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ClinicalTrials.gov public records Last synced Sep 13, 2026, 10:35 PM EDT

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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: PAH Deficiency Clear all
Recruiting Phase 3 Interventional

A Study of JNT-517 in Participants With Phenylketonuria (PKU)

NCT06971731
View directory record Official record
Conditions
Phenylketonuria
Interventions
JNT-517 Tablet, Placebo Tablet: BID
Drug
Lead sponsor
Otsuka Pharmaceutical Development & Commercialization, Inc.
Industry
Eligibility
18 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
Source: ClinicalTrials.gov public record
Updated Jul 7, 2026 · Synced Sep 13, 2026, 10:35 PM EDT
Terminated Phase 1Phase 2 Interventional

Gene Therapy Clinical Study in Adult PKU

NCT03952156
View directory record Official record
Conditions
Phenylketonurias, PAH Deficiency
Interventions
HMI-102
Genetic
Lead sponsor
Homology Medicines, Inc
Industry
Eligibility
18 Years to 55 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2023
U.S. locations
13
States / cities
Los Angeles, California • Orange, California • Tampa, Florida + 10 more
Source: ClinicalTrials.gov public record
Updated Aug 28, 2023 · Synced Sep 13, 2026, 10:35 PM EDT
Completed Phase 3 Interventional Results available

Study of Phenoptin in Subjects With Phenylketonuria Who Participated in Protocols PKU-004 or PKU-006

NCT00332189
View directory record Official record
Conditions
Phenylketonuria
Interventions
sapropterin dihydrochloride
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
4 Years and older
Enrollment
111 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2009
U.S. locations
14
States / cities
Los Angeles, California • Sacramento, California • San Jose, California + 11 more
Source: ClinicalTrials.gov public record
Updated Nov 15, 2012 · Synced Sep 13, 2026, 10:35 PM EDT
Completed Phase 2 Interventional

Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin™ Treatment in Subjects With Phenylketonuria

NCT00104260
View directory record Official record
Conditions
Phenylketonurias
Interventions
sapropterin dihydrochloride
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
8 Years and older
Enrollment
700 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2005
U.S. locations
13
States / cities
Los Angeles, California • Oakland, California • New Haven, Connecticut + 10 more
Source: ClinicalTrials.gov public record
Updated Apr 8, 2007 · Synced Sep 13, 2026, 10:35 PM EDT
Terminated Phase 1 Interventional

Safety and Tolerability of RTX-134 in Adults With Phenylketonuria

NCT04110496
View directory record Official record
Conditions
Phenylketonurias
Interventions
RTX-134
Drug
Lead sponsor
Rubius Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2022
U.S. locations
1
States / cities
Aurora, Colorado
Source: ClinicalTrials.gov public record
Updated Dec 7, 2022 · Synced Sep 13, 2026, 10:35 PM EDT
Not listed Not applicable Interventional

Additional Dietary Large Neutral Amino Acids (LNAA) for Improved Symptoms in Adult Classical Phenylktonuria (PKU)

NCT05174559
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Dec 3, 2023 · Synced Sep 13, 2026, 10:35 PM EDT
Recruiting Phase 1 Interventional Accepts healthy volunteers

A Trial to Examine if Repinatrabit is Processed Differently in Adults With Reduced Liver or Kidney Function Compared to Adults With Normal Liver and Kidney Function

NCT07713758
View directory record Official record
Conditions
Phenylketonuria
Interventions
Repinatrabit
Drug
Lead sponsor
Otsuka Pharmaceutical Development & Commercialization, Inc.
Industry
Eligibility
18 Years to 65 Years
Enrollment
32 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2026 – 2027
U.S. locations
1
States / cities
Miami, Florida
Source: ClinicalTrials.gov public record
Updated Aug 11, 2026 · Synced Sep 13, 2026, 10:35 PM EDT
Recruiting Not applicable Interventional Accepts healthy volunteers

Educational, Social Support, and Nutritional Interventions and Their Cumulative Effect on Pregnancy Outcomes and Quality of Life in Teen and Adult Women With Phenylketonuria

