NCT05265273 - A Study of Nipocalimab in Children Aged 2 to Less Than 18 Years With Generalized Myasthenia Gravis | Crick

Eligibility

Sex: ALLMin age: 2 YearsMax age: 17 Years

Medical Language ↔ Plain English

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

Locations (19)

Phoenix Children's Hospital

Phoenix, Arizona, United States

RECRUITING

Childrens Hospital Los Angeles

Los Angeles, California, United States

RECRUITING

Lucile Packard Children's Hospital Stanford

Palo Alto, California, United States

RECRUITING

UCSF Benioff Children's Hospital

San Francisco, California, United States

RECRUITING

Children's Hospital Colorado

Aurora, Colorado, United States

RECRUITING

University of South Florida Morsani Center for Advanced Healthcare

Tampa, Florida, United States

RECRUITING

University of Kansas Medical Center

Lawrence, Kansas, United States

COMPLETED

C.S. Mott Children's Hospital

Ann Arbor, Michigan, United States

RECRUITING

Penn State Milton S Hershey Medical Ctr

Hershey, Pennsylvania, United States

RECRUITING

Childrens Hospital Of Philadelphia

Philadelphia, Pennsylvania, United States

RECRUITING

...and 9 more locations

Outcomes

Primary Outcomes

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

Change from baseline in total serum IgG levels were reported.

Time frame: 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.

Time frame: 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.

Time frame: 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.

Time frame: 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.

Time frame: 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.

Time frame: 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.

Time frame: 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.

Time frame: 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.

Time frame: 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.

Time frame: 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.

Time frame: 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.

Time frame: 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.

Time frame: 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.

Time frame: Up to 3 years

Secondary Outcomes

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

Change from baseline in MG-ADL score will be reported. The MG-ADL score provides a rapid assessment of the participant's myasthenia gravis (MG) symptom severity. Eight functions (talking, chewing, swallowing, breathing, impairment of ability to brush teeth or comb hair, impairment of ability to arise from a chair, double vision, eyelid droop) are rated on a 4-point scale: 0 (no impairment) to 3 (severe impairment). The total score will be sum of eight function scores and can range from 0 to 24. A higher score indicates greater symptom severity.

Time frame: Up to 3 years

Change in the Quantitative Myasthenia Gravis (QMG) Score

The QMG score is a standardized quantitative strength assessment comprising 13 components (and is administered by a trained qualified healthcare professional \[HCP\] eg, physician, physician assistant, nurse practitioner, nurse). The quantitative results of each strength component are mapped to the following 4-point scale: 0 equals to (=) none, 1 = mild, 2 = moderate and 3 = severe. The total score will be sum of 13 components scores and can range from 0 to 39. A higher score indicates greater weakness.

Time frame: Up to 3 years

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

The EQ-5D-Y is a standardized child friendly instrument for use as a measure of health status, primarily designed for self-completion by children and adolescents, or via a proxy version to be completed by the child's caregiver. The EQ-5D-Y descriptive system comprises the following 5 dimensions: Mobility, looking after myself (washing and dressing), usual activities, pain or discomfort and feeling worried or unhappy. Each of the 5 dimensions is divided into 3 levels of perceived problems (Level 1 indicating no problem, Level 2 indicating some problems, Level 3 a lot of problems).

Time frame: Up to 3 years

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

The Neuro-QoL pediatric fatigue score will be used to assess the impact of fatigue in participants aged 10 to less than (\<) 18 years. The participant will rate each of the 11 items on a 5-point scale. Higher scores indicate greater fatigue.

Time frame: Up to 3 years

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

Overview

Conditions

Interventions

Eligibility

Locations (19)

Outcomes

Primary Outcomes

Secondary Outcomes

Central Contacts