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ClinicalTrials.gov public records Last synced Oct 15, 2026, 1:03 AM EDT

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

Showing 505–528 of 10,529 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Congenital Diseases Clear all
Not yet recruiting Phase 2 Interventional

Evaluating the Efficacy and Safety of D-galactose in PGM1-CDG (AVTX-801)

NCT05402332
View directory record Official record
Conditions
PGM1-CDG - Phosphoglucomutase 1-Related Congenital Disorder of Glycosylation
Interventions
AVTX-801, Placebo
Drug
Lead sponsor
Eva Morava-Kozicz
Other
Eligibility
18 Years to 60 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Mar 16, 2026 · Synced Oct 15, 2026, 1:03 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers Results available

Using Visual Arrays to Support Understanding of Genetic Risk

NCT06994832
View directory record Official record
Conditions
Decision Aid
Interventions
Visual Array, Narrative Visual Array, Choice, Enhanced Choice
Behavioral
Lead sponsor
Stephanie B. Seminara, MD
Other
Eligibility
18 Years and older
Enrollment
1,045 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2025
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Sep 24, 2026 · Synced Oct 15, 2026, 1:03 AM EDT
Completed No phase listed Observational

Clinical and Molecular Correlations in Spinocerebellar Ataxia Type 10 (SCA10)

NCT00004306
View directory record Official record
Conditions
Hereditary Ataxia
Interventions
Not listed
Lead sponsor
Office of Rare Diseases (ORD)
NIH
Eligibility
3 Years and older
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1999 – 2009
U.S. locations
1
States / cities
Galveston, Texas
Source: ClinicalTrials.gov public record
Updated Mar 6, 2012 · Synced Oct 15, 2026, 1:03 AM EDT
Not listed No phase listed Observational Accepts healthy volunteers

Non-Invasive Determination of Fetal Chromosome Abnormalities

NCT00891852
View directory record Official record
Conditions
Down Syndrome (Trisomy 21), Edward's Syndrome (Trisomy 18), Patau Syndrome (Trisomy 13), Klinefelter Syndrome (47, XXY), and Other Chromosome, Abnormalities.
Interventions
Not listed
Lead sponsor
Lenetix Medical Screening Laboratory
Industry
Eligibility
18 Years and older · Female only
Enrollment
1,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009
U.S. locations
7
States / cities
Rockville, Maryland • Moristown, New Jersey • Mineola, New York + 3 more
Source: ClinicalTrials.gov public record
Updated May 1, 2009 · Synced Oct 15, 2026, 1:03 AM EDT
Active, not recruiting Phase 3 Interventional Results available

HELIOS-B: A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy

NCT04153149
View directory record Official record
Conditions
Transthyretin Amyloidosis (ATTR) With Cardiomyopathy
Interventions
Vutrisiran, Sterile Normal Saline (0.9% NaCl)
Drug
Lead sponsor
Alnylam Pharmaceuticals
Industry
Eligibility
18 Years to 85 Years
Enrollment
655 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2026
U.S. locations
17
States / cities
La Mesa, California • Los Angeles, California • Stanford, California + 13 more
Source: ClinicalTrials.gov public record
Updated Jan 12, 2026 · Synced Oct 15, 2026, 1:03 AM EDT
Not listed Phase 4 Interventional

Intra-tracheal Instillation of Budesonide to Prevent Chronic Lung Disease

NCT05364385
View directory record Official record
Conditions
Infant, Extremely Premature, Acute Lung Injury, Chronic Lung Disease of Prematurity, Budesonide
Interventions
Infants in the study group (S/B group) will receive surfactant 2.5 mls/kg and Budesonide 0.5 mg (1mL), Infants in the control group (S/P group) receive placebo (Normal saline 1mL)
Drug
Lead sponsor
University of Arizona
Other
Eligibility
Up to 18 Hours
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2024
U.S. locations
1
States / cities
Tucson, Arizona
Source: ClinicalTrials.gov public record
Updated Nov 1, 2022 · Synced Oct 15, 2026, 1:03 AM EDT
Terminated Phase 2Phase 3 Interventional Results available

