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ClinicalTrials.gov public records Last synced Oct 15, 2026, 11:34 PM EDT

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Showing 697–720 of 9,409 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Genetic Disease Clear all
Completed No phase listed Observational

Registry to Monitor the Susceptibility to Aztreonam of Pseudomonas Aeruginosa Isolates From Cystic Fibrosis Patients

NCT01375036
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Not listed
Lead sponsor
Gilead Sciences
Industry
Eligibility
6 Years and older
Enrollment
510 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2016
U.S. locations
31
States / cities
Anchorage, Alaska • Tucson, Arizona • Little Rock, Arkansas + 20 more
Source: ClinicalTrials.gov public record
Updated Jan 11, 2017 · Synced Oct 15, 2026, 11:34 PM EDT
Completed Not applicable Interventional

Assessment of Bone Density and Bone Turnover Markers in Patients With Down Syndrome and Comparison to the Ts65Dn Model

NCT01148121
View directory record Official record
Conditions
Down Syndrome
Interventions
Skeletal Status Assessment of a Down Syndrome Population
Other
Lead sponsor
University of Arkansas
Other
Eligibility
18 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2015
U.S. locations
1
States / cities
Little Rock, Arkansas
Source: ClinicalTrials.gov public record
Updated Dec 7, 2016 · Synced Oct 15, 2026, 11:34 PM EDT
Completed Phase 2 Interventional Results available

Rilonacept for Treatment of Familial Mediterranean Fever (FMF)

NCT00582907
View directory record Official record
Conditions
Familial Mediterranean Fever
Interventions
Rilonacept, Placebo
Drug
Lead sponsor
The Cleveland Clinic
Other
Eligibility
4 Years and older
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2011
U.S. locations
5
States / cities
Los Angeles, California • Madera, California • Bethesda, Maryland + 2 more
Source: ClinicalTrials.gov public record
Updated Feb 11, 2013 · Synced Oct 15, 2026, 11:34 PM EDT
Completed Phase 3 Interventional

Study Evaluating rFIX; BeneFIX® in Hemophilia B

NCT00093171
View directory record Official record
Conditions
Hemophilia B
Interventions
rFIX
Drug
Lead sponsor
Wyeth is now a wholly owned subsidiary of Pfizer
Industry
Eligibility
12 Years and older
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Ends 2005
U.S. locations
7
States / cities
Los Angeles, California • Aurora, Colorado • Detroit, Michigan + 4 more
Source: ClinicalTrials.gov public record
Updated Aug 21, 2009 · Synced Oct 15, 2026, 11:34 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Improving Quality by Maintaining Accurate Problems in the EHR

NCT02596087
View directory record Official record
Conditions
Asthma, Atrial Fibrillation, Chronic Obstructive Pulmonary Disease, Coronary Artery Disease, Congestive Heart Failure, Hyperlipidemia, Hypertension, Myocardial Infarction, Sickle Cell Disease, Sleep Apnea, Smoking, Stroke, Tuberculosis
Interventions
Problem List Suggestion
Other
Lead sponsor
Brigham and Women's Hospital
Other
Eligibility
18 Years and older
Enrollment
2,386 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 2016
U.S. locations
4
States / cities
Boston, Massachusetts • Portland, Oregon • Camp Hill, Pennsylvania + 1 more
Source: ClinicalTrials.gov public record
Updated Feb 8, 2023 · Synced Oct 15, 2026, 11:34 PM EDT
Recruiting Not applicable Interventional Accepts healthy volunteers

Bundled Cancer Screening and Genetic Services Navigation

NCT07228000
View directory record Official record
Conditions
Breast Cancer Screening, Colon Cancer Screening, Genetics Predisposition
Interventions
Breast cancer screening navigation, Multicancer screening navigation, Usual care genetics referral, Pretest education + usual care genetics referral
Behavioral
Lead sponsor
Georgetown University
Other
Eligibility
45 Years to 74 Years · Female only
Enrollment
820 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2026 – 2029
U.S. locations
2
States / cities
Newark, Delaware • Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated May 8, 2026 · Synced Oct 15, 2026, 11:34 PM EDT
Completed Phase 3 Interventional Results available

A Safety and Efficacy Extension Study of a Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Patients With Hemophilia B

