Ireland
--:--IST
Recruiting Phase 4

Immune Modulation During Palynziq® Treatment in Adults (IMPALA)

NCT07477691 · tracked via the Priya Life Science USA tracker
Phase
Phase 4
Started
2026-05-29
Last updated
2026-06-17

Condition(s) studied

Phenylketonuria

Investigational drug(s) / intervention(s)

Pegvaliase →Methotrexate →

Pegvaliase: Pegvaliase (Palynziq) administered by subcutaneous injection. Dosing follows U.S. Prescribing Information and investigator judgement.

Methotrexate: Oral methotrexate 15 mg administered once weekly during the Tolerability Period and the Combination Treatment Period.

Study 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

Sex
ALL
Min age
18 Years
Max age
65 Years
Healthy volunteers
No
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

Primary outcome measure(s)

  • Change in blood Phe levels from baseline to Week 25 and Week 49 — Baseline to Week 25 and Baseline to Week 49
    To evaluate blood Phe response in adults with PKU co-administered Palynziq with MTX for 6 months
  • Anti-PEG IgG, IgM, anti-PAL IgG, IgM levels at Weeks 5, 9, 25, and 49 — Baseline to Week 5, Week 9, Week 25, and Week 49
    To evaluate the ability of MTX to modulate the immune response to Palynziq in adults with PKU

Trial sites (12)

FacilityCityRegionStatus
UCLA Dept. of Human Genetics Los Angeles California Not Yet Recruiting
Children's Hospital Colorado Aurora Colorado Not Yet Recruiting
University Of South Florida Medical Center Tampa Florida Not Yet Recruiting
Ann & Robert H. Lurie Children's Hospital of Chicago Chicago Illinois Not Yet Recruiting
Indiana University School of Medicine Indianapolis Indiana Not Yet Recruiting
University of Minnesota Medical School Minneapolis Minnesota Not Yet Recruiting
University of Rochester Medical Center (URMC) Rochester New York Not Yet Recruiting
Oregon Health & Science University (OHSU) - PARENT Portland Oregon Not Yet Recruiting
University of Pittsburgh Medical Center (UPMC) - Children's Hospital of Pittsburgh Philadelphia Pennsylvania Not Yet Recruiting
UT Southwestern Medical Center Dallas Texas Recruiting
University of Utah Hospital Salt Lake City Utah Not Yet Recruiting
Children's Hospital of Wisconsin Milwaukee Wisconsin Not Yet Recruiting
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT07477691 on ClinicalTrials.gov ↗ ← All trials in the USA