NCT01659749
View directory record Official record
Conditions
Pregnancy, Phenylketonuria
Interventions
Metabolic camp
Behavioral
Lead sponsor
Emory University
Other
Eligibility
11 Years and older · Female only
Enrollment
200 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
1995 – 2037
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated May 6, 2026 · Synced Sep 13, 2026, 10:35 PM EDT
Completed No phase listed Observational

A Non-Interventional Interview Study of Phenylketonuria (PKU) Adults

NCT03505125
View directory record Official record
Conditions
Phenylketonuria (PKU)
Interventions
Not listed
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
18 Years to 70 Years
Enrollment
63 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2019
U.S. locations
4
States / cities
Gainesville, Florida • Decatur, Georgia • Chicago, Illinois + 1 more
Source: ClinicalTrials.gov public record
Updated Jan 13, 2020 · Synced Sep 13, 2026, 10:35 PM EDT
Completed Phase 2 Interventional Results available

Kuvan Therapy in Phenylketonuria (PKU): The Effect of Blood Phenylalanine Concentration on Kuvan Response

NCT00841100
View directory record Official record
Conditions
Phenylketonuria
Interventions
Kuvan, Diet
Drug · Other
Lead sponsor
University of Miami
Other
Eligibility
4 Years and older
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2011
U.S. locations
1
States / cities
Miami, Florida
Source: ClinicalTrials.gov public record
Updated Dec 2, 2019 · Synced Sep 13, 2026, 10:35 PM EDT
Completed Phase 1Phase 2 Interventional Accepts healthy volunteers

Safety and Tolerability of SYNB1618 in Healthy Adult Volunteers and Adult Subjects With Phenylketonuria

NCT03516487
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated May 12, 2021 · Synced Sep 13, 2026, 10:35 PM EDT
Recruiting Phase 3 Interventional

A Study of Sepiapterin in Participants With Phenylketonuria (PKU)

NCT06302348
View directory record Official record
Conditions
Phenylketonuria
Interventions
Sepiapterin
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
Up to 9 Years
Enrollment
56 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2031
U.S. locations
1
States / cities
Indianapolis, Indiana
Source: ClinicalTrials.gov public record
Updated Aug 27, 2026 · Synced Sep 13, 2026, 10:35 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A Phase 1, Open Label, Placebo-Controlled Study to Evaluate the Effect of GSK1349572 on Iohexol and Para-Aminohippurate Clearance in Healthy Subjects

NCT01214993
View directory record Official record
Conditions
Infections, Human Immunodeficiency Virus and Tuberculosis
Interventions
GSK1349572, Placebo, Iohexol Injection, Para-aminohippurate infusion
Drug
Lead sponsor
ViiV Healthcare
Industry
Eligibility
18 Years to 65 Years
Enrollment
38 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2010
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jun 10, 2012 · Synced Sep 13, 2026, 10:35 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Sapropterin in Individuals With Phenylketonuria

NCT00730080
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jun 17, 2018 · Synced Sep 13, 2026, 10:35 PM EDT
Completed Phase 1 Interventional Results available

Carnitine Consumption and Augmentation in Pulmonary Arterial Hypertension

NCT04908397
View directory record Official record
Conditions
Pulmonary Arterial Hypertension, Familial Primary Pulmonary Hypertension, Primary Pulmonary Hypertension, Lung Diseases, Carnitine Nutritional Deficiency
Interventions
L-carnitine
Dietary Supplement
Lead sponsor
Vanderbilt University Medical Center
Other
Eligibility
18 Years to 85 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
1
States / cities
Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Sep 29, 2025 · Synced Sep 13, 2026, 10:35 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A Phase 1 Study to Evaluate Effects of Sapropterin Dihydrochloride on QTc Intervals in Healthy Adult Subjects

NCT00789568
View directory record Official record
Conditions
Phenylketonuria
Interventions
sapropterin dihydrochloride, Moxifloxacin, Moxifloxacin placebo
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
18 Years to 50 Years
Enrollment
56 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2008 – 2009
U.S. locations
1
States / cities
Fargo, North Dakota
Source: ClinicalTrials.gov public record
Updated Jul 22, 2021 · Synced Sep 13, 2026, 10:35 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

