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ClinicalTrials.gov public records Last synced Oct 28, 2026, 11:57 PM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Showing 1585–1608 of 10,812 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Developmental Defect Clear all
Completed Phase 1Phase 2 Interventional

Scotoma Reduction in AMD Patients Treated With Ranibizumab

NCT00467935
View directory record Official record
Conditions
Macular Degeneration
Interventions
intravitreal injection Lucentis (ranibizumab)
Drug
Lead sponsor
Retina Research Foundation
Other
Eligibility
50 Years to 90 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007
U.S. locations
1
States / cities
Slingerlands, New York
Source: ClinicalTrials.gov public record
Updated Jul 29, 2011 · Synced Oct 28, 2026, 11:57 PM EDT
Recruiting Phase 3 Interventional

A Study to Investigate the Effect of Mitapivat on Transfusion Burden in Subjects With Sickle Cell Disease (SCD)

NCT07656415
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Mitapivat Matched Placebo, Mitapivat
Drug
Lead sponsor
Agios Pharmaceuticals, Inc.
Industry
Eligibility
12 Years and older
Enrollment
159 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2030
U.S. locations
5
States / cities
Indianapolis, Indiana • Grand Rapids, Michigan • Flowood, Mississippi + 2 more
Source: ClinicalTrials.gov public record
Updated Oct 28, 2026 · Synced Oct 28, 2026, 11:57 PM EDT
Completed No phase listed Observational

The Role of Bacteria and Genetic Variations in Cystic Fibrosis

NCT00043225
View directory record Official record
Conditions
Psuedomas Infection, Cystic Fibrosis
Interventions
Not listed
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
9 Years to 80 Years
Enrollment
76 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2018
U.S. locations
4
States / cities
Bethesda, Maryland • Seattle, Washington • Madison, Wisconsin + 1 more
Source: ClinicalTrials.gov public record
Updated Jun 29, 2021 · Synced Oct 28, 2026, 11:57 PM EDT
Completed Phase 4 Interventional Accepts healthy volunteers Results available

Dupilumab Skin Barrier Function Study in Atopic Dermatitis

NCT04447417
View directory record Official record
Conditions
Atopic Dermatitis
Interventions
Dupilumab SAR231893
Drug
Lead sponsor
Sanofi
Industry
Eligibility
12 Years to 65 Years
Enrollment
52 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2021
U.S. locations
1
States / cities
Denver, Colorado
Source: ClinicalTrials.gov public record
Updated Sep 18, 2025 · Synced Oct 28, 2026, 11:57 PM EDT
Terminated Phase 3 Interventional Results available

Comparing Individualized vs. Weight Based Protocols to Treat Vaso-Occlusive Episodes in Sickle Cell Disease

NCT03933397
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Patient-Specific Protocol, Weight-based Protocol, Morphine, Hydromorphone
Other · Drug
Lead sponsor
Duke University
Other
Eligibility
18 Years and older
Enrollment
328 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
6
States / cities
Baltimore, Maryland • Detroit, Michigan • Charlotte, North Carolina + 2 more
Source: ClinicalTrials.gov public record
Updated Jul 3, 2023 · Synced Oct 28, 2026, 11:57 PM EDT
Completed No phase listed Observational Results available

Study of Long-Term Safety and Efficacy on Gene Therapy in Glycogen Storage Disease Type Ia

NCT03970278
View directory record Official record
Conditions
Glycogen Storage Disease Type IA, Von Gierke's Disease (GSD Type Ia)
Interventions
No intervention
Other
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
18 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2025
U.S. locations
3
States / cities
Farmington, Connecticut • Ann Arbor, Michigan • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Mar 18, 2026 · Synced Oct 28, 2026, 11:57 PM EDT
Not listed No phase listed Observational

A Long-term Follow-up Study to Evaluate the Safety and Efficacy of RGX-501

NCT04080050
View directory record Official record
Conditions
Homozygous Familial Hypercholesterolemia (HoFH)
Interventions
Single intravenous (IV) dose of human Low Density Lipoprotein Receptor (LDLR) Gene Therapy
Drug
Lead sponsor
REGENXBIO Inc.
Industry
Eligibility
18 Years and older
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2025
U.S. locations
4
States / cities
Columbus, Ohio • Portland, Oregon • Philadelphia, Pennsylvania + 1 more
Source: ClinicalTrials.gov public record
Updated Mar 9, 2023 · Synced Oct 28, 2026, 11:57 PM EDT
Completed Phase 1 Interventional

Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Pociredir

NCT05169580
View directory record Official record
Conditions
Sickle Cell Disease, Sickle Cell Anemia
Interventions
Pociredir oral capsule(s)
Drug
Lead sponsor
Fulcrum Therapeutics
Industry
Eligibility
18 Years to 65 Years
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2026
U.S. locations
14
States / cities
Little Rock, Arkansas • Los Angeles, California • Atlanta, Georgia + 11 more
Source: ClinicalTrials.gov public record
Updated Feb 12, 2026 · Synced Oct 28, 2026, 11:57 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

MRI Spectroscopy and Neuropsychological Functioning in Phenylketonuria

NCT03097250
View directory record Official record
Conditions
Phenylketonuria
Interventions
Not listed
Lead sponsor
Boston Children's Hospital
Other
Eligibility
12 Years to 25 Years
Enrollment
10 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2017 – 2018
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Oct 19, 2018 · Synced Oct 28, 2026, 11:57 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Detecting Dopaminergic Deficits in Individuals At-risk for Parkinsonism

NCT01358474
View directory record Official record
Conditions
Parkinson Disease, Gaucher Disease, Idiopathic Rapid Eye Movement Sleep Disorder
Interventions
Not listed
Lead sponsor
University of Minnesota
Other
Eligibility
18 Years to 85 Years
Enrollment
56 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2011 – 2018
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Apr 18, 2019 · Synced Oct 28, 2026, 11:57 PM EDT
Terminated Phase 1 Interventional Results available

Androgen Reduction in Congenital Adrenal Hyperplasia, Phase 1

NCT02574910
View directory record Official record
Conditions
Congenital Adrenal Hyperplasia
Interventions
Abiraterone acetate
Drug
Lead sponsor
University of Texas Southwestern Medical Center
Other
Eligibility
2 Years to 9 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2023
U.S. locations
4
States / cities
Los Angeles, California • Bethesda, Maryland • Ann Arbor, Michigan + 1 more
Source: ClinicalTrials.gov public record
Updated Nov 4, 2025 · Synced Oct 28, 2026, 11:57 PM EDT
Terminated Phase 2 Interventional

Safety and Efficacy of OMS643762 in Subjects With Huntington's Disease

NCT02074410
View directory record Official record
Conditions
Huntington's Disease
Interventions
OMS643762, Placebo
Drug
Lead sponsor
Omeros Corporation
Industry
Eligibility
18 Years to 65 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014
U.S. locations
9
States / cities
San Diego, California • Englewood, Colorado • Gainesville, Florida + 6 more
Source: ClinicalTrials.gov public record
Updated Oct 22, 2018 · Synced Oct 28, 2026, 11:57 PM EDT
Completed No phase listed Observational

Relationship of Brain Natriuretic Peptide (BNP) Levels to Cardiac Diagnosis, Operation Performed, Post-operative Course and Outcome

NCT00238810
View directory record Official record
Conditions
Congenital Heart Disease
Interventions
Not listed
Lead sponsor
Children's Healthcare of Atlanta
Other
Eligibility
Not listed
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2014
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Dec 2, 2014 · Synced Oct 28, 2026, 11:57 PM EDT
Completed Phase 2 Interventional

Stem Cell Transplantation With Identical Donors for Patients With Sickle Cell Disease

NCT00186810
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Busulfan, Cyclophosphamide, Horse ATG, Allogeneic stem cell transplant
Drug · Procedure
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
Up to 21 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1992 – 2007
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated May 29, 2009 · Synced Oct 28, 2026, 11:57 PM EDT
Completed Phase 2 Interventional

Safety and Efficacy Study of Q301 in Mild to Moderate Adolescents and Adults Atopic Dermatitis Patients

NCT03571620
View directory record Official record
Conditions
Atopic Dermatitis
Interventions
Q301 Cream, Vehicle
Drug
Lead sponsor
Qurient Co., Ltd.
Industry
Eligibility
12 Years to 70 Years
Enrollment
258 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2020
U.S. locations
1
States / cities
Winston-Salem, North Carolina
Source: ClinicalTrials.gov public record
Updated Apr 24, 2020 · Synced Oct 28, 2026, 11:57 PM EDT
Active, not recruiting No phase listed Observational

Neurobiological Predictors of Huntington's Disease (PREDICT-HD)

NCT00051324
View directory record Official record
Conditions
Huntington Disease
Interventions
Not listed
Lead sponsor
Jordan Schultz
Other
Eligibility
18 Years and older
Enrollment
1,700 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2025
U.S. locations
1
States / cities
Iowa City, Iowa
Source: ClinicalTrials.gov public record
Updated Jan 14, 2025 · Synced Oct 28, 2026, 11:57 PM EDT
Completed No phase listed Observational

