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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Amino Acid Metabolism, Inborn Errors Clear all
Recruiting Phase 1 Interventional

A Study of the c-Kit Specific Antibody-Drug Conjugate NN3201 for Advanced and/or Metastatic Solid Tumors Known to Express c-Kit

NCT06805825
View directory record Official record
Conditions
Gastrointestinal Stromal Tumors, Small-cell Lung Cancer, Adenoid Cystic Carcinoma, Uveal Melanoma, Neuroendocrine Tumors, Chromophobe Renal Cell Carcinoma, Clear Cell Renal Cell Carcinoma
Interventions
NN3201
Drug
Lead sponsor
Novelty Nobility, Inc.
Industry
Eligibility
18 Years and older
Enrollment
67 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
5
States / cities
Ann Arbor, Michigan • Cleveland, Ohio • Portland, Oregon + 2 more
Source: ClinicalTrials.gov public record
Updated Sep 22, 2025 · Synced Jul 23, 2026, 2:24 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 Jul 23, 2026, 2:24 PM EDT
Completed No phase listed Observational

Bone Mineral Density in Adults With Hyperphenylalaninemia

NCT01209819
View directory record Official record
Conditions
Phenylketonurias
Interventions
Not listed
Lead sponsor
The University of Texas Health Science Center, Houston
Other
Eligibility
18 Years and older
Enrollment
28 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2012
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jun 11, 2015 · Synced Jul 23, 2026, 2:24 PM EDT
Completed Phase 1 Interventional

Effect of S-adenosylmethionine (SAMe) on Blood Levels of Homocysteine

NCT00473200
View directory record Official record
Conditions
Hyperhomocysteinemia
Interventions
S-adenosylmethionine
Dietary Supplement
Lead sponsor
Baylor Research Institute
Other
Eligibility
18 Years and older
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2013
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Mar 13, 2018 · Synced Jul 23, 2026, 2:24 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 Jul 23, 2026, 2:24 PM EDT
Completed Phase 2 Interventional Results available

Study of the Safety and Tolerability of HPN-100 Compared to Sodium Phenylbutyrate in Children With Urea Cycle Disorders

NCT00947544
View directory record Official record
Conditions
Urea Cycle Disorders
Interventions
HPN-100, NaPBA
Drug
Lead sponsor
Amgen
Industry
Eligibility
6 Years to 17 Years
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2011
U.S. locations
7
States / cities
Los Angeles, California • Washington D.C., District of Columbia • New York, New York + 4 more
Source: ClinicalTrials.gov public record
Updated Jul 10, 2024 · Synced Jul 23, 2026, 2:24 PM EDT
Completed Phase 1 Interventional

Phase 1b Study to Assess Safety, Tolerability, and Pharmacokinetics of ARCT-810 in Stable Adult Subjects With Ornithine Transcarbamylase Deficiency

NCT04442347
View directory record Official record
Conditions
Ornithine Transcarbamylase Deficiency
Interventions
ARCT-810, Placebo
Biological · Other
Lead sponsor
Arcturus Therapeutics, Inc.
Industry
Eligibility
18 Years and older
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2024
U.S. locations
8
States / cities
Gainesville, Florida • Minneapolis, Minnesota • New York, New York + 5 more
Source: ClinicalTrials.gov public record
Updated Aug 1, 2024 · Synced Jul 23, 2026, 2:24 PM EDT
Completed Phase 2Phase 3 Interventional Results available

Phenylbutyrate Therapy for Maple Syrup Urine Disease

NCT01529060
View directory record Official record
Conditions
Maple Syrup Urine Disease
Interventions
Phenylbutyrate, Placebo powder
Drug
Lead sponsor
Brendan Lee
Other
Eligibility
3 Years and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2017
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Mar 18, 2019 · Synced Jul 23, 2026, 2:24 PM EDT
Recruiting No phase listed Observational

Orphan Europe Carbaglu® Surveillance Protocol

NCT03409003
View directory record Official record
Conditions
N-acetylglutamate Synthase (NAGS) Deficiency
Interventions
Not listed
Lead sponsor
Nicholas Ah Mew
Other
Eligibility
Not listed
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2027
U.S. locations
3
States / cities
Washington D.C., District of Columbia • Boston, Massachusetts • New York, New York
Source: ClinicalTrials.gov public record
Updated Feb 6, 2024 · Synced Jul 23, 2026, 2:24 PM EDT
Completed Phase 4 Interventional Results available

The Effectiveness of Kuvan in Amish PKU Patients

NCT02677870
View directory record Official record
Conditions
Phenylketonuria
Interventions
saproterin dihydrochloride, Diet treatment
Drug · Other
Lead sponsor
University Hospitals Cleveland Medical Center
Other
Eligibility
2 Years to 60 Years
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2019
U.S. locations
1
States / cities
Cleveland, Ohio
Source: ClinicalTrials.gov public record
Updated Apr 26, 2022 · Synced Jul 23, 2026, 2:24 PM EDT
Completed Phase 4 Interventional Results available

Effects of Kuvan on Melatonin Secretion

NCT01617070
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Nov 17, 2016 · Synced Jul 23, 2026, 2:24 PM EDT
Recruiting No phase listed Observational

Systemic Biomarkers of Brain Injury From Hyperammonemia

NCT04602325
View directory record Official record
Conditions
Urea Cycle Disorder, Organic Acidemia, Maple Syrup Urine Disease, Glutaric Acidemia I, Fatty Acid Oxidation Disorder, Hypoxic-Ischemic Encephalopathy
Interventions
Not listed
Lead sponsor
Children's National Research Institute
Other
Eligibility
7 Years to 18 Years
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2027
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Feb 6, 2024 · Synced Jul 23, 2026, 2:24 PM EDT
Completed Phase 2 Interventional Results available

