Join our online Q&A on exon skipping

Viewing 20 posts - 21 through 40 (of 54 total)
  • #128669
    Reply To: Join our online Q&A on exon skipping

    As for the family relocating to Europe. I believe Sarepta has a planned phase III trial for exon 53 in several EU countries but more information should come from them. Biomarin was also recruiting into their phase I/II studies, so you should check which are the trial sites for Biomarin in Europe

    Professor Francesco Muntoni, Director, Dubowitz Neuromuscular Centre, Great Ormond Street Hospital

    Professor Francesco Muntoni
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    #128670
    Reply To: Join our online Q&A on exon skipping

    Hi everyone!

    A question from Rachel Halpin in Liverpool whose son, Harry, has Duchenne muscular dystrophy:

    When will clinical trials take place for exon 44?

    Dr Jenny Sharpe, Research Communications Manager at Muscular Dystrophy UK

    Jenny Sharpe
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    #128671
    Reply To: Join our online Q&A on exon skipping

    Thanks so much for the doing this Q&A session! I am Leslie Guzmán, the mother of Diego Ramirez with Duchenne, he´s 11 years old and he’s walking. He has deletion 48-52, so he needs Exon 53. My question is – my husband is getting transferred with P&G to Europe. We can pick from Switzerland or UK. Could he join the trial under way that you are running?

    Leslie

    We currently have plans to initiate the exon 45/53 (ESSENCE) Trial in UK in the near future. Currently no study sites are planned for Switzerland.

    Dr Ed Kaye, CEO, Sarepta Therapeutics

    Dr Ed Kaye
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    #128672
    Reply To: Join our online Q&A on exon skipping

    Thank you for your answer dr. Muntoni!

    Ivana Bilic
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    #128674
    Reply To: Join our online Q&A on exon skipping

    Hi everyone!

    A question from Rachel Halpin in Liverpool whose son, Harry, has Duchenne muscular dystrophy:

    When will clinical trials take place for exon 44?

    Exon 44 is one of our future exons that we are working on; however, we have not yet determined the timeline for exon 44 clinical trials.

    Dr Ed Kaye, CEO, Sarepta Therapeutics

    Dr Ed Kaye
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    #128675
    Reply To: Join our online Q&A on exon skipping

    The FDA were critical of the methods used to quantify mRNA and dystrophin protein. There are ethical issues with multiple biopsies. Should this still be a primary measure for future trials?

    NickCatlin
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    #128676
    Reply To: Join our online Q&A on exon skipping

    Alison and Ronald Barr from the Isle of Man, have a son, James, with DMD who has a deletion amenable to exon 51. James was recently part of a clinical trial at GOSH, and his parents would like to ask Sarepta and Professor Muntoni:

    Throughout there are rigorous protocols in place while participating on a clinical trial, at all stages. It appears to be an inordinate wait for decisions to be made by the FDA or the EMA. Why ? How can we speed this process up?

    Muscular Dystrophy UK staff member

    Alexa Follen
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    #128677
    Reply To: Join our online Q&A on exon skipping

    Thanks for your response Dr. Muntoni and Dr. Kaye. So my next question is:

    1. Do the FDA approvals in US affect the plan for trials in Europe?, also.

    2. Has the Deadline passed for the recruitment that you were doing in March?

    3. As for the future plans for new trials in Europe. Are we talking weeks, months or years?. Any estimate.

    4. Can we participate in UK trial if living in Switzerland?

    Thanks again,

    Leslie

    Twinless
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    #128678
    Reply To: Join our online Q&A on exon skipping

    Hello,

    My son William was recently diagnosed with Duchenne Muscular Dystrophy. William has exon deletion 45-52. I am looking into all research which could possibly help with his condition. I am aware this is focused on exon 51. Could you inform me whether this treatment is something that could help William’s deletion in future or does he have too many exons deleted?

    Many thanks,
    Caroline Calvert

    Based on the 45-52 deletion, your son would be amenable to exon 53 skipping. We recommend that you discuss this with your physician to discuss potential treatment options for your son. We are planning exon 45 and exon 53 trials in the US and EU later this year.

