- Conditions
- Phenylketonuria (PKU)
- Interventions
- Pegvaliase
- Drug
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- 18 Years to 70 Years
- Enrollment
- 37 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2021
- U.S. locations
- 17
- States / cities
- Gainesville, Florida • Tampa, Florida • Chicago, Illinois + 14 more
Search Results
Search by objective public record fields.
Search by query text, NCT ID, condition, intervention, sponsor, city, state, recruitment status, phase, study type, healthy volunteer eligibility, sex, or age. Results come from ClinicalTrials.gov. When the live registry is unavailable, a clearly labeled stored copy may be shown.
These results were retrieved from the live ClinicalTrials.gov registry. Filters and sort order apply to this result list.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Showing 1–24
of 95
matching trials from the live ClinicalTrials.gov search.
- Conditions
- Phenylketonuria (PKU)
- Interventions
- JNT-517
- Drug
- Lead sponsor
- Otsuka Pharmaceutical Development & Commercialization, Inc.
- Industry
- Eligibility
- 4 Years and older
- Enrollment
- 240 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2028
- U.S. locations
- 8
- States / cities
- Gainesville, Florida • Tampa, Florida • Portland, Oregon + 4 more
- Conditions
- Phenylketonuria
- Interventions
- SYNB1934v1, Placebo
- Drug
- Lead sponsor
- Synlogic
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 35 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2024
- U.S. locations
- 14
- States / cities
- Culver City, California • Orange, California • Palo Alto, California + 11 more
- Conditions
- Phenylketonurias
- Interventions
- sapropterin dihydrochloride
- Drug
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- 8 Years and older
- Enrollment
- 100 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2005 – 2006
- U.S. locations
- 14
- States / cities
- Los Angeles, California • Oakland, California • Sacramento, California + 11 more
- Conditions
- Hypertension
- Interventions
- 6R-BH4 (sapropterin dihydrochloride)
- Drug
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 84 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2006 – 2008
- U.S. locations
- 24
- States / cities
- Hoover, Alabama • Huntsville, Alabama • Mobile, Alabama + 21 more
- Conditions
- Phenylketonuria (PKU)
- Interventions
- Pegvaliase
- Drug
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- Not listed
- Enrollment
- 200 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2033
- U.S. locations
- 11
- States / cities
- Aurora, Colorado • Atlanta, Georgia • Chicago, Illinois + 8 more
- Conditions
- Phenylketonuria
- Interventions
- Sapropterin
- Drug
- Lead sponsor
- Children's Hospital of Philadelphia
- Other
- Eligibility
- 18 Years to 50 Years
- Enrollment
- 6 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2011
- U.S. locations
- 1
- States / cities
- Philadelphia, Pennsylvania
- Conditions
- Tetrahydrobiopterin Deficiencies, Hyperphenylalaninemia, Non-Phenylketonuric
- Interventions
- Phenoptin
- Drug
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- Not listed
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2006 – 2009
- U.S. locations
- 9
- States / cities
- Los Angeles, California • Chicago, Illinois • Minneapolis, Minnesota + 6 more
- Conditions
- Phenylketonurias
- Interventions
- PreKUnil® LNAA Medical Food for PKU
- Other
- Lead sponsor
- University of Southern California
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2024
- U.S. locations
- 1
- States / cities
- Los Angeles, California
- Conditions
- Alcohol Dependence
- Interventions
- Not listed
- Lead sponsor
- University of Texas Southwestern Medical Center
- Other
- Eligibility
- 18 Years to 60 Years · Male only
- Enrollment
- 75 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2007 – 2012
- U.S. locations
- 1
- States / cities
- Dallas, Texas
- Conditions
- Phenylketonuria (PKU) and Hyperphenylalaninemia
- Interventions
- PheCheck, Dried Blood Spot Cards.
