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ClinicalTrials.gov public records Last synced Oct 2, 2026, 6:23 AM EDT

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Showing 1–24 of 241 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Disorder of Lipid Storage and Metabolism Clear all
Completed Phase 2 Interventional Results available

A Long-Term Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency

NCT02004704
View directory record Official record
Conditions
Sphingomyelin Lipidosis
Interventions
GZ402665
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
Not listed
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2023
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jul 3, 2024 · Synced Oct 2, 2026, 6:23 AM EDT
Active, not recruiting Phase 3 Interventional

Study to Evaluate the Efficacy and Safety of Venglustat in Adult and Pediatric Patients With Gaucher Disease Type 3

NCT05222906
View directory record Official record
Conditions
Gaucher's Disease Type III
Interventions
Venglustat, imiglucerase
Drug
Lead sponsor
Sanofi
Industry
Eligibility
12 Years and older
Enrollment
43 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
4
States / cities
New Haven, Connecticut • Iowa City, Iowa • Dallas, Texas + 1 more
Source: ClinicalTrials.gov public record
Updated Jul 13, 2026 · Synced Oct 2, 2026, 6:23 AM EDT
Completed Phase 3 Interventional

Open Label Extension of 2 mg/kg Pegunigalsidase Alfa (PRX-102) Every 4 Weeks in Adult Fabry Disease Patients

NCT03614234
View directory record Official record
Conditions
Fabry Disease
Interventions
pegunigalsidase alfa
Drug
Lead sponsor
Chiesi Farmaceutici S.p.A.
Industry
Eligibility
18 Years and older
Enrollment
29 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2026
U.S. locations
7
States / cities
Birmingham, Alabama • Atlanta, Georgia • Iowa City, Iowa + 4 more
Source: ClinicalTrials.gov public record
Updated May 22, 2026 · Synced Oct 2, 2026, 6:23 AM EDT
Completed Phase 3 Interventional Results available

A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease to Evaluate Once Daily Versus Twice Daily Dosing (EDGE)

NCT01074944
View directory record Official record
Conditions
Gaucher Disease
Interventions
Eliglustat tartrate
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
18 Years and older
Enrollment
170 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2015
U.S. locations
10
States / cities
San Diego, California • New Haven, Connecticut • Decatur, Georgia + 6 more
Source: ClinicalTrials.gov public record
Updated Feb 6, 2017 · Synced Oct 2, 2026, 6:23 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Evaluation of Biochemical Markers and Clinical Investigation of Niemann-Pick Disease, Type C

NCT00344331
View directory record Official record
Conditions
Niemann-Pick Disease, Type C
Interventions
Not listed
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
1 Day to 120 Years
Enrollment
900 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 2006
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 28, 2026 · Synced Oct 2, 2026, 6:23 AM 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 2, 2026, 6:23 AM EDT
Active, not recruiting No phase listed Observational Accepts healthy volunteers

Diffusion Tensor Imaging (DTI) in Infants With Krabbe Disease

NCT00787865
View directory record Official record
Conditions
Krabbe Disease
Interventions
Not listed
Lead sponsor
University of Pittsburgh
Other
Eligibility
Up to 17 Years
Enrollment
100 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2008 – 2027
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 6, 2026 · Synced Oct 2, 2026, 6:23 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Dose-Ranging Study of ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy in Subjects With Fabry Disease (STAAR)

NCT04046224
View directory record Official record
Conditions
Fabry Disease
Interventions
ST-920
Biological
Lead sponsor
Sangamo Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2025
U.S. locations
9
States / cities
Irvine, California • Tampa, Florida • Atlanta, Georgia + 6 more
Source: ClinicalTrials.gov public record
Updated Apr 14, 2026 · Synced Oct 2, 2026, 6:23 AM EDT
Not listed Not applicable Interventional

