Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.
COMPLETED
32 participants
OBSERVATIONAL
2019-10-11
2022-06-02
Brief Summary
Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.
Related Clinical Trials
Explore similar clinical trials based on study characteristics and research focus.
A Non-Interventional Interview Study of Phenylketonuria (PKU) Adults
NCT03505125
A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects With PKU Who Have Elevated Phenylalanine Levels
NCT00225615
PKU Skin Stripping
NCT04368624
AAV Gene Therapy Study for Subjects with PKU
NCT04480567
Nutrition Status of Adults With PKU Before and During Treatment With Pegvaliase
NCT03856203
Detailed Description
Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.
Conditions
See the medical conditions and disease areas that this research is targeting or investigating.
Study Design
Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.
COHORT
PROSPECTIVE
Eligibility Criteria
Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.
Inclusion Criteria
* 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
Exclusion Criteria
* 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
14 Years
ALL
No
Sponsors
Meet the organizations funding or collaborating on the study and learn about their roles.
BioMarin Pharmaceutical
INDUSTRY
Responsible Party
Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.
Locations
Explore where the study is taking place and check the recruitment status at each participating site.
Children's Hosptial of Colorado
Aurora, Colorado, United States
University of Florida
Gainesville, Florida, United States
University of South Florida
Tampa, Florida, United States
Emory University Hospital
Atlanta, Georgia, United States
Ann & Robert H. Lurie Chilren's Hospital
Chicago, Illinois, United States
Riley Hospital for Children
Indianapolis, Indiana, United States
University of Kentucky
Lexington, Kentucky, United States
Boston Children's Hospital
Boston, Massachusetts, United States
Morristown Medical Center
Morristown, New Jersey, United States
Oregon Health & Science University
Portland, Oregon, United States
UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States
Clinic for Special Children
Strasburg, Pennsylvania, United States
University of Texas Health Science Center at Houston
Houston, Texas, United States
Medical College of Wisconsin
Wauwatosa, Wisconsin, United States
Countries
Review the countries where the study has at least one active or historical site.
Other Identifiers
Review additional registry numbers or institutional identifiers associated with this trial.
307-902
Identifier Type: -
Identifier Source: org_study_id
More Related Trials
Additional clinical trials that may be relevant based on similarity analysis.