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Идёт набор NCT05234567

A Prospective Sub-Study of the Global Hypophosphatasia Registry

— Наблюдательное Hypophosphatasia

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: Asfotase Alfa.
Кому может быть актуально
Состояния в реестре: Hypophosphatasia. 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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Официальное название

A Prospective Observational Sub-Study of the Global Hypophosphatasia Registry to Describe the Potential Risk of Immune-Mediated Loss of Pharmacological Effect of Asfotase Alfa in Participants With Hypophosphatasia

Обзор

In this prospective observational sub-study, participants with pediatric-onset hypophosphatasia (HPP) (perinatal/infantile- or juvenile-onset) of any age will be followed for a minimum of 5 years at sites in the United States and potentially 1 or 2 other countries.

Вмешательства

  • Биопрепарат Asfotase Alfa
    All participants will receive asfotase alfa subcutaneously per standard of care. Unless otherwise specified per the Physician's standard of care, participants aged \< 2 years are recommended for a clinic visit approximately every 3 months after Enrollment until 2 years of age, after which they should have a clinic visit approximately every 6 months. Participants should be followed for 5 years, as possible.

Первичные конечные точки

  • Occurrence Of Immune-mediated Loss Of Effectiveness According To The Treating Physician [Срок оценки: Up to 5 years]
  • Occurrence Of Immune-mediated Serious Adverse Events [Срок оценки: Up to 5 years]

Критерии участия

Критерии включения

  • Any age or sex with a confirmed diagnosis of pediatric-onset HPP (that is, first HPP sign or symptom presented at < 18 years of age).
  • Currently receiving asfotase alfa treatment at Enrollment (not treatment-naïve) or the Physician has decided to resume (not treatment-naïve) or start (treatment-naïve) the participant's asfotase alfa treatment within 6 months after Enrollment.
  • Participant must have documented alkaline phosphatase (ALP) activity below the lower limit of normal for age and sex, and a documented ALPL gene mutation (Note: An exception is made for infants with clinical features of HPP plus low ALP who need to start asfotase alfa treatment right away, at the Physician's discretion, but do not yet have a genetic result. In this case, ALPL gene documentation is not required at the time of sub-study enrollment but should be documented within 6 months after Enrollment).
  • Participant or participant's parent/legally authorized representative is able to read and/or understand the informed consent and study questionnaires in the local language.
  • Participant or participant's parent/legally authorized representative must be willing and able to give signed informed consent for this sub-study, and the participant must be willing to give written informed assent, if appropriate and required by local regulations.

Критерии исключения

  • Currently participating in an Alexion-sponsored interventional clinical study. Participants who have concluded participation in an Alexion-sponsored asfotase alfa clinical study are eligible to enroll in this sub-study.

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 · 12 центров
  • Clinical Trial Site — Hartford
  • Clinical Trial Site — Chicago
  • Clinical Trial Site — Boston
  • Clinical Trial Site — Kansas City
  • Clinical Trial Site — Mineola
  • Clinical Trial Site — Cincinnati
  • Clinical Trial Site — Columbus
  • Clinical Trial Site — Pittsburgh
  • … и ещё 4 центра

Идентификаторы

NCT: NCT05234567 · ALX-HPP-501s

Первоисточники (государственные реестры)

Открыть это исследование на ClinicalTrials.gov ↗