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ClinicalTrials.gov public records Last synced Oct 16, 2026, 4:45 AM EDT

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

Showing 793–816 of 1,608 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Metabolism, Inborn Errors Clear all
Active, not recruiting No phase listed Observational

Cerliponase Alfa Observational Study in the US

NCT04476862
View directory record Official record
Conditions
Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2
Interventions
Cerliponase Alfa, Administration Kit
Drug · Device
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
Not listed
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2030
U.S. locations
16
States / cities
Little Rock, Arkansas • Orange, California • Aurora, Colorado + 12 more
Source: ClinicalTrials.gov public record
Updated Jun 30, 2026 · Synced Oct 16, 2026, 4:45 AM EDT
Completed Phase 2 Interventional

Evaluate the PK, PD, and Safety of Arhalofenate in Combination With Febuxostat for Hyperuricemia in Patients With Gout

NCT02252835
View directory record Official record
Conditions
Gout, Hyperuricemia
Interventions
Arhalofenate, Febuxostat, Colchicine
Drug
Lead sponsor
Gilead Sciences
Industry
Eligibility
18 Years to 75 Years
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014
U.S. locations
1
States / cities
Overland Park, Kansas
Source: ClinicalTrials.gov public record
Updated Sep 18, 2026 · Synced Oct 16, 2026, 4:45 AM EDT
Completed Phase 2 Interventional Results available

Trial of L-DOPA as a Treatment to Improve Vision in Albinism

NCT01176435
View directory record Official record
Conditions
Albinism
Interventions
Levodopa, Placebo
Drug
Lead sponsor
University of Minnesota
Other
Eligibility
3 Years to 60 Years
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2014
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated May 2, 2018 · Synced Oct 16, 2026, 4:45 AM EDT
Recruiting Not applicable Interventional

The ORIGIN-FH Study

NCT07470723
View directory record Official record
Conditions
Heterozygous Familial Hypercholesterolemia (HeFH), Homozygous Familial Hypercholesterolemia (HoFH), Familial Hypercholesterolemia
Interventions
Screening for FH
Diagnostic Test
Lead sponsor
University of Wisconsin, Madison
Other
Eligibility
18 Years and older
Enrollment
70 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2029
U.S. locations
1
States / cities
Madison, Wisconsin
Source: ClinicalTrials.gov public record
Updated Oct 13, 2026 · Synced Oct 16, 2026, 4:45 AM EDT
Completed Not applicable Interventional

Open Label Ganciclovir Therapy for Sight- or Life-Threatening Cytomegalovirus Disease in the Immunocompromised Patient

NCT00002025
View directory record Official record
Conditions
Cytomegalovirus Infections, Cytomegalovirus Retinitis, HIV Infections
Interventions
Ganciclovir
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
3 Months and older
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
1
States / cities
Palo Alto, California
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 16, 2026, 4:45 AM EDT
Completed Not applicable Interventional

Safety and Effectiveness of Resistance Exercise Training in Patients With Pompe Disease.

NCT02654886
View directory record Official record
Conditions
Pompe Disease, Muscle Weakness
Interventions
Resistance Exercise Training
Other
Lead sponsor
University of California, Irvine
Other
Eligibility
16 Years to 75 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2019
U.S. locations
1
States / cities
Irvine, California
Source: ClinicalTrials.gov public record
Updated Sep 26, 2019 · Synced Oct 16, 2026, 4:45 AM EDT
Available No phase listed Expanded access

Post-trial Access Program of Idursulfase-IT Along With Elaprase in Children With Hunter Syndrome

NCT05795361
View directory record Official record
Conditions
Hunter Syndrome
Interventions
Idursulfase-IT
Drug
Lead sponsor
Takeda
Industry
Eligibility
Male only
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
17
States / cities
Phoenix, Arizona • Oakland, California • Wilmington, Delaware + 14 more
Source: ClinicalTrials.gov public record
Updated Apr 13, 2026 · Synced Oct 16, 2026, 4:45 AM EDT
Terminated Phase 2 Interventional Results available

A Study to Examine the Effects of the Leptin Receptor (LEPR) Agonist Antibody REGN4461 in Adult Patients With Familial Partial Lipodystrophy (FPLD)

