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ClinicalTrials.gov public records Last synced Oct 22, 2026, 7:59 PM EDT

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Showing 769–792 of 8,007 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Genetics/Birth Defects Clear all
Completed Phase 1 Interventional

Safety and Tolerability Study of rAvPAL-PEG to Treat Phenylketonuria

NCT00634660
View directory record Official record
Conditions
Phenylketonuria
Interventions
rAvPAL-PEG
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
16 Years to 50 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2009
U.S. locations
8
States / cities
Chicago, Illinois • Minneapolis, Minnesota • St Louis, Missouri + 5 more
Source: ClinicalTrials.gov public record
Updated Feb 1, 2017 · Synced Oct 22, 2026, 7:59 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers Results available

Feasibility Study: Heavy Water Tissue Labeling Protocol

NCT01708525
View directory record Official record
Conditions
Skin Laxity
Interventions
Ulthera® System Treatment, Heavy Water
Device · Other
Lead sponsor
Ulthera, Inc
Industry
Eligibility
30 Years to 65 Years
Enrollment
2 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2013
U.S. locations
1
States / cities
Pasadena, California
Source: ClinicalTrials.gov public record
Updated Dec 11, 2017 · Synced Oct 22, 2026, 7:59 PM EDT
Terminated No phase listed Observational

Natural History Study of Children With Metachromatic Leukodystrophy

NCT01963650
View directory record Official record
Conditions
Lipid Metabolism Disorders, Metachromatic Leukodystrophy (MLD), Nervous System Diseases, Brain Diseases, Central Nervous System Diseases, Demyelinating Diseases, Metabolism, Inborn Errors, Genetic Diseases, Inborn, Sphingolipidoses, Hereditary Central Nervous System Demyelinating Diseases, Metabolic Inborn Brain Diseases, Lysosomal Storage Diseases, Metabolic Diseases, Sulfatidosis
Interventions
Not listed
Lead sponsor
Shire
Industry
Eligibility
Up to 12 Years
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2016
U.S. locations
4
States / cities
Torrance, California • Washington D.C., District of Columbia • Chicago, Illinois + 1 more
Source: ClinicalTrials.gov public record
Updated Mar 17, 2021 · Synced Oct 22, 2026, 7:59 PM EDT
Not listed No phase listed Observational

Women At Risk: The High Risk Breast Cancer Program

NCT01034891
View directory record Official record
Conditions
Breast Cancer
Interventions
Not listed
Lead sponsor
New York Presbyterian Hospital
Other
Eligibility
18 Years and older · Female only
Enrollment
2,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1991
U.S. locations
2
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Dec 18, 2009 · Synced Oct 22, 2026, 7:59 PM EDT
Not listed Not applicable Interventional

Clinical Characteristics and Temporal Properties of Individual Tics in Persistent Tic Disorder

NCT06385535
View directory record Official record
Conditions
Tics, Tic Disorders, Vocal Tic, Motor Tic, Tourette Syndrome, Tourette Syndrome in Children, Tourette Syndrome in Adolescence, Tics/Tremor
Interventions
Habit Reversal Training (HRT)
Behavioral
Lead sponsor
University of Utah
Other
Eligibility
8 Years to 12 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024
U.S. locations
1
States / cities
Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated Apr 26, 2024 · Synced Oct 22, 2026, 7:59 PM EDT
Completed Not applicable Interventional

The Safety and Effectiveness of Ganciclovir Used Alone or in Combination With Granulocyte-Macrophage Colony Stimulating Factor in the Treatment of Cytomegalovirus (CMV) of the Eye in Patients With AIDS

NCT00000989
View directory record Official record
Conditions
Cytomegalovirus Retinitis, HIV Infections
Interventions
Zidovudine, Sargramostim, Ganciclovir
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
13 Years to 65 Years
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Ends 1992
U.S. locations
4
States / cities
Los Angeles, California • Boston, Massachusetts • New York, New York + 1 more
Source: ClinicalTrials.gov public record
Updated Nov 4, 2021 · Synced Oct 22, 2026, 7:59 PM EDT
Completed No phase listed Observational

Double Push Acoustic Radiation Force (DP ARF) Ultrasound for Monitoring Degeneration in Duchenne Muscular Dystrophy

NCT01506518
View directory record Official record
Conditions
Muscular Dystrophy, Duchenne
Interventions
Not listed
Lead sponsor
University of North Carolina, Chapel Hill
Other
Eligibility
5 Years to 13 Years · Male only
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2020
U.S. locations
1
States / cities
Chapel Hill, North Carolina
Source: ClinicalTrials.gov public record
Updated Aug 2, 2021 · Synced Oct 22, 2026, 7:59 PM EDT
Not listed No phase listed Observational Accepts healthy volunteers

