Clinical Trial

Clinical Trial

  • Information Evenings


    CFI, in partnership with the Midlands Branch, held two information evenings for Parents & Carers in Portlaoise (October 21st) and Dublin (November 4th). The evenings primarily focused on new treatments in the pipelines for people with Cystic Fibrosis.

    Dr Ed McKone, CF Consultant from St Vincent's University Hospital, Dublin, was keynote speaker and shared his expert insight and knowledge regarding the treatment pipeline for people with CF in Ireland.  

    Click play on the videos below to view the presentations and discussions from the Portlaoise Information Evening, which was live streamed on the night. 

    Welcome Address by Midlands Branch


    Progress Update by Philip Watt, Chief Executive, CFI


    New Therapies in CF by Dr. Ed McKone, CF Consultant, St. Vincent's Hospital


    The Approval Process for New Therapies by Katie Murphy, Research & Development Officer, CFI


    Question & Answer Session


    More Information Evenings are planned for 2016, with details to be announced soon.

  • Extension of Orkambi to children aged 6-11

    The recent decision of the European Medicines Agency (EMA) to extend the CF drug “Orkambi” to children aged 6 to 11 years old, on the basis of clinical research emerging from two Phase III studies (NCT02514473 and NCT01897233), means that this ground breaking drug which had previously only been available to those aged 12 years and older with two copies of the F508del mutation, will now also be available to children aged 6-11 (subject to the advice of their Consultant).

    Orkambi treats the most common CF gene alteration/ mutation in Ireland (and the world) that is people who have two copies of the F508del alteration. Orkambi was approved for reimbursement by the Irish Government in April 2017. As part of that agreement, in a unique “pipeline deal”, it was agreed that future extensions of Orkambi or Kalydeco (including this EMA decision to extend Orkambi to children aged 6-11), would be made available to people with CF in Ireland.

    Commenting on the extension of Orkambi to children aged 6-11, the Minister for Health Simon Harris said:
    “It’s very positive news that Orkambi is now licenced and available for reimbursement in Ireland for CF patients aged 6 to 11.”


  • Further promising news from Vertex for those with F508del and one minimal function gene alteration.

     24 July 2017

    Further promising news from Vertex for those with F508del and one minimal function gene alteration.

    This news release covers the VX-152; VX-440 and the VX659 therapies presently undergoing clinical trials. These show very exciting outcomes for those with F508del and one minimal function gene alteration. Note: they still have to complete further phases of research before they go for approval to the European Medicines Agency (EMA) - likely timeframe for approval, if all continues to go well, is 2018/2019

    The results were from two phase 2, or midstage, clinical trials, and one phase 1 study evaluating three different triple-combination regimens. The studies tested three different experimental drugs, each on top of a regimen of two other drugs, called tezacaftor and ivacaftor.

    The primary goal of the study was improvement in FEV1, ‘forced expiratory volume’: This measures how much air someone can expel in one second.

    In the two phase 2 studies, one drug, called VX-152, showed a very exciting 9.7 percentage point average improvement in that measure, on top of the two-drug cocktail. Another, called VX-440, showed an even better 12 percentage point average improvement. A third, VX-659, in a phase 1 study, improved FEV1 by an average of 9.6 percentage points, also very encouraging.

    The next step is for Vertex to decide which regimen(s) to move into a late-stage clinical trial, expected to begin in the first half of 2018.

    ‘The results are a further important step for the treatment of cystic fibrosis but still at a relatively early stage’ said Philip Watt, CEO of CFI.

    Background Note:
    Vertex was the first company to develop a drug therapy targeting the root genetic cause of the disease; called Kalydeco, the treatment was approved by the Irish Government in 2013 and targets gene alterations accounting for about 4,500 cases of CF worldwide and 120 in Ireland. Ireland has the highest percentage of patients who benefitted from Kalydeco in the world. Around 11% of the CF population in Ireland have the G551D gene alteration and in the south west region, this rises to 25% of the total CF population. Kalydeco was expanded to 2-5 year olds and the R 117h gene alteration group as part of the April 2017 agreement in Ireland.  

    The company received approval for its second CF treatment, Orkambi, in Ireland in April 2017. A combination of Kalydeco (whose chemical name is ivacaftor and another drug, lumacaftor, potentially impacts on 25,000 patients worldwide and 550 patients in Ireland.


