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ClinicalTrials.gov public records Last synced Nov 2, 2026, 11:22 AM EST

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Showing 97–120 of 11,249 matching trials from the live ClinicalTrials.gov search.
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Recruiting Phase 1 Interventional

Feasibility and Tolerability of IMLYGIC for the Treatment of Cutaneous Neurofibromas in Adults With NF1

NCT07102394
View directory record Official record
Conditions
NF1, Neurofibromatosis, Cutaneous Neurofibroma, Neurofibroma, Von Recklinghausen Disease, Neurofibromas
Interventions
Imlygic
Drug
Lead sponsor
Johns Hopkins University
Other
Eligibility
18 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2027
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated May 8, 2026 · Synced Nov 2, 2026, 11:22 AM EST
Completed Phase 3 Interventional Results available

Efficacy and Safety of Circadin® in the Treatment of Sleep Disturbances in Children With Neurodevelopment Disabilities

NCT01906866
View directory record Official record
Conditions
Sleep Disorders
Interventions
Circadin 2/5/10 mg, Placebo
Drug
Lead sponsor
Neurim Pharmaceuticals Ltd.
Industry
Eligibility
2 Years to 17 Years
Enrollment
125 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2018
U.S. locations
18
States / cities
Phoenix, Arizona • Miami Lakes, Florida • Orange City, Florida + 14 more
Source: ClinicalTrials.gov public record
Updated Apr 23, 2024 · Synced Nov 2, 2026, 11:22 AM EST
Completed Phase 2 Interventional Results available

Everolimus for Treatment of Disfiguring Cutaneous Lesions in Neurofibromatosis1 CRAD001CUS232T

NCT02332902
View directory record Official record
Conditions
Neurofibromatosis 1
Interventions
Everolimus
Drug
Lead sponsor
The University of Texas Health Science Center, Houston
Other
Eligibility
18 Years and older
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2016
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Dec 20, 2023 · Synced Nov 2, 2026, 11:22 AM EST
Completed Phase 2 Interventional Results available

Donor Peripheral Stem Cell Transplant, Fludarabine, and Busulfan in Treating Patients With Hematologic Cancers

NCT00619645
View directory record Official record
Conditions
Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes
Interventions
busulfan, cyclosporine, fludarabine phosphate, mycophenolate mofetil, allogeneic hematopoietic stem cell transplantation, peripheral blood stem cell transplantation
Drug · Procedure
Lead sponsor
University of California, Davis
Other
Eligibility
18 Years to 120 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2013
U.S. locations
1
States / cities
Sacramento, California
Source: ClinicalTrials.gov public record
Updated Jan 10, 2018 · Synced Nov 2, 2026, 11:22 AM EST
Recruiting Phase 1 Interventional

Inhaled Ciclesonide Study in Preterm Infants

NCT06589245
View directory record Official record
Conditions
Bronchopulmonary Dysplasia
Interventions
Alvesco Inhalant Product
Drug
Lead sponsor
Venkatesh Sampath
Other
Eligibility
8 Days to 35 Days
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
Kansas City, Missouri
Source: ClinicalTrials.gov public record
Updated Sep 9, 2026 · Synced Nov 2, 2026, 11:22 AM EST
Active, not recruiting Phase 1Phase 2 Interventional

A Study to Evaluate the Safety of AB-1003 (Previously LION-101) in Subjects With Genetic Confirmation of LGMD2I/R9 (Part1)

NCT05230459
View directory record Official record
Conditions
Limb Girdle Muscular Dystrophy, Limb-Girdle Muscular Dystrophy Type 2, LGMD2I, Muscular Dystrophy, LGMD2, LGMD, FKRP, FKRP Mutation, Fukutin Related Protein
Interventions
AB-1003 dose level 1, AB-1003 dose level 2, Placebo
Genetic · Drug
Lead sponsor
AskBio Inc
Industry
Eligibility
18 Years to 65 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2032
U.S. locations
6
States / cities
Irvine, California • Iowa City, Iowa • Kansas City, Kansas + 3 more
Source: ClinicalTrials.gov public record
Updated Aug 26, 2026 · Synced Nov 2, 2026, 11:22 AM EST
Recruiting Phase 2 Interventional

