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Trials Tracker

Welcome to the Trials Tracker, bringing together all the cystic fibrosis (CF) trials currently recruiting in the UK so that you can find clinical trials you can take part in both in your region and further afield. The Trials Tracker is a new resource that we’ll be developing over time based on your feedback. If you’ve used the Trials Tracker and want to share your opinion, please complete our online poll or get in touch at clinicaltrials@cysticfibrosis.org.uk.

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Clinical trials

1-5 of 54 results for all trials

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HIT-CF Organoid Study

New innovative and effective ‘mutation-specific’ drugs are available or in development for up to 90% of people with CF (ie. those with the most common 20-30 mutations). These are called ‘CFTR modulators.’ However, this leaves an important minority (~10%) of people with CF with no modulator in development as their mutations are very rare and only a small number of individuals have these specific mutations, making studying these patients in traditional trials very challenging. Therefore, the Human Individualized Treatment for CF (HIT-CF) project has been developed to address this unmet need, ie. to develop personalised treatments for this important group. HIT-CF is a Europe-wide, multi-centre project, which comprises two parts: This IRAS submission relates to the first part - the organoid study - in which patients with rare CFTR mutations will be recruited and intestinal organoids produced from rectal biopsies from each patient will be generated. Intestinal organoids, sometimes called ‘mini-intestines,’ are cell cultures made from stem cells so they contain the same mutations as the person from whom the biopsies are derived. Using this laboratory model, candidate modulator drugs can then be tested to assess for responsiveness (thus potentially predicting response in real life). In the second part of HIT-CF - the clinical trial - these modulators will then be tested in patients identified from the organoid study as likely responders, thus increasing the likelihood of showing efficacy. We plan to test drugs on organoids derived from 500 patients across Europe.

Read more Not Applicable
  • Trial Reference Number

    119685

  • Trial status

    Open to recruitment

  • Therapeutic category

    Restore CFTR Function

Study Designed to Assess the Safety, Tolerability and PK of PTI-808 in Adults With Cystic Fibrosis

In this study, the investigational CFTR modulator medicine PTI-808 will be given in combination with one or two other investigational modulator medicines, PTI-801 and PTI-428. PTI-428, PTI-801, and PTI-808 are considered cystic fibrosis transmembrane conductance regulator (CFTR) modulators being developed to treat the underlying cause of cystic fibrosis. PTI-428 is a CFTR amplifier that increases the amount of CFTR protein in the cell, PTI-801 is a CFTR corrector that improves the processing of CFTR protein in the cell, and PTI-808 is a CFTR potentiator to enhance the activity of CFTR at the cell surface. Modulators on their own have been shown to have limited activity, and it is predicted that combining modulators will be necessary to address CFTR defects. There is a screening visit up to 4 weeks before the active portion of the study, which lasts 4 weeks. In the active portion of the study, participants will take one of the following: PTI-808 by mouth in combination with PTI-801 and PTI-428, PTI-808 in combination with PTI-801 and placebo, or all placebos for 4 weeks. The study medication is taken once a day at breakfast. Two weeks after stopping the study medication, another visit will occur.

Read more Phase II
  • Trial Reference Number

    124674

  • Age

    18+

  • Trial status

    Open to recruitment

  • Therapeutic category

    Restore CFTR Function

A Phase 1/2a Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single and Multiple Doses of IONIS-ENaCRx in Healthy Volunteers and Patients With Cystic Fibrosis

This study will be conducted in 3 parts: a single ascending dose leading to a multiple ascending dose (MAD) in healthy volunteers, followed by a MAD in participants with cystic fibrosis. The study will enroll approximately 88 subjects. The study will consist of 4 single-dose randomized cohorts. Subjects enrolled will receive a single inhaled dose of the Study Drug (IONIS-ENaCRx or placebo) on Day 1.

Read more Phase I/II
  • Trial Reference Number

    122847

  • Age

    18+

  • Trial status

    Open to recruitment

  • Therapeutic category

    Mucociliary Clearance

Phase IIa study of PC945 in Aspergillosis in Cystic Fibrosis patients

This study tests the effects of an experimental drug PC945 in people with cystic fibrosis whose lungs are infected by the fungus Aspergillus fumigatus. PC945 may be useful in treating patients infected with Aspergillus fumigatus as, unlike the usual treatments, it is inhaled into the lung and has been designed to stay there and treat the infection. Participants will continue to be treated with their usual cystic fibrosis treatment and will also receive PC945. The amount of fungus in the patients' phlegm will be measured over the course of the study. The study will take place at multiple sites in UK and will include approximately 18 participants. The maximum study duration will be about 16 weeks.

Read more Phase II
  • Trial Reference Number

    108550

  • Age

    18+

  • Trial status

    Open to recruitment

  • Therapeutic category

    Anti-Infective

VX18-121-101 Safety and Efficacy of VX-121 combination therapy in CF

The purpose of this study is to evaluate the safety, tolerability and efficacy of VX-121 combination therapy in subjects with cystic fibrosis.

Phase II
  • Trial Reference Number

    113399

  • Age

    18+

  • Trial status

    Open to recruitment

  • Therapeutic category

    Restore CFTR Function

1-5 of 54 results for all trials