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Immune Modulation During Palynziq® Treatment in Adults (IMPALA)

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Trial Status Not Yet Recruiting

Trial Identifier

NCT07477691

Condition

Phenylketonuria

The below information is taken directly from public registry websites such as ClinicalTrials.gov, EuClinicalTrials.eu, ISRCTN.com, etc. and has not been edited.

Trial Summary

Study 165-401 is a Phase 4, open-label study designed to examine the concomitant use of methotrexate (MTX) to suppress immune responses to Palynziq and improve tolerability and efficacy in adults with PKU.

Eligibility Criteria

Birth Sex

All

Age

18 Years to 65 Years

Healthy Volunteers

No

Drug/Treatment:

Pegvaliase, Methotrexate

Phase:

Phase 4

Study Type:

Interventional

Number of Participants:

12

Study Started:

2026-03-31

Study Updated:

2026-03-17

Trial Locations

  • UCLA Dept. of Human Genetics

    Los Angeles, California, United States

  • Children's Hospital Colorado

    Aurora, Colorado, United States

  • University Of South Florida Medical Center

    Tampa, Florida, United States

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

    Chicago, Illinois, United States

  • Indiana University School of Medicine

    Indianapolis, Indiana, United States

  • University of Minnesota Medical School

    Minneapolis, Minnesota, United States

  • University of Rochester Medical Center (URMC)

    Rochester, New York, United States

  • Oregon Health & Science University (OHSU) - PARENT

    Portland, Oregon, United States

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

    Philadelphia, Pennsylvania, United States

  • UT Southwestern Medical Center

    Dallas, Texas, United States

    Contact

    Markey McNutt

  • University of Utah Hospital

    Salt Lake City, Utah, United States

    Contact

    Markey McNutt

  • Children's Hospital of Wisconsin

    Milwaukee, Wisconsin, United States

    Contact

    Markey McNutt

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

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