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ClinicalTrials.gov public records Last synced Oct 15, 2026, 3:24 PM EDT

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

Showing 649–672 of 1,709 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Central Nervous System Anomalies Clear all
Active, not recruiting No phase listed Observational Accepts healthy volunteers

Phenotype, Genotype and Biomarkers 2

NCT04875416
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis, Hereditary Spastic Paraplegia, Primary Lateral Sclerosis, Progressive Muscular Atrophy, Frontotemporal Dementia
Interventions
Not listed
Lead sponsor
University of Miami
Other
Eligibility
7 Years and older
Enrollment
217 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2031
U.S. locations
3
States / cities
Miami, Florida • Kansas City, Kansas • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jun 15, 2026 · Synced Oct 15, 2026, 3:24 PM EDT
Completed Phase 3 Interventional Results available

Finding the Optimum Regimen for Duchenne Muscular Dystrophy

NCT01603407
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Prednisone, Deflazacort
Drug
Lead sponsor
University of Rochester
Other
Eligibility
4 Years to 7 Years · Male only
Enrollment
196 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2019
U.S. locations
14
States / cities
Los Angeles, California • Sacramento, California • Washington D.C., District of Columbia + 11 more
Source: ClinicalTrials.gov public record
Updated Aug 12, 2022 · Synced Oct 15, 2026, 3:24 PM EDT
Completed Phase 2 Interventional Results available

Phase 2 Basket Trial of Nab-sirolimus in Patients With Malignant Solid Tumors With Pathogenic Alterations in TSC1/TSC2 Genes (PRECISION 1)

NCT05103358
View directory record Official record
Conditions
Tumor, Tumor, Solid, Metastasis, Metastatic Cancer, Cancer, Cancer Metastatic, Tumors, Neoplasms, Neoplasm Metastasis, Solid Tumor, Advanced Solid Tumor, Advanced Cancer, Malignant Solid Tumor, Malignant Solid Neoplasm, Malignant Neoplasm, Malignant Tumor, TSC, TSC1, TSC2, Metastatic Solid Tumor, Metastatic Neoplasm
Interventions
nab-sirolimus
Drug
Lead sponsor
Aadi Bioscience, Inc.
Industry
Eligibility
12 Years and older
Enrollment
128 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
159
States / cities
Birmingham, Alabama • Mobile, Alabama • Goodyear, Arizona + 137 more
Source: ClinicalTrials.gov public record
Updated Sep 28, 2026 · Synced Oct 15, 2026, 3:24 PM EDT
Completed No phase listed Observational

Cardiac Rhythm Abnormalities in Patients With Refractory Epilepsy at High Risk for Sudden Death

NCT00203333
View directory record Official record
Conditions
Refractory Epilepsy, Arrhythmia, Sudden Death
Interventions
Medtronic REVEAL
Device
Lead sponsor
Thomas Jefferson University
Other
Eligibility
16 Years to 49 Years
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2010
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Oct 18, 2016 · Synced Oct 15, 2026, 3:24 PM EDT
Completed Phase 2 Interventional Results available

A Study of AT2101 (Afegostat Tartrate) in Adult Patients With Type 1 Gaucher Disease Currently Receiving Enzyme Replacement Therapy

NCT00433147
View directory record Official record
Conditions
Gaucher Disease, Type 1, Type 1 Gaucher Disease, Gaucher Disease
Interventions
Afegostat tartrate
Drug
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
18 Years to 74 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2008
U.S. locations
10
States / cities
San Francisco, California • Coral Springs, Florida • Decatur, Georgia + 7 more
Source: ClinicalTrials.gov public record
Updated Sep 25, 2018 · Synced Oct 15, 2026, 3:24 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

PET Scans in Normal Volunteers and Patients With Fabry Disease

NCT00005111
View directory record Official record
Conditions
Fabry Disease, Healthy
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Male only
Enrollment
24 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2000 – 2001
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 15, 2026, 3:24 PM EDT
Completed Not applicable Interventional

Body Weight Support Harness System in Spinal Muscular Atrophy

NCT05715749
View directory record Official record
Conditions
Spinal Muscular Atrophy Type I, Spinal Muscular Atrophy Type II
Interventions
In-home body weight support harness system
Other
Lead sponsor
Nationwide Children's Hospital
Other
Eligibility
Not listed
Enrollment
33 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2024
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Jul 22, 2024 · Synced Oct 15, 2026, 3:24 PM EDT
Completed Phase 2 Interventional Results available

Safety and Efficacy of Vicriviroc (MK-7690) in Combination With Pembrolizumab (MK-3475) in Participants With Advanced/Metastatic Microsatellite Stable (MSS) Colorectal Cancer (CRC) (MK-7690-046)

NCT03631407
View directory record Official record
Conditions
Colorectal Neoplasms
Interventions
Vicriviroc, Pembrolizumab
Drug · Biological
Lead sponsor
Merck Sharp & Dohme LLC
Industry
Eligibility
18 Years and older
Enrollment
41 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
6
States / cities
Scottsdale, Arizona • Encinitas, California • Fresno, California + 3 more
Source: ClinicalTrials.gov public record
Updated Nov 21, 2024 · Synced Oct 15, 2026, 3:24 PM EDT
Completed No phase listed Observational

Fabry Outcome Survey (FOS)

NCT03289065
View directory record Official record
Conditions
Fabry Disease
Interventions
Not listed
Lead sponsor
Shire
Industry
Eligibility
Not listed
Enrollment
4,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2021
U.S. locations
1
States / cities
Lexington, Massachusetts
Source: ClinicalTrials.gov public record
Updated Nov 16, 2021 · Synced Oct 15, 2026, 3:24 PM EDT
Completed Phase 3 Interventional Results available

Opioid Titration Order Sheet or Standard Care in Treating Patients With Cancer Pain

NCT00666211
View directory record Official record
Conditions
Brain and Central Nervous System Tumors, Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Lymphoproliferative Disorder, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasms, Pain, Precancerous Condition, Unspecified Adult Solid Tumor, Protocol Specific
Interventions
educational intervention, Titrated pain management, questionnaire administration
Other
Lead sponsor
Vanderbilt-Ingram Cancer Center
Other
Eligibility
21 Years and older
Enrollment
98 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2010
U.S. locations
11
States / cities
Hopkinsville, Kentucky • Owensboro, Kentucky • Chattanooga, Tennessee + 4 more
Source: ClinicalTrials.gov public record
Updated Sep 6, 2012 · Synced Oct 15, 2026, 3:24 PM EDT
Recruiting No phase listed Observational

PheCheck™ Validation Study

NCT07551921
View directory record Official record
Conditions
Phenylketonuria (PKU) and Hyperphenylalaninemia
Interventions
PheCheck, Dried Blood Spot Cards.
Device · Diagnostic Test
Lead sponsor
Aptatek BioSciences, Inc
Industry
Eligibility
1 Year and older
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2027
U.S. locations
2
States / cities
Chicago, Illinois • Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Aug 25, 2026 · Synced Oct 15, 2026, 3:24 PM EDT
Completed Phase 1 Interventional

Phase I Trial of Tariquidar (XR9576) in Combination With Doxorubicin, Vinorelbine, or Docetaxel in Pediatric Patients With Solid Tumors

NCT00011414
View directory record Official record
Conditions
Wilms' Tumor, Sarcoma, Adenaocortical Carcinoma, Refractory Cancer, Coldrhood Cancer
Interventions
Tariquidar
Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
2 Years to 18 Years
Enrollment
29 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2016
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 16, 2019 · Synced Oct 15, 2026, 3:24 PM EDT
No Longer Available No phase listed Expanded access

131I-omburtamab for the Treatment of Central Nervous System/Leptomeningeal Neoplasms in Children and Young Adults

NCT05064306
View directory record Official record
Conditions
Central Nervous System/Leptomeningeal Neoplasms
Interventions
131I-omburtamab
Drug
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
Not listed
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Aug 1, 2025 · Synced Oct 15, 2026, 3:24 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

Safety, Tolerability and Pharmacodynamics of SYNB1353 in Healthy Adult Volunteers

NCT05462132
View directory record Official record
Conditions
Homocystinuria
Interventions
SYNB1353
Drug
Lead sponsor
Synlogic
Industry
Eligibility
18 Years to 64 Years
Enrollment
31 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022
U.S. locations
1
States / cities
High Point, North Carolina
Source: ClinicalTrials.gov public record
Updated Mar 17, 2023 · Synced Oct 15, 2026, 3:24 PM EDT
Terminated No phase listed Observational Accepts healthy volunteers

Biological Sample Collection for Research and Biobanking

NCT04270604
View directory record Official record
Conditions
Parkinson Disease, Diabetes Mellitus, Type 1, Macular Degeneration, ALS, Cancer, Alzheimer Disease, Huntington Disease
Interventions
Biological sample collection
Other
Lead sponsor
New York Stem Cell Foundation Research Institute
Other
Eligibility
1 Month to 100 Years
Enrollment
1,279 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2023
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Aug 2, 2024 · Synced Oct 15, 2026, 3:24 PM EDT
Completed Not applicable Interventional

