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ClinicalTrials.gov public records Last synced Oct 6, 2026, 4:35 AM EDT

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Showing 73–96 of 2,371 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Congenital Metabolic Disorder Clear all
Completed Not applicable Interventional Accepts healthy volunteers

Universal Endometrial Cancer DNA Sequencing for Detection of Lynch Syndrome and Personalized Care

NCT03460483
View directory record Official record
Conditions
Endometrial Adenocarcinoma, Endometrial Carcinoma, Lynch Syndrome, Relatives
Interventions
Genetic Counseling, Genetic Testing, Laboratory Biomarker Analysis, Mutation Carrier Screening
Other · Procedure
Lead sponsor
Ohio State University Comprehensive Cancer Center
Other
Eligibility
18 Years and older
Enrollment
1,001 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2025
U.S. locations
9
States / cities
Akron, Ohio • Canton, Ohio • Cincinnati, Ohio + 3 more
Source: ClinicalTrials.gov public record
Updated Oct 3, 2025 · Synced Oct 6, 2026, 4:35 AM EDT
Completed Phase 3 Interventional Results available

Efficacy and Safety of Burosumab Versus Oral Phosphate and Active Vitamin D Treatment in Pediatric Patients With XLH

NCT02915705
View directory record Official record
Conditions
X-Linked Hypophosphatemia
Interventions
burosumab, Oral Phosphate Supplement, active vitamin D
Biological · Drug
Lead sponsor
Kyowa Kirin, Inc.
Industry
Eligibility
1 Year to 12 Years
Enrollment
61 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
5
States / cities
Los Angeles, California • San Francisco, California • Indianapolis, Indiana + 2 more
Source: ClinicalTrials.gov public record
Updated Aug 29, 2024 · Synced Oct 6, 2026, 4:35 AM EDT
Terminated Phase 3 Interventional Results available

Niemann-Pick Type C Treatment With Adrabetadex for Symptoms of Brain and Nervous System

NCT03643562
View directory record Official record
Conditions
Niemann-Pick Type C Disease
Interventions
Adrabetadex
Drug
Lead sponsor
Mandos LLC
Industry
Eligibility
4 Years and older
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
17
States / cities
Little Rock, Arkansas • Loma Linda, California • Los Angeles, California + 13 more
Source: ClinicalTrials.gov public record
Updated Jan 17, 2024 · Synced Oct 6, 2026, 4:35 AM EDT
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 6, 2026, 4:35 AM EDT
Terminated Phase 2 Interventional Results available

Hematopoietic Stem Cell Transplantation (HSCT) for Children With SCID Utilizing Alemtuzumab, Plerixafor & Filgrastim

NCT01182675
View directory record Official record
Conditions
Severe Combined Immunodeficiency
Interventions
Transplant Conditioning with Mobilization Only, Transplant Conditioning with Mobilization and Alemtuzumab
Drug
Lead sponsor
University of California, San Francisco
Other
Eligibility
Up to 3 Years
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2013
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Jul 31, 2018 · Synced Oct 6, 2026, 4:35 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 6, 2026, 4:35 AM EDT
Completed Not applicable Interventional Results available

Effect of Chronic Incretin-based Therapy in Cystic Fibrosis

NCT01879228
View directory record Official record
Conditions
Cystic Fibrosis, Pancreatic Insufficiency
Interventions
Sitagliptin, Placebo
Drug
Lead sponsor
University of Pennsylvania
Other
Eligibility
18 Years and older
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2020
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 8, 2022 · Synced Oct 6, 2026, 4:35 AM EDT
Completed No phase listed Observational

Mucopolysaccharidosis (MPS) VI Clinical Surveillance Program (CSP)

NCT00214773
View directory record Official record
Conditions
Mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy Syndrome)
Interventions
Not listed
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
Not listed
Enrollment
237 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2020
U.S. locations
23
States / cities
Irvine, California • Los Angeles, California • Oakland, California + 19 more
Source: ClinicalTrials.gov public record
Updated Apr 5, 2021 · Synced Oct 6, 2026, 4:35 AM EDT
Not listed No phase listed Observational