Study of Safety & PK of Luspatercept (ACE-536) in Pediatric Participants With Beta (β)-Thalassemia

NCT04143724
View directory record Official record
Conditions
Beta-Thalassemia
Interventions
ACE-536
Drug
Lead sponsor
Celgene
Industry
Eligibility
6 Years to 17 Years
Enrollment
99 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2035
U.S. locations
2
States / cities
Los Angeles, California • New York, New York
Source: ClinicalTrials.gov public record
Updated Jul 10, 2026 · Synced Nov 2, 2026, 11:22 AM EST
Recruiting No phase listed Observational

Orphan Europe Carbaglu® Surveillance Protocol

NCT03409003
View directory record Official record
Conditions
N-acetylglutamate Synthase (NAGS) Deficiency
Interventions
Not listed
Lead sponsor
Nicholas Ah Mew
Other
Eligibility
Not listed
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2027
U.S. locations
3
States / cities
Washington D.C., District of Columbia • Boston, Massachusetts • New York, New York
Source: ClinicalTrials.gov public record
Updated Feb 7, 2024 · Synced Nov 2, 2026, 11:22 AM EST
Completed Not applicable Interventional

Preliminary Feasibility and Efficacy of Behavioral Intervention to Reduce Pain-Related Disability in Pediatric SCD

NCT04388241
View directory record Official record
Conditions
Sickle Cell Disease, Pain
Interventions
The Balance Program
Behavioral
Lead sponsor
Children's National Research Institute
Other
Eligibility
8 Years to 17 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2022
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Sep 6, 2022 · Synced Nov 2, 2026, 11:22 AM EST
Recruiting No phase listed Observational

Li-Fraumeni & TP53 (LiFT UP): Understanding and Progress

NCT04541654
View directory record Official record
Conditions
Li-Fraumeni Syndrome, TP53 Gene Mutation, Hereditary Cancer Syndrome, Clonal Hematopoiesis, Mosaicism
Interventions
Data and Specimen Collection
Genetic
Lead sponsor
Dana-Farber Cancer Institute
Other
Eligibility
Not listed
Enrollment
1,500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2032
U.S. locations
3
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Mar 27, 2026 · Synced Nov 2, 2026, 11:22 AM EST
Completed Not applicable Interventional

OnTrackCF: Engagement, Feasibility, and Acceptability Study

NCT07031323
View directory record Official record
Conditions
Cystic Fibrosis (CF), Cystic Fibrosis - Complete
Interventions
OnTrackCF
Behavioral
Lead sponsor
Boston Children's Hospital
Other
Eligibility
18 Years and older
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2026
U.S. locations
3
States / cities
Birmingham, Alabama • Baltimore, Maryland • Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Oct 27, 2026 · Synced Nov 2, 2026, 11:22 AM EST
Terminated Not applicable Interventional

Measuring and Improving Coproduction Using coopeRATE

NCT04060329
View directory record Official record
Conditions
Cystic Fibrosis, Chronic Disease
Interventions
coopeRATE Prompt
Behavioral
Lead sponsor
Dartmouth-Hitchcock Medical Center
Other
Eligibility
18 Years and older
Enrollment
280 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2020
U.S. locations
9
States / cities
San Diego, California • Denver, Colorado • Kansas City, Kansas + 6 more
Source: ClinicalTrials.gov public record
Updated Jul 9, 2020 · Synced Nov 2, 2026, 11:22 AM EST
Completed Phase 3 Interventional Results available

Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) ERT Compared With Imiglucerase in Type I Gaucher Disease

NCT00553631
View directory record Official record
Conditions
Gaucher Disease, Type 1
Interventions
velaglucerase alfa, imiglucerase
Biological
Lead sponsor
Shire
Industry
Eligibility
2 Years and older
Enrollment
34 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2009
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Jun 8, 2021 · Synced Nov 2, 2026, 11:22 AM EST
Not yet recruiting Phase 2 Interventional

