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Showing 1–24 of 2,402 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Inherited Metabolic Diseases Clear all
Completed No phase listed Observational Accepts healthy volunteers

Assessing Inherited Markers of Metabolic Syndrome in the Young

NCT00966407
View directory record Official record
Conditions
Metabolic Syndrome, Diabetes Mellitus, Type 2, Obesity
Interventions
Not listed
Lead sponsor
Eric Hoffman
Other
Eligibility
18 Years to 35 Years
Enrollment
700 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2007 – 2016
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Feb 8, 2017 · Synced Sep 24, 2026, 1:02 AM EDT
Enrolling by invitation Phase 3 Interventional

Study to Evaluate the Efficacy and Safety of Lerodalcibep in Children 6 to 17 Years, With Heterozygous FH

NCT07102511
View directory record Official record
Conditions
Familial Hypercholesterolemia - Heterozygous
Interventions
lerodalcibep 300 mg Monthly SC
Biological
Lead sponsor
LIB Therapeutics LLC
Industry
Eligibility
6 Years to 17 Years
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Jul 27, 2026 · Synced Sep 24, 2026, 1:02 AM EDT
Completed No phase listed Observational

Characterization of the Patient Population With Galactosialidosis

NCT01416467
View directory record Official record
Conditions
Galactosialidosis
Interventions
Not listed
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
6 Months and older
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Oct 10, 2018 · Synced Sep 24, 2026, 1:02 AM EDT
Active, not recruiting Phase 3 Interventional

Clinical Study for Treatment-naïve IOPD Babies to Evaluate Efficacy and Safety of ERT With Avalglucosidase Alfa

NCT04910776
View directory record Official record
Conditions
Glycogen Storage Disease Type II
Interventions
avalglucosidase alfa
Drug
Lead sponsor
Sanofi
Industry
Eligibility
0 Days to 12 Months
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2027
U.S. locations
6
States / cities
Stanford, California • Minneapolis, Minnesota • Hawthorne, New York + 3 more
Source: ClinicalTrials.gov public record
Updated Apr 7, 2026 · Synced Sep 24, 2026, 1:02 AM EDT
Completed Phase 4 Interventional

Replagal Enzyme Replacement Therapy for Adults With Fabry Disease

NCT00097890
View directory record Official record
Conditions
Fabry Disease
Interventions
Replagal (Agalsidase Alfa), Replagal
Drug
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Male only
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2005
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 3, 2008 · Synced Sep 24, 2026, 1:02 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Caregiving Networks Across Disease Context and the Life Course

NCT05007990
View directory record Official record
Conditions
Inherited Metabolic Disorders, Undiagnosed Diseases, Batten's Disease, Tay Sachs, Diabetes
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
18 Years to 100 Years
Enrollment
2,800 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2030
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 14, 2026 · Synced Sep 24, 2026, 1:02 AM EDT
Completed No phase listed Observational

Screening an Orthopedic Population for Mildly-affected Individuals With Morquio Syndrome A and Maroteaux-Lamy Syndrome

NCT01961518
View directory record Official record
Conditions
Morquio Syndrome A, Maroteaux Lamy Syndrome, MPS IVA, MPS VI
Interventions
Not listed
Lead sponsor
Greenwood Genetic Center
Other
Eligibility
Up to 18 Years
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2015
U.S. locations
1
States / cities
Greenville, South Carolina
Source: ClinicalTrials.gov public record
Updated Aug 12, 2015 · Synced Sep 24, 2026, 1:02 AM EDT
Recruiting No phase listed Observational

MRI as Noninvasive Innovative Approach in Detection and Monitoring of Malignant Oral Lesions in Fanconi Anemia Patients

NCT07649031
View directory record Official record
Conditions
Fanconi Anemia
Interventions
MRIs
Diagnostic Test
Lead sponsor
University of Minnesota
Other
Eligibility
18 Years and older
Enrollment
80 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2030
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jul 1, 2026 · Synced Sep 24, 2026, 1:02 AM 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 4, 2015 · Synced Sep 24, 2026, 1:02 AM EDT
Completed Phase 2 Interventional

