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ClinicalTrials.gov public records Last synced Oct 10, 2026, 9:14 PM EDT

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Showing 49–72 of 501 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Lipid Metabolism, Inborn Errors Clear all
Completed No phase listed Observational

An Observational Study of Patients With Lysosomal Acid Lipase Deficiency/Cholesteryl Ester Storage Disease Phenotype

NCT01528917
View directory record Official record
Conditions
Cholesterol Ester Storage Disease(CESD), Lysosomal Acid Lipase Deficiency
Interventions
Not listed
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
5 Years and older
Enrollment
49 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2013
U.S. locations
6
States / cities
Stanford, California • Chicago, Illinois • Minneapolis, Minnesota + 3 more
Source: ClinicalTrials.gov public record
Updated Jul 20, 2016 · Synced Oct 10, 2026, 9:14 PM EDT
Completed Phase 3 Interventional Results available

Switchover Trial From Imiglucerase to Plant Cell Expressed Recombinant Human Glucocerebrosidase

NCT00712348
View directory record Official record
Conditions
Gaucher Disease
Interventions
Taliglucerase alfa
Drug
Lead sponsor
Pfizer
Industry
Eligibility
2 Years and older
Enrollment
31 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2013
U.S. locations
3
States / cities
Coral Springs, Florida • Decatur, Georgia • New York, New York
Source: ClinicalTrials.gov public record
Updated Oct 4, 2018 · Synced Oct 10, 2026, 9:14 PM EDT
Completed No phase listed Observational

Validating a New Severity Score System for Adults With Type 1 Gaucher Disease (GD1)

NCT01136304
View directory record Official record
Conditions
Gaucher Disease
Interventions
Imiglucerase
Drug
Lead sponsor
University Research Foundation for Lysosomal Storage Diseases, Inc.
Other
Eligibility
18 Years and older
Enrollment
173 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2013
U.S. locations
2
States / cities
Beverly Hills, California • Coral Springs, Florida
Source: ClinicalTrials.gov public record
Updated Jun 1, 2015 · Synced Oct 10, 2026, 9:14 PM 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 8, 2014 · Synced Oct 10, 2026, 9:14 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Intravitreal ERT to Prevent Retinal Disease Progression in Children With CLN2

NCT05152914
View directory record Official record
Conditions
Neuronal Ceroid Lipofuscinosis Type 2
Interventions
Cerliponase Alfa
Drug
Lead sponsor
David L Rogers, MD
Other
Eligibility
24 Months to 72 Months
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2027
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Apr 30, 2026 · Synced Oct 10, 2026, 9:14 PM EDT
Completed Phase 3 Interventional Results available

Trial to Assess the Effect of Long Term Dosing of Inclisiran in Subjects With High CV Risk and Elevated LDL-C

NCT03814187
View directory record Official record
Conditions
ASCVD, Elevated Cholesterol, Heterozygous Familial Hypercholesterolemia, Homozygous Familial Hypercholesterolemia
Interventions
Inclisiran Sodium
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
18 Years and older
Enrollment
3,275 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2023
U.S. locations
141
States / cities
Birmingham, Alabama • Foley, Alabama • Huntsville, Alabama + 109 more
Source: ClinicalTrials.gov public record
Updated Mar 6, 2024 · Synced Oct 10, 2026, 9:14 PM EDT
Not listed Phase 2 Interventional

Phase II Study of Dietary Cholesterol for Smith-Lemli-Opitz Syndrome

NCT00004347
View directory record Official record
Conditions
Smith-Lemli-Opitz Syndrome
Interventions
diet
Behavioral
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
0 Years and older
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1995
U.S. locations
1
States / cities
Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 10, 2026, 9:14 PM 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 Nov 1, 2018 · Synced Oct 10, 2026, 9:14 PM EDT
Completed Phase 4 Interventional Results available

