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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Terminated Phase 1Phase 2 Interventional Results available

A Gene Therapy Study for Homozygous Familial Hypercholesterolemia (HoFH)

NCT02651675
View directory record Official record
Conditions
Homozygous Familial Hypercholesterolemia (HoFH)
Interventions
AAV directed hLDLR gene therapy
Genetic
Lead sponsor
REGENXBIO Inc.
Industry
Eligibility
18 Years and older
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2020
U.S. locations
5
States / cities
Boca Raton, Florida • Kansas City, Kansas • Portland, Oregon + 2 more
Source: ClinicalTrials.gov public record
Updated Jul 12, 2023 · Synced Jul 21, 2026, 5:11 AM EDT
Not listed Phase 1 Interventional

Open-Label Study of Long-Term Safety and Efficacy of Intravenous Trappsol Cyclo (HPβCD) in Niemann-Pick Disease Type C

NCT03893071
View directory record Official record
Conditions
Niemann-Pick Disease, Type C1
Interventions
Hydroxypropyl-β-cyclodextrin
Drug
Lead sponsor
Cyclo Therapeutics, Inc.
Industry
Eligibility
18 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
1
States / cities
Oakland, California
Source: ClinicalTrials.gov public record
Updated Jun 17, 2021 · Synced Jul 21, 2026, 5:11 AM EDT
Completed Phase 2 Interventional

An Open-Label Clinical Trial of Replagal Enzyme Therapy in Children Ages 7-17 Years With Fabry Disease

NCT00071877
View directory record Official record
Conditions
Fabry Disease
Interventions
Replagal
Drug
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Not listed
Enrollment
25 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 3, 2008 · Synced Jul 21, 2026, 5:11 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Tweens to Teens Project at Penn State

NCT01184651
View directory record Official record
Conditions
Congenital Adrenal Hyperplasia
Interventions
Questionnaires/Interviews
Behavioral
Lead sponsor
Penn State University
Other
Eligibility
10 Years to 13 Years · Female only
Enrollment
188 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2013
U.S. locations
1
States / cities
University Park, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jan 7, 2018 · Synced Jul 21, 2026, 5:11 AM EDT
Not listed No phase listed Observational

Lynch Syndrome Can be Diagnosed Just From Somatic Mismatch Repair Mutation

NCT04516083
View directory record Official record
Conditions
Cancer Gene Mutation, Lynch Syndrome, Endometrial Cancer, Somatic Mutation
Interventions
Mismatch repair instability somatic and germline testing
Diagnostic Test
Lead sponsor
RWJ Barnabas Health at Jersey City Medical Center
Other
Eligibility
Female only
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
1
States / cities
Jersey City, New Jersey
Source: ClinicalTrials.gov public record
Updated Aug 17, 2020 · Synced Jul 21, 2026, 5:11 AM EDT
Recruiting Phase 3 Interventional

Acoramidis Transthyretin Amyloidosis Prevention Trial in the Young (ACT-EARLY) Study in Asymptomatic Carriers of a Pathogenic TTR Variant

NCT06563895
View directory record Official record
Conditions
Amyloidosis, Amyloid Cardiomyopathy, Transthyretin Amyloidosis, Cardiomyopathies, Heart Diseases, Polyneuropathies
Interventions
Acoramidis, Placebo oral tablet
Drug
Lead sponsor
Eidos Therapeutics, a BridgeBio company
Industry
Eligibility
18 Years to 75 Years
Enrollment
587 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2032
U.S. locations
39
States / cities
La Jolla, California • Los Angeles, California • San Francisco, California + 30 more
Source: ClinicalTrials.gov public record
Updated Jun 28, 2026 · Synced Jul 21, 2026, 5:11 AM EDT
Completed Phase 2 Interventional Results available

Dose-Escalation Safety Study of HPN-100 to Treat Urea Cycle Disorders

NCT00551200
View directory record Official record
Conditions
Urea Cycle Disorders
Interventions
HPN-100, BUPHENYL®
Drug
Lead sponsor
Amgen
Industry
Eligibility
18 Years and older
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2008
U.S. locations
2
States / cities
Minneapolis, Minnesota • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jul 9, 2024 · Synced Jul 21, 2026, 5:11 AM EDT
Recruiting No phase listed Observational

