A storm is coming!

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CF STORM is a ground-breaking trial to find out if people receiving Kaftrio can safely start to reduce the number of treatments they have to manage as part of their daily healthcare routine. Here we explain how the Trust has been involved from the outset and how we continue to support the development of CF STORM by involving people with cystic fibrosis (CF) and their families.

CF STORM - Clearing the air

The Cystic Fibrosis Trust was delighted to hear that the CF STORM trial has received £1.5m funding from the Government’s National Institute of Health Research (NIHR).

What’s CF STORM all about?

CF STORM, one of the first trials of its kind, will look at whether it is safe to stop so-called mucoactive nebulisers (DNase (dornase alfa) and hypertonic saline) once eligible people with CF are established on Kaftrio.

This could save people valuable time each day that is currently spent prepping, carrying out and cleaning nebulisers, which would have a positive impact on quality of life.

If people chose to give up their nebs without a trial like CF STORM, there would be no quantified evidence for clinicians to refer to and confidently advise their patients in the future.

Why is this important?

In 2015, the Cystic Fibrosis Trust supported a project with the James Lind Alliance to gather the views and opinions of people living with CF and their families to identify what aspects of life with CF they wished could be addressed by research. The responses were collated in 2017 and the number one priority was identified as ‘What are the effective ways of simplifying the treatment burden of people with cystic fibrosis?’

Prior to highly effective modulator therapies like Kaftrio, this was a particularly difficult research question to answer – how to safely reduce treatment burden without compromising stability of health. However, as more widespread access to Kaftrio started to look more likely, it brought about the opportunity to begin designing a trial.

Members of our Community Involvement group were involved again with a series of focus group discussions in December 2019 and January 2020. We asked which part of their treatment burden they would most like to stop, and would be confident to try, given the chance. Unsurprisingly, nebulisers came out as the most time-consuming and burdensome treatment, but people were less keen to stop nebulised antibiotics (such as Colistin) than mucolytic nebs like DNase and hypertonic saline.

The design focus for the trial was then established and named CF STORM (Streaming Treatments Or Reducing Medication). The name was developed by the Community Involvement team at the Trust and reflects the chance to think differently and be disruptive!

People with CF and the Trust have been involved in all aspects of designing and obtaining funding support for the CF STORM trial. After hearing about CF STORM, Candice who is a member of our Community Involvement group, has decided to join the trial steering committee. “CF STORM is something I would definitely want to get involved with, I really am curious to know whether Kaftrio could ever replace sputum thinning nebulisers!” she says.

How will the trial work?

The community has continued to be involved in the design and creation to ensure that participation in the trial is as easy as possible and not add burden into their routine, especially as its aim is to achieve the exact opposite.

The CF STORM team also designed the trial to take the current challenges of the coronavirus (COVID-19) pandemic into consideration so that it fits in with usual care (no extra hospital visits).

Clinical outcomes will be collected either remotely or in clinic, and recorded on the UK CF Registry. People with CF will not even have to attend hospital to join the trial. The Registry team has been heavily involved, building the new online system required to collect study information including consent to participate. The study data will be entered onto the Registry by clinical care teams, Trial Coordinators and participants from across the UK.

Who pays for a trial like this?

The funding for CF STORM has been approved by the UK Government through the National Institute of Health Research (NIHR). In order to apply for funding from NIHR, the CF STORM team had to prove the need for this research and provide a plan and design for how the trial would run. The need had already been demonstrated by the James Lind consultation and involvement of the CF community.

The preliminary work, to prepare for the trial and appoint a trial manager, has been funded by a grant from our Venture and Innovation Award (VIA) scheme to support the University of Liverpool and University College London. Without receiving this grant ahead of the NIHR award, the CF STORM team would not have been able to start the trial as quickly as they have managed. This has been a big partnership effort between the Trust, the Clinical Trials Unit (based in Liverpool) and academic partners across the whole UK.

Why does CF STORM only include people who are taking Kaftrio? What about Symkevi or Kalydeco?

Careful consideration was given to this question in the planning of CF STORM. It is crucial to the design of the trial that it does not try to compare too many different things at the same time. Kaftrio and Symkevi (and to a lesser extent Kalydeco) are not equally effective and so Kaftrio was chosen.

Dependent on its success, CF STORM could be the forerunner for similar trials to reduce treatment burden for others, either established on existing treatment or as they gain access to new modulator therapies that are not yet in use.

Is CF STORM taking priority over important trials for modulator therapies that may be suitable for people who can’t take Kaftrio?

CF STORM has been fortunate to receive funding now, during the pandemic, in recognition that it is a priority. There are concerns that people may be stopping treatments (such as nebs) without knowing what the consequences are for their long-term health.

CF STORM doesn’t take priority or resource away from CF trials developing new treatments or expanding access to Kaftrio.

Young white woman in jacket and wearing a lanyard

How can you find out more and get involved?

Further support for CF STORM has come from the Cystic Fibrosis Trust’s Clinical Trials Accelerator Platform and this continued support will be key in recruiting the 764 people needed to complete the trial.

The trial will open in spring 2021 and be run from many paediatric and adult CF centres across the UK. The intention is for it to be inclusive and accessible for everyone aged 12 and above who is taking Kaftrio and one or both mucolytic nebulisers (DNase and/or hypertonic saline). Further information about the trial can be found on our Trials Tracker, with the option to get in touch with the CF STORM team directly. Alternatively, you can speak to your CF care team about taking part.

Professor Kevin Southern

The Chief Investigators are Professor Kevin Southern (University of Liverpool) and Dr Gwyneth Davies (University College London), with the trial management team based at the Liverpool Clinical Trials Centre. The involvement of people living with CF will continue throughout the lifetime of the trial, with representation on the Trial Management Group and Trial Steering Committee.

To follow the progress, check out CF STORM on the Trials Tracker. If you have any questions about the trial, please contact clinicaltrials@cysticfibrosis.org.uk.

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