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ClinicalTrials.gov public records Last synced Oct 15, 2026, 9:47 AM EDT

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

Showing 601–624 of 10,531 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Congenital Diseases Clear all
Not listed Not applicable Interventional

Investigation of Supplemental L-alanine in the Management of Dietary Fructose Intolerance

NCT04022434
View directory record Official record
Conditions
Fructose Intolerance
Interventions
Placedo, Alanine
Dietary Supplement
Lead sponsor
Augusta University
Other
Eligibility
18 Years and older
Enrollment
65 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2020
U.S. locations
1
States / cities
Augusta, Georgia
Source: ClinicalTrials.gov public record
Updated Apr 15, 2020 · Synced Oct 15, 2026, 9:47 AM EDT
Completed Phase 3 Interventional Results available

Vincristine, Dactinomycin, and Doxorubicin With or Without Radiation Therapy or Observation Only in Treating Younger Patients Who Are Undergoing Surgery for Newly Diagnosed Stage I, Stage II, or Stage III Wilms' Tumor

NCT00352534
View directory record Official record
Conditions
Stage I Kidney Wilms Tumor, Stage II Kidney Wilms Tumor, Stage III Kidney Wilms Tumor
Interventions
3-Dimensional Conformal Radiation Therapy, Biospecimen Collection, Chest Radiography, Computed Tomography, Dactinomycin, Doxorubicin Hydrochloride, Echocardiography Test, Magnetic Resonance Imaging, Therapeutic Conventional Surgery, Ultrasound Imaging, Vincristine Sulfate
Radiation · Procedure · Biological + 1 more
Lead sponsor
Children's Oncology Group
Network
Eligibility
Up to 29 Years
Enrollment
808 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2026
U.S. locations
210
States / cities
Birmingham, Alabama • Mobile, Alabama • Phoenix, Arizona + 150 more
Source: ClinicalTrials.gov public record
Updated Jun 3, 2026 · Synced Oct 15, 2026, 9:47 AM EDT
Terminated Phase 2 Interventional Results available

A Study to Test the Long-term Safety of BI 655130 in Patients With Atopic Eczema Who Took Part in Study 1368-0032

NCT04086121
View directory record Official record
Conditions
Dermatitis, Atopic
Interventions
Spesolimab
Drug
Lead sponsor
Boehringer Ingelheim
Industry
Eligibility
18 Years to 75 Years
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
7
States / cities
Scottsdale, Arizona • Fort Myers, Florida • Miami, Florida + 4 more
Source: ClinicalTrials.gov public record
Updated Feb 24, 2025 · Synced Oct 15, 2026, 9:47 AM EDT
Completed Phase 3 Interventional Results available

Open-Label Safety, PK, and Efficacy Trial of Sebetralstat (KVD900) in Pediatric Patients (Ages 2-11) With HAE Type I or II

NCT06467084
View directory record Official record
Conditions
Hereditary Angioedema
Interventions
KVD900 150 mg, KVD900 300 mg, KVD900 600 mg
Drug
Lead sponsor
KalVista Pharmaceuticals, Ltd.
Industry
Eligibility
2 Years to 11 Years
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
10
States / cities
Birmingham, Alabama • Scottsdale, Arizona • San Diego, California + 7 more
Source: ClinicalTrials.gov public record
Updated Aug 5, 2026 · Synced Oct 15, 2026, 9:47 AM EDT
Completed Phase 2 Interventional

Pilot Study For Hypothermia Treatment In Hyperammonemic Encephalopathy In Neonates And Very Young Infants

NCT01624311
View directory record Official record
Conditions
Urea Cycle Disorders, Organic Acidemias
Interventions
Therapeutic Hypothermia, Standard of Care Therapy
Other
Lead sponsor
Uta Lichter-Konecki
Other
Eligibility
1 Day to 30 Days
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2015
U.S. locations
3
States / cities
Washington D.C., District of Columbia • New York, New York • Milwaukee, Wisconsin
Source: ClinicalTrials.gov public record
Updated Jun 1, 2015 · Synced Oct 15, 2026, 9:47 AM EDT
Recruiting Not applicable Interventional

Deep Brain Stimulation Therapy in Movement Disorders

NCT02119611
View directory record Official record
Conditions
Parkinson's Disease
Interventions
Deep Brain Stimulation management
Device
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
18 Years to 100 Years
Enrollment
300 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2030
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 8, 2026 · Synced Oct 15, 2026, 9:47 AM EDT
Completed Phase 2 Interventional

Safety and Efficacy of EPI-743 in Patients With Friedreich's Ataxia

NCT01728064
View directory record Official record
Conditions
Friedreich's Ataxia
Interventions
Placebo, EPI-743 400 mg, EPI-743 200 mg
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
18 Years to 45 Years
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2016
U.S. locations
3
States / cities
Los Angeles, California • Tampa, Florida • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Aug 31, 2020 · Synced Oct 15, 2026, 9:47 AM EDT
Completed Phase 2Phase 3 Interventional Results available