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

NCT00081497
View directory record Official record
Conditions
Fabry Disease
Interventions
agalsidase beta
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
16 Years and older
Enrollment
67 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2005
U.S. locations
18
States / cities
Birmingham, Alabama • Los Angeles, California • San Francisco, California + 15 more
Source: ClinicalTrials.gov public record
Updated Apr 2, 2015 · Synced Oct 10, 2026, 9:14 PM EDT
Completed No phase listed Observational

Clinical and Pathophysiological Investigations Into Erdheim Chester Disease

NCT01417520
View directory record Official record
Conditions
Myelofibrosis, Gaucher Disease, Pulmonary Fibrosis, Hermansky-Pudlak Syndrome (HPS), Cancer
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
2 Years to 80 Years
Enrollment
88 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2019
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 26, 2019 · Synced Oct 10, 2026, 9:14 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Gene Transfer Clinical Trial for Infantile and Late Infantile Krabbe Disease Treated Previously With HSCT

NCT05739643
View directory record Official record
Conditions
Krabbe Disease
Interventions
FBX-101
Biological
Lead sponsor
Forge Biologics, Inc
Industry
Eligibility
Up to 18 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2026
U.S. locations
3
States / cities
Orange, California • Ann Arbor, Michigan • Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Nov 20, 2025 · Synced Oct 10, 2026, 9:14 PM EDT
Completed Phase 2 Interventional

OGT 918-006: A Phase I/II Randomized, Controlled Study of OGT 918 in Patients With Neuronopathic Gaucher Disease

NCT00041535
View directory record Official record
Conditions
Gaucher Disease
Interventions
OGT 918
Drug
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
4 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2007
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 2, 2017 · Synced Oct 10, 2026, 9:14 PM EDT
Completed Phase 1Phase 2 Interventional

ADX-629 Therapy for Sjogren-Larsson Syndrome

NCT05443685
View directory record Official record
Conditions
Sjogren-Larsson Syndrome
Interventions
ADX-629
Drug
Lead sponsor
University of Nebraska
Other
Eligibility
5 Years to 50 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2026
U.S. locations
1
States / cities
Omaha, Nebraska
Source: ClinicalTrials.gov public record
Updated May 5, 2026 · Synced Oct 10, 2026, 9:14 PM EDT
Terminated Not applicable Interventional

Sterol and Isoprenoid Disease Research Consortium: Smith-Lemli-Opitz Syndrome

NCT01356420
View directory record Official record
Conditions
Smith-Lemli-Opitz Syndrome
Interventions
Cholesterol supplementation
Dietary Supplement
Lead sponsor
Oregon Health and Science University
Other
Eligibility
Up to 85 Years
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2015
U.S. locations
5
States / cities
Bethesda, Maryland • Omaha, Nebraska • Cincinnati, Ohio + 2 more
Source: ClinicalTrials.gov public record
Updated May 23, 2019 · Synced Oct 10, 2026, 9:14 PM EDT
Withdrawn Phase 4 Interventional

A Study in Patients With Fabry Disease Who Are on Chronic Hemodialysis Therapy for Treatment of End-stage Renal Insufficiency.

NCT00312767
View directory record Official record
Conditions
Fabry Disease
Interventions
Fabrazyme (agalsidase beta)
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
18 Years to 65 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2006
U.S. locations
1
States / cities
North Charleston, South Carolina
Source: ClinicalTrials.gov public record
Updated Feb 5, 2014 · Synced Oct 10, 2026, 9:14 PM EDT
Completed No phase listed Observational

The GBA Multimodal Study in Parkinson's Disease

NCT04101968
View directory record Official record
Conditions
Parkinson Disease, GBA Gene Mutation, Gaucher Disease
Interventions
PET scan, neuroQWERTY
Diagnostic Test
Lead sponsor
Pacific Parkinson's Research Centre
Other
Eligibility
18 Years to 80 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2026
U.S. locations
2
States / cities
Portland, Oregon • Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Sep 2, 2026 · Synced Oct 10, 2026, 9:14 PM EDT
Completed Phase 1 Interventional

