AIM-IT: Phase III trial for Idursulfase IT

Posted Friday October 25, 2013 by Melissa

I’m sitting at the National MPS Society conference and learning more about the next step in the testing of Idursulfase IT – the Phase III trial. I know families are anxiously awaiting information so I wanted to push this out there as soon as possible. If you are looking for your child to enter the trial, my advice would be to contact the expert MPS II physician at the location nearest you. I understand that the cognitive testing requires the child/parent to be proficient in English or Spanish as well. The non-U.S. sites are expected to begin first. This is the other information I have gained.

AIM-IT: Administration of Idursulfase in MPS II – Intrathecal

  1. Controlled, randomized, two-arm, open label, assessor-blinded, multicenter phase III study in Idursulfase IT
    • 42 patients randomized 2:1 (28 treated, 14 untreated)
    • 12 months of 10mg doses via IDDD (LPs allowed if IDDD problems)
    • Primary endpoint: cognitive stabilization as measured by GCA of the DAS-II
    • Key secondary endpoint: functional stabilization as measured by the Adaptive Behavioral Composite (ABC) of the Vineland scales
  2. Main population: patients 3-18 years
    • A GCA of 55-85 or
    • A documented decline of greater than or equal to 10 points over the last 12 months and GCA greater than 85
  3. Substudy
    • Less than 3 years old
    • Will not count toward goal of 42 patients
    • Will use Bayley III assessment instead of DAS-II
    • Dose adjusted for lower age
  4. Expected locations
    • USA: UNC, Chicago, Oakland
    • Mexico: Mexico City, Buenos Aires
    • Spain: Madrid
    • UK: Manchester

I hope this information is helpful and my heart is with those seeking to enter the next phase of the trial and find stabilization and a longer life for their children. Clinical trials are not for the faint of heart and I’d be happy to discuss some of the logistical challenges we faced and how we made it all work. As always, I try to be an open book.

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3 Responses to “AIM-IT: Phase III trial for Idursulfase IT”

  1. Hi Melissa,
    Is there any info if trial recruits from oversees?

    • Cetin, I think that might be up to the individual investigators. My advice would be to identify and call them, maybe starting with Dr. Muenzer, to find out if they are willing to evaluate and admit from overseas. I know the language restrictions will eliminate some, but if those are met, maybe they would consider? Let me know what you find out!