NCT07402434Toborzás még nem indult
A Prospective Study of Advanced Diagnostics in People With an Unclear Diagnosis of Cystic Fibrosis
Advanced Diagnostic Validation and Novel Clinical Evaluation Across the CFTR Spectrum (ADVANCE-CFTR): a Prospective Study of CFTR Functional Assays, Including Rectal Organoids, to Improve the Accuracy of Diagnosing Cystic Fibrosis.
Forrás: ClinicalTrials.gov · Utoljára frissítve: 2026. febr. 17.
Rövid összefoglaló (regiszter, angolul)
Sometimes it is very difficult to tell if someone has cystic fibrosis (CF), especially when they have rare CF genes. Without this certainty, they are unlikely to get the correct treatment so their health may be affected. More accurate ways to test for CF are therefore needed in this situation. The aim of this study is to develop a more accurate test using what are called "organoids" or "mini organs." Organoids are grown in the laboratory from a small piece of gut tissue. As they have the person's exact genes, they can show if the CF gene ("CFTR") is working correctly or not and thus if the person has CF. The investigators will compare the organoid response with the current more established tests, such as the sweat test and CF genetics, and other recognised specialist tests called nasal potential difference (NPD) and intestinal current measurement (ICM). The gut tissue is usually taken by a quick, relatively painless, outpatient procedure (rectal biopsy). The additional benefit of organoids is that they can also help us to work out the best treatment for that individual by testing how well the gut tissue responds to different drugs in the laboratory. The investigators wish to carry out this research to prove that gut organoids are a better way to test for CF in this situation. The goal will be to diagnose people faster and for them to get better treatment quicker, both key for leading a longer and healthier life.
- Állapot (regiszter)
- Toborzás még nem indult
- Vizsgálattípus
- Megfigyeléses
- Résztvevők száma
- 80 (tervezett)
- Első benyújtás
- 2026. febr. 4.
- Kezdés
- 2026. febr. 2.
- Elsődleges befejezés
- 2028. jún. 1.
- Fő szponzor
- Royal Brompton & Harefield NHS Foundation Trust
- Betegségek
- Cisztás fibrózis
- NCT-szám
- NCT07402434
Mit vállalsz
- Vizsgálat típusa: Megfigyeléses
- Tervezett résztvevők: 80
- 2026. febr. 2. és az elsődleges befejezés (2028. jún. 1.) között
- 1 helyszín, 1 országban
- Vizitek és vizsgálatok: kérdezd a vizsgálat csapatát, a regiszter nem sorolja fel őket
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Korábbi kezelés
- Receiving CFTR modulator treatment
- Current participation (or participation within one month of enrolment) in a clinical trial of an investigational medicinal product which affects CFTR function (e.g. CFTR modulators, genetic therapies)
- Current participation (or participation within one month of enrolment) in a clinical trial of an investigational medicinal product which affects CFTR function (e.g. CFTR modulators, genetic therapies)
Más betegség
- Contra-indication to rectal biopsy (e.g. significant bleeding diathesis)
- History of lung transplantation
- Contra-indication to rectal biopsy (e.g. significant bleeding diathesis)
- Contra-indication to rectal biopsy (e.g. significant bleeding diathesis)
Egyéb
- Group A: Difficult Cystic Fibrosis Diagnosis Cohort
- Female who is pregnant or breastfeeding
- Unable to provide informed consent
- Group B: Cystic Fibrosis Patient Cohort
- Female who is pregnant or breastfeeding
- Unable to provide informed consent
- Exclude all subjects where diagnosis of CF is in doubt
- Group C: Non-Cystic Fibrosis Patient Cohort
- Female who is pregnant or breastfeeding
- Unable to provide informed consent
Kiket keres a vizsgálat
- Group A: Difficult Cystic Fibrosis Diagnosis Cohort
- Patients with difficult-to-diagnose CF*
- Adults (≥16y)
- Willing to undergo rectal biopsy
- Able to comply with the study
- Definition: Non-diagnostic first line CFTR testing (individuals who do not fulfil CF diagnostic criteria [2 CF-causing CFTR variants and/or sweat chloride concentration ≥60 mmol/L]). Of note, there are some individuals with <2 CF-causing CFTR variants after extended CFTR genetic analysis but with a SCC >60mmol/L. Expansion of the patient pool to include these is permissible.
- Group B: Cystic Fibrosis Patient Cohort
- Patients with confirmed Cystic Fibrosis
- Adults (≥16y)
- Willing to undergo rectal biopsy for this study
- Able to comply with the study
- Group C: Non-Cystic Fibrosis Patient Cohort
- Patients without Cystic Fibrosis or CFTR-related disorder
- Adults (≥16y)
- Willing to undergo rectal biopsy for this study (if undergoing a lower GI endoscopy for other purposes)
- Able to comply with the study
Életkor a regiszterben: 16 év – - · Nem: nők és férfiak · Egészséges önkénteseket is fogad
Helyszínek (1)
Helyszínek a regiszter sorrendjében. Ha a profilodba mented a városodat, távolság szerint rendeződnek.
| Helyszín | Város | Ország | Helyszín állapota |
|---|---|---|---|
| Royal Brompton Hospital | London | Egyesült Királyság | Ismeretlen állapot |
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