A Study of Efgartigimod PH20 SC in Children Between 2 and Less Than 18 Years of Age With Generalized Myasthenia Gravis

Официальное название

An Open-label, Uncontrolled Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Safety, and Activity of Efgartigimod PH20 SC in Participants From 2 to Less Than 18 Years of Age With Generalized Myasthenia Gravis

Обзор

The purpose of this study is to measure the pharmacokinetics (PK), pharmacodynamics (PD), safety, tolerability, and immunogenicity of efgartigimod PH20 SC in pediatric participants with gMG aged 2 to \<18 years. The primary goal is to confirm an appropriate dose of efgartigimod PH20 SC for pediatric patients using PK and PD results from this study. Participants will receive injections of efgartigimod PH20 SC and will be monitored for safety until the end of the study. At the end of the follow-up period, eligible participants may roll over to an open-label extension (OLE) study. The participants will be in the study for up to 14 weeks.

Вмешательства

• Биопрепарат Efgartigimod PH20 SC
  Subcutaneous injections

Первичные конечные точки

• Efgartigimod serum concentrations as input for compartmental, model-driven analysis to determine age and size dependency of Clearance (CL) [Срок оценки: Up to 12 weeks]
• Efgartigimod serum concentrations as input for compartmental, model-driven analysis to determine age and size dependency of Volume Distribution (Vd) [Срок оценки: Up to 12 weeks]
• Total G immunoglobulins (IgG) levels as input for pharmacokinetics (PK)/pharmacodynamics (PD) modelling analysis [Срок оценки: Up to 12 weeks]
• Anti-acetylcholine receptors antibodies (AChR-Ab) as input for pharmacokinetics (PK)/ pharmacodynamics (PD) modelling analysis [Срок оценки: Up to 12 weeks]

Вторичные конечные точки (12)

• Incidence of adverse events (AEs) [Срок оценки: Up to 14 weeks]
• Severity of adverse events (AEs) [Срок оценки: Up to 14 weeks]
• Incidence of serious adverse events (SAEs) [Срок оценки: Up to 14 weeks]
• Severity of serious adverse events (SAEs) [Срок оценки: Up to 14 weeks]
• Incidence of adverse events of special interest (AESI) [Срок оценки: Up to 14 weeks]
• Severity of adverse events of special interest (AESI) [Срок оценки: Up to 14 weeks]
• Efgartigimod serum concentrations [Срок оценки: Up to 12 weeks]
• Absolute values of total Immunoglobulin G (IgG) from blood samples [Срок оценки: Up to 12 weeks]
• Change from baseline values of total Immunoglobulin G (IgG) from blood samples [Срок оценки: Up to 12 weeks]
• Percentage change from baseline values of total Immunoglobulin G (IgG) from blood samples [Срок оценки: Up to 12 weeks]
• Absolute values of anti-acetylcholine receptor antibodies (AChR-Ab) from blood samples [Срок оценки: Up to 12 weeks]
• Change from baseline values of anti-acetylcholine receptor antibodies (AChR-Ab) from blood samples [Срок оценки: Up to 12 weeks]

Критерии участия

Критерии включения

• The participant (and/or their legally authorized representative) understands the requirements of the study and is capable of providing written informed consent/assent and complying with protocol requirements
• The participant is aged 2 to <18 years at the time of informed consent/assent
• The participant has been diagnosed with generalised Myasthenia Gravis that is supported by a physical examination and confirmed seropositivity for anti-acetylcholine receptor antibodies
• The participant has had an unsatisfactory response to immunosuppressants, corticosteroids, or acetylcholinesterase inhibitors but is on stable concomitant MG therapy. If receiving corticosteroids and/or immunosuppressants, must be on a stable dose for ≥1 month before screening
• The participant agrees to use birth control consistent with local regulations and people of child-bearing potential must have a negative blood pregnancy test at screening and a negative urine pregnancy test before receiving the study drug

Критерии исключения

• Is a female adolescent of child-bearing potential who is pregnant and/or lactating or intends to become pregnant during their participation in the study
• Has worsening muscle weakness secondary to a concurrent infection or as a result of a medication
• Has a documented lack of clinical response to plasma exchange (PLEX)
• Received a live or live-attenuated vaccine within <4 weeks before screening
• Received a thymectomy within 3 months before screening or is planning to get a thymectomy during their participation in the study
• Has a known autoimmune disease or any medical condition that would interfere with an accurate assessment of clinical symptoms of generalised Myasthenia Gravis or puts the participant at undue risk
• History of malignancy, cancer, unless considered cured by adequate treatment with no evidence of recurrence for ≥3 years. Adequately treated participants with the following cancers can be included at any time: Basal cell or squamous cell skin cancer, Carcinoma in situ of the cervix, Carcinoma in situ of the breast, Incidental histological findings of prostate cancer
• Clinically significant active infection that is not sufficiently resolved in the investigator's opinion or positive serum test at screening for active infection with any of the following: Hepatitis B virus (HBV), Hepatitis C virus (HCV), HIV
• Has a positive PCR test for SARS-CoV-2 at screening
• Has/had a clinically significant disease, had recent major surgery (within 3 months of screening) or intends to have major surgery during the study, or has/had any other medical condition that, in the investigator's opinion, would confound the results of the study or put the participant at undue risk
• Has received a different study drug in another clinical study within <12 before screening
• Is currently participating in another interventional clinical study
• Has previously participated in an efgartigimod clinical study and received at least one dose of study drug
• Has a known hypersensitivity to study drug or any of its excipients
• Has a history of or current episode of alcohol, drug, or medication abuse as assessed by the investigator
• Use of some medications before screening (more information is found in the protocol)

The complete list of exclusion criteria can be found in the protocol.

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 · 3 центра

• Carolinas HealthCare System Neurosciences Institute - Neurology — Charlotte
• Neurology Rare Disease Center — Denton
• University of Virginia — Charlottesville

Израиль · 3 центра

• Hadassah Medical Center- Ein Kerem — Jerusalem
• Schneider Children's Medical Center of Israel — Petah Tikvah
• Tel Aviv Sourasky Medical Center Ichilov — Tel Aviv

Польша · 3 центра

• Uniwersyteckie Centrum Kliniczne w Gdansku — Gdansk
• Neurologia Śląska Centrum Medyczne — Katowice
• Centralny Szpital Kliniczny - Uniwersyteckie Centrum Kliniczne WUM — Warsaw

Чехия · 2 центра

• Fakultni nemocnice Brno — Brno
• Fakultni nemocnice Ostrava — Ostrava

Бельгия · 1 центр

• UZ Gent — Ghent

Канада · 1 центр

• Childrens Hospital of Eastern Ontario — Ottawa

Франция · 1 центр

• AP-HM- Hôpital de La Timone — Marseille

Германия · 1 центр

• Universitätsklinikum Essen — Essen

Италия · 1 центр

• Istituto G Gaslini Ospedale Pediatrico IRCCS — Genova

Нидерланды · 1 центр

• Leiden University Medical Center — Leiden

Испания · 1 центр

• Hospital Universitari i Politecnic La Fe de Valencia — Valencia

Швейцария · 1 центр

• Hôpital Nestlé — Lausanne

Великобритания · 1 центр

• Oxford Children's Hospital — Oxford

Идентификаторы

NCT: NCT06392386 · ARGX-113-2207 · 2023-506159-12-00

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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