Phenylketonuria (PKU) Study for Patients Taking Palynziq
Exploring Innovative Treatments
PHASE 4 STUDY SEEKING PATIENTS WITH PKU TAKING PALYNZIQ (PEGVALIASE) AND EXPERIENCING HYPERSENSITIVITY REACTIONS
Study Drug:
Palynziq (pegvaliase)
Age Group:
Adults 18 years or older
Study Duration:
Up to 30 weeks, including screening, desensitization, and follow-up
Visits:
One full-day on-site visit, followed by a next-day follow-up on-site visit Additional remote visits through Week 24, scheduled every 2–4 weeks
Location:
Uncommon Cures, Chevy Chase, MD
Compensation
Those who qualify receive all study-related laboratory tests, medical exams, and travel at no cost.
Study Overview
Uncommon Cures is conducting a Phase 4 clinical trial for adults diagnosed with Phenylketonuria (PKU) who are currently taking Palynziq (pegvaliase) and experiencing hypersensitivity reactions. Sponsored by BioMarin Pharmaceutical Inc., this study evaluates whether a rapid drug desensitization (RDD) protocol can improve treatment tolerability and long-term success for patients.
The trial is conducted at our Chevy Chase, MD location, and all travel costs will be reimbursed. Participation is at no cost to the patient.
Eligibility Criteria:
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Adults 18 years or older
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Diagnosed with Phenylketonuria (PKU)
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Currently prescribed Palynziq (pegvaliase)
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Experiencing hypersensitivity reactions to Palynziq that led to dose reduction or interruption
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Able to undergo RDD within 6 weeks of a hypersensitivity reaction
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Additional criteria may apply
Study Process
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Screening Period: Initial assessment of eligibility
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Treatment Phase: One-day on-site rapid drug desensitization protocol, followed by next-day in-person visit
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Follow-Up Period: Remote visits continue through Week 24 every 2–4 weeks
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End of Study Visit at or near Week 30
Advance Rare Treatments.
Have a Question?
FAQ
We understand that participating in a clinical trial can raise a lot of questions. Here, you’ll find answers to some of the most common questions about our process, what to expect, and how we support our patients. If you don’t see the information you’re looking for, feel free to contact us directly for more details.
The study aims to determine whether Rapid Drug Desensitization (RDD) improves the tolerability and continuation of Palynziq treatment in patients who have experienced allergic reactions.
No. If eligible, you can continue with Palynziq treatment. The desensitization process is designed to help you stay on therapy more successfully.
No. All participants will receive the RDD protocol while continuing their commercial Palynziq therapy.
There is no cost to participate in this study. Travel expenses are fully reimbursed, and all study-related procedures are covered.
There is no cost to participate in this study. Travel expenses are fully reimbursed, and all study-related procedures are covered.
Absolutely! We encourage all potential participants to discuss the trial with their healthcare provider before enrolling to ensure it aligns with their ongoing medical care.
Participants may experience better tolerance to Palynziq and improved treatment adherence. Your involvement could help improve care for other PKU patients in the future.