    Dr Ed Kaye, CEO, Sarepta Therapeutics

    Dr Ed Kaye
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    #128679
    Reply To: Join our online Q&A on exon skipping

    The FDA were critical of methods used for biomarkers and clinical outcomes. How will future trials address these issues? Please see my previous post

    NickCatlin
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    #128680
    Reply To: Join our online Q&A on exon skipping

    From the message from Nick who raises a number of important poitns

    Why are measures against placebo not showing an effect even after 48 weeks?
    THIS IS NOT SO SURPRISING AS DYSTROPHIN RESTORATION PER SE DOES NOT MAKE CHILDREN MUCH STRONGER BUT ONLY PROTECTS MUSCLE FROM FURTHER MUSCLE DEGENERATION. AS YOU SAID BEFORE, STEROIDS AND OTHER TREATEMENTS HAVE HELPED TO STABILISE DISEASE PROGRESSION TO SOME EXTENT. SO IN A RELATIVELY SHORT PERIOD OF TIME, 48 WEEKS, IT CAN BE CHALLENGING TO IDENTIFY A CLEAR DRUG EFFECT

    What is the agreed ethical time limit for continuing the use of placebos in Duchenne trials?
    THIS IS ALSO A VERY GOOD QUESTION. THE LONGEST STUDIES WITH A PLACEBO ARM AND HOWEVER A CROSSOVER, WITH ALL PATIENTS RECEIVING AN EFFECTIVE THERAPY AT SOME POINT, IS A PFIZER STUDY ON A COMPLETELY DIFFERENT MOLECULE. THIS STUDY HAS A 2 YEARS PERIOD OIN WHICH SOME CHILDREN WILL RECEIVE PLACEBO. THIS WOULD NOT BE APPLICABLE
    STUDY DESIGN FOR DMD BOYS. SO THE QUESTION IS TO THE ADVOCACY GROUPS AND PARENTS, WOULD THEY ACCEPT A PLACEBO CONTROLLED STUDY OF 2 YEARS? OF 3 YEARS? OR LONGER?
    How will trial design recognise that Duchenne patients now take a range of drugs and use physical management interventions that could also influence clinical outcomes?
    BY HAVING LONGER STUDIES

    Are the 6MWT and NSAA valid and reliable measures of clinical benefit?
    THEY HAVE BEEN USED BEFORE. THE PTC STUDY DEOMONSTRATED BENEFIT IN THE SUBSET OF BOYS WITH 300-400 METRES IN BOTH 6MWT AND NORTH STAR. AND SIMILAR DATA WERE PRESENTED BY SRAPTA AT FDA IF MY MEMORY IS CORRECT

    Professor Francesco Muntoni, Director, Dubowitz Neuromuscular Centre, Great Ormond Street Hospital

    Professor Francesco Muntoni
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    #128681
    Reply To: Join our online Q&A on exon skipping

    Hi Professor Muntoni, I saw your reply to Ms Calvert, our 5 year son has the same deletion requiring exon skipping 53. Would it be possible for him to be included in the Sarepta trials when they become available if we live in the UK?

    Kind Regards

    Emma Gauder
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    #128682
    Reply To: Join our online Q&A on exon skipping

    Two questions for Sarepta:

    1) If the FDA refuse approval of eteplirsen, what impact will this have on follow on exon skipping drugs?

    2) If it is approved by the FDA, how likely is it that the closest follow on exon skipping drugs for 45 & 53 will move through the approval pathway at a faster rate than eteplirsen?

    Dr Jenny Sharpe, Research Communications Manager at Muscular Dystrophy UK

    Jenny Sharpe
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    #128683
    Reply To: Join our online Q&A on exon skipping

    Thanks for your response Dr. Muntoni and Dr. Kaye. So my next question is:

    1. Do the FDA approvals in US affect the plan for trials in Europe?, also. We are discussing our options for filing in Europe based on multiple scenarios in regards to US.

