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ClinicalTrials.gov public records Last synced Oct 10, 2026, 12:58 PM EDT

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Showing 97–120 of 567 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Lysosomal Disease Clear all
Completed No phase listed Observational

Observational Study of Patients With Mucopolysaccharidosis (MPS) VI Who Previously Participated in ASB-00-02

NCT01387854
View directory record Official record
Conditions
MPS VI
Interventions
Not listed
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
Not listed
Enrollment
59 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2013
U.S. locations
1
States / cities
Oakland, California
Source: ClinicalTrials.gov public record
Updated Apr 2, 2014 · Synced Oct 10, 2026, 12:58 PM EDT
Completed No phase listed Observational

Gene Therapy for Tay-Sachs Disease

NCT01869270
View directory record Official record
Conditions
Tay Sachs Disease, Sandhoff Disease, Late Onset Tay Sachs Disease
Interventions
Not listed
Lead sponsor
University of Minnesota
Other
Eligibility
Not listed
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2014
U.S. locations
2
States / cities
Tampa, Florida • Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Dec 4, 2014 · Synced Oct 10, 2026, 12:58 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

PET Scans in Normal Volunteers and Patients With Fabry Disease

NCT00005111
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 10, 2026, 12:58 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 10, 2026, 12:58 PM EDT
Completed Phase 3 Interventional Results available

Study of the Safety and Efficacy of PRX-102 Compared to Agalsidase Beta on Renal Function

NCT02795676
View directory record Official record
Conditions
Fabry Disease
Interventions
PRX-102 (pegunigalsidase alfa), agalsidase beta
Biological
Lead sponsor
Protalix
Industry
Eligibility
18 Years to 60 Years
Enrollment
78 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2022
U.S. locations
15
States / cities
Birmingham, Alabama • Phoenix, Arizona • Orange, California + 11 more
Source: ClinicalTrials.gov public record
Updated Sep 13, 2023 · Synced Oct 10, 2026, 12:58 PM EDT
Completed No phase listed Observational

Immune Response in Subjects With Fabry Disease Who Are Switching From Agalsidase Alfa to Agalsidase Beta

NCT01745185
View directory record Official record
Conditions
Fabry Disease
Interventions
Not listed
Lead sponsor
O & O Alpan LLC
Other
Eligibility
7 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2016
U.S. locations
1
States / cities
Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Apr 4, 2017 · Synced Oct 10, 2026, 12:58 PM EDT
Completed Phase 2 Interventional Results available

Stem Cell Transplantation for Hurler

NCT00176917
View directory record Official record
Conditions
Mucopolysaccharidosis I, Mucopolysaccharidosis VI, Mannosidosis, Mucolipidosis Type II (I-cell Disease)
Interventions
Stem Cell Transplant, Busulfan, Cyclophosphamide, ATG
Procedure · Drug
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Not listed
Enrollment
41 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1999 – 2010
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Dec 28, 2017 · Synced Oct 10, 2026, 12:58 PM EDT
Active, not recruiting Phase 3 Interventional

A Study to Evaluate the Effect of Venglustat Tablets on Left Ventricular Mass Index in Male and Female Adult Participants With Fabry Disease

NCT05280548
View directory record Official record
Conditions
Fabry Disease
Interventions
Venglustat (GZ402671), Agalsidase alfa, Agalsidase beta (GZ419828), Migalastat
Drug
Lead sponsor
Sanofi
Industry
Eligibility
18 Years to 65 Years
Enrollment
104 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
8
States / cities
Birmingham, Alabama • Los Angeles, California • Atlanta, Georgia + 5 more
Source: ClinicalTrials.gov public record
Updated Jun 24, 2026 · Synced Oct 10, 2026, 12:58 PM EDT
Completed Phase 3 Interventional Results available

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

NCT00925301
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Oct 30, 2018 · Synced Oct 10, 2026, 12:58 PM EDT
Completed Phase 1Phase 2 Interventional Results available

