Cystic FibrosisNCT05986045
ENHANCE- Establishing Natural History in an Advanced New CF Care Era
An observational study of Cystic Fibrosis, run by Royal College of Surgeons, Ireland.
Trial summary
Measured outcomes for people with CF have improved dramatically over the last 20 years, even prior to the widespread introduction of cystic fibrosis transmembrane conductance regulator (CTFR) modulator treatments. Read more about this study
- Who is it for?
- Ages 1 month to 5 yearsHealthy volunteers can take part
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- Quality of Life
- How many people
- 550 participants
- Where
- No sites listed yet
- Study dates
- Oct 2023to 30 Sep 2028
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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 1 month to 5 years
- All sexes
- Healthy volunteers accepted
- Child
Study population: All participants with CF will be invited to participate in ENHANCE. We will ensure a presentative mix of mutation groups, sex, ages, ethnicity and location in all cohorts. We will target recruitment to cohorts 1 and 2 based on the following split of mutations: 50% F508del homozygous(FF), 20%heterozygous for F508del and a minimum function mutation(FMF), 20% heterozygous for F508del and a residual function/gating mutation or gating/other mutation and 10% with no currently treatable mutation(NON).
Who may be able to join
- Children with CF attending one of the study centres and fulling one of the following:
- Newborn infant diagnoses with cystic fibrosis through newborn screening (excludes children with an uncertain diagnosis), or having 2 documented CF disease causing mutations.
- Children with CF (sweat chloride>60mmol/L or 2 CF disease causing mutations) aged 0-6 at study initiation
- Healthy control infants without CF
Who cannot join
- Children or their parents not willing or able to complete with study procedures or assessments.
- Co-morbidities in groups 1 and 2, unrelated to CF, that in the opinion of the investigator would substantially impact on study measurements and unduly affect the veracity of the outcome data, for example a diagnosis of inflammatory bowel disease or extreme prematurity.
- Children in the control group who are carriers of CFTR mutations or have chronic medical or GI/Liver conditions that in the opinion of the investigator would unduly affect the veracity of the outcome data.
- We will not exclude someone who subsequently joins a CF Investigational drug trial if they are happy to continue, but if possible, will time their annual ENHANCE data collection to fall outside the time period of any experimental study drug administration
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
Official title: Establishing Natural History in an Advanced New CF Care Era
Measured outcomes for people with CF have improved dramatically over the last 20 years, even prior to the widespread introduction of cystic fibrosis transmembrane conductance regulator (CTFR) modulator treatments. The outlook for children with CF has improved significantly, with longer predicted survival and a lower likelihood of morbidity. This has accelerated recently. These changes have occurred within a short period of time, and there is much that we now do not understand about disease progression in children with CF and how this differs from children without CF. CF is an area which is fortunate to have well-developed and successful disease registries. CF registries have provided significant amounts of very useful data to guide improvement in treatment and outcomes over many decades. The power of registries comes from the collection of a well-defined set of important outcome measures in very large numbers of people over many years.
The outcome measures collected routinely in clinical care, which form part of the registries, are helpful in monitoring moderate-advances and symptomatic disease in people with CF. CF registries however do not tend to collect tomography(CT) scores, lung clearance index(LCI) or indeed repeated collection of biomarkers of disease activity such as sweat chloride which are increasingly relevant in an era of modulator therapies and reducing burden of symptomatic disease. We perceive an urgent need to complement registry data, cataloguing the changing natural history if early childhood CF by proactively collecting and curating sensitive, meaningful outcome data in a large cohort of children during this new era in Ireland and the UK.
The prevalence, presentation and natural history of disease manifestation of CF in young children will change significantly in the next decade with advances in the understanding and treatment of CF, including the use of therapies aimed at CFTR function. ENHANCE provides an opportunity to study these changes in real-time and in ways that are relevant to the CF community.
- Conditions
- Cystic Fibrosis
- Being tested
- Quality of Life
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Quality of LifeOther
ENHANCE will collect natural history on all children with cystic fibrosis who are enrolled over a 5 year period
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 | Newborn infants diagnosed with Cystic Fibrosis at newborn screening Receives: Other: Quality of Life | |
| Cohort 2 | Children with previous diagnosis of Cystic Fibrosis up to 5 years of age Receives: Other: Quality of Life | |
| Control | Newborn infants without cystic fibrosis Receives: Other: Quality of Life |
What is being measured
The primary outcome is the main question the study is designed to answer.
