Phase 4: testing Pegvaliase for Phenylketonuria
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 how well it works in adults with PKU.
Official registry: https://clinicaltrials.gov/study/NCT07477691 · BioMarin Pharmaceutical · enrollment listed as 12
What this phase means
Phase 4: the treatment is already approved. This study asks new questions — longer-term effects, other diseases, or how it compares in everyday care.
Phase labels are FDA/ICH categories applied to the registry Phase field. They are not a finding that this study succeeded.
What the registry says happens
If you join, the listed intervention(s) are Pegvaliase, or Methotrexate. The registry lists allocation as randomized. 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 how well it works in adults with PKU.
They plan to enroll about 12 people at 12 sites. Taking part typically means taking a study drug (or dummy pill), an infusion or injection of a biologic plus check-ins.
Official title (unrewritten)
A Phase 4 Study of Immune Modulation During Palynziq® Treatment in Adults With Phenylketonuria (PKU)
Interventions: Pegvaliase, Methotrexate
Primary outcome (registry wording): Change in blood Phe levels from baseline to Week 25 and Week 49
Who it is for (from inclusion criteria)
- Ages 18 Years to 65 Years
- Adults between 18 and 65 years old
- Have a confirmed diagnosis of phenylketonuria (PKU)
- Are in generally good health based on medical evaluation
- 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
Who the registry excludes
- 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
Public study contact (from the registry)
Trial Specialist
Published on ClinicalTrials.gov. Contact them as a possible participant. They decide eligibility.
Sites listed on ClinicalTrials.gov
12 listed- UCLA Dept. of Human Genetics — Los Angeles, California (recruiting)
- Children's Hospital Colorado — Aurora, Colorado (recruiting)
- University Of South Florida Medical Center — Tampa, Florida (recruiting)
- Ann & Robert H. Lurie Children's Hospital of Chicago — Chicago, Illinois (recruiting)
- Indiana University School of Medicine — Indianapolis, Indiana (recruiting)
- University of Minnesota Medical School — Minneapolis, Minnesota (recruiting)
- University of Rochester Medical Center (URMC) — Rochester, New York (recruiting)
- Oregon Health & Science University (OHSU) - PARENT — Portland, Oregon (recruiting)
- University of Pittsburgh Medical Center (UPMC) - Children's Hospital of Pittsburgh — Pittsburgh, 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 (recruiting)
FAQ about this NCT record
Is NCT07477691 still recruiting?
The sponsor posted status “Recruiting” on ClinicalTrials.gov at last ingest. Sites can pause independently. Confirm with the official record and the listed contact.
What phase is NCT07477691?
Phase 4. Phase labels are FDA/ICH categories applied to the registry Phase field. They are not a finding that this study succeeded.
Can PlainTrials tell me if I qualify for NCT07477691?
No. The inclusion and exclusion lists on this page are readable extracts of the public eligibility block. Only the study team can screen you.
How to read a ClinicalTrials.gov record · Questions to ask the coordinator · Glossary