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ClinicalTrials.gov public records Last synced Oct 14, 2026, 9:57 AM EDT

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Showing 385–408 of 501 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Lipid Metabolism, Inborn Errors Clear all
Withdrawn Not applicable Interventional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 14, 2026, 9:57 AM 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 14, 2026, 9:57 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 8, 2021 · Synced Oct 14, 2026, 9:57 AM EDT
Recruiting Phase 1 Interventional

A Study of VERVE-102 in Patients With Familial Hypercholesterolemia or Premature Coronary Artery Disease

NCT06164730
View directory record Official record
Conditions
Heterozygous Familial Hypercholesterolemia, Premature Coronary Heart Disease
Interventions
VERVE-102
Drug
Lead sponsor
Verve Therapeutics, Inc.
Industry
Eligibility
18 Years to 70 Years
Enrollment
85 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
10
States / cities
Dothan, Alabama • Pomona, California • Boca Raton, Florida + 7 more
Source: ClinicalTrials.gov public record
Updated Sep 2, 2026 · Synced Oct 14, 2026, 9:57 AM EDT
Recruiting No phase listed Observational

Cardiovascular Multimodality Imaging Study

NCT06610019
View directory record Official record
Conditions
Non-ischemic Cardiomyopathy, Cardiomyopathies, Hypertrophic Cardiomyopathy, Right Ventricular Arrhythmogenic Cardiomyopathy, Cardiac Amyloidosis, Anderson Fabry Disease, Sarcoidosis, Cancer Therapy-related Cardiac Dysfunction, Ventricular Arrythmia, Heart Failure
Interventions
Not listed
Lead sponsor
Montefiore Medical Center
Other
Eligibility
18 Years and older
Enrollment
10,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2031
U.S. locations
1
States / cities
The Bronx, New York
Source: ClinicalTrials.gov public record
Updated Aug 10, 2026 · Synced Oct 14, 2026, 9:57 AM EDT
Completed Phase 3 Interventional Results available

Extension Study of TKT028 Evaluating Safety and Clinical Outcomes of Replagal® in Adult Patients With Fabry Disease

NCT01124643
View directory record Official record
Conditions
Fabry Disease
Interventions
Replagal
Biological
Lead sponsor
Shire
Industry
Eligibility
18 Years and older
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2013
U.S. locations
2
States / cities
Iowa City, Iowa • Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Jun 8, 2021 · Synced Oct 14, 2026, 9:57 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Lipodystrophy Connect Patient Registry

NCT02577952
View directory record Official record
Conditions
Lipodystrophy
Interventions
Not listed
Lead sponsor
PatientCrossroads
Industry
Eligibility
Not listed
Enrollment
257 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2014 – 2018
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Apr 22, 2019 · Synced Oct 14, 2026, 9:57 AM 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 14, 2026, 9:57 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Study of IV VTS-270 for Infantile Liver Disease Associated With Niemann-Pick Disease, Type C

NCT03471143
View directory record Official record
Conditions
Niemann-Pick Disease, Type C
Interventions
VTS-270
Drug
Lead sponsor
Washington University School of Medicine
Other
Eligibility
Up to 6 Months
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2024
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Jan 5, 2026 · Synced Oct 14, 2026, 9:57 AM EDT
Active, not recruiting No phase listed Observational

Long-Term Follow Up of CLN6 Batten Disease Subjects Following Gene Transfer

NCT04273243
View directory record Official record
Conditions
CLN6, Batten Disease
Interventions
AT-GTX-501
Genetic
Lead sponsor
Emily de los Reyes
Other
Eligibility
12 Months and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2027
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Oct 2, 2025 · Synced Oct 14, 2026, 9:57 AM EDT
Completed Not applicable Interventional

A Pilot Study of a Lifestyle Intervention on the Metabolic Syndrome (3ELM Study)

NCT02233088
View directory record Official record
Conditions
Metabolic Syndrome
Interventions
ELM Group, ELM Classes, ELM Individual
Behavioral · Other
Lead sponsor
Rush University Medical Center
Other
Eligibility
18 Years to 72 Years
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2021
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Jan 12, 2023 · Synced Oct 14, 2026, 9:57 AM EDT
Active, not recruiting Phase 3 Interventional

A Phase 3 Study of Zodasiran in Adolescent and Adult Subjects With Homozygous Familial Hypercholesterolemia (YOSEMITE)

