PKU patients and caregivers can participate in study for evaluating device linked to PEG-PAL

If you live in the San Francisco Bay-area and either have PKU or care for someone with PKU, there is an opportunity to participate in a paid usability study for a potential medication delivery device.

The device at the center of the usability study would used to administer PEG-PAL, an injectable enzyme substitution developed by BioMarin Pharmaceutical that has shown to lower phe levels in PKU mice. According to the National PKU Alliance, PEG-PAL recently completed Phase 2 FDA clinical studies and all patients enrolled in that phase had experienced some measurable decrease in blood phenylalanine levels. BioMarin is moving forward with the third and final phase that would ultimately bring PEG-PAL to a commercial status, which also explains the recent interest in figuring out just how patients will react to the process of injecting themselves with the drug.

Interface Analysis Associates, the California-based human behavior consultancy firm that has partnered with BioMarin for this study, will offer two 30-minute sessions in early September 2013 and will pay participants $150 to learn how the device works and perform mock injections. There will be no needle pricks and no actual medication administered. Participants will be asked to provide feedback on their experience and preferences.

Usability Study for PKU Patients and Caregivers

Click image to enlarge.

I was asked to participate in the study but because of the 2,500 miles between East Tennessee and the West Coast I had to respectfully decline. Nonetheless, it’s a great opportunity for others in the PKU community to directly impact the design and functionality of how this exciting new treatment will be delivered. It was also smart of BioMarin to initiate this usability study – not just from a bottom-line perspective but also because patients will have an investment in the product, the opportunity to suggest design improvements, and will likely become more satisfied with how it works.

If you’re interested in participating in the study, be sure to sign-up quickly. September is right around the corner!


P.S. If you do participate in the study, you’ll have to return to PKU Parlor and let us all know how it went! 🙂


Filed under PEG-PAL, Research

2 responses to “PKU patients and caregivers can participate in study for evaluating device linked to PEG-PAL

  1. JBH

    Hi Nicole-

    I am a month into the trial at Children’s in Boston. There are clinics all over the country participating and they pay transportation/hotel/gas for all participants (no matter the distance). There are 90ish participants enrolled in Phase III nationally. According to the physician I am being followed by, “peg pal works for everyone”. Basically, they have not found a PKU patient that has not responded positively. There are some side effects, such as local rash at injection site and supposedly bone pain (have not experienced the bone pain but I guess it subsides as they titrate the dose up). Rumor has it the docs can titrate your dose up to be able to metabolize whatever diet you would like to maintain. I have not noticed a difference because I have only been getting it once a week and bc of the study can’t change my diet unless I go hypo-phe (too low). It is really cool that there may be treatment!

  2. Very cool – thanks for sharing! I’ve heard great things about PEG-PAL too. Have you had any trouble self-injecting yourself with the medication?

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