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ClinicalTrials.gov public records Last synced Oct 14, 2026, 5:31 PM EDT

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Showing 409–432 of 9,407 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Genetic Disease Clear all
Completed Phase 1 Interventional Accepts healthy volunteers

Dopaminergic Effects on Cortical Function in Tourette's (Levodopa Protocol)

NCT00634556
View directory record Official record
Conditions
Tourette Syndrome
Interventions
levodopa solution 2mg/ml for i.v. use, placebo
Drug
Lead sponsor
Washington University School of Medicine
Other
Eligibility
18 Years to 55 Years
Enrollment
49 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2006 – 2010
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Feb 12, 2018 · Synced Oct 14, 2026, 5:31 PM EDT
Terminated Phase 3 Interventional Results available

A Study Evaluating the Long Term Safety and Efficacy of VX-659 Combination Therapy

NCT03447262
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
VX-659/TEZ/IVA, IVA
Drug
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
12 Years and older
Enrollment
484 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2020
U.S. locations
48
States / cities
Birmingham, Alabama • Palo Alto, California • Aurora, Colorado + 44 more
Source: ClinicalTrials.gov public record
Updated Jan 25, 2022 · Synced Oct 14, 2026, 5:31 PM EDT
Completed Phase 1 Interventional

A Study of Ganciclovir in the Treatment of Cytomegalovirus of the Eyes

NCT00001062
View directory record Official record
Conditions
Cytomegalovirus Retinitis, HIV Infections
Interventions
Ganciclovir
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
13 Years and older
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Ends 1998
U.S. locations
6
States / cities
Los Angeles, California • Chicago, Illinois • Baltimore, Maryland + 3 more
Source: ClinicalTrials.gov public record
Updated Nov 4, 2021 · Synced Oct 14, 2026, 5:31 PM EDT
Completed Phase 2 Interventional

High-Dose Thiotepa Plus Peripheral Stem Cell Transplantation in Treating Patients With Refractory Solid Tumors

NCT00003173
View directory record Official record
Conditions
Brain and Central Nervous System Tumors, Childhood Germ Cell Tumor, Extragonadal Germ Cell Tumor, Ovarian Cancer, Retinoblastoma, Testicular Germ Cell Tumor, Unspecified Adult Solid Tumor, Protocol Specific, Unspecified Childhood Solid Tumor, Protocol Specific
Interventions
filgrastim, thiotepa, autologous bone marrow transplantation, peripheral blood stem cell transplantation
Biological · Drug · Procedure
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
Not listed
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1997 – 2003
U.S. locations
2
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Mar 7, 2013 · Synced Oct 14, 2026, 5:31 PM EDT
Completed Phase 3 Interventional Results available

CLINICAL PHASE III STUDY TO MONITOR THE SAFETY, TOLERABILITY AND EFFICACY OF SUBCUTANEOUS HUMAN IMMUNOGLOBULIN (OCTANORM) IN PATIENTS WITH PRIMARY IMMUNODEFICIENCY DISEASES, INCLUDING (BUT NOT LIMITED TO) THOSE WHO HAVE COMPLETED THE SCGAM-01 TRIAL

NCT03907241
View directory record Official record
Conditions
Primary Immunodeficiency
Interventions
Octanorm 16.5%
Drug
Lead sponsor
Octapharma
Industry
Eligibility
2 Years to 75 Years
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
6
States / cities
Irvine, California • San Diego, California • Centennial, Colorado + 3 more
Source: ClinicalTrials.gov public record
Updated Oct 27, 2020 · Synced Oct 14, 2026, 5:31 PM EDT
Enrolling by invitation Not applicable Interventional

AI Echocardiographic Screening of Cardiac Amyloidosis

NCT06664866
View directory record Official record
Conditions
Cardiac Amyloidosis
Interventions
EchoNet-LVH Assessment
Diagnostic Test
Lead sponsor
Cedars-Sinai Medical Center
Other
Eligibility
22 Years and older
Enrollment
500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
4
States / cities
Los Angeles, California • Palo Alto, California • Chicago, Illinois + 1 more
Source: ClinicalTrials.gov public record
Updated Jul 22, 2026 · Synced Oct 14, 2026, 5:31 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Magnetic Stimulation of the Human Nervous System

NCT00001780
View directory record Official record
Conditions
Demyelinating Disease, Healthy, Lysosomal Storage Disease, Motor Neuron Disease, Movement Disorder
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Not listed
Enrollment
450 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
1998 – 2005
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 14, 2026, 5:31 PM EDT
Completed Phase 2 Interventional Results available