NCT05088460
View directory record Official record
Conditions
Familial Partial Lipodystrophy, Metabolic Abnormalities
Interventions
REGN4461, Matching Placebo
Drug
Lead sponsor
Regeneron Pharmaceuticals
Industry
Eligibility
18 Years and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
5
States / cities
Boca Raton, Florida • Bethesda, Maryland • Ann Arbor, Michigan + 2 more
Source: ClinicalTrials.gov public record
Updated Oct 16, 2025 · Synced Oct 16, 2026, 4:45 AM EDT
Active, not recruiting No phase listed Observational

Analysis of Specimens From Individuals With Pulmonary Fibrosis

NCT00084305
View directory record Official record
Conditions
Pulmonary Fibrosis, Healthy Volunteers, Hermansky-Pudlak Syndrome (HPS)
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
18 Years to 115 Years
Enrollment
315 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2004
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 13, 2026 · Synced Oct 16, 2026, 4:45 AM EDT
Completed No phase listed Observational

Study of Megakaryocytes From Patients With Abnormal Platelet Vesicles

NCT00086476
View directory record Official record
Conditions
Blood Coagulation Disorders
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
2 Years to 80 Years
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2011
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 2, 2017 · Synced Oct 16, 2026, 4:45 AM EDT
Completed Phase 2 Interventional

A Phase 2 Study of JNT-517 in Adolescent Participants With Phenylketonuria

NCT06637514
View directory record Official record
Conditions
Phenylketonuria (PKU)
Interventions
JNT-517 Tablet, Placebo
Drug · Other
Lead sponsor
Otsuka Pharmaceutical Development & Commercialization, Inc.
Industry
Eligibility
12 Years to 17 Years
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2026
U.S. locations
6
States / cities
Gainesville, Florida • Tampa, Florida • Portland, Oregon + 3 more
Source: ClinicalTrials.gov public record
Updated May 13, 2026 · Synced Oct 16, 2026, 4:45 AM EDT
Enrolling by invitation No phase listed Observational Accepts healthy volunteers

Genetic Studies of Lysosomal Storage Disorders

NCT00001215
View directory record Official record
Conditions
Lysosomal Storage Disorders, Gaucher Disease, Parkinson Disease
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
1 Week to 110 Years
Enrollment
1,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 1995
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 22, 2026 · Synced Oct 16, 2026, 4:45 AM EDT
Completed Phase 3 Interventional Results available

Clinical Study of Aldurazyme in Patients With Mucopolysaccharidosis (MPS) I

NCT00912925
View directory record Official record
Conditions
Mucopolysaccharidosis I, Hurlers Syndrome, Hurler-Scheie Syndrome
Interventions
rhIDU (recombinant human-Alpha-L-Iduronidase), Placebo
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
5 Years and older
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2001
U.S. locations
2
States / cities
New York, New York • Chapel Hill, North Carolina
Source: ClinicalTrials.gov public record
Updated Apr 7, 2015 · Synced Oct 16, 2026, 4:45 AM EDT
Completed Phase 3 Interventional Results available

A Study to Evaluate Lumasiran in Children and Adults With Primary Hyperoxaluria Type 1

NCT03681184
View directory record Official record
Conditions
Primary Hyperoxaluria Type 1 (PH1)
Interventions
Placebo, Lumasiran
Drug
Lead sponsor
Alnylam Pharmaceuticals
Industry
Eligibility
6 Years and older
Enrollment
39 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2024
U.S. locations
5
States / cities
San Diego, California • Jacksonville, Florida • Rochester, Minnesota + 2 more
Source: ClinicalTrials.gov public record
Updated Aug 12, 2024 · Synced Oct 16, 2026, 4:45 AM 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 16, 2014 · Synced Oct 16, 2026, 4:45 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Safety and Dose-Finding Study of DTX401 (AAV8G6PC) in Adults With Glycogen Storage Disease Type Ia (GSDIa)

NCT03517085
View directory record Official record
Conditions
GSD1
Interventions
DTX401, steroid regimen
Genetic · Drug
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
18 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
3
States / cities
Farmington, Connecticut • Ann Arbor, Michigan • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Nov 18, 2022 · Synced Oct 16, 2026, 4:45 AM EDT
Terminated Phase 2Phase 3 Interventional Results available

