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ClinicalTrials.gov public records Last synced Oct 28, 2026, 5:05 PM EDT

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Showing 1561–1584 of 10,812 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Developmental Defect Clear all
Recruiting No phase listed Observational Accepts healthy volunteers

The HOPE Biobank Resource (BMT CTN 2402 HOPE)

NCT07227155
View directory record Official record
Conditions
Aplastic Anemias, Hemoglobinopathies, Bone Marrow Failure
Interventions
Not listed
Lead sponsor
Medical College of Wisconsin
Other
Eligibility
Not listed
Enrollment
375 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2026 – 2031
U.S. locations
14
States / cities
Duarte, California • Gainesville, Florida • Atlanta, Georgia + 10 more
Source: ClinicalTrials.gov public record
Updated Aug 27, 2026 · Synced Oct 28, 2026, 5:05 PM EDT
Terminated Phase 3 Interventional Results available

Opioid Reduction Initiative During Outpatient Pediatric Urologic Procedures Using Exparel

NCT04826484
View directory record Official record
Conditions
Urologic Diseases, Hypospadias, Undescended Testes, Chordee, Hydrocele, Orchiectomy
Interventions
Exparel 133 miligrams per 10 milliliter injection, Bupivacaine Hydrochloride
Drug
Lead sponsor
Johns Hopkins University
Other
Eligibility
6 Years and older
Enrollment
104 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Dec 26, 2023 · Synced Oct 28, 2026, 5:05 PM EDT
Completed Phase 1 Interventional

Follistatin Gene Transfer to Patients With Becker Muscular Dystrophy and Sporadic Inclusion Body Myositis

NCT01519349
View directory record Official record
Conditions
Becker Muscular Dystrophy, Sporadic Inclusion Body Myositis
Interventions
rAAV1.CMV.huFollistatin344
Biological
Lead sponsor
Nationwide Children's Hospital
Other
Eligibility
18 Years and older
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2017
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Oct 2, 2023 · Synced Oct 28, 2026, 5:05 PM EDT
Completed Phase 1 Interventional

Decitabine and Valproic Acid in Treating Patients With Refractory or Relapsed Acute Myeloid Leukemia or Previously Treated Chronic Lymphocytic Leukemia or Small Lymphocytic Lymphoma

NCT00079378
View directory record Official record
Conditions
Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Del(5q), Adult Acute Myeloid Leukemia With Inv(16)(p13;q22), Adult Acute Myeloid Leukemia With t(15;17)(q22;q12), Adult Acute Myeloid Leukemia With t(16;16)(p13;q22), Adult Acute Myeloid Leukemia With t(8;21)(q22;q22), Recurrent Adult Acute Myeloid Leukemia, Recurrent Small Lymphocytic Lymphoma, Refractory Chronic Lymphocytic Leukemia, Untreated Adult Acute Myeloid Leukemia
Interventions
decitabine, valproic acid, pharmacological study, laboratory biomarker analysis
Drug · Other
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years and older
Enrollment
84 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2004
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Sep 30, 2013 · Synced Oct 28, 2026, 5:05 PM EDT
Not listed Not applicable Interventional Accepts healthy volunteers

ABL90 FP MC Neonatal Arterial, Venous, Capillary Blood

NCT06078943
View directory record Official record
Conditions
Congenital Heart Disease
Interventions
method comparison
Device
Lead sponsor
Radiometer Medical ApS
Industry
Eligibility
1 Day to 28 Days
Enrollment
612 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2023 – 2024
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Oct 12, 2023 · Synced Oct 28, 2026, 5:05 PM EDT
Completed Phase 3 Interventional Results available

Study to Assess the Efficacy, Safety, and Tolerability of Intravitreal Aflibercept Compared to Laser Photocoagulation in Patients With Retinopathy of Prematurity

NCT04101721
View directory record Official record
Conditions
Retinopathy of Prematurity
Interventions
aflibercept, laser photocoagulation
Drug · Procedure
Lead sponsor
Regeneron Pharmaceuticals
Industry
Eligibility
Not listed
Enrollment
127 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
27
States / cities
Phoenix, Arizona • La Jolla, California • Loma Linda, California + 21 more
Source: ClinicalTrials.gov public record
Updated Jul 20, 2023 · Synced Oct 28, 2026, 5:05 PM EDT
Recruiting Phase 1Phase 2 Interventional

Safety and Efficacy of Mutation-targeted Precision Genetic Therapy for Ataxia-Telangiectasia (A-T)

NCT07215416
View directory record Official record
Conditions
Ataxia Telangiectasia
Interventions
Antisense oligonucleotide targeting the ATM gene
Drug
Lead sponsor
Timothy Yu
Other
Eligibility
0 Years to 17 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2036
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated May 28, 2026 · Synced Oct 28, 2026, 5:05 PM EDT
Completed Phase 1 Interventional

