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ClinicalTrials.gov public records Last synced Oct 31, 2026, 3:15 AM EDT

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Showing 937–960 of 2,426 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Metabolic Abnormalities Clear all
Completed Not applicable Interventional Results available

Clinical Utility of Pediatric Whole Exome Sequencing

NCT03525431
View directory record Official record
Conditions
Encephalopathy, Birth Defect, Intellectual Disability, Multiple Congenital Anomaly, Metabolic Disease, Epilepsy, Neuro-Degenerative Disease, Cerebral Palsy, Developmental Delay, Developmental Defect
Interventions
Whole Exome Sequencing
Diagnostic Test
Lead sponsor
University of California, San Francisco
Other
Eligibility
Up to 25 Years
Enrollment
529 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2022
U.S. locations
4
States / cities
Fresno, California • Oakland, California • San Francisco, California
Source: ClinicalTrials.gov public record
Updated Jul 18, 2023 · Synced Oct 31, 2026, 3:15 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 31, 2026, 3:15 AM EDT
Terminated Phase 2Phase 3 Interventional Results available

Effects of Livoletide (AZP-531) on Food-related Behaviors in Patients With Prader-Willi Syndrome

NCT03790865
View directory record Official record
Conditions
Prader-Willi Syndrome, Hyperphagia
Interventions
Livoletide, Placebo
Drug
Lead sponsor
Millendo Therapeutics SAS
Industry
Eligibility
4 Years to 65 Years
Enrollment
158 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2020
U.S. locations
15
States / cities
Birmingham, Alabama • Orange, California • San Diego, California + 12 more
Source: ClinicalTrials.gov public record
Updated Feb 17, 2021 · Synced Oct 31, 2026, 3:15 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

I-123 Brain Studies of Serotonin Metabolism in Psychiatric Patients and Normal Volunteers

NCT00001771
View directory record Official record
Conditions
Healthy, Mental Disorder, Obsessive Compulsive Disorder, Schizophrenia, Tourette Syndrome
Interventions
Not listed
Lead sponsor
National Institute of Mental Health (NIMH)
NIH
Eligibility
Not listed
Enrollment
112 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
1998 – 2003
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 31, 2026, 3:15 AM EDT
Terminated Phase 1Phase 2 Interventional Results available

Lentiviral Vector Gene Therapy - The Guard1 Trial of AVR-RD-02 for Subjects With Type 1 Gaucher Disease

NCT04145037
View directory record Official record
Conditions
Gaucher Disease
Interventions
AVR-RD-02
Drug
Lead sponsor
AVROBIO
Industry
Eligibility
18 Years to 50 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2023
U.S. locations
4
States / cities
San Diego, California • Iowa City, Iowa • Hackensack, New Jersey + 1 more
Source: ClinicalTrials.gov public record
Updated Jan 18, 2024 · Synced Oct 31, 2026, 3:15 AM EDT
Recruiting Not applicable Interventional

CAPACITY (Cardiac Amyloidosis and Physical ACtivITY) Study

NCT06096675
View directory record Official record
Conditions
Cardiac Amyloidosis
Interventions
• Cardiac Rehabilitation
Behavioral
Lead sponsor
Wake Forest University Health Sciences
Other
Eligibility
18 Years and older
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
1
States / cities
Charlotte, North Carolina
Source: ClinicalTrials.gov public record
Updated Jul 21, 2026 · Synced Oct 31, 2026, 3:15 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

BioThrax® (Anthrax) Vaccine in Pregnancy Registry

NCT01653392
View directory record Official record
Conditions
Pregnancy Complications, Pregnancy Outcome, Congenital Abnormalities
Interventions
Observational Intervention
Biological
Lead sponsor
Emergent BioSolutions
Industry
Eligibility
Female only
Enrollment
98 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2020
U.S. locations
1
States / cities
San Diego, California
Source: ClinicalTrials.gov public record
Updated Mar 15, 2024 · Synced Oct 31, 2026, 3:15 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Nexus of Risk: Sexual Assault, Alcohol Use, and Risky Sex Among College Women

NCT05892692
View directory record Official record
Conditions
Sexual Assault, Substance Use, Risky Health Behavior, Ema
Interventions
Ecological Momentary Intervention
Behavioral
Lead sponsor
University of New Mexico
Other
Eligibility
18 Years to 24 Years · Female only
Enrollment
183 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2023
U.S. locations
1
States / cities
Albuquerque, New Mexico
Source: ClinicalTrials.gov public record
Updated Apr 2, 2024 · Synced Oct 31, 2026, 3:15 AM EDT
Completed No phase listed Observational Results available

