Clinical trial NCT05265273: 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
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE2
PHASE3
Conditions: Myasthenia Gravis
Keywords: Pediatric
Lead sponsor: Janssen Research & Development, LLC
Sponsor class: INDUSTRY
First posted: 2022-03-03
Enrollment: 12
Sex: ALL
Minimum age (years): 2
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


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

Primary outcomes:
measure: Change from Baseline in Total Serum Immunoglobulin-G (IgG) Levels
timeFrame: Up to 3 years
measure: Number of Participants with Infectious Adverse Events (AEs)
timeFrame: Up to 3 years
measure: Number of Participants with Serious AEs (SAEs)
timeFrame: Up to 3 years
measure: Number of Participants with Adverse Events of Special Interests (AESIs)
timeFrame: Up to 3 years
measure: Number of Participants with Abnormalities in Clinical Laboratory Tests
timeFrame: Up to 3 years
measure: Number of Participants with Abnormalities in Vital Signs
timeFrame: Up to 3 years
measure: Number of Participants with Abnormalities in Physical Examination
timeFrame: Up to 3 years
measure: Serum Concentration of Nipocalimab over Time
timeFrame: Up to 3 years
measure: Clearance (CL) of Nipocalimab
timeFrame: Up to 3 years
measure: Volume of Distribution (V) of Nipocalimab
timeFrame: Up to 3 years
measure: Half-life (t1/2) of Nipocalimab
timeFrame: Up to 3 years
measure: Steady-state Peak Concentration (Cpeak,ss) of Nipocalimab
timeFrame: Up to 3 years
measure: Steady-state Trough concentration (Ctrough,ss) of Nipocalimab
timeFrame: Up to 3 years
measure: Steady-state Area Under the Curve (AUCss) of Nipocalimab
timeFrame: Up to 3 years
Secondary outcomes:
measure: Change from Baseline in Myasthenia Gravis -Activities of Daily Living (MG-ADL) Score
timeFrame: Up to 3 years
measure: Change in the Quantitative Myasthenia Gravis (QMG) Score
timeFrame: Up to 3 years
measure: European Quality of Life 5-Dimension Youth (EQ-5D-Y) Tool Score
timeFrame: Up to 3 years
measure: Neurological Quality of Life (Neuro-QoL) Pediatric Fatigue Score
timeFrame: Up to 3 years
measure: Patient Global Impression of Severity (PGI-S) Score
timeFrame: Up to 3 years
measure: Patient Global Impression of Change (PGI-C) Score
timeFrame: Up to 3 years
measure: Number of Participants with Anti-Drug Antibodies [ADAs] to Nipocalimab
timeFrame: Up to 3 years
measure: Number of Participants with Neutralizing Antibodies (NAbs) to Nipocalimab
timeFrame: Up to 3 years
measure: Number of Participants with Vaccine Antibody Titers to Diphtheria or Tetanus
timeFrame: Up to 3 years
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Phoenix Children's Hospital
state: Arizona
city: Los Angeles
country: United States
facility: Childrens Hospital Los Angeles
state: California
city: Palo Alto
country: United States
facility: Lucile Packard Children's Hospital Stanford
state: California
city: San Francisco
country: United States
facility: UCSF Benioff Children's Hospital
state: California
city: Aurora
country: United States
facility: Children's Hospital Colorado
state: Colorado
city: Tampa
country: United States
facility: University of South Florida Morsani Center for Advanced Healthcare
state: Florida
city: Lawrence
country: United States
facility: University of Kansas Medical Center
state: Kansas
city: Ann Arbor
country: United States
facility: C.S. Mott Children's Hospital
state: Michigan
city: Hershey
country: United States
facility: Penn State Milton S Hershey Medical Ctr
state: Pennsylvania
city: Philadelphia
country: United States
facility: Childrens Hospital Of Philadelphia
state: Pennsylvania
city: Pittsburgh
country: United States
facility: University of Pittsburgh Medical Center
state: Pennsylvania
city: Azumino-shi
country: Japan
facility: Nagano Children's Hospital
city: Chiba
country: Japan
facility: Chiba University Hospital
city: Miyazaki
country: Japan
facility: University of Miyazaki Hospital
city: Nishinomiya-Shi
country: Japan
facility: Hyogo College of Medicine Hospital
city: Saitama shi
country: Japan
facility: Saitama Prefecture Children's Medical Center
city: Shinjuku-ku
country: Japan
facility: Tokyo Women's Medical University Hospital
city: Leiden
country: Netherlands
facility: Leiden University Medical Center
city: Gdansk
country: Poland
facility: Uniwersyteckie Centrum Kliniczne
Study officials:
affiliation: Janssen Research & Development, LLC
name: Janssen Research & Development, LLC Clinical Trial
role: STUDY_DIRECTOR
References: