Independent directory Public ClinicalTrials.gov records United States
Clinical Trials Finder
Objective public record navigation
Search Results

Search by objective public record fields.

Search by query text, NCT ID, condition, intervention, sponsor, city, state, recruitment status, phase, study type, healthy volunteer eligibility, sex, or age. Results come from ClinicalTrials.gov. When the live registry is unavailable, a clearly labeled stored copy may be shown.

Clear filters

These results were retrieved from the live ClinicalTrials.gov registry. Filters and sort order apply to this result list.

ClinicalTrials.gov public records Last synced Oct 22, 2026, 8:21 PM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Showing 1–24 of 558 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Lysosomal Storage Disorder Clear all
Not listed No phase listed Observational

Podocyturia - Predictor of Renal Dysfunction in Fabry Nephropathy

NCT02994303
View directory record Official record
Conditions
Fabry Disease
Interventions
Not listed
Lead sponsor
University of Washington
Other
Eligibility
1 Day to 90 Years
Enrollment
58 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2019
U.S. locations
3
States / cities
Decatur, Georgia • Minneapolis, Minnesota • Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Aug 28, 2017 · Synced Oct 22, 2026, 8:21 PM EDT
Completed Phase 3 Interventional Results available

Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients With MPS VI

NCT00067470
View directory record Official record
Conditions
Mucopolysaccharidosis VI
Interventions
Placebo, N-acetylgalactosamine 4-sulfatase
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
7 Years and older
Enrollment
39 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2004
U.S. locations
1
States / cities
Novato, California
Source: ClinicalTrials.gov public record
Updated Dec 24, 2009 · Synced Oct 22, 2026, 8:21 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 22, 2026, 8:21 PM EDT
Completed Phase 1Phase 2 Interventional Results available

A Study to Assess the Safety and Tolerability of SOBI003 in Pediatric MPS IIIA Patients

NCT03423186
View directory record Official record
Conditions
Sanfilippo Syndrome Type A (MPS IIIA)
Interventions
SOBI003
Drug
Lead sponsor
Swedish Orphan Biovitrum
Industry
Eligibility
12 Months to 72 Months
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2019
U.S. locations
2
States / cities
Oakland, California • Chapel Hill, North Carolina
Source: ClinicalTrials.gov public record
Updated Nov 19, 2021 · Synced Oct 22, 2026, 8:21 PM 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 22, 2026, 8:21 PM EDT
Completed No phase listed Observational

Genzyme Osteopenia/Osteoporosis Study

NCT02785744
View directory record Official record
Conditions
Gaucher Disease
Interventions
Gaucher disease DNA mutation analysis
Other
Lead sponsor
NYU Langone Health
Other
Eligibility
18 Years and older
Enrollment
76 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Mar 23, 2021 · Synced Oct 22, 2026, 8:21 PM 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 22, 2026, 8:21 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

Safety and Effect of L. Reuteri on Biomarkers of Inflammation in Healthy Infants With Colic

NCT01849991
View directory record Official record
Conditions
Gastrointestinal, Colic
Interventions
Lactobacillus reuteri, Placebo
Biological
Lead sponsor
The University of Texas Health Science Center, Houston
Other
Eligibility
3 Weeks to 3 Months
Enrollment
21 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2016
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jan 30, 2018 · Synced Oct 22, 2026, 8:21 PM EDT
Completed No phase listed Observational

Pompe Telemedicine Developmental Study

NCT02950298
View directory record Official record
Conditions
Pompe Disease, Glycogen Storage Disease II
Interventions
Not listed
Lead sponsor
Duke University
Other
Eligibility
6 Years to 18 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2021
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Oct 28, 2021 · Synced Oct 22, 2026, 8:21 PM EDT
Active, not recruiting No phase listed Observational

Long-Term Follow-Up of Subjects Treated With AXO-AAV-GM2 for Tay-Sachs or Sandhoff Disease

NCT06614569
View directory record Official record
Conditions
GM2 Gangliosidosis, Tay Sachs Disease, Sandhoff Disease
Interventions
AXO-AAV-GM2
Drug
Lead sponsor
Terence Flotte
Other
Eligibility
Not listed
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2028
U.S. locations
1
States / cities
Worcester, Massachusetts
Source: ClinicalTrials.gov public record
Updated Oct 16, 2025 · Synced Oct 22, 2026, 8:21 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 22, 2026, 8:21 PM EDT
Completed Phase 2 Interventional

A Protocol to Allow Treatment With ICL670 for Patients With or at Risk of Life-threatening Complications of Transfusional Iron Overload Who Are Unable to Tolerate Other Iron Chelators Because of Documented Severe Toxicity

NCT01044186
View directory record Official record
Conditions
Transfusional Iron Overload
Interventions
ICL670
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
2 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2003
U.S. locations
8
States / cities
Oakland, California • Orange, California • Boston, Massachusetts + 4 more
Source: ClinicalTrials.gov public record
Updated Feb 23, 2017 · Synced Oct 22, 2026, 8:21 PM EDT
Completed Phase 3 Interventional Results available

A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)

NCT01275066
View directory record Official record
Conditions
MPS IV A
Interventions
BMN 110 Weekly, Placebo, BMN 110 Every Other Week
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
5 Years and older
Enrollment
177 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2012
U.S. locations
6
States / cities
Oakland, California • Wilmington, Delaware • Washington D.C., District of Columbia + 3 more
Source: ClinicalTrials.gov public record
Updated Jul 7, 2014 · Synced Oct 22, 2026, 8:21 PM EDT
Terminated Phase 1Phase 2 Interventional

A Safety and Efficacy Study of LYS-GM101 Gene Therapy in Patients With GM1 Gangliosidosis

