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ClinicalTrials.gov public records Last synced Oct 17, 2026, 10:37 PM EDT

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Showing 1009–1032 of 1,605 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Inborn Errors of Metabolism Clear all
Completed No phase listed Observational

Psychological Concomitants of Morquio A Syndrome - Longitudinal Effects of Enzyme Replacement Therapy (The MAPLE Study)

NCT02208661
View directory record Official record
Conditions
Morquio A Syndrome, Mucopolysaccharidosis IV A
Interventions
Not listed
Lead sponsor
Nadia Ali, PhD
Other
Eligibility
18 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2018
U.S. locations
1
States / cities
Decatur, Georgia
Source: ClinicalTrials.gov public record
Updated Apr 11, 2018 · Synced Oct 17, 2026, 10:37 PM EDT
Recruiting Phase 2Phase 3 Interventional

Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH

NCT02716246
View directory record Official record
Conditions
MPS IIIA, Sanfilippo Syndrome, Sanfilippo A, Mucopolysaccharidosis III
Interventions
UX111, Prophylactic Immunomodulatory (IM) Therapy, Optimized Prophylactic IM Therapy, Adjuvant IM Therapy
Biological · Drug
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
Not listed
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2029
U.S. locations
2
States / cities
Columbus, Ohio • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Sep 22, 2026 · Synced Oct 17, 2026, 10:37 PM EDT
Recruiting No phase listed Observational

Determining the Association of TTR Stabilizing Therapy With Circulating TTR Amyloid Aggregates Over Time in Patients With ATTR-CM: Longitudinal Biomarker Study

NCT07196839
View directory record Official record
Conditions
Transthyretin (TTR) Amyloid Cardiomyopathy
Interventions
Not listed
Lead sponsor
University of Texas Southwestern Medical Center
Other
Eligibility
30 Years to 80 Years
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Jan 7, 2026 · Synced Oct 17, 2026, 10:37 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 17, 2026, 10:37 PM EDT
Active, not recruiting Phase 3 Interventional

Phase 3 Study of ALXN1850 in Pediatric Participants With HPP Previously Treated With Asfotase Alfa

NCT06079372
View directory record Official record
Conditions
Hypophosphatasia
Interventions
ALXN1850, asfotase alfa
Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
2 Years to 11 Years
Enrollment
43 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2028
U.S. locations
6
States / cities
Hartford, Connecticut • Baltimore, Maryland • Minneapolis, Minnesota + 3 more
Source: ClinicalTrials.gov public record
Updated May 5, 2026 · Synced Oct 17, 2026, 10:37 PM EDT
Not listed No phase listed Observational Accepts healthy volunteers

Determining the Long-Term Effects of Prenatal Dexamethasone Treatment in Children With 21-Hydroxylase Deficiency and Their Mothers

NCT00617292
View directory record Official record
Conditions
Adrenal Hyperplasia, Congenital
Interventions
Not listed
Lead sponsor
Office of Rare Diseases (ORD)
NIH
Eligibility
12 Years and older
Enrollment
233 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2008 – 2009
U.S. locations
2
States / cities
New York, New York • Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Dec 9, 2008 · Synced Oct 17, 2026, 10:37 PM EDT
Recruiting Not applicable Interventional

Dose Response of Exercise for Arthritis Management

NCT06880653
View directory record Official record
Conditions
Arthritis, Rheumatoid Arthritis (RA), Gout, Fibromyalgia (FM), Osteoarthritis, Lupus Erythematosus, Systemic
Interventions
DREAM Adaptive Intervention
Behavioral
Lead sponsor
University of South Carolina
Other
Eligibility
18 Years and older
Enrollment
285 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2029
U.S. locations
1
States / cities
Columbia, South Carolina
Source: ClinicalTrials.gov public record
Updated Apr 3, 2026 · Synced Oct 17, 2026, 10:37 PM EDT
Completed No phase listed Observational

Sjogren-Larsson Syndrome: Natural History, Clinical Variation and Evaluation of Biochemical Markers