1. The incidence, prevalence and progression of structural lung disease
Spirometry-controlled Computed Tomography
Measured: 60 Months
2. The long-term natural history of pulmonary function and ventilation homogeneity.
Spirometry, Multiple Breath Washout
Measured: 60 Months
3. The incidence, prevalence and longitudinal progression of CF liver disease.
Liver Ultrasound, Liver Function Tests
Measured: 60 Months
4. The prevalence, natural history and progression of exocrine pancreatic dysfunction
Faecal Elastase Analysis
Measured: 60 Months
5. The longitudinal natural history of gastrointestinal symptoms, inflammation and the gut microbiome compared to a healthy control population
Microbiome Analysis, Identification of inflammatory markers, Abdominal Symptom Scores
Measured: 60 Months
6. The longitudinal natural history of annual sweat chloride levels in infants and children of different ages, the influence of different treatments on this and its association with other outcomes
Sweat chloride
Measured: 60 Months
7. The longitudinal natural history of mental health outcomes in children with CF compared to controls.
Mental Health Quality Of Life Questionnaires
Measured: 60 Months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Aug 2023
- Study startOct 2023 (estimated)
- Primary completion30 Sep 2028 (estimated)
- Study completion30 Sep 2028 (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
- Case-control
- Time perspective
- Prospective
- Enrolment
- 550 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Royal College of Surgeons, Ireland
- Sponsor type
- University, hospital or other
- Collaborators
- University Hospital of Limerick, Cork University Hospital, University College Hospital Galway, Belfast Health and Social Care Trust, NHS Lothian, Alder Hey Children's NHS Foundation Trust, Manchester University NHS Foundation Trust, Newcastle-upon-Tyne Hospitals NHS Trust, Cardiff and Vale University Health Board, Royal Brompton & Harefield NHS Foundation Trust, Erasmus University Rotterdam, Medizinische Hochschule Brandenburg Theodor Fontane, Massachusetts General Hospital, The Hospital for Sick Children, Teagasc
- Responsible party
- Sponsor
- Investigators
- Paul McNally, Principal investigator, RCSI
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05986045
- Sponsor's protocol ID
- ENHANCE
- Status last verified
- Jul 2023
- First submitted
- 3 Aug 2023
- Last updated
- 31 Aug 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 31 Aug 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Establishing Natural History in an Advanced New CF Care Era
- ClinicalTrials.gov ID
- NCT05986045
- Acronym / sponsor ID
- ENHANCE
- Conditions
- Cystic Fibrosis
- Study type
- Observational
- Recruitment status
- Not yet recruiting
- Enrolment
- 550 participants (estimated)
- Lead sponsor
- Royal College of Surgeons, IrelandUniversity, hospital or other
- Collaborators
- University Hospital of Limerick, Cork University Hospital, University College Hospital Galway, Belfast Health and Social Care Trust, NHS Lothian, Alder Hey Children's NHS Foundation Trust, Manchester University NHS Foundation Trust, Newcastle-upon-Tyne Hospitals NHS Trust, Cardiff and Vale University Health Board, Royal Brompton & Harefield NHS Foundation Trust, Erasmus University Rotterdam, Medizinische Hochschule Brandenburg Theodor Fontane, Massachusetts General Hospital, The Hospital for Sick Children, Teagasc
- Study start
- Oct 2023 (estimated)
- Primary completion
- 30 Sep 2028 (estimated)
- Study completion
- 30 Sep 2028 (estimated)
- First posted
- 14 Aug 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.
Measured outcomes for people with CF have improved dramatically over the last 20 years, even prior to the widespread introduction of cystic fibrosis transmembrane conductance regulator (CTFR) modulator treatments. The outlook for children with CF has improved significantly, with longer predicted survival and a lower likelihood of morbidity. This has accelerated recently. These changes have occurred within a short period of time, and there is much that we now do not understand about disease progression in children with CF and how this differs from children without CF. CF is an area which is fortunate to have well-developed and successful disease registries. CF registries have provided significant amounts of very useful data to guide improvement in treatment and outcomes over many decades. The power of registries comes from the collection of a well-defined set of important outcome measures in very large numbers of people over many years.
The outcome measures collected routinely in clinical care, which form part of the registries, are helpful in monitoring moderate-advances and symptomatic disease in people with CF. CF registries however do not tend to collect tomography(CT) scores, lung clearance index(LCI) or indeed repeated collection of biomarkers of disease activity such as sweat chloride which are increasingly relevant in an era of modulator therapies and reducing burden of symptomatic disease. We perceive an urgent need to complement registry data, cataloguing the changing natural history if early childhood CF by proactively collecting and curating sensitive, meaningful outcome data in a large cohort of children during this new era in Ireland and the UK.
