{"id":1210,"date":"2018-11-22T14:33:52","date_gmt":"2018-11-22T14:33:52","guid":{"rendered":"http:\/\/blogs.cardiff.ac.uk\/centre-for-trials-research\/?p=1210"},"modified":"2022-04-13T13:59:10","modified_gmt":"2022-04-13T13:59:10","slug":"flight-study-milestones-researching-immune-thrombocytopenia-itp","status":"publish","type":"post","link":"https:\/\/blogs.cardiff.ac.uk\/centre-for-trials-research\/flight-study-milestones-researching-immune-thrombocytopenia-itp\/","title":{"rendered":"FLIGHT Study Milestones: Researching Immune Thrombocytopenia (ITP)"},"content":{"rendered":"

FLIGHT<\/a> is a\u00a0multicentre randomised trial of First Line treatment pathways for newly diagnosed Immune Thrombocytopenia: Standard steroid treatment versus combined steroid and mycophenolate.\u00a0 Funded by the National Institute for Health Research (NIHR)<\/a>.<\/p>\n

Study background<\/strong><\/h2>\n

Immune Thrombocytopenia (ITP) is an illness that causes bruising and bleeding owing to a low platelet count (blood cells essential for normal clotting).\u00a0 It is an auto-immune disease and relatively rare.\u00a0 It is also non-cancerous and the rare impact on survival has prevented it being a research funding priority.\u00a0 The Chief Investigator is an enthusiastic, dedicated haematologist and ITP clinical lead at University Hospital Bristol.\u00a0 She is determined to improve first line treatment for ITP patients, which has been unchallenged for decades.\u00a0 FLIGHT is the first independently-funded UK multicentre clinical trial for IPT and represents a big step forward for research in this disease area.<\/p>\n

The study came to the Centre for Trials Research<\/a> from University Hospital Bristol via one of their statisticians, who was involved in a previous study overseen at that time by Cardiff University Haematology Cancer Trials Unit (now amalgamated into the Centre for Trials Research). The official study start date was 1 May 2017, and we submitted the study to the regulatory authorities around the same time.<\/p>\n

Current treatments and side effects<\/strong><\/h2>\n

Current first line treatment for patients are high dose steroids and most patients can suffer side effects such as difficulty sleeping, weight gain, mood swings, high blood pressure and diabetes.\u00a0 Unfortunately the illness does not get better for some patients and the majority of others get ill again when the steroids are stopped.\u00a0 About 20% of patients stay well long term.\u00a0 Mycophenolate (MMF) is often used as the next stage (or second line) ITP treatment; it is licensed for this purpose and works well.\u00a0 However it can take up to two months to work during which time patients continue to be a risk of bleeding, bruising, fatigue and usually need more steroids which they dislike intensely.\u00a0 This has a big impact on patients\u2019 quality of life, as they are required to attend hospital frequently for blood tests and most take time off sick.\u00a0\u00a0<\/strong><\/p>\n

Trial outcome<\/strong><\/h2>\n

The trial objective is to find out whether more patients would feel better sooner if everyone takes MMF at diagnosis instead of what currently happens (and what MMF is licensed for) \u2013 waiting for the illness to return.<\/p>\n

We are therefore comparing the current way patients are treated (with steroids) to a new way with all patients given MMF right at the start of treatment.<\/p>\n

How the study is progressing<\/strong><\/h2>\n

In a nutshell – we have had to work around delays in site set up and recruitment!\u00a0 Although the study open date on 27 October 2017 was 6 weeks later than planned as the FLIGHT Data Manager was not in post until 9 October 2017.<\/p>\n

Despite NIHR research set-up and delivery timelines and our best efforts in jollying site along and maintaining contact with offers of assistance, we have experienced delays owing to lack of pharmacy capacity, local R&D and lack of administrator support within the clinical teams. \u00a0We needed a large number of sites; essentially recruiting small patient numbers.\u00a0 Thirty sites were approved in the initial application.\u00a0 A further eighteen sites were added in subsequent substantial amendments.<\/p>\n

We now have 41 sites open.\u00a0 A quick look at our spreadsheets show the following breakdown in site opening timeline over the last year:<\/p>\n

27-Oct-2017 to 31-Dec-2017: 14 sites<\/p>\n

01-Jan 2018 to 30-Jun-2018: 22 sites<\/p>\n

01-Jul-2018 to date: 5 sites<\/p>\n

We have 2 remaining sites to open; both were approved in the initial application.\u00a0 One of these sites received signed Site Agreement and R&D Capability and Capacity, but then informed us they concluded the recruitment window was too tight and could not proceed with the trial after all!\u00a0 Conversely we have small NHS centres added in substantial amendments opening within 2-3 months.<\/p>\n

Without recruiting the target cohort, there is no trial and for most studies this is the biggest challenge.\u00a0 We have had a combination of 25 trial committee meetings to date and have spent a large portion of the time discussing recruitment, as well as analysing recruitment graphs, screening and recruitment spreadsheets!\u00a0 Owing to the small patient numbers seen annually in NHS centres, we are continually in contact with our sites for one reason or another, together with periodic updates from the CI \u2013 just to remind them to keep a look out for that next patient.<\/p>\n

Recruitment<\/strong><\/h2>\n

In spite of logistical challenges, we have recruited 92 patients to date with actual <\/em>recruitment exceeding expected <\/em>recruitment in February and July 2018.\u00a0 As ITP is an auto-immune disease, it can be triggered by abundance of viruses in the general population and clinicians generally expect a pattern of peak referrals in January and September\/October.\u00a0The recruitment target is 120 patients.<\/p>\n

The original recruitment deadline was 1st<\/sup> September 2018.\u00a0 With some clever statistical calculations, this has been extended to 1st<\/sup> February 2019.<\/p>\n

Our milestones so far<\/strong><\/h2>\n