Independent directory Public ClinicalTrials.gov records United States
Clear filters

These results were retrieved from the live ClinicalTrials.gov registry. Filters and sort order apply to this result list.

ClinicalTrials.gov public records Last synced Oct 14, 2026, 5:59 AM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Showing 289–312 of 1,708 matching trials from the live ClinicalTrials.gov search.
Conditions
Phenylketonuria, PKU
Interventions
Acute Moderate Intensity Exercise
Other
Lead sponsor
Oregon Health and Science University
Other
Eligibility
14 Years to 17 Years · Male only
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2014
U.S. locations
1
States / cities
Portland, Oregon
Conditions
Advanced Cancer, Metastatic Cancer, Ovarian Cancer, Primary Peritoneal Cancer, Fallopian Tube Cancer, Endometrial Cancer, Metastatic Colorectal Cancer, Pancreatic Ductal Adenocarcinoma, Acinar Cell Carcinoma
Interventions
ART0380, Gemcitabine, Irinotecan
Drug
Lead sponsor
Artios Pharma Ltd
Industry
Eligibility
18 Years and older
Enrollment
542 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2028
U.S. locations
41
States / cities
Birmingham, Alabama • Scottsdale, Arizona • Little Rock, Arkansas + 33 more
Conditions
Tourette Disorder, Chronic Tic Disorder
Interventions
D-cycloserine, Placebo
Drug
Lead sponsor
University of California, Los Angeles
Other
Eligibility
8 Years to 17 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
1
States / cities
Los Angeles, California
Conditions
Amyloidosis, Amyloid Cardiomyopathy, Transthyretin Amyloidosis, Cardiomyopathies, Heart Diseases
Interventions
acoramidis, Placebo Oral Tablet
Drug
Lead sponsor
Eidos Therapeutics, a BridgeBio company
Industry
Eligibility
18 Years to 90 Years
Enrollment
632 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2023
U.S. locations
40
States / cities
Los Angeles, California • Santa Monica, California • Aurora, Colorado + 32 more
Conditions
Glycogen Storage Disease Type II, Pompe Disease, Acid Maltase Deficiency Disease, Glycogenosis 2
Interventions
Alglucosidase alfa
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
Not listed
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003
U.S. locations
1
States / cities
New Brunswick, New Jersey
Conditions
Huntington's Disease
Interventions
Not listed
Lead sponsor
Ohio State University
Other
Eligibility
18 Years and older
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2013
U.S. locations
1
States / cities
Columbus, Ohio
Conditions
Pregnancy, Phenylketonuria
Interventions
Metabolic camp
Behavioral
Lead sponsor
Emory University
Other
Eligibility
11 Years and older · Female only
Enrollment
200 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
1995 – 2037
U.S. locations
1
States / cities
Atlanta, Georgia
Conditions
Advanced Malignant Solid Neoplasm, Recurrent Childhood Ependymoma, Recurrent Childhood Malignant Germ Cell Tumor, Recurrent Childhood Medulloblastoma, Recurrent Childhood Non-Hodgkin Lymphoma, Recurrent Childhood Osteosarcoma, Recurrent Childhood Rhabdomyosarcoma, Recurrent Childhood Soft Tissue Sarcoma, Recurrent Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor, Recurrent Hepatoblastoma, Recurrent Langerhans Cell Histiocytosis, Recurrent Malignant Glioma, Recurrent Malignant Solid Neoplasm, Recurrent Neuroblastoma, Recurrent Primary Central Nervous System Neoplasm, Recurrent Rhabdoid Tumor, Refractory Childhood Malignant Germ Cell Tumor, Refractory Childhood Osteosarcoma, Refractory Childhood Rhabdomyosarcoma, Refractory Childhood Soft Tissue Sarcoma, Refractory Ependymoma, Refractory Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor, Refractory Hepatoblastoma, Refractory Langerhans Cell Histiocytosis, Refractory Malignant Glioma, Refractory Malignant Solid Neoplasm, Refractory Medulloblastoma, Refractory Neuroblastoma, Refractory Non-Hodgkin Lymphoma, Refractory Primary Central Nervous System Neoplasm, Refractory Rhabdoid Tumor, Wilms Tumor
