Cystic FibrosisGIFT-CFJuniorNCT05699148
Gut Imaging for Function and Transit in Cystic Fibrosis 3 Junior
An observational study of Cystic Fibrosis, run by Nottingham University Hospitals NHS Trust at 1 site in United Kingdom.
Trial summary
Recently trial data has shown that the medicine KaftrioTM (Elexacaftor/ Tezacaftor/ Ivacaftor) improves lung function in children aged 6 to 11 years who have cystic fibrosis (CF). This has led to it being licensed for use in the UK in 2022 and is now being prescribed in this age group. Read more about this study
- Who is it for?
- Ages 6 to 11 yearsHealthy volunteers can take part
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- Elexacaftor / Ivacaftor / Tezacaftor
- How many people
- 17 participants
- Where
- 1 siteUnited Kingdom
- Study dates
- 1 Apr 2022to 1 Apr 2023
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 to 11 years
- All sexes
- Healthy volunteers accepted
- Child
Study population: Children with CF aged 6 to 11 years. Must have at least one copy of the Phe508del mutation. These children would be planned to start receiving ETI as part of their routine clinical care.
Who may be able to join
- Children with CF aged 6 to 11 years. Must have at least one copy of the Phe508del mutation.
- Healthy volunteers must have no previous history of gastrointestinal disease.
Who cannot join
- Healthy volunteers with a history of gastrointestinal disease.
Trial locations and contacts
1 site in 1 country.
United Kingdom1 site
Queen's Medical Centre
Nottingham, Nottinghamshire, NG7 2UH
About this study
Official title: Gut Imaging for Function and Transit in Cystic Fibrosis 3 Junior: an Evaluation of Triple Combination Therapy in Children Aged 6 to 11 Years
Recently trial data has shown that the medicine KaftrioTM (Elexacaftor/ Tezacaftor/ Ivacaftor) improves lung function in children aged 6 to 11 years who have cystic fibrosis (CF). This has led to it being licensed for use in the UK in 2022 and is now being prescribed in this age group. There is little information in trials however that shows the effect KaftrioTM (ETI) has on the gut or liver in this age group.
Previous studies in the GIFT-CF series (NCT 03566550, NCT04006873 and NCT04618185) has found differences in the functioning of the gut between adults with CF and healthy controls but it is not known whether these differences are present in those aged 6 to 11 years.
Read more
This study is a significant amendment of the GIFT-CF3 protocol (NCT04618185) that aims to measure gut function using magnetic resonance imaging (MRI) in children with CF before and after starting ETI. This study also aims to opportunistically measure lung function and structure using MRI and explore how the liver can be measured using MRI in this age group.
The study is split into 2 stages. The first is a pilot stage using the modified GIFT-CF protocol recruiting 3 children with CF before starting ETI and 3 healthy volunteers. This is to determine we are able to successfully perform these scans in these age groups. If successful, the second stage will recruit a further 12 children with CF before they start ETI. This will take our cohort up to 15 children with CF. This cohort will then be rescanned 6 months after starting ETI using the same scan protocol.
Read the detailed description
This is an observational cohort study using MRI to measure gastrointestinal (GI) function, lung structure and function and liver endpoints in children with CF aged 6 to 11 years. The study will use a modified version of the protocol used in the GIFT-CF3 study (NCT04618185) to measure GI physiology in addition to MRI measures of lung and liver structure and function. The aim is to identify whether these measures are changed before and after starting ETI.
For the CF participants, to be enrolled in the study they must be homozygous or have at least 1 copy of the Phe508del mutation, which would mean they are eligible for ETI treatment.
The modified MRI protocol, taken from NCT04618185, has been shortened to include scans that are taken over three timepoints. These timepoints are determined around the ingestion of a test meal which has been previously described. Participants will arrive fasted before having a baseline scan. After their first scan they will eat their first standardised high fat meal followed by a second standardised high fat meal around 240 minutes after.
MRI scans to assess GI function will be taken at baseline (fasted), 240 minutes post ingestion of a breakfast meal (T240) and 300 minutes post breakfast (T300). Liver scans will also be taken at baseline and lung scans taken opportunistically at the T240 timepoint. In total, participants will be asked to remain in the MRI scanner for approximately 30 minutes at a time. For comfort, participants will be able to watch multimedia programmes through the use of a specially adapted television while in the scanner.
Participants will also be asked to complete validated gastrointestinal questionnaires throughout the study day and recall their GI symptoms over the previous 2 weeks and complete a 3 day food diary.
The study will be split into two phases. The first will be a pilot phase where 3 children with CF aged 6 to 11 years and 3 healthy age and gender matched controls undergo MRI scanning. These scans will be assessed to determine whether the scan protocol can be completed by the participants and assess the quality of the images obtained. If successful, the trial will progress onto the main phase.
