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ClinicalTrials.gov public records Last synced Oct 10, 2026, 5:53 PM EDT

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Showing 121–144 of 567 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Lysosomal Disease Clear all
Recruiting Phase 1 Interventional

UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells

NCT02254863
View directory record Official record
Conditions
Adrenoleukodystrophy, Batten Disease, Mucopolysaccharidosis II, Leukodystrophy, Globoid Cell, Leukodystrophy, Metachromatic, Neimann Pick Disease, Pelizaeus-Merzbacher Disease, Sandhoff Disease, Tay-Sachs Disease, Brain Diseases, Metabolic, Inborn, Alpha-Mannosidosis, Sanfilippo Mucopolysaccharidoses
Interventions
DUOC-01
Biological
Lead sponsor
Joanne Kurtzberg, MD
Other
Eligibility
1 Week to 22 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2027
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Sep 3, 2026 · Synced Oct 10, 2026, 5:53 PM EDT
Completed No phase listed Observational

GM1 and GM2 Gangliosidosis PROspective Neurological Disease TrajectOry Study (PRONTO)

NCT05109793
View directory record Official record
Conditions
GM1 Gangliosidosis, Sandhoff Disease, Tay-Sachs Disease
Interventions
Not listed
Lead sponsor
Azafaros B.V.
Industry
Eligibility
2 Years to 20 Years
Enrollment
31 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
2
States / cities
Oakland, California • Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Sep 29, 2025 · Synced Oct 10, 2026, 5:53 PM EDT
Completed No phase listed Observational

Gait Analysis in MPS IVA

NCT01920828
View directory record Official record
Conditions
MPS IVA, Morquio Syndrome
Interventions
Not listed
Lead sponsor
Oregon Health and Science University
Other
Eligibility
1 Year and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2017
U.S. locations
1
States / cities
Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Oct 17, 2019 · Synced Oct 10, 2026, 5:53 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 10, 2026, 5:53 PM 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 10, 2026, 5:53 PM EDT
Completed Phase 2 Interventional Results available

Safety and Efficacy Study of Sebelipase Alfa in Participants With Lysosomal Acid Lipase Deficiency

NCT02112994
View directory record Official record
Conditions
Lysosomal Acid Lipase Deficiency
Interventions
Sebelipase Alfa
Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
8 Months and older
Enrollment
31 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2017
U.S. locations
3
States / cities
Chicago, Illinois • Shreveport, Louisiana • Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Dec 4, 2019 · Synced Oct 10, 2026, 5:53 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Pilot Study of Safety, Tolerability, Pharmacokinetics/Pharmacodynamics of RP103 Compared to Cystagon® in Patients With Cystinosis

NCT00872729
View directory record Official record
Conditions
Cystinosis
Interventions
Cystagon®, RP103
Drug
Lead sponsor
Amgen
Industry
Eligibility
Not listed
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009
U.S. locations
1
States / cities
San Diego, California
Source: ClinicalTrials.gov public record
Updated Dec 19, 2024 · Synced Oct 10, 2026, 5:53 PM EDT
Terminated No phase listed Observational

Screening for Lysosomal Acid Lipase Deficiency

NCT02926872
View directory record Official record
Conditions
Lysosomal Acid Lipase Deficiency
Interventions
There is no intervention in the study
Other
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
2 Years to 16 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2017
U.S. locations
2
States / cities
Atlanta, Georgia • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jun 16, 2017 · Synced Oct 10, 2026, 5:53 PM EDT
Recruiting No phase listed Observational

Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC

NCT04528355
View directory record Official record
Conditions
Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias, Inflammatory Conditions
Interventions
data collection
Drug
Lead sponsor
Paul Szabolcs
Other
Eligibility
2 Months to 60 Years
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2028
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jan 13, 2026 · Synced Oct 10, 2026, 5:53 PM EDT
Terminated Phase 1Phase 2 Interventional Results available

Open Label, Study Of Efficacy and Safety Of AVR-RD-01 for Treatment-Naive Subjects With Classic Fabry Disease

NCT03454893
View directory record Official record
Conditions
Fabry Disease
Interventions
AVR-RD-01
Drug
Lead sponsor
AVROBIO
Industry
Eligibility
16 Years to 50 Years · Male only
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2022
U.S. locations
2
States / cities
Hackensack, New Jersey • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jan 5, 2024 · Synced Oct 10, 2026, 5:53 PM EDT
Completed Phase 2 Interventional

Extension Study of Long-term Safety and Efficacy of Myozyme in Patients With Pompe Disease Who Were Previously Enrolled in Genzyme Sponsored Enzyme Replacement Therapy (ERT) Studies

NCT00763932
View directory record Official record
Conditions
Pompe Disease Infantile-Onset, Glycogen Storage Disease Type II
Interventions
Myozyme
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
18 Years and older
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2006
U.S. locations
5
States / cities
Oakland, California • Orange, California • Omaha, Nebraska + 2 more
Source: ClinicalTrials.gov public record
Updated Feb 5, 2014 · Synced Oct 10, 2026, 5:53 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Safety, Tolerability, PK, and Efficacy Evaluation of Repeat Ascending Doses of Olipudase Alfa in Pediatric Patients <18 Years of Age With Acid Sphingomyelinase Deficiency

NCT02292654
View directory record Official record
Conditions
Sphingomyelin Lipidosis
Interventions
Olipudase alfa
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
Up to 17 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2019
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Mar 23, 2022 · Synced Oct 10, 2026, 5:53 PM EDT
Completed Phase 2 Interventional Results available

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

NCT03406962
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Nov 3, 2021 · Synced Oct 10, 2026, 5:53 PM EDT
Completed Phase 3 Interventional Results available

