Clinical Trial: Newly Diagnosed Immune Thrombocytopenia Testing the Standard Steroid Treatment Against Combined Steroid & Mycophenolate

Study Status: Not yet recruiting
Recruit Status: Not yet recruiting
Study Type: Interventional

Official Title: A Multicentre Randomised Trial of First Line Treatment Pathways for Newly Diagnosed Immune Thrombocytopenia: Standard Steroid Treatment Versus Combined Steroid and Mycophe

Brief Summary:

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.


Detailed Summary:

This is a multicentre, randomised clinical trial of MMF with steroid as a first line treatment for participants with ITP against the standard care pathway involving steroids alone as first line treatment. This is not a blinded study, therefore patients and research team will know which treatment arm the participant will be randomised to.

There are no additional appointments or separate trial visits for this trial. Participants will be seen at their usual hospital appointments, which may take slightly longer than they do usually to gather all the information needed to carefully record information for the trial and to see how the participants are.

Participants will be screened and given up to one week of steroid prior to randomisation to enable sufficient time to read information, discuss and ask questions with informed consent in an appropriate setting.

Participants will then be randomised to one of either two treatment pathways below and be asked to complete quality of life questionnaires:

  1. Steroid +MMF pathway: 1mg/kg once daily prednisolone 4 days (maximum of 100mg), 40mg once daily 2 weeks, 20mg once daily 2 weeks, 10mg once daily 2 weeks, 5mg once daily 2 weeks then 5mg alternate days 2 weeks then stop, (Dexamethasone 20mg or 40mg daily for 4 days is an alternative option to prednisolone if deemed clinically more appropriate for individual circumstances).

    For the duration of steroid, patients will get a PPI (proton pump inhibitors) or H2 antagonist to protect against gastric bleeding and appropriate bone protection.

    From randomisation (alongside
    Sponsor: University Hospitals Bristol NHS Foundation Trust

    Current Primary Outcome: Time from randomisation to treatment failure. [ Time Frame: 1 year ]

    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).


    Original Primary Outcome: Same as current

    Current Secondary Outcome:

    • Remission rates [ Time Frame: Up to 12 months post randomisation ]
      Platelet count >30 x109/L and at least 2 fold increase from baseline. Complete >100x10 9/L, partial 30-100x10 9/L
    • Time to next therapy [ Time Frame: Up to 12 months post randomisation ]
      Clinically relapse (as platelet count <30x109/L) and need for further therapy


    Original Secondary Outcome:

    • Remission rates [ Time Frame: Up to 12 months post randomisation ]
      Platelet count >30 x109/L and at least 2 fold increase from baseline. Complete >100x10 9/L, partial 30-100x10 9/L
    • Time to next therapy [ Time Frame: Up to 12 months post randomisation ]
      Clinically relapse (as platelet count <30x109/L) and need for further therapy
    • Need for rescue therapies, splenectomy, time off work, hospital admissions, day unit & clinic attendances, fatigue, quality of life, medication side effects, toxicity/adverse events (including infection episodes) NHS costs, personal & social costs [ Time Frame: Up to 12 months post randomisation ]
      Number and detail of these events


    Information By: University Hospitals Bristol NHS Foundation Trust

    Dates:
    Date Received: May 2, 2017
    Date Started: September 1, 2017
    Date Completion: September 1, 2020
    Last Updated: May 16, 2017
    Last Verified: April 2017