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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Completed Phase 1Phase 2 Interventional Results available

A Study of JR-171 in Patients With Mucopolysaccharidosis I

NCT04227600
View directory record Official record
Conditions
Mucopolysaccharidosis I
Interventions
JR-171 (lepunafusp alfa)
Drug
Lead sponsor
JCR Pharmaceuticals Co., Ltd.
Industry
Eligibility
0 Years and older
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2022
U.S. locations
1
States / cities
Oakland, California
Source: ClinicalTrials.gov public record
Updated Mar 18, 2025 · Synced Jul 23, 2026, 1:09 PM EDT
Not yet recruiting Phase 1 Interventional

Study of Cabozantinib With Selumetinib for Plexiform Neurofibromas

NCT06502171
View directory record Official record
Conditions
Neurofibromatosis 1, Plexiform Neurofibroma
Interventions
Cabozantinib Oral Tablet, Selumetinib Oral Capsule
Drug
Lead sponsor
Girish Dhall, MD
Other
Eligibility
16 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2034
U.S. locations
1
States / cities
Birmingham, Alabama
Source: ClinicalTrials.gov public record
Updated Mar 11, 2026 · Synced Jul 23, 2026, 1:09 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Remote Exercise Study for Individuals With Down Syndrome

NCT04647851
View directory record Official record
Conditions
Down Syndrome
Interventions
Remote exercise intervention Down syndrome
Other
Lead sponsor
University of Nevada, Las Vegas
Other
Eligibility
18 Years to 35 Years
Enrollment
20 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2022
U.S. locations
1
States / cities
Las Vegas, Nevada
Source: ClinicalTrials.gov public record
Updated May 25, 2023 · Synced Jul 23, 2026, 1:09 PM EDT
Terminated Phase 2 Interventional Results available

Celecoxib for Thyroid Eye Disease

NCT02845336
View directory record Official record
Conditions
Thyroid Eye Disease
Interventions
Celecoxib, artificial tears
Drug
Lead sponsor
Johns Hopkins University
Other
Eligibility
18 Years and older
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2019
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 30, 2020 · Synced Jul 23, 2026, 1:09 PM EDT
Completed Not applicable Interventional Results available

Genomic Sequencing for Childhood Risk and Newborn Illness

NCT02422511
View directory record Official record
Conditions
Hereditary Disease, Genetic Predisposition to Disease
Interventions
Genomic sequencing, Family history report
Genetic · Other
Lead sponsor
Brigham and Women's Hospital
Other
Eligibility
Not listed
Enrollment
1,205 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2021
U.S. locations
2
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Apr 2, 2024 · Synced Jul 23, 2026, 1:09 PM 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 Jul 23, 2026, 1:09 PM EDT
Terminated Phase 2 Interventional Results available

Trial of Dextromethorphan in Rett Syndrome

NCT00593957
View directory record Official record
Conditions
Rett Syndrome
Interventions
Dextromethorphan
Drug
Lead sponsor
Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Other
Eligibility
2 Years to 15 Years
Enrollment
38 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2010
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Apr 22, 2014 · Synced Jul 23, 2026, 1:09 PM EDT
Completed No phase listed Observational

Biliary Atresia Research Network Northeast

NCT06184971
View directory record Official record
Conditions
Biliary Atresia
Interventions
Not listed
Lead sponsor
Yale University
Other
Eligibility
Up to 12 Years
Enrollment
320 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
14
States / cities
Hartford, Connecticut • New Haven, Connecticut • Bangor, Maine + 10 more
Source: ClinicalTrials.gov public record
Updated Jun 24, 2026 · Synced Jul 23, 2026, 1:09 PM EDT
Terminated Phase 1 Interventional

Bevacizumab and Temsirolimus Alone or in Combination With Valproic Acid or Cetuximab in Treating Patients With Advanced or Metastatic Malignancy or Other Benign Disease