In the main phase, the 12 additional children with CF will be recruited and undergo the above scanning protocol, prior to starting ETI. We will then ask participants to return for a repeat scans approximately 6 months after starting ETI.
- Conditions
- Cystic Fibrosis
- Being tested
- Elexacaftor / Ivacaftor / Tezacaftor
- Keywords
- Cystic Fibrosis, CFTR modulators, Gastrointestinal, Lung, Liver, Magnetic resonance imaging, Elexacaftor/ tezacaftor/ ivacaftor
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Elexacaftor / Ivacaftor / TezacaftorDrug
Also called Kaftrio, Trikafta
Triple combination therapy is licensed in the UK for prescription to paediatric patients aged 6 to 11 years. ETI will be started as part of routine clinical care.
| Study group | Role | What they receive |
|---|---|---|
| Children with cystic fibrosis aged 6 to 11 years | Group of children who have at least one copy of Phe508del gene mutation and are eligible for starting on the modulator elexacaftor/ tezacaftor/ ivacaftor (ETI). This group will have baseline MRI scans before starting ETI and aiming to have subsequent scans post starting ETI (6 months to 1 year post starting ETI) Receives: Drug: Elexacaftor / Ivacaftor / Tezacaftor | |
| Control Group | Age and gender matched controls with no history of cystic fibrosis or gastrointestinal disease. This group will undergo one set of scans only. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Delta Small Bowel Water (DeltaSBW)
This measurement relates to the postprandial fall in small bowel water content post second study meal. This will be adjusted to body surface area (mL/m2)
Measured: This a measure of the change in small bowel water between study time points 240 minutes and 300 minutes post first study meal.
Secondary outcomes (4)
Small bowel water content
Volume of small bowel water within the small bowel, adjusted to body surface area (mL/m2)
Measured: Will be measured at study timepoints: baseline, 240 minutes and 300 minutes and then area under the curve calculated using the three timepoints (L.min/m2)
Liver volume
Measurement of total liver volume during fasted baseline scan
Measured: Measured during fasted, baseline MRI scan
Liver elastography
Measurement of liver firmness using MRI elastography
Measured: Measured during fasted, baseline MRI scan
Lung ventilation
Measured using phase-resolved functional lung (PREFUL) MRI sequence
Measured: Measured at 240 minute time point
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Jan 2023
- Study start1 Apr 2022
- Primary completion1 Aug 2022
- Study completion1 Apr 2023 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 17 participants (actual)
Sponsor and investigators
- Lead sponsor
- Nottingham University Hospitals NHS Trust
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Andrew Prayle, Principal investigator, Associate ProfessorAlan Smyth, Principal investigator, Professor of Child Health
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05699148
- Sponsor's protocol ID
- 20CS036 sub-study
- Acronym
- GIFT-CFJunior
- Status last verified
- Jan 2023
- First submitted
- 10 Jan 2023
- Last updated
- 26 Jan 2023
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 26 Jan 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Gut Imaging for Function and Transit in Cystic Fibrosis 3 Junior: an Evaluation of Triple Combination Therapy in Children Aged 6 to 11 Years
- ClinicalTrials.gov ID
- NCT05699148
- Acronym / sponsor ID
- GIFT-CFJunior / 20CS036 sub-study
- Conditions
- Cystic Fibrosis
- Keywords
- Cystic Fibrosis, CFTR modulators, Gastrointestinal, Lung, Liver, Magnetic resonance imaging, Elexacaftor/ tezacaftor/ ivacaftor
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 17 participants (actual)
- Lead sponsor
- Nottingham University Hospitals NHS TrustUniversity, hospital or other
- Study start
- 1 Apr 2022 (actual)
- Primary completion
- 1 Aug 2022 (actual)
- Study completion
- 1 Apr 2023 (estimated)
- First posted
- 26 Jan 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Recently trial data has shown that the medicine KaftrioTM (Elexacaftor/ Tezacaftor/ Ivacaftor) improves lung function in children aged 6 to 11 years who have cystic fibrosis (CF). This has led to it being licensed for use in the UK in 2022 and is now being prescribed in this age group. There is little information in trials however that shows the effect KaftrioTM (ETI) has on the gut or liver in this age group.
Previous studies in the GIFT-CF series (NCT 03566550, NCT04006873 and NCT04618185) has found differences in the functioning of the gut between adults with CF and healthy controls but it is not known whether these differences are present in those aged 6 to 11 years.
This study is a significant amendment of the GIFT-CF3 protocol (NCT04618185) that aims to measure gut function using magnetic resonance imaging (MRI) in children with CF before and after starting ETI. This study also aims to opportunistically measure lung function and structure using MRI and explore how the liver can be measured using MRI in this age group.
