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ClinicalTrials.gov public records Last synced Oct 5, 2026, 1:22 PM EDT

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Showing 49–72 of 2,371 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Congenital Metabolic Disorder Clear all
Completed Phase 2 Interventional Results available

A Study of ALN-PCSSC in Participants With Homozygous Familial Hypercholesterolemia (HoFH)

NCT02963311
View directory record Official record
Conditions
Homozygous Familial Hypercholesterolemia
Interventions
ALN-PCSSC, Standard of Care
Drug
Lead sponsor
The Medicines Company
Industry
Eligibility
12 Years and older
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2018
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated May 18, 2020 · Synced Oct 5, 2026, 1:22 PM EDT
Completed Phase 2 Interventional Results available

Safety and Efficacy Study of Phenoptin in Subjects With Hyperphenylalaninemia Due to BH4 Deficiency

NCT00355264
View directory record Official record
Conditions
Tetrahydrobiopterin Deficiencies, Hyperphenylalaninemia, Non-Phenylketonuric
Interventions
Phenoptin
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
Not listed
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2009
U.S. locations
9
States / cities
Los Angeles, California • Chicago, Illinois • Minneapolis, Minnesota + 6 more
Source: ClinicalTrials.gov public record
Updated Sep 25, 2020 · Synced Oct 5, 2026, 1:22 PM EDT
Completed Phase 1 Interventional

Gene Therapy for the Treatment of Fanconi's Anemia Type C

NCT00001399
View directory record Official record
Conditions
Fanconi's Anemia, Pancytopenia
Interventions
Transduced CD34+ Cells
Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
5 Years and older
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1993 – 2009
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 2, 2017 · Synced Oct 5, 2026, 1:22 PM EDT
Completed No phase listed Observational Results available

Non-interventional Substudy of ALX-HPP-502 to Assess Natural History of Patients With Juvenile-onset HPP Who Served as Historical Controls in ENB-006-09

NCT02235493
View directory record Official record
Conditions
Hypophosphatasia, Bone Disease
Interventions
Retrospective Case Only
Biological
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
5 Years and older
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Mar 13, 2019 · Synced Oct 5, 2026, 1:22 PM EDT
Completed Phase 1 Interventional

Effect of ACP-501 on Safety, Tolerability, Pharmacokinetics and Pharmacodynamics in Subjects With Coronary Artery Disease

NCT01554800
View directory record Official record
Conditions
Coronary Artery Disease, Atherosclerosis
Interventions
ACP-501
Drug
Lead sponsor
MedImmune LLC
Industry
Eligibility
30 Years to 85 Years
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 14, 2019 · Synced Oct 5, 2026, 1:22 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Remote Neurocognitive and Psychological Assessment in PKU

NCT04943393
View directory record Official record
Conditions
Phenylketonurias
Interventions
nointervention
Other
Lead sponsor
University of Missouri-Columbia
Other
Eligibility
18 Years to 40 Years
Enrollment
84 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2021
U.S. locations
2
States / cities
Atlanta, Georgia • Columbia, Missouri
Source: ClinicalTrials.gov public record
Updated Apr 22, 2022 · Synced Oct 5, 2026, 1:22 PM EDT
Active, not recruiting Not applicable Interventional

Screening With Whole Body MRI For Detection Of Primary Tumors In Children And Adults With Li-Fraumeni Syndrome (LFS) And Other Cancer Predisposition Syndromes

NCT02950987
View directory record Official record
Conditions
Li-Fraumeni Syndrome
Interventions
Whole Body MRI
Device
Lead sponsor
Dana-Farber Cancer Institute
Other
Eligibility
Not listed
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2027
U.S. locations
3
States / cities
Boston, Massachusetts • New York, New York • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jan 7, 2026 · Synced Oct 5, 2026, 1:22 PM EDT
Not listed No phase listed Observational Accepts healthy volunteers

The Molecular Predisposition to Hereditary Nonpolyposis Colon Cancer (HNPCC)

NCT01447199
View directory record Official record
Conditions
Bladder Cancer, Colorectal Cancer, Endometrial Cancer, Kidney Cancer, Skin Cancer, Uterus Cancer
Interventions
Health and Diet Questionnaire
Behavioral
Lead sponsor
M.D. Anderson Cancer Center
Other
Eligibility
18 Years and older
Enrollment
2,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
1994 – 2020
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jan 7, 2020 · Synced Oct 5, 2026, 1:22 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

A Clinical Trial of PR001 (LY3884961) in Patients With Peripheral Manifestations of Gaucher Disease (PROCEED)

NCT05487599
View directory record Official record
Conditions
Gaucher Disease, Gaucher Disease, Type 1
Interventions
LY3884961
Genetic
Lead sponsor
Prevail Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2031
U.S. locations
2
States / cities
Durham, North Carolina • Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Aug 17, 2026 · Synced Oct 5, 2026, 1:22 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Innovative Ultrasound Technology in Neuromuscular Disease

