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ClinicalTrials.gov public records Last synced Sep 17, 2026, 3:49 AM EDT

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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Genetics Disease Clear all
Completed Phase 1Phase 2 Interventional Results available

PercutaneOus StEm Cell Injection Delivery Effects On Neomyogenesis in Dilated CardioMyopathy (The POSEIDON-DCM Study)

NCT01392625
View directory record Official record
Conditions
Non-ischemic Dilated Cardiomyopathy
Interventions
Autologous hMSCs, Allogeneic hMSCs
Biological
Lead sponsor
Joshua M Hare
Other
Eligibility
18 Years to 95 Years
Enrollment
37 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2017
U.S. locations
1
States / cities
Miami, Florida
Source: ClinicalTrials.gov public record
Updated Feb 14, 2018 · Synced Sep 17, 2026, 3:49 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

D-0120 Safety and PK/PD, Food Effect Study in Healthy Volunteers

NCT03291782
View directory record Official record
Conditions
Gout
Interventions
D-0120
Drug
Lead sponsor
InventisBio Co., Ltd
Industry
Eligibility
18 Years to 60 Years
Enrollment
39 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2017 – 2018
U.S. locations
1
States / cities
Daytona Beach, Florida
Source: ClinicalTrials.gov public record
Updated Dec 18, 2018 · Synced Sep 17, 2026, 3:49 AM EDT
Completed Phase 3 Interventional Results available

An Efficacy and Safety Study of Luspatercept (ACE-536) Versus Placebo in Adults Who Require Regular Red Blood Cell Transfusions Due to Beta (β) Thalassemia

NCT02604433
View directory record Official record
Conditions
Erythrocyte Transfusion, Beta-Thalassemia
Interventions
Luspatercept, Placebo
Drug · Other
Lead sponsor
Celgene
Industry
Eligibility
18 Years and older
Enrollment
336 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2021
U.S. locations
6
States / cities
Los Angeles, California • Oakland, California • Chicago, Illinois + 3 more
Source: ClinicalTrials.gov public record
Updated Apr 17, 2023 · Synced Sep 17, 2026, 3:49 AM EDT
Completed Phase 3 Interventional Results available

Combining Lesinurad With Allopurinol in Inadequate Responders

NCT01510158
View directory record Official record
Conditions
Gout
Interventions
Lesinurad, Placebo, Allopurinol
Drug
Lead sponsor
Ardea Biosciences, Inc.
Industry
Eligibility
18 Years to 85 Years
Enrollment
607 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2014
U.S. locations
165
States / cities
Athens, Alabama • Birmingham, Alabama • Gulf Shores, Alabama + 139 more
Source: ClinicalTrials.gov public record
Updated Aug 17, 2016 · Synced Sep 17, 2026, 3:49 AM EDT
Completed No phase listed Observational

Feasibility of Generating Pluripotent Stem Cells From Patients With Familial Retinoblastoma

NCT02193724
View directory record Official record
Conditions
Retinoblastoma
Interventions
Skin Biopsy, Blood Draw
Other
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
Not listed
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2019
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Oct 22, 2020 · Synced Sep 17, 2026, 3:49 AM EDT
Recruiting Phase 2 Interventional

Intraamniotic Administrations of ER004 to Male Subjects With X-linked Hypohidrotic Ectodermal Dysplasia

NCT04980638
View directory record Official record
Conditions
X-Linked Hypohidrotic Ectodermal Dysplasia (XLHED)
Interventions
ER004
Biological
Lead sponsor
EspeRare Foundation
Other
Eligibility
18 Years and older · Female only
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2032
U.S. locations
2
States / cities
Los Angeles, California • St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Apr 29, 2025 · Synced Sep 17, 2026, 3:49 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers Results available

Sickle Cell Anemia WE CARE

NCT03716726
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
WE CARE SDoH Screening Survey, Family Resource Book, Standard of care
Behavioral · Other
Lead sponsor
Boston Medical Center
Other
Eligibility
18 Years and older
Enrollment
112 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2023
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Mar 25, 2024 · Synced Sep 17, 2026, 3:49 AM EDT
Completed Phase 2 Interventional

Inhaled Mometasone to Promote Reduction in Vasoocclusive Events 2

NCT03758950
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Placebo, Mometasone
Drug
Lead sponsor
Jeffrey Glassberg
Other
Eligibility
18 Years and older
Enrollment
80 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2023
U.S. locations
2
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jun 17, 2023 · Synced Sep 17, 2026, 3:49 AM EDT
Not listed Phase 2 Interventional

