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ClinicalTrials.gov public records Last synced Oct 25, 2026, 7:00 AM EDT

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Showing 577–600 of 1,510 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Inborn Errors of Metabolism Disorders Clear all
Recruiting Not applicable Interventional

Evaluation of the Safety of Loargys Arginine Test System in Loargys-treated Patients

NCT07573059
View directory record Official record
Conditions
ARG1 Deficiency
Interventions
Loargys Arginine Test System
Device
Lead sponsor
Immedica Pharma US Inc
Industry
Eligibility
0 Years to 100 Years
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2027
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated May 7, 2026 · Synced Oct 25, 2026, 7:00 AM EDT
Completed No phase listed Observational

A Natural History of Late Onset Tay-Sachs Disease

NCT02851862
View directory record Official record
Conditions
GM2 Gangliosidosis
Interventions
Not listed
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
7 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2025
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Mar 2, 2026 · Synced Oct 25, 2026, 7:00 AM EDT
Recruiting Phase 2 Interventional

Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT

NCT01962415
View directory record Official record
Conditions
Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias, Inflammatory Conditions, Systemic Juvenile Idiopathic Arthritis (sJIA), Juvenile Rheumatoid Arthritis (JRA)
Interventions
Hydroxyurea, Alemtuzumab, Fludarabine, Melphalan, Thiotepa
Drug
Lead sponsor
Paul Szabolcs
Other
Eligibility
2 Months to 55 Years
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2027
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Dec 15, 2025 · Synced Oct 25, 2026, 7:00 AM EDT
Completed Phase 4 Interventional Results available

Pharmacokinetics of Alglucosidase Alfa in Patients With Pompe Disease

NCT01410890
View directory record Official record
Conditions
Pompe Disease, Glycogen Storage Disease Type II (GSD II)
Interventions
alglucosidase alfa
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
Not listed
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2020
U.S. locations
4
States / cities
Valhalla, New York • Cincinnati, Ohio • Salt Lake City, Utah + 1 more
Source: ClinicalTrials.gov public record
Updated Mar 28, 2022 · Synced Oct 25, 2026, 7:00 AM 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 25, 2026, 7:00 AM 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 25, 2026, 7:00 AM 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 25, 2026, 7:00 AM EDT
Terminated Phase 1Phase 2 Interventional

A Study to Assess the Safety, Pharmacokinetics, and Pharmacodynamics of mRNA-3705 in Participants With Isolated Methylmalonic Acidemia

NCT04899310
View directory record Official record
Conditions
Methylmalonic Acidemia
Interventions
mRNA-3705, Placebo
Biological
Lead sponsor
ModernaTX, Inc.
Industry
Eligibility
1 Year and older
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2026
U.S. locations
3
States / cities
Los Angeles, California • Palo Alto, California • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated May 6, 2026 · Synced Oct 25, 2026, 7:00 AM EDT
Completed Phase 2 Interventional Results available

Evaluate the Safety, Efficacy, and Pharmacokinetics of CRN04894 in Participants With Congenital Adrenal Hyperplasia (TouCAHn)

NCT05907291
View directory record Official record
Conditions
Congenital Adrenal Hyperplasia, Classic Congenital Adrenal Hyperplasia
Interventions
atumelnant (CRN04894)
Drug
Lead sponsor
Crinetics Pharmaceuticals Inc.
Industry
Eligibility
16 Years to 75 Years
Enrollment
38 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
8
States / cities
Pasadena, California • Ann Arbor, Michigan • Minneapolis, Minnesota + 5 more
Source: ClinicalTrials.gov public record
Updated Sep 10, 2026 · Synced Oct 25, 2026, 7:00 AM EDT
Recruiting Not applicable Interventional

Comparing Direct vs Indirect Methods for Cascade Screening

NCT05348564
View directory record Official record
Conditions
Long QT Syndrome, Familial Hypercholesterolemia, Ethics, Genetic Testing
Interventions
Direct contact of family members for cascade screening of relevant variants
Behavioral
Lead sponsor
University of Maryland, Baltimore
Other
Eligibility
18 Years and older
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2027
U.S. locations
1
States / cities
Lancaster, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 14, 2026 · Synced Oct 25, 2026, 7:00 AM EDT
Completed No phase listed Observational

Preliminary Study of Brain Effects of Palynziq-Related Changes in Phenylalanine in Individuals With PKU

NCT05356377
View directory record Official record
Conditions
Phenylketonurias
Interventions
Pegvaliase-Pqpz
Drug
Lead sponsor
University of Missouri-Columbia
Other
Eligibility
18 Years to 55 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
2
States / cities
Boston, Massachusetts • Columbia, Missouri
Source: ClinicalTrials.gov public record
Updated Dec 29, 2025 · Synced Oct 25, 2026, 7:00 AM EDT
Completed No phase listed Observational

Visual Function and Ocular Pigmentation in Albinism

NCT00001153
View directory record Official record
Conditions
Albinism, Albinism, Ocular, Albinism, Oculocutaneous
Interventions
Not listed
Lead sponsor
National Eye Institute (NEI)
NIH
Eligibility
Not listed
Enrollment
130 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1976 – 2000
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 25, 2026, 7:00 AM EDT
Completed No phase listed Observational Results available

Screening for Cardiac Amyloidosis With Nuclear Imaging for Minority Populations

NCT03812172
View directory record Official record
Conditions
Amyloid Cardiomyopathy, Transthyretin-Related
Interventions
99mTc-PYP or 99m Tc-HDP
Drug
Lead sponsor
Mathew S. Maurer, MD
Other
Eligibility
60 Years and older
Enrollment
646 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2024
U.S. locations
4
States / cities
New Haven, Connecticut • Boston, Massachusetts • New York, New York
Source: ClinicalTrials.gov public record
Updated Nov 19, 2025 · Synced Oct 25, 2026, 7:00 AM EDT
Completed Phase 3 Interventional Results available

A Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Therapy in Subjects With Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7)

NCT02432144
View directory record Official record
Conditions
Sly Syndrome, MPS VII, Mucopolysaccharidosis, Mucopolysaccharidosis VII
Interventions
UX003
Drug
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
5 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2019
U.S. locations
4
States / cities
Oakland, California • Orange, California • Minneapolis, Minnesota + 1 more
Source: ClinicalTrials.gov public record
Updated Jul 30, 2020 · Synced Oct 25, 2026, 7:00 AM EDT
Completed Phase 2 Interventional Results available

A Long-Term Extension Study of AT2101 (Afegostat Tartrate) in Type 1 Gaucher Patients

NCT00813865
View directory record Official record
Conditions
Gaucher Disease, Type 1 Gaucher Disease, Gaucher Disease, Type 1
Interventions
afegostat tartrate
Drug
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2012
U.S. locations
3
States / cities
Coral Springs, Florida • Decatur, Georgia • Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Aug 15, 2018 · Synced Oct 25, 2026, 7:00 AM EDT
Completed Not applicable Interventional

A Study of the Safety and Tolerance of Long-Term Therapy With Intravenous Cytovene (Ganciclovir Sodium) for Cytomegalovirus Retinitis in Persons With AIDS

NCT00002034
View directory record Official record
Conditions
Cytomegalovirus Retinitis, HIV Infections
Interventions
Zidovudine, Ganciclovir
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
13 Years and older
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
8
States / cities
Birmingham, Alabama • Oakland, California • San Francisco, California + 5 more
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 25, 2026, 7:00 AM EDT
Recruiting No phase listed Observational

Evaluation of Outcome Metrics in Alexander Disease

NCT02714764
View directory record Official record
Conditions
Alexander Disease
Interventions
Not listed
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
Not listed
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2030
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jan 15, 2026 · Synced Oct 25, 2026, 7:00 AM EDT
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 25, 2026, 7:00 AM EDT
Recruiting No phase listed Observational

Longitudinal Study of Neurodegenerative Disorders

NCT03333200
View directory record Official record
Conditions
MLD, Krabbe Disease, ALD, MPS I, MPS II, MPS III, Vanishing White Matter Disease, GM3 Gangliosidosis, PKAN, Tay-Sachs Disease, NP Deficiency, Osteopetrosis, Alpha-Mannosidosis, Sandhoff Disease, Niemann-Pick Diseases, MPS IV, Gaucher Disease, GAN, GM1 Gangliosidoses, Morquio Disease, S-Adenosylhomocysteine Hydrolase Deficiency, Batten Disease, Pelizaeus-Merzbacher Disease, Leukodystrophy, Lysosomal Storage Diseases, Purine Nucleoside Phosphorylase Deficiency, Multiple Sulfatase Deficiency Disease
Interventions
Palliative Care, Hematopoetic Stem Cell Transplantation
Other · Biological
Lead sponsor
University of Pittsburgh
Other
Eligibility
Not listed
Enrollment
1,500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2035
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Feb 9, 2026 · Synced Oct 25, 2026, 7:00 AM 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 25, 2026, 7:00 AM EDT
Recruiting Not applicable Interventional Accepts healthy volunteers

A Study Evaluating the Impact of Regular FODMAP-targeting Digestive Enzyme Blend Use on Gastrointestinal Symptoms in Individuals With Self-Reported Bloating

NCT07591584
View directory record Official record
Conditions
IBS (Irritable Bowel Syndrome), Bloating, Abdominal Pain, Gut Health
Interventions
FODMAP-targeting digestive enzyme blend, Placebo
Dietary Supplement · Other
Lead sponsor
Kiwi Health Inc
Industry
Eligibility
18 Years and older
Enrollment
150 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2026
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated May 15, 2026 · Synced Oct 25, 2026, 7:00 AM EDT
Completed Phase 2 Interventional Results available

Pegylated Recombinant Mammalian Uricase (PEG-uricase) as Treatment for Refractory Gout

NCT00111657
View directory record Official record
Conditions
Gout
Interventions
Pegloticase
Biological
Lead sponsor
John Sundy
Other
Eligibility
18 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2009
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Oct 3, 2014 · Synced Oct 25, 2026, 7:00 AM EDT
Not listed Phase 1 Interventional

Open-Label Study of Long-Term Safety and Efficacy of Intravenous Trappsol Cyclo (HPβCD) in Niemann-Pick Disease Type C

NCT03893071
View directory record Official record
Conditions
Niemann-Pick Disease, Type C1
Interventions
Hydroxypropyl-β-cyclodextrin
Drug
Lead sponsor
Cyclo Therapeutics, Inc.
Industry
Eligibility
18 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
1
States / cities
Oakland, California
Source: ClinicalTrials.gov public record
Updated Jun 18, 2021 · Synced Oct 25, 2026, 7:00 AM EDT
Completed Phase 4 Interventional

Efficacy of Proprietary Cherry Juice Blend in Osteoarthritis of the Knee

NCT00443092
View directory record Official record
Conditions
Osteoarthritis
Interventions
proprietary cherry juice blend (food), control juice (kool aid blend)
Other
Lead sponsor
CherryPharm
Industry
Eligibility
18 Years to 90 Years
Enrollment
59 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2010
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Aug 3, 2012 · Synced Oct 25, 2026, 7:00 AM EDT