NCT01552434
View directory record Official record
Conditions
Advanced Malignant Neoplasm, Castleman Disease, Digestive System Carcinoma, Erdheim-Chester Disease, Lip and Oral Cavity Carcinoma, Lymphangioleiomyomatosis, Malignant Endocrine Neoplasm, Malignant Female Reproductive System Neoplasm, Malignant Male Reproductive System Neoplasm, Malignant Neoplasm, Malignant Respiratory Tract Neoplasm, Malignant Thoracic Neoplasm, Malignant Urinary System Neoplasm, Mesothelial Neoplasm, Metastatic Malignant Neoplasm, Metastatic Urothelial Carcinoma, Neurofibromatosis Type 2, Recurrent Adult Soft Tissue Sarcoma, Recurrent Breast Carcinoma, Recurrent Childhood Soft Tissue Sarcoma, Recurrent Digestive System Carcinoma, Recurrent Female Reproductive System Carcinoma, Recurrent Male Reproductive System Carcinoma, Recurrent Malignant Neoplasm, Recurrent Pharyngeal Carcinoma, Recurrent Thyroid Gland Carcinoma, Refractory Malignant Neoplasm, Soft Tissue Neoplasm, Stage III Breast Cancer AJCC v7, Stage III Pharyngeal Cancer, Stage IIIA Breast Cancer AJCC v7, Stage IIIB Breast Cancer AJCC v7, Stage IIIC Breast Cancer AJCC v7, Stage IV Breast Cancer AJCC v6 and v7, Stage IV Pharyngeal Cancer, Stage IVA Pharyngeal Cancer, Stage IVB Pharyngeal Cancer, Stage IVC Pharyngeal Cancer, Thyroid Gland Neoplasm
Interventions
Bevacizumab, Cetuximab, Laboratory Biomarker Analysis, Pharmacological Study, Temsirolimus, Valproic Acid
Biological · Other · Drug
Lead sponsor
M.D. Anderson Cancer Center
Other
Eligibility
Not listed
Enrollment
154 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2026
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated May 14, 2026 · Synced Jul 23, 2026, 1:09 PM EDT
Terminated Phase 2 Interventional Results available

A Study of the Effect of Blood Stem Cell Transplant After Chemotherapy Alone in Patients With Fanconi Anemia

NCT03600909
View directory record Official record
Conditions
Fanconi Anemia, Myelodysplastic Syndrome (MDS), Acute Myelogenous Leukemia (AML)
Interventions
Busulfan, Fludarabine, Cyclophosphamide, Anti-Thymocyte Globulin (Rabbit), The CliniMACS device, G-CSF
Drug · Device
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
1 Month and older
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
3
States / cities
New York, New York • Cincinnati, Ohio • Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Apr 11, 2022 · Synced Jul 23, 2026, 1:09 PM EDT
Completed No phase listed Observational

A Natural History Study of Sanfilippo Syndrome Type D

NCT05648851
View directory record Official record
Conditions
Sanfilippo Syndrome Type D
Interventions
Not listed
Lead sponsor
Phoenix Nest
Industry
Eligibility
12 Months and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jul 31, 2025 · Synced Jul 23, 2026, 1:09 PM EDT
Completed Phase 2 Interventional Results available

Submucosal Bevacizumab for the Management of Recurrent Epistaxis in Patients With Hereditary Hemorrhagic Telangiectasia (HHT)

NCT01402531
View directory record Official record
Conditions
Hereditary Hemorrhagic Telangiectasia (HHT)
Interventions
Submucosal Bevacizumab
Drug
Lead sponsor
University of California, San Diego
Other
Eligibility
18 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2013
U.S. locations
1
States / cities
San Diego, California
Source: ClinicalTrials.gov public record
Updated Dec 3, 2019 · Synced Jul 23, 2026, 1:09 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Safety and Efficacy of TSHA-102 in Pediatric Females With Rett Syndrome (REVEAL Pediatric Study)

NCT06152237
View directory record Official record
Conditions
Rett Syndrome
Interventions
TSHA-102
Genetic
Lead sponsor
Taysha Gene Therapies, Inc.
Industry
Eligibility
5 Years to 8 Years · Female only
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2031
U.S. locations
3
States / cities
Chicago, Illinois • Saint Paul, Minnesota • St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Oct 14, 2025 · Synced Jul 23, 2026, 1:09 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Specimen and Data Study for Ovarian Cancer Early Detection and Prevention