NCT02104921
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis, Muscular Dystrophy, Radiculopathy, Myopathy, Polyneuropathy and Mononeuropathies, Trauma Injury, Orthopedic Disorder
Interventions
Not listed
Lead sponsor
Beth Israel Deaconess Medical Center
Other
Eligibility
18 Years to 90 Years
Enrollment
223 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2020
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Mar 11, 2022 · Synced Oct 5, 2026, 1:22 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Effects of Adalimumab in Mucopolysaccharidosis Types I, II and VI

NCT02437253
View directory record Official record
Conditions
Mucopolysaccharidosis Type I, Mucopolysaccharidosis Type II, Mucopolysaccharidosis Type VI
Interventions
Adalimumab, Placebo
Drug · Other
Lead sponsor
Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
Other
Eligibility
5 Years and older
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2016
U.S. locations
1
States / cities
Torrance, California
Source: ClinicalTrials.gov public record
Updated Sep 15, 2017 · Synced Oct 5, 2026, 1:22 PM EDT
Completed Phase 4 Interventional

N-of-1 for Beta-Blockers in Cardiac Amyloidosis

NCT05019027
View directory record Official record
Conditions
Cardiac Amyloidosis, Heart Diseases, TTR Cardiac Amyloidosis
Interventions
Beta blocker
Drug
Lead sponsor
Weill Medical College of Cornell University
Other
Eligibility
65 Years and older
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jul 29, 2026 · Synced Oct 5, 2026, 1:22 PM EDT
Completed Phase 2 Interventional Results available

Effects of Human Leptin Replacement

NCT00657605
View directory record Official record
Conditions
Obesity, Metabolic Syndrome, Diabetes
Interventions
Recombinant methionyl human leptin
Drug
Lead sponsor
University of Miami
Other
Eligibility
18 Years to 80 Years
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2010
U.S. locations
1
States / cities
Miami, Florida
Source: ClinicalTrials.gov public record
Updated Feb 13, 2020 · Synced Oct 5, 2026, 1:22 PM EDT
Recruiting Phase 1 Interventional

A Safety and Tolerability Trial Evaluating CTX310 in Participants With Refractory Dyslipidemias

NCT07491172
View directory record Official record
Conditions
Cardiovascular, Metabolic Disease, Dyslipidemias, Lipid Disorder, Hypertriglyceridemia, Heterozygous Familial Hypercholesterolemia (HeFH), Homozygous Familial Hypercholesterolemia (HoFH), Severe Hypertriglyceridemia (sHTG), Mixed Hyperlipemia, Hypercholesterolaemia
Interventions
CTX310
Drug
Lead sponsor
CRISPR Therapeutics AG
Industry
Eligibility
18 Years to 75 Years
Enrollment
90 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2028
U.S. locations
8
States / cities
Hialeah, Florida • Jacksonville, Florida • Orlando, Florida + 5 more
Source: ClinicalTrials.gov public record
Updated Sep 18, 2026 · Synced Oct 5, 2026, 1:22 PM EDT
Completed Phase 2 Interventional Results available

Long-Term Extension of Previous rAvPAL-PEG Protocols in Subjects With PKU (PAL-003)

NCT00924703
View directory record Official record
Conditions
Phenylketonuria
Interventions
rAvPAL-PEG
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
16 Years to 55 Years
Enrollment
68 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2019
U.S. locations
14
States / cities
Aurora, Colorado • Gainesville, Florida • Chicago, Illinois + 11 more
Source: ClinicalTrials.gov public record
Updated Oct 12, 2021 · Synced Oct 5, 2026, 1:22 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Feasibility, Acceptability and Directional Signal Effect on Blood Folate Levels of Iodized Salt Fortified With Folic Acid: Clinical Study

NCT05935631
View directory record Official record
Conditions
Neural Tube Defects, Folic Acid Deficiency, Fortification
Interventions
Salt with folic acid and iodine
Dietary Supplement
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
18 Years to 40 Years · Female only
Enrollment
32 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2023
U.S. locations
1
States / cities
Birmingham, Alabama
Source: ClinicalTrials.gov public record
Updated Aug 1, 2023 · Synced Oct 5, 2026, 1:22 PM EDT
Active, not recruiting No phase listed Observational Accepts healthy volunteers

The Pancreas Interception Center (PIC) for Early Detection, Prevention, and Novel Therapeutics

