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ClinicalTrials.gov public records Last synced Oct 13, 2026, 10:58 PM EDT

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Showing 145–168 of 501 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Lipid Metabolism, Inborn Errors Clear all
Completed Phase 1 Interventional Accepts healthy volunteers

Bioavailability Study of Desloratadine 5 mg Tablet of Dr. Reddy's Under Fed Conditions

NCT01314352
View directory record Official record
Conditions
Fed
Interventions
Desloratadine
Drug
Lead sponsor
Dr. Reddy's Laboratories Limited
Industry
Eligibility
18 Years to 45 Years
Enrollment
28 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2005 – 2006
U.S. locations
1
States / cities
Fort Myers, Florida
Source: ClinicalTrials.gov public record
Updated Jun 8, 2011 · Synced Oct 13, 2026, 10:58 PM EDT
Completed Phase 2Phase 3 Interventional Results available

Stem Cell Transplant for Bone Marrow Failure Syndromes

NCT00176878
View directory record Official record
Conditions
Diamond-Blackfan Anemia, Kostmann's Neutropenia, Shwachman-Diamond Syndrome
Interventions
Stem cell transplant, Fludarabine monophosphate, Total lymphoid irradiation, Busulfan, anti-thymocyte globulin
Procedure · Drug · Biological
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 35 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2009
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Dec 28, 2017 · Synced Oct 13, 2026, 10:58 PM EDT
Terminated No phase listed Observational

A Study to Identify and Characterize LAL-D Patients in High-risk Populations

NCT02345421
View directory record Official record
Conditions
Lysosomal Acid Lipase Deficiency
Interventions
Not listed
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
2 Years and older
Enrollment
640 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2015
U.S. locations
15
States / cities
Birmingham, Alabama • Phoenix, Arizona • Los Angeles, California + 12 more
Source: ClinicalTrials.gov public record
Updated May 24, 2016 · Synced Oct 13, 2026, 10:58 PM EDT
Terminated Phase 1Phase 2 Interventional

Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders

NCT01372228
View directory record Official record
Conditions
Hurler Syndrome (MPS I), Hurler-Scheie Syndrome, Hunter Syndrome (MPS II), Sanfilippo Syndrome (MPS III), Krabbe Disease (Globoid Leukodystrophy), Metachromatic Leukodystrophy (MLD), Adrenoleukodystrophy (ALD and AMN), Sandhoff Disease, Tay Sachs Disease, Pelizaeus Merzbacher (PMD), Niemann-Pick Disease, Alpha-mannosidosis
Interventions
hematopoietic stem cell infusion
Biological
Lead sponsor
Talaris Therapeutics Inc.
Industry
Eligibility
Not listed
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2016
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Apr 12, 2023 · Synced Oct 13, 2026, 10:58 PM EDT
Completed Phase 2 Interventional

Bone Response to Enzyme Replacement in Gaucher's Disease

NCT00001416
View directory record Official record
Conditions
Gaucher's Disease
Interventions
CEREDASE™
Drug
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Not listed
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1993 – 2000
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 22, 2016 · Synced Oct 13, 2026, 10:58 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Study of Gemcabene in Adults With FPLD

NCT03508687
View directory record Official record
Conditions
Familial Partial Lipodystrophy, Hypertriglyceridemia, Fatty Liver, NASH - Nonalcoholic Steatohepatitis
Interventions
300mg Gemcabene, 600mg Gemcabene
Drug
Lead sponsor
Elif Oral
Other
Eligibility
18 Years and older
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2019
U.S. locations
1
States / cities
Ann Arbor, Michigan
Source: ClinicalTrials.gov public record
Updated Aug 17, 2020 · Synced Oct 13, 2026, 10:58 PM EDT
Available No phase listed Expanded access

Treatment Protocol of Plozasiran in Adults With High-Risk Severe Hypertriglyceridemia (SHTG) and in Adults and Adolescents With FCS

NCT06796426
View directory record Official record
Conditions
Familial Chylomicronemia, High Risk Severe Hypertriglyceridemia (SHTG)
Interventions
Plozasiran
Drug
Lead sponsor
Arrowhead Pharmaceuticals
Industry
Eligibility
15 Years and older
U.S. locations
1
States / cities
Pasadena, California
Source: ClinicalTrials.gov public record
Updated Mar 3, 2026 · Synced Oct 13, 2026, 10:58 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A Study to Evaluate Pharmacokinetic Parameters of Eliglustat in Healthy Volunteers Who Are CYP2D6 Extensive or Poor Metabolizers

