Pittsburgh, PANCT07241234Now EnrollingIRB Ready

Phenylketonuria Clinical Trial in Pittsburgh, PA

Access cutting-edge phenylketonuria treatment through this clinical trial at a research site in Pittsburgh. Study-provided care at no cost to qualified participants.

Sponsored by Agios Pharmaceuticals, Inc.

Quick Self-Assessment

See if you qualify for this Pittsburgh location

Preparing your pre-screening questions…

Expert Care in Pittsburgh

Access phenylketonuria specialists at no cost

IRB Approved

This study follows strict safety protocols and ethical guidelines

No-Cost Care

All study-related phenylketonuria treatment provided free

Apply for This Pittsburgh Location

Check if you qualify for this phenylketonuria clinical trial in Pittsburgh, PA

Secure & Confidential

Your information is protected and will only be shared with the research team.

Why Participate?

  • No-Cost Study Care

  • Local to Pittsburgh

    Convenient for PA residents

  • Cutting-Edge Treatment

    Access to innovative therapies

  • Expert Medical Care

    Close monitoring by specialists

  • Possible Compensation*

    For time and travel

*Compensation varies by study. Confirm details with coordinator.

Simple Process

  1. 1Submit this form
  2. 2Phone screening
  3. 3Visit Pittsburgh site if eligible
  4. 4Begin participation

About This Phenylketonuria Study in Pittsburgh

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

Sponsor: Agios Pharmaceuticals, Inc.

Who Can Participate

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. Key

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.

Not sure if you qualify? Submit your interest and a study coordinator will help determine your eligibility.

Frequently Asked Questions

Q:Is this study available in Pittsburgh?

Yes, this clinical trial (NCT07241234) has an active research site in Pittsburgh, PA that is currently enrolling participants.

Q:Is it safe to participate?

Clinical trials follow strict safety guidelines and ethical standards. This study has been reviewed and approved, and participants are closely monitored by medical professionals. You can withdraw at any time.

Q:Will I be compensated?

Many clinical trials offer compensation for your time and travel expenses. Specific compensation details will be discussed during the screening process. All study-related medical care is provided at no cost.

Q:Can I leave the trial if I change my mind?

Absolutely. Participation is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty.

Still have questions? Our study coordinators are here to help.

Phenylketonuria Treatment Options in Pittsburgh, PA

If you're searching for phenylketonuria treatment options in Pittsburgh, PA, this clinical trial (NCT07241234) may be an excellent opportunity. Clinical trials provide access to cutting-edge treatments that aren't yet available to the general public, often at no cost to participants.

Our Pittsburgh research site is actively enrolling participants for this clinical trial. You'll receive care from experienced phenylketonuria specialists who are at the forefront of medical research. All study-related care, including examinations, treatments, and monitoring, is provided at no cost to qualified participants.

Looking for more options? Browse all phenylketonuria clinical trials near you to find additional studies recruiting in your area.

More Essential Tremor Trials in Pittsburgh, PA

See all essential tremor clinical trials recruiting in Pittsburgh — not just this study.

Browse Essential Tremor Trials in Pittsburgh →

Browse More Trials by Condition

Essential Tremor TrialsPulmonary Hypertension TrialsPKU TrialsAll Conditions →

Ready to Join in Pittsburgh?

Take the first step toward participating in this groundbreaking clinical trial

Secure · Expert Care · Pittsburgh, PA