The study is split into 2 stages. The first is a pilot stage using the modified GIFT-CF protocol recruiting 3 children with CF before starting ETI and 3 healthy volunteers. This is to determine we are able to successfully perform these scans in these age groups. If successful, the second stage will recruit a further 12 children with CF before they start ETI. This will take our cohort up to 15 children with CF. This cohort will then be rescanned 6 months after starting ETI using the same scan protocol.
Detailed description
This is an observational cohort study using MRI to measure gastrointestinal (GI) function, lung structure and function and liver endpoints in children with CF aged 6 to 11 years. The study will use a modified version of the protocol used in the GIFT-CF3 study (NCT04618185) to measure GI physiology in addition to MRI measures of lung and liver structure and function. The aim is to identify whether these measures are changed before and after starting ETI.
For the CF participants, to be enrolled in the study they must be homozygous or have at least 1 copy of the Phe508del mutation, which would mean they are eligible for ETI treatment.
The modified MRI protocol, taken from NCT04618185, has been shortened to include scans that are taken over three timepoints. These timepoints are determined around the ingestion of a test meal which has been previously described. Participants will arrive fasted before having a baseline scan. After their first scan they will eat their first standardised high fat meal followed by a second standardised high fat meal around 240 minutes after.
MRI scans to assess GI function will be taken at baseline (fasted), 240 minutes post ingestion of a breakfast meal (T240) and 300 minutes post breakfast (T300). Liver scans will also be taken at baseline and lung scans taken opportunistically at the T240 timepoint. In total, participants will be asked to remain in the MRI scanner for approximately 30 minutes at a time. For comfort, participants will be able to watch multimedia programmes through the use of a specially adapted television while in the scanner.
Participants will also be asked to complete validated gastrointestinal questionnaires throughout the study day and recall their GI symptoms over the previous 2 weeks and complete a 3 day food diary.
The study will be split into two phases. The first will be a pilot phase where 3 children with CF aged 6 to 11 years and 3 healthy age and gender matched controls undergo MRI scanning. These scans will be assessed to determine whether the scan protocol can be completed by the participants and assess the quality of the images obtained. If successful, the trial will progress onto the main phase.
In the main phase, the 12 additional children with CF will be recruited and undergo the above scanning protocol, prior to starting ETI. We will then ask participants to return for a repeat scans approximately 6 months after starting ETI.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Children with cystic fibrosis aged 6 to 11 years | – | Drug: Elexacaftor / Ivacaftor / TezacaftorGroup of children who have at least one copy of Phe508del gene mutation and are eligible for starting on the modulator elexacaftor/ tezacaftor/ ivacaftor (ETI). This group will have baseline MRI scans before starting ETI and aiming to have subsequent scans post starting ETI (6 months to 1 year post starting ETI) |
| Control Group | – | –Age and gender matched controls with no history of cystic fibrosis or gastrointestinal disease. This group will undergo one set of scans only. |
Who may qualify
- Ages
- 6 to 11 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child
- Study population
- Children with CF aged 6 to 11 years. Must have at least one copy of the Phe508del mutation. These children would be planned to start receiving ETI as part of their routine clinical care.
Key inclusion criteria (2 listed)
- Children with CF aged 6 to 11 years. Must have at least one copy of the Phe508del mutation.
- Healthy volunteers must have no previous history of gastrointestinal disease.
Key exclusion criteria (1 listed)
- Healthy volunteers with a history of gastrointestinal disease.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Elexacaftor / Ivacaftor / TezacaftorAlso: Kaftrio, TrikaftaTriple combination therapy is licensed in the UK for prescription to paediatric patients aged 6 to 11 years. ETI will be started as part of routine clinical care. | Drug | Children with cystic fibrosis aged 6 to 11 years |
Primary outcome
Delta Small Bowel Water (DeltaSBW)
Time frame: This a measure of the change in small bowel water between study time points 240 minutes and 300 minutes post first study meal.
This measurement relates to the postprandial fall in small bowel water content post second study meal. This will be adjusted to body surface area (mL/m2)
4 secondary outcomes
Small bowel water content
Time frame: Will be measured at study timepoints: baseline, 240 minutes and 300 minutes and then area under the curve calculated using the three timepoints (L.min/m2)
Liver volume
Time frame: Measured during fasted, baseline MRI scan
Liver elastography
Time frame: Measured during fasted, baseline MRI scan
Lung ventilation
Time frame: Measured at 240 minute time point
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Jan 2023.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Andrew Prayle principal investigator
Associate Professor
Alan Smyth principal investigator
Professor of Child Health
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Cystic Fibrosis, run by Nottingham University Hospitals NHS Trust at 1 site in United Kingdom.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 26 Jan 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Delta Small Bowel Water (DeltaSBW) (time frame: This a measure of the change in small bowel water between study time points 240 minutes and 300 minutes post first study meal.). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Jan 2023.