"Safety, Tolerability and Pharmacokinetics of MP-376 Administered for 14 Days to Stable Pediatric (CF) Patients"

NCT00840333
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
MP-376 (Levofloxacin solution for Inhalation)
Drug
Lead sponsor
Amgen
Industry
Eligibility
6 Years to 16 Years
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009
U.S. locations
6
States / cities
Mobile, Alabama • San Diego, California • Orlando, Florida + 3 more
Source: ClinicalTrials.gov public record
Updated Dec 4, 2024 · Synced Oct 28, 2026, 5:05 PM EDT
Terminated No phase listed Observational

CEC/EPC and Cardiovascular Risk in Renal Transplant Recipients

NCT00365833
View directory record Official record
Conditions
Transplantation, Kidney, Cardiovascular Abnormalities, Cardiovascular Diseases
Interventions
Not listed
Lead sponsor
University of Florida
Other
Eligibility
18 Years to 80 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2007
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Sep 30, 2011 · Synced Oct 28, 2026, 5:05 PM EDT
Completed Not applicable Interventional

Timing of Delivery in Fetal Growth Restriction of Uncomplicated Women

NCT03046355
View directory record Official record
Conditions
Fetal Growth Restriction
Interventions
Labor induction at 37.0 to 37.6 weeks of gestation, Expectant monitoring until delivery
Procedure
Lead sponsor
The University of Texas Health Science Center, Houston
Other
Eligibility
18 Years to 39 Years · Female only
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2019
U.S. locations
3
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jun 13, 2019 · Synced Oct 28, 2026, 5:05 PM EDT
Enrolling by invitation No phase listed Observational

Long-term Follow-up Study of Male Adults With Hemophilia B Previously Treated With Etranacogene Dezaparvovec (CSL222)

NCT05962398
View directory record Official record
Conditions
Hemophilia B
Interventions
AAV5-hFIXco-Padua
Genetic
Lead sponsor
CSL Behring
Industry
Eligibility
18 Years and older · Male only
Enrollment
56 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2035
U.S. locations
15
States / cities
Phoenix, Arizona • Little Rock, Arkansas • Los Angeles, California + 11 more
Source: ClinicalTrials.gov public record
Updated Jul 18, 2025 · Synced Oct 28, 2026, 5:05 PM EDT
Terminated Early Phase 1 Interventional

Gene Therapy for Chronic Granulomatous Disease

NCT00394316
View directory record Official record
Conditions
Chronic Granulomatous Disease
Interventions
Phagocyte Oxidase Subunit Transduced CD34 Hematopoietic Stem Cells
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
3 Years to 55 Years · Male only
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2014
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 5, 2018 · Synced Oct 28, 2026, 5:05 PM 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 28, 2026, 5:05 PM EDT
Terminated No phase listed Observational

Pulmonary Resistance in Fontan Who Have Undergone Heart Transplantation

NCT00268047
View directory record Official record
Conditions
Congenital Disorders
Interventions
Not listed
Lead sponsor
Children's Healthcare of Atlanta
Other
Eligibility
Up to 21 Years
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Jul 6, 2011 · Synced Oct 28, 2026, 5:05 PM EDT
Completed Phase 3 Interventional Results available

A Study of Lanadelumab to Prevent Hereditary Angioedema (HAE) Attacks in Children

NCT04070326
View directory record Official record
Conditions
Hereditary Angioedema
Interventions
Lanadelumab
Drug
Lead sponsor
Shire
Industry
Eligibility
2 Years to 11 Years
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
11
States / cities
Santa Monica, California • Walnut Creek, California • Centennial, Colorado + 8 more
Source: ClinicalTrials.gov public record
Updated May 26, 2022 · Synced Oct 28, 2026, 5:05 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A Study to Assess the Pharmacokinetics and Safety of Co-administered Oral Galicaftor, Navocaftor, and ABBV-576 in Healthy Adults for the Treatment of Cystic Fibrosis

NCT05530278
View directory record Official record
Conditions
Healthy Volunteers
Interventions
Galicaftor, ABBV-576, Navocaftor, Midazolam
Drug
Lead sponsor
AbbVie
Industry
Eligibility
18 Years to 65 Years · Female only
Enrollment
24 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022
U.S. locations
4
States / cities
Anaheim, California • Miami, Florida • Las Vegas, Nevada + 1 more
Source: ClinicalTrials.gov public record
Updated Feb 21, 2023 · Synced Oct 28, 2026, 5:05 PM EDT
Active, not recruiting Phase 2 Interventional

