July 9, 2013 mcfadyena

Phase II/III Intrathecal Trial Update

Boston Red Sox GameThe Phase II/III is getting closer! For those families who have been waiting, it couldn’t come soon enough, I know. Here is an update of what I know:
Dose: 10mg every 4 weeks. Trey has been receiving 30mg, so this dose will be different from what Trey is currently receiving.
Criteria: An intracranial pressure of 30 or over will likely remain exclusionary. IQ must be greater than 55, possibly less than 85, the 55 is certain but the upper ceiling is not yet solidified.
Timeline: The Phase II/III trial will be 12 months long. This differs from the Phase I/II as it was 6 months long. This also means that the control (no drug) group will receive 12 months of no drug. I don’t know if there will be re-qualifications after the 12 month control is done, as was done in Phase I/II.
Device: The new device will likely be ready to implant in August on a boy in the Phase I/II trial.
Locations: Definitely Chapel Hill, Chicago, and Oakland. There may be other US locations. There will also be locations in the UK, Argentina, and Spain, possibly a few other Spanish speaking countries, to be determined.
Languages: English & Spanish.
Age: My understanding is that ages 3-18 will be included, with an addition. There will be a sub-study for boys less than 3 years of age whose mutation predicts them to have severe disease.
Natural History Study: The trial has begun a Natural History Study to follow boys with MPS II who may be eligible for the intrathecal trial. Boys who are not in the study will not be excluded from the trial, this study just allows the trial folk to follow the boys and pick them out if they might qualify.
Time to market: The trial is 12 months long, it is estimated it will take 12 months to fully enrol, that’s two years. Then with approval processes, it looks as though this drug is 3-4 years to market.
My word is not set in stone, I am not the boss, I am a mom who is sharing this info so that other moms and dads can do what they need to do in order to get their boys into this trial. If you want to chat, I am here. This is not an easy road and if I can walk side by side with you for a while to make it a little easier, don’t hesitate to ask. My heart is with all of you who are fighting to get this for your boys.


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