NCT02053792
View directory record Official record
Conditions
Hemophilia B
Interventions
rIX-FP
Biological
Lead sponsor
CSL Behring
Industry
Eligibility
Up to 70 Years · Male only
Enrollment
97 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2021
U.S. locations
4
States / cities
Aurora, Colorado • Indianapolis, Indiana • Philadelphia, Pennsylvania + 1 more
Source: ClinicalTrials.gov public record
Updated Jul 14, 2022 · Synced Oct 15, 2026, 11:34 PM EDT
Not listed Phase 2 Interventional

Efficacy and Safety of Pirfenidone Treatment in HPS-ILD

NCT04193592
View directory record Official record
Conditions
Hermansky Pudlak Syndrome, Interstitial Lung Disease
Interventions
Pirfenidone
Drug
Lead sponsor
Jesse Roman
Other
Eligibility
18 Years to 75 Years
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Dec 10, 2019 · Synced Oct 15, 2026, 11:34 PM EDT
Completed Phase 3 Interventional

Study to Evaluate Efficacy and Safety of PF-04965842 With or Without Topical Medications in Subjects Aged 12 Years and Older With Moderate to Severe Atopic Dermatitis

NCT03422822
View directory record Official record
Conditions
Dermatitis, Atopic
Interventions
Abrocitinib 100 mg, Abrocitinib 200 mg, Placebo
Drug
Lead sponsor
Pfizer
Industry
Eligibility
12 Years and older
Enrollment
3,166 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2025
U.S. locations
156
States / cities
Birmingham, Alabama • Anaheim, California • Encinitas, California + 101 more
Source: ClinicalTrials.gov public record
Updated Jan 15, 2026 · Synced Oct 15, 2026, 11:34 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Pilot Study of Safety, Tolerability, Pharmacokinetics/Pharmacodynamics of RP103 Compared to Cystagon® in Patients With Cystinosis

NCT00872729
View directory record Official record
Conditions
Cystinosis
Interventions
Cystagon®, RP103
Drug
Lead sponsor
Amgen
Industry
Eligibility
Not listed
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009
U.S. locations
1
States / cities
San Diego, California
Source: ClinicalTrials.gov public record
Updated Dec 19, 2024 · Synced Oct 15, 2026, 11:34 PM EDT
Completed Not applicable Interventional

Prospective Randomized Trial of Streamlined Genetic Education and Testing for Ovarian Cancer Patients

NCT04537702
View directory record Official record
Conditions
Ovarian Cancer, Epithelial, Peritoneal Cancer, Fallopian Tube Cancer
Interventions
Streamlined Education, Traditional Education
Behavioral
Lead sponsor
Duke University
Other
Eligibility
18 Years and older · Female only
Enrollment
64 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Aug 26, 2021 · Synced Oct 15, 2026, 11:34 PM EDT
Recruiting No phase listed Observational

Study of Selected X-Linked Disorders: Aicardi Syndrome

NCT00697411
View directory record Official record
Conditions
Aicardi Syndrome, Brain Disorders
Interventions
Syndrome cause identification
Other
Lead sponsor
Baylor College of Medicine
Other
Eligibility
Not listed
Enrollment
500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2030
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated May 5, 2026 · Synced Oct 15, 2026, 11:34 PM EDT
Completed Phase 3 Interventional Results available

A Placebo-Controlled Study of Safety and Effectiveness of Myozyme (Alglucosidase Alfa) in Patients With Late-Onset Pompe Disease

NCT00158600
View directory record Official record
Conditions
Pompe Disease (Late-onset), Glycogen Storage Disease Type II (GSD-II), Acid Maltase Deficiency Disease, Glycogenosis 2
Interventions
alglucosidase alfa, Placebo
Biological · Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
8 Years and older
Enrollment
90 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2007
U.S. locations
5
States / cities
Beverly Hills, California • Washington D.C., District of Columbia • St Louis, Missouri + 2 more
Source: ClinicalTrials.gov public record
Updated Apr 28, 2015 · Synced Oct 15, 2026, 11:34 PM EDT
Completed Phase 2 Interventional

Efficacy and Safety of MCO-010 Optogenetic Therapy in Adults With Retinitis Pigmentosa [RESTORE]