The BROADEN Study: A Study of Volanesorsen (Formerly IONIS-APOCIIIRx) in Participants With Familial Partial Lipodystrophy

NCT02527343
View directory record Official record
Conditions
Familial Partial Lipodystrophy
Interventions
volanesorsen, Placebo
Drug
Lead sponsor
Akcea Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2019
U.S. locations
6
States / cities
Ann Arbor, Michigan • Rochester, Minnesota • St Louis, Missouri + 3 more
Source: ClinicalTrials.gov public record
Updated Oct 18, 2021 · Synced Oct 15, 2026, 1:03 AM EDT
Completed No phase listed Observational

Umbilical Cord Blood Proteomic Analysis and Neonatal Brain Injury

NCT00375908
View directory record Official record
Conditions
Hypoxia-Ischemia, Brain, Periventricular Leukomalacia
Interventions
Not listed
Lead sponsor
Johns Hopkins University
Other
Eligibility
Not listed
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2008
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 1, 2015 · Synced Oct 15, 2026, 1:03 AM EDT
Active, not recruiting Phase 3 Interventional

Clinical Study for Treatment-naïve IOPD Babies to Evaluate Efficacy and Safety of ERT With Avalglucosidase Alfa

NCT04910776
View directory record Official record
Conditions
Glycogen Storage Disease Type II
Interventions
avalglucosidase alfa
Drug
Lead sponsor
Sanofi
Industry
Eligibility
0 Days to 12 Months
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2027
U.S. locations
6
States / cities
Stanford, California • Minneapolis, Minnesota • Hawthorne, New York + 3 more
Source: ClinicalTrials.gov public record
Updated Apr 8, 2026 · Synced Oct 15, 2026, 1:03 AM EDT
Completed Phase 3 Interventional Results available

Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With DMD (RACER53)

NCT04060199
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Viltolarsen, Placebo
Drug
Lead sponsor
NS Pharma, Inc.
Industry
Eligibility
4 Years to 7 Years · Male only
Enrollment
77 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2023
U.S. locations
2
States / cities
Sacramento, California • Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Dec 11, 2024 · Synced Oct 15, 2026, 1:03 AM EDT
Completed Phase 2 Interventional Results available

A Phase 2, Multicenter, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy, Safety, and Tolerability of TAK-935 (OV935) as an Adjunctive Therapy in Pediatric Participants With Developmental and/or Epileptic Encephalopathies

NCT03650452
View directory record Official record
Conditions
Epilepsy, Dravet Syndrome, Lennox-Gastaut Syndrome
Interventions
TAK-935, Placebo
Drug
Lead sponsor
Takeda
Industry
Eligibility
2 Years to 17 Years
Enrollment
141 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2020
U.S. locations
15
States / cities
Phoenix, Arizona • Los Angeles, California • Aurora, Colorado + 12 more
Source: ClinicalTrials.gov public record
Updated Sep 14, 2026 · Synced Oct 15, 2026, 1:03 AM EDT
Not listed Phase 3 Interventional

Phase III Trial Assessing the Efficacy and Safety of PXT3003 in CMT1A Patients

NCT04762758
View directory record Official record
Conditions
Charcot-Marie-Tooth Disease
Interventions
(RS)-baclofen, naltrexone hydrochloride and D-sorbitol, Placebo
Drug
Lead sponsor
Pharnext S.C.A.
Other
Eligibility
16 Years to 65 Years
Enrollment
350 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
20
States / cities
Los Angeles, California • Sacramento, California • New Britain, Connecticut + 16 more
Source: ClinicalTrials.gov public record
Updated Apr 3, 2024 · Synced Oct 15, 2026, 1:03 AM EDT
Completed Phase 1 Interventional