NCT06188325
View directory record Official record
Conditions
Gaucher's Disease
Interventions
Eliglustat
Drug
Lead sponsor
Sanofi
Industry
Eligibility
18 Years to 65 Years
Enrollment
18 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Jan 10, 2024 · Synced Oct 13, 2026, 10:58 PM EDT
Recruiting No phase listed Observational

MAGNETIC RESONANCE SPECTROSCOPY BIOMARKERS IN TYPE 3 GAUCHER DISEASE (GD3)

NCT05586243
View directory record Official record
Conditions
Gaucher Disease, Type 3
Interventions
No intervention
Other
Lead sponsor
University of Minnesota
Other
Eligibility
18 Years to 80 Years
Enrollment
5 participants
Timeline
2023 – 2026
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Aug 4, 2026 · Synced Oct 13, 2026, 10:58 PM EDT
Not listed Not applicable Interventional

Identification of Undiagnosed Lysosomal Acid Lipase Deficiency

NCT01716728
View directory record Official record
Conditions
Lysosomal Acid Lipase Deficiency, Cholesterol Ester Storage Disease
Interventions
enzyme analysis
Other
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
Not listed
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2012
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Aug 13, 2013 · Synced Oct 13, 2026, 10:58 PM EDT
Completed Phase 2 Interventional

Alternative Dosing and Regimen of Replagal to Treat Fabry Disease

NCT00075244
View directory record Official record
Conditions
Fabry Disease
Interventions
Replagal
Drug
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Male only
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2005
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 13, 2026, 10:58 PM EDT
Completed Phase 2 Interventional

Phase II Randomized Study of Alendronate Sodium for Osteopenia in Patients With Gaucher's Disease

NCT00004488
View directory record Official record
Conditions
Gaucher's Disease, Osteopenia
Interventions
alendronate sodium, calcium carbonate, cholecalciferol
Drug
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
18 Years to 50 Years
Enrollment
82 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1998 – 2002
U.S. locations
2
States / cities
Cincinnati, Ohio • Dayton, Ohio
Source: ClinicalTrials.gov public record
Updated Mar 25, 2015 · Synced Oct 13, 2026, 10:58 PM EDT
Not listed No phase listed Observational

SRT in Comparison to ERT on Immune Aspects and Bone Involvement in Gaucher Disease

NCT02605603
View directory record Official record
Conditions
Gaucher Disease
Interventions
Not listed
Lead sponsor
Lysosomal and Rare Disorders Research and Treatment Center, Inc.
Other
Eligibility
18 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2020
U.S. locations
1
States / cities
Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Aug 13, 2019 · Synced Oct 13, 2026, 10:58 PM EDT
Completed Phase 1 Interventional

Effect of ACP-501 on Safety, Tolerability, Pharmacokinetics and Pharmacodynamics in Subjects With Coronary Artery Disease

NCT01554800
View directory record Official record
Conditions
Coronary Artery Disease, Atherosclerosis
Interventions
ACP-501
Drug
Lead sponsor
MedImmune LLC
Industry
Eligibility
30 Years to 85 Years
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 14, 2019 · Synced Oct 13, 2026, 10:58 PM EDT
Enrolling by invitation Not applicable Interventional

Penn Family Screening for Familial Hypercholesterolemia

NCT05750667
View directory record Official record
Conditions
Familial Hypercholesterolemia
Interventions
Cascade screening
Behavioral
Lead sponsor
Northwestern University
Other
Eligibility
18 Years and older
Enrollment
800 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2027
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Feb 9, 2026 · Synced Oct 13, 2026, 10:58 PM EDT
Completed Phase 2 Interventional Results available

Open Label Extension of ISIS 301012 (Mipomersen) to Treat Familial Hypercholesterolemia

NCT00477594
View directory record Official record
Conditions
Lipid Metabolism, Inborn Errors, Hypercholesterolemia, Autosomal Dominant, Hyperlipidemias, Metabolic Diseases, Hyperlipoproteinemia Type II, Metabolism, Inborn Errors, Genetic Diseases, Inborn, Infant, Newborn, Diseases, Metabolic Disorder, Congenital Abnormalities, Hypercholesterolemia, Hyperlipoproteinemias, Dyslipidemias, Lipid Metabolism Disorders
Interventions
mipomersen sodium
Drug
Lead sponsor
Kastle Therapeutics, LLC
Industry
Eligibility
12 Years and older
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2011
U.S. locations
5
States / cities
Chicago, Illinois • Auburn, Maine • Biddeford, Maine + 2 more
Source: ClinicalTrials.gov public record
Updated Sep 9, 2016 · Synced Oct 13, 2026, 10:58 PM EDT
Completed Phase 4 Interventional Results available

A Prospective Study to Investigate Safety and Tolerability of Shorter Infusion of Fabrazyme