- Device · Diagnostic Test
- Lead sponsor
- Aptatek BioSciences, Inc
- Industry
- Eligibility
- 1 Year and older
- Enrollment
- 60 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2027
- U.S. locations
- 2
- States / cities
- Chicago, Illinois • Boston, Massachusetts
- Conditions
- Phenylketonuria (PKU)
- Interventions
- Pegvaliase, Diet Only
- Drug · Other
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- 12 Years to 17 Years
- Enrollment
- 55 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2027
- U.S. locations
- 13
- States / cities
- Phoenix, Arizona • Little Rock, Arkansas • Aurora, Colorado + 10 more
- Conditions
- Phenylketonuria
- Interventions
- BMN 165 (rAvPAL-PEG)
- Biological
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- 16 Years to 70 Years
- Enrollment
- 24 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2015
- U.S. locations
- 6
- States / cities
- Aurora, Colorado • Gainesville, Florida • Boston, Massachusetts + 3 more
- Conditions
- Phenylketonuria
- Interventions
- PTC923, Placebo
- Drug
- Lead sponsor
- PTC Therapeutics
- Industry
- Eligibility
- Not listed
- Enrollment
- 157 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2023
- U.S. locations
- 12
- States / cities
- Stanford, California • Aurora, Colorado • Gainesville, Florida + 9 more
- Conditions
- Phenylketonuria
- Interventions
- JNT-517, JNT-517 Matching Placebo
- Drug
- Lead sponsor
- Otsuka Pharmaceutical Development & Commercialization, Inc.
- Industry
- Eligibility
- 4 Years and older
- Enrollment
- 120 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2028
- U.S. locations
- 10
- States / cities
- Los Angeles, California • Gainesville, Florida • Tampa, Florida + 7 more
- Conditions
- Phenylketonuria (PKU)
- Interventions
- Kuvan, Large Neutral Amino Acid Therapy
- Drug · Dietary Supplement
- Lead sponsor
- University of Southern California
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2014
- U.S. locations
- 1
- States / cities
- Los Angeles, California
- Conditions
- Phenylketonuria
- Interventions
- Sapropterin (Kuvan)
- Drug
- Lead sponsor
- Washington University School of Medicine
- Other
- Eligibility
- 6 Years to 50 Years
- Enrollment
- 45 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2008 – 2018
- U.S. locations
- 1
- States / cities
- St Louis, Missouri
- Conditions
- Phenylketonuria, Healthy
- Interventions
- SYNB1618, Placebo
- Drug
- Lead sponsor
- Synlogic
- Industry
- Eligibility
- 18 Years to 64 Years
- Enrollment
- 70 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2018 – 2019
- U.S. locations
- 4
- States / cities
- Boston, Massachusetts • Pittsburgh, Pennsylvania • Nashville, Tennessee + 1 more
- Conditions
- BH4 Deficiency, Hyperphenylalaninemia
- Interventions
- PTC923
- Drug
- Lead sponsor
- PTC Therapeutics
- Industry
- Eligibility
- 12 Months and older
- Enrollment
- 8 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2020
- U.S. locations
- 4
- States / cities
- Minneapolis, Minnesota • Dallas, Texas • Salt Lake City, Utah + 1 more
- Conditions
- Phenylketonuria
- Interventions
- SAR444836
- Drug
- Lead sponsor
- Sanofi
- Industry
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 32 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2030
- U.S. locations
- 7
- States / cities
- San Francisco, California • Aurora, Colorado • Gainesville, Florida + 4 more
- Conditions
- COPD, Tetrahydrobiopterin Deficiency, Oxidative Stress
- Interventions
- Tetrahydrobiopterin, Placebo oral tablet
- Drug
- Lead sponsor
- University of Massachusetts, Amherst
- Other
- Eligibility
- 18 Years to 85 Years
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2021 – 2022
- U.S. locations
- 1
- States / cities
- Amherst, Massachusetts
- Conditions
- Phenylketonuria (PKU)
- Interventions
- Pegvaliase
- Drug
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- Not listed
- Enrollment
- 450 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2033
- U.S. locations
- 12
- States / cities
- Aurora, Colorado • Atlanta, Georgia • Chicago, Illinois + 9 more
- Conditions
- Phenylketonurias, PAH Deficiency, Phenylketonuria
- Interventions
- HMI-103
- Drug
- Lead sponsor
- Homology Medicines, Inc
- Industry
- Eligibility
- 18 Years to 55 Years
- Enrollment
- 3 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2023
- U.S. locations
- 2
- States / cities
- Topeka, Indiana • Lancaster, Pennsylvania
- Conditions
- Phenylketonuria
- Interventions
- Administration of CANTAB and Subject Global Assessment
- Other
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- 18 Years to 70 Years
- Enrollment
- 9 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2017
- U.S. locations
- 4
- States / cities
- Lexington, Kentucky • Omaha, Nebraska • Oklahoma City, Oklahoma + 1 more