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ClinicalTrials.gov public records Last synced Oct 17, 2026, 12:22 PM EDT

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

Showing 1033–1056 of 10,533 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Congenital Diseases Clear all
Recruiting No phase listed Observational

Non-interventional Study of Patients With Transthyretin (ATTR) Amyloidosis

NCT06465810
View directory record Official record
Conditions
Transthyretin Amyloidosis, ATTR-CM, ATTRv-PN, ATTR, ATTR-Mixed, hATTR
Interventions
Treatment of transthyretin (ATTR) amyloidosis in observational study setting
Drug
Lead sponsor
AstraZeneca
Industry
Eligibility
18 Years to 130 Years
Enrollment
1,850 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2031
U.S. locations
33
States / cities
La Jolla, California • Los Angeles, California • San Francisco, California + 24 more
Source: ClinicalTrials.gov public record
Updated Sep 23, 2026 · Synced Oct 17, 2026, 12:22 PM EDT
Terminated Phase 3 Interventional

Moxifloxacin in Preventing Bacterial Infections in Patients Who Have Undergone Donor Stem Cell Transplant

NCT00324324
View directory record Official record
Conditions
Breast Cancer, Chronic Myeloproliferative Disorders, Gestational Trophoblastic Tumor, Infection, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasms, Neuroblastoma, Ovarian Cancer, Testicular Germ Cell Tumor
Interventions
moxifloxacin hydrochloride, Placebo
Drug
Lead sponsor
OHSU Knight Cancer Institute
Other
Eligibility
18 Years to 120 Years
Enrollment
240 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2012
U.S. locations
1
States / cities
Portland, Oregon
Source: ClinicalTrials.gov public record
Updated May 9, 2017 · Synced Oct 17, 2026, 12:22 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A Study to Compare the Relative Bioavailability of Two Iberdomide (CC-220) Formulations and to Assess The Effect Of Food on the Drug Levels of Iberdomide in Healthy Adult Participants

NCT05899738
View directory record Official record
Conditions
Healthy Volunteers
Interventions
Iberdomide
Drug
Lead sponsor
Bristol-Myers Squibb
Industry
Eligibility
18 Years to 65 Years
Enrollment
32 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2023
U.S. locations
1
States / cities
Daytona Beach, Florida
Source: ClinicalTrials.gov public record
Updated Jul 26, 2024 · Synced Oct 17, 2026, 12:22 PM EDT
Recruiting Phase 4 Interventional

Dupilumab De-escalation in Pediatric Atopic Dermatitis

NCT06116526
View directory record Official record
Conditions
Atopic Dermatitis
Interventions
Dupilumab - Discontinuation, Dupilumab - Dose Reduction, Dupilumab - Standard Dose
Drug
Lead sponsor
Johns Hopkins University
Other
Eligibility
1 Year to 17 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Apr 20, 2026 · Synced Oct 17, 2026, 12:22 PM EDT
Recruiting Phase 2Phase 3 Interventional

Alpelisib in Pediatric and Adult Patients With Lymphatic Malformations Associated With a PIK3CA Mutation.

NCT05948943
View directory record Official record
Conditions
Lymphatic Malformations
Interventions
Alpelisib, Placebo
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
0 Years to 100 Years
Enrollment
232 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2033
U.S. locations
18
States / cities
Oakland, California • Orange, California • Palo Alto, California + 13 more
Source: ClinicalTrials.gov public record
Updated Oct 9, 2026 · Synced Oct 17, 2026, 12:22 PM EDT
Completed Phase 3 Interventional

A Study to Evaluate the Efficacy and Safety of Subcutaneous Amlitelimab Monotherapy Compared With Placebo in Participants Aged 12 Years and Older With Moderate-to-severe Atopic Dermatitis (COAST 2)

NCT06181435
View directory record Official record
Conditions
Dermatitis Atopic
Interventions
Amlitelimab, Placebo
Drug
Lead sponsor
Sanofi
Industry
Eligibility
12 Years and older
Enrollment
589 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2026
U.S. locations
43
States / cities
Scottsdale, Arizona • Tucson, Arizona • Lomita, California + 38 more
Source: ClinicalTrials.gov public record
Updated Aug 13, 2026 · Synced Oct 17, 2026, 12:22 PM EDT
Not listed No phase listed Observational

The Effect of Bethanechol on Tracheobronchomalacia

NCT05299008
View directory record Official record
Conditions
Tracheobronchomalacia
Interventions
Bethanechol
Drug
Lead sponsor
Arkansas Children's Hospital Research Institute
Other
Eligibility
Not listed
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
1
States / cities
Little Rock, Arkansas
Source: ClinicalTrials.gov public record
Updated Jan 30, 2024 · Synced Oct 17, 2026, 12:22 PM EDT
Completed Phase 4 Interventional Results available

