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ClinicalTrials.gov public records Last synced Oct 15, 2026, 2:37 PM EDT

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Showing 385–408 of 567 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Lysosomal Disease Clear all
Completed Phase 2 Interventional Results available

Study of BMN 110 in Pediatric Patients < 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome)

NCT01515956
View directory record Official record
Conditions
Mucopolysaccharidosis IVA, Morquio A Syndrome, MPS IVA
Interventions
BMN 110
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
Up to 5 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2016
U.S. locations
2
States / cities
Oakland, California • Manhasset, New York
Source: ClinicalTrials.gov public record
Updated Aug 10, 2017 · Synced Oct 15, 2026, 2:37 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 15, 2026, 2:37 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 15, 2026, 2:37 PM EDT
Completed Phase 1 Interventional Results available

Administration of IV Laronidase Post Bone Marrow Transplant in Hurler

NCT01173016
View directory record Official record
Conditions
Hurler Syndrome
Interventions
Laronidase
Drug
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 14 Years
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2016
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Mar 20, 2020 · Synced Oct 15, 2026, 2:37 PM 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 15, 2026, 2:37 PM EDT
Completed Phase 4 Interventional Accepts healthy volunteers

Curcumin (Tumeric) in the Treatment of Irritable Bowel Syndrome: A Randomized-Controlled Trial

NCT00779493
View directory record Official record
Conditions
Irritable Bowel Syndrome
Interventions
curcumin, placebo
Dietary Supplement
Lead sponsor
Kaiser Permanente
Other
Eligibility
18 Years and older
Enrollment
17 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2008 – 2009
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Mar 11, 2015 · Synced Oct 15, 2026, 2:37 PM EDT
Completed Phase 1Phase 2 Interventional Results available

First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221

NCT02675465
View directory record Official record
Conditions
Pompe Disease
Interventions
ATB200, AT2221
Drug
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
18 Years to 75 Years
Enrollment
29 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2024
U.S. locations
12
States / cities
Phoenix, Arizona • Orange, California • Gainesville, Florida + 9 more
Source: ClinicalTrials.gov public record
Updated Oct 23, 2025 · Synced Oct 15, 2026, 2:37 PM EDT
Completed Phase 2 Interventional Results available

Drug-drug Interaction Study

NCT01380743
View directory record Official record
Conditions
Pompe Disease
Interventions
duvoglustat, rhGAA
Drug
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
18 Years to 65 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2013
U.S. locations
11
States / cities
Phoenix, Arizona • Scottsdale, Arizona • Orange, California + 8 more
Source: ClinicalTrials.gov public record
Updated Jun 26, 2025 · Synced Oct 15, 2026, 2:37 PM EDT
Completed Phase 3 Interventional Results available

A Study of Two Fabrazyme (Agalsidase Beta) Dosing Regimens in Treatment-naïve, Male Pediatric Patients Without Severe Symptoms

NCT00701415
View directory record Official record
Conditions
Fabry Disease
Interventions
Agalsidase beta
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
5 Years to 18 Years · Male only
Enrollment
31 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2015
U.S. locations
3
States / cities
Decatur, Georgia • Cincinnati, Ohio • Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Jun 29, 2016 · Synced Oct 15, 2026, 2:37 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 15, 2026, 2:37 PM EDT
Completed No phase listed Observational

Diagnosis of Mucopolysaccharidosis Disorders in Patients Presenting With Bilateral Hip Disease

NCT01707433
View directory record Official record
Conditions
Mucopolysaccharidosis IV A, Mucopolysaccharidosis VI
Interventions
Enzyme testing
Other
Lead sponsor
Children's Hospitals and Clinics of Minnesota
Other
Eligibility
Up to 21 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2015
U.S. locations
2
States / cities
Minneapolis, Minnesota • Saint Paul, Minnesota
Source: ClinicalTrials.gov public record
Updated Aug 4, 2015 · Synced Oct 15, 2026, 2:37 PM EDT
Completed Phase 3 Interventional

A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease

NCT00074971
View directory record Official record
Conditions
Fabry Disease
Interventions
Fabrazyme (agalsidase beta)
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
16 Years and older
Enrollment
58 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1999 – 2004
U.S. locations
14
States / cities
Los Angeles, California • San Francisco, California • Coral Springs, Florida + 11 more
Source: ClinicalTrials.gov public record
Updated Dec 4, 2013 · Synced Oct 15, 2026, 2:37 PM EDT
Recruiting No phase listed Observational

A Global Prospective Observational Registry of Patients With Pompe Disease

NCT06121011
View directory record Official record
Conditions
Pompe Disease
Interventions
Cipaglucosidase alfa, Miglustat, Alglucosidase alfa or Avalglucosidase alfa, Untreated
Biological · Drug · Other
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
Not listed
Enrollment
500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2034
U.S. locations
15
States / cities
Little Rock, Arkansas • Irvine, California • Jacksonville, Florida + 10 more
Source: ClinicalTrials.gov public record
Updated Mar 10, 2026 · Synced Oct 15, 2026, 2:37 PM EDT
Completed No phase listed Observational Results available