    ‘Minimal function alterations’ are gene changes that leave the CFTR protein minimally functional or unable to function at all.

  • Keeping up with Clinical Trials!

    There has been a very welcome surge in the number of clinical trials being conducted globally over the past decade – and Cystic Fibrosis is very much a part of this growth. Due to increasing awareness and interest in new therapies and clinical trials we have seen an increase in the number of queries around these studies.

    We are always delighted to provide support and information but another great way to keep up-to-date and to read about on-going clinical trials is to visit is a registry and results database of publicly and privately supported clinical studies of human participants conducted around the world.

    Red Blue Pill

    If you log on to the above website and search 'Cystic Fibrosis' – you will see detailed descriptions of status of trials, which phase they are in, where they are taking place, results (if any), etc. It is a great tool for interested people to stay informed and up-to-date.

  • Publication of the Orkambi Clinical Trial Data 

    On 17th of May 2015, the The New England Journal of Medicine (NEJM) published results on a new treatment combining ivacaftor (Kalydeco) with another compound called lumacaftor. This combination is now known as Orkambi and it targets the underlying cause of the CF in people with two copies of the F508del mutation, which represents 56.5% (654) people with CF in Ireland1.

    Orkambi clinical trial data

    Published results show a significant increase in FEV1 in patients of between 2.6 - 4% (p<0.001). Further analysis also revealed that the rate of pulmonary exacerbations was 30 to 39% lower in patients who were in receipt of the combination therapy compared to those who received placebo. The rate of events leading to hospitalisation or the use of intravenous antibiotics was also reduced for people being treated with the combination therapy.

    The publication of the study comes on the heels of a recommendation last week from an advisory committee of the U.S. Food and Drug Administration (FDA) that the FDA approve the drug for people with CF ages 12 and older with two copies of F508del. While the committee’s recommendation is an important part of the FDA’s review process, the decision is not binding. The FDA has said it will make its final decision by July 5, 2015.

    The drug is currently being considered for licencing by the European Medicines Agency, with a decision expected in the autumn. If there is a positive decision from the EMA then individual countries have to consider reimbursement of the drug, should it be approved by the EMA.

    CFI will continue to keep our members updated on the progress of this and other potential therapies developed for use in CF care.

    You can listen to This email address is being protected from spambots. You need JavaScript enabled to view it., Research and Development Officer from CFI, speak about these results on RTE Radio 1 news.

    1Cystic Fibrosis Registry of Ireland (2013) Annual Report

  • Vertex Announces Data from 12-Week Phase 2 Safety Study of VX-661 in Combination with Ivacaftor in People with Cystic Fibrosis Who Have Two Copies of the F508del Mutation

    The primary objective of this Phase IIb trial was to assess and ensure patient safety when taking the combination of VX-661 and Ivacaftor. The study evaluated two doses of VX-661 (100 mg once daily or 50 mg every 12 hours) in combination with Ivacaftor (Kalydeco) (150 mg every 12 hours). The study showed that the combination regimen was generally well tolerated, and all patients completed 12 weeks of treatment. The most common adverse events were pulmonary exacerbation, and cough.


    Secondary endpoints evaluated the effect of the combination on lung function. The patient group who received the 100 mg dose of VX-661 plus Kalydeco, patients reported a 4.4% improvement in lung function at 4 weeks, and a 3% improvement at 12 weeks. Consistent with prior Phase 2 studies that evaluated 4 weeks of treatment with VX-661 in combination with Ivacaftor, this study showed a rapid improvement in lung function within four weeks of treatment, and after patients completed treatment, lung function returned to baseline.

    These safety and efficacy data, together with other data from multiple previously completed Phase 2 studies of VX-661, support Vertex's ongoing Phase 3 program of VX-661 in combination with Ivacaftor. The Phase 3 program is evaluating VX-661 (100 mg once daily) in combination with Ivacaftor (150 mg every 12 hours) and consists of four Phase 3 studies, including a study in people with two copies of the F508del mutation that began enrolment in February. The other three studies will enroll people with CF who have one copy of the F508del mutation and a second mutation that is either a gating mutation, residual function mutation or a mutation that results in minimal CFTR function.

    To read the full release, please click here.