A Study to Assess the Safety, Tolerability, and Efficacy of Long-term SOBI003 Treatment in Pediatric MPS IIIA Patients

NCT03811028
View directory record Official record
Conditions
Sanfilippo Syndrome Type A (MPS IIIA)
Interventions
SOBI003
Drug
Lead sponsor
Swedish Orphan Biovitrum
Industry
Eligibility
18 Months to 78 Months
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
2
States / cities
Oakland, California • Chapel Hill, North Carolina
Source: ClinicalTrials.gov public record
Updated Feb 25, 2022 · Synced Oct 10, 2026, 12:58 PM EDT
Withdrawn Phase 2 Interventional

Safety and Efficacy of Gabapentin for Neuropathic Pain in Fabry Disease

NCT01588314
View directory record Official record
Conditions
Fabry Disease, Neuropathic Pain
Interventions
Gabapentin, placebo
Drug
Lead sponsor
University of Minnesota
Other
Eligibility
18 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2014
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated May 29, 2019 · Synced Oct 10, 2026, 12:58 PM EDT
Recruiting Phase 3 Interventional

A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease, GM1 Gangliosidosis or GM2 Gangliosidosis

NCT07054515
View directory record Official record
Conditions
Niemann-Pick Type C Disease, GM1 Gangliosidosis, GM2 Gangliosidosis
Interventions
AZ-3102, Placebo
Drug
Lead sponsor
Azafaros B.V.
Industry
Eligibility
4 Years and older
Enrollment
147 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
5
States / cities
Oakland, California • Minneapolis, Minnesota • Rochester, Minnesota + 2 more
Source: ClinicalTrials.gov public record
Updated Sep 24, 2026 · Synced Oct 10, 2026, 12:58 PM EDT
Completed Phase 2 Interventional

Open-Label Study of Efficacy and Safety of Recombinant Human N-acetylgalactosamine 4-sulfatase in Patients With MPS VI

NCT00048711
View directory record Official record
Conditions
Mucopolysaccharidosis VI
Interventions
N-acetylgalactosamine 4-sulfatase
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
5 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2006
U.S. locations
1
States / cities
Novato, California
Source: ClinicalTrials.gov public record
Updated Aug 25, 2009 · Synced Oct 10, 2026, 12:58 PM EDT
Terminated No phase listed Observational

Study to Detect Unrecognized Mucopolysaccharidosis in Children Visiting Rheumatology, Hand or Skeletal Dysplasia Clinics

NCT01675674
View directory record Official record
Conditions
Mucopolysaccharidoses, Mucopolysaccharidosis I, Mucopolysaccharidosis II, Mucopolysaccharidosis IV, Mucopolysaccharidosis VI
Interventions
Dried blood spot test for MPS
Other
Lead sponsor
National MPS Society
Other
Eligibility
6 Months to 18 Years
Enrollment
3,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2014
U.S. locations
2
States / cities
New Brunswick, New Jersey • New York, New York
Source: ClinicalTrials.gov public record
Updated May 24, 2013 · Synced Oct 10, 2026, 12:58 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 10, 2026, 12:58 PM EDT
Active, not recruiting Phase 3 Interventional

A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of GM1 Gangliosidosis or GM2 Gangliosidosis

NCT07082543
View directory record Official record
Conditions
Gangliosidoses, GM2, Gangliosidosis, GM1
Interventions
AZ-3102, Placebo
Drug
Lead sponsor
Azafaros B.V.
Industry
Eligibility
4 Years and older
Enrollment
75 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
5
States / cities
Oakland, California • Minneapolis, Minnesota • Rochester, Minnesota + 2 more
Source: ClinicalTrials.gov public record
Updated Sep 22, 2026 · Synced Oct 10, 2026, 12:58 PM EDT
Completed Phase 4 Interventional

A Multicenter Study of the Efficacy of Cerezyme in Testing Skeletal Disease in Patients With Type I Gaucher Disease.