NCT00005095
View directory record Official record
Conditions
Cervical Cancer, Endometrial Cancer, Fallopian Tube Cancer, Hereditary Breast/Ovarian Cancer (brca1, brca2), Ovarian Cancer, Sarcoma, Uterine Leiomyomata, Vaginal Cancer, Vulvar Cancer
Interventions
Ca-125, screening questionnaire administration, Surgery
Other · Procedure
Lead sponsor
Northwestern University
Other
Eligibility
18 Years to 80 Years · Female only
Enrollment
6,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2000 – 2040
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Feb 27, 2025 · Synced Jul 23, 2026, 1:09 PM EDT
Completed Phase 2 Interventional

An Open-Label Clinical Trial of Replagal Enzyme Therapy in Children Ages 7-17 Years With Fabry Disease

NCT00071877
View directory record Official record
Conditions
Fabry Disease
Interventions
Replagal
Drug
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Not listed
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2005
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 3, 2008 · Synced Jul 23, 2026, 1:09 PM EDT
Completed Not applicable Interventional Results available

High-Dose Chemotherapy With or Without Total-Body Irradiation Followed by Autologous Stem Cell Transplant in Treating Patients With Hematologic Cancer or Solid Tumors

NCT00536601
View directory record Official record
Conditions
Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Myeloid Leukemia in Remission, 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), Adult Nasal Type Extranodal NK/T-cell Lymphoma, Childhood Acute Lymphoblastic Leukemia in Remission, Childhood Acute Myeloid Leukemia in Remission, Childhood Burkitt Lymphoma, Childhood Diffuse Large Cell Lymphoma, Childhood Immunoblastic Large Cell Lymphoma, Childhood Nasal Type Extranodal NK/T-cell Lymphoma, Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor (PNET), Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue, Hepatosplenic T-cell Lymphoma, Intraocular Lymphoma, Nodal Marginal Zone B-cell Lymphoma, Peripheral T-cell Lymphoma, Plasma Cell Neoplasm, Primary Systemic Amyloidosis, Recurrent Adult Acute Lymphoblastic Leukemia, Recurrent Adult Acute Myeloid Leukemia, Recurrent Adult Burkitt Lymphoma, Recurrent Adult Diffuse Large Cell Lymphoma, Recurrent Adult Diffuse Mixed Cell Lymphoma, Recurrent Adult Diffuse Small Cleaved Cell Lymphoma, Recurrent Adult Grade III Lymphomatoid Granulomatosis, Recurrent Adult Hodgkin Lymphoma, Recurrent Adult Immunoblastic Large Cell Lymphoma, Recurrent Adult Lymphoblastic Lymphoma, Recurrent Childhood Acute Lymphoblastic Leukemia, Recurrent Childhood Acute Myeloid Leukemia, Recurrent Childhood Anaplastic Large Cell Lymphoma, Recurrent Childhood Grade III Lymphomatoid Granulomatosis, Recurrent Childhood Large Cell Lymphoma, Recurrent Childhood Lymphoblastic Lymphoma, Recurrent Childhood Small Noncleaved Cell Lymphoma, Recurrent Cutaneous T-cell Non-Hodgkin Lymphoma, Recurrent Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor, Recurrent Grade 1 Follicular Lymphoma, Recurrent Grade 2 Follicular Lymphoma, Recurrent Grade 3 Follicular Lymphoma, Recurrent Malignant Testicular Germ Cell Tumor, Recurrent Mantle Cell Lymphoma, Recurrent Marginal Zone Lymphoma, Recurrent Neuroblastoma, Recurrent Small Lymphocytic Lymphoma, Recurrent/Refractory Childhood Hodgkin Lymphoma, Refractory Chronic Lymphocytic Leukemia, Refractory Multiple Myeloma, Regional Neuroblastoma, Splenic Marginal Zone Lymphoma, Testicular Lymphoma, Unspecified Adult Solid Tumor, Protocol Specific, Unspecified Childhood Solid Tumor, Protocol Specific, Waldenström Macroglobulinemia
Interventions
etoposide, cyclophosphamide, carmustine, melphalan, busulfan, carboplatin, thiotepa, total-body irradiation, autologous hematopoietic stem cell transplantation, autologous-autologous tandem hematopoietic stem cell transplantation
Drug · Radiation · Procedure
Lead sponsor
Roswell Park Cancer Institute
Other
Eligibility
4 Years and older
Enrollment
174 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2018
U.S. locations
1
States / cities
Buffalo, New York
Source: ClinicalTrials.gov public record
Updated Apr 21, 2021 · Synced Jul 23, 2026, 1:09 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