A Study to Evaluate Subcutaneously Administered rAvPAL-PEG in Patients With Phenylketonuria for 24 Weeks

NCT01560286
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Aug 5, 2019 · Synced Jul 23, 2026, 2:24 PM EDT
Completed Phase 2 Interventional Results available

Dose-Finding Study to Evaluate the Safety, Efficacy, & Tolerability of Multiple Doses of rAvPAL-PEG in Subjects With PKU

NCT00925054
View directory record Official record
Conditions
Phenylketonuria
Interventions
rAvPAL-PEG 0.001 mg/kg, rAvPAL-PEG 0.003 mg/kg, rAvPAL-PEG 0.01 mg/kg, rAvPAL-PEG 0.03 mg/kg, rAvPAL-PEG 0.1 mg/kg
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
16 Years to 55 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2015
U.S. locations
11
States / cities
Aurora, Colorado • Decatur, Georgia • Chicago, Illinois + 8 more
Source: ClinicalTrials.gov public record
Updated Feb 25, 2019 · Synced Jul 23, 2026, 2:24 PM EDT
Completed Phase 3 Interventional Results available

Study of Phenoptin to Increase Phenylalanine Tolerance in Phenylketonuric Children on a Phenylalanine-restricted Diet

NCT00272792
View directory record Official record
Conditions
Phenylketonurias
Interventions
Sapropterin Dihydrochloride, Placebo
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
4 Years to 12 Years
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006
U.S. locations
9
States / cities
Los Angeles, California • Oakland, California • Sacramento, California + 6 more
Source: ClinicalTrials.gov public record
Updated Aug 12, 2015 · Synced Jul 23, 2026, 2:24 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

The Effects of Kuvan on Functional Brain Connectivity in Individuals With Phenylketonuria (PKU)

NCT00964236
View directory record Official record
Conditions
Phenylketonuria
Interventions
Sapropterin
Drug
Lead sponsor
University of Missouri-Columbia
Other
Eligibility
6 Years and older
Enrollment
20 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2011
U.S. locations
1
States / cities
Columbia, Missouri
Source: ClinicalTrials.gov public record
Updated Jan 3, 2018 · Synced Jul 23, 2026, 2:24 PM EDT
Completed Not applicable Interventional Results available

The Brain, Neurological Features and Neuropsychological Functioning in Adults With Phenylketonuria: A Pilot Study

NCT01917344
View directory record Official record
Conditions
Phenylketonuria
Interventions
MRI
Other
Lead sponsor
Boston Children's Hospital
Other
Eligibility
18 Years to 55 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2014
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jun 18, 2015 · Synced Jul 23, 2026, 2:24 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 Jul 23, 2026, 2:24 PM EDT
Completed Phase 3 Interventional Results available

A Study of PTC923 in Participants With Phenylketonuria

NCT05099640
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jan 9, 2024 · Synced Jul 23, 2026, 2:24 PM EDT
Active, not recruiting No phase listed Observational

Long Term Follow Up to Evaluate DTX301 in Adults With Late-Onset OTC Deficiency

NCT03636438
View directory record Official record
Conditions
Ornithine Transcarbamylase (OTC) Deficiency
Interventions
No Intervention
Other
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
18 Years and older
Enrollment
11 participants
Timeline
2018 – 2029
U.S. locations
4
States / cities
Aurora, Colorado • Boston, Massachusetts • New York, New York + 1 more
Source: ClinicalTrials.gov public record
Updated May 31, 2026 · Synced Jul 23, 2026, 2:24 PM EDT
Completed Phase 4 Interventional Results available

A Study of the Safety, Efficacy and Pharmacokinetics of Glycerol Phenylbutyrate in Pediatric Subjects Under 2 Years of Age With Urea Cycle Disorders

NCT02246218
View directory record Official record
Conditions
Urea Cycle Disorder
Interventions
RAVICTI
Drug
Lead sponsor
Amgen
Industry
Eligibility
1 Day to 2 Years
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2017
U.S. locations
16
States / cities
Los Angeles, California • Palo Alto, California • Washington D.C., District of Columbia + 13 more
Source: ClinicalTrials.gov public record
Updated Jun 30, 2024 · Synced Jul 23, 2026, 2:24 PM EDT
Recruiting No phase listed Observational

Clinical and Basic Investigations Into Hermansky-Pudlak Syndrome

NCT00001456
View directory record Official record
Conditions
Hermansky-Pudlak Syndrome (HPS)
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
1 Month to 115 Years
Enrollment
600 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1995
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 16, 2026 · Synced Jul 23, 2026, 2:24 PM EDT
Completed Phase 3 Interventional

Preventing Cytomegalovirus (CMV) Organ Damage With Valganciclovir in People With HIV

NCT00006145
View directory record Official record
Conditions
Cytomegalovirus Infections, HIV Infections
Interventions
Valganciclovir
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
13 Years and older
Enrollment
350 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2006
U.S. locations
56
States / cities
Birmingham, Alabama • Los Angeles, California • Palo Alto, California + 33 more
Source: ClinicalTrials.gov public record
Updated Oct 31, 2021 · Synced Jul 23, 2026, 2:24 PM EDT
Terminated No phase listed Observational

Adeno-Associated Virus (AAV) Antibody Study in Subjects OTC Deficiency, GSDIa, and Wilson Disease

NCT04909346
View directory record Official record
Conditions
Ornithine Transcarbamylase Deficiency, Wilson Disease, Glycogen Storage Disease Type IA
Interventions
Not listed
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
18 Years and older
Enrollment
51 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2022
U.S. locations
1
States / cities
Akron, Ohio
Source: ClinicalTrials.gov public record
Updated Jun 11, 2023 · Synced Jul 23, 2026, 2:24 PM EDT