NCT00365131
View directory record Official record
Conditions
Gaucher Disease Type I, Cerebroside Lipidosis Syndrome, Clucocerebrosidase Deficiency Disease, Glucosylceramide Beta-Glucosidase Deficiency Disease, Gaucher Disease, Non-Neuronopathic Form
Interventions
Cerezyme (imiglucerase for injection)
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
10 Years to 65 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1997 – 2004
U.S. locations
1
States / cities
Coral Springs, Florida
Source: ClinicalTrials.gov public record
Updated Mar 5, 2015 · Synced Oct 10, 2026, 12:58 PM EDT
No Longer Available No phase listed Expanded access

BPX-501 T Cells Infused Post Stem Cell Transplant in Pediatrics With Non-Malignant Disorders Ineligible for BPU004 Study

NCT03639844
View directory record Official record
Conditions
Hurler Syndrome, Inherited Metabolic Disorder, Lysosomal Storage Disorder, Metachromatic Leukodystrophy, Inborn Errors of Metabolism
Interventions
rivogenlecleucel, rimiducid
Biological · Drug
Lead sponsor
Bellicum Pharmaceuticals
Industry
Eligibility
3 Months to 21 Years
U.S. locations
2
States / cities
Los Angeles, California • Palo Alto, California
Source: ClinicalTrials.gov public record
Updated Oct 5, 2020 · Synced Oct 10, 2026, 12:58 PM EDT
Enrolling by invitation No phase listed Observational

A Long-Term Follow-Up Study of Participants With Cystinosis Who Previously Received CTNS-RD-04

NCT05146830
View directory record Official record
Conditions
Cystinosis
Interventions
Safety and Efficacy Assessments
Other
Lead sponsor
Stephanie Cherqui
Other
Eligibility
14 Years to 50 Years
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2036
U.S. locations
1
States / cities
La Jolla, California
Source: ClinicalTrials.gov public record
Updated Jul 3, 2024 · Synced Oct 10, 2026, 12:58 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Safety and Efficacy of Albuterol in Individuals With Late-onset Pompe Disease

NCT01885936
View directory record Official record
Conditions
Pompe Disease
Interventions
Albuterol, Placebo
Drug
Lead sponsor
Duke University
Other
Eligibility
18 Years and older
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2016
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Jul 15, 2019 · Synced Oct 10, 2026, 12:58 PM 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 10, 2026, 12:58 PM EDT
Active, not recruiting No phase listed Observational

Evaluation of Intravenous Laronidase Pharmacokinetics Before and After Hematopoietic Cell Transplantation in Patients With Mucopolysaccharidosis Type IH.

NCT05634512
View directory record Official record
Conditions
Hematopoietic Cell Transplantation, Mucopolysaccharidosis Type I
Interventions
Laronidase therapy and a stem cell transplant
Drug
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
0 Years to 3 Years
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Oct 30, 2025 · Synced Oct 10, 2026, 12:58 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 in Mucopolysaccharidosis III, Type B (MPS IIIB)

NCT02324049
View directory record Official record
Conditions
Mucopolysaccharidosis IIIB
Interventions
SBC-103
Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
2 Years to 12 Years
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
2
States / cities
Minneapolis, Minnesota • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Aug 21, 2018 · Synced Oct 10, 2026, 12:58 PM EDT
Completed No phase listed Observational

Functional Near-Infrared Spectroscopy (fNIRS) Combined With Diffuse Correlation Spectroscopy (DCS) in Neurocognitive Disease as Compared to Healthy Neurotypical Controls

NCT05642221
View directory record Official record
Conditions
Juvenile Neuronal Ceroid Lipofuscinosis, Smith-Lemli-Opitz Syndrome, Creatine Transporter Deficiency, Niemann-Pick Disease Type C1
Interventions
Not listed
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
6 Months to 110 Years
Enrollment
73 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 8, 2026 · Synced Oct 10, 2026, 12:58 PM EDT