Interventions
Biospecimen Collection, Bone Marrow Aspiration and Biopsy, Bone Scan, Computed Tomography, Erdafitinib, Laboratory Biomarker Analysis, Magnetic Resonance Imaging, Pharmacological Study, Positron Emission Tomography, Radionuclide Imaging, X-Ray Imaging
Procedure · Drug · Other
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
12 Months to 21 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2026
U.S. locations
114
States / cities
Birmingham, Alabama • Anchorage, Alaska • Mesa, Arizona + 91 more
Conditions
Macular Degeneration
Interventions
intravitreal injection Lucentis (ranibizumab)
Drug
Lead sponsor
Retina Research Foundation
Other
Eligibility
50 Years to 90 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007
U.S. locations
1
States / cities
Slingerlands, New York
Conditions
Gaucher's Disease
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Not listed
Enrollment
70 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1991 – 2008
U.S. locations
1
States / cities
Bethesda, Maryland
Conditions
Lennox Gastaut Syndrome (LGS)
Interventions
Placebo, Soticlestat
Drug
Lead sponsor
Takeda
Industry
Eligibility
2 Years to 55 Years
Enrollment
270 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
24
States / cities
Phoenix, Arizona • Tucson, Arizona • Los Angeles, California + 17 more
Conditions
Fabry Disease, Fabry Disease, Cardiac Variant
Interventions
ST-920
Biological
Lead sponsor
Sangamo Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2039
U.S. locations
8
States / cities
Irvine, California • Tampa, Florida • Atlanta, Georgia + 5 more
Conditions
Mucopolysaccharidosis Disorders, Hurler Syndrome, Hunter Syndrome, Maroteaux Lamy Syndrome, Sly Syndrome, Alpha-Mannosidosis, Fucosidosis, Aspartylglucosaminuria, Glycoprotein Metabolic Disorders, Sphingolipidoses, Recessive Leukodystrophies, Globoid Cell Leukodystrophy, Metachromatic Leukodystrophy, Niemann-Pick B, Niemann-Pick C Subtype 2, Sphingomyelin Deficiency, Peroxisomal Disorders, Adrenoleukodystrophy With Cerebral Involvement, Zellweger Syndrome, Neonatal Adrenoleukodystrophy, Infantile Refsum Disease, Acyl-CoA Oxidase Deficiency, D-Bifunctional Enzyme Deficiency, Multifunctional Enzyme Deficiency, Alpha-methylacyl-CoA Racmase Deficiency, Mitochondrial Neurogastrointestingal Encephalopathy, Severe Osteopetrosis, Hereditary Leukoencephalopathy With Axonal Spheroids (HDLS; CSF1R Mutation), Inherited Metabolic Disorders
Interventions
Stem Cell Transplantation, IMD Preparative Regimen, Osteopetrosis Only Preparative Regimen, Osteopetrosis Haploidentical Only Preparative Regimen, cALD SR-A (Standard-Risk, Regimen A), cALD SR-B (Standard-Risk, Regimen B), cALD HR-D (High-Risk, Regimen C), cALD HR-D (High-Risk, Regimen D)
Biological · Drug
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 55 Years
Enrollment
149 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2029
U.S. locations
1
States / cities
Minneapolis, Minnesota
Enrolling by invitation No phase listed Observational