Collection and Study of Cerebrospinal Fluid in Patients With Hunter Syndrome

NCT01449240
View directory record Official record
Conditions
Hunter Syndrome
Interventions
No treatment
Other
Lead sponsor
Shire
Industry
Eligibility
Up to 70 Years · Male only
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2013
U.S. locations
5
States / cities
Decatur, Georgia • Chicago, Illinois • Minneapolis, Minnesota + 2 more
Source: ClinicalTrials.gov public record
Updated Jun 9, 2021 · Synced Oct 15, 2026, 2:37 PM EDT
Withdrawn Phase 4 Interventional

The Effect of Velaglucerase Alfa (Vpriv) on Skeletal Development in Pediatric Gaucher Disease

NCT02528617
View directory record Official record
Conditions
Gaucher Disease Type 1, Gaucher Disease Type 3
Interventions
Velaglucerase alfa
Drug
Lead sponsor
Baylor Research Institute
Other
Eligibility
4 Years to 14 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Mar 2, 2018 · Synced Oct 15, 2026, 2:37 PM EDT
Completed Phase 1Phase 2 Interventional

Open-label Study of Anakinra in MPS III

NCT04018755
View directory record Official record
Conditions
Mucopolysaccharidosis III
Interventions
anakinra
Biological
Lead sponsor
Lynda E Polgreen
Other
Eligibility
4 Years and older
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2023
U.S. locations
1
States / cities
Torrance, California
Source: ClinicalTrials.gov public record
Updated Oct 22, 2024 · Synced Oct 15, 2026, 2:37 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers Results available

Assessment of Small Fiber Neuropathy in Rare Diseases Using Sudoscan

NCT02985710
View directory record Official record
Conditions
Small Fiber Neuropathy, Fabry Disease, Ehlers Danlos Syndrome, Mitochondrial Disease
Interventions
Sudoscan, Skin biopsy, QSART
Device · Procedure
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
8 Years to 70 Years
Enrollment
102 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2016 – 2020
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Oct 7, 2021 · Synced Oct 15, 2026, 2:37 PM EDT
Completed No phase listed Observational

Longitudinal Study of Cognition With Niemann-Pick Disease, Type C

NCT01899950
View directory record Official record
Conditions
Niemann-Pick Disease, Type C
Interventions
Not listed
Lead sponsor
Mayo Clinic
Other
Eligibility
2 Years to 99 Years
Enrollment
70 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2014
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Jan 6, 2016 · Synced Oct 15, 2026, 2:37 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Safety and Efficacy of Voxzogo for Growth Deficits in MPS IVA and VI

NCT05845749
View directory record Official record
Conditions
MPS IVA, MPS VI
Interventions
Vosoritide Injection [Voxzogo]
Drug
Lead sponsor
University of California, San Francisco
Other
Eligibility
5 Years to 10 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2029
U.S. locations
1
States / cities
Oakland, California
Source: ClinicalTrials.gov public record
Updated Mar 10, 2026 · Synced Oct 15, 2026, 2:37 PM EDT
Not yet recruiting Phase 1Phase 2 Interventional

Single Injection of rAAV9-CMV-hNAGLUop Gene for Patients With Mucopolysaccharidosis (MPS) IIIB

NCT07818759
View directory record Official record
Conditions
Sanfilippo Syndrome (MPS III)
Interventions
rAAV9-CMV-hNAGLUop
Biological
Lead sponsor
NeuroGT
Industry
Eligibility
6 Months and older
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2027 – 2029
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Sep 14, 2026 · Synced Oct 15, 2026, 2:37 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Pregnancy With Morquio Syndrome - What Are Patients' Perspectives and Has ERT Changed Them?

NCT03150069
View directory record Official record
Conditions
Morquio Disease, MPS - Mucopolysaccharidosis
Interventions
Interview
Other
Lead sponsor
Emory University
Other
Eligibility
15 Years to 100 Years · Female only
Enrollment
18 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2017 – 2018
U.S. locations
1
States / cities
Decatur, Georgia
Source: ClinicalTrials.gov public record
Updated Jan 20, 2021 · Synced Oct 15, 2026, 2:37 PM EDT
Completed No phase listed Observational

A Retrospective Chart Review of Deceased Patients With Mucopolysaccharidosis Type IIIB

NCT02293382
View directory record Official record
Conditions
MPS IIIB (Sanfilippo Syndrome)
Interventions
Not listed
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
Not listed
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2015
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Feb 3, 2017 · Synced Oct 15, 2026, 2:37 PM EDT
Active, not recruiting Phase 1 Interventional

Phase 1 Study of GC1130A in Patients With Sanfilippo Syndrome Type A (MPS IIIA)

NCT06567769
View directory record Official record
Conditions
Sanfilippo Syndrome Type A
Interventions
GC1130A
Drug
Lead sponsor
GC Biopharma Corp
Industry
Eligibility
12 Months to 18 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
2
States / cities
Oakland, California • Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Sep 8, 2026 · Synced Oct 15, 2026, 2:37 PM EDT