Cystic fibrosis Nutritional, Metabolic, Endocrine Cystic fibrosis
Conditions
Interventions
CF STORM will be a randomised open-label trial, undertaken on a national patient registry, to evaluate whether people with cystic fibrosis (pwCF) can rationalise their daily treatment without a signif
this takes place after all baseline measurements have been completed.
Percent predicted Forced Expiratory Volume in One Second (ppFEV1), need for extra antibiotic treatment, quality of life (QoL), an
Sponsors
Alder Hey Children's NHS Foundation Trust
Eligibility
Sex/Gender
All
Age
6 Years to 110 Years
Inclusion criteria
Inclusion criteria: Current key inclusion criteria as of 25/09/2025: 1. Clear diagnosis and clinical features of Cystic Fibrosis 2. One or two Phe508del variants 3. Established on daily mucoactive nebulised therapy (hypertonic saline or Dornase alfa or both) for at least 3 months 4. 6 years of age or older 5. Established on Kaftrio™ for at least 3 months 6. Enrolled in the UK CF Registry 7. Able to undertake spirometry 8. No need for extra antibiotics (oral or intravenous) in the previous 2 weeks 9. Completed informed consent and assent if applicable, obtained from the participant, participant's parent, or legal representative and agreement of the participant to comply with the requirements of the study Previous key inclusion criteria: 1. Clear diagnosis and clinical features of Cystic Fibrosis 2. One or two Phe508del variants 3. Established on daily mucoactive nebulised therapy (hypertonic saline or Dornase alfa or both) for at least 3 months 4. Aged =12 years 5. Established on Kaftrio™ for at least 3 months 6. Enrolled in the UK CF Registry 7. Able to undertake spirometry 8. No need for extra antibiotics (oral or intravenous) in the previous 6 weeks 9. Completed informed consent and assent if applicable, obtained from the participant, participant's parent, or legal representative and agreement of the participant to comply with the requirements of the study
Exclusion criteria
Exclusion criteria: 1. Significant adverse reaction to Kaftrio™ requiring dose change during the previous 3 months 2. ppFEV1 below 40% after Kaftrio™ therapy at any point during the previous 3 months 3. History of solid organ transplant or placed on the active transplant waiting list 4. Other non-CF condition that, in the opinion of the local CF team, significantly impacts on clinical progress 5. Participation in another Clinical Trial of an Investigational Medicinal Product (CTIMP) within the previous 3 months (the 3-month period does not apply to open-label Kaftrio™ CTIMPs) 6. Prescribed Mannitol dry powder for inhalation as part of usual daily CF care within the previous 6 weeks
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Change in percent predicted Forced Expiratory Volume in One Second (ppFEV1) measured using spirometry at baseline, 52 weeks, and any other relevant encounters during the trial period | — |
Secondary
| Measure | Time frame |
|---|---|
| 1. Change in respiratory function measured as the incidence of Forced Expiratory Flow between 25-75% of vital capacity using spirometry at baseline and 52 weeks 2. Need for extra antibiotic treatment measured using the number of courses and the total number of days of extra antibiotics (oral, intravenous and nebulised) at baseline and 52 weeks 3. Need for extra chronic medications measured using the number of courses and the total number of days of chronic medications (oral and nebulised) at baseline and 52 weeks 4. Number and proportion of respiratory cultures positive for significant pathogens measured using respiratory cultures at baseline and 52 weeks and any other relevant encounters during the trial period 5. Need for hospital admission measured from the number of separate hospital inpatient stays at 52 weeks and any other relevant encounters during the trial period 6. Change in nutritional status measured using Body Mass Index (BMI) calculated from weight (kg) and height (cm) at baseline and 52 weeks 7. Number of clinician-determined pulmonary exacerbations measured from participant records at baseline and 52 weeks and any other relevant encounters during the trial period 8. Change in disease-specific Quality of Life measured using Cystic Fibrosis Questionnaire-Revised (CFQ-R) at baseline and 52 weeks 9. Adverse events relating to a large drop in respiratory function or treatment of pulmonary exacerbation with IV antibiotics measured using spirometry to calculate ppFEV1 and use of intravenous antibiotics (including dates and drug name) within 4 weeks of the event and at 52 weeks 10. Costs to the NHS measured using treatment costs and compliance to allocation at 52 weeks and any other relevant encounters during the trial period 11. To determine if the ‘STOP’ intervention represents value for money measured using Incremental cost per QALY gained compared to ‘CONTINUE’ arm, estimated using the EQ-5D-5L QoL measure at baseline, 17, 34, 50, and 52 weeks | — |
Countries
England, Northern Ireland, United Kingdom
Contacts
Public ContactAbigail Williams
Outcome results
None listed