BEGIN 새로운 이미징 바이오마커 (BEGINNING)
To determine the treatment effect of triple-combination therapy in 6-8 year olds after presumed FDA approval, using rapid structural and functional pulmonary and abdominal MRI (UTE and 129Xe).
연구 개요
상세 설명
The overall hypothesis is that multi-organ MRI will provide more sensitive, robust outcome measures in young CF patients than traditional measures employed in the BEGIN study and that these novel measures will be more sensitive to treatment effects, tested here by comparison before and after triple-combination modulator therapy. By understanding the nature of early lung obstruction and characteristic changes in the liver and pancreas over time, we continue to lay the groundwork for more personalized medicine in the future.
Assessing treatment response and clinical benefit in children with CF who are clinically normal per standard outcomes (e.g., spirometry, pancreatic function) will become paramount as triplecombination therapy is extended to younger patients with milder CF clinical presentation than their historic peers. Here the sensitivity and profile free of ionizing-radiation exposure of MRI can be leveraged to follow an individual with CF over time to quantify changes with therapy-with additional spatial resolution unavailable from standard clinical testing.
연구 유형
등록 (실제)
단계
- 4단계
연락처 및 위치
연구 장소
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Kansas
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Kansas City, Kansas, 미국, 66160
- University of Kansas Medical Center
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Ohio
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Cincinnati, Ohio, 미국, 45229
- Carrie Stevens
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Virginia
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Charlottesville, Virginia, 미국, 22903
- University of Virginia
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참여기준
자격 기준
공부할 수 있는 나이
건강한 자원 봉사자를 받아들입니다
연구 대상 성별
설명
Inclusion Criteria:
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Written informed consent (and assent where appropriate) obtained from the subject or subject's legal representative.
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Willingness to adhere to the study-visit schedule and other protocol requirements.
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Ages 6-8 years old at baseline MRI visit (may be enrolled up to 60 days before 6th birthday).
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Documentation of CF diagnosis as evidenced by one or more clinical features consistent with the CF phenotype and one or more of the following criteria:
- Sweat chloride equal to or greater than 60 mEq/liter by quantitative pilocarpine iontophoresis test
- Two well-characterized mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene
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Physician intent to prescribe triple-combination therapy
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Clinically-stable with no respiratory tract infection at the time of enrollment.
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No change in chronic maintenance therapies in the 28 days prior to enrollment.
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Ability to cooperate with MRI procedures
Exclusion Criteria:
- Individuals currently on ivacaftor therapy (including Kalydeco, Orkambi, and Symdeko) and with at least one gating mutation. Gating mutations include G551D, G178R, S549N, S549R, G551S, G970R, G1244E, S1251N, S1255P, or G1349D.
- Acute respiratory symptoms (e.g. wheezing) at the time of the MRI.
- Acute respiratory infection, defined as increased cough, wheezing or respiratory rate in the 28 days prior to enrollment.
- Chronic lung disease not related to CF
- Chronic liver disease not related to CF
- Acute pancreatitis, defined by clinical criteria (45).
- Chronic pancreatic disease not related to CF.
- Physical findings that would compromise the safety of the subject or the quality of the study data as determined at the discretion of the site investigator.
- Any other condition that, in the opinion of the Site Investigator/designee, would preclude informed consent or assent, make study participation unsafe, complicate interpretation of study outcome data, or otherwise interfere with achieving the study objectives.
공부 계획
연구는 어떻게 설계됩니까?
디자인 세부사항
- 주 목적: 특수 증상
- 할당: 무작위화되지 않음
- 중재 모델: 크로스오버 할당
- 마스킹: 하나의
무기와 개입
참가자 그룹 / 팔 |
개입 / 치료 |
|---|---|
|
실험적: Pre Trikafta
129Xe MRI |
Rapid spatial mapping of lung, liver, and pancreatic structure and function is now possible with a combination of hyperpolarized 129Xe and traditional proton MRI, all absent sedation and ionizing radiation. |
|
실험적: Post Trikafta
129Xe MRI |
Rapid spatial mapping of lung, liver, and pancreatic structure and function is now possible with a combination of hyperpolarized 129Xe and traditional proton MRI, all absent sedation and ionizing radiation. |
연구는 무엇을 측정합니까?
주요 결과 측정
결과 측정 |
측정값 설명 |
기간 |
|---|---|---|
|
Ventilation Defect Percentage change from baseline
기간: 1 year
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For pulmonary MRI, the primary outcome measure is the change in 129Xe ventilation defect percentage (VDP) from pre-therapy baseline to the one-year follow-up visit. |
1 year
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Pancreas volume
기간: 1 year
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For pancreatic MRI, the primary outcome measure is change in pancreas volume normalized to BSA between pre-therapy baseline and one-year follow-up visit. |
1 year
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2차 결과 측정
결과 측정 |
측정값 설명 |
기간 |
|---|---|---|
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Abdominal T1 values
기간: 1 year
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Changes in MRI T1 average in the liver and pancreas, from baseline to follow up at 1 year |
1 year
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Lung reader score
기간: 1 year
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Changes in reader score for visible structural defects from proton MRI, from baseline to follow up at 1 year |
1 year
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공동 작업자 및 조사자
수사관
- 수석 연구원: Jason Woods, PhD, Children's Hospital Medical Center, Cincinnati
연구 기록 날짜
연구 주요 날짜
연구 시작 (실제)
기본 완료 (추정된)
연구 완료 (추정된)
연구 등록 날짜
최초 제출
QC 기준을 충족하는 최초 제출
처음 게시됨 (실제)
연구 기록 업데이트
마지막 업데이트 게시됨 (실제)
QC 기준을 충족하는 마지막 업데이트 제출
마지막으로 확인됨
추가 정보
이 연구와 관련된 용어
추가 관련 MeSH 약관
기타 연구 ID 번호
- 2021-0325
개별 참가자 데이터(IPD) 계획
개별 참가자 데이터(IPD)를 공유할 계획입니까?
약물 및 장치 정보, 연구 문서
미국 FDA 규제 의약품 연구
미국 FDA 규제 기기 제품 연구
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