Loading clinical trials...

NOT_YET_RECRUITINGPHASE1

A Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AG-181 in Subjects With Phenylketonuria

A Phase 1b, Open-label, Multicenter, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Study of AG-181 in Subjects With Phenylketonuria

NCT07241234•Agios Pharmaceuticals, Inc.

View on ClinicalTrials.gov

Summary

The primary purpose of this study is to assess the safety and tolerability of AG-181 in subjects with Phenylketonuria (PKU).

Eligibility

Age

18 - 69 years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•Diagnosis of PKU, defined as documented presence of 2 mutant alleles in the phenylalanine hydroxylase (PAH) gene, of which at least 1 is the R408W mutation, as determined during Screening per the genotyping performed by the study central genotyping laboratory.
•At least 1 plasma Phe concentration greater than (\>) 600 micromoles per liter (μmol/L) in the 52 weeks before providing informed consent.
•Average concentration of plasma Phe \> 600 μmol/L in Phe samples taken during Screening, with no individual assessment below 360 μmol/L. Any Phe samples taken after Day -20 will not be included.
•Body mass index (BMI) greater than or equal to (≥) 18.0 kilograms per meter square (kg/m\^2) to lesser than or equal to (≤) 35.0 kg/m\^2 and weight ≥ 50 kilograms (kg) at any time during the Screening Period.
•Documented approval from a dietitian confirming that the subject can maintain their diet consistent in protein and Phe intake throughout the study as outlined in the Diet Manual.

Exclusion Criteria

•Prior exposure to AG-181.
•Receiving inhibitors of P-glycoprotein (P-gp) that have not been stopped for ≥ 5 days or a timeframe equivalent to 5 half-lives (whichever is longer) before administration of the first dose of study drug.
•Receiving products that are strong inhibitors or strong inducers of cytochrome P450 CYP1A2, CYP2C8, or CYP3A that have not been stopped for ≥ 28 days before administration of the first dose of study drug.
•Receiving treatment with an acid-reducing agent, including but not limited to proton pump inhibitors and H2 blockers. Short-acting acid-reducing agents such as calcium carbonate are permitted.
•Any preexisting condition that could (in the opinion of the Investigator) interfere with gastrointestinal anatomy or motility that may disrupt the absorption, metabolism, and/or excretion of the study drug.
•Any preexisting condition that could (in the opinion of the Investigator) interfere with hepatic or renal function that may disrupt the absorption, metabolism, and/or excretion of the study drug.
•Inability to tolerate oral medication.
•Unwillingness to washout from tetrahydrobiopterin (BH4) supplementation (eg, sapropterin dihydrochloride, Kuvan), pegvaliase-pqpz (Palynziq), or any other PKU therapy by Day -30 during Screening.

Key Dates

Start Date

March 1, 2026

Primary Completion Date

January 17, 2028

Completion Date

January 17, 2028

Last Updated

March 2, 2026

Enrollment

ESTIMATED participants

Conditions

Phenylketonuria

Interventions

AG-181

DRUG

Contacts

Agios Medical Affairs

CONTACT

833-228-8474 medinfo@agios.com

Development of Novel Psychological Assessment Tools and Anxiety Intervention for Phenylketonuria

NCT06560736

Phenylketonurias

View study

RECRUITINGPHASE1/PHASE2

AAV Gene Therapy Clinical Study in Adult Classic PKU (PHEdom)

NCT06332807

Phenylketonurias

View study

RECRUITINGPHASE4

Rapid Drug Desensitization Study in Adults Experiencing Hypersensitivity Reactions to Palynziq

NCT06780332

Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: March 2, 2026Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

View ClinicalTrials.gov Terms and Conditions

A Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AG-181 in Subjects With Phenylketonuria

Inclusion Criteria

Exclusion Criteria

Development of Novel Psychological Assessment Tools and Anxiety Intervention for Phenylketonuria

AAV Gene Therapy Clinical Study in Adult Classic PKU (PHEdom)

Rapid Drug Desensitization Study in Adults Experiencing Hypersensitivity Reactions to Palynziq

Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria

AAV Gene Therapy Study for Subjects with PKU

Evaluation of the Express Plus Range