The Brain and Neuropsychological Functioning in Adults With Sapropterin Dihydrochloride Treated Phenylketonuria

NCT02297347
View directory record Official record
Conditions
Phenylketonuria
Interventions
Not listed
Lead sponsor
Boston Children's Hospital
Other
Eligibility
18 Years to 55 Years
Enrollment
10 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2017
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Feb 4, 2018 · Synced Sep 13, 2026, 10:35 PM EDT
Recruiting No phase listed Observational

A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace)

NCT05813678
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Mar 24, 2026 · Synced Sep 13, 2026, 10:35 PM EDT
Terminated Not applicable Interventional

Moderate Intensity Exercise and Phenylketonuria

NCT01904708
View directory record Official record
Conditions
Phenylketonuria, PKU
Interventions
Acute Moderate Intensity Exercise
Other
Lead sponsor
Oregon Health and Science University
Other
Eligibility
14 Years to 17 Years · Male only
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2014
U.S. locations
1
States / cities
Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Oct 15, 2014 · Synced Sep 13, 2026, 10:35 PM EDT
Completed No phase listed Observational

Study to Evaluate the Effects of Kuvan on Individuals With Phenylketonuria (PKU) With Maladaptive Behaviors

NCT00728676
View directory record Official record
Conditions
Phenylketonuria
Interventions
Not listed
Lead sponsor
Shoji Yano
Other
Eligibility
18 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2010
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Jan 17, 2013 · Synced Sep 13, 2026, 10:35 PM EDT
Terminated No phase listed Observational

Behavioral Effects of Kuvan in Children With Mild Phenylketonuria

NCT00827762
View directory record Official record
Conditions
Phenylketonuria
Interventions
Kuvan
Drug
Lead sponsor
Washington University School of Medicine
Other
Eligibility
6 Years to 18 Years
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2010
U.S. locations
4
States / cities
Chicago, Illinois • Columbia, Missouri • St Louis, Missouri + 1 more
Source: ClinicalTrials.gov public record
Updated Dec 4, 2013 · Synced Sep 13, 2026, 10:35 PM EDT
Completed Phase 1 Interventional

ALlogeneic Cardiosphere-derived Stem Cells (CDCs) for Pulmonary Hypertension therApy

NCT03145298
View directory record Official record
Conditions
Pulmonary Arterial Hypertension (PAH)
Interventions
Allogeneic Human Cardiosphere-Derived Stem Cells, Placebo
Biological
Lead sponsor
Cedars-Sinai Medical Center
Other
Eligibility
18 Years to 75 Years
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2023
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Jun 11, 2023 · Synced Sep 13, 2026, 10:35 PM EDT
Completed Phase 2 Interventional

Efficacy and Safety of SYNB1618 and SYNB1934 in Adult Patients With Phenylketonuria

NCT04534842
View directory record Official record
Conditions
Phenylketonuria
Interventions
SYNB1618, SYNB1934
Drug
Lead sponsor
Synlogic
Industry
Eligibility
18 Years and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2022
U.S. locations
11
States / cities
Aurora, Colorado • Boca Raton, Florida • Gainesville, Florida + 7 more
Source: ClinicalTrials.gov public record
Updated Oct 16, 2022 · Synced Sep 13, 2026, 10:35 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Evaluation of Behavior, Executive Function, Neurotransmitter Function and Genomic Expression Kuvan Nonresponders

NCT01274026
View directory record Official record
Conditions
Phenylketonuria, Behavior and Behavior Mechanisms, PAH Gene Expression
Interventions
sapropterin dihydrochloride
Drug
Lead sponsor
Tulane University School of Medicine
Other
Eligibility
2 Years to 21 Years
Enrollment
21 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2011 – 2013
U.S. locations
1
States / cities
New Orleans, Louisiana
Source: ClinicalTrials.gov public record
Updated Aug 13, 2023 · Synced Sep 13, 2026, 10:35 PM EDT