Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria

Part of paid clinical trials in Phoenix, Arizona.

Sponsor
BioMarin Pharmaceutical
Study ID
NCT05270837
Phase
PHASE3
Status
Active Not Recruiting

Conditions

  • Phenylketonuria (PKU)

Eligibility Criteria

Sex
ALL
Age
12 Years - 17 Years
Healthy Volunteers
Not accepted

Interventions

  • Pegvaliase — DRUG
    Pegvaliase 2.5mg/10mg/20mg/40mg/60mg self-administered from 1 time up to 7 times a week
  • Diet Only — OTHER
    Diet Control

Study Details

This is a Phase 3 open-label randomized controlled study enrolling approximately 54 adolescents with PKU. The study is designed to assess the safety and efficacy of pegvaliase injections.

Key Dates

Start date
Jun 17, 2022
Status verified
Sep 2025
Primary completion
Jan 14, 2025
Completion
Oct 31, 2027

Study Design

Enrollment
55 participants (estimated)
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

Arms

  • Experimental: Pegvaliase
  • Other: Diet Only
    Participants will be managing their PKU with diet alone. Participants in the diet-only control arm will be required to maintain and adjust dietary and medical protein food intake through Week 72, initiating pegvaliase treatment beginning Week 73 and, from Weeks 73 through 215.

Primary Outcome Measure

Change in blood Phe concentration [ Time Frame: Treatment naïve baseline following 72 weeks on study ]

Locations (13)

FacilityCityStateZIPSite coordinators
Phoenix Children's HospitalPhoenixArizona85016-
Arkansas Children's HospitalLittle RockArkansas72202-
Children's Hospital of ColoradoAuroraColorado80045-
University of South FloridaTampaFlorida33606-
Ann and Robert H. Lurie Children's Hospital of ChicagoChicagoIllinois60611-
IU Health University HospitalIndianapolisIndiana46202-
University of Kentucky College of MedicineLexingtonKentucky40506-
Boston Children's HospitalBostonMassachusetts02115-
University of Pittsburgh Medical CenterPittsburghPennsylvania15224-
McGovern Medical School, University of Texas Health Science Center at Houston (UTHealth)HoustonTexas77030-
University of Utah Medical CenterSalt Lake CityUtah84132-
University of Virginia School of MedicineCharlottesvilleVirginia22903-
Children's Hospital of WisconsinMilwaukeeWisconsin53226-

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Phoenix Children's Hospital· Phoenix, AZArkansas Children's Hospital· Little Rock, ARChildren's Hospital of Colorado· Aurora, COUniversity of South Florida· Tampa, FLAnn and Robert H. Lurie Children's Hospital of Chicago· Chicago, ILIU Health University Hospital· Indianapolis, IN

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