Safety and Efficacy of Self Administered Injections of Pegvaliase (>40mg/Day Dose) in Adults With PKU

Part of paid clinical trials in Gainesville, Florida.

Sponsor
BioMarin Pharmaceutical
Study ID
NCT03694353
Phase
PHASE3
Status
Completed

Conditions

  • Phenylketonuria (PKU)

Eligibility Criteria

Sex
ALL
Age
18 Years - 70 Years
Healthy Volunteers
Not accepted

Interventions

Study Details

This is a Phase 3 open-label extension study enrolling adult patients with PKU who were previously treated with pegvaliase in Studies PAL-003 (NCT00924703) or 165-302 (NCT02468570). The study is designed to evaluate the long-term safety and efficacy of pegvaliase administered at doses \> 40 mg/day to 60 mg/day.

Key Dates

Start date
Sep 13, 2018
Status verified
Feb 2022
Primary completion
Jan 13, 2021
Completion
Jan 13, 2021

Study Design

Enrollment
37 participants (actual)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Pegvaliase
    Beginning on Day 1, subjects will receive the same dose and regimen of pegvaliase they were receiving in 165-302 or PAL-003 (pegvaliase dosing should continue without interruption from the previous study). Subsequent revisions to dosing regimens are allowed following consultation with the medical monitor.

Primary Outcome Measure

Number of Participants With Treatment-emergent Adverse Events (TEAEs) as Assessed by CTCAE v5.0 [ Time Frame: Up to Day 741 (approximately Week 106) ]

Locations (17)

FacilityCityStateZIPSite coordinators
University of FloridaGainesvilleFlorida32610-
University of South FloridaTampaFlorida33606-
Ann & Robert H. Lurie Children's Hospital of ChicagoChicagoIllinois60611-
Indiana CTSI Clinical Research CenterIndianapolisIndiana46202-
University of Kentucky Medical CenterLexingtonKentucky40536-
Wayne State University, Center for Molecular Medicine and Genetics, Pediatrics and PathologyDetroitMichigan48201-
University of Missouri Health CareColumbiaMissouri65201-
Washington University School of MedicineSt LouisMissouri63110-
University of Nebraska Medical CenterOmahaNebraska68198-
Morristown Medical CenterMorristownNew Jersey07960-
University of Oklahoma Health Science CenterOklahoma CityOklahoma73104-
Oregon Health and Science UniversityPortlandOregon97239-
St. Christopher's Hospital for ChildrenPhiladelphiaPennsylvania19134-
UPMC Children's Hospital of PittsburghPittsburghPennsylvania15224-
Vanderbilt University Medical CenterNashvilleTennessee37232-
University of Texas Health Science Center at HoustonHoustonTexas77030-
Medical College of MilwaukeeMilwaukeeWisconsin53226-

Find similar trials in Gainesville, FL

By research site
University of Florida· Gainesville, FLUniversity of South Florida· Tampa, FLAnn & Robert H. Lurie Children's Hospital of Chicago· Chicago, ILIndiana CTSI Clinical Research Center· Indianapolis, INUniversity of Kentucky Medical Center· Lexington, KYWayne State University, Center for Molecular Medicine and Genetics, Pediatrics and Pathology· Detroit, MI

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