NCT04273269
View directory record Official record
Conditions
GM1 Gangliosidosis
Interventions
LYS-GM101
Genetic
Lead sponsor
LYSOGENE
Industry
Eligibility
Up to 3 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
1
States / cities
Orange, California
Source: ClinicalTrials.gov public record
Updated Jun 9, 2023 · Synced Oct 22, 2026, 8:21 PM EDT
Not listed No phase listed Observational Accepts healthy volunteers

Immune Biomarkers Related to Bone Pathology in Patients With Type 1 Gaucher Disease

NCT04055831
View directory record Official record
Conditions
Gaucher Disease Type 1
Interventions
Not listed
Lead sponsor
Lysosomal and Rare Disorders Research and Treatment Center, Inc.
Other
Eligibility
16 Years to 90 Years
Enrollment
40 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2020
U.S. locations
1
States / cities
Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Aug 15, 2019 · Synced Oct 22, 2026, 8:21 PM 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 22, 2026, 8:21 PM EDT
Completed Not applicable Interventional

A Prospective and Retrospective Cohort Study in Patients With Chronic Forms of Acid Sphingomyelinase Deficiency (ASMD)

NCT04106544
View directory record Official record
Conditions
Sphingomyelin Lipidosis
Interventions
Investigational Procedures
Procedure
Lead sponsor
Sanofi
Industry
Eligibility
Not listed
Enrollment
84 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2023
U.S. locations
3
States / cities
Atlanta, Georgia • The Bronx, New York • Valhalla, New York
Source: ClinicalTrials.gov public record
Updated Aug 16, 2023 · Synced Oct 22, 2026, 8:21 PM EDT
Completed Not applicable Interventional

Implementation and Evaluation of a Rare Disease Algorithm to Identify Persons at Risk of Gaucher Disease Using Data From Electronic Health Records (EHRs) in the United States (Project Searchlight)

NCT05908656
View directory record Official record
Conditions
Gaucher Disease
Interventions
Investigational procedure
Procedure
Lead sponsor
Sanofi
Industry
Eligibility
Not listed
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024
U.S. locations
3
States / cities
Gainesville, Florida • New York, New York • Milwaukee, Wisconsin
Source: ClinicalTrials.gov public record
Updated Nov 19, 2024 · Synced Oct 22, 2026, 8:21 PM EDT
Completed No phase listed Observational

Identification of Tongue Involvement in Late-Onset Pompe Disease

NCT02765828
View directory record Official record
Conditions
Myopathy, Neuropathy, Glycogen Storage Disease Type II (Late-onset Pompe Disease)
Interventions
Observational study
Other
Lead sponsor
Duke University
Other
Eligibility
12 Years and older
Enrollment
73 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Jul 6, 2021 · Synced Oct 22, 2026, 8:21 PM EDT
Enrolling by invitation Phase 2Phase 3 Interventional

An Extension Study of the Long-Term Safety, Tolerability, and Efficacy of Tividenofusp Alfa (DNL310) in Participants With Mucopolysaccharidosis Type II (MPS II) From Study DNLI-E-0002 or Study DNLI-E-0007

NCT06075537
View directory record Official record
Conditions
Mucopolysaccharidosis II
Interventions
tividenofusp alfa
Drug
Lead sponsor
Denali Therapeutics Inc.
Industry
Eligibility
Up to 18 Years
Enrollment
99 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2027
U.S. locations
8
States / cities
Oakland, California • Chicago, Illinois • Hackensack, New Jersey + 5 more
Source: ClinicalTrials.gov public record
Updated Jun 11, 2026 · Synced Oct 22, 2026, 8:21 PM EDT
Completed Phase 1Phase 2 Interventional

Prophylactic Ranibizumab for Exudative Age-related Macular Degeneration

NCT02140151
View directory record Official record
Conditions
Age-related Macular Degeneration
Interventions
Ranibizumab 0.5mg
Drug
Lead sponsor
Southern California Desert Retina Consultants, MC
Other
Eligibility
50 Years and older
Enrollment
108 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2020
U.S. locations
4
States / cities
Mountain View, California • Palm Desert, California • Baltimore, Maryland + 1 more
Source: ClinicalTrials.gov public record
Updated Aug 25, 2022 · Synced Oct 22, 2026, 8:21 PM EDT
Completed No phase listed Observational

The GBA Multimodal Study in Parkinson's Disease

NCT04101968
View directory record Official record
Conditions
Parkinson Disease, GBA Gene Mutation, Gaucher Disease
Interventions
PET scan, neuroQWERTY
Diagnostic Test
Lead sponsor
Pacific Parkinson's Research Centre
Other
Eligibility
18 Years to 80 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2026
U.S. locations
2
States / cities
Portland, Oregon • Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Sep 2, 2026 · Synced Oct 22, 2026, 8:21 PM EDT
Completed Not applicable Interventional

Effect of Warfarin in the Treatment of Metachromatic Leukodystrophy

NCT00683189
View directory record Official record
Conditions
Metachromatic Leukodystrophy
Interventions
Warfarin
Drug
Lead sponsor
The Cooper Health System
Other
Eligibility
1 Year to 10 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2008
U.S. locations
1
States / cities
Camden, New Jersey
Source: ClinicalTrials.gov public record
Updated Mar 21, 2011 · Synced Oct 22, 2026, 8:21 PM EDT
Completed No phase listed Observational

Study to Collect Data on Fabry Disease Patients With Enhanceable Alpha-Galactosidase A Activity

NCT00106912
View directory record Official record
Conditions
Fabry Disease
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
18 Years and older · Male only
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2008
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 2, 2017 · Synced Oct 22, 2026, 8:21 PM EDT