The prevalence, presentation and natural history of disease manifestation of CF in young children will change significantly in the next decade with advances in the understanding and treatment of CF, including the use of therapies aimed at CFTR function. ENHANCE provides an opportunity to study these changes in real-time and in ways that are relevant to the CF community.
Study design
- Observational model
- Case-control
- Time perspective
- Prospective
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 | – | Other: Quality of LifeNewborn infants diagnosed with Cystic Fibrosis at newborn screening |
| Cohort 2 | – | Other: Quality of LifeChildren with previous diagnosis of Cystic Fibrosis up to 5 years of age |
| Control | – | Other: Quality of LifeNewborn infants without cystic fibrosis |
Who may qualify
- Ages
- 1 month to 5 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child
- Study population
- All participants with CF will be invited to participate in ENHANCE. We will ensure a presentative mix of mutation groups, sex, ages, ethnicity and location in all cohorts. We will target recruitment to cohorts 1 and 2 based on the following split of mutations: 50% F508del homozygous(FF), 20%heterozygous for F508del and a minimum function mutation(FMF), 20% heterozygous for F508del and a residual function/gating mutation or gating/other mutation and 10% with no currently treatable mutation(NON).
Key inclusion criteria (4 listed)
- Newborn infant diagnoses with cystic fibrosis through newborn screening (excludes children with an uncertain diagnosis), or having 2 documented CF disease causing mutations.
- Children with CF (sweat chloride>60mmol/L or 2 CF disease causing mutations) aged 0-6 at study initiation
- Healthy control infants without CF
Key exclusion criteria (4 listed)
- Children or their parents not willing or able to complete with study procedures or assessments.
- Co-morbidities in groups 1 and 2, unrelated to CF, that in the opinion of the investigator would substantially impact on study measurements and unduly affect the veracity of the outcome data, for example a diagnosis of inflammatory bowel disease or extreme prematurity.
- Children in the control group who are carriers of CFTR mutations or have chronic medical or GI/Liver conditions that in the opinion of the investigator would unduly affect the veracity of the outcome data.
- We will not exclude someone who subsequently joins a CF Investigational drug trial if they are happy to continue, but if possible, will time their annual ENHANCE data collection to fall outside the time period of any experimental study drug administration
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 |
|---|---|---|
| Quality of LifeENHANCE will collect natural history on all children with cystic fibrosis who are enrolled over a 5 year period | Other | Cohort 1, Cohort 2, Control |
Primary outcome
1. The incidence, prevalence and progression of structural lung disease
Time frame: 60 Months
Spirometry-controlled Computed Tomography
2. The long-term natural history of pulmonary function and ventilation homogeneity.
Time frame: 60 Months
Spirometry, Multiple Breath Washout
3. The incidence, prevalence and longitudinal progression of CF liver disease.
Time frame: 60 Months
Liver Ultrasound, Liver Function Tests
4. The prevalence, natural history and progression of exocrine pancreatic dysfunction
Time frame: 60 Months
Faecal Elastase Analysis
5. The longitudinal natural history of gastrointestinal symptoms, inflammation and the gut microbiome compared to a healthy control population
Time frame: 60 Months
Microbiome Analysis, Identification of inflammatory markers, Abdominal Symptom Scores
6. The longitudinal natural history of annual sweat chloride levels in infants and children of different ages, the influence of different treatments on this and its association with other outcomes
Time frame: 60 Months
Sweat chloride
7. The longitudinal natural history of mental health outcomes in children with CF compared to controls.
Time frame: 60 Months
Mental Health Quality Of Life Questionnaires
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 Aug 2023.
- Individual participant dataSharing planned: No
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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.
Paul McNally principal investigator
RCSI
Questions about this trial
What is this clinical trial studying?
An observational study of Cystic Fibrosis, run by Royal College of Surgeons, Ireland.
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 4 inclusion and 4 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: opening soon, as of the sponsor's last update on 31 Aug 2023. Status can change between updates, so confirm with the study contact before making plans.
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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Karen Lester, PhD ((01) 4096500, karenlester@rcsi.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
1. The incidence, prevalence and progression of structural lung disease (time frame: 60 Months). The study lists 7 primary outcomes in all. 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 31 Aug 2023.