Inhaled Therapy Adherence and Outcomes to Kaftrio in Cystic Fibrosis

NCT05519020 | Completed

• 1 other identifier: STH22060
• Study Type: observational
• Target: 1,385
• Locations: 1 country
• Sites: 1

Brief Summary

Cystic fibrosis (CF) is a life-limiting and life-long genetic condition which requires intensive preventative treatment to manage the symptoms and progression of disease. While preventative treatments target the effects of cystic fibrosis, precision medicines target the underlying dysfunction of the cystic fibrosis transmembrane regulator (CFTR) protein at a cell level. The first of these expensive precision medicines also known as modulator therapies, Ivacaftor, was shown to be highly effective in clinical trials with an increase of over 10% in lung function. Real-world studies showed an increase of only 6% and a return to baseline lung function by year five of treatment. Preventative therapies were continued during the Ivacaftor clinical trials whereas there is real world evidence of declining inhaled preventative therapy use following Ivacaftor initiation. This is a potential explanation for the efficacy-effectiveness gap. The first study in the National Efficacy Effectiveness Modulator Optimisation (NEEMO) programme is exploring this (REC ref: 21/HRA/4940, IRAS 301975). Ivacaftor/Tezacaftor/Elexacaftor is the most recent modulator available, commissioned in the UK (United Kingdom) in 2020, and suitable for around 90% of people with cystic fibrosis. It is not yet known if the efficacy effectiveness gap seen with Ivacaftor also exists for Ivacaftor/Tezacaftor/Elexacaftor. There is also uncertainty about the continued need for preventative inhaled therapy alongside the prescription of Ivacaftor/Tezacaftor/Elexacaftor. This second study in the NEEMO programme is a cohort, observational study and will explore adherence to inhaled preventative therapies in adults with cystic fibrosis before and after commencing Ivacaftor/Tezacaftor/Elexacaftor, and in those not prescribed Ivacaftor/Tezacaftor/Elexacaftor. It will also look at the relationship between adherence to preventative inhaled therapy and outcome for adults with CF taking Ivacaftor/Tezacaftor/Elexacaftor. The analysis will use routinely collected pseudo anonymised data from the CFHealthHub learning health system (CFHealthHub), alongside anonymised data from the CF registry and routinely collected clinical data.

Conditions

Cystic Fibrosis

Keywords

CFTR modulators; Kaftrio; Trikafta; Inhaled therapy; Adherence

Outcome Measures

Primary Outcomes (1)

• Change in Forced Expiratory Volume (FEV)

  Change from Baseline in Forced Expiratory Volume at 5 years

  5 years

Secondary Outcomes (4)

• Change in Body Mass Index (BMI)

  5 years

• Change in unadjusted adherence to preventative inhaled therapy

  5 years

• Change in effective adherence to preventative inhaled therapy

  5 years

• Change in adherence to Ivacaftor/Tezacaftor/Elexacaftor.

  5 years

Study Arms (2)

Ivacaftor/Tezacaftor/Elexacaftor eligible

People with cystic fibrosis who are eligible for and taking Ivacaftor/Tezacaftor/Elexacaftor

Ivacaftor/Tezacaftor/Elexacaftor ineligible

People with cystic fibrosis who are not taking Ivacaftor/Tezacaftor/Elexacaftor for any reason

Eligibility Criteria

• Age: 16 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Adults with cystic fibrosis, who attend one of the seventeen CF centres within, or previously participating in, the CFHealthHub learning health system (REC ref: 17/LO/0032, IRAS 216782) who are, or have ever been, prescribed Ivacaftor/Tezacaftor/Elexacaftor will be identified by their local centre. Data will also be analysed from these centres for people with CF who do not participate in CFHealthHub and those who have not been prescribed Ivacaftor/Tezacaftor/Elexacaftor but who consent to share their data with the CF Registry (REC ref: 07/Q0104/01, IRAS 35220).

You may qualify if:

• Diagnosis of cystic fibrosis
• Attend one of the seventeen specialist CF centres who are part of, or have previously been part of, the CFHealthHub learning health system
• Have given consent for routinely collected clinical data to be used as per the CFHealthHub trials within cohorts consent and/or the CF registry consent
• Aged 16 or over (attend an adult CF centre)

You may not qualify if:

• \. Has not given consent for routinely collected clinical data to be used as per the CFHealthHub trials within cohorts consent and/or the CF registry consent. This will be determined at a site level.

Sponsors & Collaborators

• Sheffield Teaching Hospitals NHS Foundation Trust: lead

Study Sites (1)

Sheffield Teaching Hospitals NHS Trust

Sheffield, South Yorkshire, S5 7AU, United Kingdom

Location

Related Links

• CFHealthHub website

MeSH Terms

Conditions

Cystic Fibrosis

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Study Officials

• Tracey Daniels

  York & Scarborough NHS Foundation Trust

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: OTHER
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 25, 2022
• First Posted: August 29, 2022
• Study Start: July 27, 2022
• Primary Completion: October 31, 2024
• Study Completion: October 31, 2024
• Last Updated: February 21, 2025

Record last verified: 2025-02

Locations

GB (1)