Amyloid Plaque and Tangle Imaging in Alzheimer's Disease and Down Syndrome

NCT00965835
View directory record Official record
Conditions
Down Syndrome, Alzheimer's Disease
Interventions
Not listed
Lead sponsor
National Institute on Aging (NIA)
NIH
Eligibility
21 Years and older
Enrollment
40 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2011
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Dec 29, 2009 · Synced Oct 22, 2026, 7:59 PM EDT
Recruiting No phase listed Observational

Mucopolysaccharidosis VII Disease Monitoring Program

NCT03604835
View directory record Official record
Conditions
Mucopolysaccharidosis VII, MPS VII, MPS 7, Sly Syndrome
Interventions
No Intervention
Other
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
Not listed
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2032
U.S. locations
7
States / cities
Orange, California • Washington D.C., District of Columbia • Chicago, Illinois + 4 more
Source: ClinicalTrials.gov public record
Updated Aug 27, 2026 · Synced Oct 22, 2026, 7:59 PM EDT
Completed No phase listed Observational

Clinical Outcomes of Triple Combination Therapy in Severe Cystic Fibrosis Disease.

NCT04038710
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Triple combination therapy
Drug
Lead sponsor
National Jewish Health
Other
Eligibility
12 Years and older
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
1
States / cities
Denver, Colorado
Source: ClinicalTrials.gov public record
Updated Mar 11, 2021 · Synced Oct 22, 2026, 7:59 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers

A Test to Predict the Hemolytic Potential of Drugs in G6PD Deficiency

NCT00076323
View directory record Official record
Conditions
Glucosephosphate Dehydrogenase Deficiency, Favism
Interventions
Primaquine
Drug
Lead sponsor
Walter Reed Army Institute of Research (WRAIR)
Federal
Eligibility
18 Years to 60 Years
Enrollment
14 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2003 – 2006
U.S. locations
1
States / cities
Silver Spring, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 27, 2010 · Synced Oct 22, 2026, 7:59 PM EDT
Completed Phase 1Phase 2 Interventional Results available

NORTHERA (DROXIDOPA) for Dysautonomia in Adult Survivors of Menkes Disease and Occipital Horn Syndrome

NCT04977388
View directory record Official record
Conditions
Menkes Disease, Occipital Horn Syndrome
Interventions
Droxidopa, Placebo
Drug · Other
Lead sponsor
Stephen G. Kaler, MD
Other
Eligibility
18 Years to 50 Years
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Apr 13, 2026 · Synced Oct 22, 2026, 7:59 PM EDT
Completed Phase 1 Interventional

Sickle Cell Pain: Intervention With Capsaicin Exposure

NCT03899246
View directory record Official record
Conditions
Neuropathic Pain, Sickle Cell Disease
Interventions
Eight Percent Topical Capsaicin
Drug
Lead sponsor
Children's Hospital of Michigan
Other
Eligibility
14 Years to 21 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2020
U.S. locations
1
States / cities
Detroit, Michigan
Source: ClinicalTrials.gov public record
Updated Jul 29, 2020 · Synced Oct 22, 2026, 7:59 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers Results available

PeLear CCC: Proyecto Latino Contra Cancer Colorrectal

NCT06426927
View directory record Official record
Conditions
Colorectal Cancer, Rectal Cancer, Colon Cancer, Colon Rectal Cancer, Rectal Neoplasms, Colon Adenocarcinoma, Hereditary Nonpolyposis Colon Cancer
Interventions
Colorectal Cancer Educational Videos in Spanish
Other
Lead sponsor
University of North Carolina, Chapel Hill
Other
Eligibility
18 Years and older
Enrollment
60 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2024 – 2025
U.S. locations
1
States / cities
Chapel Hill, North Carolina
Source: ClinicalTrials.gov public record
Updated Jan 21, 2026 · Synced Oct 22, 2026, 7:59 PM EDT
Completed No phase listed Observational

VCRC Patient Contact Registry Patient-Reported Data Validation Study

NCT02190942
View directory record Official record
Conditions
Behcet's Disease, Churg-Strauss Syndrome, Giant Cell Arteritis, Wegener Granulomatosis, Microscopic Polyangiitis, Polyarteritis Nodosa, Takayasu's Arteritis
Interventions
Not listed
Lead sponsor
University of South Florida
Other
Eligibility
Not listed
Enrollment
198 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2019
U.S. locations
1
States / cities
Tampa, Florida
Source: ClinicalTrials.gov public record
Updated Oct 16, 2019 · Synced Oct 22, 2026, 7:59 PM EDT
Completed Phase 1 Interventional

