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ClinicalTrials.gov public records Last synced Oct 24, 2026, 1:43 PM EDT

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Showing 769–792 of 10,802 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Developmental Defect Clear all
Active, not recruiting Phase 3 Interventional

Phase 3 Study of ALXN1850 in Treatment-Naïve Pediatric Participants With HPP

NCT06079359
View directory record Official record
Conditions
Hypophosphatasia
Interventions
ALXN1850, Placebo
Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
2 Years to 11 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2028
U.S. locations
3
States / cities
Baltimore, Maryland • Kansas City, Missouri • Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated May 28, 2026 · Synced Oct 24, 2026, 1:43 PM EDT
Completed Phase 2 Interventional Results available

A Pilot Study To Evaluate The Effects of Everolimus on Brain mTOR Activity and Cortical Hyperexcitability in TSC and FCD

NCT02451696
View directory record Official record
Conditions
Epilepsy, Tuberous Sclerosis Complex, Focal Cortical Dysplasia
Interventions
Everolimus
Drug
Lead sponsor
NYU Langone Health
Other
Eligibility
2 Years to 40 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2017
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Sep 1, 2021 · Synced Oct 24, 2026, 1:43 PM EDT
Terminated Phase 1Phase 2 Interventional Results available

Safety, Tolerability, and Pharmacokinetics of UX053 in Patients With Glycogen Storage Disease Type III (GSD III)

NCT04990388
View directory record Official record
Conditions
Glycogen Storage Disease Type III
Interventions
UX053, Placebo, Antipyretic, H2 Blocker, H1 Blocker
Biological · Other · Drug
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
18 Years and older
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
4
States / cities
Orange, California • Atlanta, Georgia • Philadelphia, Pennsylvania + 1 more
Source: ClinicalTrials.gov public record
Updated Apr 16, 2024 · Synced Oct 24, 2026, 1:43 PM EDT
Completed No phase listed Observational

Stress, Hypothalamic-pituitary-adrenal (HPA) Dysfunction, and Relapse in Alcoholism

NCT00744588
View directory record Official record
Conditions
Alcohol Dependence
Interventions
Not listed
Lead sponsor
University of Texas Southwestern Medical Center
Other
Eligibility
18 Years to 60 Years · Male only
Enrollment
75 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2012
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Jan 3, 2013 · Synced Oct 24, 2026, 1:43 PM EDT
Recruiting Phase 1 Interventional

Neonatal White Matter Injury Trial (WRAP)

NCT07688746
View directory record Official record
Conditions
White Matter Injury, Brain Injury, Fetus and Neonate, Neonatal Brain Injury, Periventricular Leukomalacia, Periventricular White Matter Abnormalities
Interventions
Clemastine fumarate
Drug
Lead sponsor
Bridget LaMonica Ostrem, M.D., Ph.D.
Other
Eligibility
3 Weeks to 20 Weeks
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2031
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Jul 28, 2026 · Synced Oct 24, 2026, 1:43 PM EDT
Not listed Phase 3 Interventional

Safety, Efficacy and Pharmacokinetics of GreenGene™ F to Previously Treated Patients With Severe Hemophilia A

NCT01619046
View directory record Official record
Conditions
Hemophilia A
Interventions
GreenGene™ F and an approved recombinant Factor VIII product, GreenGene™ F
Biological
Lead sponsor
Green Cross Corporation
Industry
Eligibility
12 Years and older
Enrollment
124 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2015
U.S. locations
10
States / cities
Little Rock, Arkansas • Los Angeles, California • Torrance, California + 7 more
Source: ClinicalTrials.gov public record
Updated Jul 3, 2014 · Synced Oct 24, 2026, 1:43 PM EDT
Active, not recruiting Phase 1 Interventional

Patch vs. No Patch Fetoscopic Meningomyelocele Repair Study

NCT03794011
View directory record Official record
Conditions
Neural Tube Defects
Interventions
fetoscopy
Device
Lead sponsor
Baylor College of Medicine
Other
Eligibility
18 Years to 64 Years · Female only
Enrollment
38 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2027
U.S. locations
2
States / cities
Stanford, California • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Oct 1, 2026 · Synced Oct 24, 2026, 1:43 PM EDT
Completed No phase listed Observational

Prospective Study of Scheuermann's Kyphosis

NCT00738309
View directory record Official record
Conditions
Scheuermann's Kyphosis
Interventions
Not listed
Lead sponsor
Setting Scoliosis Straight Foundation
Other
Eligibility
Up to 21 Years
Enrollment
147 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2013
U.S. locations
8
States / cities
Sacramento, California • San Diego, California • Wilmington, Delaware + 5 more
Source: ClinicalTrials.gov public record
Updated Apr 19, 2016 · Synced Oct 24, 2026, 1:43 PM EDT
Completed Not applicable Interventional Results available

