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ClinicalTrials.gov public records Last synced Oct 25, 2026, 3:34 PM EDT

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Showing 1009–1032 of 10,802 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Developmental Defect Clear all
Completed Phase 3 Interventional Results available

OASIS-HAE: A Study to Evaluate the Safety and Efficacy of Donidalorsen (ISIS 721744 or IONIS-PKK-LRx) in Participants With Hereditary Angioedema (HAE)

NCT05139810
View directory record Official record
Conditions
Hereditary Angioedema
Interventions
Donidalorsen, Placebo
Drug
Lead sponsor
Ionis Pharmaceuticals, Inc.
Industry
Eligibility
12 Years and older
Enrollment
91 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
15
States / cities
Scottsdale, Arizona • San Diego, California • Santa Monica, California + 12 more
Source: ClinicalTrials.gov public record
Updated Mar 6, 2025 · Synced Oct 25, 2026, 3:34 PM EDT
Terminated Phase 4 Interventional

Safety Study of Galantamine in Tic Disorders

NCT00226824
View directory record Official record
Conditions
Tourette's Syndrome, Motor Tic Disorder, Vocal Tic Disorder
Interventions
galantamine
Drug
Lead sponsor
Parkinson's Disease and Movement Disorders Center
Other
Eligibility
18 Years to 50 Years
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2007
U.S. locations
1
States / cities
Albany, New York
Source: ClinicalTrials.gov public record
Updated Aug 25, 2009 · Synced Oct 25, 2026, 3:34 PM EDT
Completed Not applicable Interventional Results available

Spinal Stimulation and Mobility Devices

NCT05520359
View directory record Official record
Conditions
Cerebral Palsy, Stroke, Premature Birth, Hypoxic-Ischemic Encephalopathy, Periventricular Leukomalacia
Interventions
Mobility Device, Spinal Stimulation
Device
Lead sponsor
University of Washington
Other
Eligibility
4 Years to 70 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2023
U.S. locations
1
States / cities
Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Sep 18, 2026 · Synced Oct 25, 2026, 3:34 PM EDT
Completed Phase 3 Interventional

Study of SRP-4045 (Casimersen) and SRP-4053 (Golodirsen) in Participants With Duchenne Muscular Dystrophy (DMD)

NCT02500381
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
SRP-4045, SRP-4053, Placebo
Drug
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
6 Years to 13 Years · Male only
Enrollment
228 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2025
U.S. locations
23
States / cities
Phoenix, Arizona • Los Angeles, California • San Diego, California + 19 more
Source: ClinicalTrials.gov public record
Updated Nov 18, 2025 · Synced Oct 25, 2026, 3:34 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers Results available

MRI for Non-Invasive Imaging in Neonates and Children

NCT02163681
View directory record Official record
Conditions
Healthy, Cystic Fibrosis (CF), Asthma, Bronchopulmonary Dysplasia (BPD)
Interventions
Hyperpolarized Helium-3 MRI of the chest
Drug
Lead sponsor
University of Virginia
Other
Eligibility
4 Months to 65 Years
Enrollment
45 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2011 – 2018
U.S. locations
1
States / cities
Charlottesville, Virginia
Source: ClinicalTrials.gov public record
Updated Apr 19, 2022 · Synced Oct 25, 2026, 3:34 PM EDT
Recruiting Phase 1 Interventional Accepts healthy volunteers

A Research Study Looking at How Different Doses of Study Medicine (Inno8) Work in the Body of People With Haemophilia A

NCT07220564
View directory record Official record
Conditions
Haemophilia A
Interventions
NNC0442-0344 A
Drug
Lead sponsor
Novo Nordisk A/S
Industry
Eligibility
18 Years to 64 Years · Male only
Enrollment
30 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2025 – 2027
U.S. locations
5
States / cities
Aurora, Colorado • Indianapolis, Indiana • Iowa City, Iowa + 2 more
Source: ClinicalTrials.gov public record
Updated Sep 18, 2026 · Synced Oct 25, 2026, 3:34 PM EDT
Terminated Phase 1Phase 2 Interventional Results available

Safety and Efficacy Study of Sotatercept in Adults With Transfusion Dependent Diamond Blackfan Anemia

NCT01464164
View directory record Official record
Conditions
Diamond Blackfan Anemia
Interventions
Sotatercept, Sotatercept with prednisone boost
Drug
Lead sponsor
Northwell Health
Other
Eligibility
18 Years and older
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2021
U.S. locations
1
States / cities
Manhasset, New York
Source: ClinicalTrials.gov public record
Updated Mar 17, 2023 · Synced Oct 25, 2026, 3:34 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Integrated Cancer Repository for Cancer Research

