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ClinicalTrials.gov public records Last synced Oct 9, 2026, 2:53 PM EDT

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Showing 73–96 of 1,608 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Metabolism, Inborn Errors Clear all
Recruiting No phase listed Observational

Analysis of Velmanase Alfa (Lamzede®)'s Effects in the Body of Children With Alpha-Mannosidosis Under the Age 3

NCT06184503
View directory record Official record
Conditions
Alpha-Mannosidosis
Interventions
Velmanase Alfa
Drug
Lead sponsor
Chiesi Farmaceutici S.p.A.
Industry
Eligibility
Up to 3 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2029
U.S. locations
2
States / cities
Miami, Florida • Greenwood, South Carolina
Source: ClinicalTrials.gov public record
Updated May 16, 2025 · Synced Oct 9, 2026, 2:53 PM EDT
Not listed No phase listed Observational Accepts healthy volunteers

Role of the Protein Osteoprotegerin in the Bone Health of Women With Congenital Adrenal Hyperplasia

NCT00694525
View directory record Official record
Conditions
Adrenal Hyperplasia, Congenital
Interventions
Not listed
Lead sponsor
Office of Rare Diseases (ORD)
NIH
Eligibility
20 Years to 35 Years · Female only
Enrollment
40 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2008 – 2009
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jun 2, 2009 · Synced Oct 9, 2026, 2:53 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Biomarker Validation for Niemann-Pick Disease, Type C: Safety and Efficacy of N-Acetyl Cysteine

NCT00975689
View directory record Official record
Conditions
Niemann-Pick Disease, Type C
Interventions
N-Acetyl Cysteine
Drug
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
12 Months and older
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2010
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated May 6, 2013 · Synced Oct 9, 2026, 2:53 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers

A Test to Predict the Hemolytic Potential of Drugs in G6PD Deficiency

NCT00076323
View directory record Official record
Conditions
Glucosephosphate Dehydrogenase Deficiency, Favism
Interventions
Primaquine
Drug
Lead sponsor
Walter Reed Army Institute of Research (WRAIR)
Federal
Eligibility
18 Years to 60 Years
Enrollment
14 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2003 – 2006
U.S. locations
1
States / cities
Silver Spring, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 27, 2010 · Synced Oct 9, 2026, 2:53 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Resistance Exercise in Barth Syndrome

NCT01629459
View directory record Official record
Conditions
Barth Syndrome
Interventions
Resistance exercise and protein supplementation
Behavioral
Lead sponsor
Washington University School of Medicine
Other
Eligibility
15 Years to 35 Years · Male only
Enrollment
12 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2019
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated May 13, 2020 · Synced Oct 9, 2026, 2:53 PM EDT
Not listed Phase 1 Interventional Accepts healthy volunteers

Safety and Efficacy Study of Intravenous Uricase-PEG 20

NCT01021241
View directory record Official record
Conditions
Gout, Hyperuricemia
Interventions
Uricase-PEG 20
Biological
Lead sponsor
EnzymeRx
Industry
Eligibility
40 Years to 75 Years
Enrollment
20 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2010
U.S. locations
2
States / cities
Miami, Florida • Duncansville, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Feb 23, 2010 · Synced Oct 9, 2026, 2:53 PM EDT
Terminated Phase 3 Interventional Results available

Efficacy and Safety of ALXN1840 Administered for 48 Weeks Versus Standard of Care in Participants With Wilson Disease

NCT03403205
View directory record Official record
Conditions
Wilson Disease
Interventions
ALXN1840, SoC Therapy
Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
12 Years and older
Enrollment
214 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2023
U.S. locations
6
States / cities
Los Angeles, California • New Haven, Connecticut • Chicago, Illinois + 3 more
Source: ClinicalTrials.gov public record
Updated Jun 17, 2024 · Synced Oct 9, 2026, 2:53 PM EDT
Enrolling by invitation Not applicable Interventional

AI Echocardiographic Screening of Cardiac Amyloidosis

NCT06664866
View directory record Official record
Conditions
Cardiac Amyloidosis
Interventions
EchoNet-LVH Assessment
Diagnostic Test
Lead sponsor
Cedars-Sinai Medical Center
Other
Eligibility
22 Years and older
Enrollment
500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
4
States / cities
Los Angeles, California • Palo Alto, California • Chicago, Illinois + 1 more
Source: ClinicalTrials.gov public record
Updated Jul 22, 2026 · Synced Oct 9, 2026, 2:53 PM EDT
Completed No phase listed Observational