NCT07037771
View directory record Official record
Conditions
Homozygous Familial Hypercholesterolemia
Interventions
zodasiran Injection, Placebo
Drug
Lead sponsor
Arrowhead Pharmaceuticals
Industry
Eligibility
12 Years and older
Enrollment
70 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
4
States / cities
Park Ridge, Illinois • New York, New York • Cincinnati, Ohio + 1 more
Source: ClinicalTrials.gov public record
Updated Aug 12, 2026 · Synced Oct 14, 2026, 9:57 AM EDT
Terminated Phase 4 Interventional Results available

Study of the Effects of Fabrazyme Treatment on Lactation and Infants

NCT00230607
View directory record Official record
Conditions
Fabry Disease, Alpha Galactosidase A Deficiency
Interventions
agalsidase beta
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
Not listed
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2024
U.S. locations
3
States / cities
Decatur, Georgia • Fairfax, Virginia • Milwaukee, Wisconsin
Source: ClinicalTrials.gov public record
Updated Apr 10, 2024 · Synced Oct 14, 2026, 9:57 AM EDT
Completed No phase listed Observational

Patterns of Inclisiran Use in the Real World: An Analysis of US Databases

NCT07023445
View directory record Official record
Conditions
Atherosclerotic Cardiovascular Disease, Familial Hypercholesterolemia
Interventions
Not listed
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
18 Years and older
Enrollment
37,688 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
1
States / cities
East Hanover, New Jersey
Source: ClinicalTrials.gov public record
Updated Jun 17, 2025 · Synced Oct 14, 2026, 9:57 AM EDT
Terminated Phase 1 Interventional

Safety Study of rhASM Enzyme Replacement Therapy in Adults With Acid Sphingomyelinase Deficiency (Niemann-Pick Disease)

NCT00410566
View directory record Official record
Conditions
Acid Sphingomyelinase Deficiency, Niemann-Pick Disease
Interventions
rhASM
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
18 Years to 65 Years
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2009
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Mar 19, 2015 · Synced Oct 14, 2026, 9:57 AM EDT
Completed Phase 2 Interventional Results available

Safety Study of Replagal® Therapy in Children With Fabry Disease

NCT01363492
View directory record Official record
Conditions
Fabry Disease
Interventions
Replagal (agalsidase alfa)
Biological
Lead sponsor
Shire
Industry
Eligibility
7 Years to 17 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2013
U.S. locations
5
States / cities
Decatur, Georgia • Durham, North Carolina • Dallas, Texas + 2 more
Source: ClinicalTrials.gov public record
Updated Jun 9, 2021 · Synced Oct 14, 2026, 9:57 AM EDT
Terminated Phase 4 Interventional Results available

Synergistic Enteral Regimen for Treatment of the Gangliosidoses

NCT02030015
View directory record Official record
Conditions
GM1 Gangliosidoses, GM2 Gangliosidoses, Tay-Sachs Disease, Sandhoff Disease
Interventions
miglustat, Ketogenic Diet
Drug · Other
Lead sponsor
University of Minnesota
Other
Eligibility
Up to 204 Months
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2019
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Apr 14, 2021 · Synced Oct 14, 2026, 9:57 AM EDT
Active, not recruiting Not applicable Interventional

Randomized Controlled Trial of Alert-Based Computerized Decision Support for Optimizing Low-Density Lipoprotein Management

NCT05088759
View directory record Official record
Conditions
Hyperlipemia, Mixed, ASCVD
Interventions
Alert
Behavioral
Lead sponsor
Brigham and Women's Hospital
Other
Eligibility
18 Years and older
Enrollment
400 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2026
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Sep 29, 2026 · Synced Oct 14, 2026, 9:57 AM EDT
Recruiting Phase 3 Interventional

A Study of Mavorixafor in Participants With Congenital and Acquired Primary Autoimmune and Idiopathic Chronic Neutropenic Disorders Who Are Experiencing Recurrent and/or Serious Infections

NCT06056297
View directory record Official record
Conditions
Neutropenia
Interventions
Mavorixafor, Placebo
Drug
Lead sponsor
X4 Pharmaceuticals
Industry
Eligibility
12 Years and older
Enrollment
176 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
22
States / cities
Phoenix, Arizona • Irvine, California • La Jolla, California + 19 more
Source: ClinicalTrials.gov public record
Updated Aug 6, 2026 · Synced Oct 14, 2026, 9:57 AM EDT
Recruiting No phase listed Observational