Efficacy and Safety of Lucerastat Oral Monotherapy in Adult Subjects With Fabry Disease

NCT03425539
View directory record Official record
Conditions
Fabry Disease
Interventions
Lucerastat, Placebo
Drug
Lead sponsor
Idorsia Pharmaceuticals Ltd.
Industry
Eligibility
18 Years and older
Enrollment
118 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
17
States / cities
Birmingham, Alabama • Irvine, California • Oakland, California + 14 more
Source: ClinicalTrials.gov public record
Updated Aug 9, 2024 · Synced Oct 10, 2026, 5:53 PM EDT
Terminated No phase listed Observational

A Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)

NCT00787995
View directory record Official record
Conditions
MPS IV A, Mucopolysaccharidosis IVA, Morquio A Syndrome
Interventions
Not listed
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
Not listed
Enrollment
353 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2014
U.S. locations
2
States / cities
Oakland, California • Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Dec 8, 2014 · Synced Oct 10, 2026, 5:53 PM EDT
Terminated Phase 1Phase 2 Interventional Results available

Lentiviral Vector Gene Therapy - The Guard1 Trial of AVR-RD-02 for Subjects With Type 1 Gaucher Disease

NCT04145037
View directory record Official record
Conditions
Gaucher Disease
Interventions
AVR-RD-02
Drug
Lead sponsor
AVROBIO
Industry
Eligibility
18 Years to 50 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2023
U.S. locations
4
States / cities
San Diego, California • Iowa City, Iowa • Hackensack, New Jersey + 1 more
Source: ClinicalTrials.gov public record
Updated Jan 18, 2024 · Synced Oct 10, 2026, 5:53 PM EDT
Completed Phase 2 Interventional Results available

A Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease

NCT00214500
View directory record Official record
Conditions
Fabry Disease
Interventions
migalastat HCl
Drug
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
18 Years to 55 Years · Male only
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2008
U.S. locations
5
States / cities
Los Angeles, California • Decatur, Georgia • Bethesda, Maryland + 2 more
Source: ClinicalTrials.gov public record
Updated Oct 30, 2018 · Synced Oct 10, 2026, 5:53 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

Safety and Pharmacokinetics of AT1001 (Migalastat HCl) in Healthy Subjects and Subjects With Impaired Renal Function

NCT01730469
View directory record Official record
Conditions
Fabry Disease
Interventions
AT1001 150 mg
Drug
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
18 Years to 75 Years
Enrollment
32 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2011 – 2012
U.S. locations
4
States / cities
Costa Mesa, California • Miami, Florida • Orlando, Florida
Source: ClinicalTrials.gov public record
Updated Aug 3, 2017 · Synced Oct 10, 2026, 5:53 PM EDT
Completed No phase listed Observational

Farber Disease Natural History Study

NCT03233841
View directory record Official record
Conditions
Farber Disease, Farber's Disease, Farber Lipogranulomatosis, Acid Ceramidase Deficiency, Ceramidase Deficiency, N-Laurylsphingosine Deacylase Deficiency, ASAH1 Mutation
Interventions
Not listed
Lead sponsor
Sumitomo Pharma Switzerland GmbH
Industry
Eligibility
Not listed
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2019
U.S. locations
2
States / cities
Oakland, California • Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Jan 18, 2020 · Synced Oct 10, 2026, 5:53 PM EDT
Recruiting Phase 4 Interventional Accepts healthy volunteers

Effects of Bisphosphonates on OI-Related Hearing Loss

NCT04152551
View directory record Official record
Conditions
Osteogenesis Imperfecta
Interventions
Risedronate Oral Tablet
Drug
Lead sponsor
Hospital for Special Surgery, New York
Other
Eligibility
6 Years to 100 Years
Enrollment
100 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2027
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Apr 28, 2026 · Synced Oct 10, 2026, 5:53 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

SPL Insufficiency Syndrome (SPLIS)/NPHS14: a SPLIS Observational Study and Patient Registry (International)

NCT04885179
View directory record Official record
Conditions
Sphingolipidoses, Enzyme Deficiency
Interventions
no intervention
Other
Lead sponsor
University of California, San Francisco
Other
Eligibility
Not listed
Enrollment
120 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2025 – 2028
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Oct 24, 2025 · Synced Oct 10, 2026, 5:53 PM EDT
No Longer Available No phase listed Expanded access

Single Patient Expanded Access Protocol: Metabolic Boost

NCT02021266
View directory record Official record
Conditions
Metachromatic Leukodystrophy
Interventions
Enriched Hematopoetic Stem Cell Infusion
Biological
Lead sponsor
Talaris Therapeutics Inc.
Industry
Eligibility
Female only
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Oct 8, 2020 · Synced Oct 10, 2026, 5:53 PM EDT
Completed No phase listed Observational

The Natural History and Pathogenesis of Mucolipidosis Type IV

NCT00015782
View directory record Official record
Conditions
Mucolipidosis Type IV
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Not listed
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2008
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 2, 2017 · Synced Oct 10, 2026, 5:53 PM EDT
Completed Phase 4 Interventional Results available

A Study Evaluating Glycosphingolipid Clearance in Patients Treated With Agalsidase Alfa Who Switch to Agalsidase Beta

NCT01650779
View directory record Official record
Conditions
Fabry Disease
Interventions
Agalsidase beta
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
Male only
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2013
U.S. locations
6
States / cities
Coral Springs, Florida • Decatur, Georgia • Baltimore, Maryland + 3 more
Source: ClinicalTrials.gov public record
Updated Jul 10, 2014 · Synced Oct 10, 2026, 5:53 PM EDT