Genotype-Phenotype Correlations of Late Infantile Neuronal Ceroid Lipofuscinosis

NCT01035424
View directory record Official record
Conditions
Batten Disease, Late Infantile Neuronal Ceroid Lipofuscinosis
Interventions
Not listed
Lead sponsor
Weill Medical College of Cornell University
Other
Eligibility
2 Years to 18 Years
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2016
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jul 29, 2020 · Synced Oct 9, 2026, 2:53 PM EDT
Active, not recruiting Phase 3 Interventional

Phase 3 Study of ALXN1850 in Treatment-Naïve Pediatric Participants With HPP

NCT06079359
View directory record Official record
Conditions
Hypophosphatasia
Interventions
ALXN1850, Placebo
Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
2 Years to 11 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2028
U.S. locations
3
States / cities
Baltimore, Maryland • Kansas City, Missouri • Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated May 28, 2026 · Synced Oct 9, 2026, 2:53 PM EDT
Completed Phase 2 Interventional

Long-Term Safety and Efficacy Evaluation of EPI-743 in Children With Leigh Syndrome

NCT02352896
View directory record Official record
Conditions
Leigh Syndrome
Interventions
EPI-743
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
1 Year to 18 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2023
U.S. locations
4
States / cities
Stanford University, California • Akron, Ohio • Houston, Texas + 1 more
Source: ClinicalTrials.gov public record
Updated Oct 23, 2024 · Synced Oct 9, 2026, 2:53 PM EDT
Completed Phase 2 Interventional Results available

Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NBI-74788 in Adults With Congenital Adrenal Hyperplasia

NCT03525886
View directory record Official record
Conditions
CAH - Congenital Adrenal Hyperplasia
Interventions
NBI-74788
Drug
Lead sponsor
Neurocrine Biosciences
Industry
Eligibility
18 Years to 50 Years
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2020
U.S. locations
6
States / cities
Aurora, Colorado • Indianapolis, Indiana • Ann Arbor, Michigan + 3 more
Source: ClinicalTrials.gov public record
Updated May 3, 2022 · Synced Oct 9, 2026, 2:53 PM EDT
Completed No phase listed Observational

The Fabrazyme® and Arbs and ACE Inhibitor Treatment (FAACET) Study

NCT00446862
View directory record Official record
Conditions
Fabry Disease, Proteinuria
Interventions
enalapril and other angiotensin converting enzyme inhibitors; losartan and other angiotensin receptor blockers
Drug
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
19 Years to 85 Years
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2012
U.S. locations
5
States / cities
Birmingham, Alabama • Atlanta, Georgia • Chicago, Illinois + 2 more
Source: ClinicalTrials.gov public record
Updated Nov 19, 2013 · Synced Oct 9, 2026, 2:53 PM EDT
Active, not recruiting No phase listed Observational

The REPLACE Registry for Cholbam® (Cholic Acid)

NCT03115086
View directory record Official record
Conditions
Bile Acid Synthesis Disorders
Interventions
Cholbam
Drug
Lead sponsor
Mirum Pharmaceuticals, Inc.
Industry
Eligibility
Not listed
Enrollment
55 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2039
U.S. locations
24
States / cities
Birmingham, Alabama • Palo Alto, California • San Francisco, California + 18 more
Source: ClinicalTrials.gov public record
Updated Jul 31, 2026 · Synced Oct 9, 2026, 2:53 PM EDT
Completed Phase 1Phase 2 Interventional

RGX-121 Gene Therapy in Children 5 Years of Age and Over With MPS II (Hunter Syndrome)

NCT04571970
View directory record Official record
Conditions
Mucopolysaccharidosis Type II (MPS II)
Interventions
RGX-121
Genetic
Lead sponsor
REGENXBIO Inc.
Industry
Eligibility
5 Years to 17 Years · Male only
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
1
States / cities
Oakland, California
Source: ClinicalTrials.gov public record
Updated Jan 28, 2025 · Synced Oct 9, 2026, 2:53 PM EDT
Completed Not applicable Interventional

Diet and Exercise in Pompe Disease

NCT02363153
View directory record Official record
Conditions
Pompe Disease, Glycogen Storage Disease Type II, Lysosomal Storage Diseases
Interventions
Diet and Exercise
Other
Lead sponsor
University of Florida
Other
Eligibility
15 Years to 55 Years
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2021
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated May 14, 2021 · Synced Oct 9, 2026, 2:53 PM EDT
Completed Phase 2 Interventional Results available

