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ClinicalTrials.gov public records Last synced Jul 22, 2026, 4:48 AM EDT

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Showing 1–24 of 501 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
Filters: Condition: Lipid Metabolism, Inborn Errors Clear all
Completed Phase 3 Interventional Results available

PLUTO: Pediatric Lipid-redUction Trial of rOsuvastatin

NCT00355615
View directory record Official record
Conditions
Familial Hypercholesterolemia
Interventions
Rosuvastatin, Placebo
Drug
Lead sponsor
AstraZeneca
Industry
Eligibility
10 Years to 17 Years
Enrollment
173 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2008
U.S. locations
4
States / cities
Los Angeles, California • Hyde Park, New York • Cincinnati, Ohio + 1 more
Source: ClinicalTrials.gov public record
Updated Aug 30, 2011 · Synced Jul 22, 2026, 4:48 AM EDT
No Longer Available No phase listed Expanded access

Compassionate Use of Triheptanoin (C7) for Inherited Disorders of Energy Metabolism

NCT01461304
View directory record Official record
Conditions
Very Long-chain acylCoA Dehydrogenase (VLCAD) Deficiency, Carnitine Palmitoyltransferase Deficiencies (CPT1, CPT2), Mitochondrial Trifunctional Protein Deficiency, Long-chain Hydroxyacyl-CoA Dehydrogenase Deficiency, Glycogen Storage Disorders, Pyruvate Carboxylase Deficiency Disease, ACYL-CoA DEHYDROGENASE FAMILY, MEMBER 9, DEFICIENCY of, Barth Syndrome
Interventions
triheptanoin
Drug
Lead sponsor
Jerry Vockley, MD, PhD
Other
Eligibility
1 Month and older
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Dec 9, 2021 · Synced Jul 22, 2026, 4:48 AM EDT
Recruiting No phase listed Observational

NYSCF Scientific Discovery Biobank

NCT06203106
View directory record Official record
Conditions
ALS, Amyotrophic Lateral Sclerosis, Alzheimer Disease, Alzheimer Disease, Early Onset, Alzheimer Disease, Late Onset, Batten Disease, Corticobasal Degeneration, Dementia, Frontotemporal Dementia, Huntington Disease, Lewy Body Disease, Multiple Sclerosis, Multiple System Atrophy, Parkinson Disease, Parkinson's Disease and Parkinsonism, Progressive Supranuclear Palsy, INAD, Diabetes, Diabetes Mellitus, Diabetes Mellitus, Type 2, Diabetes Mellitus, Type 1, Macular Degeneration, Ovarian Cancer, Cervical Cancer, Uterine Cancer, Vaginal Cancer, Vulvar Cancer, PTSD, Post Traumatic Stress Disorder
Interventions
Biological Sample Collection
Other
Lead sponsor
New York Stem Cell Foundation Research Institute
Other
Eligibility
30 Days and older
Enrollment
10,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2045
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Mar 2, 2025 · Synced Jul 22, 2026, 4:48 AM EDT
Terminated Phase 1Phase 2 Interventional

A Fabry Disease Gene Therapy Study

NCT04040049
View directory record Official record
Conditions
Fabry Disease, Lysosomal Storage Diseases
Interventions
FLT190
Genetic
Lead sponsor
Spur Therapeutics
Industry
Eligibility
18 Years and older · Male only
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2023
U.S. locations
4
States / cities
Los Angeles, California • New York, New York • Pittsburgh, Pennsylvania + 1 more
Source: ClinicalTrials.gov public record
Updated Jun 4, 2023 · Synced Jul 22, 2026, 4:48 AM EDT
Terminated Phase 3 Interventional

ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases

NCT00654433
View directory record Official record
Conditions
Inherited Metabolic Diseases, Lysosomal Storage Disorders, Peroxisomal Storage Diseases, Inborn Errors of Metabolism, Mucopolysaccharidosis
Interventions
ALD-101
Biological
Lead sponsor
Aldagen
Industry
Eligibility
Up to 16 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2011
U.S. locations
3
States / cities
Los Angeles, California • New York, New York • Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Jul 7, 2014 · Synced Jul 22, 2026, 4:48 AM EDT
Not listed Not applicable Interventional

HDL Acute Lipid Optimization in Homozygous Familial Hypercholesterolemia

NCT03135184
View directory record Official record
Conditions
Homozygous Familial Hypercholesterolemia, HoFH
Interventions
HDL Therapeutics PDS-2 System
Device
Lead sponsor
HDL Therapeutics
Industry
Eligibility
12 Years and older
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2019
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated May 22, 2019 · Synced Jul 22, 2026, 4:48 AM EDT
Completed Phase 1Phase 2 Interventional

