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ClinicalTrials.gov public records Last synced Jul 23, 2026, 11:25 AM EDT

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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Conditions
Adrenoleukodystrophy, Batten Disease, Mucopolysaccharidosis II, Leukodystrophy, Globoid Cell, Leukodystrophy, Metachromatic, Neimann Pick Disease, Pelizaeus-Merzbacher Disease, Sandhoff Disease, Tay-Sachs Disease, Brain Diseases, Metabolic, Inborn, Alpha-Mannosidosis, Sanfilippo Mucopolysaccharidoses
Interventions
DUOC-01
Biological
Lead sponsor
Joanne Kurtzberg, MD
Other
Eligibility
1 Week to 22 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2026
U.S. locations
1
States / cities
Durham, North Carolina
Recruiting Not applicable Interventional

Neurologic Stem Cell Treatment Study

NCT02795052
Conditions
Neurologic Disorders, Nervous System Diseases, Neurodegenerative Diseases, Neurological Disorders, Stroke, Traumatic Brain Injury, Cadasil, Chronic Traumatic Encephalopathy, Cerebral Infarction, Cerebral Ischemia, Cerebral Stroke, Cerebral Hemorrhage, Parkinson, Multi-System Degeneration, MSA - Multiple System Atrophy, Progressive Supranuclear Palsy, ALS, Amyotrophic Lateral Sclerosis, Neuropathy, Diabetic Neuropathies, Alzheimer Disease, Dementia, Frontotemporal Dementia, Lewy Body Disease, Cognitive Impairment, Lewy Body Variant of Alzheimer Disease
Interventions
Intravenous and Intranasal BMSC
Procedure
Lead sponsor
MD Stem Cells
Industry
Eligibility
18 Years and older
Enrollment
500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2028
U.S. locations
2
States / cities
Westport, Connecticut • Coral Springs, Florida
Conditions
PPA, Non-fluent Aphasia, Apraxia of Speech, Primary Progressive Non-fluent Aphasia, Primary Progressive Aphasia
Interventions
AV-1451
Drug
Lead sponsor
Mayo Clinic
Other
Eligibility
18 Years and older
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2028
U.S. locations
1
States / cities
Rochester, Minnesota
Conditions
I Cell Disease, Fucosidosis, Globoid Cell Leukodystrophy, Adrenoleukodystrophy, Mannosidosis, Niemann-Pick Disease, Pulmonary Complications, Mucopolysaccharidosis I, Mucopolysaccharidosis VI, Metachromatic Leukodystrophy, Gaucher's Disease, Wolman Disease
Interventions
Not listed
Lead sponsor
Fairview University Medical Center
Other
Eligibility
0 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1999
U.S. locations
1
States / cities
Minneapolis, Minnesota
Conditions
Monocarboxylate Transporter 8 Deficiency, Allan-Herndon-Dudley Syndrome
Interventions
Tiratricol, Placebo
Drug
Lead sponsor
Rare Thyroid Therapeutics International AB
Industry
Eligibility
4 Years and older · Male only
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
6
States / cities
Kissimmee, Florida • Atlanta, Georgia • St Louis, Missouri + 3 more
Conditions
Monocarboxylate Transporter 8 Deficiency, Allan-Herndon-Dudley Syndrome
Interventions
Tiratricol
Drug
Lead sponsor
Rare Thyroid Therapeutics International AB
Industry
Eligibility
Not listed
U.S. locations
17
States / cities
Birmingham, Alabama • Madera, California • San Diego, California + 13 more
