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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT03694353
Recruitment Status : Completed
First Posted : October 3, 2018
Last Update Posted : February 4, 2021
Information provided by (Responsible Party):
BioMarin Pharmaceutical

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

Condition or disease Intervention/treatment Phase
Phenylketonuria (PKU) Drug: Pegvaliase Phase 3

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 37 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-label Extension Study to Evaluate the Safety and Efficacy of Subcutaneous Injections of Pegvaliase (> 40 mg/Day Dose) in Adults With Phenylketonuria
Actual Study Start Date : September 13, 2018
Actual Primary Completion Date : January 13, 2021
Actual Study Completion Date : January 13, 2021

Arm Intervention/treatment
Experimental: Pegvaliase Drug: Pegvaliase

Primary Outcome Measures :
  1. Incidence of treatment-emergent adverse events as assessed by CTCAE v5.0 [ Time Frame: Week 0 - Week 121 ]
  2. Change in blood Phe concentration [ Time Frame: Week 0 - Week 121 ]

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   18 Years to 70 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion criteria:

  • Must be enrolled in PAL-003 or 165-302 Part 4 at the time of screening for 165-304 and most recently receiving pegvaliase at a dose > 40 mg/day
  • Has identified a competent person or persons ≥ 18 years of age who can observe the subject during study drug administration and for a minimum of 1 hour following administration in situations required per protocol
  • For females with childbearing potential, must have negative pregnancy test at screening and be willing to have additional pregnancy tests during the study.
  • If sexually active and not planning to become pregnant (self or partner), must be willing to use 2 acceptable methods of contraception while participating in the study and for 4 weeks after the study.

Exclusion criteria:

  • Use of any investigational product (except pegvaliase) or investigational medical device within 30 days prior to screening or requirement for any investigational agent prior to completion of all scheduled study assessments.
  • Use of any medication (except pegvaliase) intended to treat PKU, including the use of large neutral amino acids, within 2 days prior to the administration of pegvaliase (Day 1).
  • Use or planned use of any injectable drugs containing PEG (other than pegvaliase), including medroxyprogesterone injection, within 3 months prior to screening and during study participation.
  • Current participation in the Kuvan® registry study (PKU Demographics, Outcomes and Safety [PKUDOS])
  • A history of organ transplantation or on chronic immunosuppressive therapy

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT03694353

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United States, Florida
University of Florida
Gainesville, Florida, United States, 32610
University of South Florida
Tampa, Florida, United States, 33606
United States, Illinois
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611
United States, Indiana
Indiana CTSI Clinical Research Center
Indianapolis, Indiana, United States, 46202
United States, Kentucky
University of Kentucky Medical Center
Lexington, Kentucky, United States, 40536
United States, Missouri
University of Missouri Health Care
Columbia, Missouri, United States, 65201
Washington University School of Medicine
Saint Louis, Missouri, United States, 63110
United States, Nebraska
University of Nebraska Medical Center
Omaha, Nebraska, United States, 68198
United States, New Jersey
Morristown Medical Center
Morristown, New Jersey, United States, 07960
United States, Oklahoma
University of Oklahoma Health Science Center
Oklahoma City, Oklahoma, United States, 73104
United States, Oregon
Oregon Health and Science University
Portland, Oregon, United States, 97239
United States, Pennsylvania
St. Christopher's Hospital for Children
Philadelphia, Pennsylvania, United States, 19134
UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States, 15224
United States, Tennessee
Vanderbilt University Medical Center
Nashville, Tennessee, United States, 37232
United States, Texas
University of Texas Health Science Center at Houston
Houston, Texas, United States, 77030
United States, Wisconsin
Medical College of Milwaukee
Milwaukee, Wisconsin, United States, 53226
Sponsors and Collaborators
BioMarin Pharmaceutical
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Study Director: Medical Director, MD BioMarin Pharmaceutical
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Responsible Party: BioMarin Pharmaceutical Identifier: NCT03694353    
Other Study ID Numbers: 165-304
First Posted: October 3, 2018    Key Record Dates
Last Update Posted: February 4, 2021
Last Verified: February 2021

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Amino Acid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Metabolic Diseases