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ClinicalTrials.gov public records Last synced Nov 6, 2026, 11:52 AM EST

All trial information on this site comes from the official ClinicalTrials.gov public API (clinicaltrials.gov/api/v2), run by the U.S. National Library of Medicine. We only display that public information; we do not add opinions, endorsements, or any other intent. Always review the official ClinicalTrials.gov record for the latest information.

Showing 1–24 of 319 matching trials from the live ClinicalTrials.gov search.

Filters: Condition: Lipoidosis Clear all

Conditions: Fabry Disease
Interventions: Not listed
Lead sponsor: Boston Children's Hospital; Other
Eligibility: 1 Year and older
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2030
U.S. locations: 1
States / cities: Boston, Massachusetts

Conditions: Fabry Disease, Chronic Kidney Disease, Stage IV (Severe)
Interventions: Fabrazyme (agalsidase beta); Biological
Lead sponsor: Genzyme, a Sanofi Company; Industry
Eligibility: 16 Years and older
Enrollment: 20 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2002 – 2003
U.S. locations: 9
States / cities: Farmington, Connecticut • Coral Springs, Florida • Kansas City, Kansas + 6 more

Conditions: GM1 Gangliosidosis, GM2 Gangliosidosis, Gaucher Disease, Type 2, Tay-Sachs Disease, AB Variant Gangliosidosis GM2, Sandhoff Disease
Interventions: Not listed
Lead sponsor: Idorsia Pharmaceuticals Ltd.; Industry
Eligibility: 0 Months and older
Enrollment: 226 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2021
U.S. locations: 3
States / cities: Chicago, Illinois • Rochester, Minnesota • Fairfax, Virginia

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

Conditions: Fabry Disease
Interventions: migalastat hydrochloride, Placebo; Drug
Lead sponsor: Amicus Therapeutics; Industry
Eligibility: 16 Years to 74 Years
Enrollment: 67 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2009 – 2014
U.S. locations: 13
States / cities: Los Angeles, California • San Francisco, California • Decatur, Georgia + 10 more

Conditions: Lipoidosis
Interventions: SCH-58235; Drug
Lead sponsor: National Heart, Lung, and Blood Institute (NHLBI); NIH
Eligibility: Not listed
Enrollment: 5 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2001 – 2004
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Fabry Disease
Interventions: migalastat HCl, ERT; Drug
Lead sponsor: Amicus Therapeutics; Industry
Eligibility: 18 Years and older
Enrollment: 450 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2032
U.S. locations: 8
States / cities: Birmingham, Alabama • Little Rock, Arkansas • Atlanta, Georgia + 5 more

Conditions: Fabry Disease
Interventions: migalastat HCl; Drug
Lead sponsor: Amicus Therapeutics; Industry
Eligibility: 18 Years and older
Enrollment: 23 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2007 – 2012
U.S. locations: 3
States / cities: Decatur, Georgia • New York, New York • Dallas, Texas

Conditions: Gaucher's Disease
Interventions: Lysodase; Drug
Lead sponsor: National Institute of Mental Health (NIMH); NIH
Eligibility: Not listed
Enrollment: 18 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 1993 – 2001
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Infantile Globoid Cell Leukodystrophy
Interventions: Not listed
Lead sponsor: Zymenex A/S; Industry
Eligibility: Up to 2 Years
Enrollment: 6 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2009 – 2014
U.S. locations: 1
States / cities: Pittsburgh, Pennsylvania

Conditions: Primary Immune Deficiency Disorders, Hemophagocytic Lymphohistiocytosis, Inherited Bone Marrow Failure Syndrome, Hemoglobinopathies, Metabolic Disorders
Interventions: BPX-501 and Rimiducid; Biological
Lead sponsor: Bellicum Pharmaceuticals; Industry
Eligibility: 4 Months to 55 Years
Enrollment: 1 participant
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2018
U.S. locations: 1
States / cities: Seattle, Washington

Conditions: Lysosomal Acid Lipase Deficiency, Wolman Disease
Interventions: Not listed
Lead sponsor: Alexion Pharmaceuticals, Inc.; Industry
Eligibility: Not listed
Enrollment: 40 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2010 – 2013
U.S. locations: 7
States / cities: Los Angeles, California • Palo Alto, California • Minneapolis, Minnesota + 2 more

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

Conditions: GM1 Gangliosidosis, GM1 Gangliosidosis, Type I, GM1 Gangliosidosis, Type 2, Beta-Galactosidase-1 (GLB1) Deficiency
Interventions: PBGM01; Biological
Lead sponsor: Gemma Biotherapeutics; Industry
Eligibility: 1 Month to 24 Months
Enrollment: 26 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2029
U.S. locations: 4
States / cities: Oakland, California • Minneapolis, Minnesota • New Brunswick, New Jersey + 1 more

Conditions: Niemann-Pick Disease, Type C
Interventions: IB1001; Drug
Lead sponsor: IntraBio Inc; Industry
Eligibility: 6 Years and older
Enrollment: 33 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2022
U.S. locations: 1
States / cities: Rochester, Minnesota