Statins and Lupus: Effects of Statins on Clinical Lupus Parameters, Serological Markers and Toll-like Receptors

NCT00519363
View directory record Official record
Conditions
Systemic Lupus Erythematosus
Interventions
atorvastatin
Drug
Lead sponsor
The Center for Rheumatic Disease, Allergy, & Immunology
Other
Eligibility
18 Years to 60 Years · Female only
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2008
U.S. locations
1
States / cities
Kansas City, Missouri
Source: ClinicalTrials.gov public record
Updated Mar 23, 2010 · Synced Jul 22, 2026, 4:48 AM EDT
Completed Phase 3 Interventional Results available

Safety, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged >12 Years) With Fabry Disease

NCT04049760
View directory record Official record
Conditions
Fabry Disease
Interventions
migalastat HCl 150 mg
Drug
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
12 Years to 17 Years
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2024
U.S. locations
5
States / cities
Tampa, Florida • Atlanta, Georgia • Minneapolis, Minnesota + 2 more
Source: ClinicalTrials.gov public record
Updated Feb 4, 2026 · Synced Jul 22, 2026, 4:48 AM EDT
Completed Phase 3 Interventional Results available

Study to Compare the Efficacy and Safety of Oral AT1001 and Enzyme Replacement Therapy in Patients With Fabry Disease

NCT01218659
View directory record Official record
Conditions
Fabry Disease
Interventions
migalastat hydrochloride, agalsidase
Drug · Biological
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
16 Years to 74 Years
Enrollment
68 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2015
U.S. locations
8
States / cities
Los Angeles, California • Aurora, Colorado • Decatur, Georgia + 5 more
Source: ClinicalTrials.gov public record
Updated Oct 31, 2018 · Synced Jul 22, 2026, 4:48 AM EDT
Recruiting No phase listed Observational

A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding

NCT04252066
View directory record Official record
Conditions
Fabry Disease
Interventions
migalastat
Drug
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
Female only
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2030
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jul 7, 2025 · Synced Jul 22, 2026, 4:48 AM EDT
Recruiting No phase listed Observational

A Long-term Follow-up Study of Gaucher Disease

NCT03190837
View directory record Official record
Conditions
Gaucher Disease
Interventions
Not listed
Lead sponsor
Duke University
Other
Eligibility
Not listed
Enrollment
200 participants
Timeline
2017 – 2027
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Sep 21, 2025 · Synced Jul 22, 2026, 4:48 AM EDT
Recruiting Phase 1Phase 2 Interventional

A Phase 1/2 Study of Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 Gangliosidosis

NCT03952637
View directory record Official record
Conditions
Lysosomal Diseases, Gangliosidosis, GM1
Interventions
AAV9-GLB1, Abdominal ultrasound, Rituximab, Sirolimus, Methylprednisolone, Prednisone, Audiology assessment with ABR, Bone density scan (DEXA), Electrocardiogram (EKG), Echocardiogram, Electroencephalogram (EEG) awake and extended overnight, Laboratory tests, Lumbar puncture, Brain MRI/MRS/fMRI, Neurocognitive testing, Neurology exam, PICC or other Central line placement, Skeletal survey, Skin biopsy, Speech and modified barium swallow study, Ophthalmology exam
Biological · Procedure · Drug + 3 more
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
6 Months to 12 Years
Enrollment
54 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2028
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Dec 10, 2025 · Synced Jul 22, 2026, 4:48 AM EDT
Completed No phase listed Observational

Sjogren-Larsson Syndrome: Natural History, Clinical Variation and Evaluation of Biochemical Markers

NCT01971957
View directory record Official record
Conditions
Sjogren-Larsson Syndrome (SLS)
Interventions
Not listed
Lead sponsor
University of Nebraska
Other
Eligibility
Not listed
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2023
U.S. locations
2
States / cities
Omaha, Nebraska • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Oct 7, 2024 · Synced Jul 22, 2026, 4:48 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

Comparison of KADIAN 100 mg When Dosed With Alcohol Under Fasting and Fed Conditions Compared to Water

