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ClinicalTrials.gov public records Last synced Oct 25, 2026, 1:09 PM EDT

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Showing 985–1008 of 10,802 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Developmental Defect Clear all
Completed Phase 1Phase 2 Interventional Results available

Study of Vitamin D in Children With Sickle Cell Disease

NCT01276587
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Vitamin D3
Drug
Lead sponsor
Gary M Brittenham, MD
Other
Eligibility
3 Years to 20 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Apr 12, 2023 · Synced Oct 25, 2026, 1:09 PM EDT
Completed Not applicable Interventional Results available

Medication Adherence in Children, Adolescents and Adults With Neurofibromatosis Type 1(NF1) on Clinical Treatment Trials

NCT03531814
View directory record Official record
Conditions
Neurofibromatosis 1, Neurofibroma, Plexiform
Interventions
Medication Event Monitoring System (MEMS^TM), Questionnaires
Behavioral · Other
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
3 Years to 59 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2023
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jun 11, 2024 · Synced Oct 25, 2026, 1:09 PM EDT
Completed Not applicable Interventional

Improving Therapeutic Patient Education for Atopic Dermatitis: Evaluation of a Parent Handbook

NCT03722706
View directory record Official record
Conditions
Dermatitis, Atopic
Interventions
Educational handbook
Other
Lead sponsor
Boston Children's Hospital
Other
Eligibility
30 Days to 16 Years
Enrollment
246 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2020
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jul 17, 2020 · Synced Oct 25, 2026, 1:09 PM EDT
Withdrawn Phase 2 Interventional

IV Acetaminophen in Children Undergoing Palatoplasty

NCT01760330
View directory record Official record
Conditions
Cleft Palate
Interventions
IV acetaminophen, Placebo
Drug
Lead sponsor
Nationwide Children's Hospital
Other
Eligibility
Not listed
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2016
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Mar 8, 2016 · Synced Oct 25, 2026, 1:09 PM EDT
Completed Phase 4 Interventional Results available

Efficacy, Safety and Tolerability of Icatibant for the Treatment of HAE

NCT01457430
View directory record Official record
Conditions
Hereditary Angioedema
Interventions
Icatibant
Drug
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
18 Years and older
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2014
U.S. locations
5
States / cities
La Jolla, California • Los Angeles, California • Wheaton, Maryland + 2 more
Source: ClinicalTrials.gov public record
Updated Oct 18, 2016 · Synced Oct 25, 2026, 1:09 PM EDT
Recruiting No phase listed Observational

Non-Invasive Detection of Right-to-Left Cardiac Shunts

NCT07420400
View directory record Official record
Conditions
Patent Foramen Ovale (PFO)
Interventions
Point-of-care ultrasound
Diagnostic Test
Lead sponsor
Scripps Health
Other
Eligibility
18 Years and older
Enrollment
75 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2027
U.S. locations
1
States / cities
La Jolla, California
Source: ClinicalTrials.gov public record
Updated Feb 19, 2026 · Synced Oct 25, 2026, 1:09 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Prophylactic Continuous Positive Airway Pressure (CPAP) in Delivery Room, in the Prevention of Transient Tachypnea of Newborn (TTN): A Randomized Controlled Trial

NCT01082900
View directory record Official record
Conditions
Transient Tachypnea
Interventions
Prophylactic CPAP Administration
Other
Lead sponsor
New York Hospital Queens
Other
Eligibility
2 Minutes to 30 Minutes
Enrollment
138 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2013
U.S. locations
1
States / cities
Flushing, New York
Source: ClinicalTrials.gov public record
Updated Oct 6, 2015 · Synced Oct 25, 2026, 1:09 PM EDT
Not listed No phase listed Observational

Natural History Study in Huntington Disease Gene Expansion Carriers (HDGECs) - SHIELD HD

NCT04406636
View directory record Official record
Conditions
Huntington Disease
Interventions
Not listed
Lead sponsor
CHDI Foundation, Inc.
Other
Eligibility
18 Years to 63 Years
Enrollment
70 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2023
U.S. locations
5
States / cities
San Diego, California • Englewood, Colorado • Boston, Massachusetts + 2 more
Source: ClinicalTrials.gov public record
Updated Sep 23, 2022 · Synced Oct 25, 2026, 1:09 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 25, 2026, 1:09 PM EDT
Completed Phase 1 Interventional

Repeated Infusions of Mesenchymal Stromal Cells in Children With Osteogenesis Imperfecta

