Immune Modulation During Palynziq® Treatment in Adults (IMPALA)
ConditionPhenylketonuria
Trial statusRecruiting
Trial phasePhase 4
Trial typeInterventional
Biological sexAll
Age18-65
SponsorBioMarin Pharmaceutical
About this trial
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
Qualifiers
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
Disqualifiers
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)
Trial design
Treatments tested in this trial
- Pegvaliase
- Methotrexate
Treatment groups
12 Participants
are divided into 2 treatment groupsSponsors and collaborators
Source ClinicalTrials.gov