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ClinicalTrials.gov public records Last synced Sep 19, 2026, 10:32 PM EDT

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Showing 1–24 of 293 matching trials from the live ClinicalTrials.gov search.
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Completed Early Phase 1 Interventional

Baclofen Treatment of Ataxia Telangiectasia

NCT00640003
View directory record Official record
Conditions
Ataxia Telangiectasia
Interventions
Baclofen, Placebo
Drug
Lead sponsor
Johns Hopkins University
Other
Eligibility
12 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2011
U.S. locations
2
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Dec 19, 2017 · Synced Sep 19, 2026, 10:32 PM EDT
Completed Phase 2 Interventional Results available

A Study to Learn About the Effects and Safety of RTA 408 (Omaveloxolone) in People Aged 16 to 40 With Friedreich's Ataxia

NCT02255435
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
Omaveloxolone Capsules, 2.5 mg, Omaveloxolone Capsules, 5 mg, Omaveloxolone Capsules, 10 mg, Placebo, Omaveloxolone Capsules, 20 mg, Omaveloxolone Capsules, 40 mg, Omaveloxolone Capsules, 80 mg, Omaveloxolone Capsules, 160 mg, Omaveloxolone Capsules, 300 mg, Omaveloxolone Capsules, 150 mg
Drug
Lead sponsor
Biogen
Industry
Eligibility
16 Years to 40 Years
Enrollment
172 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2025
U.S. locations
7
States / cities
Los Angeles, California • Gainesville, Florida • Tampa, Florida + 4 more
Source: ClinicalTrials.gov public record
Updated Sep 2, 2026 · Synced Sep 19, 2026, 10:32 PM EDT
Completed Early Phase 1 Interventional Results available

Donepezil in Treating Young Patients With Primary Brain Tumors Previously Treated With Radiation Therapy to the Brain

NCT00452868
View directory record Official record
Conditions
Brain and Central Nervous System Tumors, Cognitive/Functional Effects, Long-term Effects Secondary to Cancer Therapy in Children, Neurotoxicity, Psychosocial Effects of Cancer and Its Treatment, Radiation Toxicity
Interventions
Donepezil
Drug
Lead sponsor
Wake Forest University Health Sciences
Other
Eligibility
8 Years to 17 Years
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2010
U.S. locations
2
States / cities
Boston, Massachusetts • Winston-Salem, North Carolina
Source: ClinicalTrials.gov public record
Updated Sep 6, 2018 · Synced Sep 19, 2026, 10:32 PM EDT
Completed Phase 2Phase 3 Interventional Results available

Trial in Adult Participants With Spinocerebellar Ataxia (SCA)

NCT02960893
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Oct 7, 2025 · Synced Sep 19, 2026, 10:32 PM EDT
Completed Not applicable Interventional

Coronary Artery Disease in Patients With Friedreich's Ataxia

NCT04649866
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
plethysmography
Diagnostic Test
Lead sponsor
University of Pittsburgh
Other
Eligibility
18 Years to 79 Years
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Sep 7, 2023 · Synced Sep 19, 2026, 10:32 PM EDT
Completed Phase 2 Interventional

Phenylacetate in Treating Children With Recurrent or Progressive Brain Tumors

NCT00003241
View directory record Official record
Conditions
Brain and Central Nervous System Tumors
Interventions
phenylacetate
Drug
Lead sponsor
Texas Children's Cancer Center
Other
Eligibility
2 Years to 21 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
1998 – 2004
U.S. locations
6
States / cities
Los Angeles, California • San Francisco, California • Washington D.C., District of Columbia + 3 more
Source: ClinicalTrials.gov public record
Updated Jun 25, 2013 · Synced Sep 19, 2026, 10:32 PM EDT
Completed Not applicable Interventional Results available

Cerebellar tDCS and Balance Training in PwMS

NCT04391023
View directory record Official record
Conditions
Multiple Sclerosis
Interventions
Sham Transcranial Direct Current Stimulation (tDCS), Balance Training, 2 mA Transcranial Direct Current Stimulation, 4 mA Transcranial Direct Current Stimulation
Device · Behavioral
Lead sponsor
University of Iowa
Other
Eligibility
18 Years to 70 Years
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2023
U.S. locations
1
States / cities
Iowa City, Iowa
Source: ClinicalTrials.gov public record
Updated Jun 17, 2024 · Synced Sep 19, 2026, 10:32 PM EDT
Active, not recruiting Phase 1 Interventional

A Study to Find Out How BIIB141 (Omaveloxolone) is Processed in the Body and to Learn More About Its Safety in Participants With Friedreich's Ataxia Aged 2 to 15 Years Old

