MRI Parameters as Biomarkers in Cystic Fibrosis (FEMPTO) (FEMPTO)
A Feasibility Study Evaluating MRI Parameters as Biomarkers of the Effect of a Secretagogue in Cystic Fibrosis (FEMPTO)
Cystic fibrosis (CF) is an inherited condition that affects several organs, including the gut. People with CF often experience uncomfortable digestive symptoms such as bloating, gas, abdominal pain, and constipation. The root cause is that the gut produces thicker-than-normal mucus, which slows things down and makes it harder for the bowel to work properly.
Current treatments such as laxatives, enemas, or surgery can be inconvenient and don't always work well. There is a real need for better options, and for a clearer understanding of how the CF gut behaves and responds to treatment.
This study will use MRI, a non-invasive imaging technique, to look at how fluid moves through the gut in adults with CF and the impact of linaclotide. Linaclotide is a licensed medication used to treat chronic constipation and irritable bowel syndrome. It works by encouraging the gut to release more fluid, helping things move more easily. It acts locally in the gut and is not absorbed into the bloodstream. Early research suggests it may help in CF in a way that does not rely on correcting the underlying genetic fault, making it a promising avenue to explore.
Participants will include people with different bowel habits, including those who tend towards constipation and those who tend towards diarrhoea. The scans will help researchers check whether certain MRI measurements can reliably detect changes in gut fluid and movement.
This is a feasibility study; its main objective is to test whether these MRI measurements are useful and practical, and support a sample size calculation, in future CF studies investigating linaclotide effects.
연구 개요
상태
상태
정황
정황
개입 / 치료
개입 / 치료
상세 설명
Background: The recent James Lind Alliance priority setting listed "How can we relieve gastro-intestinal symptoms, such as stomach pain, bloating and nausea?" as number 3 priority despite treatment with CFTR modulators. The investigators have shown using magnetic resonance imaging (MRI) that pwCF have abnormal small bowel contents which empty more slowly into the colon. This is most likely due to impaired pancreatic, biliary and intestinal secretions leading to hyperviscosity of contents. Linaclotide is a drug which stimulates bicarbonate secretion in the small intestine which has been shown to accelerate transit in mice models of CF but not in patients. Its action would be predicted to reduce mucus viscosity and normalise small bowel contents and transit.
Aims: to perform a mechanistic pilot study using MRI to show that linaclotide normalises small bowel contents and transit times.
Methods: A randomised, placebo-controlled trial of linaclotide in pwCF. Linaclotide 290ug will be taken on the day before and on the MRI study day. Patients will then undergo serial MRI scanning for 6 hours following dosing at time T 0 , with a standard rice pudding meal at time T60 and a second 700kcal meal at T 300 minutes with the final scan at time T360minutes as the investigators have done previously. The investigators will measure small bowel water content (SBWC) as well as time for chyme to enter the colon (orocaecal transit time [OCTT]) and T1 ascending colon(T1AC), a measure of colonic water content. Primary endpoint: SBWC AUC 0-6hours . Secondary endpoints: change in SBWC after second meal from T300 to T360 ( ∆ SBWC), OCTT, T1 AC AUC 0-6 hours, bowel frequency and stool consistency on study day and the following 3 days.
Impact: If successful this will provide a mechanistic rationale for a full 12 week clinical study of linaclotide as adjunctive treatment for pwCF.
연구 유형
연구 유형
등록 (추정된)
등록
단계
단계
- 해당 없음
연락처 및 위치
연구 연락처
연구 연락처
- 이름: Abi Spicer, PhD
- 전화번호: +44-7855 193162
- 이메일: MS-fempto@nottingham.ac.uk
연구 장소
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Gloucestershire
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Nottingham, Gloucestershire, 영국, NG7 2UH
- Sir Peter Mansfield Imaging Centre
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연락하다:
- Abi Spicer, PhD
- 전화번호: +447855 193162
- 이메일: MS-fempto@nottingham.ac.uk
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연락하다:
- Robin Spiller, MD
- 전화번호: +44-7824328319
- 이메일: robin.spiller@nottingham.ac.uk
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-
참여기준
자격 기준
자격 기준
공부할 수 있는 나이
- 성인
건강한 자원 봉사자를 받아들입니다
설명
Inclusion Criteria:
• Participant is willing and able to give informed consent for participation in the study.
