Recruiting PKU Studies
Thank you for your interest in learning about ongoing PKU studies!
To support PKU research, NPKUA helps to connect community members who are interested in participating in research with active PKU studies through the PKU Patient Registry. Featured studies need to undergo a rigorous approval process before sharing with the PKU community. If you would like to receive email notifications for recruiting studies, you can do so through the PKU Patient Registry. If you have any questions, please email [email protected].
Please see the list of approved PKU studies that are actively recruiting:
Phase 4 Study Seeking Patients with PKU Taking Palynziq (Pegvaliase) and Experiencing Hypersensitivity Reactions
Study Contact(s)
Eligibility Criteria
- Adults 18 years or older with a confirmed diagnosis of PKU
- Currently prescribed commercial Palynziq treatment
- Experiencing hypersensitivity reactions (allergic reactions such as joint pains, injection site redness or swelling, swelling of the eyes, lips, tongue or throat, rashes, hives or anaphylaxis) to Palynziq leading to interruption or reduction of dose or dosing frequency or inability to advance dose or dosing frequency
- Able to undergo rapid drug desensitization (RDD) within 6 weeks of a hypersensitivity reaction
- Other criteria apply (see flyer for more information)
PHEdom (NGGT002-P-2301) – AAV Gene Therapy for PKU
Study Contact(s)
Eligibility Criteria
- Diagnosis of classic PKU due to PAH mutation
- Individuals aged 18-55 years
- At least 2 documented Phe levels > 600 umol/L
- Not on any PKU medications at least 28 days prior to participation
