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Inhaled Therapy Adherence and Outcomes to Kaftrio in Cystic Fibrosis

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ClinicalTrials.gov Identifier: NCT05519020
Recruitment Status : Recruiting
First Posted : August 29, 2022
Last Update Posted : August 29, 2022
Sponsor:
Information provided by (Responsible Party):
Sheffield Teaching Hospitals NHS Foundation Trust

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.


Condition or disease
Cystic Fibrosis

Show Show detailed description

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Study Type : Observational
Estimated Enrollment : 1500 participants
Observational Model: Cohort
Time Perspective: Other
Official Title: Exploring Adherence to Preventative Inhaled Therapy and Outcomes for People With Cystic Fibrosis Who Take Ivacaftor/Tezacaftor/Elexacaftor.
Actual Study Start Date : July 27, 2022
Estimated Primary Completion Date : December 31, 2026
Estimated Study Completion Date : December 31, 2026

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Cystic Fibrosis
Drug Information available for: Ivacaftor

Group/Cohort
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



Primary Outcome Measures :
  1. Change in Forced Expiratory Volume (FEV) [ Time Frame: 5 years ]
    Change from Baseline in Forced Expiratory Volume at 5 years


Secondary Outcome Measures :
  1. Change in Body Mass Index (BMI) [ Time Frame: 5 years ]
    Change from Baseline in BMI at 5 years

  2. Change in unadjusted adherence to preventative inhaled therapy [ Time Frame: 5 years ]
    The percentage of preventative inhaled therapy taken compared with the prescription at 5 years

  3. Change in effective adherence to preventative inhaled therapy [ Time Frame: 5 years ]
    The percentage of preventative inhaled therapy compared with the ideal prescription at 5 years

  4. Change in adherence to Ivacaftor/Tezacaftor/Elexacaftor. [ Time Frame: 5 years ]
    Medicines possession ratio of Ivacaftor/Tezacaftor/Elexacaftor at 5 years



Information from the National Library of Medicine

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Ages Eligible for Study:   16 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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).
Criteria

Inclusion Criteria:

  1. Diagnosis of cystic fibrosis
  2. Attend one of the seventeen specialist CF centres who are part of, or have previously been part of, the CFHealthHub learning health system
  3. 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
  4. Aged 16 or over (attend an adult CF centre)

Exclusion Criteria:

1. 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.


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05519020


Contacts
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Contact: Tracey E Daniels, MSc 07866 206204 traceydaniels1@nhs.net
Contact: Sarah Birchall sarah.birchall4@nhs.net

Locations
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United Kingdom
Sheffield Teaching Hospitals NHS Trust Recruiting
Sheffield, South Yorkshire, United Kingdom, S5 7AU
Contact: Martin Wildman       martin.wildman3@nhs.net   
Sponsors and Collaborators
Sheffield Teaching Hospitals NHS Foundation Trust
Investigators
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Principal Investigator: Tracey Daniels York & Scarborough NHS Foundation Trust
Additional Information:
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Responsible Party: Sheffield Teaching Hospitals NHS Foundation Trust
ClinicalTrials.gov Identifier: NCT05519020    
Other Study ID Numbers: STH22060
First Posted: August 29, 2022    Key Record Dates
Last Update Posted: August 29, 2022
Last Verified: August 2022

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Sheffield Teaching Hospitals NHS Foundation Trust:
CFTR modulators
Kaftrio
Trikafta
Inhaled therapy
Adherence
Additional relevant MeSH terms:
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Cystic Fibrosis
Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases