Search | Clinical Trials Finder

These results were retrieved from the live ClinicalTrials.gov registry. Filters and sort order apply to this result list.

ClinicalTrials.gov public records Last synced Oct 11, 2026, 4:52 AM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Showing 97–120 of 260 matching trials from the live ClinicalTrials.gov search.

Filters: Condition: Amino Acid Metabolism, Inborn Errors Clear all

Conditions: Cytomegalovirus Infections, HIV Infections
Interventions: Zidovudine, Didanosine, Ganciclovir; Drug
Lead sponsor: Hoffmann-La Roche; Industry
Eligibility: 18 Years and older
Enrollment: 24 participants
Healthy volunteers: Healthy volunteers not accepted
U.S. locations: 3
States / cities: Irvine, California • Washington D.C., District of Columbia • Galveston, Texas

Conditions: Cytomegalovirus Retinitis, HIV Infections
Interventions: Sevirumab; Drug
Lead sponsor: Sandoz; Industry
Eligibility: 18 Years and older
Healthy volunteers: Healthy volunteers not accepted
U.S. locations: 2
States / cities: Boston, Massachusetts • Galveston, Texas

Conditions: Hermansky Pudlak Syndrome
Interventions: Pulmonary function test, Chest CT, Sample collection; Other
Lead sponsor: Vanderbilt University; Other
Eligibility: 12 Years to 90 Years
Enrollment: 55 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2019
U.S. locations: 5
States / cities: Maywood, Illinois • Boston, Massachusetts • New York, New York + 2 more

Conditions: Phenylketonuria, Maternal
Interventions: Pegvaliase; Drug
Lead sponsor: BioMarin Pharmaceutical; Industry
Eligibility: Female only
Enrollment: 50 participants
Timeline: 2022 – 2032
U.S. locations: 1
States / cities: Wilmington, North Carolina

Conditions: Phenylketonuria
Interventions: Kuvan, Meal Challenge; Drug · Other
Lead sponsor: Emory University; Other
Eligibility: 10 Years to 45 Years
Enrollment: 12 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2011 – 2013
U.S. locations: 1
States / cities: Decatur, Georgia

Conditions: ARG1 Deficiency
Interventions: Loargys Arginine Test System; Device
Lead sponsor: Immedica Pharma US Inc; Industry
Eligibility: 0 Years to 100 Years
Enrollment: 100 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2027
U.S. locations: 1
States / cities: Atlanta, Georgia

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

Conditions: Phenylketonuria
Interventions: Pegvaliase, Methotrexate; Biological · Drug
Lead sponsor: BioMarin Pharmaceutical; Industry
Eligibility: 18 Years to 65 Years
Enrollment: 12 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2027
U.S. locations: 12
States / cities: Los Angeles, California • Aurora, Colorado • Tampa, Florida + 9 more

Conditions: Cytomegalovirus Infections, HIV Infections
Interventions: Ganciclovir; Drug
Lead sponsor: National Institute of Allergy and Infectious Diseases (NIAID); NIH
Eligibility: 14 Days to 20 Years
Enrollment: 32 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: Ends 1998
U.S. locations: 18
States / cities: Los Angeles, California • Oakland, California • San Diego, California + 10 more

Conditions: Cytomegalovirus Infections
Interventions: Valganciclovir, Ganciclovir; Drug
Lead sponsor: National Institute of Allergy and Infectious Diseases (NIAID); NIH
Eligibility: Up to 30 Days
Enrollment: 24 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2002 – 2007
U.S. locations: 21
States / cities: Birmingham, Alabama • Little Rock, Arkansas • Los Angeles, California + 18 more

Conditions: Endothelial Dysfunction
Interventions: Sapropterin Dihydrochloride, Sapropterin Dihydrochloride and Vitamin C; Drug
Lead sponsor: BioMarin Pharmaceutical; Industry
Eligibility: 18 Years to 75 Years
Enrollment: 52 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2007 – 2009
U.S. locations: 1
States / cities: Hackensack, New Jersey

Conditions: OTC Deficiency
Interventions: DTX301, Placebo, Oral Corticosteroids, Placebo for oral corticosteroids, Sodium Acetate; Genetic · Other · Drug
Lead sponsor: Ultragenyx Pharmaceutical Inc; Industry
Eligibility: 12 Years and older
Enrollment: 37 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2031
U.S. locations: 4
States / cities: Los Angeles, California • Aurora, Colorado • Chicago, Illinois + 1 more

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

Conditions: Cytomegalovirus Infections, HIV Infections
Interventions: Ganciclovir; Drug
Lead sponsor: Hoffmann-La Roche; Industry
Eligibility: 13 Years and older
Enrollment: 700 participants
Healthy volunteers: Healthy volunteers not accepted
U.S. locations: 19
States / cities: Berkeley, California • Los Angeles, California • San Diego, California + 12 more