NCT01061099
View directory record Official record
Conditions
Osteogenesis Imperfecta Type II, Osteogenesis Imperfecta Type III
Interventions
Mesenchymal Stromal Cells
Biological
Lead sponsor
Nationwide Children's Hospital
Other
Eligibility
Up to 19 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2014
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 24, 2015 · Synced Oct 25, 2026, 1:09 PM EDT
Active, not recruiting Phase 2Phase 3 Interventional

Open Label Extension to Assess the Long-Term Safety and Tolerability of ZYN002 in Children and Adolescents With FXS

NCT03802799
View directory record Official record
Conditions
Fragile X Syndrome
Interventions
ZYN002 - Cannabidiol Transdermal Gel
Drug
Lead sponsor
Zynerba Pharmaceuticals, Inc.
Industry
Eligibility
3 Years to 29 Years
Enrollment
476 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2026
U.S. locations
19
States / cities
Phoenix, Arizona • Culver City, California • Irvine, California + 16 more
Source: ClinicalTrials.gov public record
Updated May 27, 2026 · Synced Oct 25, 2026, 1:09 PM EDT
Completed No phase listed Observational

Developing the Family Map: Looking at Communal Coping

NCT01633021
View directory record Official record
Conditions
Sickle Cell, Diabetes, Cancer, Cardiovascular Disease, Genetic Screening
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
18 Years to 100 Years
Enrollment
1,061 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2012
U.S. locations
3
States / cities
Bethesda, Maryland • Cincinnati, Ohio • Lewisburg, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Oct 2, 2026 · Synced Oct 25, 2026, 1:09 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers

3D Modalities for Medical Student Education of Congenital Heart Disease

NCT07295730
View directory record Official record
Conditions
Tetralogy of Fallot (TOF), Hypoplastic Left Heart Syndrome (HLHS)
Interventions
3D-printed heart models and augmented reality
Other
Lead sponsor
Case Western Reserve University
Other
Eligibility
18 Years and older
Enrollment
26 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2025
U.S. locations
1
States / cities
Cleveland, Ohio
Source: ClinicalTrials.gov public record
Updated Dec 26, 2025 · Synced Oct 25, 2026, 1:09 PM EDT
Recruiting Phase 2 Interventional

Human Craniomaxillofacial Allotransplantation

NCT01889381
View directory record Official record
Conditions
Facial Injuries, Traumatic Wounds and Injuries, Craniofacial Injuries, Craniofacial Defects, Facial Transplantation, Facial Deformity
Interventions
Bone marrow cell-based therapy & 1-drug immunosuppression.
Drug
Lead sponsor
Johns Hopkins University
Other
Eligibility
18 Years to 65 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2041
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 25, 2026 · Synced Oct 25, 2026, 1:09 PM EDT
Completed Not applicable Interventional

The VetSeq Study: a Pilot Study of Genome Sequencing in Veteran Care

NCT03380819
View directory record Official record
Conditions
Rare Diseases, Genetic Disease
Interventions
Genome sequencing
Diagnostic Test
Lead sponsor
VA Boston Healthcare System
Federal
Eligibility
Not listed
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2022
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Apr 26, 2023 · Synced Oct 25, 2026, 1:09 PM EDT
Terminated No phase listed Observational

Clinical Survey Study to Assess Physical Function and the Incidence of Hypoglycemia in Participants With Glycogen Storage Disease Type III

NCT05196165
View directory record Official record
Conditions
Glycogen Storage Disease Type III
Interventions
No Intervention
Other
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
1 Year and older
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2023
U.S. locations
3
States / cities
Philadelphia, Pennsylvania • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Sep 28, 2023 · Synced Oct 25, 2026, 1:09 PM EDT
Not listed Not applicable Interventional

Stem Cell Ophthalmology Treatment Study

NCT01920867
View directory record Official record
Conditions
Retinal Disease, Macular Degeneration, Hereditary Retinal Dystrophy, Optic Nerve Disease, Glaucoma
Interventions
RB (Retrobulbar), ST (Subtenon), IV (Intravenous), IVIT (Intravitreal), IO (Intraocular)
Procedure
Lead sponsor
MD Stem Cells
Industry
Eligibility
18 Years and older
Enrollment
300 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2020
U.S. locations
1
States / cities
Westport, Connecticut
Source: ClinicalTrials.gov public record
Updated Oct 23, 2019 · Synced Oct 25, 2026, 1:09 PM EDT
Not listed Phase 1Phase 2 Interventional