Acamprosate in Youth With Fragile X Syndrome

NCT01300923
View directory record Official record
Conditions
Fragile X Syndrome, Autism Spectrum Disorders
Interventions
Acamprosate
Drug
Lead sponsor
Indiana University
Other
Eligibility
5 Years to 17 Years
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2011
U.S. locations
1
States / cities
Indianapolis, Indiana
Source: ClinicalTrials.gov public record
Updated Jul 30, 2019 · Synced Oct 14, 2026, 5:31 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 14, 2026, 5:31 PM EDT
Terminated Phase 1 Interventional Accepts healthy volunteers

Comparison of Nikkomycin Z Bioavailability Under Fed and Fasting Conditions

NCT01647256
View directory record Official record
Conditions
Healthy
Interventions
Nikkomycin Z
Drug
Lead sponsor
University of Arizona
Other
Eligibility
18 Years to 40 Years
Enrollment
5 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2013
U.S. locations
1
States / cities
Tucson, Arizona
Source: ClinicalTrials.gov public record
Updated Sep 17, 2013 · Synced Oct 14, 2026, 5:31 PM EDT
Completed Phase 4 Interventional Results available

Infusion Duration Study To Assess Tolerability of Pegloticase Administered With a Shorter Infusion Duration in Subjects With Uncontrolled Gout Receiving Methotrexate

NCT04511702
View directory record Official record
Conditions
Chronic Uncontrolled Gout, Gout, Uncontrolled Gout
Interventions
Pegloticase with MTX
Biological
Lead sponsor
Amgen
Industry
Eligibility
18 Years and older
Enrollment
191 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2024
U.S. locations
39
States / cities
Birmingham, Alabama • Anchorage, Alaska • Gilbert, Arizona + 33 more
Source: ClinicalTrials.gov public record
Updated Oct 20, 2025 · Synced Oct 14, 2026, 5:31 PM EDT
Active, not recruiting Phase 2 Interventional

A Trial to Investigate Different Doses of Lonapegsomatropin Compared to Somatropin in Individuals With Turner Syndrome

NCT05690386
View directory record Official record
Conditions
Turner Syndrome
Interventions
Lonapegsomatropin, Somatropin
Biological · Drug
Lead sponsor
Ascendis Pharma Endocrinology Division A/S
Industry
Eligibility
1 Year to 10 Years · Female only
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2027
U.S. locations
19
States / cities
Palo Alto, California • San Diego, California • Aurora, Colorado + 16 more
Source: ClinicalTrials.gov public record
Updated May 13, 2026 · Synced Oct 14, 2026, 5:31 PM EDT
Completed Phase 2Phase 3 Interventional Results available

Arimoclomol Prospective Study in Participants Diagnosed With Niemann-Pick Disease Type C

NCT02612129
View directory record Official record
Conditions
Niemann-Pick Disease, Type C
Interventions
arimoclomol, Placebo
Drug
Lead sponsor
ZevraDenmark
Industry
Eligibility
2 Years to 18 Years
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2024
U.S. locations
2
States / cities
Oakland, California • Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Nov 29, 2024 · Synced Oct 14, 2026, 5:31 PM EDT
Recruiting Phase 4 Interventional

Dupixent and Narrowband UVB for Atopic Dermatitis

NCT05285839
View directory record Official record
Conditions
Atopic Dermatitis
Interventions
Dupixent
Drug
Lead sponsor
Psoriasis Treatment Center of Central New Jersey
Other
Eligibility
18 Years and older
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
1
States / cities
East Windsor, New Jersey
Source: ClinicalTrials.gov public record
Updated Mar 15, 2024 · Synced Oct 14, 2026, 5:31 PM EDT
Terminated Phase 1 Interventional

Alternating Systemic Chemotherapy and Intra-Arterial Melphalan (IAM) Chemotherapy in Children With Intra-Ocular Retinoblastoma

NCT02116959
View directory record Official record
Conditions
Advanced Intra-Ocular Retinoblastoma, Retinoblastoma
Interventions
Melphalan, Carboplatin, Etoposide, Vincristine
Drug
Lead sponsor
University of California, San Francisco
Other
Eligibility
3 Months to 18 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2019
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Apr 16, 2020 · Synced Oct 14, 2026, 5:31 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Cerebral Autosomal Dominant Arteriopathy With Subcortical Infarcts and Leukoencephalopathy (CADASIL) Study

NCT05677880
View directory record Official record
Conditions
CADASIL
Interventions
Study Procedures
Other
Lead sponsor
University of Wisconsin, Madison
Other
Eligibility
18 Years and older
Enrollment
660 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2027
U.S. locations
12
States / cities
Los Angeles, California • San Francisco, California • Denver, Colorado + 9 more
Source: ClinicalTrials.gov public record
Updated Jan 15, 2026 · Synced Oct 14, 2026, 5:31 PM EDT
Terminated Phase 2 Interventional Results available