Genetic Modifiers of Cystic Fibrosis: Sibling Study

NCT00037778
View directory record Official record
Conditions
Lung Diseases, Cystic Fibrosis
Interventions
Not listed
Lead sponsor
Johns Hopkins University
Other
Eligibility
Up to 100 Years
Enrollment
3,459 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2013
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 31, 2016 · Synced Oct 28, 2026, 11:57 PM EDT
Completed No phase listed Observational

Molecular Mechanisms and Diagnosis of Mastocytosis

NCT00336076
View directory record Official record
Conditions
Mastocytosis
Interventions
Collection of blood and bone marrow
Other
Lead sponsor
University of Michigan
Other
Eligibility
Not listed
Enrollment
136 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2009
U.S. locations
1
States / cities
Ann Arbor, Michigan
Source: ClinicalTrials.gov public record
Updated Nov 7, 2016 · Synced Oct 28, 2026, 11:57 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Angelman Syndrome Video Assessment (ASVA) Source Material Study

NCT05637697
View directory record Official record
Conditions
Angelman Syndrome
Interventions
Not listed
Lead sponsor
The Emmes Company, LLC
Industry
Eligibility
1 Year and older
Enrollment
73 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2023
U.S. locations
1
States / cities
Kingston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Dec 13, 2023 · Synced Oct 28, 2026, 11:57 PM EDT
Completed No phase listed Observational Results available

Observational Safety Study for KALBITOR (Ecallantide) in the Treatment of Acute Attacks of Hereditary Angioedema

NCT01059526
View directory record Official record
Conditions
Hereditary Angioedema (HAE)
Interventions
ecallantide
Drug
Lead sponsor
Shire
Industry
Eligibility
16 Years and older
Enrollment
81 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2014
U.S. locations
40
States / cities
Little Rock, Arkansas • Bell Gardens, California • Granada Hills, California + 35 more
Source: ClinicalTrials.gov public record
Updated Jun 8, 2021 · Synced Oct 28, 2026, 11:57 PM EDT
Terminated Not applicable Interventional

Post Approval Study: NOVATION Ceramic Articulation Hip System

NCT00715143
View directory record Official record
Conditions
Total Hip Replacement (Non-inflammatory)
Interventions
Novation Ceramic
Device
Lead sponsor
Exactech
Industry
Eligibility
21 Years and older
Enrollment
126 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2021
U.S. locations
5
States / cities
Fort Lauderdale, Florida • Cumming, Georgia • Worcester, Massachusetts + 2 more
Source: ClinicalTrials.gov public record
Updated Aug 31, 2022 · Synced Oct 28, 2026, 11:57 PM EDT
Completed Phase 4 Interventional Results available

Allopurinol Outcome Study

NCT01391325
View directory record Official record
Conditions
Gout
Interventions
Allopurinol
Drug
Lead sponsor
Ardea Biosciences, Inc.
Industry
Eligibility
18 Years to 85 Years
Enrollment
1,735 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2013
U.S. locations
127
States / cities
Birmingham, Alabama • Bringham, Alabama • Calera, Alabama + 113 more
Source: ClinicalTrials.gov public record
Updated Jun 17, 2014 · Synced Oct 28, 2026, 11:57 PM EDT
No Longer Available No phase listed Expanded access

Treatment Use of 3,4-Diaminopyridine

NCT01765140
View directory record Official record
Conditions
Myasthenic Syndromes, Congenital
Interventions
3,4-diaminopyridine
Drug
Lead sponsor
Vern C. Juel, M.D.
Other
Eligibility
Not listed
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Jan 20, 2025 · Synced Oct 28, 2026, 11:57 PM EDT
Completed Phase 2 Interventional Results available

Safety and Efficacy of Pitolisant on Excessive Daytime Sleepiness and Other Non-Muscular Symptoms in Patients With Myotonic Dystrophy Type 1

NCT04886518
View directory record Official record
Conditions
Myotonic Dystrophy 1, Excessive Daytime Sleepiness
Interventions
Pitolisant Oral Tablet, Placebo oral tablet
Drug
Lead sponsor
Harmony Biosciences Management, Inc.
Industry
Eligibility
18 Years to 65 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
11
States / cities
Irvine, California • Aurora, Colorado • Tampa, Florida + 8 more
Source: ClinicalTrials.gov public record
Updated Sep 4, 2026 · Synced Oct 28, 2026, 11:57 PM EDT