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

Официальное название

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

Обзор

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.

Вмешательства

• Препарат Nipocalimab
  Nipocalimab will be administered as an IV infusion.

Первичные конечные точки

• Change from Baseline in Total Serum Immunoglobulin-G (IgG) Levels [Срок оценки: Up to 3 years]
• Number of Participants with Infectious Adverse Events (AEs) [Срок оценки: Up to 3 years]
• Number of Participants with Serious AEs (SAEs) [Срок оценки: Up to 3 years]
• Number of Participants with Adverse Events of Special Interests (AESIs) [Срок оценки: Up to 3 years]
• Number of Participants with Abnormalities in Clinical Laboratory Tests [Срок оценки: Up to 3 years]
• Number of Participants with Abnormalities in Vital Signs [Срок оценки: Up to 3 years]
• Number of Participants with Abnormalities in Physical Examination [Срок оценки: Up to 3 years]
• Serum Concentration of Nipocalimab over Time [Срок оценки: Up to 3 years]
• Clearance (CL) of Nipocalimab [Срок оценки: Up to 3 years]
• Volume of Distribution (V) of Nipocalimab [Срок оценки: Up to 3 years]

Вторичные конечные точки (9)

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

Критерии участия

Критерии включения

• 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

Критерии исключения

• 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

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Не применимо

Модель

Одна группа

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

США · 11 центров

• 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
• … и ещё 3 центра

Япония · 6 центров

• 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

Нидерланды · 1 центр

• Leiden University Medical Center — Leiden

Польша · 1 центр

• Uniwersyteckie Centrum Kliniczne — Gdansk

Идентификаторы

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

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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