CorEvitas International Adolescent Atopic Dermatitis (AD) Drug Safety and Effectiveness Registry

NCT06238765
View directory record Official record
Conditions
Atopic Dermatitis
Interventions
Not listed
Lead sponsor
CorEvitas
Network
Eligibility
10 Years to 15 Years
Enrollment
1,500 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2023 – 2099
U.S. locations
1
States / cities
Waltham, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jun 28, 2026 · Synced Jul 23, 2026, 1:09 PM EDT
Withdrawn No phase listed Observational

Measurement of Carboxyhemoglobin by Gas Chromatography as an Index of Hemolysis

NCT00917007
View directory record Official record
Conditions
ABO Incompatibility, Hemolytic Disease of Newborn, Neonatal Jaundice, Neonatal Hyperbilirubinemia
Interventions
Not listed
Lead sponsor
State University of New York - Upstate Medical University
Other
Eligibility
Up to 28 Days
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2010
U.S. locations
2
States / cities
Syracuse, New York
Source: ClinicalTrials.gov public record
Updated Oct 24, 2021 · Synced Jul 23, 2026, 1:09 PM EDT
Recruiting No phase listed Observational

A Long-Term Observational Study of Patients With Fucosidosis

NCT07615400
View directory record Official record
Conditions
Fucosidosis
Interventions
Not listed
Lead sponsor
JCR Pharmaceuticals Co., Ltd.
Industry
Eligibility
Not listed
Enrollment
57 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2031
U.S. locations
2
States / cities
Orange, California • Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated May 28, 2026 · Synced Jul 23, 2026, 1:09 PM EDT
Completed Not applicable Interventional Results available

Reduced-Intensity Hematopoietic Stem Cell Transplant for High Risk Lysosomal and Peroxisomal Disorders

NCT01626092
View directory record Official record
Conditions
Lysosomal Storage Disease, Peroxisomal Disorder
Interventions
Campath-1H, Clofarabine, Melphalan, Total Body Irradiation with Marrow Boosting, Hematopoietic stem cell transplantation, Cyclosporine A, Mycophenolate mofetil
Drug · Radiation · Biological
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 55 Years
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2013
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Dec 4, 2017 · Synced Jul 23, 2026, 1:09 PM EDT
Withdrawn Not applicable Interventional

FFP Versus PCC in Intracranial Hemorrhage

NCT02429453
View directory record Official record
Conditions
Intracranial Hemorrhage, Traumatic, Intracranial Hemorrhage, Spontaneous
Interventions
Four Factor Prothrombin Complex Concentrate, Fresh Frozen Plasma
Drug · Biological
Lead sponsor
University of Utah
Other
Eligibility
18 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2016
U.S. locations
1
States / cities
Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated Nov 15, 2016 · Synced Jul 23, 2026, 1:09 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

NSAID Drug Interaction Study

NCT01884272
View directory record Official record
Conditions
Gout
Interventions
lesinurad 400 mg, naproxen 250 mg, indomethacin 25 mg
Drug
Lead sponsor
Ardea Biosciences, Inc.
Industry
Eligibility
18 Years to 55 Years · Male only
Enrollment
24 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013
U.S. locations
1
States / cities
Kalamazoo, Michigan
Source: ClinicalTrials.gov public record
Updated Nov 7, 2013 · Synced Jul 23, 2026, 1:09 PM EDT
Active, not recruiting Phase 1 Interventional

Safety of Blood Stem Cell Mobilization With Plerixafor in Patients With Sickle Cell Disease

NCT03664830
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Plerixafor
Drug
Lead sponsor
City of Hope Medical Center
Other
Eligibility
18 Years to 40 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2026
U.S. locations
1
States / cities
Duarte, California
Source: ClinicalTrials.gov public record
Updated Sep 15, 2025 · Synced Jul 23, 2026, 1:09 PM EDT