Sedation During Muscle Biopsy in Patients With Duchenne Muscular Dystrophy

NCT01645098
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Ketamine, Dexmedetomidine
Drug
Lead sponsor
Nationwide Children's Hospital
Other
Eligibility
Male only
Enrollment
53 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2013
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Feb 26, 2015 · Synced Oct 24, 2026, 1:43 PM EDT
Active, not recruiting No phase listed Observational

A Study to Evaluate Impact of Efanesoctocog Alfa on Long-term Joint Health in Participants With Hemophilia A

NCT05911763
View directory record Official record
Conditions
Hemophilia A
Interventions
Efanesoctocog Alfa BIVV001
Drug
Lead sponsor
Sanofi
Industry
Eligibility
Not listed
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2030
U.S. locations
28
States / cities
Gilbert, Arizona • Loma Linda, California • Los Angeles, California + 23 more
Source: ClinicalTrials.gov public record
Updated Sep 29, 2026 · Synced Oct 24, 2026, 1:43 PM EDT
Completed Phase 2 Interventional

A Phase 2 Study of JNT-517 in Adolescent Participants With Phenylketonuria

NCT06637514
View directory record Official record
Conditions
Phenylketonuria (PKU)
Interventions
JNT-517 Tablet, Placebo
Drug · Other
Lead sponsor
Otsuka Pharmaceutical Development & Commercialization, Inc.
Industry
Eligibility
12 Years to 17 Years
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2026
U.S. locations
6
States / cities
Gainesville, Florida • Tampa, Florida • Portland, Oregon + 3 more
Source: ClinicalTrials.gov public record
Updated May 13, 2026 · Synced Oct 24, 2026, 1:43 PM EDT
Not listed No phase listed Observational

Improving P. Aeruginosa Detection With Breath-based Diagnostics (IMPACT-Breath)

NCT04735952
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Not listed
Lead sponsor
University of British Columbia
Other
Eligibility
3 Years and older
Enrollment
300 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2024
U.S. locations
5
States / cities
Phoenix, Arizona • Aurora, Colorado • Denver, Colorado + 2 more
Source: ClinicalTrials.gov public record
Updated Dec 5, 2023 · Synced Oct 24, 2026, 1:43 PM EDT
Completed Phase 2 Interventional Results available

Phase 2 Basket Trial of Nab-sirolimus in Patients With Malignant Solid Tumors With Pathogenic Alterations in TSC1/TSC2 Genes (PRECISION 1)

NCT05103358
View directory record Official record
Conditions
Tumor, Tumor, Solid, Metastasis, Metastatic Cancer, Cancer, Cancer Metastatic, Tumors, Neoplasms, Neoplasm Metastasis, Solid Tumor, Advanced Solid Tumor, Advanced Cancer, Malignant Solid Tumor, Malignant Solid Neoplasm, Malignant Neoplasm, Malignant Tumor, TSC, TSC1, TSC2, Metastatic Solid Tumor, Metastatic Neoplasm
Interventions
nab-sirolimus
Drug
Lead sponsor
Aadi Bioscience, Inc.
Industry
Eligibility
12 Years and older
Enrollment
128 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
159
States / cities
Birmingham, Alabama • Mobile, Alabama • Goodyear, Arizona + 137 more
Source: ClinicalTrials.gov public record
Updated Sep 28, 2026 · Synced Oct 24, 2026, 1:43 PM EDT
Completed No phase listed Observational

Screening Protocol to Evaluate Acid Alpha-Glucosidase (GAA) Activity and GAA Gene Mutations in Patients With Late Onset Pompe Disease

NCT00113035
View directory record Official record
Conditions
Pompe Disease
Interventions
Not listed
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
8 Years and older
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005
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 May 5, 2015 · Synced Oct 24, 2026, 1:43 PM EDT
Completed Phase 2 Interventional Results available

Long Term Open-label Study Evaluating Safety of Diacerein 1% Ointment Topical Formulation in Subjects With Epidermolysis Bullosa Simplex

NCT03389308
View directory record Official record
Conditions
Epidermolysis Bullosa, Epidermolysis Bullosa Simplex
Interventions
diacerein 1% ointment
Drug
Lead sponsor
Castle Creek Pharmaceuticals, LLC
Industry
Eligibility
6 Months to 99 Years
Enrollment
51 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2020
U.S. locations
3
States / cities
Chicago, Illinois • Chapel Hill, North Carolina • San Antonio, Texas
Source: ClinicalTrials.gov public record
Updated Sep 16, 2020 · Synced Oct 24, 2026, 1:43 PM EDT
Not listed No phase listed Observational Accepts healthy volunteers

Clinical Trial Readiness for SCA1 and SCA3

NCT03487367
View directory record Official record
Conditions
Spinocerebellar Ataxia Type 1, Spinocerebellar Ataxia 3
Interventions
Not listed
Lead sponsor
The Methodist Hospital Research Institute
Other
Eligibility
18 Years to 65 Years
Enrollment
200 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2023
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jan 19, 2022 · Synced Oct 24, 2026, 1:43 PM EDT
Completed Not applicable Interventional Results available