Conditions
PSP, CBD, PCA, LPA, Semantic Dementia, Semantic Aphasia, Behavioral Variant of Frontotemporal Dementia, FTD, PPA, Apraxia of Speech, MSA - Multiple System Atrophy, Parkinson Disease
Interventions
C-11 PiB, AV1451 Tau, C-11 ER176
Drug
Lead sponsor
Mayo Clinic
Other
Eligibility
21 Years and older
Enrollment
500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2050
U.S. locations
1
States / cities
Rochester, Minnesota
Conditions
PPA, Primary Progressive Aphasia, Aphasia, Non-fluent Aphasia, Semantic Dementia, Apraxia of Speech, Primary Progressive Nonfluent Aphasia, Progressive Aphasia
Interventions
C-11 PiB, F-18 FDG
Drug
Lead sponsor
Mayo Clinic
Other
Eligibility
18 Years and older
Enrollment
168 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2015
U.S. locations
1
States / cities
Rochester, Minnesota
Conditions
HIV Infections, Sinusitis
Interventions
Pseudoephedrine hydrochloride, Guaifenesin, Cefuroxime axetil, Beclomethasone dipropionate
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
12 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
Ends 1993
U.S. locations
10
States / cities
San Diego, California • San Francisco, California • The Bronx, New York + 1 more
Conditions
Hurler Syndrome (MPS I), Hurler-Scheie Syndrome, Hunter Syndrome (MPS II), Sanfilippo Syndrome (MPS III), Krabbe Disease (Globoid Leukodystrophy), Metachromatic Leukodystrophy (MLD), Adrenoleukodystrophy (ALD and AMN), Sandhoff Disease, Tay Sachs Disease, Pelizaeus Merzbacher (PMD), Niemann-Pick Disease, Alpha-mannosidosis
Interventions
hematopoietic stem cell infusion
Biological
Lead sponsor
Talaris Therapeutics Inc.
Industry
Eligibility
Not listed
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2016
U.S. locations
1
States / cities
Durham, North Carolina
Conditions
Hurler's Syndrome, Maroteaux-Lamy Syndrome, Sly Syndrome, Alpha Mannosidosis, Fucosidosis, Aspartylglucosaminuria, Sphingolipidoses, Krabbe Disease, Wolman's Disease, Niemann-Pick Disease Type B, Niemann-Pick Disease, Type C
Interventions
Stem Cell Transplantation, Cyclophosphamide, Campath-1H, Busulfan
Procedure · Drug
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 21 Years
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2010
U.S. locations
1
States / cities
Minneapolis, Minnesota
Conditions
Apraxia of Speech, Non-fluent Aphasia, Primary Progressive Aphasia, Primary Progressive Nonfluent Aphasia
Interventions
Testing protocol for the study
Diagnostic Test
Lead sponsor
Mayo Clinic
Other
Eligibility
18 Years and older
Enrollment
47 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2027
U.S. locations
1
States / cities
Rochester, Minnesota
Conditions
Primary Progressive Aphasia, Logopenic Progressive Aphasia, Non-Fluent Primary Progressive Aphasia
Interventions
Active tDCS + Language Therapy, Sham tDCS + Language Therapy
Device
Lead sponsor
Johns Hopkins University
Other
Eligibility
50 Years to 90 Years
Enrollment
180 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2024 – 2028
U.S. locations
2
States / cities
Baltimore, Maryland • Philadelphia, Pennsylvania
Recruiting No phase listed Observational Accepts healthy volunteers

