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Идёт набор NCT06191640

Sinus Disease in Young Children With Cystic Fibrosis

— Наблюдательное Cystic Fibrosis in Children Cystic Fibrosis Chronic Rhinosinusitis (Diagnosis) Olfactory Disorder

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: Ivacaftor or elexacaftor/tezacaftor/ivacaftor.
Кому может быть актуально
Состояния в реестре: Cystic Fibrosis in Children, Cystic Fibrosis, Chronic Rhinosinusitis (Diagnosis), Olfactory Disorder. Базовые параметры: 2 лет — 8 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 a prospective, observational study examining the impact of highly effective cystic fibrosis transmembrane conductance regulator (CFTR) modulators on chronic rhinosinusitis (CRS) and olfactory dysfunction (OD) in young children with cystic fibrosis (YCwCF). This study involves two groups: children 2-8 years old, inclusive at initial visit, receiving highly effective modulator therapy (HEMT), and a control group of children 2-8 years old, inclusive at initial visit, not receiving HEMT. Outcomes will include sinus magnetic resonance imaging (MRI) scans, olfactory tests, and quality of life surveys obtained over a two-year period.

Подробное описание

This multi-center, prospective, observational study investigates the effects of highly effective modulator therapy (HEMT) on chronic rhinosinusitis (CRS) and olfactory dysfunction (OD) in young children with cystic fibrosis (YCwCF). The study spans two years and includes two distinct groups of children with cystic fibrosis: children ≤ 8 years old receiving HEMT and a control group of children ≤ 8 not receiving HEMT. The study aims to assess the efficacy of HEMT in improving sinus health and olfactory capabilities in this young demographic.

Key assessments include magnetic resonance imaging (MRI) sinus opacification, olfactory bulb volume measured via MRI, objective olfactory testing, and various quality (QOL) surveys. This investigation seeks to characterize the severity of CRS and OD in YCwCF, and to elucidate if early initiation of HEMT improves CRS and OD .

In the HEMT group, participants will have a pre-HEMT assessment followed by 1-year and 2-year post-HEMT evaluations. In the control/non-HEMT group, participants will undergo parallel assessments at baseline, 1-year, and 2-year intervals to track the natural progression of CRS and OD without HEMT.

Вмешательства

  • Препарат Ivacaftor or elexacaftor/tezacaftor/ivacaftor
    HEMT's are prescribed at the discretion of the treating physician and is not dictated by the principal investigator of this study.

Первичные конечные точки

  • Change in MRI sinus opacification [Срок оценки: Baseline, 1-year, and 2-year follow-up]
  • Change in olfactory bulb volume [Срок оценки: Baseline, 1-year, and 2-year follow-up]
Вторичные конечные точки (5)
  • Change in Pediatric Smell Wheel (PSW) Scores [Срок оценки: Baseline, 1-year, and 2-year follow-up]
  • Olfactory Cleft Opacification [Срок оценки: Baseline, 1-year, and 2-year follow-up]
  • Change in Brief Questionnaire of Olfactory Disorders (BQOD) Scores [Срок оценки: Baseline, 1-year, and 2-year follow-up]
  • Change in Sinus and Nasal Quality of Life Survey (SN-5) - Symptom Frequency Domains [Срок оценки: Baseline, 1-year, and 2-year follow-up]
  • Change in Sinus and Nasal Quality of Life Survey (SN-5) - Overall Quality of Life (QOL) Domain [Срок оценки: Baseline, 1-year, and 2-year follow-up]

Критерии участия

Критерии включения

HEMT Group:

  • Children with documentation of a CF diagnosis
  • Age 2-8 years old at first study visit
  • CFTR mutation consistent with FDA labeled indication of highly effective modulator therapy (ivacaftor or elexacaftor/tezacaftor/ivacaftor)
  • Clinician intent to prescribe ivacaftor or ETI so that enrollment is before start of HEMT

Non-HEMT/Control Group:

  • Children with documentation of a CF diagnosis
  • Age 2-8 years at first study visit
  • Ineligible for highly effective modulator therapy (ivacaftor or elexacaftor/tezacaftor/ivacaftor) based on CFTR mutation or clinical decision not to initiate HEMT if eligible

Критерии исключения

For Both Groups:

  • Use of an investigational drug within 28 days prior to the first study visit
  • Use of ivacaftor or elexacaftor/tezacaftor/ivacaftor within the 180 days prior to and including the first study visit
  • Use of chronic oral corticosteroids within the 28 days prior to and including the first study visit.
  • Sinus surgery within 180 days prior to the first study visit

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 · 6 центров
  • Children's Hospital Colorado — Aurora
  • University of Iowa — Iowa City
  • University of Kansas Medical Center — Kansas City
  • Cincinnati Children's Hospital Medical Center — Cincinnati
  • University of Vermont — Colchester
  • University of Virginia — Charlottesville

Публикации

  • Liu CM, Fischer JL, Zemanick ET, Woods JC, Markarian KK, Fain SB, Froh D, Heltshe SL, Hoffman LR, Humphries SM, Kramer EL, Ode KL, Lewis M, Li DA, Mata J, Milla SS, Niedbalski PJ, Sawatzky BD, Sim MS, Sullivan JS, Trout AT, Goss CH, Taylor-Cousar JL, Beswick DM. The impact of highly effective modulator therapy on sinusitis and dysosmia in young children with cystic fibrosis: a prospective study pr PMID 39811548

Идентификаторы

NCT: NCT06191640 · 22-000594

Первоисточники (государственные реестры)

Открыть это исследование на ClinicalTrials.gov ↗