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Showing 1–24 of 1,247 matching trials from the live ClinicalTrials.gov search.
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Completed Early Phase 1 Interventional

PET/MRI Evaluation of Cardiac Amyloid

NCT03119558
View directory record Official record
Conditions
Cardiac Amyloidosis
Interventions
18F-Florbetaben (Neuraceq®) PET/MRI
Drug
Lead sponsor
Stanford University
Other
Eligibility
18 Years and older
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2018
U.S. locations
1
States / cities
Stanford, California
Source: ClinicalTrials.gov public record
Updated Jul 16, 2019 · Synced Jun 10, 2026, 6:14 PM EDT
Recruiting Not applicable Interventional

A Study to Evaluate the Feasibility of a Physiologic Biomarker to Assess Pain and Other Sensory Problems Using Pupillometry in Participants With Neurofibromatosis Type 1 (NF1)

NCT06507748
View directory record Official record
Conditions
Neurofibromatosis Type 1
Interventions
AlgometRx Nociometer
Device
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
1 Year to 120 Years
Enrollment
70 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2027
U.S. locations
2
States / cities
Washington D.C., District of Columbia • Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jun 8, 2026 · Synced Jun 10, 2026, 6:14 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Genetics of Charcot Marie Tooth (CMT) - Modifiers of CMT1A, New Causes of CMT2

NCT01193088
View directory record Official record
Conditions
Charcot-Marie-Tooth Disease, Type Ia (Disorder), HMSN
Interventions
Not listed
Lead sponsor
University of Iowa
Other
Eligibility
Not listed
Enrollment
1,050 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2010 – 2026
U.S. locations
17
States / cities
Los Angeles, California • Palo Alto, California • Aurora, Colorado + 13 more
Source: ClinicalTrials.gov public record
Updated Oct 6, 2025 · Synced Jun 10, 2026, 6:14 PM EDT
Completed Early Phase 1 Interventional

Methylprednisolone Treatment of Friedreich Ataxia

NCT02424435
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
Methylprednisolone
Drug
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
5 Years and older
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2018
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 24, 2021 · Synced Jun 10, 2026, 6:14 PM EDT
Completed No phase listed Observational

Eligibility Screening for a NCI Pediatric Oncology Branch Research Study

NCT00026780
View directory record Official record
Conditions
Ewing Sarcoma, Osteosarcoma, Neuroblastoma, Acute Lymphoblastic Leukemia, Neurofibromatosis Type 1
Interventions
Not listed
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
Up to 35 Years
Enrollment
1,720 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2020
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 7, 2020 · Synced Jun 10, 2026, 6:14 PM EDT
Completed Phase 2 Interventional Results available

Interferon Gamma-1b in Friedreich Ataxia (FRDA)

NCT01965327
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
Interferon Gamma-1b
Drug
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
5 Years to 17 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2014
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 12, 2021 · Synced Jun 10, 2026, 6:14 PM EDT
Completed Phase 3 Interventional Results available

Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome (LAVENDER™)

NCT04181723
View directory record Official record
Conditions
Rett Syndrome
Interventions
Trofinetide, Placebo
Drug · Other
Lead sponsor
ACADIA Pharmaceuticals Inc.
Industry
Eligibility
5 Years to 20 Years · Female only
Enrollment
187 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
21
States / cities
Birmingham, Alabama • Phoenix, Arizona • La Jolla, California + 18 more
Source: ClinicalTrials.gov public record
Updated Apr 7, 2024 · Synced Jun 10, 2026, 6:14 PM EDT
Completed No phase listed Observational

Neurofibromatosis Type 1 (NF1) and Tibial Dysplasia

NCT00303368
View directory record Official record
Conditions
Neurofibromatosis Type 1, Tibial Dysplasia
Interventions
Not listed
Lead sponsor
Shriners Hospitals for Children
Other
Eligibility
Not listed
Enrollment
395 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2016
U.S. locations
1
States / cities
Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated Feb 24, 2019 · Synced Jun 10, 2026, 6:14 PM EDT
Terminated Phase 1 Interventional

A Pharmacokinetics and Safety Study of BIIB132 in Adults With Spinocerebellar Ataxia 3

