
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2/Phase 3

Countries of recruitment

Canada, Japan, Netherlands, Poland, United States

Contacts

• Name: Janssen Research & Development, LLC Clinical Trial
• Affiliation: Janssen Research & Development, LLC

• Name: Study Contact

Key inclusion & exclusion 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


Age minimum: 2 Years
Age maximum: 17 Years
Gender: All

Health Condition(s) or Problem(s) studied

Myasthenia Gravis

Intervention(s)

Drug: Nipocalimab

Primary Outcome(s)

Number of Participants with Abnormalities in Vital Signs [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]

Steady-state Trough concentration (Ctrough,ss) of Nipocalimab [Time Frame: Up to 3 years]

Steady-state Peak Concentration (Cpeak,ss) of Nipocalimab [Time Frame: Up to 3 years]

Serum Concentration of Nipocalimab over Time [Time Frame: Up to 3 years]

Steady-state Area Under the Curve (AUCss) of Nipocalimab [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 Adverse Events of Special Interests (AESIs) [Time Frame: Up to 3 years]

Number of Participants with Abnormalities in Physical Examination [Time Frame: Up to 3 years]

Volume of Distribution (V) of Nipocalimab [Time Frame: Up to 3 years]

Change from Baseline in Total Serum Immunoglobulin-G (IgG) Levels [Time Frame: Up to 3 years]

Clearance (CL) of Nipocalimab [Time Frame: Up to 3 years]

Half-life (t1/2) of Nipocalimab [Time Frame: Up to 3 years]

Secondary Outcome(s)

European Quality of Life 5-Dimension Youth (EQ-5D-Y) Tool Score [Time Frame: Up to 3 years]

Patient Global Impression of Change (PGI-C) Score [Time Frame: Up to 3 years]

Change from Baseline in Myasthenia Gravis -Activities of Daily Living (MG-ADL) Score [Time Frame: Up to 3 years]

Neurological Quality of Life (Neuro-QoL) Pediatric Fatigue 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 Vaccine Antibody Titers to Diphtheria or Tetanus [Time Frame: Up to 3 years]

Number of Participants with Neutralizing Antibodies (NAbs) to Nipocalimab [Time Frame: Up to 3 years]

Patient Global Impression of Severity (PGI-S) Score [Time Frame: Up to 3 years]

Change in the Quantitative Myasthenia Gravis (QMG) Score [Time Frame: Up to 3 years]

Secondary ID(s)

2021-002479-20

CR109137

80202135MYG2001

2022-502539-21-00

Source(s) of Monetary Support

Please refer to primary and secondary sponsors