NCT02012699
View directory record Official record
Conditions
Pancreatic Cancer, Thyroid Cancer, Lung Cancer, Esophageal Cancer, Thymus Cancer, Colon Cancer, Rectal Cancer, Gastrointestinal Stromal Tumors, Anal Cancer, Bile Duct Cancer, Duodenal Cancer, Gallbladder Cancer, Gastric Cancer, Liver Cancer, Small Intestine Cancer, Peritoneal Surface Malignancies, Familial Adenomatous Polyposis, Lynch Syndrome, Bladder Cancer, Kidney Cancer, Penile Cancer, Prostate Cancer, Testicular Cancer, Ureter Cancer, Urethral Cancer, Hypopharyngeal Cancer, Laryngeal Cancer, Lip Cancer, Oral Cavity Cancer, Nasopharyngeal Cancer, Oropharyngeal Cancer, Paranasal Sinus Cancer, Nasal Cavity Cancer, Salivary Gland Cancer, Skin Cancer, Central Nervous System Tumor, Central Nervous System Cancer, Mesothelioma, Breast Cancer, Leukemia, Melanoma, Sarcoma, Unknown Primary Tumor, Multiple Myeloma, Ovarian Cancer, Endometrial Cancer, Vaginal Cancer, Neuroendocrine Tumors, Plasma Cell Dyscrasia, Healthy Control
Interventions
Not listed
Lead sponsor
University of Nebraska
Other
Eligibility
19 Years to 110 Years
Enrollment
999,999 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2099
U.S. locations
42
States / cities
Greenwood Village, Colorado • Manchester, Connecticut • Daytona Beach, Florida + 34 more
Source: ClinicalTrials.gov public record
Updated Jun 29, 2026 · Synced Oct 25, 2026, 3:34 PM EDT
Not listed No phase listed Observational

Variables That Are Correlated to Developing Multiple Endocrine Neoplasia (MEN) and Pancreatic Neuroendocrine Tumors (PNET)

NCT03053999
View directory record Official record
Conditions
Multiple Endocrine Neoplasia, Pancreatic Neuroendocrine Tumors, Hyperparathyroidism
Interventions
Genome Sequencing, Data Review
Other
Lead sponsor
M.D. Anderson Cancer Center
Other
Eligibility
Not listed
Enrollment
53 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2023
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Sep 12, 2019 · Synced Oct 25, 2026, 3:34 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Familial Partial Lipodystrophy Study

NCT02858830
View directory record Official record
Conditions
Lipodystrophy, Familial Partial
Interventions
High fat mixed meal
Other
Lead sponsor
Mayo Clinic
Other
Eligibility
18 Years and older
Enrollment
12 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2016 – 2017
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Nov 24, 2021 · Synced Oct 25, 2026, 3:34 PM EDT
Completed Not applicable Interventional

Decompression Coring Versus Conservative Therapy in Patients With Avascular Necrosis of the Hip Related to Sickle Cell Disease

NCT00006130
View directory record Official record
Conditions
Bone Avascular Necrosis, Sickle Cell Anemia
Interventions
Core decompression
Procedure
Lead sponsor
UCSF Benioff Children's Hospital Oakland
Other
Eligibility
10 Years and older
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1999
U.S. locations
2
States / cities
Oakland, California • Chapel Hill, North Carolina
Source: ClinicalTrials.gov public record
Updated Feb 15, 2017 · Synced Oct 25, 2026, 3:34 PM EDT
Completed Phase 2 Interventional Results available

Study of Burosumab (KRN23) in Adults With Tumor-Induced Osteomalacia (TIO) or Epidermal Nevus Syndrome (ENS)

NCT02304367
View directory record Official record
Conditions
Tumor Induced Osteomalacia (TIO), Epidermal Nevus Syndrome (ENS)
Interventions
Burosumab
Biological
Lead sponsor
Kyowa Kirin, Inc.
Industry
Eligibility
18 Years and older
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2021
U.S. locations
7
States / cities
Golden, Colorado • New Haven, Connecticut • Indianapolis, Indiana + 4 more
Source: ClinicalTrials.gov public record
Updated May 6, 2024 · Synced Oct 25, 2026, 3:34 PM EDT
Not listed No phase listed Observational

Cure Cystinosis International Registry

NCT01327807
View directory record Official record
Conditions
Cystinosis, Nephropathic Cystinosis, Renal Fanconi Syndrome
Interventions
Not listed
Lead sponsor
Cystinosis Research Foundation
Other
Eligibility
Not listed
Enrollment
750 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2022
U.S. locations
1
States / cities
San Diego, California
Source: ClinicalTrials.gov public record
Updated Mar 12, 2014 · Synced Oct 25, 2026, 3:34 PM EDT
Enrolling by invitation No phase listed Observational

EEG Dynamics in Lennox-Gastaut Syndrome Patients Undergoing Fenfluramine Treatment

NCT07555171
View directory record Official record
Conditions
Lennox Gastaut Syndrome (LGS)
Interventions
fenfluramine HCl
Drug
Lead sponsor
University of Chicago
Other
Eligibility
2 Years to 35 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Aug 6, 2026 · Synced Oct 25, 2026, 3:34 PM EDT
Completed No phase listed Observational