Choline Supplementation as a Neurodevelopmental Intervention in Fetal Alcohol Spectrum Disorders Study

NCT05108974
View directory record Official record
Conditions
Fetal Alcohol Spectrum Disorders
Interventions
Choline Bitartrate
Drug
Lead sponsor
Jeff Wozniak
Other
Eligibility
30 Months to 72 Months
Enrollment
62 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2027
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jul 8, 2026 · Synced Oct 28, 2026, 5:05 PM EDT
Completed No phase listed Observational

Journey of Patients With Vasculitis From First Symptom to Diagnosis

NCT03410290
View directory record Official record
Conditions
Vasculitis, Systemic Vasculitis, Behcet's Disease, CNS Vasculitis, Cryoglobulinemic Vasculitis, Eosinophilic Granulomatous Vasculitis, Temporal Arteritis, Giant Cell Arteritis, Granulomatosis With Polyangiitis, Wegener Granulomatosis, Henoch Schonlein Purpura, IgA Vasculitis, Microscopic Polyangiitis, Polyarteritis Nodosa, Takayasu Arteritis, Urticarial Vasculitis
Interventions
Online Questionnaire
Other
Lead sponsor
University of Pennsylvania
Other
Eligibility
Not listed
Enrollment
456 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018
U.S. locations
1
States / cities
Tampa, Florida
Source: ClinicalTrials.gov public record
Updated Jun 6, 2018 · Synced Oct 28, 2026, 5:05 PM EDT
Recruiting No phase listed Observational

Natural History of MADD

NCT07734090
View directory record Official record
Conditions
Multiple Acyl-CoA Dehydrogenase Deficiency
Interventions
Not listed
Lead sponsor
Icahn School of Medicine at Mount Sinai
Other
Eligibility
Not listed
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2031
U.S. locations
2
States / cities
Aurora, Colorado • New York, New York
Source: ClinicalTrials.gov public record
Updated Sep 16, 2026 · Synced Oct 28, 2026, 5:05 PM EDT
Completed No phase listed Observational

Disability Severity Scale (DSI) and Hereditary Motor and Sensory Neuropathy Overall Disability Scale (HMSN-R-ODS)

NCT02194010
View directory record Official record
Conditions
Charcot Marie Tooth Disease, Inherited Peripheral Neuropathy
Interventions
Not listed
Lead sponsor
University of Iowa
Other
Eligibility
10 Years and older
Enrollment
563 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2019
U.S. locations
2
States / cities
Iowa City, Iowa • Rochester, New York
Source: ClinicalTrials.gov public record
Updated Feb 7, 2019 · Synced Oct 28, 2026, 5:05 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

An Open-label, Phase 1/2 Trial of Gene Therapy 4D-310 in Adults With Fabry Disease

NCT04519749
View directory record Official record
Conditions
Fabry Disease
Interventions
4D-310
Biological
Lead sponsor
4D Molecular Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2030
U.S. locations
4
States / cities
La Jolla, California • Atlanta, Georgia • Pittsburgh, Pennsylvania + 1 more
Source: ClinicalTrials.gov public record
Updated Apr 8, 2024 · Synced Oct 28, 2026, 5:05 PM EDT
Terminated Phase 2 Interventional Results available

A Study Evaluating if Pridopidine is Safe, Efficacious, and Tolerable in Patients With Huntington's Disease

NCT02494778
View directory record Official record
Conditions
Huntington's Disease
Interventions
Pridopidine
Drug
Lead sponsor
Prilenia
Industry
Eligibility
21 Years and older
Enrollment
248 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2018
U.S. locations
11
States / cities
Los Angeles, California • Englewood, Colorado • Washington D.C., District of Columbia + 8 more
Source: ClinicalTrials.gov public record
Updated Sep 17, 2021 · Synced Oct 28, 2026, 5:05 PM EDT
Completed Not applicable Interventional

SMART Mobile Application Technology Utilization in the Treatment of Sickle Cell Disease Post Day Hospital Discharge

NCT02475993
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
SMART app
Other
Lead sponsor
Duke University
Other
Eligibility
18 Years and older
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Oct 18, 2019 · Synced Oct 28, 2026, 5:05 PM EDT
Completed Phase 2Phase 3 Interventional Results available

A Study to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Participants With Friedreich Ataxia

NCT04577352
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
Vatiquinone, Placebo
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
7 Years and older
Enrollment
146 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2023
U.S. locations
5
States / cities
Los Angeles, California • Gainesville, Florida • Tampa, Florida + 2 more
Source: ClinicalTrials.gov public record
Updated Apr 13, 2026 · Synced Oct 28, 2026, 5:05 PM EDT