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ClinicalTrials.gov public records Last synced Jun 11, 2026, 1:47 AM EDT

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

Fludarabine Phosphate, Cyclophosphamide, and Total-Body Irradiation Followed by Donor Bone Marrow Transplant, Mycophenolate Mofetil, and Cyclosporine in Treating Patients With Fanconi Anemia

NCT00453388
View directory record Official record
Conditions
Acute Myeloid Leukemia in Remission, de Novo Myelodysplastic Syndrome, Fanconi Anemia, Previously Treated Myelodysplastic Syndrome
Interventions
Allogeneic Bone Marrow Transplantation, Cyclophosphamide, Cyclosporine, Fludarabine Phosphate, Laboratory Biomarker Analysis, Mycophenolate Mofetil, Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation, Total-Body Irradiation
Procedure · Drug · Other + 1 more
Lead sponsor
Fred Hutchinson Cancer Center
Other
Eligibility
Not listed
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2013
U.S. locations
4
States / cities
Oakland, California • Nashville, Tennessee • Seattle, Washington + 1 more
Source: ClinicalTrials.gov public record
Updated Jan 28, 2020 · Synced Jun 11, 2026, 1:47 AM EDT
Recruiting No phase listed Observational

Fabry Disease Registry & Pregnancy Sub-registry

NCT00196742
View directory record Official record
Conditions
Fabry Disease
Interventions
Not listed
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
Not listed
Enrollment
9,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2034
U.S. locations
92
States / cities
Birmingham, Alabama • Phoenix, Arizona • Tucson, Arizona + 72 more
Source: ClinicalTrials.gov public record
Updated Apr 27, 2026 · Synced Jun 11, 2026, 1:47 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 Jun 11, 2026, 1:47 AM EDT
Not listed Phase 2 Interventional

Umbilical Cord Blood for Stem Cell Transplantation in Treating Young Patients With Malignant or Nonmalignant Diseases

NCT00084695
View directory record Official record
Conditions
Childhood Langerhans Cell Histiocytosis, Fanconi Anemia, Leukemia, Lymphoma, Myelodysplastic Syndromes, Neuroblastoma, Sarcoma, Unspecified Childhood Solid Tumor, Protocol Specific
Interventions
anti-thymocyte globulin, busulfan, cyclophosphamide, fludarabine phosphate, melphalan, methylprednisolone, radiation therapy
Biological · Drug · Radiation
Lead sponsor
Milton S. Hershey Medical Center
Other
Eligibility
Up to 21 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2003
U.S. locations
1
States / cities
Hershey, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jan 9, 2014 · Synced Jun 11, 2026, 1:47 AM EDT
Completed No phase listed Observational Results available

Response to Diaphragmatic Pacing in Subjects With Pompe Disease

NCT02354651
View directory record Official record
Conditions
Pompe Disease
Interventions
Diaphragm conditioning via phrenic nerve stimulation
Other
Lead sponsor
University of Florida
Other
Eligibility
2 Years to 65 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2023
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Jul 31, 2023 · Synced Jun 11, 2026, 1:47 AM EDT
Recruiting Not applicable Interventional

Interfacing With NeuroTechnology to Expand Neural Throughput (INTENT)

NCT07521930
View directory record Official record
Conditions
Tetraplegia/Tetraparesis, Amyotrophic Lateral Sclerosis (ALS), Muscular Disorders, Atrophic, Brain Stem Stroke, Spinal Cord Injuries (SCI), Muscular Dystrophies, Muscular Atrophy, Progressive, Pontine Hemorrhage, Pontine Infarction
Interventions
INTENT Neural Interface System
Device
Lead sponsor
Johns Hopkins University
Other
Eligibility
18 Years to 80 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Apr 12, 2026 · Synced Jun 11, 2026, 1:47 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

Primaquine Enantiomers in G6PD Deficient Human Volunteers

NCT04073953
View directory record Official record
Conditions
G6PD Deficiency
Interventions
RPQ, SPQ, Placebo
Drug
Lead sponsor
University of Mississippi, Oxford
Other
Eligibility
18 Years to 65 Years
Enrollment
18 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2023
U.S. locations
1
States / cities
University, Mississippi
Source: ClinicalTrials.gov public record
Updated Nov 20, 2024 · Synced Jun 11, 2026, 1:47 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Maternal Obesity and Small for Gestational Age Infants

NCT00371657
View directory record Official record
Conditions
Obesity, Small for Gestational Age
Interventions
Not listed
Lead sponsor
Oklahoma State University Center for Health Sciences
Other
Eligibility
18 Years to 50 Years · Female only
Enrollment
420 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2006
U.S. locations
1
States / cities
Tulsa, Oklahoma
Source: ClinicalTrials.gov public record
Updated Feb 3, 2008 · Synced Jun 11, 2026, 1:47 AM EDT
Completed Phase 3 Interventional Results available

Study of the Effects of Oral AT1001 (Migalastat Hydrochloride) in Patients With Fabry Disease