Sildenafil Exercise: Role of PDE5 Inhibition

NCT04039087
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Sildenafil 40mg oral capsule, Placebo Oral capsule
Drug
Lead sponsor
National Jewish Health
Other
Eligibility
9 Years and older
Enrollment
31 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2024
U.S. locations
2
States / cities
Denver, Colorado • Augusta, Georgia
Source: ClinicalTrials.gov public record
Updated Aug 28, 2026 · Synced Oct 15, 2026, 9:47 AM EDT
Completed Phase 2 Interventional

Lymphocyte Infusion in Treating Patients With Relapsed Cancer After Bone Marrow or Peripheral Stem Cell Transplantation

NCT00003887
View directory record Official record
Conditions
Breast Cancer, Chronic Myeloproliferative Disorders, Gestational Trophoblastic Tumor, Kidney Cancer, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Neuroblastoma, Ovarian Cancer, Sarcoma, Testicular Germ Cell Tumor
Interventions
peripheral blood lymphocyte therapy
Biological
Lead sponsor
Fred Hutchinson Cancer Center
Other
Eligibility
1 Year to 70 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
1998 – 2003
U.S. locations
1
States / cities
Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Nov 30, 2011 · Synced Oct 15, 2026, 9:47 AM EDT
Terminated Not applicable Interventional

Customized Orthosis for Children With Clubfoot

NCT03853811
View directory record Official record
Conditions
Congenital Talipes Equinovarus
Interventions
Ankle Foot Orthosis (AFO)
Device
Lead sponsor
Medical College of Wisconsin
Other
Eligibility
Up to 12 Months
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
1
States / cities
Milwaukee, Wisconsin
Source: ClinicalTrials.gov public record
Updated Apr 11, 2022 · Synced Oct 15, 2026, 9:47 AM EDT
Not listed Not applicable Interventional

Blood Conservation in Patients (3.5-12kg) Undergoing Congenital Cardiac Surgery

NCT05881564
View directory record Official record
Conditions
Congenital Heart Disease
Interventions
blood use for priming cardiopulmonary bypass circuit, clear prime for cardiopulmonary bypass
Other
Lead sponsor
Augusta University
Other
Eligibility
Not listed
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2026
U.S. locations
1
States / cities
Augusta, Georgia
Source: ClinicalTrials.gov public record
Updated Jun 1, 2023 · Synced Oct 15, 2026, 9:47 AM EDT
Completed Not applicable Interventional

Impact of an Orthotic Intervention in Children With Peripheral Neuropathy

NCT03655587
View directory record Official record
Conditions
Gait, Drop Foot, Chemotherapy-induced Peripheral Neuropathy, Acute Lymphocytic Leukemia, Pediatric, Rhabdomyosarcoma, Wilms Tumor
Interventions
Solid ankle foot orthotic, Resting night splint
Device
Lead sponsor
Children's Hospitals and Clinics of Minnesota
Other
Eligibility
5 Years to 14 Years
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2022
U.S. locations
2
States / cities
Minneapolis, Minnesota • Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Feb 10, 2023 · Synced Oct 15, 2026, 9:47 AM EDT
Terminated No phase listed Observational

Kidney Information Network for Disease Research and Education

NCT03321604
View directory record Official record
Conditions
ESRD, Fabry Disease, Rare Diseases, Kidney Diseases, End Stage Renal Disease, Genetic Diseases, X-Linked
Interventions
Not listed
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
18 Years to 65 Years
Enrollment
338 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2019
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Feb 24, 2021 · Synced Oct 15, 2026, 9:47 AM EDT
Completed Phase 2 Interventional Results available

Can Ondansetron Prevent Neonatal Abstinence Syndrome (NAS) in Babies Born to Narcotic-dependent Women

NCT01965704
View directory record Official record
Conditions
Narcotic Addiction, Neonatal Abstinence Syndrome
Interventions
Ondansetron, Placebo
Drug
Lead sponsor
Stanford University
Other
Eligibility
Up to 45 Years
Enrollment
196 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2020
U.S. locations
8
States / cities
San Francisco, California • San Jose, California • Stanford, California + 5 more
Source: ClinicalTrials.gov public record
Updated Jun 7, 2022 · Synced Oct 15, 2026, 9:47 AM EDT
Terminated Phase 2 Interventional Results available

Celecoxib for Thyroid Eye Disease

NCT02845336
View directory record Official record
Conditions
Thyroid Eye Disease
Interventions
Celecoxib, artificial tears
Drug
Lead sponsor
Johns Hopkins University
Other
Eligibility
18 Years and older
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2019
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 31, 2020 · Synced Oct 15, 2026, 9:47 AM EDT
Completed No phase listed Observational Results available

An Observational Study Evaluating Anti-Idursulfase Serum Antibody Response in Hunter Syndrome Patients

NCT00882921
View directory record Official record
Conditions
Hunter Syndrome
Interventions
Idursulfase
Biological
Lead sponsor
Shire
Industry
Eligibility
5 Years and older · Male only
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2013
U.S. locations
2
States / cities
Oakland, California • Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jun 8, 2021 · Synced Oct 15, 2026, 9:47 AM EDT
Completed Phase 2 Interventional Results available