Study of SPR001 in Adults With Classic Congenital Adrenal Hyperplasia

NCT03257462
View directory record Official record
Conditions
Congenital Adrenal Hyperplasia, CAH - Congenital Adrenal Hyperplasia
Interventions
SPR001
Drug
Lead sponsor
Spruce Biosciences
Industry
Eligibility
18 Years and older
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2019
U.S. locations
9
States / cities
Orange, California • San Diego, California • Melbourne, Florida + 6 more
Source: ClinicalTrials.gov public record
Updated Oct 22, 2025 · Synced Oct 9, 2026, 2:53 PM EDT
Completed Phase 2 Interventional Results available

A Study of AT2101 (Afegostat Tartrate) in Adult Patients With Type 1 Gaucher Disease Currently Receiving Enzyme Replacement Therapy

NCT00433147
View directory record Official record
Conditions
Gaucher Disease, Type 1, Type 1 Gaucher Disease, Gaucher Disease
Interventions
Afegostat tartrate
Drug
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
18 Years to 74 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2008
U.S. locations
10
States / cities
San Francisco, California • Coral Springs, Florida • Decatur, Georgia + 7 more
Source: ClinicalTrials.gov public record
Updated Sep 25, 2018 · Synced Oct 9, 2026, 2:53 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Gene Therapy for Children With CLN3 Batten Disease

NCT03770572
View directory record Official record
Conditions
CLN3, Batten Disease
Interventions
Low dose CLN-301, High dose CLN-301
Genetic
Lead sponsor
Neela Therapeutics
Industry
Eligibility
3 Years to 10 Years
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2029
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Jun 10, 2026 · Synced Oct 9, 2026, 2:53 PM EDT
Completed Phase 3 Interventional Results available

Effectiveness of Paricalcitol in Reducing Parathyroid Hormone (PTH) Levels in X-linked Hypophosphatemic Rickets

NCT00417612
View directory record Official record
Conditions
Hypophosphatemia, Familial, Hyperparathyroidism
Interventions
Paricalcitol, Placebo
Drug · Other
Lead sponsor
Yale University
Other
Eligibility
9 Years and older
Enrollment
33 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2012
U.S. locations
1
States / cities
New Haven, Connecticut
Source: ClinicalTrials.gov public record
Updated Mar 17, 2020 · Synced Oct 9, 2026, 2:53 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional Accepts healthy volunteers

PK and Safety Evaluation Study of SAP-001 in Adult Subjects With Normal and Impaired Renal Function

NCT06729853
View directory record Official record
Conditions
Gout, Hyperuricemia
Interventions
SAP-001
Drug
Lead sponsor
Shanton Pharma Pte. Ltd.
Industry
Eligibility
18 Years to 80 Years
Enrollment
32 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2024 – 2025
U.S. locations
2
States / cities
Tampa, Florida • Saint Paul, Minnesota
Source: ClinicalTrials.gov public record
Updated Dec 11, 2024 · Synced Oct 9, 2026, 2:53 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Safety and Efficacy of Deferasirox (ICL670) in Patients With Iron Overload Resulting From Hereditary Hemochromatosis

NCT00395629
View directory record Official record
Conditions
Iron Overload, Hereditary Hemochromatosis
Interventions
Deferasirox (ICL670)
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
18 Years and older
Enrollment
49 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2009
U.S. locations
9
States / cities
Long Beach, California • Farmington, Connecticut • Detroit, Michigan + 6 more
Source: ClinicalTrials.gov public record
Updated May 30, 2011 · Synced Oct 9, 2026, 2:53 PM EDT
Recruiting No phase listed Observational

LCAT (Lecithin Cholesterol Acyl Transferase) Natural History Study

NCT06217588
View directory record Official record
Conditions
LCAT Deficiency
Interventions
Demographics, diagnosis type, genotype, lipid profile, renal function profile, treatment allocation, ophthalmology exam, country of residence.
Other
Lead sponsor
University of Pennsylvania
Other
Eligibility
Not listed
Enrollment
40 participants
Timeline
2022 – 2028
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated May 6, 2026 · Synced Oct 9, 2026, 2:53 PM EDT
Recruiting Not applicable Interventional Accepts healthy volunteers

Home Exercise for Individuals With Neurodegenerative Disease

NCT05443906
View directory record Official record
Conditions
Neurodegenerative Diseases, Leukodystrophy, Ataxia, LBSL, Leukoencephalopathy With Brain Stem and Spinal Cord Involvement and High Lactate Syndrome (Disorder), Leukoencephalopathy With Brainstem and Spinal Cord Involvement and Lactate Elevation, Cerebellar Ataxia, Adrenomyeloneuropathy
Interventions
Home exercise
Behavioral
Lead sponsor
Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Other
Eligibility
5 Years to 75 Years
Enrollment
30 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2023 – 2028
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 16, 2026 · Synced Oct 9, 2026, 2:53 PM EDT