NCT06054893
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
Omaveloxolone
Drug
Lead sponsor
Biogen
Industry
Eligibility
2 Years to 15 Years
Enrollment
33 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2030
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Sep 2, 2026 · Synced Sep 19, 2026, 10:32 PM EDT
Completed Phase 1 Interventional

Safety Study of Idebenone to Treat Friedreich's Ataxia

NCT00015808
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
Idebenone
Drug
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Not listed
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2006
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 11, 2006 · Synced Sep 19, 2026, 10:32 PM EDT
Terminated Phase 1 Interventional

A Study to Assess Nomlabofusp in Adolescents and Children With Friedreich's Ataxia

NCT06681766
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
Nomlabofusp, Placebo
Drug
Lead sponsor
Larimar Therapeutics, Inc.
Industry
Eligibility
2 Years to 17 Years
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2025
U.S. locations
1
States / cities
Chevy Chase, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 9, 2026 · Synced Sep 19, 2026, 10:32 PM EDT
Not yet recruiting Phase 2Phase 3 Interventional

Double-Blind Trial of Everolimus for Improving Social Abilities in PTEN Germline Mutations

NCT07218575
View directory record Official record
Conditions
Cowden's Disease, Cowden's Syndrome, Lhermitte-Duclos Disease, Cerebellum Dysplastic Gangliocytoma, Bannayan Zonana Syndrome, Myhre Riley Smith Syndrome, Riley Smith Syndrome, PTEN Hamartoma Tumor Syndrome, Bannayan Riley Ruvalcaba Syndrome
Interventions
Everolimus blinded oral capsules, Placebo
Drug
Lead sponsor
Boston Children's Hospital
Other
Eligibility
5 Years to 45 Years
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2030
U.S. locations
3
States / cities
Stanford, California • Boston, Massachusetts • Cleveland, Ohio
Source: ClinicalTrials.gov public record
Updated Jun 3, 2026 · Synced Sep 19, 2026, 10:32 PM EDT
Completed Phase 1 Interventional

Colony-Stimulating Factors in Treating Children With Recurrent or Refractory Solid Tumors

NCT00003597
View directory record Official record
Conditions
Cancer
Interventions
recombinant human thrombopoietin, carboplatin, etoposide, ifosfamide, G-CSF
Biological · Drug
Lead sponsor
Children's Oncology Group
Network
Eligibility
1 Year to 21 Years
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1998 – 2005
U.S. locations
23
States / cities
Long Beach, California • Los Angeles, California • Orange, California + 16 more
Source: ClinicalTrials.gov public record
Updated Jul 23, 2014 · Synced Sep 19, 2026, 10:32 PM EDT
Completed Phase 1 Interventional

Imetelstat Sodium in Treating Young Patients With Refractory or Recurrent Solid Tumors or Lymphoma

NCT01273090
View directory record Official record
Conditions
Brain and Central Nervous System Tumors, Lymphoma, Lymphoproliferative Disorder, Small Intestine Cancer, Unspecified Childhood Solid Tumor, Protocol Specific
Interventions
imetelstat sodium, laboratory biomarker analysis, pharmacological study
Drug · Other
Lead sponsor
Children's Oncology Group
Network
Eligibility
1 Year to 21 Years
Enrollment
34 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2013
U.S. locations
21
States / cities
Birmingham, Alabama • Orange, California • San Francisco, California + 18 more
Source: ClinicalTrials.gov public record
Updated Jan 29, 2014 · Synced Sep 19, 2026, 10:32 PM EDT
Recruiting Not applicable Interventional

Cerebellar TMS and Satiety in Prader-Willi Syndrome

NCT05938543
View directory record Official record
Conditions
Prader-Willi Syndrome
Interventions
Repetitive Transcranial Magnetic Stimulation (rTMS)
Device
Lead sponsor
Brigham and Women's Hospital
Other
Eligibility
18 Years to 64 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2026
U.S. locations
1
States / cities
Belmont, Massachusetts
Source: ClinicalTrials.gov public record
Updated Dec 4, 2025 · Synced Sep 19, 2026, 10:32 PM EDT
Completed Not applicable Interventional Results available

Transcranial Magnetic Stimulation for Facial Pain

NCT02633306
View directory record Official record
Conditions
Trigeminal Nerve Injuries, Post-herpetic Neuralgia, Facial Pain, Nervus Intermedius Neuralgia
Interventions
transcranial magnetic stimulation
Device
Lead sponsor
University of Minnesota
Other
Eligibility
18 Years to 110 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Apr 7, 2021 · Synced Sep 19, 2026, 10:32 PM EDT
Recruiting Not applicable Interventional Accepts healthy volunteers