NCT05692596
View directory record Official record
Conditions
Pancreatic Ductal Adenocarcinoma, Pancreatic Cyst, Chronic Pancreatitis, Fatty Pancreas, Genetic Pancreatic Cancer, Genetic Pancreatitis, BRCA Mutation, Lynch Syndrome, FAP, Familial Atypical Multiple Mole-Melanoma, PALB2 Gene Mutation, Peutz-Jeghers Syndrome, Ataxia Telangiectasia
Interventions
Data collection
Other
Lead sponsor
H. Lee Moffitt Cancer Center and Research Institute
Other
Eligibility
18 Years and older
Enrollment
317 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2027
U.S. locations
1
States / cities
Tampa, Florida
Source: ClinicalTrials.gov public record
Updated Sep 15, 2026 · Synced Oct 5, 2026, 1:22 PM EDT
Completed Early Phase 1 Interventional Results available

Calcitriol Monotherapy for X-Linked Hypophosphatemia

NCT03748966
View directory record Official record
Conditions
X-linked Hypophosphatemia, Hypophosphatemic Rickets, Hypophosphatemic Rickets, X-Linked Dominant
Interventions
Calcitriol
Drug
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
3 Years to 70 Years
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2026
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated May 7, 2026 · Synced Oct 5, 2026, 1:22 PM EDT
Recruiting Phase 2 Interventional

A Study to Investigate the Clinical Effect and the Safety of PRX-115 Infused Intravenously at Different Dosing Regimens, With and Without Methotrexate, Versus Placebo in Adults Gout Patients (RELEASE)

NCT07280156
View directory record Official record
Conditions
Gout
Interventions
PRX-115, Methotrexate (MTX), PRX-115 placebo, Placebo-Methotrexate
Biological · Drug · Other
Lead sponsor
Protalix
Industry
Eligibility
18 Years and older
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
26
States / cities
Glendale, Arizona • Little Rock, Arkansas • Anaheim, California + 20 more
Source: ClinicalTrials.gov public record
Updated Sep 29, 2026 · Synced Oct 5, 2026, 1:22 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Comparison of Two Forms of Hydrocortisone in Patients With Congenital Adrenal Hyperplasia

NCT00519818
View directory record Official record
Conditions
Congenital Adrenal Hyperplasia, 21-Hydroxylase Deficiency, Adrenogenital Syndrome
Interventions
Chronocort, Cortef
Drug
Lead sponsor
Neurocrine UK Limited
Industry
Eligibility
16 Years to 60 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2009
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Apr 26, 2022 · Synced Oct 5, 2026, 1:22 PM EDT
Completed No phase listed Observational Accepts healthy volunteers Results available

Early-onset Obesity and Cognitive Impairment in Children With Pseudohypoparathyroidism

NCT02411461
View directory record Official record
Conditions
Pseudohypoparathyroidism, Albright Hereditary Osteodystrophy
Interventions
Not listed
Lead sponsor
Vanderbilt University Medical Center
Other
Eligibility
6 Years to 18 Years
Enrollment
39 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2014 – 2017
U.S. locations
1
States / cities
Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Nov 13, 2019 · Synced Oct 5, 2026, 1:22 PM EDT
Active, not recruiting Phase 1 Interventional

Phase 1 Study of GC1130A in Patients With Sanfilippo Syndrome Type A (MPS IIIA)

NCT06567769
View directory record Official record
Conditions
Sanfilippo Syndrome Type A
Interventions
GC1130A
Drug
Lead sponsor
GC Biopharma Corp
Industry
Eligibility
12 Months to 18 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
2
States / cities
Oakland, California • Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Sep 8, 2026 · Synced Oct 5, 2026, 1:22 PM EDT
Recruiting Phase 3 Interventional

The Cancer of the Pancreas Screening-5 CAPS5)Study

NCT02000089
View directory record Official record
Conditions
Pancreas Cancer, Peutz-Jeghers Syndrome (PJS), Gene Mutation, Germline Mutation Carrier, Lynch Syndrome
Interventions
Secretin, MRI, Tumor marker gene test with CA19-9
Drug · Diagnostic Test · Other
Lead sponsor
Johns Hopkins University
Other
Eligibility
18 Years and older
Enrollment
9,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2029
U.S. locations
9
States / cities
New Haven, Connecticut • Baltimore, Maryland • Boston, Massachusetts + 5 more
Source: ClinicalTrials.gov public record
Updated Oct 9, 2025 · Synced Oct 5, 2026, 1:22 PM EDT
Completed No phase listed Observational

Wolman/CESD Natural History Chart Review and Longitudinal Follow-Up

NCT01884220
View directory record Official record
Conditions
Wolman Disease, Cholesterol Ester Storage Disease, Acid Cholesteryl Ester Hydrolase Deficiency, Type 2
Interventions
There are no interventions in this study.
Other
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
Not listed
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2014
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Jul 28, 2015 · Synced Oct 5, 2026, 1:22 PM EDT