NCT06019728
View directory record Official record
Conditions
Fabry's Disease
Interventions
AGALSIDASE BETA (GZ419828), Acetaminophen, Diphenhydramine, Dexamethasone, Montelukast, Loratadine, Cetirizine, Fexofenadine
Drug
Lead sponsor
Sanofi
Industry
Eligibility
2 Years to 65 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
5
States / cities
Santa Monica, California • Atlanta, Georgia • Grand Rapids, Michigan + 2 more
Source: ClinicalTrials.gov public record
Updated Oct 21, 2025 · Synced Oct 13, 2026, 10:58 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 13, 2026, 10:58 PM EDT
Completed Phase 2 Interventional Results available

CLINICAL PROTOCOL to Investigate the Long-term Safety and Efficacy of Metreleptin in Various Forms of Partial Lipodystrophy

NCT02654977
View directory record Official record
Conditions
Familial Partial Lipodystrophy, Nonalcoholic Steatohepatitis, NAFLD
Interventions
Metreleptin
Drug
Lead sponsor
University of Michigan
Other
Eligibility
5 Years and older
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2019
U.S. locations
1
States / cities
Ann Arbor, Michigan
Source: ClinicalTrials.gov public record
Updated Nov 24, 2020 · Synced Oct 13, 2026, 10:58 PM EDT
Completed Phase 2 Interventional Results available

Trial to Evaluate the Effect of ALN-PCSSC Treatment on Low Density Lipoprotein Cholesterol (LDL-C)

NCT02597127
View directory record Official record
Conditions
Atherosclerotic Cardiovascular Disease, Familial Hypercholesterolemia, Diabetes
Interventions
ALN-PCSSC, Normal Saline
Drug
Lead sponsor
The Medicines Company
Industry
Eligibility
18 Years to 99 Years
Enrollment
501 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2017
U.S. locations
8
States / cities
Jacksonville, Florida • Indianapolis, Indiana • New York, New York + 4 more
Source: ClinicalTrials.gov public record
Updated May 17, 2019 · Synced Oct 13, 2026, 10:58 PM EDT
Not listed No phase listed Observational Accepts healthy volunteers

Screening of Lysosomal Storage Disorders Diseases in Minority Groups

NCT03812042
View directory record Official record
Conditions
Lysosomal Storage Diseases
Interventions
Enzyme assay and molecular sequencing
Diagnostic Test
Lead sponsor
Lysosomal and Rare Disorders Research and Treatment Center, Inc.
Other
Eligibility
Not listed
Enrollment
100,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2016 – 2019
U.S. locations
1
States / cities
Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Jan 22, 2019 · Synced Oct 13, 2026, 10:58 PM EDT
Completed Phase 4 Interventional

A Multicenter Study of the Efficacy of Cerezyme in Testing Skeletal Disease in Patients With Type I Gaucher Disease.

NCT00365131
View directory record Official record
Conditions
Gaucher Disease Type I, Cerebroside Lipidosis Syndrome, Clucocerebrosidase Deficiency Disease, Glucosylceramide Beta-Glucosidase Deficiency Disease, Gaucher Disease, Non-Neuronopathic Form
Interventions
Cerezyme (imiglucerase for injection)
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
10 Years to 65 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1997 – 2004
U.S. locations
1
States / cities
Coral Springs, Florida
Source: ClinicalTrials.gov public record
Updated Mar 5, 2015 · Synced Oct 13, 2026, 10:58 PM EDT
Completed Phase 1Phase 2 Interventional

Unrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood Cells

NCT00730314
View directory record Official record
Conditions
Sickle Cell Disease, Thalassemia, Anemia, Granuloma, Wiskott-Aldrich Syndrome, Chediak Higashi Syndrome, Osteopetrosis, Neutropenia, Thrombocytopenia, Hurler Disease, Niemann-Pick Disease, Fucosidosis
Interventions
Hematopoietic stem cell transplantation
Procedure
Lead sponsor
Children's Hospital Los Angeles
Other
Eligibility
Up to 21 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2015
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Jun 23, 2016 · Synced Oct 13, 2026, 10:58 PM EDT
Withdrawn Phase 4 Interventional

Pharmacokinetics, Pharmacodynamics And Safety Study Of Elelyso(tm) In Pediatric Subjects With Type 1 Gaucher Disease

NCT03021941
View directory record Official record
Conditions
Type 1 Gaucher Disease
Interventions
Elelyso 60 units/kg
Drug
Lead sponsor
Pfizer
Industry
Eligibility
Up to 12 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2020
U.S. locations
1
States / cities
Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Sep 24, 2019 · Synced Oct 13, 2026, 10:58 PM EDT