Phase II Study of Dietary Cholesterol for Smith-Lemli-Opitz Syndrome

NCT00004347
View directory record Official record
Conditions
Smith-Lemli-Opitz Syndrome
Interventions
diet
Behavioral
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
0 Years and older
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1995
U.S. locations
1
States / cities
Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Jun 23, 2005 · Synced Sep 17, 2026, 3:49 AM EDT
Recruiting Phase 1Phase 2 Interventional

A Study to Evaluate Safety, Tolerability, and Efficacy of AB-1009 Gene Therapy (GAA Gene) in Adult Participants With Late-Onset Pompe Disease (PROGRESS-GT LOPD)

NCT07282847
View directory record Official record
Conditions
Pompe Disease (Late-onset), Pompe Disease Late-Onset, LOPD
Interventions
AB-1009 (GAA Gene)
Genetic
Lead sponsor
AskBio Inc
Industry
Eligibility
18 Years to 65 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2032
U.S. locations
9
States / cities
Phoenix, Arizona • Irvine, California • Palo Alto, California + 6 more
Source: ClinicalTrials.gov public record
Updated Jul 19, 2026 · Synced Sep 17, 2026, 3:49 AM EDT
Completed No phase listed Observational

Goal Attainment Scaling (GAS) Sub-Study

NCT06484426
View directory record Official record
Conditions
Alzheimer's Disease in Down Syndrome
Interventions
Not listed
Lead sponsor
LuMind IDSC Foundation
Other
Eligibility
35 Years and older
Enrollment
46 participants
Timeline
2021 – 2024
U.S. locations
5
States / cities
San Diego, California • Park Ridge, Illinois • Baltimore, Maryland + 2 more
Source: ClinicalTrials.gov public record
Updated Jul 2, 2024 · Synced Sep 17, 2026, 3:49 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Sickle Cell Disease and the Genomic and Gene Therapy Needs of Stakeholders

NCT04416178
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Not listed
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
13 Years and older
Enrollment
352 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2026
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Apr 30, 2026 · Synced Sep 17, 2026, 3:49 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Radiofrequency and Ultrasound for Improvement of Skin Laxity and Wrinkles: Efficacy and Safety Evaluation

NCT05929625
View directory record Official record
Conditions
Skin Laxity
Interventions
BTL-585-2 RF+US, BTL-585-2 RF
Device
Lead sponsor
BTL Industries Ltd.
Industry
Eligibility
21 Years and older
Enrollment
41 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2022
U.S. locations
2
States / cities
Birmingham, Michigan • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jul 2, 2023 · Synced Sep 17, 2026, 3:49 AM EDT
Recruiting Phase 1 Interventional

A Study of the c-Kit Specific Antibody-Drug Conjugate NN3201 for Advanced and/or Metastatic Solid Tumors Known to Express c-Kit

NCT06805825
View directory record Official record
Conditions
Gastrointestinal Stromal Tumors, Small-cell Lung Cancer, Adenoid Cystic Carcinoma, Uveal Melanoma, Neuroendocrine Tumors, Chromophobe Renal Cell Carcinoma, Clear Cell Renal Cell Carcinoma
Interventions
NN3201
Drug
Lead sponsor
Novelty Nobility, Inc.
Industry
Eligibility
18 Years and older
Enrollment
67 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
5
States / cities
Ann Arbor, Michigan • Cleveland, Ohio • Portland, Oregon + 2 more
Source: ClinicalTrials.gov public record
Updated Sep 22, 2025 · Synced Sep 17, 2026, 3:49 AM EDT
Completed Phase 2 Interventional Results available

Proton Pump Inhibitors (PPI) and Fat Absorption in Cystic Fibrosis (CF) and Exocrine Pancreatic Insufficiency (EPI)

NCT03551691
View directory record Official record
Conditions
Pancreatic Insufficiency, Cystic Fibrosis
Interventions
Omeprazole 20mg Capsule, Placebo oral capsule
Drug
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
12 Years and older
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2022
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 29, 2024 · Synced Sep 17, 2026, 3:49 AM EDT
Terminated Not applicable Interventional

Defining the Role of Insulin Resistance in 'Idiopathic' Dilated Cardiomyopathy

NCT00466713
View directory record Official record
Conditions
Dilated Cardiomyopathy
Interventions
Rosiglitazone therapy
Drug
Lead sponsor
Stanford University
Other
Eligibility
18 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007
U.S. locations
1
States / cities
Stanford, California
Source: ClinicalTrials.gov public record
Updated May 12, 2014 · Synced Sep 17, 2026, 3:49 AM EDT
Completed Phase 1 Interventional

