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

NCT05265273 | Recruiting Phase 2 DMC FDA Drug

• 4 other identifiers: CR10913780202135MYG20012021-002479-202022-502539-21-00
• Study Type: interventional
• Target: 12
• Locations: 4 countries
• Sites: 19

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.

Conditions

Myasthenia Gravis

Keywords

Pediatric

Outcome Measures

Primary Outcomes (14)

• Change from Baseline in Total Serum Immunoglobulin-G (IgG) Levels

  Change from baseline in total serum IgG levels were reported.

  Up to 3 years

• Number of Participants with Infectious Adverse Events (AEs)

  Number of participants with infectious AEs will be reported. An AE is any untoward medical occurrence in a participant participating in a clinical study that does not necessarily have a causal relationship with the pharmaceutical/biological agent under study.

  Up to 3 years

• Number of Participants with Serious AEs (SAEs)

  Number of participants with SAEs will be reported. A SAE is any AE that results in: death, persistent or significant disability/incapacity, requires inpatient hospitalization or prolongation of existing hospitalization, is life-threatening experience, is a congenital anomaly/birth defect, is suspected transmission of any infectious agent via a medicinal product, is medically important to prevent one of the outcomes listed above.

  Up to 3 years

• Number of Participants with Adverse Events of Special Interests (AESIs)

  Number of participants with AESIs will be reported. Treatment-emergent AEs associated with the following situations are considered an AESI: a) infections that are severe or require intravenous (IV) anti-infective or operative/invasive intervention; b) hypoalbuminemia with albumin less than (\<)20 grams per liter (g/L) \[\<\] 2.0 grams per deciliter \[g/dL\]) c) opportunistic infections and d) Serious and non-serious deep-vein thrombosis (DVT) and/or pulmonary embolism (PE). Any AE occurring at or after the initial administration of study intervention through end of study is treatment emergent.

  Up to 3 years

• Number of Participants with Abnormalities in Clinical Laboratory Tests

  Number of participants with abnormalities in clinical laboratory tests (including chemistry, hematology, coagulation, and urinalysis) will be reported.

  Up to 3 years

• Number of Participants with Abnormalities in Vital Signs

  Number of participants with abnormalities in vital signs including sitting pulse/heart rate, sitting systolic and diastolic blood pressure, and oral temperature (degrees Celsius) will be reported.

  Up to 3 years

• Number of Participants with Abnormalities in Physical Examination

  Number of participants with abnormalities in physical examinations including height, weight, assessments of the skin, head, eyes, ears, nose, throat, neck, thyroid, lungs, heart, abdomen, lymph nodes and extremities will be reported.

  Up to 3 years

• Serum Concentration of Nipocalimab over Time

  Serum samples will be analyzed to determine concentrations of nipocalimab using a validated, specific, and sensitive immunoassay method.

  Up to 3 years

• Clearance (CL) of Nipocalimab

  CL is defined as the volume of serum from which nipocalimab is completely removed per unit time.

  Up to 3 years

• Volume of Distribution (V) of Nipocalimab

  V is defined as the representation of nipocalimab's propensity to either remain in the serum or redistribute to other tissue compartments.

  Up to 3 years

• Half-life (t1/2) of Nipocalimab

  t1/2 is defined as the time it takes for nipocalimab's active substance in the body to reduce by half.

  Up to 3 years

• Steady-state Peak Concentration (Cpeak,ss) of Nipocalimab

  Cpeak,ss is defined as the peak serum concentration of nipocalimab at steady state.

  Up to 3 years

• Steady-state Trough concentration (Ctrough,ss) of Nipocalimab

  Ctrough,ss will be reported. It is defined as the observed serum concentration of nipocalimab just prior to the beginning of a dosing interval at steady state.

  Up to 3 years

• Steady-state Area Under the Curve (AUCss) of Nipocalimab

  AUCss is defined as the area under the curve for nipocalimab at steady state.

