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ClinicalTrials.gov public records Last synced Oct 10, 2026, 12:20 AM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Showing 121–144 of 2,374 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Congenital Metabolic Disorder Clear all
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 10, 2026, 12:20 AM 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 10, 2026, 12:20 AM EDT
Completed Not applicable Interventional

Leucine-enriched Essential Amino Acid Intake to Optimize Protein Anabolism in Children With Cystic Fibrosis

NCT01172301
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Essential amino acid intake + Leucine vs total AA supplement
Dietary Supplement
Lead sponsor
Texas A&M University
Other
Eligibility
10 Years to 21 Years
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2013
U.S. locations
1
States / cities
Little Rock, Arkansas
Source: ClinicalTrials.gov public record
Updated Aug 11, 2015 · Synced Oct 10, 2026, 12:20 AM 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 10, 2026, 12:20 AM 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 10, 2026, 12:20 AM 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 10, 2026, 12:20 AM EDT
Completed No phase listed Observational

International Ataxia Rating Scale in Younger Patients

NCT01942850
View directory record Official record
Conditions
Ataxia Telangiectasia
Interventions
Not listed
Lead sponsor
Quince Therapeutics S.p.A.
Industry
Eligibility
6 Years to 18 Years
Enrollment
63 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2014
U.S. locations
1
States / cities
Tampa, Florida
Source: ClinicalTrials.gov public record
Updated Dec 31, 2014 · Synced Oct 10, 2026, 12:20 AM 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 10, 2026, 12:20 AM 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 10, 2026, 12:20 AM 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 10, 2026, 12:20 AM 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 10, 2026, 12:20 AM EDT
Completed Not applicable Interventional

EnVision CF Multicenter Study of Glucose Tolerance in Cystic Fibrosis

NCT03650712
View directory record Official record
Conditions
Cystic Fibrosis-related Diabetes
Interventions
Oral glucose tolerance test, Continuous glucose monitoring, Dexa scan
Diagnostic Test
Lead sponsor
Katie Larson Ode
Other
Eligibility
6 Years and older
Enrollment
317 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2023
U.S. locations
4
States / cities
Aurora, Colorado • Iowa City, Iowa • Minneapolis, Minnesota + 1 more
Source: ClinicalTrials.gov public record
Updated Oct 2, 2026 · Synced Oct 10, 2026, 12:20 AM EDT
Completed Phase 2 Interventional Results available

Cardiac T2* in Beta-thalassemia Patients on Deferasirox Treatment

NCT00447694
View directory record Official record
Conditions
Beta-thalassemia, Iron Overload
Interventions
Deferasirox
Drug
Lead sponsor
Novartis
Industry
Eligibility
10 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2009
U.S. locations
3
States / cities
Los Angeles, California • Oakland, California • Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Jun 14, 2021 · Synced Oct 10, 2026, 12:20 AM EDT
Terminated No phase listed Observational Accepts healthy volunteers

Clinical and Laboratory Investigation of Humans With Informative Iron or Erythroid Phenotypes

NCT00102245
View directory record Official record
Conditions
Hemoglobinopathies, Hemolysis, Iron Deficiency and Overload, Anemias
Interventions
Not listed
Lead sponsor
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
NIH
Eligibility
1 Year and older
Enrollment
334 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2005 – 2017
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Feb 14, 2018 · Synced Oct 10, 2026, 12:20 AM 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 10, 2026, 12:20 AM 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 10, 2026, 12:20 AM EDT
Recruiting Phase 3 Interventional

Study to Evaluate Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Type 10 (HYPERION)

NCT06891443
View directory record Official record
Conditions
Leber Congenital Amaurosis 10, Blindness, Leber Congenital Amaurosis, Sensation Disorders, Vision Disorder, Neurological Manifestations, Eye Diseases, Hereditary, Eye Diseases, Eye Disorders Congenital, Retinal Disease
Interventions
sepofarsen, Placebo IVT
Drug · Other
Lead sponsor
Laboratoires Thea
Industry
Eligibility
6 Years and older
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
5
States / cities
San Francisco, California • Miami, Florida • Iowa City, Iowa + 2 more
Source: ClinicalTrials.gov public record
Updated Jun 25, 2026 · Synced Oct 10, 2026, 12:20 AM EDT
Recruiting Phase 1Phase 2 Interventional

A Study to Evaluate Safety, Tolerability, and Efficacy of AB-1009 Gene Therapy (GAA Gene) in Adult Participants With Late-Onset Pompe Disease (PROGRESS-GT LOPD)

NCT07282847
View directory record Official record
Conditions
Pompe Disease (Late-onset), Pompe Disease Late-Onset, LOPD
Interventions
AB-1009 (GAA Gene)
Genetic
Lead sponsor
AskBio Inc
Industry
Eligibility
18 Years to 65 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2032
U.S. locations
9
States / cities
Phoenix, Arizona • Irvine, California • Palo Alto, California + 6 more
Source: ClinicalTrials.gov public record
Updated Jul 20, 2026 · Synced Oct 10, 2026, 12:20 AM 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 10, 2026, 12:20 AM 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 10, 2026, 12:20 AM EDT
Recruiting No phase listed Observational

Longitudinal Prospective Natural History Study of Retinopathy in Zellweger Spectrum Disorder

NCT06190626
View directory record Official record
Conditions
Zellweger Spectrum Disorder
Interventions
Not listed
Lead sponsor
McGill University Health Centre/Research Institute of the McGill University Health Centre
Other
Eligibility
Not listed
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2029
U.S. locations
1
States / cities
Rochester, New York
Source: ClinicalTrials.gov public record
Updated Jan 21, 2026 · Synced Oct 10, 2026, 12:20 AM EDT
Terminated Phase 1Phase 2 Interventional

Liver Cell Transplant for Phenylketonuria

NCT01465100
View directory record Official record
Conditions
Phenylketonuria
Interventions
Preparative Radiation Therapy, Hepatocyte Transplant, Immunosuppression, Liver Evaluation, Neuro-psychological Assessment, Whole body Phe oxidation testing, Liver Biopsy
Radiation · Procedure · Drug + 3 more
Lead sponsor
Ira Fox
Other
Eligibility
14 Years to 55 Years
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2016
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Aug 16, 2023 · Synced Oct 10, 2026, 12:20 AM EDT
Completed Phase 4 Interventional Accepts healthy volunteers Results available

Study of Febuxostat Effect on Blood Pressure in Patients With High Normal Blood Pressure

NCT01472692
View directory record Official record
Conditions
Prehypertension, Gout, Pulse Wave Velocity, Hypertension, 24 Hour Blood Pressure
Interventions
Febuxostat
Drug
Lead sponsor
University of California, San Diego
Other
Eligibility
18 Years to 50 Years
Enrollment
47 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2011 – 2014
U.S. locations
1
States / cities
San Diego, California
Source: ClinicalTrials.gov public record
Updated Dec 16, 2019 · Synced Oct 10, 2026, 12:20 AM EDT