NCT00925301
View directory record Official record
Conditions
Fabry Disease
Interventions
migalastat hydrochloride, Placebo
Drug
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
16 Years to 74 Years
Enrollment
67 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2014
U.S. locations
13
States / cities
Los Angeles, California • San Francisco, California • Decatur, Georgia + 10 more
Source: ClinicalTrials.gov public record
Updated Oct 29, 2018 · Synced Jun 11, 2026, 1:47 AM EDT
Terminated Phase 1Phase 2 Interventional Results available

Extension Study to Study PQ-110-001 (NCT03140969)

NCT03913130
View directory record Official record
Conditions
Leber Congenital Amaurosis 10, Blindness, Leber Congenital Amaurosis, Vision Disorders, Sensation Disorders, Neurologic Manifestations, Eye Diseases, Eye Diseases, Hereditary, Eye Disorders Congenital, Retinal Disease
Interventions
QR-110
Drug
Lead sponsor
Laboratoires Thea
Industry
Eligibility
6 Years and older
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
2
States / cities
Iowa City, Iowa • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Oct 14, 2024 · Synced Jun 11, 2026, 1:47 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Research Study in Healthy Volunteers of Patients With Fanconi Anemia, Myeloproliferative Disorders, or Myeloma

NCT00900055
View directory record Official record
Conditions
Chronic Myeloproliferative Disorders, Fanconi Anemia, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic/Myeloproliferative Neoplasms
Interventions
microarray analysis, polyacrylamide gel electrophoresis, polymerase chain reaction, protein expression analysis, reverse transcriptase-polymerase chain reaction, western blotting, chromatography, high performance liquid chromatography, immunoenzyme technique
Genetic · Other
Lead sponsor
OHSU Knight Cancer Institute
Other
Eligibility
1 Year to 55 Years
Enrollment
213 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
1975 – 2016
U.S. locations
1
States / cities
Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Jul 4, 2022 · Synced Jun 11, 2026, 1:47 AM EDT
Completed Phase 3 Interventional Results available

Extension Study of TKT028 Evaluating Safety and Clinical Outcomes of Replagal® in Adult Patients With Fabry Disease

NCT01124643
View directory record Official record
Conditions
Fabry Disease
Interventions
Replagal
Biological
Lead sponsor
Shire
Industry
Eligibility
18 Years and older
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2013
U.S. locations
2
States / cities
Iowa City, Iowa • Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Jun 7, 2021 · Synced Jun 11, 2026, 1:47 AM EDT
Completed Phase 1 Interventional

Cyclophosphamide in Treating Patients Who Are Undergoing a Donor Bone Marrow Transplant for Fanconi's Anemia

NCT00317876
View directory record Official record
Conditions
Fanconi Anemia
Interventions
cyclophosphamide, cyclosporine, methotrexate, allogeneic bone marrow transplantation, nonmyeloablative allogeneic hematopoietic stem cell transplantation
Drug · Procedure
Lead sponsor
Fred Hutchinson Cancer Center
Other
Eligibility
Not listed
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1998
U.S. locations
2
States / cities
Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Apr 19, 2012 · Synced Jun 11, 2026, 1:47 AM EDT
Completed No phase listed Observational

Study to Evaluate Biomarkers and Clinical Manifestations in Individuals With Glycogen Storage Disease Type III (GSD III)

NCT04574830
View directory record Official record
Conditions
Glycogen Storage Disease Type III
Interventions
No Intervention
Other
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
5 Years to 65 Years
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2022
U.S. locations
4
States / cities
Irvine, California • Orange, California • Aurora, Colorado + 1 more
Source: ClinicalTrials.gov public record
Updated Sep 12, 2022 · Synced Jun 11, 2026, 1:47 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Impact of the Severity of Pediatric Psoriasis on Childhood Body Mass Index

NCT00879944
View directory record Official record
Conditions
Psoriasis, Atopic Dermatitis
Interventions
Blood pressure, Height, Weight, BMI, Waist Circumference
Other
Lead sponsor
Northwestern University
Other
Eligibility
5 Years to 17 Years
Enrollment
409 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2012
U.S. locations
9
States / cities
San Diego, California • San Francisco, California • Stanford, California + 6 more
Source: ClinicalTrials.gov public record
Updated Apr 13, 2015 · Synced Jun 11, 2026, 1:47 AM EDT
Active, not recruiting Phase 3 Interventional

A Clinical Evaluation of Non-Invasive Vagus Nerve Stimulation for Temper Outbursts in People With PWS

NCT06144645
View directory record Official record
Conditions
Prader-Willi Syndrome
Interventions
tVNS, intermittent stimulation, tVNS, continuous stimulation
Device
Lead sponsor
Foundation for Prader-Willi Research
Other
Eligibility
10 Years to 40 Years
Enrollment
102 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
18
States / cities
Palo Alto, California • San Diego, California • Aurora, Colorado + 14 more
Source: ClinicalTrials.gov public record
Updated Apr 5, 2026 · Synced Jun 11, 2026, 1:47 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Evaluation and Treatment of Patients With Connective Tissue Disease

