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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Active, not recruiting Phase 2 Interventional

Study to Determine Optimal Dose and Evaluate Safety, Tolerability, and Pharmacokinetics of Progerinin in Patients With Hutchinson-Gilford Progeria Syndrome (HGPS)

NCT06775041
View directory record Official record
Conditions
Hutchinson-Gilford Progeria Syndrome
Interventions
Progerinin, Lonafarnib
Drug
Lead sponsor
PRG Science & Technology Co., Ltd.
Industry
Eligibility
1 Year and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2026
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Feb 8, 2026 · Synced Sep 18, 2026, 9:18 AM EDT
Recruiting Not applicable Interventional

Atlantic Lipid Lowering Treatment Optimization Program

NCT06439654
View directory record Official record
Conditions
Familial Hypercholesterolemia, Lipoprotein Types--Lp System Lp(A) Hyperlipoproteinemia, Apolipoprotein B 100, Familial Defective, High Density Lipoprotein Deficiency, Low-Density-Lipoid-Type Hyperlipoproteinemia
Interventions
Supportive care
Other
Lead sponsor
Atlantic Health System
Other
Eligibility
18 Years to 120 Years
Enrollment
250 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
2
States / cities
Clark, New Jersey • Springfield, New Jersey
Source: ClinicalTrials.gov public record
Updated Aug 2, 2025 · Synced Sep 18, 2026, 9:18 AM EDT
Active, not recruiting Phase 2 Interventional Accepts healthy volunteers

Naive T Cell Depletion for Preventing Chronic Graft-versus-Host Disease in Children and Young Adults With Blood Cancers Undergoing Donor Stem Cell Transplant

NCT03779854
View directory record Official record
Conditions
Acute Biphenotypic Leukemia, Acute Leukemia, Acute Leukemia of Ambiguous Lineage, Acute Lymphoblastic Leukemia, Acute Undifferentiated Leukemia, Allogeneic Hematopoietic Stem Cell Transplantation Recipient, Blastic Plasmacytoid Dendritic Cell Neoplasm, Blasts Under 25 Percent of Bone Marrow Nucleated Cells, Blasts Under 5 Percent of Bone Marrow Nucleated Cells, Mixed Phenotype Acute Leukemia, Myelodysplastic Syndrome With Excess Blasts-1, Myelodysplastic Syndrome/Acute Myeloid Leukemia, Burkitt Leukemia, Chronic Monocytic Leukemia, Lymphoblastic Lymphoma, Mast Cell Leukemia, Myeloproliferative Neoplasm
Interventions
Total-Body Irradiation, Thiotepa, Fludarabine, Cyclophosphamide, Busulfan, Allogeneic Bone Marrow Transplantation, Tacrolimus, Methotrexate, Naive T Cell-Depleted Hematopoietic Stem Cell Transplantation, Echocardiography, Biospecimen Collection, Bone Marrow Aspiration, Bone Marrow Biopsy
Radiation · Drug · Procedure
Lead sponsor
Fred Hutchinson Cancer Center
Other
Eligibility
6 Months to 26 Years
Enrollment
45 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2028
U.S. locations
9
States / cities
Los Angeles, California • Atlanta, Georgia • Iowa City, Iowa + 5 more
Source: ClinicalTrials.gov public record
Updated Sep 2, 2026 · Synced Sep 18, 2026, 9:18 AM EDT
Completed Not applicable Interventional

Theta Burst Stimulation to Improve Inhibitory Motor Physiology in Tourette Syndrome

NCT05628805
View directory record Official record
Conditions
Tourette Syndrome, Tourette Syndrome in Children, Tourette Syndrome in Adolescence, Tourette Syndrome, Modifier of
Interventions
iTBS
Device
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
10 Years to 21 Years
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Mar 17, 2026 · Synced Sep 18, 2026, 9:18 AM EDT
Recruiting Phase 3 Interventional

BEACON - Phase III Clinical Study of Rugonersen in Angelman Syndrome.