The Efficacy and Population Pharmacokinetics of Tranexamic Acid for Craniosynostosis Surgery

NCT02188576
View directory record Official record
Conditions
Craniosynostosis
Interventions
high dose TXA, Low dose TXA
Drug
Lead sponsor
Boston Children's Hospital
Other
Eligibility
3 Months to 6 Years
Enrollment
66 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2018
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jan 31, 2020 · Synced Oct 17, 2026, 12:22 PM EDT
Terminated Phase 1Phase 2 Interventional

A Multiple Ascending Dose Study of ACN00177 (Pegtarviliase) in Subjects With CBS Deficiency

NCT05154890
View directory record Official record
Conditions
Homocystinuria Due to Cystathionine Beta-Synthase Deficiency
Interventions
Pegtarviliase IV, Pegtarviliase SC
Drug
Lead sponsor
Aeglea Biotherapeutics
Industry
Eligibility
12 Years and older
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Jul 27, 2023 · Synced Oct 17, 2026, 12:22 PM EDT
Completed Phase 1 Interventional

Study of Human Central Nervous System (CNS) Stem Cells Transplantation in Pelizaeus-Merzbacher Disease (PMD) Subjects

NCT01005004
View directory record Official record
Conditions
Pelizaeus-Merzbacher Disease
Interventions
HuCNS-SC cells
Biological
Lead sponsor
StemCells, Inc.
Industry
Eligibility
6 Months to 5 Years · Male only
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2012
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Jan 15, 2015 · Synced Oct 17, 2026, 12:22 PM EDT
Completed Phase 3 Interventional Results available

A Study of VX-121 Combination Therapy in Participants With Cystic Fibrosis (CF) Who Are Homozygous for F508del, Heterozygous for F508del and a Gating (F/G) or Residual Function (F/RF) Mutation, or Have At Least 1 Other Triple Combination Responsive (TCR) CFTR Mutation and No F508del Mutation

NCT05076149
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
VX-121/TEZ/D-IVA, ELX/TEZ/IVA, IVA, Placebo (matched to VX-121/TEZ/D-IVA), Placebo (matched to ELX/TEZ/IVA), Placebo (matched to IVA)
Drug
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
12 Years and older
Enrollment
597 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
62
States / cities
Birmingham, Alabama • Tucson, Arizona • Little Rock, Arkansas + 55 more
Source: ClinicalTrials.gov public record
Updated Jun 13, 2024 · Synced Oct 17, 2026, 12:22 PM EDT
Not yet recruiting Phase 3 Interventional

Safety and Efficacy of Fetoscopic Endoluminal Tracheal Occlusion in Congenital Diaphragmatic Hernia

NCT06946576
View directory record Official record
Conditions
Congenital Diaphragmatic Hernia
Interventions
Fetoscopic Endoluminal Tracheal Occlusion (FETO) Procedure
Device
Lead sponsor
Oregon Health and Science University
Other
Eligibility
18 Years and older · Female only
Enrollment
80 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2030
U.S. locations
1
States / cities
Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Sep 2, 2025 · Synced Oct 17, 2026, 12:22 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Weigh Easy: Simplifying Home Weight Monitoring for Infants

NCT04384276
View directory record Official record
Conditions
Cleft Lip and Palate
Interventions
The Weigh Easy System, Retrospective Control
Other
Lead sponsor
Children's Mercy Hospital Kansas City
Other
Eligibility
Up to 12 Weeks
Enrollment
157 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2022
U.S. locations
1
States / cities
Kansas City, Missouri
Source: ClinicalTrials.gov public record
Updated Jan 17, 2023 · Synced Oct 17, 2026, 12:22 PM EDT
Completed Phase 2 Interventional Results available

A Study of Tanezumab in Adults With Chronic Low Back Pain

NCT00876187
View directory record Official record
Conditions
Low Back Pain
Interventions
Tanezumab 20 mg IV, Placebo for naproxen, Tanezumab 10 mg IV, Tanezumab 5 mg IV, Placebo for tanezumab, Naproxen
Biological · Drug
Lead sponsor
Pfizer
Industry
Eligibility
18 Years and older
Enrollment
1,359 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2011
U.S. locations
135
States / cities
Anniston, Alabama • Birmingham, Alabama • Hueytown, Alabama + 101 more
Source: ClinicalTrials.gov public record
Updated Jul 7, 2021 · Synced Oct 17, 2026, 12:22 PM EDT
Completed Phase 3 Interventional

Evaluation of Repeat Administration of Purified Poloxamer 188

NCT02449616
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
MST-188
Drug
Lead sponsor
Mast Therapeutics, Inc.
Industry
Eligibility
4 Years to 65 Years
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2015
U.S. locations
9
States / cities
San Diego, California • Fort Myers, Florida • Hollywood, Florida + 6 more
Source: ClinicalTrials.gov public record
Updated Oct 28, 2016 · Synced Oct 17, 2026, 12:22 PM EDT
Not listed Phase 2 Interventional