- Proven diagnosis of CF.
- Aged between 18-60 years.
- Ability to conform to the study protocol, including overnight fasting, dietary and lifestyle restriction, administering linaclotide and placebo intervention, MRI scanning, consuming the two study meals and blue muffins, and rating stool frequency and appearance.
Exclusion Criteria:
- Pregnant (current or planned during the study duration) or breastfeeding
- Currently taking regular medicines (i.e., pain relief medication like ibuprofen/paracetamol) apart from cystic fibrosis medicines (i.e., pancreatic enzyme replacement therapy (PERT) medication, CFTR modulators). There are some other medicines it is OK to take such as:
- Some antidepressants (SSRIs or low-dose tricyclics)
- Antihistamines (for allergies)
- The contraceptive pill
- Any resections (surgery) of the intestines, not including removal of the appendix
- Serious medical conditions including any other digestive disorder unrelated to CF.
- Taken a course of intravenous or oral antibiotics in the last 2 weeks (except prophylactic antibiotics such as azithromycin).
- Inability to stop opiates
- Inability to have MRI scans (this could be due to some metallic implants, pacemaker, history of metallic foreign body in your eye and penetrating eye injury)
- Night shift work during the week before study days
- BMI under 18.5 or above 35 kg/m2.
- Taken part in a CTIMP study in the last 4 weeks involving invasive procedures.
- currently has chronic diarrhoea symptoms.
공부 계획
연구는 어떻게 설계됩니까?
디자인 세부사항
- 주 목적: 기초 과학
- 할당: 무작위
- 중재 모델: 크로스오버 할당
- 마스킹: 삼루타
팔의 수
무기와 개입
참가자 그룹 / 팔참가자 그룹 / 팔 |
개입 / 치료개입 / 치료 |
|---|---|
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위약 비교기: Placebo
encapsulated lactose
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lactose 20mg
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활성 비교기: linaclotide
linaclotide 290 ug over-encapsulated identical appearance to placebo
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linaclotide
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연구는 무엇을 측정합니까?
주요 결과 측정
주요 결과 측정
결과 측정 |
측정값 설명 |
기간 |
|---|---|---|
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Small Bowel Water Content AUC 0-6hours
기간: from enrolment until study end at approximately 4 weeks
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Area under curve Small Bowel Water Content
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from enrolment until study end at approximately 4 weeks
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2차 결과 측정
2차 결과 측정
결과 측정 |
측정값 설명 |
기간 |
|---|---|---|
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Change in Small Bowel Water Content (SBWC)
기간: from enrolment until study end at approximately 4 weeks
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change in SBWC after second meal from T300 to T360 ( ∆ SBWC),
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from enrolment until study end at approximately 4 weeks
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Orocaecal transit time (OCTT)
기간: from enrolment until study end at approximately 4 weeks
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Time from meal ingestion to appearance of head of meal in caecum
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from enrolment until study end at approximately 4 weeks
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T1 ascending colon
기간: from enrolment until study end at approximately 4 weeks
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T1 ascending colon(T1AC), a measure of colonic water content.
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from enrolment until study end at approximately 4 weeks
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공동 작업자 및 조사자
협력자
협력자
연구 기록 날짜
연구 주요 날짜
연구 시작 (추정된)
연구 시작
기본 완료 (추정된)
기본 완료
연구 완료 (추정된)
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
추가 관련 MeSH 약관
기타 연구 ID 번호
기타 연구 ID 번호
- FMHS 185-0526
개별 참가자 데이터(IPD) 계획
개별 참가자 데이터(IPD)를 공유할 계획입니까?
IPD 계획 설명
IPD 공유 기간
IPD 공유 액세스 기준
IPD 공유 지원 정보 유형
- 연구_프로토콜
- CSR
약물 및 장치 정보, 연구 문서
미국 FDA 규제 의약품 연구
미국 FDA 규제 기기 제품 연구
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