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ClinicalTrials.gov public records Last synced Oct 14, 2026, 10:36 AM EDT

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Showing 409–432 of 501 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Lipid Metabolism, Inborn Errors Clear all
Completed Phase 3 Interventional

Pharmacokinetics, Safety and Tolerability of Zavesca (Miglustat) in Patients With Infantile Onset Gangliosidosis: Single and Steady State Oral Doses

NCT00672022
View directory record Official record
Conditions
GM2 Gangliosidoses, Tay-Sachs, Sandhoff Disease
Interventions
Zavesca (Miglustat)
Drug
Lead sponsor
Children's National Research Institute
Other
Eligibility
6 Months to 5 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2007
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated May 6, 2008 · Synced Oct 14, 2026, 10:36 AM EDT
Terminated Phase 2Phase 3 Interventional Results available

Adrabetadex to Treat Niemann-Pick Type C1 (NPC1) Disease

NCT04958642
View directory record Official record
Conditions
Niemann-Pick Disease, Type C
Interventions
Adrabetadex
Drug
Lead sponsor
Mandos LLC
Industry
Eligibility
4 Years to 21 Years
Enrollment
66 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2022
U.S. locations
15
States / cities
Birmingham, Alabama • Orange, California • San Francisco, California + 12 more
Source: ClinicalTrials.gov public record
Updated Aug 29, 2023 · Synced Oct 14, 2026, 10:36 AM EDT
Active, not recruiting No phase listed Observational Accepts healthy volunteers

Diffusion Tensor Imaging (DTI) in Infants With Krabbe Disease

NCT00787865
View directory record Official record
Conditions
Krabbe Disease
Interventions
Not listed
Lead sponsor
University of Pittsburgh
Other
Eligibility
Up to 17 Years
Enrollment
100 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2008 – 2027
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 6, 2026 · Synced Oct 14, 2026, 10:36 AM EDT
Completed Phase 3 Interventional

A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease

NCT00074971
View directory record Official record
Conditions
Fabry Disease
Interventions
Fabrazyme (agalsidase beta)
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
16 Years and older
Enrollment
58 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1999 – 2004
U.S. locations
14
States / cities
Los Angeles, California • San Francisco, California • Coral Springs, Florida + 11 more
Source: ClinicalTrials.gov public record
Updated Dec 4, 2013 · Synced Oct 14, 2026, 10:36 AM EDT
Recruiting Phase 3 Interventional

Long-term Safety and Tolerability of Inclisiran in Participants With HeFH or HoFH Who Have Completed the Pediatric ORION-16, ORION-13, ORION-20, or ORION-19 Studies

NCT05682378
View directory record Official record
Conditions
Heterozygous or Homozygous Familial Hypercholesterolemia
Interventions
Inclisiran
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
2 Years to 100 Years
Enrollment
195 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2032
U.S. locations
5
States / cities
Boca Raton, Florida • New York, New York • Cincinnati, Ohio + 2 more
Source: ClinicalTrials.gov public record
Updated Sep 18, 2026 · Synced Oct 14, 2026, 10:36 AM EDT
Completed Phase 2Phase 3 Interventional Results available

Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Sebelipase Alfa in Children With Growth Failure Due to Lysosomal Acid Lipase Deficiency

NCT01371825
View directory record Official record
Conditions
Lysosomal Acid Lipase Deficiency, Wolman Disease
Interventions
Sebelipase alfa (SBC-102)
Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
Up to 24 Months
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2018
U.S. locations
1
States / cities
Irvine, California
Source: ClinicalTrials.gov public record
Updated Jan 30, 2019 · Synced Oct 14, 2026, 10:36 AM EDT
Completed Phase 3 Interventional Results available

Efficacy and Safety Study of ISIS 301012 (Mipomersen) as Add-on in Familial Hypercholesterolemic Patients With Coronary Artery Disease

NCT00706849
View directory record Official record
Conditions
Heterozygous Familial Hypercholesterolemia, Coronary Artery Disease
Interventions
mipomersen sodium, placebo
Drug
Lead sponsor
Kastle Therapeutics, LLC
Industry
Eligibility
18 Years and older
Enrollment
124 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2010
U.S. locations
37
States / cities
La Jolla, California • Los Angeles, California • Mission Viejo, California + 31 more
Source: ClinicalTrials.gov public record
Updated Sep 9, 2016 · Synced Oct 14, 2026, 10:36 AM EDT
Completed Phase 2 Interventional Results available

A Study of the Efficacy and Safety of Enclitide Chloride (MK-0616 Oral PCSK9 Inhibitor) in Adults With Hypercholesterolemia (MK-0616-008)

