새로운 구조적 및 기능적 MRI를 사용한 낭포성 섬유증 폐 질환의 CFTR 조절제 치료 평가
This is a prospective, longitudinal observational study conducted at The Hospital for Sick Children (SickKids). The purpose of this study is to evaluate the ability of MRI to detect changes in lung structure and function in CF patients receiving CFTR-modulator therapy. Participants who meet eligibility criteria and consent to the study will undergo study procedures which include spirometry and body plethysmography (optional), multiple breath washout testing, MRI and measurement of quality of life. The study will consist of three to six study visits for Group 1, 2 and 3: baseline, one month (±1 week) and 6 month (±2 week) and if they re-consent then 3 additional visits of 1 year (±6 months). (2 years (±6 months) and 3 years (±6 months) follow-up. Some participants in Group 2 may transition to Group 3.
This study includes three populations of interest: Group 1: 25 healthy volunteers, Group 2: 25 patients with stable CF lung disease , Group 3: 20 patients with CF lung disease anticipated to receive treatment with CFTR-modulator therapy and Group 4: 18 patients with CF who are between 4-8 years old and are starting triple combination modulator therapy using the rapid lung and abdominal imaging methods.
연구 개요
상태
상태
정황
정황
연구 유형
연구 유형
등록 (실제)
등록
연락처 및 위치
연구 장소
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Ontario
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Toronto, Ontario, 캐나다, M5G 1X8
- The Hospital for Sick Children
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참여기준
자격 기준
자격 기준
공부할 수 있는 나이
건강한 자원 봉사자를 받아들입니다
연구 대상 성별
샘플링 방법
연구 인구
This study includes four populations of interest: Group 1: 25 healthy volunteers, Group 2: 25 patients with stable CF lung disease and Group 3: 20 patients with CF lung disease anticipated to receive treatment with CFTR-modulator therapy and Group 4: 18 patients with CF who are between 4-8 years old and are starting triple combination modulator therapy using the rapid lung and abdominal imaging methods
설명
Inclusion Criteria:
- Participants must be greater than or equal to 4 years of age
- Informed consent by patient or parent/guardian consent and participant assent when appropriate.
- Able to perform reproducible spirometry and achieve a breath hold duration sufficient for MRI acquisition
Exclusion Criteria:
- Medical instability that would preclude the ability to undergo the required investigations
- FEV1 % predicted < 40%
- Severe claustrophobia
- Does not meet MRI screening criteria
- Cough within the past 3 days prior to study visit
- Usage of oral antibiotics within 3 weeks prior to study visit
공부 계획
연구는 어떻게 설계됩니까?
디자인 세부사항
- 관찰 모델: OTHER
- 시간 관점: PROSPECTIVE
그룹/코호트 수
코호트 및 개입
그룹/코호트그룹/코호트 |
|---|
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Group 1
Healthy participants without lung disease |
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Group 2
Participants with stable cystic fibrosis |
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Group 3
Participants with cystic fibrosis, anticipated to receive treatment with CFTR-modulator therapy. Please note: treatment is determined by your physician as part of your normal therapy plan. |
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Group 4
Participants with CF who are between 4-8 years old and are starting triple combination modulator therapy using the rapid lung and abdominal imaging methods. |
연구는 무엇을 측정합니까?
주요 결과 측정
주요 결과 측정
결과 측정 |
측정값 설명 |
기간 |
|---|---|---|
|
Reproducibility
기간: 36 months
|
Establish the interscan reproducibility of fractional ventilation maps and 1H MRI over three and up to six visits in healthy volunteers and in patients with stable CF lung disease and compare to conventional spirometry and LCI. |
36 months
|
|
Correlation
기간: 12 months
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Correlate changes in lung structure and function detected by MRI imaging with clinical outcomes including FEV1 rate of decline, frequency of pulmonary exacerbations, days of IV antibiotics and hospital days to treat pulmonary exacerbation and change in health-related quality of life measures (CFQ-R scores and CF-QUEST) in the setting of patients with stable CF lung disease and those receiving CFTR-modulator therapy. |
12 months
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Responsiveness
기간: 36 months
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Assess responsiveness of MRI to treatment effects over three and up to six visits visits in CF patients receiving CFTR-modulator therapy and compare to conventional breathing tests |
36 months
|
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Younger cohort
기간: 24 months
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Extension of above objectives originally only explored in children ages 8-18 years old, to younger cohort of CF participants ages 4-8 years old at baseline prior to initiation of CFTR modulator therapy, as well as at 12 and 24 months after therapy initiation. |
24 months
|
공동 작업자 및 조사자
수사관
수사관
- 수석 연구원: Giles Santyr, PhD, The Hospital for Sick Children
연구 기록 날짜
연구 주요 날짜
연구 시작 (실제)
연구 시작
기본 완료 (실제)
기본 완료
연구 완료 (실제)
연구 완료
연구 등록 날짜
최초 제출
최초 제출
QC 기준을 충족하는 최초 제출
QC 기준을 충족하는 최초 제출
처음 게시됨 (실제)
처음 게시됨
연구 기록 업데이트
마지막 업데이트 게시됨 (실제)
마지막 업데이트 게시됨
QC 기준을 충족하는 마지막 업데이트 제출
QC 기준을 충족하는 마지막 업데이트 제출
마지막으로 확인됨
마지막으로 확인됨
추가 정보
이 연구와 관련된 용어
키워드
기타 연구 ID 번호
기타 연구 ID 번호
- 1000063021
개별 참가자 데이터(IPD) 계획
개별 참가자 데이터(IPD)를 공유할 계획입니까?
IPD 계획 설명
Data will be shared between the two participating sites in the study. A data transfer agreement has been implemented to enable this transfer smoothly.
IPD 공유 기간
The data will be available after enrolling the first participant and will be available for the duration of the study
IPD 공유 지원 정보 유형
- 연구_프로토콜
- 수액
- ICF
- CSR
약물 및 장치 정보, 연구 문서
미국 FDA 규제 의약품 연구
미국 FDA 규제 기기 제품 연구
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