    Very encouraging Late Stage Study Results for forthcoming CF Triple-Combination drugs

    On 27 November Vertex pharmaceuticals released the initial Phase 3 clinical trial data for one of two next-generation, triple-combination drugs that are undergoing late stage clinical trials (Phase 3 trials). These are further part of the CFTR family of drugs that includes Klaydeco; Orkambi and most recently Symkevi.

    ‘The results are very encouraging’  said Philip Watt, CEO Cystic Fibrosis Ireland (CFI). 'They are still at clinical trial stage but they point to the increasingly strong possibility of further drugs coming down the line for those already on a CFTR drug and for some of those that are not. It continues to be a time of hope for people with CF. We are determined to try and ensure that no one gets left behind and that there will be a CFTR drug available to as many patients as possible in Ireland’

    In further, unconnected news - Novartis has sold TOBI Solution and Tobi Pod inhaler to US pharma firm, Mylan which also has a significant presence in Ireland (see information at end of this article).

    The results
    Positive results were announced from late-stage studies of a potential triple-combination CFTR drug for people with cystic fibrosis. Vertex Pharmaceuticals released Phase 3 clinical trial data for two studies of the next-generation modulator VX-659 in combination with ivacaftor and tezacaftor. 

    The first study tested this triple-combination drug on people with CF ages 12 years and older who have one copy of the most common gene alteration (mutation), F508del, and one minimal function mutation

    Results showed that those who received VX-659 combined with ivacaftor and tezacaftor had a 14 percent increase in lung function compared to participants taking a placebo. Researchers believe that anyone with at least one copy of the F508del mutation -- regardless of their second mutation -- could benefit from next-generation modulators.

    The second study included people with CF ages 12 years and older who have two copies of the F508del mutation. The trial compared the effectiveness of Symkevi (tezacaftor/ivacaftor) to VX-659 combined with tezacaftor and ivacaftor. Participants who were given VX-659 combined with tezacaftor and ivacaftor had a 10 percent improvement in lung function over those who were only given Symkevi® (tezacaftor/ivacaftor).

    Just as important are the wider impacts that these drugs will likely have including overall quality of life and reduction in exacerbations and weight gain. It’s too early to be definitive but the signs at this stage are very encouraging.

    Vertex will decide which of the two next-generation drugs they will submit to the U.S. Food and Drug Administration (FDA) for potential approval as a new drug. If approved, it is estimated that triple-combination CFTR modulators could potentially bring the benefits of therapies that treat the underlying cause of the disease to more than 90 percent of people with CF.

    Michael P. Boyle, M.D., senior vice president of therapeutics in the US Cystic Fibrosis Foundation stated:

    ‘It is an exciting time for the CF community, as we approach a milestone that seemed impossible even just a few years ago. Today's announcement represents an important step in our journey to developing treatments for the underlying cause of this disease for all people with cystic fibrosis. We are enthusiastic about the clinical benefit VX-659 demonstrated in these studies and look forward to seeing how these compare to the other next-generation modulator in clinical trials, VX-445.’

    CF Hospital Centres in Ireland have played a key role in supporting clinical trials for these medications. It is unclear yet when they will be submitted for approval to the FDA in the US and the EMA in Europe.

    There are presently 3 Vertex CFTR drugs approved for reimbursement in Ireland. These are ground-breaking drugs that treat the underlying cause of CF and include:

    Kalydeco (2013) primarily for those patients with the G551D gene alteration

    Orkambi (2017)for those patients with two copies of the F508del gene alteration

    Symkevi (2018) for those patients with two copies of the F508del gene alteration (alternate drug to Orkambi)

    In relation to Symkevi, CFI understands that a protocol is being developed. It is likely that those who are doing well on Orkambi (and the feedback to date is generally very positive) will remain on Orkambi.

    The impact of these drugs are closely monitored in Ireland. CFI will continue to provide impartial and informed information about these and future CF Drugs

    Further Drugs News: Novartis sells Tobramycin to Mylan
    In September 2018 Mylan, a Pennsylvania pharma company paid Novartis, the Swiss based pharm-company, 463 million dollars for its CF therapies including TOBI solution and TOBI pod-inhaler. This coincides with TOBI coming off patent. Nebulised Tobramycin was introduced in 1998 and has proven especially effective against Pseudomonos. Meanwhile Teva an international Pharma company has introduced a generic form of Nebulised Tobramycin.  These developments will likely result in significant price reductions in Nebulised Tobramycin to the HSE.