NCT01971957
View directory record Official record
Conditions
Sjogren-Larsson Syndrome (SLS)
Interventions
Not listed
Lead sponsor
University of Nebraska
Other
Eligibility
Not listed
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2023
U.S. locations
2
States / cities
Omaha, Nebraska • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Oct 8, 2024 · Synced Oct 17, 2026, 10:37 PM EDT
Recruiting Phase 1Phase 2 Interventional

64Cu-GRIP B in Patients With Advanced Malignancies

NCT05888532
View directory record Official record
Conditions
Prostate Cancer, Renal Cancer, Urethral Cancer, Advanced Solid Tumor, Metastatic Castration-resistant Prostate Cancer, Solid Tumor, Adult
Interventions
Copper-64 labeled Granzyme B (64Cu-GRIP B), Positron Emission Tomography (PET)
Drug · Procedure
Lead sponsor
Rahul Aggarwal
Other
Eligibility
18 Years and older
Enrollment
91 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2027
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Mar 17, 2026 · Synced Oct 17, 2026, 10:37 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 17, 2026, 10:37 PM EDT
Enrolling by invitation No phase listed Observational Accepts healthy volunteers

Tissue Sample Study for Mitochondrial Disorders

NCT01803906
View directory record Official record
Conditions
Mitochondrial Disorders, Mitochondrial Disease, Melas, Kearns Sayer, NARP, MNGIE, LHON, Mitochondrial Depletion Syndrome, Leigh's Disease
Interventions
Not listed
Lead sponsor
Columbia University
Other
Eligibility
Not listed
Enrollment
6,900 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2026
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jan 7, 2026 · Synced Oct 17, 2026, 10:37 PM EDT
Recruiting Phase 2 Interventional

Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)

NCT07123155
View directory record Official record
Conditions
Pompe Disease
Interventions
S-606001, Placebo
Drug
Lead sponsor
Shionogi
Industry
Eligibility
18 Years and older
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
9
States / cities
Irvine, California • Gainesville, Florida • Atlanta, Georgia + 6 more
Source: ClinicalTrials.gov public record
Updated Jun 23, 2026 · Synced Oct 17, 2026, 10:37 PM EDT
Completed Phase 2 Interventional Results available

Study of KRN23 (Burosumab), a Recombinant Fully Human Monoclonal Antibody Against Fibroblast Growth Factor 23 (FGF23), in Pediatric Subjects With X-linked Hypophosphatemia (XLH)

NCT02163577
View directory record Official record
Conditions
X-linked Hypophosphatemia
Interventions
burosumab
Biological
Lead sponsor
Kyowa Kirin, Inc.
Industry
Eligibility
5 Years to 12 Years
Enrollment
52 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2018
U.S. locations
4
States / cities
San Francisco, California • New Haven, Connecticut • Indianapolis, Indiana + 1 more
Source: ClinicalTrials.gov public record
Updated May 6, 2024 · Synced Oct 17, 2026, 10:37 PM EDT
Completed Phase 4 Interventional Results available

A Phase 4 Two Dose Level Study of Naglazyme(TM) (Galsulfase) in Infants With MPS VI

NCT00299000
View directory record Official record
Conditions
Mucopolysaccharidosis VI, Maroteaux-Lamy Syndrome
Interventions
Naglazyme
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
Up to 1 Year
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2009
U.S. locations
2
States / cities
Los Angeles, California • Oakland, California
Source: ClinicalTrials.gov public record
Updated Jul 22, 2011 · Synced Oct 17, 2026, 10:37 PM EDT
Completed Not applicable Interventional

MRI and Ultrasound Findings in Patients With Gout and Normal Plain Radiographs

NCT00584311
View directory record Official record
Conditions
Gout
Interventions
MRI and Ultrasound
Other
Lead sponsor
University of South Florida
Other
Eligibility
18 Years to 80 Years
Enrollment
33 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2008
U.S. locations
1
States / cities
Tampa, Florida
Source: ClinicalTrials.gov public record
Updated Jun 9, 2008 · Synced Oct 17, 2026, 10:37 PM EDT
Completed Phase 2 Interventional