Bone Marrow Transplant With Abatacept for Non-Malignant Diseases

NCT01917708
View directory record Official record
Conditions
Hurler Syndrome, Fanconi Anemia, Glanzmann Thrombasthenia, Wiskott-Aldrich Syndrome, Chronic Granulomatous Disease, Severe Congenital Neutropenia, Leukocyte Adhesion Deficiency, Shwachman-Diamond Syndrome, Diamond-Blackfan Anemia, Dyskeratosis-congenita, Chediak-Higashi Syndrome, Severe Aplastic Anemia, Thalassemia Major, Hemophagocytic Lymphohistiocytosis, Sickle Cell Disease
Interventions
Abatacept
Drug
Lead sponsor
Emory University
Other
Eligibility
Up to 21 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2019
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Dec 26, 2019 · Synced Oct 10, 2026, 9:14 PM EDT
Terminated No phase listed Observational

Kidney Information Network for Disease Research and Education

NCT03321604
View directory record Official record
Conditions
ESRD, Fabry Disease, Rare Diseases, Kidney Diseases, End Stage Renal Disease, Genetic Diseases, X-Linked
Interventions
Not listed
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
18 Years to 65 Years
Enrollment
338 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2019
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Feb 24, 2021 · Synced Oct 10, 2026, 9:14 PM EDT
Withdrawn Not applicable Interventional

Cross-over Pilot Study of Blood Cholesterol Response to Dietary Saturated Fat in Patients With Autosomal Dominant Hypercholesterolemia

NCT03756779
View directory record Official record
Conditions
Familial Hypercholesterolemia
Interventions
Dietary intervention
Other
Lead sponsor
University of Texas Southwestern Medical Center
Other
Eligibility
18 Years to 80 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2020
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Nov 28, 2018 · Synced Oct 10, 2026, 9:14 PM EDT
Recruiting No phase listed Observational

Oxidative Stress and Inflammatory Biomarkers in Gaucher Disease

NCT02437396
View directory record Official record
Conditions
Gaucher Disease Type I, Oxidative Stress, Inflammation
Interventions
Not listed
Lead sponsor
University of Minnesota
Other
Eligibility
18 Years to 75 Years
Enrollment
34 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2026
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jul 13, 2026 · Synced Oct 10, 2026, 9:14 PM EDT
Completed Phase 2Phase 3 Interventional Results available

A Trial to Evaluate Safety, Tolerability and Efficacy of Elamipretide in Subjects With Barth Syndrome

NCT03098797
View directory record Official record
Conditions
Barth Syndrome
Interventions
Elamipretide, Placebo
Drug
Lead sponsor
Stealth BioTherapeutics Inc.
Industry
Eligibility
12 Years and older · Male only
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2021
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Apr 16, 2024 · Synced Oct 10, 2026, 9:14 PM EDT
Terminated Not applicable Interventional Results available

SLOS: The Effect of Simvastatin in Patients Receiving Cholesterol Supplementation

NCT01434745
View directory record Official record
Conditions
Smith-Lemli-Opitz Syndrome
Interventions
Simvastatin, Lactose
Drug · Dietary Supplement
Lead sponsor
Oregon Health and Science University
Other
Eligibility
1 Year to 89 Years
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2014
U.S. locations
1
States / cities
Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Oct 1, 2019 · Synced Oct 10, 2026, 9:14 PM EDT
Terminated No phase listed Observational

Cardiovasculorenal Phenotyping in Fabry Disease Through Noninvasive Testing

NCT05699265
View directory record Official record
Conditions
Fabry Disease
Interventions
Measures of arterial stiffness and endothelial function, Ambulatory blood pressure monitoring, Cardiopulmonary exercise testing (CPET), Serum and urine biomarkers
Diagnostic Test
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
8 Years and older
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Jun 19, 2025 · Synced Oct 10, 2026, 9:14 PM EDT
Completed Phase 2 Interventional Results available

Dosing Study of Replagal in Patients With Fabry Disease

NCT00068107
View directory record Official record
Conditions
Fabry Disease
Interventions
Replagal
Drug
Lead sponsor
Baylor Research Institute
Other
Eligibility
Male only
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2013
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 31, 2015 · Synced Oct 10, 2026, 9:14 PM EDT