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

NCT ID NCT07477691

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

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.

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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

Contacts and locations

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Study contacts

Contact

Phone: •••-•••-•••• 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

Contact
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

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