A Prospective Clinical Study of Phenylketonuria (PKU)
1 other identifier
observational
32
1 country
14
Brief Summary
This is a study for adults and children ≥ 14 years old who have Phenylketonuria (PKU) with uncontrolled plasma Phe levels. No clinical intervention or study drug is provided by BioMarin in this study.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for all trials
Started Oct 2019
Typical duration for all trials
14 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
Study Start
First participant enrolled
October 11, 2019
CompletedFirst Submitted
Initial submission to the registry
June 26, 2020
CompletedFirst Posted
Study publicly available on registry
June 30, 2020
CompletedPrimary Completion
Last participant's last visit for primary outcome
June 2, 2022
CompletedStudy Completion
Last participant's last visit for all outcomes
June 2, 2022
CompletedMarch 14, 2023
September 1, 2022
2.6 years
June 26, 2020
March 13, 2023
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Change in Plasma Phe Levels
through study completion, an average of 2 years
Secondary Outcomes (1)
Change in dietary protein intake from medical and intact food
through study completion, an average of 2 years
Eligibility Criteria
PKU Patients 14 years or older with uncontrolled plasma Phe levels
You may qualify if:
- Male and female subjects with diagnosis of PKU which is a condition characterized by PAH deficiency
- Ability and willingness to maintain dietary protein intake consistent with baseline intake
- Willingness and capable per investigator opinion to comply with study procedures and requirements
- Plasma Phe Levels \> 600umol/L
You may not qualify if:
- Clinically significant liver dysfunction or disease
- Prior treatment with gene therapy
- Any condition that, in the opinion of the investigator or Sponsor, would prevent the subject from fully complying with the requirements of the study
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (14)
Children's Hosptial of Colorado
Aurora, Colorado, 80045, United States
University of Florida
Gainesville, Florida, 32605, United States
University of South Florida
Tampa, Florida, 33606, United States
Emory University Hospital
Atlanta, Georgia, 30322, United States
Ann & Robert H. Lurie Chilren's Hospital
Chicago, Illinois, 60611, United States
Riley Hospital for Children
Indianapolis, Indiana, 46202, United States
University of Kentucky
Lexington, Kentucky, 40536-0293, United States
Boston Children's Hospital
Boston, Massachusetts, 02115, United States
Morristown Medical Center
Morristown, New Jersey, 07960, United States
Oregon Health & Science University
Portland, Oregon, 97239, United States
UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224, United States
Clinic for Special Children
Strasburg, Pennsylvania, 17579, United States
University of Texas Health Science Center at Houston
Houston, Texas, 77030, United States
Medical College of Wisconsin
Wauwatosa, Wisconsin, 53226, United States
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Design
- Study Type
- observational
- Observational Model
- COHORT
- Time Perspective
- PROSPECTIVE
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
June 26, 2020
First Posted
June 30, 2020
Study Start
October 11, 2019
Primary Completion
June 2, 2022
Study Completion
June 2, 2022
Last Updated
March 14, 2023
Record last verified: 2022-09