Carotid Structure and Function in MPS Syndromes: A Multicenter Study of the Lysosomal Disease Network

NCT01586871
View directory record Official record
Conditions
Mucopolysaccharidoses
Interventions
Not listed
Lead sponsor
University of Minnesota
Other
Eligibility
3 Years to 18 Years
Enrollment
1,147 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2017
U.S. locations
2
States / cities
Orange, California • Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Feb 24, 2021 · Synced Oct 31, 2026, 3:15 AM EDT
Completed Phase 2 Interventional Results available

Controlled Trial of Panhematin in Treatment of Acute Attacks of Porphyria

NCT02180412
View directory record Official record
Conditions
Acute Porphyrias
Interventions
Panhematin, Glucose
Biological · Other
Lead sponsor
The University of Texas Medical Branch, Galveston
Other
Eligibility
18 Years to 100 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2022
U.S. locations
1
States / cities
Galveston, Texas
Source: ClinicalTrials.gov public record
Updated Apr 11, 2025 · Synced Oct 31, 2026, 3:15 AM EDT
Recruiting Not applicable Interventional

Exercise Training in Transthyretin Cardiac Amyloidosis

NCT05797857
View directory record Official record
Conditions
Amyloid Cardiomyopathy, Transthyretin Cardiac Amyloidosis
Interventions
Exercise Training
Other
Lead sponsor
Brigham and Women's Hospital
Other
Eligibility
60 Years to 90 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2030
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Mar 18, 2025 · Synced Oct 31, 2026, 3:15 AM EDT
Recruiting Not applicable Interventional Accepts healthy volunteers

Identifying and Caring for Individuals With Inherited Cancer Syndrome

NCT04494945
View directory record Official record
Conditions
BRCA1/2-Associated Hereditary Breast and Ovarian Cancer Syndrome, Breast Ductal Carcinoma In Situ, Hematopoietic and Lymphoid System Neoplasm, Hereditary Neoplastic Syndrome, Lynch Syndrome, Malignant Solid Neoplasm
Interventions
Biospecimen Collection, Genetic Counseling, Genetic Testing, Survey Administration
Procedure · Other
Lead sponsor
OHSU Knight Cancer Institute
Other
Eligibility
18 Years and older
Enrollment
27,500 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2027
U.S. locations
2
States / cities
Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Jul 14, 2026 · Synced Oct 31, 2026, 3:15 AM EDT
Completed Not applicable Interventional

Evaluation of PKU Sphere

NCT03419819
View directory record Official record
Conditions
Phenylketonurias
Interventions
PKU Sphere
Dietary Supplement
Lead sponsor
Vitaflo International, Ltd
Industry
Eligibility
3 Years and older
Enrollment
28 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2019
U.S. locations
1
States / cities
Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Feb 16, 2024 · Synced Oct 31, 2026, 3:15 AM EDT
Completed Phase 3 Interventional Results available

Safety/Effectiveness Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Cysteamine Treatment Naive Patients With Cystinosis

NCT01744782
View directory record Official record
Conditions
Cystinosis
Interventions
RP103
Drug
Lead sponsor
Amgen
Industry
Eligibility
Up to 6 Years
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2016
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Dec 27, 2024 · Synced Oct 31, 2026, 3:15 AM EDT
Completed Phase 1Phase 2 Interventional Results available

A Repeated Study of KRN23 in Adults With X-Linked Hypophosphatemia

NCT01340482
View directory record Official record
Conditions
X-linked Hypophosphatemia
Interventions
KRN23
Drug
Lead sponsor
Kyowa Kirin Co., Ltd.
Industry
Eligibility
18 Years and older
Enrollment
29 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2013
U.S. locations
5
States / cities
San Francisco, California • New Haven, Connecticut • Indianapolis, Indiana + 2 more
Source: ClinicalTrials.gov public record
Updated Jun 18, 2024 · Synced Oct 31, 2026, 3:15 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Extension Study of PRX-102 for up to 60 Months

NCT01981720
View directory record Official record
Conditions
Fabry Disease
Interventions
PRX-102 (pegunigalsidase alfa)
Biological
Lead sponsor
Protalix
Industry
Eligibility
18 Years and older
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2021
U.S. locations
5
States / cities
Gainesville, Florida • Iowa City, Iowa • Baltimore, Maryland + 2 more
Source: ClinicalTrials.gov public record
Updated Sep 13, 2023 · Synced Oct 31, 2026, 3:15 AM EDT
Completed Phase 3 Interventional Results available

