Cure My Disease

Don't miss your cure!

 Log in / Sign up

Can a common arthritis drug make PKU treatment safer and more effective?

NCT ID NCT07477691

Disease control Sponsor: BioMarin Pharmaceutical Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tests whether adding methotrexate (a drug that calms the immune system) can help adults with PKU tolerate and respond better to Palynziq, a protein-based treatment. About 12 adults who either haven't taken Palynziq before or have high blood Phe levels despite treatment will receive both drugs for 6 months. The goal is to see if methotrexate reduces immune reactions that can limit Palynziq's effectiveness.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 4

Runs after approval, following long-term safety and how well the treatment works in everyday use.

Participants

About 12 people

The number the study aims to enrol. It can still change while the study runs.

Started

May 2026

Expected to finish

Sep 2027

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 to 65 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Adults between 18 and 65 years old * Have a confirmed diagnosis of phenylketonuria (PKU) * Are in generally good health based on medical evaluation * Are willing and medically eligible to receive Palynziq and methotrexate (MTX) Cohort A: Have never taken Palynziq before and are willing to start it during the study Cohort B: Have blood \> 600 μmol/L after taking Palynziq for at least 24 weeks, are on a daily dose of at least 20mg and unable to increase the dose further * Agree to use required contraception if they or their partner could become pregnant * Are willing to carry two epinephrine devices at all times during Palynziq treatment Exclusion Criteria: * Pregnant, breastfeeding, planning to become pregnant, planning to father a child, or not using effective birth control if applicable * Have a known severe allergy or hypersensitivity reaction to methotrexate (MTX), Palynziq, or other PEG-containing medications * Have a serious active infection or a history of severe or recurrent infections * Have significant medical conditions that may affect safety or participation (such as serious heart, lung, liver, kidney, immune, neurological, psychiatric, or cancer-related conditions) * Have a history of substance or alcohol abuse within the past 12 months * Have had an organ transplant or are taking chronic immunosuppressive medications * Are currently taking medications that are not allowed in the study, including other PKU treatments besides Palynziq * Are using, or plan to use, injectable PEG-containing medications other than Palynziq during the study * Have major surgery planned during the study participation period * Are currently participating in another clinical study involving Palynziq * In the opinion of the study doctor, are not a suitable candidate for the study or may have difficulty complying with study requirements

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for Phenylketonuria are added.

Our safety recommendation!

By submitting, you agree to our Terms of use

Conditions

The condition(s) this trial relates to.

phenylketonuria

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Phenylketonuria

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    12 sites. The list below names each one and where it is.

  3. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  4. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Study contacts

  • Contact

    Email: •••••@•••••

Locations

  • Ann & Robert H. Lurie Children's Hospital of Chicago

    NOT_YET_RECRUITING

    Chicago, Illinois, 60611, United States

  • Children's Hospital Colorado

    NOT_YET_RECRUITING

    Aurora, Colorado, 80045, United States

  • Children's Hospital of Wisconsin

    NOT_YET_RECRUITING

    Milwaukee, Wisconsin, 53226, United States

  • Indiana University School of Medicine

    NOT_YET_RECRUITING

    Indianapolis, Indiana, 46202, United States

  • Oregon Health & Science University (OHSU) - PARENT

    NOT_YET_RECRUITING

    Portland, Oregon, 97239, United States

  • UCLA Dept. of Human Genetics

    NOT_YET_RECRUITING

    Los Angeles, California, 90095, United States

  • UT Southwestern Medical Center

    RECRUITING

    Dallas, Texas, 75390, United States

  • University Of South Florida Medical Center

    NOT_YET_RECRUITING

    Tampa, Florida, 33613, United States

  • University of Minnesota Medical School

    NOT_YET_RECRUITING

    Minneapolis, Minnesota, 55455, United States

  • University of Pittsburgh Medical Center (UPMC) - Children's Hospital of Pittsburgh

    NOT_YET_RECRUITING

    Philadelphia, Pennsylvania, 19104, United States

  • University of Rochester Medical Center (URMC)

    NOT_YET_RECRUITING

    Rochester, New York, 14642, United States

  • University of Utah Hospital

    NOT_YET_RECRUITING

    Salt Lake City, Utah, 84108, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.