Immune Modulation During Palynziq® Treatment in Adults (IMPALA)
Condition(s) studied
Investigational drug(s) / intervention(s)
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
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)
| Facility | City | Region | Status |
|---|---|---|---|
| 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 |
More BioMarin Pharmaceutical trials in the USA
Other trials for the same condition
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