A clinical trial for ‘left out’ cystic fibrosis patients

3 June 2024

An exciting clinical trial at Royal Brompton and Harefield hospitals has recruited its first patient to a study which aims to find a treatment for the subset of patients who do not benefit from the recent treatment advancements in cystic fibrosis (CF).

Led by Professor Jane Davies, respiratory consultant, the early phase trial will evaluate how safe, tolerable, and effective an investigational inhaled drug is in people with CF.

Early phase trials can be notoriously difficult as they involve investigational drugs that are being tested in people with disease for the first time, and therefore require the cooperation of a whole host of staff across different departments to make it work.


Modulator therapies

CF transmembrane conductance regulator (CFTR) modulator therapies are a class of drugs that are designed to correct the defective CFTR protein found in patients with CF.

These modulator therapies have revolutionised the lives of people living with CF, with people living longer and having healthier lives.

However, CF is caused by a large number of genetic variants and so far, the modulators that have been developed do not work on every type of CF disease.

Approximately 5,000 people with CF are not suitable for CFTR modulator therapy, which is where this new trial comes in, to develop a drug which could potentially provide an alternative for these patients.


The clinical trial

The first patient for the study was recruited in early March 2024 and involved an extensive and meticulous series of visits and tests over a 28 day period.

The early days of the clinical trial involved the patient staying on the hospital ward overnight so their response to the investigational agent could be very closely monitored. Unlike the modulator therapies, which are taken as a tablet, this investigational treatment is administered through inhalation.

The patient then underwent a series of tests including lung function, blood tests, chest CT and an ECG.

Yasmine Needham, a research practitioner coordinating the study, explained why the patient was required to have an initial stay in the hospital:

“This is the first time the investigational drug is being tested in people, so we don’t know how it’ll affect the body. Because of this, it’s key to have the participant stay in the hospital. It lets us watch closely for any reactions or symptoms and ensures they can quickly get any help they need from our team.”

The series of visits involved a team of staff including doctors, pharmacists to dispense the investigational drug, nurses to undertake tests, administrators to coordinate the visits and the bronchoscopy team to take lung samples to see how the investigational drug affects the airways. Many of these staff members had to be on deck and available to support the study even over evenings and weekends.

Professor Davies remarked on the hard work undertaking the trial and said:

“We’ve been heavily involved in CF clinical trials for over 20 years, and thanks to our multidisciplinary team members and their incredible commitment to improving the lives of people living with CF, are one of the most active trials sites across the Europe Network. For this particular trial, watching the way the team pulled together to tackle a really new challenge was very impressive.

“It would be remiss of me though not to also thank the people with CF who volunteer for these trials; we really couldn’t do any of it without you!

Professor Davies went on to explain the importance of these early phase studies in providing potential solutions for patients who do not otherwise benefit from currently available treatments aimed at the underlying cause of the disease. She said:

“CFTR modulators have had even greater clinical benefits than we’d ever expected, but the gap in health and wellbeing between patients with CF receiving them and the ~5,000 people who can’t is growing ever larger. Globally, this group is recognised as the major unmet need in CF. The current trial is one in a series of exciting new ‘genetic therapies’ studies that the team will be undertaking, so we anticipate a very busy year or two.”

The next step for the study team is to recruit more patients over the next year. If the data looks promising the study may enter the next phase of development which could involve a larger number of patients.  


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