NCT07605429
View directory record Official record
Conditions
Angelman Syndrome
Interventions
rugonersen, Sham procedure
Drug · Procedure
Lead sponsor
OHB Pediatrics Ltd.
Industry
Eligibility
1 Year to 50 Years
Enrollment
165 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2031
U.S. locations
3
States / cities
San Diego, California • Chicago, Illinois • Chapel Hill, North Carolina
Source: ClinicalTrials.gov public record
Updated Sep 9, 2026 · Synced Sep 18, 2026, 9:18 AM EDT
Terminated No phase listed Observational

Comparison of Bispectral Index Values in Patients With and Without Down's Syndrome

NCT02288702
View directory record Official record
Conditions
Down Syndrome
Interventions
monitoring BIS in both groups
Device
Lead sponsor
Milton S. Hershey Medical Center
Other
Eligibility
2 Years to 17 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2017
U.S. locations
1
States / cities
Hershey, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jan 22, 2023 · Synced Sep 18, 2026, 9:18 AM EDT
Completed Phase 1Phase 2 Interventional

Treatment of Exudative and Vasogenic Chorioretinal Diseases Including Variants of AMD and Other CNV Related Maculopathy

NCT00470977
View directory record Official record
Conditions
Coats' Disease, Idiopathic Retinal Telangiectasia, Retinal Angiomatous Proliferation, Polypoidal Choroidal Vasculopathy, Pseudoxanthoma Elasticum, Pathological Myopia, Multi-focal Choroiditis, Rubeosis Iridis, Von Hippel Lindau Disease, BEST VITELLIFORM MACULAR DYSTROPHY, MULTIFOCAL (Disorder)
Interventions
ranibizumab injection (0.5 mg)
Drug
Lead sponsor
Manhattan Eye, Ear & Throat Hospital
Other
Eligibility
18 Years and older
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2010
U.S. locations
2
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Oct 24, 2012 · Synced Sep 18, 2026, 9:18 AM EDT
Completed Not applicable Interventional Results available

Treadmill Training and Orthotic Use in Infants With Down Syndrome

NCT00825175
View directory record Official record
Conditions
Down Syndrome
Interventions
Treadmill Training, Supramalleolar Orthoses
Behavioral · Device
Lead sponsor
University of Michigan
Other
Eligibility
6 Months to 36 Months
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2008
U.S. locations
1
States / cities
Ann Arbor, Michigan
Source: ClinicalTrials.gov public record
Updated Dec 3, 2017 · Synced Sep 18, 2026, 9:18 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A Trial to Investigate Teprotumumab (High-concentration Formulation) Subcutaneous Administration in Healthy Adult Non-Japanese and Japanese Participants

NCT06674941
View directory record Official record
Conditions
Healthy Volunteers
Interventions
Teprotumumab
Drug
Lead sponsor
Amgen
Industry
Eligibility
18 Years to 55 Years
Enrollment
44 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2023
U.S. locations
1
States / cities
Las Vegas, Nevada
Source: ClinicalTrials.gov public record
Updated Nov 4, 2024 · Synced Sep 18, 2026, 9:18 AM EDT
Completed Not applicable Interventional

Effects of Innovative Aerobic Exercise Training in Cystic Fibrosis

NCT04543929
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
exercise
Behavioral
Lead sponsor
University of Kansas Medical Center
Other
Eligibility
18 Years and older
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020
U.S. locations
1
States / cities
Kansas City, Kansas
Source: ClinicalTrials.gov public record
Updated Oct 9, 2024 · Synced Sep 18, 2026, 9:18 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers Results available

Validation of a Diagnostic Test for Glucose-6-phosphate Dehydrogenase (G6PD) Deficiency in Anti-coagulated Blood