NCT04945772
View directory record Official record
Conditions
Retinitis Pigmentosa, Retinitis, Retinal Diseases, Eye Diseases, Eye Diseases, Hereditary, Retinal Dystrophies, Retinal Degeneration
Interventions
Gene Therapy Product-MCO-010, Sham Injection
Biological · Procedure
Lead sponsor
Nanoscope Therapeutics Inc.
Industry
Eligibility
18 Years and older
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
5
States / cities
Beverly Hills, California • Pensacola, Florida • Fargo, North Dakota + 2 more
Source: ClinicalTrials.gov public record
Updated Mar 22, 2024 · Synced Oct 15, 2026, 11:34 PM EDT
Recruiting No phase listed Observational

Natural History Study for Charcot Marie Tooth Disease

NCT05902351
View directory record Official record
Conditions
Charcot-Marie-Tooth Disease, Charcot-Marie-Tooth, Charcot-Marie-Tooth Disease, Type IA, Charcot-Marie-Tooth Disease Type 2A, Charcot-Marie-Tooth Disease Type 2, Charcot-Marie-Tooth Disease, Type 2C, Charcot-Marie-Tooth Disease Type 2A2B, Charcot-Marie-Tooth Disease Type 2B2, Charcot-Marie-Tooth Disease Type 2A1, Charcot-Marie-Tooth Disease Type 4B1, Charcot-Marie-Tooth Disease, Type IB, Charcot-Marie-Tooth Disease Type 2B1, Charcot-Marie-Tooth Disease Type 2U (Diagnosis), Charcot-Marie-Tooth Disease Type 4A, Charcot-Marie-Tooth Disease, Type 4A, Axonal Form, Charcot-Marie-Tooth Disease Type 2A2A, Charcot-Marie-Tooth Disease Type 2S (Disorder), Charcot-Marie-Tooth Disease and Deafness, Charcot-Marie-Tooth Disease Type 4B2, Charcot-Marie-Tooth Disease Type 4H, Charcot-Marie-Tooth Disease Type 1F, Charcot-Marie-Tooth Disease Type 4C, Charcot-Marie-Tooth Disease Type 4E, Charcot-Marie-Tooth Disease Type 1D, Charcot-Marie-Tooth Disease Type 2Q (Diagnosis), Charcot-Marie-Tooth Disease Type 2A2, Charcot-Marie-Tooth Disease Type 2N (Diagnosis), Charcot-Marie-Tooth Disease Type 2B5, Charcot-Marie-Tooth Disease Type 2D, Charcot-Marie-Tooth Disease Type 4D, Charcot-Marie-Tooth Disease Type 2K, Charcot-Marie-Tooth Disease Type 2L (Diagnosis), Charcot-Marie-Tooth Disease Type 2T, Charcot-Marie-Tooth Disease Type 2I, Charcot-Marie-Tooth Disease Type 2J, Charcot-Marie-Tooth Disease Type 2E, Charcot-Marie-Tooth Disease Type 2G, Charcot-Marie-Tooth Disease Type 1C, Charcot-Marie-Tooth Disease Type 2R, Charcot-Marie-Tooth Disease Type 2O (Diagnosis), Charcot-Marie-Tooth Disease Type 2M, Charcot-Marie-Tooth Disease Type 2P, Charcot-Marie-Tooth Disease Type 2Y, Charcot-Marie-Tooth Disease Type 4F (Diagnosis), Charcot-Marie-Tooth Disease Type 4B3, Charcot-Marie-Tooth Disease Type 2H, HNPP, X-Linked Charcot-Marie-Tooth Disease, Charcot Marie Tooth Disease (CMT)
Interventions
Not listed
Lead sponsor
Hereditary Neuropathy Foundation
Government
Eligibility
Not listed
Enrollment
10,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2029
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Sep 17, 2026 · Synced Oct 15, 2026, 11:34 PM EDT
Active, not recruiting Phase 2 Interventional Results available

Testing GDC-0032 (Taselisib) as a Potential Targeted Treatment in Cancers With PIK3CA Genetic Changes (MATCH-Subprotocol I)