A Long-Term Observational Study of Patients With Fucosidosis

NCT07615400
View directory record Official record
Conditions
Fucosidosis
Interventions
Not listed
Lead sponsor
JCR Pharmaceuticals Co., Ltd.
Industry
Eligibility
Not listed
Enrollment
57 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2031
U.S. locations
2
States / cities
Orange, California • Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated May 28, 2026 · Synced Jul 21, 2026, 5:11 AM EDT
Completed Phase 2Phase 3 Interventional Results available

Iduronate-2-sulfatase Enzyme Replacement Therapy in Mucopolysaccharidosis II (MPS II)

NCT00069641
View directory record Official record
Conditions
Mucopolysaccharidosis II
Interventions
iduronate-2-sulfatase enzyme replacement therapy, Placebo
Biological
Lead sponsor
Shire
Industry
Eligibility
5 Years to 25 Years · Male only
Enrollment
96 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2005
U.S. locations
4
States / cities
Oakland, California • St Louis, Missouri • Chapel Hill, North Carolina + 1 more
Source: ClinicalTrials.gov public record
Updated Jun 9, 2021 · Synced Jul 21, 2026, 5:11 AM EDT
Completed Phase 1 Interventional

Stem Cell Gene Therapy to Treat X-Linked Severe Combined Immunodeficiency (XSCID)

NCT00028236
View directory record Official record
Conditions
Severe Combined Immunodeficiency
Interventions
Gene-Transduced Autologous CD34+ Stem Cells
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
18 Months to 20 Years
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2011
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 1, 2017 · Synced Jul 21, 2026, 5:11 AM EDT
Completed No phase listed Observational

Antiproteinuric Agents and Fabry Disease

NCT00343577
View directory record Official record
Conditions
Fabry Disease, Proteinuria
Interventions
Not listed
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
14 Years to 95 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2006
U.S. locations
1
States / cities
Birmingham, Alabama
Source: ClinicalTrials.gov public record
Updated Nov 18, 2013 · Synced Jul 21, 2026, 5:11 AM EDT
Completed Phase 1 Interventional

Re-administration of Intramuscular AAV9 in Patients With Late-Onset Pompe Disease

NCT02240407
View directory record Official record
Conditions
Pompe Disease
Interventions
Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase, Rapamycin, saline, Rituxan, Diphenhydramine, Acetaminophen, Lidocaine, LMX 4 Topical Cream
Genetic · Drug · Other
Lead sponsor
University of Florida
Other
Eligibility
18 Years to 50 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 Apr 4, 2022 · Synced Jul 21, 2026, 5:11 AM EDT
Completed No phase listed Observational

Natural History and Functional Status Study of Patients With Lafora Disease

NCT03876522
View directory record Official record
Conditions
Lafora Disease
Interventions
Not listed
Lead sponsor
Ionis Pharmaceuticals, Inc.
Industry
Eligibility
5 Years and older
Enrollment
33 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
2
States / cities
Los Angeles, California • Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Sep 27, 2022 · Synced Jul 21, 2026, 5:11 AM EDT
Completed Not applicable Interventional Results available

Weight Management for Adolescents With IDD

NCT02561754
View directory record Official record
Conditions
Developmental Disabilities, Weight Loss, Body Weight, Down Syndrome, Body Weight Changes, Intellectual Disability
Interventions
Enhanced Stop Light Diet (eSLD), Conventional Diet (CD), Face-to-face (FTF), Technology (TECH), iPad with FaceTime
Dietary Supplement · Behavioral · Other
Lead sponsor
University of Kansas Medical Center
Other
Eligibility
13 Years to 21 Years
Enrollment
110 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2021
U.S. locations
2
States / cities
Kansas City, Kansas • Lawrence, Kansas
Source: ClinicalTrials.gov public record
Updated Feb 9, 2023 · Synced Jul 21, 2026, 5:11 AM EDT
Completed Phase 3 Interventional Results available

Comparison of Chronocort Versus Standard Hydrocortisone Replacement Therapy in Participants Aged 16 Years and Over With Congenital Adrenal Hyperplasia

NCT05063994
View directory record Official record
Conditions
Congenital Adrenal Hyperplasia
Interventions
Chronocort, Cortef, Placebo
Drug · Other
Lead sponsor
Neurocrine UK Limited
Industry
Eligibility
16 Years and older
Enrollment
55 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
11
States / cities
Los Angeles, California • Orange, California • Jacksonville, Florida + 8 more
Source: ClinicalTrials.gov public record
Updated Feb 24, 2025 · Synced Jul 21, 2026, 5:11 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 Jul 21, 2026, 5:11 AM EDT
Completed Phase 2 Interventional Accepts healthy volunteers Results available

