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Идёт набор NCT06193928

Long-Term SafEty and Clinical Outcomes of LivmArli in Patients in the United States (LEAP-US)

— Наблюдательное Alagille Syndrome Progressive Familial Intrahepatic Cholestasis

Ориентир для пациента и семьи

Простыми словами

Автоматическая сводка по структурированным данным реестра. Она помогает сориентироваться, но не заменяет официальный протокол или оценку врача.

Что изучают
В протоколе указаны: Livmarli, Livmarli.
Кому может быть актуально
Состояния в реестре: Alagille Syndrome, Progressive Familial Intrahepatic Cholestasis. Базовые параметры: Без ограничений · Все.
Что важно проверить
Возраст, диагноз и пол — только базовые ориентиры. Предыдущее лечение, анализы и другие обязательные условия указаны ниже в критериях участия.
Где проводится
США
Следующий шаг
Сохраните исследование, покажите его лечащему врачу и уточните актуальный статус у исследовательского центра. Расходы, документы и поездка →

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Обзор

The objective of this 5-year, prospective, observational cohort study is to evaluate the long-term safety and clinical outcomes of patients with Alagille syndrome (ALGS) or Progressive familial intrahepatic cholestasis (PFIC) treated with Livmarli.

Подробное описание

Livmarli® is a novel, minimally absorbed, pharmacological product that inhibits the ileal bile acid transporter (IBAT) in the terminal ileum, leading to reduced levels of bile acids. Livmarli (maralixibat) has been developed by Mirum Pharmaceuticals and was the first treatment approved by the US Food and Drug Administration (FDA) for the treatment of cholestatic pruritus in patients 3 months of age and older with Alagille syndrome (ALGS). Subsequently, Livmarli was approved by the FDA for the treatment of cholestatic pruritus in patients 12 months of age and older with Progressive familial intrahepatic cholestasis (PFIC). To be eligible for the study, participants must meet the following criteria:

* A clinically and/or genetically confirmed ALGS diagnosis or PFIC diagnosis * Prescribed Livmarli

Вмешательства

  • Препарат Livmarli
    The recommended dosage is 380 mcg/kg once daily.
  • Препарат Livmarli
    The recommended dosage us 570 mcg/kg twice daily.

Первичные конечные точки

  • Incidence of Long-Term Clinical Outcomes [Срок оценки: Long-term clinical outcomes (SBD, LT, portal hypertension, all-cause mortality) up to 180 days after discontinuation of Livmarli will be recorded.]
  • Liver Transplant Indication and Waitlist Status [Срок оценки: LT waitlist status will be collected at enrollment and every 6 months for 5 years.]
  • Assessment of Growth and Development [Срок оценки: Weight (kilograms) and height (centimeters) z-scores will be collected every year for 5 years.]
  • Incidence of Clinical Events Potentially Related to Fat-Soluble Vitamin Deficiencies and Their Long-Term Sequelae [Срок оценки: The incidence of events will be assessed and reported every year for 5 years.]

Критерии участия

Критерии включения

  • A clinically and/or genetically confirmed ALGS diagnosis or PFIC diagnosis
  • Participant prescribed Livmarli

Критерии исключения

  • Refusal to provide informed consent/assent (if required by the local IRB)
  • Previously or currently on Livmarli through participation in a clinical study or expanded access program
  • Participants who have previously received an SBD or LT
  • Any condition or abnormalities that, in the opinion of the investigator, may interfere with the participant participating in or completing the study
  • Participants who have received an investigational drug within 30 days of the first dose of Livmarli

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

Здоровые добровольцы: Нет

Дизайн исследования

Модель наблюдения
Когортное

Центры проведения

США · 8 центров
  • Children's Hospital Los Angeles CHLA — Los Angeles
  • Section of Gastroenterology, Hepatology and Nutrition, Department of Pediatrics and the Di — Aurora
  • Children's Healthcare of Atlanta - Emory University School of Medicine — Atlanta
  • Children's Mercy Kansas City, Department of Gastroenterology, Section of Hepatology — Kansas City
  • Oregon Health and Science University, Division of Pediatric Gastroenterology, Department o — Portland
  • Children's Hospital of Philadelphia — Philadelphia
  • Children Hospital of Pittsburgh — Pittsburgh
  • University of Utah, Division of Pediatric Gastroenterology, Hepatology and Nutrition — Salt Lake City

Идентификаторы

NCT: NCT06193928 · MRX-310

Первоисточники (государственные реестры)

Открыть это исследование на ClinicalTrials.gov ↗