Conditions: Schizophrenia, Psychotic Disorders
Interventions: Glycine administration; Dietary Supplement
Lead sponsor: Mclean Hospital; Other
Eligibility: 18 Years to 55 Years
Enrollment: 21 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2010 – 2013
U.S. locations: 1
States / cities: Belmont, Massachusetts

Conditions: Oculocutaneous Albinism
Interventions: Not listed
Lead sponsor: National Human Genome Research Institute (NHGRI); NIH
Eligibility: 1 Year to 80 Years
Enrollment: 206 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2008 – 2019
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Homocystinuria
Interventions: SYNB1353; Drug
Lead sponsor: Synlogic; Industry
Eligibility: 18 Years to 64 Years
Enrollment: 31 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2022
U.S. locations: 1
States / cities: High Point, North Carolina

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

Conditions: Idiopathic Pulmonary Fibrosis
Interventions: No intervention; Other
Lead sponsor: 23andMe, Inc.; Industry
Eligibility: 18 Years and older
Enrollment: 690 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2023
U.S. locations: 1
States / cities: Sunnyvale, California

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

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

Conditions: Urea Cycle Disorders, Carbamoyl-Phosphate Synthase I Deficiency
Interventions: LNP.UCD.ABE; Biological
Lead sponsor: Rebecca Ahrens-Nicklas; Other
Eligibility: 24 Hours to 5 Years
Enrollment: 7 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2028
U.S. locations: 1
States / cities: Philadelphia, Pennsylvania

Conditions: Colitis, HIV Infections
Interventions: Ganciclovir; Drug
Lead sponsor: Hoffmann-La Roche; Industry
Eligibility: 21 Years and older · Male only
Healthy volunteers: Healthy volunteers not accepted
U.S. locations: 1
States / cities: Palo Alto, California

Conditions: Cytomegalovirus Retinitis, HIV Infections
Interventions: Aldesleukin; Drug
Lead sponsor: Chiron Corporation; Industry
Eligibility: 18 Years and older
Healthy volunteers: Healthy volunteers not accepted
U.S. locations: 1
States / cities: Toledo, Ohio

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A Phase I Pharmacokinetic Study in HIV-Positive Subjects of Oral Ganciclovir and Concomitant Antiretroviral Zidovudine and Didanosine

A Phase I/II Trial to Assess the Safety and Tolerance of Escalating Doses of a Human Anti-Cytomegalovirus Monoclonal Antibody (SDZ MSL-109) in Patients With the Acquired Immunodeficiency Syndrome and CMV Retinitis

A Longitudinal Study of Hermansky-Pudlak Syndrome Pulmonary Fibrosis

A Global, Multicenter Study to Assess Maternal, Fetal and Infant Outcomes of Exposure to Palynziq® (Pegvaliase) During Pregnancy and Breastfeeding

Phenylketonuria, Oxidative Stress, and BH4

Evaluation of the Safety of Loargys Arginine Test System in Loargys-treated Patients

A Phase 3 Substudy to Evaluate Executive Function in Adults With PKU Who Are Participating in the Phase 3 Study, 165-302

Immune Modulation During Palynziq® Treatment in Adults (IMPALA)

A Pharmacokinetic and Tolerance Study of Oral Ganciclovir in HIV-Infected Children With Asymptomatic Cytomegalovirus Infection and Low CD4 Cell Counts or Quiescent Cytomegalovirus Disease

Valganciclovir in Congenital CMV Infants

A Phase 2, Pharmacokinetic (PK) Study of 6R-BH4 Alone or 6R-BH4 With Vitamin C in Subjects With Endothelial Dysfunction

Clinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC) Deficiency

Safety and Efficacy of Self Administered Injections of Pegvaliase (>40mg/Day Dose) in Adults With PKU

A Randomized, Double-Blind Study of the Efficacy and Safety of Oral Ganciclovir for the Prevention of CMV Disease in People Infected With the Human Immunodeficiency Virus

Acute Glycine Pharmacodynamic Study

Clinical, Cellular, and Molecular Investigation Into Oculocutaneous Albinism

Safety, Tolerability and Pharmacodynamics of SYNB1353 in Healthy Adult Volunteers

Clinical and Basic Investigations Into Hermansky-Pudlak Syndrome

23andMe IPF Research Study

Orphan Europe Carbaglu® Surveillance Protocol

Moderate Intensity Exercise and Phenylketonuria

Study of LNP.UCD.ABE in Patients With Urea Cycle Disorders

A Study of Ganciclovir in the Treatment of Cytomegalovirus of the Large Intestine in Patients With AIDS

A Phase I Study of the Safety of Proleukin (Aldesleukin) in Combination With Ganciclovir and Antiretroviral Therapy in HIV Seropositive Patients With Cytomegalovirus (CMV) Retinitis