Actively Recruiting
A Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AG-181 in Subjects With Phenylketonuria
Led by Agios Pharmaceuticals, Inc. · Updated on 2026-04-24
20
Participants Needed
1
Research Sites
90 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
The primary purpose of this study is to assess the safety and tolerability of AG-181 in subjects with Phenylketonuria (PKU).
CONDITIONS
Official Title
A Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AG-181 in Subjects With Phenylketonuria
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Diagnosis of PKU confirmed by documented presence of 2 mutant alleles in the PAH gene, with at least 1 being the R408W mutation, as verified by central genotyping.
- At least one plasma phenylalanine level above 600 micromoles per liter within 52 weeks before consent.
- Average plasma phenylalanine concentration above 600 micromoles per liter during screening with no individual measurement below 360 micromoles per liter (excluding samples after Day -20).
- Body mass index between 18.0 and 35.0 kg/m2 and weight of at least 50 kilograms during screening.
- Dietitian approval confirming ability to maintain consistent protein and phenylalanine intake as per the study diet manual.
You will not qualify if you...
- Prior treatment with AG-181.
- Use of P-glycoprotein inhibitors not stopped for at least 5 days or 5 half-lives before first dose.
- Use of strong inhibitors or inducers of CYP1A2, CYP2C8, or CYP3A not stopped for at least 28 days before first dose.
- Treatment with acid-reducing agents such as proton pump inhibitors or H2 blockers (short-acting agents like calcium carbonate allowed).
- Any preexisting condition affecting gastrointestinal anatomy or motility that may interfere with drug absorption or metabolism.
- Any preexisting condition affecting liver or kidney function that may interfere with drug absorption or metabolism.
- Inability to tolerate oral medication.
- Unwillingness to discontinue tetrahydrobiopterin supplementation, pegvaliase-pqpz, or other PKU therapies by Day -30 during screening.
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
University of Texas Southwestern Medical Center (UTSW)
Dallas, Texas, United States, 75390
Actively Recruiting
Research Team
A
Agios Medical Affairs
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
2
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