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ClinicalTrials.gov public records Last synced Jun 26, 2026, 5:05 AM EDT

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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: CNS Metabolic Disorders Clear all
Withdrawn No phase listed Observational Accepts healthy volunteers

Genetic Study of Familial and Sporadic ALS/Motor Neuron Disease, Miyoshi Myopathy and Other Neuromuscular Disorders

NCT01459302
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis, Frontotemporal Dementia, PLS, Motor Neuron Disease, Lou Gehrigs Disease, Familial Disease, Amyotrophic Lateral Sclerosis, Sporadic, Muscular Dystrophy, Miyoshi Myopathy, Amyotrophic Lateral Sclerosis With Dementia
Interventions
Not listed
Lead sponsor
University of Massachusetts, Worcester
Other
Eligibility
Not listed
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2024
U.S. locations
1
States / cities
Worcester, Massachusetts
Source: ClinicalTrials.gov public record
Updated Sep 18, 2024 · Synced Jun 26, 2026, 5:05 AM EDT
Terminated Phase 3 Interventional Results available

Extension Treatment Using EryDex System in Patients With AT Who Participated in the ATTeST-IEDAT-02-2015 Study

NCT03563053
View directory record Official record
Conditions
Ataxia Telangiectasia, Genetic Syndrome
Interventions
EryDex System
Combination Product
Lead sponsor
Quince Therapeutics S.p.A.
Industry
Eligibility
6 Years and older
Enrollment
104 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2022
U.S. locations
4
States / cities
Los Angeles, California • Baltimore, Maryland • Cincinnati, Ohio + 1 more
Source: ClinicalTrials.gov public record
Updated Oct 7, 2024 · Synced Jun 26, 2026, 5:05 AM EDT
Recruiting Phase 1 Interventional

A Study to Investigate the Safety and Pharmacodynamics of a Single Intrathecal Injection (IT) of INS1202 in Participants With Amyotrophic Lateral Sclerosis (ALS)

NCT07290062
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis
Interventions
INS1202
Genetic
Lead sponsor
Insmed Gene Therapy LLC
Industry
Eligibility
18 Years to 79 Years
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2030
U.S. locations
5
States / cities
La Jolla, California • Palo Alto, California • Columbia, Missouri + 2 more
Source: ClinicalTrials.gov public record
Updated Jun 11, 2026 · Synced Jun 26, 2026, 5:05 AM EDT
Not listed No phase listed Observational

Flicker App for Minimal Hepatic Encephalopathy

NCT04415294
View directory record Official record
Conditions
Hepatic Encephalopathy
Interventions
Critical flicker frequency
Diagnostic Test
Lead sponsor
University of Washington
Other
Eligibility
18 Years to 75 Years
Enrollment
75 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2023
U.S. locations
1
States / cities
Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Jan 10, 2022 · Synced Jun 26, 2026, 5:05 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

A Phase 1/Phase 2 Open-label Study to Evaluate the Safety, Tolerability, and Efficacy of a Single Intravenous Administration of SAR444836 in Adult Participants With Phenylketonuria

NCT05972629
View directory record Official record
Conditions
Phenylketonuria
Interventions
SAR444836
Drug
Lead sponsor
Sanofi
Industry
Eligibility
18 Years to 65 Years
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2030
U.S. locations
7
States / cities
San Francisco, California • Aurora, Colorado • Gainesville, Florida + 4 more
Source: ClinicalTrials.gov public record
Updated Sep 22, 2025 · Synced Jun 26, 2026, 5:05 AM EDT
Completed Phase 2Phase 3 Interventional Accepts healthy volunteers Results available

Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency

NCT02004691
View directory record Official record
Conditions
Sphingomyelin Lipidosis
Interventions
placebo (saline), Olipudase alfa
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
18 Years and older
Enrollment
36 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2023
U.S. locations
3
States / cities
San Francisco, California • Decatur, Georgia • The Bronx, New York
Source: ClinicalTrials.gov public record
Updated Nov 6, 2024 · Synced Jun 26, 2026, 5:05 AM EDT
Withdrawn Phase 2 Interventional

Exogenous Sodium Lactate Infusion in Traumatic Brain Injury (ELI-TBI)

NCT02776488
View directory record Official record
Conditions
Brain Injuries, Traumatic
Interventions
Sodium Lactate, Placebo
Drug
Lead sponsor
University of California, Los Angeles
Other
Eligibility
18 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2026
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Nov 14, 2021 · Synced Jun 26, 2026, 5:05 AM EDT
Completed No phase listed Observational