Identification of Undiagnosed Gaucher Disease

NCT01716741
View directory record Official record
Conditions
Gaucher Disease
Interventions
Enzyme analysis
Other
Lead sponsor
Brigham and Women's Hospital
Other
Eligibility
18 Years and older
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2012
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Oct 30, 2012 · Synced Oct 2, 2026, 6:23 AM EDT
Terminated Phase 2Phase 3 Interventional Results available

Adrabetadex to Treat Niemann-Pick Type C1 (NPC1) Disease

NCT04958642
View directory record Official record
Conditions
Niemann-Pick Disease, Type C
Interventions
Adrabetadex
Drug
Lead sponsor
Mandos LLC
Industry
Eligibility
4 Years to 21 Years
Enrollment
66 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2022
U.S. locations
15
States / cities
Birmingham, Alabama • Orange, California • San Francisco, California + 12 more
Source: ClinicalTrials.gov public record
Updated Aug 29, 2023 · Synced Oct 2, 2026, 6:23 AM EDT
Terminated Phase 1 Interventional

A Study in Type 1 Gaucher Patients to Evaluate the Pharmacokinetics, Safety and Pharmacodynamics of AT2101

NCT00875160
View directory record Official record
Conditions
Type 1 Gaucher Disease
Interventions
AT2101
Drug
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
18 Years to 65 Years
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009
U.S. locations
1
States / cities
Miramar, Florida
Source: ClinicalTrials.gov public record
Updated Aug 19, 2010 · Synced Oct 2, 2026, 6:23 AM EDT
Recruiting Phase 2 Interventional

Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT

NCT01962415
View directory record Official record
Conditions
Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias, Inflammatory Conditions, Systemic Juvenile Idiopathic Arthritis (sJIA), Juvenile Rheumatoid Arthritis (JRA)
Interventions
Hydroxyurea, Alemtuzumab, Fludarabine, Melphalan, Thiotepa
Drug
Lead sponsor
Paul Szabolcs
Other
Eligibility
2 Months to 55 Years
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2027
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Dec 15, 2025 · Synced Oct 2, 2026, 6:23 AM EDT
Completed Not applicable Interventional Results available

Reduced-Intensity Hematopoietic Stem Cell Transplant for High Risk Lysosomal and Peroxisomal Disorders

NCT01626092
View directory record Official record
Conditions
Lysosomal Storage Disease, Peroxisomal Disorder
Interventions
Campath-1H, Clofarabine, Melphalan, Total Body Irradiation with Marrow Boosting, Hematopoietic stem cell transplantation, Cyclosporine A, Mycophenolate mofetil
Drug · Radiation · Biological
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 55 Years
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2013
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Dec 5, 2017 · Synced Oct 2, 2026, 6:23 AM EDT
Completed Phase 2Phase 3 Interventional Accepts healthy volunteers Results available

Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency

NCT02004691
View directory record Official record
Conditions
Sphingomyelin Lipidosis
Interventions
placebo (saline), Olipudase alfa
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
18 Years and older
Enrollment
36 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2023
U.S. locations
3
States / cities
San Francisco, California • Decatur, Georgia • The Bronx, New York
Source: ClinicalTrials.gov public record
Updated Nov 7, 2024 · Synced Oct 2, 2026, 6:23 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 2, 2026, 6:23 AM EDT
Terminated Phase 2 Interventional Results available

Severe Renal Disease Study in Fabry Patients Treated With Fabrazyme

NCT00837824
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Apr 7, 2015 · Synced Oct 2, 2026, 6:23 AM EDT
Suspended Phase 1Phase 2 Interventional

Clinical Trial of Ambroxol in Patients With Type I Gaucher Disease

NCT01463215
View directory record Official record
Conditions
Type I Gaucher Disease
Interventions
Ambroxol
Drug
Lead sponsor
Exsar Corporation
Industry
Eligibility
16 Years and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2015
U.S. locations
1
States / cities
Monmouth Junction, New Jersey
Source: ClinicalTrials.gov public record
Updated Feb 15, 2013 · Synced Oct 2, 2026, 6:23 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Safety, Tolerability and Pharmacokinetics of SBC-102 (Sebelipase Alfa) in Adult Participants With Lysosomal Acid Lipase Deficiency