CMV Retinitis Retreatment Trial

NCT00000766
View directory record Official record
Conditions
Cytomegalovirus Retinitis, HIV Infections
Interventions
Foscarnet sodium, Ganciclovir
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
18 Years and older
Enrollment
300 participants
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
9
States / cities
La Jolla, California • Los Angeles, California • San Francisco, California + 4 more
Source: ClinicalTrials.gov public record
Updated Feb 28, 2011 · Synced Sep 24, 2026, 1:02 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Irradiated Blood Versus Non Irradiated Blood Transfusions in Craniosynostosis Repair

NCT02483702
View directory record Official record
Conditions
Graft Versus Host Disease, Hyperkalemia, Craniectomy, Synostectomy, FOA, Congenital Malformations
Interventions
Irradiated Blood Transfusions, Non-Irradiated Blood Transfusions
Other
Lead sponsor
Valley Anesthesiology Consultants
Other
Eligibility
2 Months to 6 Months
Enrollment
20 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2017
U.S. locations
1
States / cities
Phoenix, Arizona
Source: ClinicalTrials.gov public record
Updated Oct 18, 2018 · Synced Sep 24, 2026, 1:02 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Effect of G6PD Deficiency on Red Blood Cell Storage

NCT04081272
View directory record Official record
Conditions
G6PD Deficiency
Interventions
Sodium Chromate Cr51
Drug
Lead sponsor
Columbia University
Other
Eligibility
18 Years to 70 Years · Male only
Enrollment
40 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2017
U.S. locations
2
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Sep 16, 2019 · Synced Sep 24, 2026, 1:02 AM EDT
Not listed No phase listed Observational

RCDP Natural History Study

NCT04031287
View directory record Official record
Conditions
Rhizomelic Chondrodysplasia Punctata
Interventions
Not listed
Lead sponsor
MED-LIFE DISCOVERIES LP
Industry
Eligibility
6 Months to 21 Years
Enrollment
75 participants
Timeline
2019 – 2021
U.S. locations
1
States / cities
Wilmington, Delaware
Source: ClinicalTrials.gov public record
Updated Aug 1, 2019 · Synced Sep 24, 2026, 1:02 AM EDT
Completed Phase 1Phase 2 Interventional

Statins and Lupus: Effects of Statins on Clinical Lupus Parameters, Serological Markers and Toll-like Receptors

NCT00519363
View directory record Official record
Conditions
Systemic Lupus Erythematosus
Interventions
atorvastatin
Drug
Lead sponsor
The Center for Rheumatic Disease, Allergy, & Immunology
Other
Eligibility
18 Years to 60 Years · Female only
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2008
U.S. locations
1
States / cities
Kansas City, Missouri
Source: ClinicalTrials.gov public record
Updated Mar 23, 2010 · Synced Sep 24, 2026, 1:02 AM EDT
Recruiting No phase listed Observational

Adrenoleukodystrophy National Registry Study

NCT03789721
View directory record Official record
Conditions
ALD (Adrenoleukodystrophy), Adrenoleukodystrophy, Cerebral Adrenoleukodystrophy
Interventions
Medical Record Abstraction, Biospecimen Sample Collection
Other
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Not listed
Enrollment
1,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2030
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated May 11, 2026 · Synced Sep 24, 2026, 1:02 AM EDT
Terminated No phase listed Observational

A Study to Identify and Characterize LAL-D Patients in High-risk Populations

NCT02345421
View directory record Official record
Conditions
Lysosomal Acid Lipase Deficiency
Interventions
Not listed
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
2 Years and older
Enrollment
640 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2015
U.S. locations
15
States / cities
Birmingham, Alabama • Phoenix, Arizona • Los Angeles, California + 12 more
Source: ClinicalTrials.gov public record
Updated May 23, 2016 · Synced Sep 24, 2026, 1:02 AM EDT
Completed No phase listed Observational

Study of Smith-Lemli-Opitz Syndrome

NCT00001721
View directory record Official record
Conditions
Smith-Lemli-Opitz Syndrome
Interventions
Cholesterol Suspension, CRH, Cholesterol
Drug
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
Not listed
Enrollment
130 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1998
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 15, 2026 · Synced Sep 24, 2026, 1:02 AM EDT
Enrolling by invitation Phase 2 Interventional Accepts healthy volunteers

