Меню
Идёт набор NCT05040178

An Observational Study of Carbaglu® for the Treatment of MMA and PA in Adults and Pediatrics

— Наблюдательное Hyperammonemia Methylmalonic Acidemia Propionic Acidemia

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: Carglumic Acid.
Кому может быть актуально
Состояния в реестре: Hyperammonemia, Methylmalonic Acidemia, Propionic Acidemia. 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 →

Не всё понятно в терминах? Прочитайте наш гид для пациентов →

Официальное название

A Non-Interventional Post-Authorization Safety Study (PASS) of Carbaglu® for the Treatment of Hyperammonemia Due to Methylmalonic Acidemia (MMA) and Propionic Acidemia (PA) in Adult and Pediatric Patient Populations

Обзор

To obtain short-term and long-term clinical safety information, in pediatric and adult patients with PA and MMA treated with Carbaglu®.

Подробное описание

This study is being conducted to obtain short-term and long-term clinical safety information from adult and pediatric patients treated for hyperammonemia due to Methylmalonic Acidemia (MMA) and Propionic Acidemia (PA). This is an observational/non-interventional study. Patients will be treated per the prescribing information and routine medical practice.

Only available data will be collected as part of the study including developmental outcomes, details of treatment with Carbaglu® and other treatments for hyperammonemia including dietary and protein management, plasma ammonia levels, pregnancy and maternal complications, adverse effects on the developing fetus and neonate, adverse effects on the infant through first year of life.

Вмешательства

  • Препарат Carglumic Acid
    Current or previous treatment with Carbaglu, the dose of Carbaglu® prescribed will be determined by the investigator for each individual patient.

Первичные конечные точки

  • Effects of Carbaglu® on plasma ammonia levels [Срок оценки: Patients treated with Carbaglu® will be managed chronically (out-patient) or acutely (hospital in-patient). In both cases, data will be collected for approximately 1 year following discontinuation of Carbaglu treatment.]
  • Adverse Event frequency and severity [Срок оценки: Patients treated with Carbaglu® will be managed chronically (out-patient) or acutely (hospital in-patient). In both cases, data will be collected for approximately 1 year following discontinuation of Carbaglu treatment.]
Вторичные конечные точки (1)
  • Fetal Outcomes and Pregnancy Outcomes [Срок оценки: Collection of pregnancy information for patients who becomes pregnant while participating in the trial or at time of enrollment. Pregnancy reports and reports involving neonates and infants up to 1 year of age must be reported to RRD Pharmacovigilance.]

Критерии участия

Критерии включения

  • Provision of signed and dated informed consent/assent form
  • Prescribed and treated with Carbaglu®
  • Have an established diagnosis of PA or MMA defined as follows:
  • Diagnosed with PA by semi quantitative urine organic acid analysis, defined as presence of elevated methylcitric acid and normal methylmalonic acid levels and no evidence of biotin related disorders in the organic acid analysis; OR
  • Diagnosed with MMA by semi quantitative urine organic acid analysis, defined as elevation of methylmalonic acid and no evidence of vitamin B12 dependent disorder on plasma amino acid analysis (vitamin B12 dependency is defined by documented vitamin B12 responsiveness).

AND/OR

  • Confirmation by molecular genetic testing

Критерии исключения

  • None

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 · 5 центров
  • Children's National Hospital — Washington D.C.
  • University of South Florida — Tampa
  • Ann & Robert H. Lurie Children's Hospital of Chicago — Chicago
  • Riley Children's Hospital — Indianapolis
  • Icahn School of Medicine at Mt. Sinai — New York

Идентификаторы

NCT: NCT05040178 · CARBAGLU-RRDUS-PASS-0573

Первоисточники (государственные реестры)

Открыть это исследование на ClinicalTrials.gov ↗