Phase 2B Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)

NCT00847379
View directory record Official record
Conditions
Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
Interventions
Ataluren
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
5 Years and older · Male only
Enrollment
173 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2010
U.S. locations
15
States / cities
Sacramento, California • Aurora, Colorado • Gulf Breeze, Florida + 12 more
Source: ClinicalTrials.gov public record
Updated Jul 15, 2020 · Synced Oct 14, 2026, 5:31 PM EDT
Recruiting Not applicable Interventional Accepts healthy volunteers

New Non-invasive Modalities for Assessing Retinal Structure and Function

NCT03475173
View directory record Official record
Conditions
Ischemic Optic Neuropathy, Branch Retinal Artery Occlusion, Hemianopia, Leber Hereditary Optic Neuropathy, Acute Zonal Occult Outer Retinopathy
Interventions
LSFG-NAVI
Device
Lead sponsor
Randy Kardon
Other
Eligibility
18 Years to 99 Years
Enrollment
500 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2028
U.S. locations
1
States / cities
Iowa City, Iowa
Source: ClinicalTrials.gov public record
Updated Sep 8, 2026 · Synced Oct 14, 2026, 5:31 PM EDT
Completed Phase 2 Interventional Results available

OPTION: A Trial to Assess the Safety & Efficacy of MS1819 in Patients With Exocrine Pancreatic Insufficiency Due to Cystic Fibrosis

NCT03746483
View directory record Official record
Conditions
Exocrine Pancreatic Insufficiency (EPI), Cystic Fibrosis (CF)
Interventions
MS1819, Porcine PERT
Drug
Lead sponsor
Entero Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
41 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019
U.S. locations
11
States / cities
Long Beach, California • Altamonte Springs, Florida • Miami, Florida + 8 more
Source: ClinicalTrials.gov public record
Updated Jun 6, 2022 · Synced Oct 14, 2026, 5:31 PM EDT
Active, not recruiting Phase 4 Interventional

Effects of the Contraceptive Implant in Women With Sickle Cell Disease

NCT05730205
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Nexplanon 68 MG Drug Implant
Drug
Lead sponsor
University of Pennsylvania
Other
Eligibility
18 Years to 45 Years · Female only
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2027
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jun 12, 2026 · Synced Oct 14, 2026, 5:31 PM EDT
Active, not recruiting No phase listed Observational

Analysis of Specimens From Individuals With Pulmonary Fibrosis

NCT00084305
View directory record Official record
Conditions
Pulmonary Fibrosis, Healthy Volunteers, Hermansky-Pudlak Syndrome (HPS)
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
18 Years to 115 Years
Enrollment
315 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2004
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 13, 2026 · Synced Oct 14, 2026, 5:31 PM EDT
Terminated Phase 2 Interventional Results available

A 4-week Study to Test Different Doses of BI 1265162 in Adolescents and Adults With Cystic Fibrosis Using the Respimat® Inhaler - BALANCE - CF™1

NCT04059094
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
BI 1265162, Placebo
Drug
Lead sponsor
Boehringer Ingelheim
Industry
Eligibility
12 Years and older
Enrollment
52 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2020
U.S. locations
8
States / cities
Orlando, Florida • Indianapolis, Indiana • Chapel Hill, North Carolina + 5 more
Source: ClinicalTrials.gov public record
Updated Jun 4, 2021 · Synced Oct 14, 2026, 5:31 PM EDT
Completed Not applicable Interventional

Assessment of Patient Experience With Auto-Captioning Glasses in NF2-Related-Schwannomatosis

NCT07420751
View directory record Official record
Conditions
NF2-related Schwannomatosis
Interventions
Auto-captioning Glasses
Device
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
18 Years and older
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Aug 25, 2026 · Synced Oct 14, 2026, 5:31 PM EDT
Completed Phase 2 Interventional

Study of SAR447537 (INBRX-101) Compared to Plasma-derived A1PI Therapy in Adults With AATD Emphysema

NCT05856331
View directory record Official record
Conditions
Alpha 1-Antitrypsin Deficiency, Emphysema
Interventions
SAR447537, Zemaira
Drug
Lead sponsor
Sanofi
Industry
Eligibility
18 Years to 80 Years
Enrollment
99 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
39
States / cities
Birmingham, Alabama • Phoenix, Arizona • Los Angeles, California + 18 more
Source: ClinicalTrials.gov public record
Updated Aug 11, 2025 · Synced Oct 14, 2026, 5:31 PM EDT