CD34+ (Malignant) Stem Cell Selection for Patients Receiving Allogenic Stem Cell Transplant

NCT02061800
View directory record Official record
Conditions
Chronic Myeloid Leukemia (CML), Acute Myelogenous Leukemia (AML), Myelodysplastic Syndrome (MDS), Juvenile Myelomonocytic Leukemia (JMML), Acute Lymphoblastic Leukemia (ALL), Lymphoma (Hodgkin's and Non-Hodgkin's)
Interventions
CliniMACS CD34+ Reagent System, Thiotepa, Cyclophosphamide, Alemtuzumab, Tacrolimus, Melphalan, Busulfan, Fludarabine, Methylprednisolone
Device · Drug
Lead sponsor
Diane George
Other
Eligibility
Up to 22 Years
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2026
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jun 4, 2024 · Synced Oct 25, 2026, 1:09 PM EDT
Completed No phase listed Observational

Neurobehavioral Phenotypes in MPS III

NCT01873911
View directory record Official record
Conditions
Sanfilippo Syndrome Type A, Sanfilippo Syndrome Type B, Hurler Syndrome
Interventions
Not listed
Lead sponsor
University of Minnesota
Other
Eligibility
2 Years to 12 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2014
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Nov 1, 2019 · Synced Oct 25, 2026, 1:09 PM EDT
Completed Not applicable Interventional

The Effects of Lutein and Zeaxanthin Supplementation on Vision in Patients With Albinism

NCT02200263
View directory record Official record
Conditions
Ocular Albinism (OA), Oculocutaneous Albinism (OCA)
Interventions
Lutein plus Zeaxanthin, Placebo
Dietary Supplement
Lead sponsor
Johns Hopkins University
Other
Eligibility
12 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2018
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Dec 28, 2018 · Synced Oct 25, 2026, 1:09 PM EDT
Completed Phase 2Phase 3 Interventional Results available

Study of Recombinant Factor IX Product, IB1001, in Subjects With Hemophilia B

NCT00768287
View directory record Official record
Conditions
Hemophilia B
Interventions
IB1001, nonacog alfa
Biological
Lead sponsor
Medexus Pharma, Inc.
Industry
Eligibility
5 Years and older
Enrollment
77 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2016
U.S. locations
10
States / cities
Duarte, California • Los Angeles, California • Atlanta, Georgia + 7 more
Source: ClinicalTrials.gov public record
Updated Apr 6, 2021 · Synced Oct 25, 2026, 1:09 PM EDT
Completed No phase listed Observational

Prognostic Markers of Inflammation in Infants Undergoing Cardiopulmonary Bypass

NCT03143348
View directory record Official record
Conditions
Congenital Heart Defect, Cardiopulmonary Bypass, Inflammatory Response, Low Cardiac Output Syndrome, Inflammation
Interventions
Single blood draw, Multiple blood draws
Procedure
Lead sponsor
University of Oklahoma
Other
Eligibility
Up to 6 Months
Enrollment
38 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2019
U.S. locations
1
States / cities
Oklahoma City, Oklahoma
Source: ClinicalTrials.gov public record
Updated Apr 30, 2019 · Synced Oct 25, 2026, 1:09 PM EDT
Completed Phase 2 Interventional Results available

Stem Cell Transplant w/Laronidase for Hurler

NCT00176891
View directory record Official record
Conditions
Mucopolysaccharidosis I, Hurler Syndrome
Interventions
Stem Cell Transplant, Laronidase ERT
Procedure · Drug
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 7 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2012
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jul 11, 2019 · Synced Oct 25, 2026, 1:09 PM EDT
Completed Phase 2Phase 3 Interventional Results available

Azacitidine With or Without Nivolumab or Midostaurin, or Decitabine and Cytarabine Alone in Treating Older Patients With Newly Diagnosed Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome

NCT03092674
View directory record Official record
Conditions
Acute Myeloid Leukemia, Myelodysplastic Syndrome, Myelodysplastic Syndrome/Acute Myeloid Leukemia
Interventions
Azacitidine, Cytarabine, Decitabine, Laboratory Biomarker Analysis, Midostaurin, Nivolumab
Drug · Other · Biological
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
60 Years and older
Enrollment
76 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2024
U.S. locations
506
States / cities
Anchorage, Alaska • Tucson, Arizona • Hot Springs, Arkansas + 337 more
Source: ClinicalTrials.gov public record
Updated Jan 16, 2025 · Synced Oct 25, 2026, 1:09 PM EDT