NCT05261126
View directory record Official record
Conditions
Hypercholesterolemia, Familial Hypercholesterolemia
Interventions
Enclitide Chloride, Placebo
Drug
Lead sponsor
Merck Sharp & Dohme LLC
Industry
Eligibility
18 Years to 80 Years
Enrollment
381 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022
U.S. locations
20
States / cities
Beverly Hills, California • Sacramento, California • Santa Ana, California + 17 more
Source: ClinicalTrials.gov public record
Updated Dec 9, 2024 · Synced Oct 14, 2026, 10:36 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Caregiving Networks Across Disease Context and the Life Course

NCT05007990
View directory record Official record
Conditions
Inherited Metabolic Disorders, Undiagnosed Diseases, Batten's Disease, Tay Sachs, Diabetes
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
18 Years to 100 Years
Enrollment
2,800 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2030
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 15, 2026 · Synced Oct 14, 2026, 10:36 AM EDT
Active, not recruiting No phase listed Observational

Longitudinal Assessment of Atypical Tripeptidyl Peptidase 1 Enzyme Deficiency Patients

NCT04098211
View directory record Official record
Conditions
Neuronal Ceroid-Lipofuscinoses, Neuronal Ceroid Lipofuscinosis CLN2, Spinocerebellar Ataxia, Autosomal Recessive 7
Interventions
Not listed
Lead sponsor
Children's Hospital of Orange County
Other
Eligibility
4 Years and older
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2026
U.S. locations
1
States / cities
Orange, California
Source: ClinicalTrials.gov public record
Updated Apr 8, 2026 · Synced Oct 14, 2026, 10:36 AM EDT
Completed Phase 3 Interventional

A Study Comparing The Efficacy & Safety Of Torcetrapib/Atorvastatin And Atorvastatin In Subjects With High Triglycerides

NCT00134498
View directory record Official record
Conditions
Hypertriglyceridemia, Hyperlipoproteinemia Type IV
Interventions
torcetrapib/atorvastatin, atorvastatin
Drug
Lead sponsor
Pfizer
Industry
Eligibility
18 Years and older
Enrollment
160 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2006
U.S. locations
39
States / cities
Anaheim, California • Huntington Beach, California • Los Angeles, California + 36 more
Source: ClinicalTrials.gov public record
Updated Nov 16, 2007 · Synced Oct 14, 2026, 10:36 AM EDT
Completed No phase listed Observational

Clinical Studies of Progeria

NCT00094393
View directory record Official record
Conditions
Progeria
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
6 Months to 70 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2018
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Nov 15, 2019 · Synced Oct 14, 2026, 10:36 AM EDT
Completed No phase listed Observational

GM1 and GM2 Gangliosidosis PROspective Neurological Disease TrajectOry Study (PRONTO)

NCT05109793
View directory record Official record
Conditions
GM1 Gangliosidosis, Sandhoff Disease, Tay-Sachs Disease
Interventions
Not listed
Lead sponsor
Azafaros B.V.
Industry
Eligibility
2 Years to 20 Years
Enrollment
31 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
2
States / cities
Oakland, California • Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Sep 29, 2025 · Synced Oct 14, 2026, 10:36 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Androgenetic Alopecia in Fabry Disease

NCT01295008
View directory record Official record
Conditions
Fabry Disease
Interventions
Not listed
Lead sponsor
Baylor Research Institute
Other
Eligibility
18 Years to 64 Years · Male only
Enrollment
107 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2010 – 2015
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Jan 13, 2016 · Synced Oct 14, 2026, 10:36 AM EDT
Completed No phase listed Observational

Intravenous ACP-501 for Familial LCAT Deficiency (rhLCAT)

NCT04737720
View directory record Official record
Conditions
Familial LCAT Deficiency
Interventions
Recombinant LCAT
Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
18 Years to 100 Years
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2013
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 8, 2026 · Synced Oct 14, 2026, 10:36 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Dose-Ranging Study of ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy in Subjects With Fabry Disease (STAAR)

NCT04046224
View directory record Official record
Conditions
Fabry Disease
Interventions
ST-920
Biological
Lead sponsor
Sangamo Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2025
U.S. locations
9
States / cities
Irvine, California • Tampa, Florida • Atlanta, Georgia + 6 more
Source: ClinicalTrials.gov public record
Updated Apr 14, 2026 · Synced Oct 14, 2026, 10:36 AM EDT
Recruiting No phase listed Observational

Natural History of Sphingosine Phosphate Lyase Insufficiency Syndrome (SPLIS)