NCT04054661
View directory record Official record
Conditions
G6PD Deficiency
Interventions
SD Biosensor STANDARD G6PD Test, Pointe Scientific Test Kit, HemoCue System
Diagnostic Test
Lead sponsor
PATH
Other
Eligibility
18 Years to 65 Years
Enrollment
229 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2020
U.S. locations
1
States / cities
Reading, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Nov 8, 2021 · Synced Sep 18, 2026, 9:18 AM EDT
Terminated Phase 1 Interventional

Gene Transfer Clinical Study in Crigler-Najjar Syndrome

NCT03223194
View directory record Official record
Conditions
Crigler-Najjar Syndrome
Interventions
AT342
Genetic
Lead sponsor
Audentes Therapeutics
Industry
Eligibility
1 Year and older
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2021
U.S. locations
2
States / cities
The Bronx, New York • Strasburg, Pennsylvania
Source: ClinicalTrials.gov public record
Updated May 17, 2022 · Synced Sep 18, 2026, 9:18 AM EDT
Completed No phase listed Observational

Prognostic Value of Transforming Growth Factor-Beta 1 in Normal Pressure Hydrocephalus

NCT00600795
View directory record Official record
Conditions
Normal Pressure Hydrocephalus
Interventions
CSF collection
Other
Lead sponsor
Vanderbilt University Medical Center
Other
Eligibility
18 Years and older
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2011
U.S. locations
1
States / cities
Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Apr 3, 2017 · Synced Sep 18, 2026, 9:18 AM EDT
Completed No phase listed Observational

T Cell Memory Fuels the Innate Response in Chronic CF Lung Disease

NCT04397861
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Not listed
Lead sponsor
National Jewish Health
Other
Eligibility
18 Years and older
Enrollment
112 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2021
U.S. locations
1
States / cities
Denver, Colorado
Source: ClinicalTrials.gov public record
Updated Sep 28, 2022 · Synced Sep 18, 2026, 9:18 AM EDT
Completed Phase 3 Interventional

A Clinical Study of Intravenous Immunoglobulin

NCT00468273
View directory record Official record
Conditions
Immunologic Deficiency Syndromes
Interventions
Immune Globulin Intravenous (Human) Omr-IgG-am IGIV
Drug
Lead sponsor
FFF Enterprises
Industry
Eligibility
3 Years to 75 Years
Enrollment
57 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2009
U.S. locations
8
States / cities
Los Angeles, California • Centennial, Colorado • North Palm Beach, Florida + 5 more
Source: ClinicalTrials.gov public record
Updated Aug 10, 2014 · Synced Sep 18, 2026, 9:18 AM EDT
Terminated Phase 1Phase 2 Interventional Results available

A Study of FCX-007 for Recessive Dystrophic Epidermolysis Bullosa (RDEB)

NCT02810951
View directory record Official record
Conditions
Epidermolysis Bullosa Dystrophica, Recessive
Interventions
FCX-007
Genetic
Lead sponsor
Castle Creek Biosciences, LLC.
Industry
Eligibility
7 Years and older
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2022
U.S. locations
2
States / cities
Stanford, California • Aurora, Colorado
Source: ClinicalTrials.gov public record
Updated Feb 7, 2023 · Synced Sep 18, 2026, 9:18 AM EDT
Completed Phase 2 Interventional Results available

RAPAMYCIN FOR KIDNEY ANGIOMYOLIPOMAS

NCT00126672
View directory record Official record
Conditions
Nonmalignant Neoplasm, Tuberous Sclerosis, Lymphangioleimyomatosis, Kidney Angiomyolipoma
Interventions
Rapamycin
Drug
Lead sponsor
Dana-Farber Cancer Institute
Other
Eligibility
3 Years to 65 Years
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2010
U.S. locations
7
States / cities
Loma Linda, California • Hartford, Connecticut • Boston, Massachusetts + 4 more
Source: ClinicalTrials.gov public record
Updated Apr 24, 2022 · Synced Sep 18, 2026, 9:18 AM EDT
Recruiting No phase listed Observational