NCT00076830
View directory record Official record
Conditions
Connective Tissue Disorders, Osteogenesis Imperfecta, Bone Diseases, Metabolic, Melorheostosis
Interventions
Not listed
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
Up to 70 Years
Enrollment
180 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 2004
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jun 7, 2026 · Synced Jun 11, 2026, 1:47 AM EDT
Active, not recruiting Phase 3 Interventional

Trial of Dichloroacetate in Pyruvate Dehydrogenase Complex Deficiency:

NCT02616484
View directory record Official record
Conditions
Pyruvate Dehydrogenase Complex Deficiency
Interventions
Dichloroacetate (DCA), Placebo, Genotype
Drug · Other · Genetic
Lead sponsor
Saol Therapeutics Inc
Industry
Eligibility
6 Months to 17 Years
Enrollment
34 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2025
U.S. locations
10
States / cities
Orange, California • Stanford, California • Washington D.C., District of Columbia + 7 more
Source: ClinicalTrials.gov public record
Updated May 3, 2025 · Synced Jun 11, 2026, 1:47 AM EDT
Completed Phase 3 Interventional Results available

Atorvastatin Three Year Pediatric Study

NCT00827606
View directory record Official record
Conditions
Familial Hypercholesterolemia
Interventions
atorvastatin
Drug
Lead sponsor
Pfizer's Upjohn has merged with Mylan to form Viatris Inc.
Industry
Eligibility
6 Years to 15 Years
Enrollment
272 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2013
U.S. locations
7
States / cities
Phoenix, Arizona • Washington D.C., District of Columbia • Gainesville, Florida + 3 more
Source: ClinicalTrials.gov public record
Updated Feb 20, 2021 · Synced Jun 11, 2026, 1:47 AM EDT
Completed Phase 1Phase 2 Interventional Results available

An Open-label Safety, Pharmacokinetic, and Efficacy Study of Miglustat for the Treatment of Subjects With Batten Ceroid Lipofuscinosis, Neuronal 3 (CLN3) Disease

NCT05174039
View directory record Official record
Conditions
Batten Disease
Interventions
Miglustat 100 milligrams (mg) Oral Capsule
Drug
Lead sponsor
Beyond Batten Disease Foundation
Other
Eligibility
17 Years and older
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Sep 8, 2025 · Synced Jun 11, 2026, 1:47 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Frequency of Endometrial Cancer Precursors Associated with Lynch Syndrome

NCT05257057
View directory record Official record
Conditions
Lynch Syndrome, Endometrial Cancer, Endometrial Hyperplasia, Mismatch Repair Deficiency, Microsatellite Instability
Interventions
Immunohistochemical staining
Diagnostic Test
Lead sponsor
WellSpan Health
Other
Eligibility
18 Years to 99 Years · Female only
Enrollment
91 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2023
U.S. locations
1
States / cities
York, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 27, 2025 · Synced Jun 11, 2026, 1:47 AM EDT
Completed Phase 4 Interventional Results available

Study of KRYSTEXXA® (Pegloticase) Plus Methotrexate in Participants With Uncontrolled Gout

NCT03994731
View directory record Official record
Conditions
Gout
Interventions
Pegloticase, methotrexate, placebo, folic acid, gout flare prophylaxis regimen, fexofenadine, acetaminophen, methylprednisolone
Biological · Drug · Dietary Supplement
Lead sponsor
Amgen
Industry
Eligibility
18 Years and older
Enrollment
152 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
60
States / cities
Birmingham, Alabama • Anchorage, Alaska • Flagstaff, Arizona + 54 more
Source: ClinicalTrials.gov public record
Updated Jun 25, 2024 · Synced Jun 11, 2026, 1:47 AM EDT
Completed Phase 2 Interventional Results available

Efficacy and Safety of Deferasirox in Non-transfusion Dependent Thalassemia Patients With Iron Overload and a One Year Open-label Extension Study

NCT00873041
View directory record Official record
Conditions
Non-transfusion Dependent Thalassemia
Interventions
deferasirox, placebo
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
10 Years and older
Enrollment
166 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2012
U.S. locations
3
States / cities
Oakland, California • Chicago, Illinois • New York, New York
Source: ClinicalTrials.gov public record
Updated Jul 8, 2013 · Synced Jun 11, 2026, 1:47 AM EDT
Completed No phase listed Observational

Health-related Quality of Life in Rare Kidney Stone

NCT02124395
View directory record Official record
Conditions
Primary Hyperoxaluria, Cystinuria, Adenine Phosphoribosyl Transferase Deficiency, Dent Disease
Interventions
Health-related Quality of Life in Rare Kidney Stone Questionnaire
Other
Lead sponsor
NYU Langone Health
Other
Eligibility
5 Years and older
Enrollment
365 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2022
U.S. locations
2
States / cities
Rochester, Minnesota • New York, New York
Source: ClinicalTrials.gov public record
Updated Sep 1, 2022 · Synced Jun 11, 2026, 1:47 AM EDT