NCT05160558
View directory record Official record
Conditions
Spinocerebellar Ataxia Type 3
Interventions
BIIB132, BIIB132-Matching Placebo
Drug
Lead sponsor
Biogen
Industry
Eligibility
18 Years to 70 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2023
U.S. locations
10
States / cities
Los Angeles, California • San Francisco, California • Gainesville, Florida + 7 more
Source: ClinicalTrials.gov public record
Updated Aug 3, 2023 · Synced Jun 10, 2026, 6:14 PM EDT
Recruiting No phase listed Observational

Tuberous Sclerosis Complex and Lymphangioleiomyomatosis Pregnancy Registry (TSC-LAM Registry)

NCT06160310
View directory record Official record
Conditions
Tuberous Sclerosis Complex, Lymphangioleiomyomatosis
Interventions
Not listed
Lead sponsor
David M. Ritter
Other
Eligibility
Female only
Enrollment
300 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2029
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Jan 22, 2026 · Synced Jun 10, 2026, 6:14 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers Results available

Single and Multiple Ascending Dose Study of MTR-601 in Healthy Individuals

NCT06117020
View directory record Official record
Conditions
Muscle Spasticity, Hypertonia, Muscle, Cerebral Palsy, Multiple Sclerosis, Stroke, Hereditary Spastic Paraplegia, Dystonia, Spinal Cord Injuries
Interventions
MTR-601
Drug
Lead sponsor
Motric Bio
Industry
Eligibility
18 Years to 45 Years
Enrollment
89 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2023 – 2024
U.S. locations
1
States / cities
San Antonio, Texas
Source: ClinicalTrials.gov public record
Updated Jun 4, 2026 · Synced Jun 10, 2026, 6:14 PM EDT
Withdrawn Phase 1Phase 2 Interventional

Safety and Efficacy Study of Neratinib and Cetuximab to Treat Patients With Quadruple Wild-Type Metastatic Colorectal Cancer

NCT01960023
View directory record Official record
Conditions
Colorectal Cancer
Interventions
Cetuximab, Neratinib
Drug
Lead sponsor
NSABP Foundation Inc
Network
Eligibility
18 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2016
U.S. locations
12
States / cities
Gainesville, Florida • Orlando, Florida • Boise, Idaho + 7 more
Source: ClinicalTrials.gov public record
Updated Oct 26, 2021 · Synced Jun 10, 2026, 6:14 PM EDT
Completed Phase 1 Interventional

Open-Trial of EPI-743 for Adults With Tourette Syndrome

NCT01719523
View directory record Official record
Conditions
Tourette Syndrome
Interventions
EPI-743
Drug
Lead sponsor
Yale University
Other
Eligibility
18 Years to 65 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2013
U.S. locations
1
States / cities
New Haven, Connecticut
Source: ClinicalTrials.gov public record
Updated Mar 4, 2014 · Synced Jun 10, 2026, 6:14 PM EDT
Terminated Phase 2 Interventional

PTC299 for Treatment of Neurofibromatosis Type 2

NCT00911248
View directory record Official record
Conditions
Neurofibromatosis 2
Interventions
PTC299
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2012
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Apr 11, 2019 · Synced Jun 10, 2026, 6:14 PM EDT
Withdrawn No phase listed Observational

Breast Cancer BRCA1 Carriers: a Pilot Study

NCT05062174
View directory record Official record
Conditions
BRCA1 Mutation, High-grade Serous Ovarian Cancer, TNBC - Triple-Negative Breast Cancer
Interventions
Mifepristone 200 MG, Prophylactic mastectomy
Drug · Procedure
Lead sponsor
Indiana University
Other
Eligibility
18 Years and older · Female only
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
2
States / cities
Carmel, Indiana • Indianapolis, Indiana
Source: ClinicalTrials.gov public record
Updated Dec 14, 2022 · Synced Jun 10, 2026, 6:14 PM EDT
Withdrawn Not applicable Interventional

Turmeric as Treatment in Epilepsy

NCT03254680
View directory record Official record
Conditions
Epilepsy, Dravet Syndrome, Lennox-Gastaut Syndrome, Tuberous Sclerosis, Focal Seizures
Interventions
Turmeric
Dietary Supplement
Lead sponsor
NYU Langone Health
Other
Eligibility
1 Year to 70 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Apr 29, 2018 · Synced Jun 10, 2026, 6:14 PM EDT
Completed Phase 3 Interventional