Pancreas Registry and High Risk Registry

NCT02775461
View directory record Official record
Conditions
Pancreas Cancer, Pancreatitis, Chronic Pancreatitis, Pancreatic Cyst, Family History of Pancreas Cancer, Genetic Mutations
Interventions
Not listed
Lead sponsor
Icahn School of Medicine at Mount Sinai
Other
Eligibility
18 Years and older
Enrollment
1,368 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2033
U.S. locations
2
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Feb 10, 2026 · Synced Sep 18, 2026, 9:18 AM EDT
Completed Not applicable Interventional

Study of Total Body Irradiation and Fludarabine Followed By Allogeneic Peripheral Blood Stem Cell or Bone Marrow Transplantation in Combination With Cyclosporine and Mycophenolate Mofetil in Patients With Inherited Disorders

NCT00010361
View directory record Official record
Conditions
Metabolism, Inborn Errors, Granulomatous Disease, Chronic
Interventions
cyclosporine, fludarabine, mycophenolate mofetil
Drug
Lead sponsor
Fred Hutchinson Cancer Center
Other
Eligibility
Up to 55 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2000
U.S. locations
1
States / cities
Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Dec 8, 2014 · Synced Sep 18, 2026, 9:18 AM EDT
Completed No phase listed Observational

Observational Study of Patients With Mucopolysaccharidosis (MPS) VI Who Previously Participated in ASB-00-02

NCT01387854
View directory record Official record
Conditions
MPS VI
Interventions
Not listed
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
Not listed
Enrollment
59 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2013
U.S. locations
1
States / cities
Oakland, California
Source: ClinicalTrials.gov public record
Updated Apr 1, 2014 · Synced Sep 18, 2026, 9:18 AM EDT
Completed Phase 1 Interventional

Study of Ruxolitinib Cream in Children With Atopic Dermatitis

NCT05034822
View directory record Official record
Conditions
Atopic Dermatitis
Interventions
Ruxolitinib cream
Drug
Lead sponsor
Incyte Corporation
Industry
Eligibility
2 Years to 11 Years
Enrollment
29 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
15
States / cities
Gilbert, Arizona • Hot Springs, Arkansas • Anaheim, California + 12 more
Source: ClinicalTrials.gov public record
Updated Sep 14, 2023 · Synced Sep 18, 2026, 9:18 AM EDT
Withdrawn Phase 2 Interventional

Treatment of Chronic Itch in Atopic Dermatitis With Opioid Antagonist Naltrexone

NCT04325802
View directory record Official record
Conditions
Atopic Dermatitis, Pruritus, Pruritus Chronic, Dermatitis
Interventions
Naltrexone, Placebo
Drug · Other
Lead sponsor
University of Minnesota
Other
Eligibility
18 Years to 70 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Apr 3, 2023 · Synced Sep 18, 2026, 9:18 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Spectralis HRA+OCT Imaging of the Retina With Autofluorescence in Sickle Cell Disease

NCT01123369
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Not listed
Lead sponsor
Medical University of South Carolina
Other
Eligibility
12 Years and older
Enrollment
8 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2010
U.S. locations
2
States / cities
Charleston, South Carolina
Source: ClinicalTrials.gov public record
Updated Sep 13, 2010 · Synced Sep 18, 2026, 9:18 AM EDT
Completed Early Phase 1 Interventional

Netarsudil Use After Descemetorhexis Without Endothelial Keratoplasty

NCT04057053
View directory record Official record
Conditions
Fuchs' Endothelial Dystrophy, Cataract
Interventions
Netarsudil 0.02% Ophthalmic Solution
Drug
Lead sponsor
Massachusetts Eye and Ear Infirmary
Other
Eligibility
50 Years to 70 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2020
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jan 25, 2021 · Synced Sep 18, 2026, 9:18 AM EDT