A Study of RG1662 in Individuals With Down Syndrome

NCT01436955
View directory record Official record
Conditions
Down Syndrome
Interventions
Placebo, RG1662
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
18 Years to 30 Years
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2013
U.S. locations
8
States / cities
Phoenix, Arizona • Little Rock, Arkansas • La Jolla, California + 5 more
Source: ClinicalTrials.gov public record
Updated Mar 3, 2015 · Synced Oct 15, 2026, 1:03 AM EDT
Active, not recruiting Phase 2Phase 3 Interventional

A Study to Evaluate Efficacy, Safety, Tolerability and Exposure After a Repeat-dose of Sepofarsen (QR-110) in LCA10 (ILLUMINATE)

NCT03913143
View directory record Official record
Conditions
Leber Congenital Amaurosis 10, Blindness, Leber Congenital Amaurosis, Vision Disorders, Sensation Disorders, Neurologic Manifestations, Eye Diseases, Eye Diseases, Hereditary, Eye Disorders Congenital, Retinal Disease
Interventions
sepofarsen, Sham
Drug · Other
Lead sponsor
ProQR Therapeutics
Industry
Eligibility
8 Years and older
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2023
U.S. locations
1
States / cities
Iowa City, Iowa
Source: ClinicalTrials.gov public record
Updated Mar 17, 2022 · Synced Oct 15, 2026, 1:03 AM EDT
Active, not recruiting Phase 3 Interventional

Roll-over Study to Collect and Assess Long-term Safety of Everolimus in Patients With TSC and Refractory Seizures Who Have Completed the EXIST-3 Study [CRAD001M2304] and Who Are Benefitting From Continued Treatment

NCT02962414
View directory record Official record
Conditions
Tuberous Sclerosis Complex
Interventions
everolimus
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
2 Years to 65 Years
Enrollment
206 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2026
U.S. locations
14
States / cities
Phoenix, Arizona • Los Angeles, California • Oakland, California + 11 more
Source: ClinicalTrials.gov public record
Updated Aug 21, 2026 · Synced Oct 15, 2026, 1:03 AM EDT
Completed Not applicable Interventional

Improving Critical Congenital Heart Disease Screening With Addition of Perfusion Measurements

NCT04056104
View directory record Official record
Conditions
Congenital Heart Disease
Interventions
SpO2 and PIx Measurement
Diagnostic Test
Lead sponsor
University of California, Davis
Other
Eligibility
Up to 21 Days
Enrollment
543 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
6
States / cities
Los Angeles, California • Sacramento, California • San Francisco, California + 2 more
Source: ClinicalTrials.gov public record
Updated Feb 3, 2022 · Synced Oct 15, 2026, 1:03 AM EDT
Withdrawn Not applicable Interventional Accepts healthy volunteers

Evaluating Skin Barrier Dysfunction in Infants at High Risk of Atopy

NCT03089476
View directory record Official record
Conditions
Atopic Dermatitis, Eczema, Food Allergy
Interventions
Evaluating atopy in infants, Evaluating TEWL and STS in adults
Other
Lead sponsor
National Jewish Health
Other
Eligibility
34 Weeks to 50 Years
Healthy volunteers
Accepts healthy volunteers
Timeline
2017 – 2018
U.S. locations
1
States / cities
Denver, Colorado
Source: ClinicalTrials.gov public record
Updated Jun 12, 2018 · Synced Oct 15, 2026, 1:03 AM EDT
Not listed Not applicable Interventional

Safety and Efficacy Study of the Draeger Babylog VN500 Device in HFOV Mode in VLBW Neonates

NCT02445040
View directory record Official record
Conditions
Respiratory Distress Syndrome In Premature Infants
Interventions
Babylog VN500 in HFOV Mode
Device
Lead sponsor
Draeger Medical Systems, Inc.
Industry
Eligibility
23 Weeks to 30 Weeks
Enrollment
225 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2020
U.S. locations
14
States / cities
Little Rock, Arkansas • San Diego, California • Iowa City, Iowa + 9 more
Source: ClinicalTrials.gov public record
Updated Sep 19, 2019 · Synced Oct 15, 2026, 1:03 AM EDT
Completed Phase 4 Interventional Accepts healthy volunteers Results available

