Идёт набор NCT06168201

VIrtual STudy in Achondroplasia for the US (VISTA)

— Наблюдательное Achondroplasia

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: Это наблюдательное исследование: исследуемое лечение участникам по протоколу не назначают.
Кому может быть актуально: Состояния в реестре: Achondroplasia. Базовые параметры: 0 мес. — 18 лет · 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

This is an observational study of individuals with achondroplasia in the United States. The primary study population consists of pediatric individuals treated and untreated with VOXZOGO™. Study enrollment started in February 2023. The projected total duration of the study is approximately 5 years at minimum from start of study recruitment in February 2023, with the duration of individual prospective follow-up differing depending on the time of enrollment. The study duration may be extended based on decisions by the study sponsor. Data will be collected in two formats for the primary pediatric study population: 1. Participant-mediated access to electronic health records(including medical imaging, when available) which will enable retrospective and prospective collection of secondary data reflecting real-life treatment use and clinical care. . 2. Primary data collection of Clinical Outcome Assessments (COAs) and questionnaire data. Data will be collected in the following format for the adult cohort: \- Participant-mediated access to electronic health records (including medical imaging, when available) which will enable retrospective and prospective collection of secondary data reflecting real-life treatment use and clinical care. The primary study population will include approximately 150 pediatric individuals with achondroplasia regardless of their treatment status with VOXZOGO™. Individuals may change status from untreated to treated during the prospective period of the study (or vice versa) however they will only be counted once, based on their treated status at the point of enrollment. The secondary study population will include 20 adolescent and adult participants.

Первичные конечные точки

Height [Срок оценки: Retrospective data and prospective data for up to 5 years starting from time of study enrolment]
Weight [Срок оценки: Retrospective data and prospective data for up to 5 years starting from time of study enrolment]
Body Mass Index [Срок оценки: Retrospective data and prospective data for up to 5 years starting from time of study enrolment]
Head Circumference [Срок оценки: Retrospective data and prospective data for up to 5 years starting from time of study enrolment]
Annualized growth velocity (AGV) [Срок оценки: Retrospective data and prospective data for up to 5 years starting from time of study enrolment]
Medical diagnoses by age [Срок оценки: Retrospective data and prospective data for up to 5 years starting from time of study enrolment]
Surgical procedures and/or medical interventions by age [Срок оценки: Retrospective data and prospective data for up to 5 years starting from time of study enrolment]
Changes in physical functioning [Срок оценки: every 6 months starting from time of study enrolment up to 5 years]
Changes in health-related quality of life (HRQoL) [Срок оценки: every 6 months starting from time of study enrolment up to 5 years]
Adherence to treatment with VOXZOGO™ (vosoritide), if prescribed [Срок оценки: every 6 months starting from time of study enrolment up to 5 years]

Критерии участия

Критерии включения

Physician diagnosis of achondroplasia
Age at time of enrollment:

Primary Pediatric Cohort: ≤13 years old Secondary Adolescent and Adult Cohort: ≥14 years old

Receiving medical care in the United States
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