  Up to 3 years

Secondary Outcomes (9)

• Change from Baseline in Myasthenia Gravis -Activities of Daily Living (MG-ADL) Score

  Up to 3 years

• Change in the Quantitative Myasthenia Gravis (QMG) Score

  Up to 3 years

• European Quality of Life 5-Dimension Youth (EQ-5D-Y) Tool Score

  Up to 3 years

• Neurological Quality of Life (Neuro-QoL) Pediatric Fatigue Score

  Up to 3 years

• Patient Global Impression of Severity (PGI-S) Score

  Up to 3 years

Study Arms (1)

Nipocalimab

EXPERIMENTAL

Participants age 2 to less than (\<) 18 years of age (globally) and 8 to \<18 years of age (for US sites only) will be divided into 2 cohorts as per their age-adolescents 12 to \<18 years and children 2 to \<12 years and will receive nipocalimab once every two weeks for 24 weeks. After Week 24, all participants will have the option to enroll in long term extension (LTE).

Drug: Nipocalimab

Interventions

Nipocalimab DRUG

Nipocalimab will be administered as an IV infusion.

Also known as: M281/JNJ-80202135

Nipocalimab

Eligibility Criteria

• Age: 2 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Janssen Research & Development, LLC: lead

Study Sites (19)

Phoenix Children's Hospital

Phoenix, Arizona, 85016, United States

RECRUITING

Childrens Hospital Los Angeles

Los Angeles, California, 90027, United States

RECRUITING

Lucile Packard Children's Hospital Stanford

Palo Alto, California, 94304, United States

RECRUITING

UCSF Benioff Children's Hospital

San Francisco, California, 94158, United States

RECRUITING

Children's Hospital Colorado

Aurora, Colorado, 80045, United States

RECRUITING

University of South Florida Morsani Center for Advanced Healthcare

Tampa, Florida, 33613, United States

RECRUITING

University of Kansas Medical Center

Lawrence, Kansas, 66045, United States

COMPLETED

C.S. Mott Children's Hospital

Ann Arbor, Michigan, 48109, United States

RECRUITING

Penn State Milton S Hershey Medical Ctr

Hershey, Pennsylvania, 17033, United States

RECRUITING

Childrens Hospital Of Philadelphia

Philadelphia, Pennsylvania, 19106, United States

RECRUITING

University of Pittsburgh Medical Center

Pittsburgh, Pennsylvania, 15224, United States

TERMINATED

Nagano Children's Hospital

Azumino-shi, 399-8288, Japan

RECRUITING

Chiba University Hospital

Chiba, 260 8677, Japan

COMPLETED

University of Miyazaki Hospital

Miyazaki, 889-1692, Japan

RECRUITING

Hyogo College of Medicine Hospital

Nishinomiya-Shi, 663-8501, Japan

RECRUITING

Saitama Prefecture Children's Medical Center

Saitama Shi, 330-8777, Japan

RECRUITING

Tokyo Women's Medical University Hospital

Shinjuku-ku, 162-8666, Japan

RECRUITING

Leiden University Medical Center

Leiden, 2333 ZA, Netherlands

RECRUITING

Uniwersyteckie Centrum Kliniczne

Gdansk, 80 211, Poland

RECRUITING

MeSH Terms

Conditions

Myasthenia Gravis

Condition Hierarchy (Ancestors)

Paraneoplastic Syndromes, Nervous System; Nervous System Neoplasms; Neoplasms by Site; Neoplasms; Paraneoplastic Syndromes; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Neurodegenerative Diseases; Neuromuscular Junction Diseases; Neuromuscular Diseases; Autoimmune Diseases; Immune System Diseases

Study Officials

• Janssen Research & Development, LLC Clinical Trial

  Janssen Research & Development, LLC

  STUDY DIRECTOR

Central Study Contacts

Study Contact

CONTACT

844-434-4210; Participate-In-This-Study1@its.jnj.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR
• Expanded Access: Yes

Study Record Dates

• First Submitted: February 22, 2022
• First Posted: March 3, 2022
• Study Start: July 20, 2022
• Primary Completion (Estimated): June 26, 2026
• Study Completion (Estimated): July 2, 2029
• Last Updated: March 13, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will share

Locations

US (11); NL (1); JP (6); PL (1)