Mirdametinib to Reduce the Size of Large/Giant Congenital Melanocytic Nevi in Children, Adolescents and Young Adults

NCT07891286
View directory record Official record
Conditions
LGCMN, Large and Giant Congenital Melanocytic Nevi, NF1, Solid Tumor, Pediatric Tumors, Neurocutaneous Melanocytosis, Congenital Melanocytic Nevus, Arachnoid Cysts, Astrocytoma, Ependymoma, Melanoma
Interventions
Mirdametinib
Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
3 Years to 40 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2027 – 2032
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 29, 2026 · Synced Nov 2, 2026, 11:22 AM EST
Withdrawn Phase 3 Interventional

Study of a Gene Therapy Treatment for Hemophilia A

NCT06297486
View directory record Official record
Conditions
Hemophilia A
Interventions
SPK-8011
Genetic
Lead sponsor
Spark Therapeutics, Inc.
Industry
Eligibility
18 Years and older · Male only
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2035
U.S. locations
27
States / cities
Phoenix, Arizona • Loma Linda, California • Los Angeles, California + 23 more
Source: ClinicalTrials.gov public record
Updated Dec 13, 2024 · Synced Nov 2, 2026, 11:22 AM EST
Recruiting Phase 4 Interventional

A Study to Test a Medicine (Fitusiran) for Preventing Bleeds in People With Severe Hemophilia Who Previously Received Preventive Treatment With Emicizumab

NCT06145373
View directory record Official record
Conditions
Hemophilia A
Interventions
Fitusiran (SAR439774), Clotting factor concentrates (CFC) or bypassing agents (BPA), Antithrombin concentrate (ATIIIC), Emicizumab
Drug · Biological
Lead sponsor
Sanofi
Industry
Eligibility
12 Years and older · Male only
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2030
U.S. locations
2
States / cities
Los Angeles, California • Flowood, Mississippi
Source: ClinicalTrials.gov public record
Updated Sep 23, 2026 · Synced Nov 2, 2026, 11:22 AM EST
Active, not recruiting No phase listed Observational

Childhood Allergy and the Neonatal Environment

NCT04215783
View directory record Official record
Conditions
Asthma in Children, Allergy
Interventions
Not listed
Lead sponsor
University of Wisconsin, Madison
Other
Eligibility
Not listed
Enrollment
499 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2027
U.S. locations
4
States / cities
Detroit, Michigan • St Louis, Missouri • Nashville, Tennessee + 1 more
Source: ClinicalTrials.gov public record
Updated Sep 11, 2026 · Synced Nov 2, 2026, 11:22 AM EST
Completed Phase 1Phase 2 Interventional Results available

An Extension Study of HGT-HIT-045 Evaluating Long-Term Safety and Clinical Outcomes of Idursulfase-IT in Conjunction With Elaprase in Pediatric Participants With Hunter Syndrome and Cognitive Impairment

NCT01506141
View directory record Official record
Conditions
Hunter Syndrome
Interventions
Idursulfase-IT, Elaprase
Drug
Lead sponsor
Takeda
Industry
Eligibility
3 Years to 18 Years · Male only
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2024
U.S. locations
7
States / cities
Chicago, Illinois • Chapel Hill, North Carolina • Portland, Oregon + 4 more
Source: ClinicalTrials.gov public record
Updated Aug 6, 2025 · Synced Nov 2, 2026, 11:22 AM EST
Completed Phase 2 Interventional Results available

Study of Dupilumab and Immune Responses in Adults With Atopic Dermatitis (AD)

NCT02210780
View directory record Official record
Conditions
Atopic Dermatitis
Interventions
Dupilumab, Placebo
Drug
Lead sponsor
Regeneron Pharmaceuticals
Industry
Eligibility
18 Years to 64 Years
Enrollment
194 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2015
U.S. locations
42
States / cities
Birmingham, Alabama • Fort Smith, Arkansas • Long Beach, California + 39 more
Source: ClinicalTrials.gov public record
Updated May 7, 2020 · Synced Nov 2, 2026, 11:22 AM EST
Completed Phase 3 Interventional Results available

