Immune Modulation During Palynziq® Treatment in Adults (IMPALA)

Part of paid clinical trials in Los Angeles, California.

Sponsor
BioMarin Pharmaceutical
Study ID
NCT07477691
Phase
PHASE4
Status
Not Yet Recruiting

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Conditions

Eligibility Criteria

Sex
ALL
Age
18 Years - 65 Years
Healthy Volunteers
Not accepted

Interventions

  • Pegvaliase — BIOLOGICAL
    Pegvaliase (Palynziq) administered by subcutaneous injection. Dosing follows U.S. Prescribing Information and investigator judgement.
  • Methotrexate — DRUG
    Oral methotrexate 15 mg administered once weekly during the Tolerability Period and the Combination Treatment Period.

Study Details

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.

Key Dates

Start date
Mar 31, 2026
Status verified
Mar 2026
Primary completion
Sep 30, 2027
Completion
Sep 30, 2027

Study Design

Enrollment
12 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

Arms

  • Experimental: Palynziq naïve
    Individuals with uncontrolled blood phenylalanine (Phe) \> 600 μmol/L on existing management who have never been treated with Palynziq. Participants will have a 4-week Screening Period, followed by a 4-week MTX Tolerability Period during which only MTX will be administered. If MTX is tolerated during the MTX Tolerability Period, the participant will then enter the 24-week Combination Treatment Period during which MTX + Palynziq will be administered concurrently. After completion of Week 24 (Combination Treatment Period), MTX will be discontinued for both cohorts and Palynziq only will continue for another 24 weeks (to Week 49).
  • Experimental: Palynziq experienced
    Individuals who have uncontrolled blood Phe \> 600 μmol/L after ≥ 24 consecutive weeks of treatment with Palynziq, are on a current dose of ≥ 20mg/day and are unable to further dose escalate (Palynziq experienced). Participants will have a 4-week Screening Period. Participants will then have 24 weeks of concurrent MTX + Palynziq treatment (Combination Treatment Period): participants will be assessed for MTX tolerability during the first 4 weeks of MTX + Palynziq (MTX Tolerability Period) and then receive another 20 weeks of MTX + Palynziq. After completion of Week 24 (Combination Treatment Period), MTX will be discontinued for both cohorts and Palynziq only will continue for another 24 weeks (to Week 49).

Primary Outcome Measure

Change in blood Phe levels from baseline to Week 25 and Week 49 [ Time Frame: Baseline to Week 25 and Baseline to Week 49 ]

Central Contacts

Locations (12)

FacilityCityStateZIPSite coordinators
UCLA Dept. of Human GeneticsLos AngelesCalifornia90095-
Children's Hospital ColoradoAuroraColorado80045-
University Of South Florida Medical CenterTampaFlorida33613-
Ann & Robert H. Lurie Children's Hospital of ChicagoChicagoIllinois60611-
Indiana University School of MedicineIndianapolisIndiana46202-
University of Minnesota Medical SchoolMinneapolisMinnesota55455-
University of Rochester Medical Center (URMC)RochesterNew York14642-
Oregon Health & Science University (OHSU) - PARENTPortlandOregon97239-
University of Pittsburgh Medical Center (UPMC) - Children's Hospital of PittsburghPhiladelphiaPennsylvania19104-
UT Southwestern Medical CenterDallasTexas75390
Markey McNutt
University of Utah HospitalSalt Lake CityUtah84108-
Children's Hospital of WisconsinMilwaukeeWisconsin53226-

Find similar trials in Los Angeles, CA

By research site
UCLA Dept. of Human Genetics· Los Angeles, CAChildren's Hospital Colorado· Aurora, COUniversity Of South Florida Medical Center· Tampa, FLAnn & Robert H. Lurie Children's Hospital of Chicago· Chicago, ILIndiana University School of Medicine· Indianapolis, INUniversity of Minnesota Medical School· Minneapolis, MN

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