The BROADEN Study: A Study of Volanesorsen (Formerly IONIS-APOCIIIRx) in Participants With Familial Partial Lipodystrophy

NCT02527343
View directory record Official record
Conditions
Familial Partial Lipodystrophy
Interventions
volanesorsen, Placebo
Drug
Lead sponsor
Akcea Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2019
U.S. locations
6
States / cities
Ann Arbor, Michigan • Rochester, Minnesota • St Louis, Missouri + 3 more
Source: ClinicalTrials.gov public record
Updated Oct 18, 2021 · Synced Oct 16, 2026, 4:45 AM EDT
Completed Phase 3 Interventional Results available

Study of the Safety and Efficacy of PRX-102 Compared to Agalsidase Beta on Renal Function

NCT02795676
View directory record Official record
Conditions
Fabry Disease
Interventions
PRX-102 (pegunigalsidase alfa), agalsidase beta
Biological
Lead sponsor
Protalix
Industry
Eligibility
18 Years to 60 Years
Enrollment
78 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2022
U.S. locations
15
States / cities
Birmingham, Alabama • Phoenix, Arizona • Orange, California + 11 more
Source: ClinicalTrials.gov public record
Updated Sep 13, 2023 · Synced Oct 16, 2026, 4:45 AM EDT
Completed Not applicable Interventional

A Collection of Case Studies in Infants With UCD to Evaluate Infant Growth and the Safety of a New Medical Food for UCD

NCT03911089
View directory record Official record
Conditions
Urea Cycle Disorder, Inborn Errors of Metabolism
Interventions
UCD Anamix Infant
Other
Lead sponsor
Nutricia Research
Industry
Eligibility
Up to 30 Days
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2019
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Dec 9, 2021 · Synced Oct 16, 2026, 4:45 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Analysis of the Nervous System in Patients With Fabry's Disease

NCT00001491
View directory record Official record
Conditions
Fabry's Disease
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Not listed
Enrollment
325 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
1995 – 2008
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 2, 2017 · Synced Oct 16, 2026, 4:45 AM EDT
Terminated Phase 1 Interventional Accepts healthy volunteers

Response to Phenylketonuria to Tetrahydrobiopterin (BH4)

NCT00244218
View directory record Official record
Conditions
Phenylketonuria
Interventions
tetrahydrobiopterin (BH4)
Drug
Lead sponsor
The University of Texas Medical Branch, Galveston
Other
Eligibility
10 Years and older
Enrollment
57 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2005 – 2015
U.S. locations
1
States / cities
Galveston, Texas
Source: ClinicalTrials.gov public record
Updated Sep 21, 2021 · Synced Oct 16, 2026, 4:45 AM EDT
Terminated No phase listed Observational

Study to Detect Unrecognized Mucopolysaccharidosis in Children Visiting Rheumatology, Hand or Skeletal Dysplasia Clinics

NCT01675674
View directory record Official record
Conditions
Mucopolysaccharidoses, Mucopolysaccharidosis I, Mucopolysaccharidosis II, Mucopolysaccharidosis IV, Mucopolysaccharidosis VI
Interventions
Dried blood spot test for MPS
Other
Lead sponsor
National MPS Society
Other
Eligibility
6 Months to 18 Years
Enrollment
3,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2014
U.S. locations
2
States / cities
New Brunswick, New Jersey • New York, New York
Source: ClinicalTrials.gov public record
Updated May 24, 2013 · Synced Oct 16, 2026, 4:45 AM 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 23, 2021 · Synced Oct 16, 2026, 4:45 AM EDT
Recruiting No phase listed Observational

Artificial Intelligence Guided Echocardiographic Screening of Rare Diseases (EchoNet-Screening)

NCT05139797
View directory record Official record
Conditions
Cardiac Amyloidosis
Interventions
EchoNet-LVH screening for cardiac amyloidosis
Other
Lead sponsor
Cedars-Sinai Medical Center
Other
Eligibility
18 Years and older
Enrollment
300 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2027
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Jul 22, 2026 · Synced Oct 16, 2026, 4:45 AM EDT