NCT04439175
View directory record Official record
Conditions
Advanced Lymphoma, Advanced Malignant Solid Neoplasm, Hematopoietic and Lymphoid Cell Neoplasm, Refractory Lymphoma, Refractory Malignant Solid Neoplasm, Refractory Multiple Myeloma
Interventions
Taselisib
Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years and older
Enrollment
70 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2027
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated May 6, 2026 · Synced Oct 15, 2026, 11:34 PM EDT
Completed Not applicable Interventional Results available

Argus® II Retinal Stimulation System Feasibility Protocol

NCT00407602
View directory record Official record
Conditions
Retinitis Pigmentosa
Interventions
Implant of Argus II Retinal Prosthesis
Device
Lead sponsor
Second Sight Medical Products
Industry
Eligibility
18 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2019
U.S. locations
7
States / cities
Los Angeles, California • San Francisco, California • Baltimore, Maryland + 3 more
Source: ClinicalTrials.gov public record
Updated Jan 20, 2022 · Synced Oct 15, 2026, 11:34 PM EDT
Completed Not applicable Interventional

Randomized Study of Folic Acid Therapy for Hyperhomocysteinemia in Patients With End Stage Renal Disease Receiving Hemodialysis

NCT00004495
View directory record Official record
Conditions
End Stage Renal Disease, Hyperhomocysteinemia
Interventions
cyanocobalamin, folic acid, pyridoxine
Drug
Lead sponsor
Georgetown University
Other
Eligibility
21 Years to 89 Years
Enrollment
84 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1999 – 2000
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Mar 25, 2015 · Synced Oct 15, 2026, 11:34 PM EDT
Completed No phase listed Observational

Internet Support Group for Parents of a Child With Neurofibromatosis Type 1

NCT02153931
View directory record Official record
Conditions
Neurofibromatosis 1
Interventions
Not listed
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years to 99 Years
Enrollment
33 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2019
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jan 31, 2019 · Synced Oct 15, 2026, 11:34 PM EDT
Completed Phase 2 Interventional

FR901228 in Treating Patients With Refractory or Progressive Small Cell Lung Cancer or Non-small Cell Lung Cancer

NCT00020202
View directory record Official record
Conditions
Extensive Stage Small Cell Lung Cancer, Stage IIIA Non-small Cell Lung Cancer, Stage IV Non-small Cell Lung Cancer, Recurrent Small Cell Lung Cancer, Stage IIIB Non-small Cell Lung Cancer, Recurrent Non-small Cell Lung Cancer
Interventions
FR901228
Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years and older
Timeline
2000 – 2007
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2024 · Synced Oct 15, 2026, 11:34 PM EDT
Active, not recruiting Phase 2 Interventional Results available

Testing Ipatasertib as Potentially Targeted Treatment in Cancers With AKT Genetic Changes (MATCH - Subprotocol Z1K)

NCT06400251
View directory record Official record
Conditions
Advanced Lymphoma, Advanced Malignant Solid Neoplasm, Refractory Lymphoma, Refractory Malignant Solid Neoplasm, Refractory Multiple Myeloma
Interventions
Biopsy Procedure, Biospecimen Collection, Computed Tomography, Ipatasertib, Magnetic Resonance Imaging
Procedure · Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years and older
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2027
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jun 1, 2026 · Synced Oct 15, 2026, 11:34 PM EDT
Completed Phase 2 Interventional Results available

Etidronate for Arterial Calcifications Due to Deficiency in CD73 (ACDC)

NCT01585402
View directory record Official record
Conditions
Arterial Calcification, CD73 Deficiency
Interventions
Etidronate, MRI Scan
Drug · Device
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
18 Years to 80 Years
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2021
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 4, 2022 · Synced Oct 15, 2026, 11:34 PM EDT
Withdrawn Phase 1 Interventional

Pharmacokinetics of Vancomycin for Inhalation in Cystic Fibrosis

NCT01509339
View directory record Official record
Conditions
Cystic Fibrosis, Methicillin-resistant Staphylococcus Aureus
Interventions
Vancomycin
Drug
Lead sponsor
Case Western Reserve University
Other
Eligibility
18 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2021
U.S. locations
1
States / cities
Cleveland, Ohio
Source: ClinicalTrials.gov public record
Updated Jan 12, 2022 · Synced Oct 15, 2026, 11:34 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 15, 2026, 11:34 PM EDT