Trial of Late Surfactant for Prevention of Bronchopulmonary Dysplasia

NCT01022580
View directory record Official record
Conditions
Bronchopulmonary Dysplasia
Interventions
Infasurf surfactant (ONY, Inc.), Sham (No Treatment)
Drug
Lead sponsor
Roberta Ballard
Other
Eligibility
7 Days to 14 Days
Enrollment
511 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2016
U.S. locations
21
States / cities
Little Rock, Arkansas • Berkeley, California • Oakland, California + 15 more
Source: ClinicalTrials.gov public record
Updated May 13, 2021 · Synced Nov 2, 2026, 11:22 AM EST
Completed Phase 3 Interventional Results available

Safety and Efficacy of Turoctocog Alfa in Haemophilia A Subjects

NCT00840086
View directory record Official record
Conditions
Congenital Bleeding Disorder, Haemophilia A
Interventions
turoctocog alfa
Drug
Lead sponsor
Novo Nordisk A/S
Industry
Eligibility
12 Years to 65 Years · Male only
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2011
U.S. locations
18
States / cities
Little Rock, Arkansas • Long Beach, California • Tampa, Florida + 15 more
Source: ClinicalTrials.gov public record
Updated Mar 17, 2017 · Synced Nov 2, 2026, 11:22 AM EST
Terminated Phase 2Phase 3 Interventional Results available

Clinical Trial of Growth Hormone in MPS I, II, and VI

NCT00748969
View directory record Official record
Conditions
Mucopolysaccharidosis I, Mucopolysaccharidosis II, Mucopolysaccharidosis VI
Interventions
Somatropin (DNA origin)
Drug
Lead sponsor
Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
Other
Eligibility
5 Years to 17 Years
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2013
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Sep 5, 2018 · Synced Nov 2, 2026, 11:22 AM EST
Terminated Phase 2 Interventional

A Study of SerpinPC in Participants with Hemophilia B (HemB) with Inhibitors

NCT05789537
View directory record Official record
Conditions
Hemophilia B with Inhibitor, Hemophilia B
Interventions
SerpinPC
Drug
Lead sponsor
ApcinteX Ltd
Industry
Eligibility
12 Years to 65 Years · Male only
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
3
States / cities
Aurora, Colorado • Tampa, Florida • Greenville, North Carolina
Source: ClinicalTrials.gov public record
Updated Mar 11, 2025 · Synced Nov 2, 2026, 11:22 AM EST
Terminated Phase 2 Interventional Results available

Decitabine Followed by Idarubicin and Cytarabine in Treating Patients With Relapsed or Refractory AML and MDS

NCT01607645
View directory record Official record
Conditions
Adult Acute Megakaryoblastic Leukemia (M7), Adult Acute Monoblastic Leukemia (M5a), Adult Acute Monocytic Leukemia (M5b), Adult Acute Myeloblastic Leukemia With Maturation (M2), Adult Acute Myeloblastic Leukemia Without Maturation (M1), Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Inv(16)(p13;q22), Adult Acute Myeloid Leukemia With t(16;16)(p13;q22), Adult Acute Myeloid Leukemia With t(8;21)(q22;q22), Adult Acute Myelomonocytic Leukemia (M4), Adult Erythroleukemia (M6a), Adult Pure Erythroid Leukemia (M6b), Previously Treated Myelodysplastic Syndromes, Recurrent Adult Acute Myeloid Leukemia, Refractory Anemia With Excess Blasts
Interventions
decitabine, idarubicin, cytarabine
Drug
Lead sponsor
Fred Hutchinson Cancer Center
Other
Eligibility
18 Years and older
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2013
U.S. locations
1
States / cities
Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Mar 31, 2017 · Synced Nov 2, 2026, 11:22 AM EST