RDEA3170 Monotherapy in Subjects With Gout

NCT01927198
View directory record Official record
Conditions
Gout
Interventions
Placebo, RDEA3170 5 mg, RDEA3170 10 mg, RDEA3170 12.5 mg
Drug
Lead sponsor
Ardea Biosciences, Inc.
Industry
Eligibility
18 Years to 70 Years
Enrollment
172 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2014
U.S. locations
49
States / cities
Birmingham, Alabama • Gulf Shores, Alabama • Mobile, Alabama + 45 more
Source: ClinicalTrials.gov public record
Updated Jul 1, 2015 · Synced Oct 17, 2026, 10:37 PM EDT
Completed No phase listed Observational

A Natural History Study of Canavan Disease

NCT02851563
View directory record Official record
Conditions
Canavan Disease
Interventions
Not listed
Lead sponsor
NYU Langone Health
Other
Eligibility
Up to 99 Years
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
2
States / cities
Boston, Massachusetts • New York, New York
Source: ClinicalTrials.gov public record
Updated Nov 1, 2019 · Synced Oct 17, 2026, 10:37 PM EDT
Completed No phase listed Observational Results available

A Study of Patients With Sanfilippo Syndrome Type A (MPS IIIA)

NCT01047306
View directory record Official record
Conditions
Sanfilippo Syndrome Type A
Interventions
assessment
Other
Lead sponsor
Shire
Industry
Eligibility
1 Year and older
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2013
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jun 11, 2021 · Synced Oct 17, 2026, 10:37 PM EDT
Not listed No phase listed Observational Accepts healthy volunteers

Assessment and Validation of Electronic Gas and Bloating Diary

NCT04684849
View directory record Official record
Conditions
Gas, Bloating, Distension, Constipation, Diarrhea
Interventions
APP Diary, Paper Diary
Other
Lead sponsor
Augusta University
Other
Eligibility
18 Years to 80 Years
Enrollment
40 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2024
U.S. locations
1
States / cities
Augusta, Georgia
Source: ClinicalTrials.gov public record
Updated Oct 24, 2022 · Synced Oct 17, 2026, 10:37 PM EDT
Terminated Phase 1 Interventional

Intrathecal Enzyme Replacement Therapy for Spinal Cord Compression in Mucopolysaccharidosis (MPS) I

NCT00215527
View directory record Official record
Conditions
Mucopolysaccharidosis I, Lysosomal Storage Diseases, Spinal Cord Compression
Interventions
laronidase
Drug
Lead sponsor
Patricia I. Dickson, M.D.
Individual
Eligibility
8 Years and older
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2011
U.S. locations
1
States / cities
Torrance, California
Source: ClinicalTrials.gov public record
Updated Feb 21, 2013 · Synced Oct 17, 2026, 10:37 PM EDT
Not listed No phase listed Observational

A Prospective Natural History Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)

NCT03227042
View directory record Official record
Conditions
Mucopolysaccharidosis Type IIIB
Interventions
Not listed
Lead sponsor
Allievex Corporation
Industry
Eligibility
Up to 18 Years
Enrollment
44 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2025
U.S. locations
1
States / cities
Oakland, California
Source: ClinicalTrials.gov public record
Updated Oct 26, 2022 · Synced Oct 17, 2026, 10:37 PM EDT
Terminated Phase 1Phase 2 Interventional Results available

Pilot Study of a Multi-Drug Regimen for Severe Pulmonary Fibrosis in Hermansky-Pudlak Syndrome

NCT00467831
View directory record Official record
Conditions
Hermansky-Pudlak Syndrome (HPS), Pulmonary Fibrosis, Oculocutaneous Albinism, Platelet Storage Pool Deficiency, Metabolic Disease
Interventions
Losartan, Zileuton, N-Acetylcysteine, Pravastatin, Erythromycin
Drug
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
18 Years to 70 Years
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2012
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 2, 2013 · Synced Oct 17, 2026, 10:37 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Early Detection of Neuropathy in ATTRv

NCT05311488
View directory record Official record
Conditions
Hereditary Amyloidosis, Transthyretin-Related
Interventions
neurofilament light chain, In-vivo Meissner Corpuscle imaging, Nerve conduction study
Diagnostic Test
Lead sponsor
University of Pennsylvania
Other
Eligibility
30 Years to 90 Years
Enrollment
47 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2026
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 12, 2026 · Synced Oct 17, 2026, 10:37 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 17, 2026, 10:37 PM EDT