Management of Severe Acute Malnutrition in SCD, in Northern Nigeria

NCT03634488
View directory record Official record
Conditions
Sickle Cell Anemia, Severe Acute Malnutrition
Interventions
hydroxyurea (20mg/kg/day), Ready-to-use therapeutic food
Drug · Dietary Supplement
Lead sponsor
Vanderbilt University Medical Center
Other
Eligibility
5 Years to 12 Years
Enrollment
132 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2022
U.S. locations
1
States / cities
Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Mar 26, 2024 · Synced Jul 21, 2026, 5:11 AM EDT
Completed Phase 2Phase 3 Interventional Results available

Open-Label Study of Asfotase Alfa in Infants and Children ≤ 5 Years of Age With Hypophosphatasia (HPP)

NCT01176266
View directory record Official record
Conditions
Hypophosphatasia
Interventions
asfotase alfa
Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
1 Minute to 5 Years
Enrollment
69 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2016
U.S. locations
3
States / cities
Oakland, California • Cincinnati, Ohio • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 12, 2019 · Synced Jul 21, 2026, 5:11 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Intranasal Oxytocin for Infants With Prader-Willi Syndrome

NCT03245762
View directory record Official record
Conditions
Prader-Willi Syndrome
Interventions
Oxytocin, Placebo
Drug
Lead sponsor
University of Florida
Other
Eligibility
1 Week to 6 Months
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2018
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Mar 25, 2020 · Synced Jul 21, 2026, 5:11 AM EDT
Completed No phase listed Observational

Nutrition Status of Adults Treated With Pegvaliase

NCT03792451
View directory record Official record
Conditions
Phenylketonurias
Interventions
Pegvaliase
Drug
Lead sponsor
Boston Children's Hospital
Other
Eligibility
18 Years to 65 Years
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2020
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Mar 3, 2021 · Synced Jul 21, 2026, 5:11 AM EDT
Terminated Phase 2 Interventional Accepts healthy volunteers Results available

Phenylketonuria, Oxidative Stress, and BH4

NCT01395394
View directory record Official record
Conditions
Phenylketonuria
Interventions
Kuvan, Meal Challenge
Drug · Other
Lead sponsor
Emory University
Other
Eligibility
10 Years to 45 Years
Enrollment
12 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2011 – 2013
U.S. locations
1
States / cities
Decatur, Georgia
Source: ClinicalTrials.gov public record
Updated Aug 27, 2014 · Synced Jul 21, 2026, 5:11 AM EDT
Completed No phase listed Observational

Liver Fibrosis in Sickle Cell Disease

NCT02007746
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Liver transient elastography (Fibroscan), Ferriscan, Liver biopsy in iron overloaded subjects.
Other · Procedure
Lead sponsor
University of Miami
Other
Eligibility
10 Years to 65 Years
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2017
U.S. locations
1
States / cities
Miami, Florida
Source: ClinicalTrials.gov public record
Updated May 25, 2021 · Synced Jul 21, 2026, 5:11 AM EDT
Completed No phase listed Observational

Pilot Study: Urea Cycle Disorders Practice Patterns and Outcomes Assessment

NCT02311283
View directory record Official record
Conditions
Urea Cycle Disorders
Interventions
Not listed
Lead sponsor
University of South Florida
Other
Eligibility
Not listed
Enrollment
87 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2016
U.S. locations
1
States / cities
Tampa, Florida
Source: ClinicalTrials.gov public record
Updated Mar 6, 2018 · Synced Jul 21, 2026, 5:11 AM EDT
Completed Not applicable Interventional

Impact of a Ketogenic Diet on Metabolic and Psychiatric Health in Patients With Bipolar or Schizophrenia Illness

NCT03935854
View directory record Official record
Conditions
Obesity, Ketogenic Dieting, Metabolic Syndrome, Bipolar Disorder, Schizophrenia, Weight Gain, Psychotropic Agents Causing Adverse Effects in Therapeutic Use, Brain Metabolic Disorder
Interventions
LCHF, Ketogenic Diet
Other
Lead sponsor
Stanford University
Other
Eligibility
18 Years to 75 Years
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
1
States / cities
Stanford, California
Source: ClinicalTrials.gov public record
Updated Dec 16, 2024 · Synced Jul 21, 2026, 5:11 AM EDT