Xeroderma Pigmentosum Patient Experiences

NCT01123694
View directory record Official record
Conditions
Xeroderma Pigmentosum
Interventions
Survey Study
Other
Lead sponsor
Henry Ford Health System
Other
Eligibility
Not listed
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2010
U.S. locations
1
States / cities
Detroit, Michigan
Source: ClinicalTrials.gov public record
Updated May 14, 2010 · Synced Oct 6, 2026, 4:35 AM EDT
Completed Phase 2 Interventional

Hormone Replacement Therapy to Treat Turner Syndrome

NCT00013546
View directory record Official record
Conditions
Osteoporosis, Turner's Syndrome
Interventions
TMTDS
Drug
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
Female only
Enrollment
92 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2002
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 6, 2026, 4:35 AM EDT
Recruiting No phase listed Observational

Natural History and Structural Functional Relationships in Fabry Renal Disease Treatment Outcomes(Changes)in Fabry Renal Disease Study

NCT01581424
View directory record Official record
Conditions
Fabry Disease
Interventions
Not listed
Lead sponsor
University of Minnesota
Other
Eligibility
1 Year to 75 Years
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2030
U.S. locations
2
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Aug 14, 2026 · Synced Oct 6, 2026, 4:35 AM EDT
Recruiting Phase 1Phase 2 Interventional

Reduced Intensity Conditioning and Familial HLA-Mismatched BMT for Non-Malignant Disorders

NCT03128996
View directory record Official record
Conditions
Severe Sickle Cell Disease, Bone Marrow Failure Syndromes, Metabolic Disorders, Immunologic Disorders, Hemoglobinopathies, Non-malignant Disorders
Interventions
RIC regimen, GVHD prophylaxis regimen
Drug
Lead sponsor
Washington University School of Medicine
Other
Eligibility
1 Day to 21 Years
Enrollment
29 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2033
U.S. locations
4
States / cities
New Haven, Connecticut • Wilmington, Delaware • Grand Rapids, Michigan + 1 more
Source: ClinicalTrials.gov public record
Updated May 29, 2026 · Synced Oct 6, 2026, 4:35 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Sapropterin in Individuals With Phenylketonuria

NCT00730080
View directory record Official record
Conditions
Phenylketonuria
Interventions
Sapropterin (Kuvan)
Drug
Lead sponsor
Washington University School of Medicine
Other
Eligibility
6 Years to 50 Years
Enrollment
45 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2008 – 2018
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Jun 18, 2018 · Synced Oct 6, 2026, 4:35 AM EDT
Completed No phase listed Observational

Studying Phosphorus Metabolism

NCT00066183
View directory record Official record
Conditions
Phosphorus Metabolism
Interventions
Not listed
Lead sponsor
National Institute of Dental and Craniofacial Research (NIDCR)
NIH
Eligibility
Not listed
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2005
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 6, 2026, 4:35 AM EDT
Completed Phase 2 Interventional Accepts healthy volunteers Results available

Understanding the Role of Gut Microbiota in Hyperphagia in Prader-Willi Syndrome

NCT05541003
View directory record Official record
Conditions
Prader-Willi Syndrome, Obesity
Interventions
NBT-NM108
Drug
Lead sponsor
Rutgers, The State University of New Jersey
Other
Eligibility
18 Years to 35 Years
Enrollment
12 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2023
U.S. locations
1
States / cities
New Brunswick, New Jersey
Source: ClinicalTrials.gov public record
Updated Jul 16, 2024 · Synced Oct 6, 2026, 4:35 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Gene Therapy For Children With Variant Late Infantile Neuronal Ceroid Lipofuscinosis 6 (vLINCL6) Disease

NCT02725580
View directory record Official record
Conditions
Variant Late-Infantile Neuronal Ceroid Lipofuscinosis
Interventions
AT-GTX-501
Genetic
Lead sponsor
Emily de los Reyes
Other
Eligibility
1 Year and older
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2024
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Oct 24, 2025 · Synced Oct 6, 2026, 4:35 AM EDT
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 6, 2026, 4:35 AM EDT
Completed Phase 3 Interventional Results available

Comparison of Chronocort® With Standard Glucocorticoid Therapy in Patients With Congenital Adrenal Hyperplasia