A Gene Therapy Study for Hemophilia B

NCT02484092
View directory record Official record
Conditions
Hemophilia B
Interventions
SPK-9001
Biological
Lead sponsor
Pfizer
Industry
Eligibility
18 Years and older · Male only
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2019
U.S. locations
9
States / cities
Sacramento, California • Jackson, Mississippi • Madison, Mississippi + 2 more
Source: ClinicalTrials.gov public record
Updated Jun 16, 2020 · Synced Oct 15, 2026, 9:47 AM EDT
Completed Phase 4 Interventional

TESTO: Testosterone Effects on Short-Term Outcomes in Infants With XXY

NCT03325647
View directory record Official record
Conditions
Klinefelter Syndrome
Interventions
Testosterone Cypionate 200 Milligram/Milliliter Injectable Solution, Placebo injectable saline
Drug
Lead sponsor
University of Colorado, Denver
Other
Eligibility
31 Days to 90 Days · Male only
Enrollment
72 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2021
U.S. locations
1
States / cities
Aurora, Colorado
Source: ClinicalTrials.gov public record
Updated May 6, 2022 · Synced Oct 15, 2026, 9:47 AM EDT
Completed Phase 1 Interventional

Donor Mesenchymal Stem Cell Infusion in Treating Patients With Acute or Chronic Graft-Versus-Host Disease After Undergoing a Donor Stem Cell Transplant

NCT00361049
View directory record Official record
Conditions
Cancer
Interventions
graft versus host disease prophylaxis/therapy, fluorescence in situ hybridization, immunoenzyme technique, immunohistochemistry staining method, laboratory biomarker analysis, in vitro-treated bone marrow transplantation, management of therapy complications
Biological · Genetic · Other + 1 more
Lead sponsor
Case Comprehensive Cancer Center
Other
Eligibility
18 Years and older
Enrollment
49 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2010
U.S. locations
8
States / cities
Cleveland, Ohio
Source: ClinicalTrials.gov public record
Updated Nov 5, 2010 · Synced Oct 15, 2026, 9:47 AM EDT
Completed Phase 3 Interventional Results available

A Study of PTC923 in Participants With Phenylketonuria

NCT05099640
View directory record Official record
Conditions
Phenylketonuria
Interventions
PTC923, Placebo
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
Not listed
Enrollment
157 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
12
States / cities
Stanford, California • Aurora, Colorado • Gainesville, Florida + 9 more
Source: ClinicalTrials.gov public record
Updated Jan 10, 2024 · Synced Oct 15, 2026, 9:47 AM EDT
Active, not recruiting Phase 3 Interventional

Evaluation of Maralixibat in Pruritus Associated With General Cholestatic Liver Disease (EXPAND)

NCT06553768
View directory record Official record
Conditions
Cholestatic Liver Disease (Except ALGS, PFIC, PBC and PSC)
Interventions
Maralixibat, Placebo
Drug · Other
Lead sponsor
Mirum Pharmaceuticals, Inc.
Industry
Eligibility
6 Months and older
Enrollment
90 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
8
States / cities
Los Angeles, California • Palo Alto, California • Chicago, Illinois + 4 more
Source: ClinicalTrials.gov public record
Updated Oct 6, 2026 · Synced Oct 15, 2026, 9:47 AM EDT
Completed No phase listed Observational

Natural History of Patients With PH3 and a History of Stone Events

NCT04542590
View directory record Official record
Conditions
Primary Hyperoxaluria Type 3
Interventions
Not listed
Lead sponsor
Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
Industry
Eligibility
2 Years and older
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
2
States / cities
Boston, Massachusetts • New York, New York
Source: ClinicalTrials.gov public record
Updated Dec 2, 2024 · Synced Oct 15, 2026, 9:47 AM EDT
Recruiting Phase 2 Interventional

Phase 2 Study to Assess the Safety and Efficacy of ANG003

NCT07450547
View directory record Official record
Conditions
Exocrine Pancreatic Insufficiency (EPI), Cystic Fibrosis (CF)
Interventions
ANG003 Dose A, ANG003 Dose B, Creon
Drug
Lead sponsor
Anagram Therapeutics, Inc.
Industry
Eligibility
12 Years and older
Enrollment
113 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2027
U.S. locations
27
States / cities
Long Beach, California • Los Angeles, California • Denver, Colorado + 23 more
Source: ClinicalTrials.gov public record
Updated Oct 6, 2026 · Synced Oct 15, 2026, 9:47 AM EDT
Terminated Not applicable Interventional

Osteoporosis and MRI Study in Hemophilia

NCT01460147
View directory record Official record
Conditions
Bone Mineral Density, Hemophilia A, Hemophilia B
Interventions
DXA scan + MRI
Other
Lead sponsor
Baxalta now part of Shire
Industry
Eligibility
25 Years and older · Male only
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2012
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Apr 22, 2021 · Synced Oct 15, 2026, 9:47 AM EDT