Continuous Versus Cyclical OCP Use in PCOS

NCT03819140
View directory record Official record
Conditions
Polycystic Ovary Syndrome
Interventions
Yasmin
Drug
Lead sponsor
University of California, San Francisco
Other
Eligibility
15 Years to 40 Years · Female only
Enrollment
51 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2022
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Nov 14, 2025 · Synced Oct 15, 2026, 1:03 AM EDT
Recruiting No phase listed Observational

Specimen Collection From Pregnant Women at Increased Risk for Fetal Aneuploidy

NCT01429389
View directory record Official record
Conditions
Down Syndrome, Fetal Aneuploidy
Interventions
Not listed
Lead sponsor
Sequenom, Inc.
Industry
Eligibility
18 Years and older · Female only
Enrollment
2,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2028
U.S. locations
12
States / cities
Birmingham, Alabama • Los Angeles, California • San Diego, California + 6 more
Source: ClinicalTrials.gov public record
Updated Sep 9, 2026 · Synced Oct 15, 2026, 1:03 AM EDT
Recruiting Phase 1Phase 2 Interventional

A Safety and Efficacy of Intrathecally Administered ION283 in Patients With Lafora Disease

NCT06609889
View directory record Official record
Conditions
Lafora Disease
Interventions
ION283
Genetic
Lead sponsor
Berge Minassian
Other
Eligibility
10 Years to 18 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2028
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Dec 18, 2025 · Synced Oct 15, 2026, 1:03 AM EDT
Recruiting Phase 1Phase 2 Interventional

A Study Assessing HMB-002 in Participants With Von Willebrand Disease

NCT06754852
View directory record Official record
Conditions
Von Willebrand Disease (VWD), Von Willebrand Disease (VWD), Type 1, Von Willebrand Disease (VWD), Type 2, Von Willebrand Disease (VWD), Type 3
Interventions
HMB-002 (Part A), HMB-002 (Part B), HMB-002 with Concomitant Factor Concentrate (Part C) (Not Applicable in US)
Drug
Lead sponsor
Hemab ApS
Industry
Eligibility
16 Years to 69 Years
Enrollment
108 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
13
States / cities
Phoenix, Arizona • Little Rock, Arkansas • Los Angeles, California + 10 more
Source: ClinicalTrials.gov public record
Updated Jun 30, 2026 · Synced Oct 15, 2026, 1:03 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Postnatal Choline Supplementation in Children With Prenatal Alcohol Exposure

NCT01149538
View directory record Official record
Conditions
Fetal Alcohol Spectrum Disorders, Fetal Alcohol Syndrome, Partial Fetal Alcohol Syndrome, Alcohol Related Neurodevelopmental Disorder, Prenatal Alcohol Exposure
Interventions
Choline bitartrate, Placebo for choline bitartrate
Drug · Dietary Supplement
Lead sponsor
University of Minnesota
Other
Eligibility
2 Years to 5 Years
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2014
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Dec 19, 2016 · Synced Oct 15, 2026, 1:03 AM EDT
Recruiting Phase 3 Interventional

Study to Evaluate Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Type 10 (HYPERION)

NCT06891443
View directory record Official record
Conditions
Leber Congenital Amaurosis 10, Blindness, Leber Congenital Amaurosis, Sensation Disorders, Vision Disorder, Neurological Manifestations, Eye Diseases, Hereditary, Eye Diseases, Eye Disorders Congenital, Retinal Disease
Interventions
sepofarsen, Placebo IVT
Drug · Other
Lead sponsor
Laboratoires Thea
Industry
Eligibility
6 Years and older
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
5
States / cities
San Francisco, California • Miami, Florida • Iowa City, Iowa + 2 more
Source: ClinicalTrials.gov public record
Updated Jun 25, 2026 · Synced Oct 15, 2026, 1:03 AM EDT