Fabry Patient's Experience Of PegunigaLsidasE Alfa Monthly Infusion

NCT05186324
View directory record Official record
Conditions
Fabry Disease
Interventions
Interview
Other
Lead sponsor
Chiesi Farmaceutici S.p.A.
Industry
Eligibility
Not listed
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022
U.S. locations
7
States / cities
Birmingham, Alabama • Atlanta, Georgia • Iowa City, Iowa + 4 more
Source: ClinicalTrials.gov public record
Updated Mar 20, 2023 · Synced Jun 26, 2026, 5:05 AM EDT
Completed Phase 1 Interventional

Safety and Tolerability Study of rAvPAL-PEG to Treat Phenylketonuria

NCT00634660
View directory record Official record
Conditions
Phenylketonuria
Interventions
rAvPAL-PEG
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
16 Years to 50 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2009
U.S. locations
8
States / cities
Chicago, Illinois • Minneapolis, Minnesota • St Louis, Missouri + 5 more
Source: ClinicalTrials.gov public record
Updated Jan 31, 2017 · Synced Jun 26, 2026, 5:05 AM EDT
No Longer Available No phase listed Expanded access

Expanded Access Protocol: Repeated Administration of Nurown® (Autologous MSC-NTF Cells) for the Treatment of ALS

NCT04681118
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis
Interventions
NurOwn (MSC-NTF cells)
Biological
Lead sponsor
Brainstorm-Cell Therapeutics
Industry
Eligibility
18 Years to 63 Years
U.S. locations
5
States / cities
Irvine, California • San Francisco, California • Boston, Massachusetts + 2 more
Source: ClinicalTrials.gov public record
Updated Apr 22, 2024 · Synced Jun 26, 2026, 5:05 AM EDT
Completed No phase listed Observational

Longitudinal Study of Cognition With Niemann-Pick Disease, Type C

NCT01899950
View directory record Official record
Conditions
Niemann-Pick Disease, Type C
Interventions
Not listed
Lead sponsor
Mayo Clinic
Other
Eligibility
2 Years to 99 Years
Enrollment
70 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2014
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Jan 5, 2016 · Synced Jun 26, 2026, 5:05 AM EDT
Recruiting Not applicable Interventional

Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular Disease

NCT07478172
View directory record Official record
Conditions
Neuromuscular Diseases (NMD), Amyotrophic Lateral Sclerosis, Myasthenia Gravis, Lambert-eaton Myasthenic Syndrome, Primary Lateral Sclerosis, Spinal Muscular Atrophy, Charcot Marie Tooth Disease (CMT), Fascioscapulohumeral Muscular Dystrophy, Inclusion Body Myositis, Mitochondrial Myopathy, Nemaline Myopathy, Centronuclear Myopathy, Postpolio Syndrome, Pompe Disease (Late-onset), Chronic Inflammatory Demyelinating Polyneuropathy, Hereditary Spastic Paraplegia, Postural Orthostatic Tachycardia Syndrome (POTS), Progressive Muscular Atrophy
Interventions
Whole-body Electrical Muscle Stimulation Exercise
Device
Lead sponsor
University of Missouri-Columbia
Other
Eligibility
18 Years and older
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2031
U.S. locations
1
States / cities
Columbia, Missouri
Source: ClinicalTrials.gov public record
Updated Mar 16, 2026 · Synced Jun 26, 2026, 5:05 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Study of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Participants With GM1 Gangliosidosis

NCT04713475
View directory record Official record
Conditions
GM1 Gangliosidosis, GM1 Gangliosidosis, Type I, GM1 Gangliosidosis, Type 2, Beta-Galactosidase-1 (GLB1) Deficiency
Interventions
PBGM01
Biological
Lead sponsor
Gemma Biotherapeutics
Industry
Eligibility
1 Month to 24 Months
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2029
U.S. locations
4
States / cities
Oakland, California • Minneapolis, Minnesota • New Brunswick, New Jersey + 1 more
Source: ClinicalTrials.gov public record
Updated May 20, 2025 · Synced Jun 26, 2026, 5:05 AM EDT
Completed Phase 3 Interventional Results available

A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With Late-Onset Pompe Disease (LOPD)

NCT04138277
View directory record Official record
Conditions
Pompe Disease (Late-onset)
Interventions
AT2221, ATB200
Drug · Biological
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
119 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2024
U.S. locations
18
States / cities
Little Rock, Arkansas • Irvine, California • Gainesville, Florida + 14 more
Source: ClinicalTrials.gov public record
Updated Mar 4, 2026 · Synced Jun 26, 2026, 5:05 AM EDT
Completed No phase listed Observational