Posterior Cervical Fixation Study

NCT04770571
Conditions
Degenerative Disc Disease, Craniocervical Injuries, Cervical Radiculopathy, Cervical Disc Disease, Cervical Fusion, Cervical Spine Disease, Cervical Myelopathy, Cervical Instabilities Spine, Thoracic Injury
Interventions
observational study
Device
Lead sponsor
NuVasive
Industry
Eligibility
18 Years and older
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2028
U.S. locations
7
States / cities
Orange, California • Hartford, Connecticut • Hyattsville, Maryland + 4 more
Recruiting No phase listed Observational Accepts healthy volunteers

Biomarkers in Friedreich's Ataxia

NCT02497534
Conditions
Friedreich's Ataxia
Interventions
Not listed
Lead sponsor
University of Florida
Other
Eligibility
6 Years to 70 Years
Enrollment
203 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2030
U.S. locations
1
States / cities
Gainesville, Florida
Completed No phase listed Observational

Pompe Telemedicine Developmental Study

NCT02950298
Conditions
Pompe Disease, Glycogen Storage Disease II
Interventions
Not listed
Lead sponsor
Duke University
Other
Eligibility
6 Years to 18 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2021
U.S. locations
1
States / cities
Durham, North Carolina
Completed Phase 2Phase 3 Interventional Results available

Trial in Adult Participants With Spinocerebellar Ataxia (SCA)

NCT02960893
Conditions
Spinocerebellar Ataxias, Spinocerebellar Ataxia Genotype Type 1, Spinocerebellar Ataxia Genotype Type 2, Spinocerebellar Ataxia Genotype Type 3, Spinocerebellar Ataxia Genotype Type 6, Spinocerebellar Ataxia Genotype Type 7, Spinocerebellar Ataxia Genotype Type 8, Spinocerebellar Ataxia Genotype Type 10
Interventions
Troriluzole, Placebo
Drug
Lead sponsor
Biohaven Pharmaceuticals, Inc.
Industry
Eligibility
18 Years to 75 Years
Enrollment
141 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2024
U.S. locations
18
States / cities
Phoenix, Arizona • Long Beach, California • Los Angeles, California + 13 more
Conditions
Friedreich Ataxia, Healthy Volunteers
Interventions
Buccal Swabs, Blood Draws and Skin Punch Biopsy
Diagnostic Test
Lead sponsor
Larimar Therapeutics, Inc.
Industry
Eligibility
18 Years to 50 Years
Enrollment
60 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2022
U.S. locations
1
States / cities
Eatontown, New Jersey
Conditions
Tourette Syndrome
Interventions
pramipexole 0.125 mg BID, pramipexole 0.0625 mg QD, pramipexole 0.125 mg TID, pramipexole 0.25 mg BID, pramipexole 0.0625 mg BID
Drug
Lead sponsor
Boehringer Ingelheim
Industry
Eligibility
6 Years to 17 Years
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2008
U.S. locations
13
States / cities
Bradenton, Florida • Tampa, Florida • Columbus, Georgia + 9 more
Terminated Phase 3 Interventional Results available

Open-Label Phase 3 Long-Term Safety Study of Migalastat

NCT01458119
Conditions
Fabry Disease
Interventions
migalastat hydrochloride
Drug
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
16 Years and older
Enrollment
85 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2016
U.S. locations
11
States / cities
Atlanta, Georgia • Chicago, Illinois • Kansas City, Kansas + 8 more
Active, not recruiting No phase listed Observational Accepts healthy volunteers

Wearable Gait Sensors

NCT06628934
Conditions
Spinocerebellar Ataxia
Interventions
Not listed
Lead sponsor
University of Chicago
Other
Eligibility
18 Years to 75 Years
Enrollment
600 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2017 – 2026
U.S. locations
1
States / cities
Chicago, Illinois
Conditions
Fabry Disease
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Female only
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2003
U.S. locations
1
States / cities
Bethesda, Maryland
Active, not recruiting Not applicable Interventional

Grand Valley State University (GVSU) Skills on Wheels

NCT05339932
Conditions
Spina Bifida, Cerebral Palsy, Genetic Disease, Spinal Cord Injuries, Gross Motor Development Delay, Motor Skills Disorders
Interventions
Manual Wheelchair Skills Training
Other
Lead sponsor
Grand Valley State University
Other
Eligibility
5 Years to 17 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
1
States / cities
Grand Rapids, Michigan
Conditions
Pregnant Women, Prenatal Care
Interventions
Not listed
Lead sponsor
Verinata Health, Inc.
Industry
Eligibility
18 Years and older · Female only
Enrollment
10,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2011
U.S. locations
11
States / cities
Los Angeles, California • San Francisco, California • Fort Collins, Colorado + 8 more