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Набор по приглашению NCT05308927

French Registry of Children Treated With Norditropin® for Short Stature Associated With Noonan Syndrome

— Наблюдательное Noonan Syndrome

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: Norditropin.
Кому может быть актуально
Состояния в реестре: Noonan Syndrome. 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
Франция
Следующий шаг
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Обзор

This is a non-interventional registry of children treated with Norditropin® for short stature due to Noonan Syndrome (NS). This study aims to provide data on long-term growth evolution and safety of Norditropin® as well as Health Related Quality of Life (HRQoL) data. This registry will include the entirety of children treated with Norditropin® for short stature due to NS over the inclusion period. The decision to initiate treatment with commercially available Norditropin® is made by the patient/parents/Legally Acceptable Representative (LAR) and the treating physician before and independently from the decision to include the patient in this study.

Вмешательства

  • Препарат Norditropin
    Patients will be treated with commercially available Norditropin® according to routine clinical practice at the discretion of the treating physician.

Первичные конечные точки

  • Change in height standard deviation score [Срок оценки: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)]
Вторичные конечные точки (12)
  • Number of adverse drug reactions (ADR) [Срок оценки: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)]
  • Number of serious adverse drug reactions (SADR) [Срок оценки: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)]
  • Number of serious adverse events (SAE) [Срок оценки: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)]
  • Number of adverse events (AE) [Срок оценки: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)]
  • Education [Срок оценки: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)]
  • School level [Срок оценки: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)]
  • Educational measures [Срок оценки: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)]
  • Tanner stage [Срок оценки: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)]
  • Change in pulse rate [Срок оценки: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)]
  • Change in diastolic blood pressure [Срок оценки: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)]
  • Change in systolic blood pressure [Срок оценки: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)]
  • Change in corrected QT-interval (Electrocardiogram) [Срок оценки: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)]

Критерии участия

Критерии включения

  • Patients with a clinical and/or genetic diagnosis of NS
  • Patients who are treated with Norditropin® (already treated or initiating) and who are followed in a participating center
  • The decision to initiate treatment with commercially available Norditropin® has been made by the patient/parents/Legally Acceptable Representative (LAR) and the treating physician before and independently from the decision to include the patient in this study

Критерии исключения

  • Patients/Parents/LAR opposed to the collection and processing of their children's medical data
  • Mental incapacity, unwillingness or language barriers precluding adequate understanding or cooperation

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

Здоровые добровольцы: Нет

Дизайн исследования

Модель наблюдения
Когортное

Центры проведения

Франция · 3 центра
  • Centre Hospitalier Universitaire D'Angers-2 — Angers
  • Ap-Hp-Hopital de Bicetre-2 — Le Kremlin-Bicêtre
  • Hopital Des Enfants-2 — Toulouse

Идентификаторы

NCT: NCT05308927 · GH-4831 · U1111-1264-1805

Первоисточники (государственные реестры)

Открыть это исследование на ClinicalTrials.gov ↗