Compassionate Treatment of Patients With Inborn Errors of Bile Acid Metabolism With Cholic Acid

NCT00007020
View directory record Official record
Conditions
Infantile Refsum's Disease, Zellweger Syndrome, Adrenoleukodystrophy, Peroxisomal Disorders, Cholestasis
Interventions
Cholic Acids
Drug
Lead sponsor
Mirum Pharmaceuticals, Inc.
Industry
Eligibility
Not listed
Enrollment
85 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1992 – 2009
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Oct 3, 2023 · Synced Oct 31, 2026, 3:15 AM EDT
Active, not recruiting Phase 3 Interventional Results available

Combination Chemotherapy With or Without Atezolizumab in Treating Patients With Stage III Colon Cancer and Deficient DNA Mismatch Repair

NCT02912559
View directory record Official record
Conditions
Colon Adenocarcinoma, DNA Repair Disorder, Lynch Syndrome, Stage III Colon Cancer AJCC v8
Interventions
Atezolizumab, Biospecimen Collection, Computed Tomography, Fluorouracil, Leucovorin Calcium, Magnetic Resonance Imaging, Oxaliplatin, Quality-of-Life Assessment
Drug · Procedure · Other
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
12 Years and older
Enrollment
712 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2026
U.S. locations
1026
States / cities
Anchorage, Alaska • Fairbanks, Alaska • Phoenix, Arizona + 683 more
Source: ClinicalTrials.gov public record
Updated Oct 28, 2026 · Synced Oct 31, 2026, 3:15 AM EDT
Recruiting No phase listed Observational

Fabry Disease Registry & Pregnancy Sub-registry

NCT00196742
View directory record Official record
Conditions
Fabry Disease
Interventions
Not listed
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
Not listed
Enrollment
9,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2034
U.S. locations
91
States / cities
Birmingham, Alabama • Phoenix, Arizona • Tucson, Arizona + 70 more
Source: ClinicalTrials.gov public record
Updated Oct 14, 2026 · Synced Oct 31, 2026, 3:15 AM EDT
Completed Phase 1 Interventional

Safety and Dose Ranging Study of Insulin Receptor MAb-IDUA Fusion Protein in Patients With MPS I

NCT02371226
View directory record Official record
Conditions
Mucopolysaccharidosis I
Interventions
AGT-181 (HIRMAb-IDUA)
Drug
Lead sponsor
ArmaGen, Inc
Industry
Eligibility
18 Years and older
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
6
States / cities
Oakland, California • Orange, California • Decatur, Georgia + 3 more
Source: ClinicalTrials.gov public record
Updated Feb 21, 2023 · Synced Oct 31, 2026, 3:15 AM EDT
Completed Phase 3 Interventional Results available

Open-Label Extension Study of the Long-Term Effects of Migalastat HCL in Patients With Fabry Disease

NCT02194985
View directory record Official record
Conditions
Fabry Disease
Interventions
migalastat HCl 150 mg
Drug
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
84 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2019
U.S. locations
7
States / cities
Atlanta, Georgia • Kansas City, Kansas • Grand Rapids, Michigan + 4 more
Source: ClinicalTrials.gov public record
Updated Dec 21, 2020 · Synced Oct 31, 2026, 3:15 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 31, 2026, 3:15 AM EDT
Completed No phase listed Observational

Characteristics of Prader-Willi Syndrome and Early-onset Morbid Obesity

NCT00375089
View directory record Official record
Conditions
Prader-Willi Syndrome, Obesity
Interventions
Group 1, Group 2
Other
Lead sponsor
University of Florida
Other
Eligibility
Up to 60 Years
Enrollment
392 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2014
U.S. locations
4
States / cities
Orange, California • Gainesville, Florida • Kansas City, Kansas + 1 more
Source: ClinicalTrials.gov public record
Updated Sep 22, 2014 · Synced Oct 31, 2026, 3:15 AM EDT
Terminated Not applicable Interventional

Optimizing the Social Engagement System in Prader-Willi Syndrome: Insights From the Polyvagal Theory

NCT03101826
View directory record Official record
Conditions
Prader-Willi Syndrome
Interventions
Listening Project Protocol
Behavioral
Lead sponsor
Indiana University
Other
Eligibility
13 Years to 25 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2023
U.S. locations
1
States / cities
Brewster, Massachusetts
Source: ClinicalTrials.gov public record
Updated Apr 3, 2023 · Synced Oct 31, 2026, 3:15 AM EDT