A Study of JNJ-64251330 in Participants With Familial Adenomatous Polyposis

NCT05014360
View directory record Official record
Conditions
Adenomatous Polyposis Coli
Interventions
JNJ-64251330
Drug
Lead sponsor
Janssen Research & Development, LLC
Industry
Eligibility
18 Years and older
Enrollment
42 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
5
States / cities
Duarte, California • Miami, Florida • Boston, Massachusetts + 1 more
Source: ClinicalTrials.gov public record
Updated Jun 1, 2023 · Synced Sep 17, 2026, 3:49 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Adherence to Colon Cancer Screening (ACCS)

NCT02112747
View directory record Official record
Conditions
Colon Cancer, Rectal Cancer
Interventions
Patient Navigator
Other
Lead sponsor
Ohio State University Comprehensive Cancer Center
Other
Eligibility
25 Years to 75 Years
Enrollment
1,900 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2020
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Mar 9, 2021 · Synced Sep 17, 2026, 3:49 AM EDT
Active, not recruiting Not applicable Interventional

Early Feasibility Evaluation of the AccuCinch® Ventricular Restoration System in Patients With Heart Failure and Reduced Ejection Fraction

NCT03533517
View directory record Official record
Conditions
Heart Failure, Cardiomyopathy, Dilated
Interventions
AccuCinch® Ventricular Restoration
Device
Lead sponsor
Ancora Heart, Inc.
Industry
Eligibility
18 Years and older
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2026
U.S. locations
18
States / cities
San Francisco, California • Atlanta, Georgia • Louisville, Kentucky + 14 more
Source: ClinicalTrials.gov public record
Updated Jan 29, 2025 · Synced Sep 17, 2026, 3:49 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

High Risk Multiple Gestation Study

NCT02278874
View directory record Official record
Conditions
Trisomy 13, Trisomy 18, Trisomy 21, Sex Chromosome Abnormalities
Interventions
Not listed
Lead sponsor
Natera, Inc.
Industry
Eligibility
18 Years and older
Enrollment
99 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2014 – 2019
U.S. locations
4
States / cities
Boston, Massachusetts • Glen Cove, New York • New York, New York + 1 more
Source: ClinicalTrials.gov public record
Updated Jun 12, 2019 · Synced Sep 17, 2026, 3:49 AM EDT
Withdrawn No phase listed Observational

Breast Cancer BRCA1 Carriers: a Pilot Study

NCT05062174
View directory record Official record
Conditions
BRCA1 Mutation, High-grade Serous Ovarian Cancer, TNBC - Triple-Negative Breast Cancer
Interventions
Mifepristone 200 MG, Prophylactic mastectomy
Drug · Procedure
Lead sponsor
Indiana University
Other
Eligibility
18 Years and older · Female only
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
2
States / cities
Carmel, Indiana • Indianapolis, Indiana
Source: ClinicalTrials.gov public record
Updated Dec 14, 2022 · Synced Sep 17, 2026, 3:49 AM EDT
Recruiting Phase 2 Interventional

A Long Term Extension Study to Evaluate the Safety and Efficacy of Afimkibart (RO7790121) in Participants With Atopic Dermatitis

NCT07223697
View directory record Official record
Conditions
Atopic Dermatitis
Interventions
Afimkibart
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
18 Years and older
Enrollment
120 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2033
U.S. locations
6
States / cities
Los Angeles, California • Tampa, Florida • Alpharetta, Georgia + 2 more
Source: ClinicalTrials.gov public record
Updated Sep 3, 2026 · Synced Sep 17, 2026, 3:49 AM EDT
Completed Not applicable Interventional

Lingual Muscle Training in Late-Onset Pompe Disease (LOPD)

NCT03255213
View directory record Official record
Conditions
Glycogen Storage Disease
Interventions
Lingual Muscle Training
Other
Lead sponsor
Duke University
Other
Eligibility
12 Years and older
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2020
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Jul 27, 2021 · Synced Sep 17, 2026, 3:49 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Natural History of Amyloid Deposition in Adults With Down Syndrome

NCT01303133
View directory record Official record
Conditions
Down Syndrome
Interventions
Not listed
Lead sponsor
University of Pittsburgh
Other
Eligibility
30 Years and older
Enrollment
81 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2018
U.S. locations
2
States / cities
Pittsburgh, Pennsylvania • Madison, Wisconsin
Source: ClinicalTrials.gov public record
Updated Jan 8, 2019 · Synced Sep 17, 2026, 3:49 AM EDT