NCT02716818
View directory record Official record
Conditions
Congenital Adrenal Hyperplasia
Interventions
Chronocort®, standard glucocorticoid therapy
Drug
Lead sponsor
Neurocrine UK Limited
Industry
Eligibility
18 Years and older
Enrollment
122 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2018
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 2, 2026 · Synced Oct 6, 2026, 4:35 AM EDT
Completed Phase 3 Interventional Results available

Study of ARO-APOC3 (Plozasiran) in Adults With Familial Chylomicronemia Syndrome (FCS)

NCT05089084
View directory record Official record
Conditions
Familial Chylomicronemia
Interventions
Plozasiran, Placebo
Drug
Lead sponsor
Arrowhead Pharmaceuticals
Industry
Eligibility
18 Years and older
Enrollment
75 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2026
U.S. locations
9
States / cities
Boca Raton, Florida • Suwanee, Georgia • Indianapolis, Indiana + 5 more
Source: ClinicalTrials.gov public record
Updated Jun 5, 2026 · Synced Oct 6, 2026, 4:35 AM EDT
Active, not recruiting Not applicable Interventional

Daily Caloric Restriction in ADPKD

NCT04907799
View directory record Official record
Conditions
Autosomal Dominant Polycystic Kidney, Overweight and Obesity
Interventions
Daily caloric restriction, Standard advice control
Behavioral · Other
Lead sponsor
University of Colorado, Denver
Other
Eligibility
18 Years to 65 Years
Enrollment
125 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2027
U.S. locations
1
States / cities
Aurora, Colorado
Source: ClinicalTrials.gov public record
Updated Aug 12, 2025 · Synced Oct 6, 2026, 4:35 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Validation of Biomarkers in Amyotrophic Lateral Sclerosis (ALS)

NCT00677768
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis, Lou Gehrig's Disease, Primary Lateral Sclerosis, Nervous System Diseases, Hereditary Spastic Paraparesis
Interventions
No intervention
Other
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
30 Years to 80 Years
Enrollment
475 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2008 – 2015
U.S. locations
29
States / cities
Phoenix, Arizona • Orange, California • Jacksonville, Florida + 24 more
Source: ClinicalTrials.gov public record
Updated Jun 3, 2016 · Synced Oct 6, 2026, 4:35 AM EDT
Not listed Phase 2 Interventional

Cholesterol and Antioxidant Treatment in Patients With Smith-Lemli-Opitz Syndrome (SLOS)

NCT01773278
View directory record Official record
Conditions
Smith-Lemli-Opitz Syndrome, Cone-Rod Dystrophy, Hearing Loss
Interventions
Antioxidants, Cholesterol
Drug
Lead sponsor
University of Colorado, Denver
Other
Eligibility
Up to 65 Years
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2025
U.S. locations
1
States / cities
Aurora, Colorado
Source: ClinicalTrials.gov public record
Updated May 8, 2024 · Synced Oct 6, 2026, 4:35 AM EDT
Completed Phase 1Phase 2 Interventional

AAVRh.10 Administered to Children With Late Infantile Neuronal Ceroid Lipofuscinosis

NCT01414985
View directory record Official record
Conditions
Late Infantile Neuronal Ceroid Lipofuscinosis, Batten Disease
Interventions
AAVrh.10CUCLN2
Biological
Lead sponsor
Weill Medical College of Cornell University
Other
Eligibility
3 Years to 18 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2017
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Aug 31, 2020 · Synced Oct 6, 2026, 4:35 AM EDT
Completed Phase 2 Interventional Results available

Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders

NCT01043640
View directory record Official record
Conditions
Mucopolysaccharidosis, Hurler Syndrome, Hunter Syndrome, Maroteaux-Lamy Syndrome, Sly Syndrome, Alpha Mannosidosis, Fucosidosis, Aspartylglucosaminuria, Adrenoleukodystrophy (ALD), Krabbe Disease, Metachromatic Leukodystrophy (MLD), Sphingolipidoses, Peroxisomal Disorders
Interventions
Campath-1H, Cyclophosphamide, Busulfan, Allogeneic stem cell transplantation, Cyclosporine A, Mycophenolate Mofetil
Drug · Procedure
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 21 Years
Enrollment
46 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2017
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Feb 5, 2018 · Synced Oct 6, 2026, 4:35 AM EDT