Conditions: Type 2 Gaucher Disease
Interventions: Not listed
Lead sponsor: National Institute of Neurological Disorders and Stroke (NINDS); NIH
Eligibility: Up to 5 Years · Male only
Enrollment: 1 participant
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2005 – 2006
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Fabry Disease
Interventions: Migalastat HCl, Agalsidase Beta, Agalsidase Alfa; Drug · Biological
Lead sponsor: Amicus Therapeutics; Industry
Eligibility: 18 Years to 65 Years · Male only
Enrollment: 20 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2011 – 2012
U.S. locations: 5
States / cities: Birmingham, Alabama • Decatur, Georgia • Iowa City, Iowa + 2 more

Conditions: Late Infantile Neuronal Ceroid Lipofuscinosis, Batten Disease
Interventions: Group 1; Other
Lead sponsor: Weill Medical College of Cornell University; Other
Eligibility: 2 Years to 8 Years
Enrollment: 32 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2011 – 2015
U.S. locations: 1
States / cities: New York, New York

Conditions: Gaucher Disease, Type 1
Interventions: Eliglustat tartrate, Imiglucerase; Drug
Lead sponsor: Genzyme, a Sanofi Company; Industry
Eligibility: 18 Years and older
Enrollment: 160 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2009 – 2015
U.S. locations: 17
States / cities: Beverly Hills, California • San Francisco, California • Aurora, Colorado + 13 more

Conditions: Inherited Metabolic Disorders (IMD)
Interventions: MGTA-456; Drug
Lead sponsor: Magenta Therapeutics, Inc.; Industry
Eligibility: 0 Years to 17 Years
Enrollment: 8 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2021
U.S. locations: 2
States / cities: Minneapolis, Minnesota • Cincinnati, Ohio

Conditions: Fabry Disease
Interventions: agalsidase alfa; Biological
Lead sponsor: Shire; Industry
Eligibility: Not listed
U.S. locations: 25
States / cities: Tucson, Arizona • La Jolla, California • Los Angeles, California + 21 more

Conditions: Fabry Disease, Healthy
Interventions: Not listed
Lead sponsor: National Institute of Neurological Disorders and Stroke (NINDS); NIH
Eligibility: Male only
Enrollment: 24 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2000 – 2001
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Neurological Regression, Myoclonus, Cherry Red Spot, Brain Atrophy
Interventions: Not listed
Lead sponsor: National Human Genome Research Institute (NHGRI); NIH
Eligibility: 1 Day to 100 Years
Enrollment: 200 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: Started 2002
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Tay-Sachs Disease, Porphyria, Erythropoietic, Leukodystrophy, Globoid Cell, Metabolism, Inborn Errors
Interventions: Not listed
Lead sponsor: National Center for Research Resources (NCRR); NIH
Eligibility: 0 Years and older
Enrollment: 50 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: Started 1999
U.S. locations: 1
States / cities: New York, New York

Search by objective public record fields.

Evaluation of Phenotypic Variability in Fabry Disease

Severe Renal Disease Study in Fabry Patients Treated With Fabrazyme

Natural History Study for Pediatric Patients With Early Onset of Either GM1 Gangliosidosis, GM2 Gangliosidoses, or Gaucher Disease Type 2

Identification of Undiagnosed Lysosomal Acid Lipase Deficiency

Study of the Effects of Oral AT1001 (Migalastat Hydrochloride) in Patients With Fabry Disease

SCH-58235 (Ezetimibe) to Treat Homozygous Sitosterolemia

A Study of Patients With Fabry Disease (US Specific)

Open-label Long-term Safety Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease Who Have Completed a Previous AT1001 Study

PEG-Glucocerebrosidase for the Treatment of Gaucher Disease

The Natural History of Infantile Globoid Cell Leukodystrophy

Safety Study of Gene Modified Donor T Cell Infusion After Stem Cell Transplant for Non-Malignant Diseases

A Retrospective Natural History Study of Patients With Lysosomal Acid Lipase Deficiency/Wolman Phenotype

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

Study of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Participants With GM1 Gangliosidosis

N-Acetyl-L-Leucine for Niemann-Pick Disease, Type C (NPC)

Convection-Enhanced Delivery of Glucocerebrosidase to Treat Type 2 Gaucher Disease

Drug-Drug Interaction Study Between AT1001 (Migalastat Hydrochloride) and Agalsidase in Participants With Fabry Disease

Collection of Cerebrospinal Fluid in Healthy Children

A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease Who Have Reached Therapeutic Goals With Enzyme Replacement Therapy (ENCORE)

MGTA-456 in Patients With Inherited Metabolic Disorders Undergoing Hematopoietic Stem Cell Transplantation (HSCT)

Treatment Protocol of Replagal for Patients With Fabry Disease

PET Scans in Normal Volunteers and Patients With Fabry Disease

Natural History of Glycosphingolipid Storage Disorders and Glycoprotein Disorders

Diagnostic and Screening Study of Genetic Disorders