Immune Thrombocytopenia Clinical Trial
— FLIGHTOfficial title:
A Multicentre Randomised Trial of First Line Treatment Pathways for Newly Diagnosed Immune Thrombocytopenia: Standard Steroid Treatment Versus Combined Steroid and Mycophenolate
Verified date | October 2022 |
Source | University Hospitals Bristol and Weston NHS Foundation Trust |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
This is a study of two treatment pathways [Standard steroid treatment versus combined steroid and Mycophenolate (MMF)] for subjects with newly diagnosed Immune Thrombocytopenia (ITP). ITP is an illness that causes bruising and bleeding due to a low platelet count (blood cells essential for normal clotting). Patients are first given high dose steroids but most suffer side effects (e.g. difficulty sleeping, weight gain, moods swings, high blood pressure and diabetes). In addition, the majority of patients become ill again when the steroids are stopped - only about 20% stay well long term. ITP is relatively rare, non-cancerous in nature and the rare impact on survival of ITP have prevented it from being a priority for research funding, with first line treatment being unsatisfactory and unchallenged for decades. This underestimates the profound adverse impact an ITP diagnosis and its treatment has on individual patients, many of whom are young. MMF is often used as the next stage treatment for ITP and it works well. However, it can take up to 2 months to work during which patients continue to be at risk of bleeding, bruising, fatigue and usually need more steroids which they find intolerable. They are required to come to hospital for weekly blood tests and for many this impacts on work. We want to find out whether it would benefit more patients if everyone takes MMF at diagnosis instead of current practice (waiting for the illness to come back). We plan to test this by comparing the current way we treat patients to a new way with patients given MMF right at the start of their treatment. 120 patients from 20 different hospitals will be asked to take part and half will be randomly chosen for the new pathway.
Status | Completed |
Enrollment | 123 |
Est. completion date | March 5, 2020 |
Est. primary completion date | March 5, 2020 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 16 Years and older |
Eligibility | Inclusion Criteria: - Patients (males and females) >16 years old with a diagnosis of ITP, a pl count <30x109/L AND a clinical need for first line treatment. - Patients have provided written informed consent Exclusion Criteria: - The exclusion criteria include pregnancy and breastfeeding - Patients with HIV, Hepatitis B or C, or Common Variable immunodeficiency. - Women of child bearing potential require a pregnancy test result within 7 days prior to randomisation (as per 7.1 below) to rule out unintended pregnancy - Contraindications to MMF or steroid (see SPC, Appendix 2) including patients with hypersensitivity to mycophenolate mofetil, mycophenolic acid or to any of the excipients or active significant infection - Patients not capable of giving informed consent (e.g. due to incapacity) - Patients unwilling to follow contraceptive advice if allocated to MMF treatment arm. |
Country | Name | City | State |
---|---|---|---|
United Kingdom | University Hospital Bristol NHS Foundation Trust | Bristol |
Lead Sponsor | Collaborator |
---|---|
University Hospitals Bristol and Weston NHS Foundation Trust | Cardiff University |
United Kingdom,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Time from randomisation to treatment failure. | To include patients who are refractory (platelet count <30x109/L in spite of 2 weeks treatment in the steroid arm or platelet count <30x109/L in spite of 2 months treatment in the steroid +MMF arm) or who initially respond but then relapse (defined clinically as platelet count <30x109/L and need for further therapy). | 1 year | |
Secondary | Medication side effects, toxicity and other adverse events (including infection episodes) | Most participants experience side effects from the medication, data will be collected on treatment side effects and adverse events as assessed by CTCAE V4.0 | Up to 12 months post randomisation | |
Secondary | Bleeding events | To be analysed as number of bleeding events per patient recorded by 12 months. In addition we will collect site and type of bleeding, treatment required for bleeding, whether hospital admission was required, whether ITP rescue treatments were needed to be used in the calculating of costs for the economic evaluation. | up to 12 months post randomisation | |
Secondary | Remission rates | Platelet count >30 x109/L and at least 2 fold increase from baseline. Complete >100x10 9/L, partial 30-100x10 9/L | Up to 12 months post randomisation | |
Secondary | Time to relapse and time to next therapy | period of time between relapse and time of next therapy | Up to 12 months post randomisation | |
Secondary | Cumulative cortiocosteroid dose | Total steroid dose from randomisation | Up to 12 months post randomisation | |
Secondary | Need for rescue therapies | To be analysed as number patients who required rescue therapies between randomisation and 12 months post randomisation. We will also record the mean number of rescue therapies, why they were needed their type and cost for use in the economic evaluation. | up to 12 months post randomisation | |
Secondary | Need for splenectomy | Whether a participant has undergone splenectomy procedure | Up to 12 months post randomisation | |
Secondary | Socioeconomic costs | NHS, personal and social costs | Up to 12 months post randomisation | |
Secondary | Patient reported outcomes - Quality of Life | To be assessed using the utility score of the ICECAP (A) to calculate area under the curve using the trapezium method over a 12 month time frame from date of randomisation. | Up to 12 months post randomisation | |
Secondary | Patient reported outcomes - Fatigue | To be assessed using the utility score of the FACIT-F (Version 4) to calculate area under the curve over a 12 month time frame from date of randomisation. | Up to 12 months post randomisation | |
Secondary | Patient reported outcomes - Impact of bleeding | To be assessed using the utility score of the FACT-Th6 (Version 4) to calculate area under the curve over a 12 month time frame from date of randomisation. | Up to 12 months post randomisation | |
Secondary | Patient reported outcomes - Care Costs | To be assessed using the utility score of the Thrombocytopenia care costs V1 questionnaire to calculate area under the curve over a 12 month time frame from date of randomisation. | Up to 12 months post randomisation |
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