Actively Recruiting
A Phase 1b, Open-label, Multicenter Study Evaluating Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AG-181 in Adults With Phenylketonuria
Led by Agios Pharmaceuticals, Inc. · Updated on 2026-05-29
20
Participants Needed
2
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the safety and tolerability of AG-181, a drug being studied in people with Phenylketonuria (PKU), a genetic condition characterized by a specific mutation in the phenylalanine hydroxylase gene. This Phase 1b, open-label study is sponsored by Agios Pharmaceuticals, Inc. and aims to understand the drug's effects and behavior in the body in subjects aged 18 to 69 who have elevated phenylalanine levels. Participants will receive AG-181 tablets daily from Day 1 to Day 28 during the treatment period. The study includes two cohorts, both receiving the investigational drug without randomization or placebo control. The trial monitors various pharmacokinetic parameters, such as plasma concentration, peak concentration time, and drug clearance, alongside changes in phenylalanine levels. During the study, participants will be closely observed for any adverse events up to Day 42 to assess safety. Blood samples will be taken to measure drug levels and phenylalanine concentration, with dietary consistency maintained throughout the trial. The total participation time includes treatment and follow-up periods to ensure thorough monitoring of safety and drug effects.
CONDITIONS
Brief 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 presence of 2 mutant alleles in the PAH gene, including at least 1 R408W mutation
- At least one plasma phenylalanine concentration greater than 600 micromoles per liter within the past 52 weeks
- Average plasma phenylalanine concentration above 600 micromoles per liter during screening, with no measurements below 360 micromoles per liter
- Body mass index between 18.0 and 35.0 kg/m² and weight of at least 50 kilograms during screening
- Approval from a dietitian confirming ability to maintain consistent protein and phenylalanine intake throughout the study as per the Diet Manual
You will not qualify if you...
- Previous exposure to AG-181
- Use of P-glycoprotein inhibitors not stopped at least 5 days before first dose
- Use of strong inhibitors or inducers of CYP1A2, CYP2C8, or CYP3A not stopped at least 28 days before first dose
- Current treatment with acid-reducing agents except short-acting agents like calcium carbonate
- Any condition interfering with gastrointestinal anatomy or motility affecting drug absorption or metabolism
- Any condition interfering with liver or kidney function affecting drug processing
- Inability to tolerate oral medication
- Unwillingness to stop other PKU treatments like BH4 supplementation or pegvaliase by 30 days before screening
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - 28 days
Participants receive AG-181 tablets daily to evaluate safety, tolerability, pharmacokinetics, and pharmacodynamics.
Daily dosing with multiple visits during treatment
Duration - Up to 14 days after treatment
Participants are monitored for safety and adverse events after treatment completion.
1 to 2 visits for safety assessment
Trial Site Locations
Total: 2 locations
1
University of Texas Southwestern Medical Center (UTSW)
Dallas, Texas, United States, 75390
Actively Recruiting
2
Centrum Wsparcia Badań Klinicznych Pomorskiego Uniwersytetu Medycznego w Szczecinie
Szczecin, West Pomeranian Voivodeship, Poland, 71-252
Actively Recruiting
Research Team
A
Agios Medical Affairs
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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