Validation of a Novel Cerebellar-striatal Satiety Circuit in Humans

NCT06105164
View directory record Official record
Conditions
Appetitive Behavior, Obesity
Interventions
Repetitive Transcranial Magnetic Stimulation (rTMS), Sham repetitive Transcranial Magnetic Stimulation (rTMS)
Device
Lead sponsor
Brigham and Women's Hospital
Other
Eligibility
18 Years to 65 Years
Enrollment
150 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2024 – 2029
U.S. locations
2
States / cities
Belmont, Massachusetts • Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Dec 4, 2025 · Synced Sep 19, 2026, 10:32 PM EDT
Active, not recruiting No phase listed Observational

Natural History of Spinocerebellar Ataxia Type 7 (SCA7)

NCT02741440
View directory record Official record
Conditions
Spinocerebellar Ataxia
Interventions
Not listed
Lead sponsor
National Eye Institute (NEI)
NIH
Eligibility
12 Years to 100 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2028
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 14, 2026 · Synced Sep 19, 2026, 10:32 PM EDT
No Longer Available No phase listed Expanded access

EPI-743 for Mitochondrial Respiratory Chain Diseases

NCT01370447
View directory record Official record
Conditions
Mitochondrial Diseases
Interventions
EPI-743
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
1 Year and older
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
16
States / cities
Los Angeles, California • Orange, California • Palo Alto, California + 12 more
Source: ClinicalTrials.gov public record
Updated Jan 22, 2024 · Synced Sep 19, 2026, 10:32 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Biomarkers in Friedreich's Ataxia

NCT02497534
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Sep 22, 2025 · Synced Sep 19, 2026, 10:32 PM EDT
Completed Phase 4 Interventional Results available

Long Term Study of Avonex Therapy Following a First Attack of Multiple Sclerosis

NCT00179478
View directory record Official record
Conditions
Multiple Sclerosis, Optic Neuritis, Transverse Myelitis, Acute Brainstem/Cerebellar Syndrome
Interventions
interferon beta 1a 30 ug IM once weekly
Drug
Lead sponsor
Beth Israel Deaconess Medical Center
Other
Eligibility
18 Years and older
Enrollment
155 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2009
U.S. locations
20
States / cities
Derby, Connecticut • Tampa, Florida • Atlanta, Georgia + 16 more
Source: ClinicalTrials.gov public record
Updated Sep 5, 2017 · Synced Sep 19, 2026, 10:32 PM EDT
Completed Not applicable Interventional Results available

Using Real-time fMRI Neurofeedback and Motor Imagery to Enhance Motor Timing and Precision in Cerebellar Ataxia

NCT05436262
View directory record Official record
Conditions
Cerebellar Ataxia, Spinocerebellar Ataxias, Cerebellar Degeneration
Interventions
Real-time fMRI with neurofeedback of motor imagery
Device
Lead sponsor
Johns Hopkins University
Other
Eligibility
18 Years to 100 Years
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Jan 7, 2025 · Synced Sep 19, 2026, 10:32 PM EDT
Terminated Not applicable Interventional

Cell-Based Approaches For Modeling and Treating Ataxia-Telangiectasia

NCT02246491
View directory record Official record
Conditions
Ataxia-Telangiectasia (A-T)
Interventions
A-T iPS cell line, Carrier patients iPS cell line
Other
Lead sponsor
Johns Hopkins University
Other
Eligibility
3 Years to 100 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2018
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 18, 2019 · Synced Sep 19, 2026, 10:32 PM EDT
Completed Phase 2 Interventional

Thiotepa Followed by Peripheral Stem Cell or Bone Marrow Transplant in Treating Patients With Malignant Glioma

NCT00008008
View directory record Official record
Conditions
Brain and Central Nervous System Tumors
Interventions
filgrastim, sargramostim, cyclophosphamide, thiotepa, autologous bone marrow transplantation, bone marrow ablation with stem cell support, peripheral blood stem cell transplantation
Biological · Drug · Procedure
Lead sponsor
Herbert Irving Comprehensive Cancer Center
Other
Eligibility
Not listed
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1997 – 2008
U.S. locations
2
States / cities
Paterson, New Jersey • New York, New York
Source: ClinicalTrials.gov public record
Updated Feb 3, 2013 · Synced Sep 19, 2026, 10:32 PM EDT
Recruiting Phase 2 Interventional

Study of LX2006 Gene Therapy in Friedreich Ataxia Cardiomyopathy

NCT07721025
View directory record Official record
Conditions
Friedreich Ataxia, Cardiomyopathy, Secondary
Interventions
LX2006, Usual Care
Genetic · Other
Lead sponsor
Lexeo Therapeutics
Industry
Eligibility
6 Years and older
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2032
U.S. locations
1
States / cities
Tampa, Florida
Source: ClinicalTrials.gov public record
Updated Sep 1, 2026 · Synced Sep 19, 2026, 10:32 PM EDT