Gait Analysis in MPS IVA

NCT01920828
View directory record Official record
Conditions
MPS IVA, Morquio Syndrome
Interventions
Not listed
Lead sponsor
Oregon Health and Science University
Other
Eligibility
1 Year and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2017
U.S. locations
1
States / cities
Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Oct 17, 2019 · Synced Oct 25, 2026, 3:34 PM EDT
Active, not recruiting Phase 1 Interventional

Long-term Follow-up (LTFU) of Patients Treated With Genome-edited Autologous Hematopoietic Stem and Progenitor Cells (HSPC)

NCT06155500
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
OTQ923
Biological
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
18 Years to 100 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2039
U.S. locations
3
States / cities
Chicago, Illinois • New York, New York • Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Jul 28, 2026 · Synced Oct 25, 2026, 3:34 PM EDT
Completed No phase listed Observational

Airway Clearance Therapy on Hyperpolarized 129Xenon and MRI

NCT03593434
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
hyperpolarized Xenon gas
Drug
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
6 Years to 21 Years
Enrollment
20 participants
Timeline
2018 – 2021
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Mar 10, 2022 · Synced Oct 25, 2026, 3:34 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

BioEnergetics and Metabolomics in Cystic Fibrosis

NCT02225899
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Not listed
Lead sponsor
Emory University
Other
Eligibility
18 Years to 50 Years
Enrollment
50 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2014 – 2018
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Nov 29, 2019 · Synced Oct 25, 2026, 3:34 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Exercise Capacity in Pediatric Sickle Cell Anemia

NCT01527799
View directory record Official record
Conditions
Sickle Cell Anemia
Interventions
Not listed
Lead sponsor
Ann & Robert H Lurie Children's Hospital of Chicago
Other
Eligibility
8 Years to 21 Years
Enrollment
60 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2015
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Feb 4, 2016 · Synced Oct 25, 2026, 3:34 PM EDT
Recruiting Phase 2 Interventional

A Study of ROC-101 in Patients With Pulmonary Arterial Hypertension (PAH) and Pulmonary Hypertension Associated With Interstitial Lung Disease (ILD-PH) (ROCSTAR STUDY)

NCT07175038
View directory record Official record
Conditions
Pulmonary Arterial Hypertension (PAH), Pulmonary Hypertension, Interstitial Lung Disease
Interventions
ROC-101
Drug
Lead sponsor
AllRock Bio, Inc.
Industry
Eligibility
18 Years to 80 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2029
U.S. locations
11
States / cities
Phoenix, Arizona • Rancho Cordova, California • Aurora, Colorado + 8 more
Source: ClinicalTrials.gov public record
Updated Oct 8, 2026 · Synced Oct 25, 2026, 3:34 PM EDT
Completed No phase listed Observational

Effects of Enzyme Replacement in Gaucher's Disease

NCT00001289
View directory record Official record
Conditions
Gaucher's Disease
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Not listed
Enrollment
70 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1991 – 2008
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 2, 2017 · Synced Oct 25, 2026, 3:34 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Safety and Efficacy of Clenbuterol in Individuals With Late-onset Pompe Disease and Receiving Enzyme Replacement Therapy

NCT01942590
View directory record Official record
Conditions
Pompe Disease
Interventions
Clenbuterol, Placebo
Drug
Lead sponsor
Dwight Koeberl, M.D., Ph.D.
Other
Eligibility
18 Years and older
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2016
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Jul 2, 2019 · Synced Oct 25, 2026, 3:34 PM EDT
Completed Phase 2 Interventional Results available

A Multi-Center Study of Riociguat in Patients With Sickle Cell Diseases

NCT02633397
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Riociguat, Placebo
Drug
Lead sponsor
Mark Gladwin
Other
Eligibility
18 Years and older
Enrollment
130 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2022
U.S. locations
20
States / cities
Oakland, California • Washington D.C., District of Columbia • Miami, Florida + 17 more
Source: ClinicalTrials.gov public record
Updated Jul 19, 2023 · Synced Oct 25, 2026, 3:34 PM EDT
Terminated No phase listed Observational

Genome-wide Analysis of Single Nucleotide Polymorphisms of Brain Arteriovenous Malformations and Cerebral Aneurysm

NCT01801488
View directory record Official record
Conditions
Arteriovenous Malformations, Intracranial Aneurysm, Subarachnoid Hemorrhage
Interventions
Not listed
Lead sponsor
State University of New York - Upstate Medical University
Other
Eligibility
18 Years to 89 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2011
U.S. locations
1
States / cities
Syracuse, New York
Source: ClinicalTrials.gov public record
Updated Nov 25, 2015 · Synced Oct 25, 2026, 3:34 PM EDT