ALS Research Collaborative

NCT06885918
Conditions
Amyotrophic Lateral Sclerosis, ALS (Amyotrophic Lateral Sclerosis), ALS With Frontotemporal Dementia (ALS/FTD), Motor Neuron Disease, Motor Neuron Disease, Amyotrophic Lateral Sclerosis
Interventions
Not listed
Lead sponsor
ALS Therapy Development Institute
Other
Eligibility
18 Years and older
Enrollment
2,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2014 – 2035
U.S. locations
1
States / cities
Watertown, Massachusetts
Completed Not applicable Interventional Accepts healthy volunteers Results available

Rural Dementia Caregiver Project

NCT04428112
Conditions
Depression, Stress, Psychological, Self Efficacy, Loneliness, Social Isolation, Health Care Utilization, Dementia, Dementia Alzheimers, Dementia of Alzheimer Type, Dementia, Vascular, Dementia Frontal, Dementia, Lewy Body, Dementia, Mixed, Dementia in Parkinsons Disease, Dementia, HIV, Dementia Frontotemporal, Dementia, Multi-Infarct
Interventions
Building Better Caregivers Workshop, Attention Control
Behavioral
Lead sponsor
University of California, San Francisco
Other
Eligibility
18 Years and older
Enrollment
418 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2023
U.S. locations
1
States / cities
San Francisco, California
Conditions
Lysosomal Acid Lipase Deficiency, Wolman Disease
Interventions
Sebelipase alfa (SBC-102)
Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
Up to 24 Months
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2018
U.S. locations
1
States / cities
Irvine, California
Conditions
Juvenile Neuronal Ceroid Lipofuscinosis, Smith-Lemli-Opitz Syndrome, Creatine Transporter Deficiency, Niemann-Pick Disease Type C1
Interventions
Not listed
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
6 Months to 110 Years
Enrollment
73 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
1
States / cities
Bethesda, Maryland
Conditions
Sickle Cell Disease, Thalassemia, Anemia, Granuloma, Wiskott-Aldrich Syndrome, Chediak Higashi Syndrome, Osteopetrosis, Neutropenia, Thrombocytopenia, Hurler Disease, Niemann-Pick Disease, Fucosidosis
Interventions
Hematopoietic stem cell transplantation
Procedure
Lead sponsor
Children's Hospital Los Angeles
Other
Eligibility
Up to 21 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2015
U.S. locations
1
States / cities
Los Angeles, California
Conditions
Lysosomal Acid Lipase Deficiency
Interventions
Sebelipase Alfa
Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
Up to 8 Months
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2018
U.S. locations
1
States / cities
Phoenix, Arizona
Conditions
Progressive Supranuclear Palsy Richardson Syndrome (PSP-RS)
Interventions
NIO752, Placebo
Other · Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
41 Years to 81 Years
Enrollment
300 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2031
U.S. locations
4
States / cities
San Francisco, California • Englewood, Colorado • Jacksonville, Florida + 1 more
Conditions
Behavioral Variant of Frontotemporal Dementia, Caregiver Stress Syndrome
Interventions
Health Information + Health Coaching
Behavioral
Lead sponsor
University of Pennsylvania
Other
Eligibility
18 Years and older
Enrollment
43 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2021
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Conditions
Frontotemporal Lobar Degeneration (FTLD), Frontotemporal Dementia (FTD), Corticobasal Degeneration, Corticobasal Syndrome (CBS)
Interventions
Not listed
Lead sponsor
BioSensics
Industry
Eligibility
40 Years and older
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2030
U.S. locations
2
States / cities
Lutherville, Maryland • Boston, Massachusetts
Completed No phase listed Observational Accepts healthy volunteers

4-Repeat Tauopathy Neuroimaging Initiative - Cycle 2

NCT02966145
Conditions
Corticobasal Degeneration (CBD), Corticobasal Syndrome (CBS), Cortical-basal Ganglionic Degeneration (CBGD), Progressive Supranuclear Palsy (PSP), Nonfluent Variant Primary Progressive Aphasia (nfvPPA), Oligosymptomatic/Variant Progressive Supranuclear Palsy (o/vPSP)
Interventions
Observational Study
Other
Lead sponsor
University of California, San Francisco
Other
Eligibility
40 Years to 80 Years
Enrollment
293 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2016 – 2024
U.S. locations
7
States / cities
San Diego, California • San Francisco, California • Baltimore, Maryland + 4 more
Recruiting No phase listed Observational Accepts healthy volunteers

UPenn Observational Research Repository on Neurodegenerative Disease

NCT04715399
Conditions
Frontotemporal Degeneration(FTD), Primary Progressive Aphasia(PPA), Familial Frontotemporal Lobar Degeneration (fFTLD), Amyotrophic Lateral Sclerosis(ALS), Lewy Body Disease(LBD), Progressive Supranuclear Palsy(PSP), Corticobasal Syndrome(CBS), Posterior Cortical Atrophy(PCA), Alzheimer's Disease(AD)
Interventions
No intervention
Other
Lead sponsor
University of Pennsylvania
Other
Eligibility
18 Years and older
Enrollment
1,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2070
U.S. locations
1
States / cities
Philadelphia, Pennsylvania