Theophylline for Treatment of Pseudohypoparathyroidism

NCT04240821
View directory record Official record
Conditions
Pseudohypoparathyroidism, Pseudohypoparathyroidism Type 1a, Albright Hereditary Osteodystrophy
Interventions
Theophylline ER
Drug
Lead sponsor
Jaclyn Tamaroff
Other
Eligibility
2 Years to 99 Years
Enrollment
34 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2026
U.S. locations
1
States / cities
Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Sep 1, 2026 · Synced Sep 24, 2026, 1:02 AM EDT
Terminated No phase listed Observational

Behavioral Effects of Kuvan in Children With Mild Phenylketonuria

NCT00827762
View directory record Official record
Conditions
Phenylketonuria
Interventions
Kuvan
Drug
Lead sponsor
Washington University School of Medicine
Other
Eligibility
6 Years to 18 Years
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2010
U.S. locations
4
States / cities
Chicago, Illinois • Columbia, Missouri • St Louis, Missouri + 1 more
Source: ClinicalTrials.gov public record
Updated Dec 4, 2013 · Synced Sep 24, 2026, 1:02 AM EDT
Completed Phase 2 Interventional Results available

Interferon Gamma-1b in Friedreich Ataxia (FRDA)

NCT01965327
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
Interferon Gamma-1b
Drug
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
5 Years to 17 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2014
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 12, 2021 · Synced Sep 24, 2026, 1:02 AM EDT
Recruiting Not applicable Interventional

Atlantic Lipid Lowering Treatment Optimization Program

NCT06439654
View directory record Official record
Conditions
Familial Hypercholesterolemia, Lipoprotein Types--Lp System Lp(A) Hyperlipoproteinemia, Apolipoprotein B 100, Familial Defective, High Density Lipoprotein Deficiency, Low-Density-Lipoid-Type Hyperlipoproteinemia
Interventions
Supportive care
Other
Lead sponsor
Atlantic Health System
Other
Eligibility
18 Years to 120 Years
Enrollment
250 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
2
States / cities
Clark, New Jersey • Springfield, New Jersey
Source: ClinicalTrials.gov public record
Updated Aug 2, 2025 · Synced Sep 24, 2026, 1:02 AM EDT
Completed Phase 3 Interventional Results available

Study of the Safety, Pharmacokinetics and Efficacy of HPN-100, in Pediatric Subjects With Urea Cycle Disorders (UCDs)

NCT01347073
View directory record Official record
Conditions
Urea Cycle Disorders
Interventions
HPN-100
Drug
Lead sponsor
Amgen
Industry
Eligibility
29 Days to 6 Years
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2013
U.S. locations
8
States / cities
Los Angeles, California • Washington D.C., District of Columbia • Portland, Maine + 5 more
Source: ClinicalTrials.gov public record
Updated Jul 10, 2024 · Synced Sep 24, 2026, 1:02 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers Results available

RDEA3170 Bioavailability Study

NCT02336594
View directory record Official record
Conditions
Gout
Interventions
RDEA3170 10 mg, RDEA3170 2.5 mg
Drug
Lead sponsor
Ardea Biosciences, Inc.
Industry
Eligibility
18 Years to 65 Years · Male only
Enrollment
15 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2014 – 2015
U.S. locations
1
States / cities
Austin, Texas
Source: ClinicalTrials.gov public record
Updated Oct 12, 2017 · Synced Sep 24, 2026, 1:02 AM EDT
Completed Phase 4 Interventional

Phase IV Study to Gather More Information About the Safety of ACZONE Gel, 5% in Treating Subjects With Acne Who Have G6PD Deficiency

NCT00243542
View directory record Official record
Conditions
Acne Vulgaris
Interventions
ACZONE Gel, 5%, Vehicle
Drug
Lead sponsor
Allergan
Industry
Eligibility
12 Years and older
Enrollment
64 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2006
U.S. locations
29
States / cities
Birmingham, Alabama • San Diego, California • San Francisco, California + 24 more
Source: ClinicalTrials.gov public record
Updated May 29, 2011 · Synced Sep 24, 2026, 1:02 AM EDT