NCT01307098
View directory record Official record
Conditions
Cholesterol Ester Storage Disease(CESD), Lysosomal Acid Lipase Deficiency, LAL-Deficiency
Interventions
Sebelipase alfa 0.35 mg/kg, Sebelipase alfa 1 mg/kg, Sebelipase alfa 3 mg/kg
Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
18 Years to 65 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2012
U.S. locations
3
States / cities
Stanford, California • New York, New York • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Dec 11, 2018 · Synced Oct 2, 2026, 6:23 AM EDT
Completed Phase 2Phase 3 Interventional Results available

Stem Cell Transplant for Inborn Errors of Metabolism

NCT00176904
View directory record Official record
Conditions
Adrenoleukodystrophy, Metachromatic Leukodystrophy, Globoid Cell Leukodystrophy, Gaucher's Disease, Fucosidosis, Wolman Disease, Niemann-Pick Disease, Batten Disease, GM1 Gangliosidosis, Tay Sachs Disease, Sandhoff Disease
Interventions
Stem Cell Transplant, Busulfan, Cyclophosphamide, Antithymocyte Globulin
Procedure · Drug
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Not listed
Enrollment
135 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1995 – 2010
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Dec 28, 2017 · Synced Oct 2, 2026, 6:23 AM EDT
Completed Phase 4 Interventional Results available

A Study of the Safety and Efficacy of Fabrazyme (Agalsidase Beta) as Compared to Placebo in Patients With Advanced Fabry Disease

NCT00074984
View directory record Official record
Conditions
Fabry Disease
Interventions
Fabrazyme (agalsidase beta), Placebo
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
16 Years and older
Enrollment
82 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2004
U.S. locations
19
States / cities
Birmingham, Alabama • Los Angeles, California • San Francisco, California + 16 more
Source: ClinicalTrials.gov public record
Updated Dec 27, 2013 · Synced Oct 2, 2026, 6:23 AM EDT
Recruiting No phase listed Observational

Longitudinal Study of Neurodegenerative Disorders

NCT03333200
View directory record Official record
Conditions
MLD, Krabbe Disease, ALD, MPS I, MPS II, MPS III, Vanishing White Matter Disease, GM3 Gangliosidosis, PKAN, Tay-Sachs Disease, NP Deficiency, Osteopetrosis, Alpha-Mannosidosis, Sandhoff Disease, Niemann-Pick Diseases, MPS IV, Gaucher Disease, GAN, GM1 Gangliosidoses, Morquio Disease, S-Adenosylhomocysteine Hydrolase Deficiency, Batten Disease, Pelizaeus-Merzbacher Disease, Leukodystrophy, Lysosomal Storage Diseases, Purine Nucleoside Phosphorylase Deficiency, Multiple Sulfatase Deficiency Disease
Interventions
Palliative Care, Hematopoetic Stem Cell Transplantation
Other · Biological
Lead sponsor
University of Pittsburgh
Other
Eligibility
Not listed
Enrollment
1,500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2035
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Feb 9, 2026 · Synced Oct 2, 2026, 6:23 AM 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 2, 2026, 6:23 AM EDT
Completed No phase listed Observational

Natural History Study of Infantile and Juvenile GM1 Gangliosidosis (GM1) Patients

NCT04041102
View directory record Official record
Conditions
GM1 Gangliosidosis
Interventions
Not listed
Lead sponsor
University of Pennsylvania
Other
Eligibility
Not listed
Enrollment
31 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2024
U.S. locations
2
States / cities
Oakland, California • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Oct 24, 2025 · Synced Oct 2, 2026, 6:23 AM EDT