AMPLATZER Duct Occluder II Clinical Study

NCT00713700
View directory record Official record
Conditions
Patent Ductus Arteriosus
Interventions
AMPLATZER Duct Occluder II
Device
Lead sponsor
Abbott Medical Devices
Industry
Eligibility
6 Months to 18 Years
Enrollment
192 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2016
U.S. locations
25
States / cities
San Diego, California • San Francisco, California • Aurora, Colorado + 22 more
Source: ClinicalTrials.gov public record
Updated Feb 4, 2019 · Synced Oct 24, 2026, 1:43 PM EDT
Recruiting No phase listed Observational

Danon Disease Natural History Study

NCT06214507
View directory record Official record
Conditions
Danon Disease
Interventions
No intervention
Other
Lead sponsor
Rocket Pharmaceuticals Inc.
Industry
Eligibility
8 Years and older
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2030
U.S. locations
9
States / cities
Phoenix, Arizona • La Jolla, California • Aurora, Colorado + 6 more
Source: ClinicalTrials.gov public record
Updated Oct 19, 2026 · Synced Oct 24, 2026, 1:43 PM EDT
Withdrawn No phase listed Observational

Changes in Cerebral Oxygenation During Laparoscopy in Patients With Single Ventricle Anatomy

NCT02614664
View directory record Official record
Conditions
Congenital Heart Disease, Single Ventricle
Interventions
NIRS
Device
Lead sponsor
Roby Sebastian
Other
Eligibility
Not listed
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Aug 6, 2018 · Synced Oct 24, 2026, 1:43 PM EDT
Completed Phase 2 Interventional

Phase II Study to Evaluate Efficacy and Safety of D-0120 in Combination With Allopurinol in Subjects With Gout

NCT05665699
View directory record Official record
Conditions
Gout
Interventions
D-0120, Allopurinol
Drug
Lead sponsor
InventisBio Co., Ltd
Industry
Eligibility
18 Years to 75 Years
Enrollment
80 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
19
States / cities
Birmingham, Alabama • Plantation, Florida • Evergreen Park, Illinois + 16 more
Source: ClinicalTrials.gov public record
Updated Jul 9, 2026 · Synced Oct 24, 2026, 1:43 PM EDT
Completed Phase 2Phase 3 Interventional Results available

Efficacy and Safety of Inotersen in Familial Amyloid Polyneuropathy

NCT01737398
View directory record Official record
Conditions
FAP, Familial Amyloid Polyneuropathy, TTR, Transthyretin, Amyloidosis
Interventions
Inotersen, Placebo
Drug
Lead sponsor
Ionis Pharmaceuticals, Inc.
Industry
Eligibility
18 Years to 82 Years
Enrollment
173 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2017
U.S. locations
9
States / cities
Orange, California • Indianapolis, Indiana • Baltimore, Maryland + 5 more
Source: ClinicalTrials.gov public record
Updated Jul 17, 2019 · Synced Oct 24, 2026, 1:43 PM EDT
Not listed No phase listed Observational

Using Wearable and Mobile Data to Diagnose and Monitor Movement Disorders

NCT04231487
View directory record Official record
Conditions
Essential Tremor, Parkinson Disease, Huntington Disease, Dystonia, Primary, Spinocerebellar Ataxias, Movement Disorders
Interventions
Not listed
Lead sponsor
Rutgers, The State University of New Jersey
Other
Eligibility
18 Years to 80 Years
Enrollment
210 participants
Timeline
2019 – 2022
U.S. locations
1
States / cities
Newark, New Jersey
Source: ClinicalTrials.gov public record
Updated Sep 21, 2022 · Synced Oct 24, 2026, 1:43 PM EDT
Completed Phase 2 Interventional Results available

Bevacizumab for Symptomatic Vestibular Schwannoma in Neurofibromatosis Type 2 (NF2)

NCT01207687
View directory record Official record
Conditions
Vestibular Schwannoma, Neurofibromatosis Type 2
Interventions
bevacizumab, laboratory biomarker analysis, quality-of-life assessment
Biological · Other · Procedure
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
12 Years and older
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2014
U.S. locations
3
States / cities
Baltimore, Maryland • Rockville, Maryland • Charlestown, Massachusetts
Source: ClinicalTrials.gov public record
Updated Aug 27, 2018 · Synced Oct 24, 2026, 1:43 PM EDT
Completed Phase 1 Interventional

Hydroxyurea and Erythropoietin to Treat Sickle Cell Anemia

NCT00270478
View directory record Official record
Conditions
Sickle Cell Disease, Chronic Kidney Disease, Pulmonary Hypertension
Interventions
Erythropoietin and Hydroxyurea
Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
18 Years and older
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2009
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Dec 17, 2019 · Synced Oct 24, 2026, 1:43 PM EDT