Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.
COMPLETED
24 participants
OBSERVATIONAL
2020-10-21
2023-06-28
Brief Summary
Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.
* Patients with CF who are p.Phe508del homozygotes will already be receiving the less effective CFTR modulator drug Symkevi (Tezacaftor / Ivacaftor) and will switch to KaftrioTM.
* Patients who are who are compound heterozygotes with at least 1 copy of p.Phe508del currently have access to no effective CFTR modulator and will be starting a CFTR modulator (Kaftrio) for the first time.
Participants attend a study visit before Kaftrio treatment commences, followed by visits at 12 and 24 weeks after starting treatment. At each visit they will be scanned before and after standardised meals in the morning and mid-day (11 scans in total over 6 hours). No intravenous contrast or bowel preparation will be used. Participants will complete questionnaires on gastrointestinal symptoms as well as providing stool and sputum samples for assessment of microbiome and stool for inflammatory mediators and pancreatic function (elastase).
\*\*Following an extension, participants had a further visit at 76 weeks post starting Kaftrio, updated in detailed description\*\*
Related Clinical Trials
Explore similar clinical trials based on study characteristics and research focus.
Gut Imaging for Function and Transit in Cystic Fibrosis 3 Junior
NCT05699148
Gut Imaging for Function & Transit in Cystic Fibrosis Study 2
NCT04006873
Real-world Efficacy and Safety of Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Modulator Therapy in Adult Patients With Cystic Fibrosis (CF)
NCT05526027
Study of Ivacaftor in Subjects With Cystic Fibrosis (CF) Who Have a Non-G551D CF Transmembrane Conductance Regulator (CFTR) Gating Mutation
NCT01614470
Description of the Short-term Effects of KAFTRIO® by Continuous Monitoring With the PHEAL-CR-K Application in Real Life in Patients With Cystic Fibrosis Eligible for KAFTRIO® Treatment
NCT05295524
Detailed Description
Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.
Participants will attend 4 times, following an extension (pre-Kaftrio, at 12 weeks of Kaftrio at 24 weeks of Kaftrio and 76 weeks of Kaftrio) for MRI scanning at the Sir Peter Mansfield Imaging Centre, after an overnight fast. On this day, participants will be asked to withhold any medicines directly altering bowel habit such as laxatives. They will continue to take pancreatic enzyme replacement therapy and other medications for CF.
The Investigators will use the same MRI protocol as described in GIFT-CF (NCT03566550 and NCT04006873). Participants will have their first MRI scan fasted. After the first scan, they will eat a first standardised meal . They will then have 7 MRI scans at half hourly intervals and 3 MRI scans at hourly intervals. Participants will be given a second standardised meal after their ninth MRI scan. Each MRI scan will last approximately 15 minutes. After each MRI scan, participants will complete a validated gastrointestinal symptom questionnaire (Gastrointestinal Symptom Rating Scale). In between scans, participants will have access to an adjacent room with Wifi and television access.
They will also complete gut symptom questionnaires relating to the preceding 2 weeks and a 3 day food diary. They will also provide a sputum and stool sample.
Infection control requirements mean that only 1 participant will attend for MRI scanning per day. Extra COVID-19 precautions will also be taken throughout scan days.
\*\*\*Please note, due to the timing of EMA approval for Kaftrio and ethics approval for the study granted on 21st Ocober 2020, the clinicaltrials.gov registration was created on 21st October 2020 and approved by the clinicaltrials.gov PRS team on 4th November 2020.\*\*\*
Conditions
See the medical conditions and disease areas that this research is targeting or investigating.
Study Design
Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.
COHORT
PROSPECTIVE
Study Groups
Review each arm or cohort in the study, along with the interventions and objectives associated with them.
p.Phe508del homozygous genotype
People with CF with 2 copies of p.Phe508del and previously eligible for Symkevi (Tezacaftor/Ivacaftor)
Magnetic Resonance Imaging (MRI)
MRI to study gut function and transit without the risk of exposure to ionising radiation.
p.Phe508del heterozygous genotype
People with CF with 1 copy of p.Phe508del and not previously eligible for any CFTR modulator
Magnetic Resonance Imaging (MRI)
MRI to study gut function and transit without the risk of exposure to ionising radiation.
Interventions
Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.
Magnetic Resonance Imaging (MRI)
MRI to study gut function and transit without the risk of exposure to ionising radiation.
Eligibility Criteria
Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.
Inclusion Criteria
* Capacity to consent, or to understand the requirements of the study where parental consent is needed.
* Confirmed diagnosis of CF, either by sweat test or genetic testing.
* Genotype homozygous p.Phe508del or compound heterozygous with at least 1 copy of p.Phe508del.
* Eligible for KaftrioTM (Elexacaftor / Tezacaftor / Ivacaftor) treatment but not yet commenced Kaftrio treatment as part of routine care.
Exclusion Criteria
* FEV1 \< 40% (% predicted using Global Lung Initiative values)
* Unable to stop medications directly prescribed to alter bowel habit, such as laxatives of anti-diarrhoeals, on the study day
* Previous resection of small bowel \>20cm in length
* Intestinal stoma
* Diagnosis of inflammatory bowel disease or coeliac disease, confirmed by biopsy
* Gastrointestinal malignancy
* Unable to comply with dietary restrictions required for the study
* Pregnancy - tests are available at the SPMIC if participants are unsure
* Unable to speak or understand English
12 Years
60 Years
ALL
No
Sponsors
Meet the organizations funding or collaborating on the study and learn about their roles.
Manchester Metropolitan University
OTHER
Vertex Pharmaceuticals Incorporated
INDUSTRY
Nottingham University Hospitals NHS Trust
OTHER
Responsible Party
Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.
Principal Investigators
Learn about the lead researchers overseeing the trial and their institutional affiliations.
Alan Smyth
Role: PRINCIPAL_INVESTIGATOR
University of Nottingham
Locations
Explore where the study is taking place and check the recruitment status at each participating site.
Nottingham University Hospitals NHS Trust
Nottingham, Nottinghamshire, United Kingdom
Countries
Review the countries where the study has at least one active or historical site.
Other Identifiers
Review additional registry numbers or institutional identifiers associated with this trial.
20CS036
Identifier Type: -
Identifier Source: org_study_id
More Related Trials
Additional clinical trials that may be relevant based on similarity analysis.