Time-Restricted Eating in Huntington's Disease: A Clinical Pilot Study

NCT06490367
View directory record Official record
Conditions
Huntington Disease
Interventions
Time-Restricted Eating Diet
Behavioral
Lead sponsor
Oregon Health and Science University
Other
Eligibility
21 Years and older
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2025
U.S. locations
1
States / cities
Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Jun 4, 2025 · Synced Oct 15, 2026, 3:24 PM EDT
Completed Phase 2Phase 3 Interventional Results available

Dose-Ranging Efficacy and Safety Study of Topical Rapamycin Cream for Facial Angiofibroma Associated With Tuberous Sclerosis Complex

NCT03826628
View directory record Official record
Conditions
Facial Angiofibroma, Tuberous Sclerosis
Interventions
rapamycin, placebo
Drug
Lead sponsor
Dermatology Specialties Limited Partnership
Industry
Eligibility
6 Years to 65 Years
Enrollment
107 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
6
States / cities
Phoenix, Arizona • La Jolla, California • St. Petersburg, Florida + 3 more
Source: ClinicalTrials.gov public record
Updated Sep 8, 2023 · Synced Oct 15, 2026, 3:24 PM EDT
Recruiting No phase listed Observational

Nutritional Impacts of Palynziq on Patients With Phenylketonuria (PKU)

NCT04404530
View directory record Official record
Conditions
Phenylketonurias
Interventions
Palynziq
Drug
Lead sponsor
Emory University
Other
Eligibility
16 Years and older
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2028
U.S. locations
2
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Jan 14, 2026 · Synced Oct 15, 2026, 3:24 PM EDT
Active, not recruiting No phase listed Observational Accepts healthy volunteers

Diffusion Tensor Imaging (DTI) in Infants With Krabbe Disease

NCT00787865
View directory record Official record
Conditions
Krabbe Disease
Interventions
Not listed
Lead sponsor
University of Pittsburgh
Other
Eligibility
Up to 17 Years
Enrollment
100 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2008 – 2027
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 6, 2026 · Synced Oct 15, 2026, 3:24 PM EDT
Completed Phase 3 Interventional Results available

A Study to Assess Efficacy, Long Term Safety and Tolerability of RT001 in Subjects With Friedreich's Ataxia

NCT04102501
View directory record Official record
Conditions
Friedreich's Ataxia
Interventions
RT001, Placebo
Drug
Lead sponsor
Biojiva LLC
Industry
Eligibility
12 Years to 50 Years
Enrollment
65 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
6
States / cities
Long Beach, California • Los Angeles, California • Gainesville, Florida + 3 more
Source: ClinicalTrials.gov public record
Updated May 27, 2022 · Synced Oct 15, 2026, 3:24 PM EDT
Completed No phase listed Observational

Antiproteinuric Agents and Fabry Disease

NCT00343577
View directory record Official record
Conditions
Fabry Disease, Proteinuria
Interventions
Not listed
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
14 Years to 95 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2006
U.S. locations
1
States / cities
Birmingham, Alabama
Source: ClinicalTrials.gov public record
Updated Nov 19, 2013 · Synced Oct 15, 2026, 3:24 PM EDT
Completed Not applicable Interventional

Evaluation of an Online Comprehensive Behavioral Intervention for Tics (CBIT) Therapist Training Program

NCT05547854
View directory record Official record
Conditions
Tics, Tic Disorders, Tourette Syndrome
Interventions
Behavior Therapy Training Institute, CBIT-Trainer
Behavioral
Lead sponsor
University of Utah
Other
Eligibility
7 Years and older
Enrollment
284 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
2
States / cities
Salt Lake City, Utah • Milwaukee, Wisconsin
Source: ClinicalTrials.gov public record
Updated Oct 5, 2026 · Synced Oct 15, 2026, 3:24 PM EDT
Withdrawn Phase 4 Interventional

Pharmacokinetics, Pharmacodynamics And Safety Study Of Elelyso(tm) In Pediatric Subjects With Type 1 Gaucher Disease

NCT03021941
View directory record Official record
Conditions
Type 1 Gaucher Disease
Interventions
Elelyso 60 units/kg
Drug
Lead sponsor
Pfizer
Industry
Eligibility
Up to 12 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2020
U.S. locations
1
States / cities
Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Sep 24, 2019 · Synced Oct 15, 2026, 3:24 PM EDT