AAV2/8-LSPhGAA (ACTUS-101) in Late-Onset Pompe Disease

NCT03533673
View directory record Official record
Conditions
Pompe Disease
Interventions
ACTUS-101
Genetic
Lead sponsor
AskBio Inc
Industry
Eligibility
18 Years and older
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2026
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Apr 13, 2026 · Synced Oct 22, 2026, 7:59 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A Phase 1, Non-Randomized, Open-Label, Single-Dose Study To Evaluate The Pharmacokinetics, Safety, And Tolerability Of Dimebon [PF 01913539] In Subjects With Severely-Impaired And Normal Renal Function

NCT00824590
View directory record Official record
Conditions
Alzheimer's Disease, Huntington's Disease
Interventions
Dimebon
Drug
Lead sponsor
Pfizer
Industry
Eligibility
18 Years to 75 Years
Enrollment
20 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009
U.S. locations
2
States / cities
Miami, Florida • Saint Paul, Minnesota
Source: ClinicalTrials.gov public record
Updated Dec 30, 2009 · Synced Oct 22, 2026, 7:59 PM EDT
Recruiting No phase listed Observational

International Registry of Patients With Alpha Thalassemia

NCT04872179
View directory record Official record
Conditions
Alpha-Thalassemia, Alpha Thalassemia Major, Alpha Thalassemia Minor
Interventions
Not listed
Lead sponsor
University of California, San Francisco
Other
Eligibility
Not listed
Enrollment
500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2037
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Sep 2, 2026 · Synced Oct 22, 2026, 7:59 PM EDT
Completed Phase 2 Interventional

Study of High-Dose Melphalan and Autologous Stem Cell Transplantation in Patients With Primary Light Chain Amyloidosis

NCT00017680
View directory record Official record
Conditions
Amyloidosis
Interventions
Melphalan, Autologous Stem Cell Transplantation
Drug · Procedure
Lead sponsor
Herbert Irving Comprehensive Cancer Center
Other
Eligibility
18 Years to 70 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1999 – 2004
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jun 9, 2008 · Synced Oct 22, 2026, 7:59 PM EDT
Active, not recruiting No phase listed Observational

Defining Endpoints in Becker Muscular Dystrophy

NCT05257473
View directory record Official record
Conditions
Becker Muscular Dystrophy, Muscular Dystrophies, Muscular Dystrophy in Children, Muscular Dystrophy, Becker
Interventions
Not listed
Lead sponsor
Virginia Commonwealth University
Other
Eligibility
6 Years and older · Male only
Enrollment
80 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
9
States / cities
Orange, California • Aurora, Colorado • Iowa City, Iowa + 6 more
Source: ClinicalTrials.gov public record
Updated Sep 3, 2026 · Synced Oct 22, 2026, 7:59 PM EDT
Completed Early Phase 1 Interventional

Pilot Study Evaluating Sulforaphane in Atypical Nevi-Precursor Lesions

NCT01568996
View directory record Official record
Conditions
Atypical Nevi, Melanoma
Interventions
broccoli sprout extract - sulforaphane (BSE-SFN)
Drug
Lead sponsor
John Kirkwood
Other
Eligibility
18 Years and older
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2016
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jan 9, 2018 · Synced Oct 22, 2026, 7:59 PM EDT
Completed Phase 3 Interventional

Phase 3 Study of Immune Globulin Intravenous (Human)IVIG-SN™ in Subjects With Primary Immunodeficiency

NCT01406470
View directory record Official record
Conditions
Immunologic Deficiency Syndrome
Interventions
Immune Globulin Intravenous (Human) 5% Liquid, IVIG-SN™
Drug
Lead sponsor
Green Cross Corporation
Industry
Eligibility
2 Years to 70 Years
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2013
U.S. locations
9
States / cities
Birmingham, Alabama • North Palm Beach, Florida • Chicago, Illinois + 5 more
Source: ClinicalTrials.gov public record
Updated Jan 10, 2014 · Synced Oct 22, 2026, 7:59 PM EDT
Completed Phase 2 Interventional

DMT210 Topical Gel in the Treatment of Atopic Dermatitis

NCT02949960
View directory record Official record
Conditions
Atopic Dermatitis
Interventions
DMT210 Topical Gel, Vehicle Control
Drug
Lead sponsor
Dermata Therapeutics
Industry
Eligibility
12 Years and older
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2017
U.S. locations
2
States / cities
Houston, Texas • Norfolk, Virginia
Source: ClinicalTrials.gov public record
Updated Apr 18, 2018 · Synced Oct 22, 2026, 7:59 PM EDT
Completed Phase 2 Interventional Results available

Study of Idebenone in the Treatment of Mitochondrial Encephalopathy Lactic Acidosis & Stroke-like Episodes

NCT00887562
View directory record Official record
Conditions
MELAS Syndrome
Interventions
Idebenone, Placebo
Drug · Other
Lead sponsor
Michio Hirano
Other
Eligibility
8 Years to 65 Years
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2012
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Oct 26, 2016 · Synced Oct 22, 2026, 7:59 PM EDT