NCT00768183
View directory record Official record
Conditions
Healthy
Interventions
KADIAN Capsule + alcohol (under fasting conditions), KADIAN Capsule + water (under fasting conditions), morphine sulfate IR oral solution + water, KADIAN Capsule + alcohol (under fed conditions)
Drug
Lead sponsor
Actavis Inc.
Industry
Eligibility
21 Years to 40 Years · Male only
Enrollment
32 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2006
U.S. locations
1
States / cities
Lincoln, Nebraska
Source: ClinicalTrials.gov public record
Updated Jul 15, 2020 · Synced Jul 22, 2026, 4:48 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 29, 2026 · Synced Jul 22, 2026, 4:48 AM EDT
Completed Phase 2 Interventional Results available

Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of SBC-102 (Sebelipase Alfa) in Adult Subjects With Lysosomal Acid Lipase Deficiency

NCT01488097
View directory record Official record
Conditions
Cholesterol Ester Storage Disease (CESD), Lysosomal Acid Lipase Deficiency, LAL-Deficiency
Interventions
sebelipase alfa
Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2017
U.S. locations
5
States / cities
Eureka, California • Sacramento, California • San Francisco, California + 2 more
Source: ClinicalTrials.gov public record
Updated Jul 19, 2018 · Synced Jul 22, 2026, 4:48 AM EDT
Recruiting Phase 1Phase 2 Interventional

64Cu-GRIP B in Patients With Advanced Malignancies

NCT05888532
View directory record Official record
Conditions
Prostate Cancer, Renal Cancer, Urethral Cancer, Advanced Solid Tumor, Metastatic Castration-resistant Prostate Cancer, Solid Tumor, Adult
Interventions
Copper-64 labeled Granzyme B (64Cu-GRIP B), Positron Emission Tomography (PET)
Drug · Procedure
Lead sponsor
Rahul Aggarwal
Other
Eligibility
18 Years and older
Enrollment
91 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2027
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Mar 16, 2026 · Synced Jul 22, 2026, 4:48 AM EDT
Completed Phase 2 Interventional Results available

Short-term Behavioral Effects of Cholesterol Therapy in Smith-Lemli-Opitz Syndrome

NCT00114634
View directory record Official record
Conditions
Smith-Lemli-Opitz Syndrome
Interventions
Egg yolk preparation with cholesterol, Egg substitute, without cholesterol
Dietary Supplement
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
4 Years to 17 Years
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2009
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jan 28, 2016 · Synced Jul 22, 2026, 4:48 AM EDT
Completed Phase 2Phase 3 Interventional Results available

Study of GA-GCB Enzyme Replacement Therapy in Type 1 Gaucher Disease Patients Previously Treated With Imiglucerase

NCT00478647
View directory record Official record
Conditions
Gaucher Disease
Interventions
GA-GCB (velaglucerase alfa)
Biological
Lead sponsor
Shire
Industry
Eligibility
2 Years and older
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2009
U.S. locations
11
States / cities
Los Angeles, California • Oakland, California • Decatur, Georgia + 8 more
Source: ClinicalTrials.gov public record
Updated Jun 9, 2021 · Synced Jul 22, 2026, 4:48 AM EDT
Completed Phase 3 Interventional Results available

Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) ERT Compared With Imiglucerase in Type I Gaucher Disease

NCT00553631
View directory record Official record
Conditions
Gaucher Disease, Type 1
Interventions
velaglucerase alfa, imiglucerase
Biological
Lead sponsor
Shire
Industry
Eligibility
2 Years and older
Enrollment
34 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2009
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Jun 7, 2021 · Synced Jul 22, 2026, 4:48 AM EDT
Not listed Phase 2 Interventional

Phase II Study of Glucocerebrosidase in Patients With Gaucher Disease

NCT00004293
View directory record Official record
Conditions
Gaucher's Disease
Interventions
glucocerebrosidase
Drug
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
18 Years to 65 Years
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1999
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jun 23, 2005 · Synced Jul 22, 2026, 4:48 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 20, 2021 · Synced Jul 22, 2026, 4:48 AM EDT
Completed No phase listed Observational

Registry of Fabry Disease - A Multicenter Observational Study

NCT00055016
View directory record Official record
Conditions
Fabry Disease
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Not listed
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2008
U.S. locations
2
States / cities
Bethesda, Maryland • Cambridge, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jul 1, 2017 · Synced Jul 22, 2026, 4:48 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Pre-Clinical White Matter Changes and Associated Connectivity Effects in Fabry Disease

NCT03678324
View directory record Official record
Conditions
Fabry Disease
Interventions
Functional MRI and fast bound-pool fraction imaging
Other
Lead sponsor
University of Utah
Other
Eligibility
18 Years and older
Enrollment
40 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2022
U.S. locations
1
States / cities
Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated May 25, 2022 · Synced Jul 22, 2026, 4:48 AM EDT