The Myelin Disorders Biorepository Project

NCT03047369
View directory record Official record
Conditions
Leukodystrophy, White Matter Disease, Leukoencephalopathies, 4H Syndrome, Adrenoleukodystrophy, AMN, ALD, ALD Gene Mutation, ALD (Adrenoleukodystrophy), X-linked Adrenoleukodystrophy, X-ALD, Adrenomyeloneuropathy, Aicardi Goutieres Syndrome, AGS, Alexander Disease, Alexanders Leukodystrophy, AxD, ADLD, Canavan Disease, CTX, Cerebrotendinous Xanthomatoses, Krabbe Disease, GALC Deficiency, Globoid Leukodystrophy, TUBB4A-Related Leukodystrophy, H-ABC - Hypomyelination, Atrophy of Basal Ganglia and Cerebellum, HBSL, HBSL - Hypomyelination, Brain Stem, Spinal Cord, Leg Spasticity, LBSL, Leukoencephalopathy With Brain Stem and Spinal Cord Involvement and High Lactate Syndrome (Disorder), Leukoencephalopathy With Brainstem and Spinal Cord Involvement and Lactate Elevation, ALSP, CSF1R Gene Mutation, HCC - Hypomyelination and Congenital Cataract, MLC1, Megalencephalic Leukoencephalopathy With Subcortical Cysts, MLD, Metachromatic Leukodystrophy, PMD, Pelizaeus-Merzbacher Disease, PLP1 Null Syndrome, PLP1 Gene Duplication | Blood or Tissue | Mutations, Pelizaeus Merzbacher Like Disease, Peroxisomal Biogenesis Disorder, Zellweger Syndrome, Refsum Disease, Salla Disease, Sialic Storage Disease, Sjögren, Sjogren-Larsson Syndrome, Van Der Knapp Disease, Vanishing White Matter Disease, Charcot-Marie-Tooth, CMT, Mct8 (Slc16A2)-Specific Thyroid Hormone Cell Transporter Deficiency, Allan-Herndon-Dudley Syndrome, Cadasil, Cockayne Syndrome, Multiple Sulfatase Deficiency, Gangliosidoses, GM2 Gangliosidosis, BPAN, Labrune Syndrome, LCC, Mucopolysaccharidoses, TBCK-Related Intellectual Disability Syndrome
Interventions
Not listed
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
Not listed
Enrollment
12,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2030
U.S. locations
23
States / cities
Los Angeles, California • Orange, California • Palo Alto, California + 18 more
Source: ClinicalTrials.gov public record
Updated Oct 23, 2025 · Synced Oct 14, 2026, 9:57 AM EDT
Completed No phase listed Observational

Equitable Implementation of Cascade Screening for Familial Hypercholesterolemia

NCT05430191
View directory record Official record
Conditions
Familial Hypercholesterolemia
Interventions
Cascade Screening
Other
Lead sponsor
Northwestern University
Other
Eligibility
18 Years and older
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2023
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Sep 29, 2023 · Synced Oct 14, 2026, 9:57 AM EDT
Completed No phase listed Observational Results available

Cerebrotendinous Xanthomatosis (CTX) Prevalence Study

NCT02638220
View directory record Official record
Conditions
Cerebrotendinous Xanthomatosis (CTX)
Interventions
Not listed
Lead sponsor
Travere Therapeutics, Inc.
Industry
Eligibility
2 Years to 21 Years
Enrollment
442 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2021
U.S. locations
44
States / cities
Birmingham, Alabama • La Jolla, California • Los Angeles, California + 38 more
Source: ClinicalTrials.gov public record
Updated Sep 19, 2024 · Synced Oct 14, 2026, 9:57 AM EDT
Not listed No phase listed Observational

Pulmonary Disease and Exercise Tolerance in Boys With Fabry Disease

NCT01304875
View directory record Official record
Conditions
Fabry Disease
Interventions
Not listed
Lead sponsor
Cedars-Sinai Medical Center
Other
Eligibility
8 Years to 18 Years · Male only
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2010
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Jun 27, 2013 · Synced Oct 14, 2026, 9:57 AM EDT
Completed No phase listed Observational

A Natural History of Late Onset Tay-Sachs Disease

NCT02851862
View directory record Official record
Conditions
GM2 Gangliosidosis
Interventions
Not listed
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
7 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2025
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Mar 2, 2026 · Synced Oct 14, 2026, 9:57 AM EDT