A Safety and Efficacy Study of Dimebon in Patients With Huntington Disease

NCT00920946
View directory record Official record
Conditions
Huntington Disease
Interventions
Dimebon, Placebo
Drug · Other
Lead sponsor
Medivation, Inc.
Industry
Eligibility
30 Years and older
Enrollment
403 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2009
U.S. locations
24
States / cities
Scottsdale, Arizona • Davis, California • Irvine, California + 21 more
Source: ClinicalTrials.gov public record
Updated Oct 11, 2016 · Synced Jun 10, 2026, 6:14 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Caregiving Networks Across Disease Context and the Life Course

NCT05007990
View directory record Official record
Conditions
Inherited Metabolic Disorders, Undiagnosed Diseases, Batten's Disease, Tay Sachs, Diabetes
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
18 Years to 100 Years
Enrollment
2,800 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2030
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jun 7, 2026 · Synced Jun 10, 2026, 6:14 PM EDT
Completed Not applicable Interventional

Incorporating teleCBIT Into a Hospital-Based Tic Program

NCT04007913
View directory record Official record
Conditions
Tic Disorders, Tourette Syndrome
Interventions
teleCBIT
Behavioral
Lead sponsor
San Jose State University
Other
Eligibility
5 Years to 65 Years
Enrollment
31 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
2
States / cities
San Jose, California • Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Jul 4, 2019 · Synced Jun 10, 2026, 6:14 PM EDT
Completed Phase 2 Interventional Results available

SARC031: MEK Inhibitor Selumetinib (AZD6244) in Combination With the mTOR Inhibitor Sirolimus for Patients With Malignant Peripheral Nerve Sheath Tumors

NCT03433183
View directory record Official record
Conditions
Malignant Peripheral Nerve Sheath Tumors, Neurofibromatosis 1
Interventions
Selumetinib, Sirolimus
Drug
Lead sponsor
Sarcoma Alliance for Research through Collaboration
Other
Eligibility
12 Years and older
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2023
U.S. locations
5
States / cities
Washington D.C., District of Columbia • Baltimore, Maryland • Bethesda, Maryland + 2 more
Source: ClinicalTrials.gov public record
Updated May 26, 2026 · Synced Jun 10, 2026, 6:14 PM EDT
Completed Phase 2 Interventional Results available

Mexiletine for Muscle Cramps in Charcot Marie Tooth Disease

NCT02561702
View directory record Official record
Conditions
Charcot Marie Tooth Disease
Interventions
Placebo, Mexiletine
Other · Drug
Lead sponsor
University of Rochester
Other
Eligibility
18 Years and older
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2016
U.S. locations
1
States / cities
Rochester, New York
Source: ClinicalTrials.gov public record
Updated Mar 25, 2018 · Synced Jun 10, 2026, 6:14 PM EDT
Enrolling by invitation Phase 1 Interventional

Assessing the Efficacy of Repeat, Monthly Treatments of Deoxycholate for NF1 Associated Cutaneous Neurofibromas (cNFs)

NCT06300502
View directory record Official record
Conditions
Neurofibromas, Cutaneous, Neurofibromatosis 1
Interventions
Kybella
Drug
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
18 Years to 85 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Apr 21, 2026 · Synced Jun 10, 2026, 6:14 PM 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 10, 2026, 6:14 PM EDT
Completed Phase 2 Interventional Results available

Open-Label Safety and Tolerability Study of NBI-98854 for the Treatment of Pediatric Subjects With Tourette Syndrome

NCT03444038
View directory record Official record
Conditions
Tourette Syndrome
Interventions
Valbenazine
Drug
Lead sponsor
Neurocrine Biosciences
Industry
Eligibility
6 Years to 18 Years
Enrollment
85 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2019
U.S. locations
36
States / cities
Sun City, Arizona • Anaheim, California • San Diego, California + 31 more
Source: ClinicalTrials.gov public record
Updated Feb 22, 2022 · Synced Jun 10, 2026, 6:14 PM EDT