F 18 T807 Tau PET Imaging in Familial Amyotrophic Lateral Sclerosis

NCT02414230
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis (ALS)
Interventions
Drug: F 18 T807
Drug
Lead sponsor
Tammie L. S. Benzinger, MD, PhD
Other
Eligibility
18 Years and older
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2020
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Sep 21, 2020 · Synced Jun 26, 2026, 5:05 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Long Term Follow-up Study of Type-1 Gaucher Subjects Post FLT201 Dose (GALILEO-2)

NCT06545136
View directory record Official record
Conditions
Gaucher Disease, Type 1
Interventions
FLT201
Genetic
Lead sponsor
Spur Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
75 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2029
U.S. locations
1
States / cities
Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Nov 3, 2025 · Synced Jun 26, 2026, 5:05 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A Study of VRG50635 in Healthy Volunteers

NCT06286475
View directory record Official record
Conditions
Healthy Volunteers
Interventions
VRG50635, Placebo
Drug
Lead sponsor
Verge Genomics
Industry
Eligibility
19 Years to 55 Years
Enrollment
22 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2024
U.S. locations
1
States / cities
Lincoln, Nebraska
Source: ClinicalTrials.gov public record
Updated Dec 14, 2025 · Synced Jun 26, 2026, 5:05 AM EDT
Not listed No phase listed Observational

SRT in Comparison to ERT on Immune Aspects and Bone Involvement in Gaucher Disease

NCT02605603
View directory record Official record
Conditions
Gaucher Disease
Interventions
Not listed
Lead sponsor
Lysosomal and Rare Disorders Research and Treatment Center, Inc.
Other
Eligibility
18 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2020
U.S. locations
1
States / cities
Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Aug 12, 2019 · Synced Jun 26, 2026, 5:05 AM EDT
Completed Phase 2Phase 3 Interventional Results available

HEALEY ALS Platform Trial - Regimen F ABBV-CLS-7262

NCT05740813
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis
Interventions
ABBV-CLS-7262 Dose 1, ABBV-CLS-7262 Dose 2, Matching Placebo
Drug
Lead sponsor
Merit E. Cudkowicz, MD
Other
Eligibility
18 Years to 100 Years
Enrollment
310 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Nov 13, 2025 · Synced Jun 26, 2026, 5:05 AM EDT
Recruiting Phase 4 Interventional

Effects of Semaglutide on Intracranial Blood Flow and Brain-Barrier Permeability in Type-2 Diabetes

NCT05780905
View directory record Official record
Conditions
Diabetes Mellitus, Type 2, Stroke (CVA) or Transient Ischemic Attack
Interventions
Semaglutide Auto-Injector, Placebo
Drug · Other
Lead sponsor
University of Washington
Other
Eligibility
40 Years to 65 Years
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
1
States / cities
Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Jun 4, 2024 · Synced Jun 26, 2026, 5:05 AM EDT
Completed No phase listed Observational

Natural History and Biomarkers of Amyotrophic Lateral Sclerosis and Frontotemporal Dementia Caused by the C9ORF72 Gene Mutation

NCT01925196
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis, Frontotemporal Lobar Degeneration
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
18 Years and older
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2013
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jun 23, 2026 · Synced Jun 26, 2026, 5:05 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Longitudinal Evaluation of Familial Frontotemporal Dementia Subjects

NCT02372773
View directory record Official record
Conditions
Familial Frontotemporal Dementia
Interventions
Not listed
Lead sponsor
Mayo Clinic
Other
Eligibility
18 Years to 90 Years
Enrollment
398 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2020
U.S. locations
7
States / cities
San Francisco, California • Jacksonville, Florida • Charlestown, Massachusetts + 4 more
Source: ClinicalTrials.gov public record
Updated Aug 2, 2020 · Synced Jun 26, 2026, 5:05 AM EDT
Recruiting No phase listed Observational

Analysis of Human ALS Tissues and Registry of ALS Patients

NCT05067179
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis
Interventions
Not listed
Lead sponsor
University of Illinois at Chicago
Other
Eligibility
18 Years to 90 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2030
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Sep 21, 2025 · Synced Jun 26, 2026, 5:05 AM EDT
Completed Not applicable Interventional Results available

Effectiveness of Expiratory Muscle Strength Training for Improving Communication in ALS

NCT05003167
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis
Interventions
Expiratory Muscle Strength Training (EMST-150)
Device
Lead sponsor
Purdue University
Other
Eligibility
40 Years to 70 Years
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2022
U.S. locations
1
States / cities
West Lafayette, Indiana
Source: ClinicalTrials.gov public record
Updated Jun 7, 2023 · Synced Jun 26, 2026, 5:05 AM EDT