NCT06669949
View directory record Official record
Conditions
Sphingosine Phosphate Lyase Insufficiency Syndrome (SPLIS)
Interventions
no intervention
Other
Lead sponsor
University of California, San Francisco
Other
Eligibility
Not listed
Enrollment
28 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2030
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Aug 4, 2026 · Synced Oct 14, 2026, 10:36 AM EDT
Completed Phase 1 Interventional

Study of the Pharmacokinetics of Trappsol and Effects on Potential Biomarkers of Niemann-Pick C1 (NPC1)

NCT02939547
View directory record Official record
Conditions
Niemann-Pick Disease, Type C1
Interventions
Hydroxypropyl-beta-cyclodextrin
Drug
Lead sponsor
Cyclo Therapeutics, Inc.
Industry
Eligibility
18 Years and older
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2020
U.S. locations
2
States / cities
Oakland, California • Morristown, New Jersey
Source: ClinicalTrials.gov public record
Updated Feb 21, 2021 · Synced Oct 14, 2026, 10:36 AM EDT
Suspended Phase 1Phase 2 Interventional

Study of Safety, Tolerability and Efficacy of PBKR03 in Pediatric Subjects With Early Infantile Krabbe Disease

NCT04771416
View directory record Official record
Conditions
Leukodystrophy, Globoid Cell
Interventions
PBKR03
Biological
Lead sponsor
Gemma Biotherapeutics
Industry
Eligibility
1 Month to 9 Months
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2030
U.S. locations
4
States / cities
Chicago, Illinois • New York, New York • Philadelphia, Pennsylvania + 1 more
Source: ClinicalTrials.gov public record
Updated Jun 30, 2026 · Synced Oct 14, 2026, 10:36 AM EDT
Active, not recruiting Phase 3 Interventional

A Study to Evaluate the Effect of Venglustat Tablets on Left Ventricular Mass Index in Male and Female Adult Participants With Fabry Disease

NCT05280548
View directory record Official record
Conditions
Fabry Disease
Interventions
Venglustat (GZ402671), Agalsidase alfa, Agalsidase beta (GZ419828), Migalastat
Drug
Lead sponsor
Sanofi
Industry
Eligibility
18 Years to 65 Years
Enrollment
104 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
8
States / cities
Birmingham, Alabama • Los Angeles, California • Atlanta, Georgia + 5 more
Source: ClinicalTrials.gov public record
Updated Jun 24, 2026 · Synced Oct 14, 2026, 10:36 AM EDT
Completed Phase 4 Interventional Results available

Immunogenicity of Metreleptin in Patients With Generalized Lipodystrophy

NCT04026178
View directory record Official record
Conditions
Generalized Lipodystrophy
Interventions
Metreleptin
Drug
Lead sponsor
Aegerion Pharmaceuticals, Inc.
Industry
Eligibility
1 Year and older
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2024
U.S. locations
8
States / cities
Birmingham, Alabama • New Orleans, Louisiana • Ann Arbor, Michigan + 4 more
Source: ClinicalTrials.gov public record
Updated Nov 26, 2025 · Synced Oct 14, 2026, 10:36 AM EDT
Completed Phase 3 Interventional Results available

Acid Lipase Replacement Investigating Safety and Efficacy (ARISE) in Participants With Lysosomal Acid Lipase Deficiency

NCT01757184
View directory record Official record
Conditions
Lysosomal Acid Lipase Deficiency
Interventions
Sebelipase Alfa, Placebo
Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
4 Years and older
Enrollment
66 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2018
U.S. locations
11
States / cities
Tucson, Arizona • Palo Alto, California • San Francisco, California + 8 more
Source: ClinicalTrials.gov public record
Updated Dec 29, 2020 · Synced Oct 14, 2026, 10:36 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Cancer in Inherited Bone Marrow Failure Syndromes

NCT00027274
View directory record Official record
Conditions
Diamond Blackfan Anemia, Dyskeratosis Congenita, Fanconi Anemia, Shwachman Diamond Syndrome, Inherited Bone Marrow Failure Syndrome, Aplastic Anemia
Interventions
Not listed
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
1 Day to 100 Years
Enrollment
4,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 2001
U.S. locations
2
States / cities
Bethesda, Maryland • Rockville, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 8, 2026 · Synced Oct 14, 2026, 10:36 AM EDT
Recruiting Not applicable Interventional

Computerized Decision Support for Identification and Management of Familial Hypercholesterolemia

NCT06743659
View directory record Official record
Conditions
Familial Hypercholesterolaemia
Interventions
Alert-based computerized decision support
Device
Lead sponsor
Brigham and Women's Hospital
Other
Eligibility
Not listed
Enrollment
450 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Sep 29, 2026 · Synced Oct 14, 2026, 10:36 AM EDT