Study of Eblasakimab in Male or Female Moderate-to-Severe Atopic Dermatitis Patients Previously Treated With Dupilumab

NCT05694884
View directory record Official record
Conditions
Atopic Dermatitis
Interventions
Placebo, ASLAN004
Drug
Lead sponsor
ASLAN Pharmaceuticals
Other
Eligibility
18 Years and older
Enrollment
75 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
24
States / cities
Birmingham, Alabama • Encino, California • Fountain Valley, California + 20 more
Source: ClinicalTrials.gov public record
Updated Jan 18, 2024 · Synced Oct 17, 2026, 12:22 PM EDT
Completed Not applicable Interventional

Endoscopic Goniotomy for Infantile Glaucoma

NCT00338533
View directory record Official record
Conditions
Glaucoma
Interventions
endoscopic goniotomy
Device
Lead sponsor
Vanderbilt University
Other
Eligibility
1 Week to 3 Years
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1997 – 2007
U.S. locations
1
States / cities
Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Jan 8, 2013 · Synced Oct 17, 2026, 12:22 PM EDT
Terminated Not applicable Interventional

Evaluating the Effect of Bathing Additives in Atopic Dermatitis

NCT04001855
View directory record Official record
Conditions
Atopic Dermatitis, Eczema
Interventions
Dilute vinegar (acetic acid), Dilute bleach
Other
Lead sponsor
University of Arizona
Other
Eligibility
5 Years and older
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2020
U.S. locations
1
States / cities
Tucson, Arizona
Source: ClinicalTrials.gov public record
Updated Jun 4, 2021 · Synced Oct 17, 2026, 12:22 PM EDT
Active, not recruiting Phase 3 Interventional Results available

Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis

NCT00999609
View directory record Official record
Conditions
Inherited Retinal Dystrophy Due to RPE65 Mutations, Leber Congenital Amaurosis
Interventions
AAV2-hRPE65v2,voretigene neparvovec-rzyl
Biological
Lead sponsor
Spark Therapeutics, Inc.
Industry
Eligibility
3 Years and older
Enrollment
31 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2030
U.S. locations
2
States / cities
Iowa City, Iowa • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 23, 2025 · Synced Oct 17, 2026, 12:22 PM EDT
Completed No phase listed Observational

Study of the Experiences and Needs of Parents Continuing a Pregnancy Following a Prenatal Diagnosis of Holopresencephaly

NCT00005016
View directory record Official record
Conditions
Holoprosencephaly
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
Not listed
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2001
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 17, 2026, 12:22 PM EDT
Completed Phase 3 Interventional Results available

Study to Investigate Immunogenicity, Efficacy and Safety of Treatment With Human-cl rhFVIII

NCT01992549
View directory record Official record
Conditions
Severe Hemophilia A
Interventions
Human-cl rhFVIII
Biological
Lead sponsor
Octapharma
Industry
Eligibility
Male only
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2018
U.S. locations
1
States / cities
Sacramento, California
Source: ClinicalTrials.gov public record
Updated Jan 19, 2021 · Synced Oct 17, 2026, 12:22 PM EDT
Completed Phase 2 Interventional Results available

B244 Topical Spray for the Treatment of Pruritus in Adults With a History of Atopic Dermatitis

NCT04490109
View directory record Official record
Conditions
Atopic Dermatitis, Pruritus
Interventions
B244, Vehicle
Biological
Lead sponsor
AOBiome LLC
Industry
Eligibility
18 Years to 65 Years
Enrollment
547 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2022
U.S. locations
56
States / cities
Birmingham, Alabama • Phoenix, Arizona • Scottsdale, Arizona + 50 more
Source: ClinicalTrials.gov public record
Updated Jan 28, 2025 · Synced Oct 17, 2026, 12:22 PM EDT
Terminated Phase 2 Interventional Results available

Effects of Perioperative Nesiritide or Milrinone Infusion on Recovery From Fontan Surgery

NCT00543309
View directory record Official record
Conditions
Heart Defects, Congenital
Interventions
nesiritide, milrinone, placebo
Drug
Lead sponsor
John M Costello
Other
Eligibility
Not listed
Enrollment
106 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2013
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jun 25, 2018 · Synced Oct 17, 2026, 12:22 PM EDT
Withdrawn Early Phase 1 Interventional

Topical and Local TXA in Facelifts - A Randomized Controlled Double Blinded Study

NCT06345833
View directory record Official record
Conditions
Hemophilia, Hemorrhage, Facelift Surgery
Interventions
1%Tranexamic acid with standard local, 3% TXA, 1% TXA with local plus 3% TXA-soaked pledgets
Drug
Lead sponsor
University of Minnesota
Other
Eligibility
18 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jul 23, 2026 · Synced Oct 17, 2026, 12:22 PM EDT