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    #1

    Help/advice needed

    Hi all

    First post here, I was diagnosed in January and after genetic testing it was identified as FUS MND.

    I was informed of a clinical trial taking place with the doctor optimistic of the results. Great news I thought.

    Then I didn’t meet the threshold in the breathing test, not too worry there was still the compassionate use pathway. Until I was told no, it’s against company policy to grant compassionate use while a trial is ongoing 🤮 this made me feel sick and angry.

    I guess my question is does anyone have any experience of this? Is there anything I can do? I know my consultant is still pushing for access but it has been almost 2 months and no changes as of yet.

    Thanks
    Ryan
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    #2
    RyanFisher93 A warm welcome to the forum, Ryan, sorry to meet you in such circumstances...

    Those Clinical Trial exclusion criteria can be so arbitrarily cruel, with no room for nuance - I'm sorry you were turned down for the trial.

    Presumably the rejection came from Ionis, not King's?

    If King's don't get enough participants, might the powers that be reconsider you?

    IDK what your SVC is but, if it's borderline, could you/your Neuro appeal?

    What about contacting Ionis directly?

    ​Diagnosed 03/2007. Sporadic Definite ALS/MND Spinal (hand) Onset.
    Significant bulbar impairment - No functional limbs - No speech - Feeding tube - Overnight NIV - Eye gaze user    .
    ​
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      #3
      Ryan.

      Hi there.

      I don't know who your consultant is or who you have as a contact in Kings. Have you spoken to the trial consultant?

      i have just checked the trial. It is 77 patients globally. It says King's is not now recruiting.

      77 is not many, and I suspect they may have a full trial.

      It can be frustrating, but the ethics of trials are complicated and although can seem illogical there are good reasons for the processes. Trials of new, potentially harmful drugs/treatments require serious monitoring and processing etc.

      Trial inclusion criteria are well thought out, and trials need to maximise contiguous data, and with drop out rates key. The trial could fail just because of lack of data if the inclusion criteria are not strict. Our desire to attend trials can backfire if drop out rates exceed the protocol statistical plan.

      But yes don't give up. I will ask someone who knows the trial for any advice.

      best wishes
      Lee
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        #4
        Hi both

        Yes the rejection came from Ionis. My results were between 30-35% so 15-20% below the threshold.

        The consultant is Keith Mayl (apologies probably incorrect terminology). I met with Ionis in July (virtually) that’s when they mentioned company policy.

        I was under the impression compassionate use what be separate from the trial anyway.

        Thanks for your responses.
        • 👍 2

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