    2. Has the Deadline passed for the recruitment that you were doing in March? Please clarify the question.

    3. As for the future plans for new trials in Europe. Are we talking weeks, months or years?. Any estimate. We plan to initiate in the coming months for exons 45 and 53.

    4. Can we participate in UK trial if living in Switzerland? This may depend on local regulations which we do not have information. We will investigate further and respond by email if you send question to: CDiJohnson@sarepta.com.

    Thanks again,

    Leslie

    Dr Ed Kaye, CEO, Sarepta Therapeutics

    Dr Ed Kaye
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    #128684
    Reply To: Join our online Q&A on exon skipping

    In England, the Government recently introduced the Early Access to Medicines Scheme which companies can apply for to deliver their drug at an earlier stage of the process, pre EMA MAA. Is this something that Sarepta may look to explore?

    Muscular Dystrophy UK staff member

    Alexa Follen
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    #128685
    Reply To: Join our online Q&A on exon skipping

    Two questions for Sarepta:

    1) If the FDA refuse approval of eteplirsen, what impact will this have on follow on exon skipping drugs? Depending on the outcome of FDA decision, the Sarepta Management team will evaluate the impact on follow-on exon skipping clinical development. At the moment, we are moving ahead, as planned.

    2) If it is approved by the FDA, how likely is it that the closest follow on exon skipping drugs for 45 & 53 will move through the approval pathway at a faster rate than eteplirsen? Since this is a confirmatory trial requiring a double-blind, placebo control we do not anticipate the approval pathway will be faster. However, we would anticipate that the follow-on exon drugs may have a shorter regulatory review time.

    Dr Ed Kaye, CEO, Sarepta Therapeutics

    Dr Ed Kaye
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    #128686
    Reply To: Join our online Q&A on exon skipping

    Dystrophin as a biomarker in future clinical trials.
    We apprectiate that muscle biopsies are invasive and , while we work hard to minimise risks, there is always a small risk associated with the procedures. When developing a new drug however as exon 53 in SKIP NMD, however we felt essential to assess if the drug (after a period of 48 weeks, so when the children in the eteplirsen study all had dystrophin expression) is working in the way we anticipate it to work. It might not be necessary to do muscle biopsies in each and every study, but at this stage of development this is still providing the investigators and the company essential information. We and Sarepta have taken a very great care in esnuring that problems encountered in rpevious trials with loss of samples and so on, do not occurr in current and future studies

    Professor Francesco Muntoni, Director, Dubowitz Neuromuscular Centre, Great Ormond Street Hospital

    Professor Francesco Muntoni
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    #128687
    Reply To: Join our online Q&A on exon skipping

    In England, the Government recently introduced the Early Access to Medicines Scheme which companies can apply for to deliver their drug at an earlier stage of the process, pre EMA MAA. Is this something that Sarepta may look to explore?

    We are exploring early access to medicine programs in a variety of countries in Europe, including UK.

    Dr Ed Kaye, CEO, Sarepta Therapeutics

    Dr Ed Kaye
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    #128688
    Reply To: Join our online Q&A on exon skipping

    Another question from one of our supporters whose son has Duchenne muscular dystrophy:

    I’ve read that participation on a trial for SMT C1100 is an exclusionary criterion for exon skipping trials – is this for a specific period of time or is a child to be excluded if they have ever taken part in a SMT C1100 trial and if so, why?

    Dr Jenny Sharpe, Research Communications Manager at Muscular Dystrophy UK

    Jenny Sharpe
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    #128689
    Reply To: Join our online Q&A on exon skipping

    Hi
    I am always careful in promising things of which I have no contrtol! (ie.. at the moment this other trial still has to be set up, although discussion i believe has started). There will be multiple sites in UK which will recruit children in the exon 53 study run by Sarepta, London most likely, but other sites as well, so you should follow both the Sarepta webpages and contact the name provided by Dr Kaye; and also our study coordinator in the second part of this year (her name is in the webpages of our centre)

    Professor Francesco Muntoni, Director, Dubowitz Neuromuscular Centre, Great Ormond Street Hospital

    Professor Francesco Muntoni
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