- Conditions
- Mindfulness Based Stress Reduction, Caregiver Burnout, Caregiver Stress Syndrome, Dementia, Dementia Frontal, Frontotemporal Dementia, Frontotemporal Lobar Degeneration
- Interventions
- Mindfulness-Based Stress Reduction, Self-Guided Mindfulness eCourse
- Other
- Lead sponsor
- University of California, San Francisco
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 40 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2021 – 2022
- U.S. locations
- 1
- States / cities
- San Francisco, California
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Showing 25–48
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- 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
- 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
- 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
- Primary Progressive Aphasia
- Interventions
- Active tDCS plus Speech-Language Therapy, Sham plus Speech-Language Therapy
- Device
- Lead sponsor
- Johns Hopkins University
- Other
- Eligibility
- 50 Years to 90 Years
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2013 – 2025
- U.S. locations
- 1
- States / cities
- Baltimore, Maryland
- 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
- Mucopolysaccharidosis I, Mucopolysaccharidosis VI, Adrenoleukodystrophy, Niemann-Pick Disease, Metachromatic Leukodystrophy, Wolman Disease, Krabbe's Disease, Gaucher's Disease, Fucosidosis, Batten Disease, Severe Aplastic Anemia, Diamond-Blackfan Anemia, Amegakaryocytic Thrombocytopenia, Myelodysplastic Syndrome, Acute Myelogenous Leukemia, Acute Lymphocytic Leukemia
- Interventions
- Human Placental Derived Stem Cell
- Drug
- Lead sponsor
- New York Medical College
- Other
- Eligibility
- 0 Years to 55 Years
- Enrollment
- 43 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2022
- U.S. locations
- 3
- States / cities
- Denver, Colorado • Valhalla, New York • Salt Lake City, Utah
- 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
- 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
- Conditions
- Frontotemporal Dementia, Caregiver Burden, Primary Progressive Aphasia (PPA), Corticobasal Syndrome
- Interventions
- STELLA-FTD Control, STELLA-FTD Test
- Behavioral
- Lead sponsor
- Oregon Health and Science University
- Other
- Eligibility
- 18 Years to 100 Years
- Enrollment
- 640 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2025 – 2029
- U.S. locations
- 1
- States / cities
- Portland, Oregon
- Conditions
- Down Syndrome, Alzheimer's Disease
- Interventions
- Not listed
- Lead sponsor
- Michael Rafii
- Other
- Eligibility
- 30 Years to 60 Years
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2017
- U.S. locations
- 1
- States / cities
- La Jolla, California
- Conditions
- Primary Progressive Aphasia, Frontotemporal Dementia
- Interventions
- Monthly Check-in Session
- Behavioral
- Lead sponsor
- University of Chicago
- Other
- Eligibility
- Not listed
- Enrollment
- 57 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2020
- U.S. locations
- 1
- States / cities
- Chicago, Illinois
- 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
- Primary Tauopathies, Corticobasal Degeneration Syndrome, Frontotemporal Lobar Degeneration With Tau Inclusions, MAPT Mutation Carriers, Symptomatic, Traumatic Encephalopathy Syndrome, Nonfluent Aphasia, Progressive
- Interventions
- BIIB092, Placebo
- Drug · Other
- Lead sponsor
- University of California, San Francisco
- Other
- Eligibility
- 35 Years to 80 Years
- Enrollment
- 22 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2019
- U.S. locations
- 1
- States / cities
- San Francisco, California
- 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
- Conditions
- Frontotemporal Degeneration (FTD), ALS (Amyotrophic Lateral Sclerosis), Primary Progressive Aphasia (PPA), Familial Frontotemporal Lobar Degeneration (fFTLD), Fronto-temporal Lobar Dementia, Dementia With Lewy Bodies (DLB), Cortical Basal Syndrome (CBS), Alzheimer's Disease (AD), Progressive Supranuclear Palsy(PSP), Parkinson's Disease (PD), Parkinson's Disease Dementia (PDD), Limbic-predominant Age-related TDP-43 Encephalopathy (LATE), Posterior Cortical Atrophy (PCA), Primary Lateral Sclerosis (PLS), Primary Muscular Atrophy (PMA)
- Interventions
- Not listed
- Lead sponsor
- University of Pennsylvania
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 1,000 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2025 – 2070
- U.S. locations
- 1
- States / cities
- Philadelphia, Pennsylvania
- 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
- 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
- 5
- States / cities
- Scottsdale, Arizona • San Francisco, California • Englewood, Colorado + 2 more
- Conditions
- Spinal Muscular Atrophy, Fragile X Syndrome, Fragile X - Premutation, Duchenne Muscular Dystrophy, Hyperinsulinemic Hypoglycemia, Familial 1, Diabetes Mellitus, Adrenoleukodystrophy, Neonatal, Medium-chain Acyl-CoA Dehydrogenase Deficiency, Very Long Chain Acyl Coa Dehydrogenase Deficiency, Beta-ketothiolase Deficiency, Severe Combined Immunodeficiency Due to Adenosine Deaminase Deficiency, Primary Hyperoxaluria Type 1, Congenital Bile Acid Synthesis Defect Type 2, Pyridoxine-Dependent Epilepsy, Hereditary Fructose Intolerance, Hypophosphatasia, Hyperargininemia, Mucopolysaccharidosis Type 6, Argininosuccinic Aciduria, Citrullinemia, Type I, Wilson Disease, Maple Syrup Urine Disease, Type 1A, Maple Syrup Urine Disease, Type 1B, Biotinidase Deficiency, Neonatal Severe Primary Hyperparathyroidism, Intrinsic Factor Deficiency, Usher Syndrome Type 1D/F Digenic (Diagnosis), Cystic Fibrosis, Stickler Syndrome Type 2, Stickler Syndrome Type 1, Alport Syndrome, Autosomal Recessive, Alport Syndrome, X-Linked, Carbamoyl Phosphate Synthetase I Deficiency Disease, Carnitine Palmitoyl Transferase 1A Deficiency, Carnitine Palmitoyltransferase II Deficiency, Cystinosis, Chronic Granulomatous Disease, Cerebrotendinous Xanthomatoses, Maple Syrup Urine Disease, Type 2, Severe Combined Immunodeficiency Due to DCLRE1C Deficiency, Thyroid Dyshormonogenesis 6, Thyroid Dyshormonogenesis 5, Supravalvar Aortic Stenosis, Factor X Deficiency, Hemophilia A, Hemophilia B, Tyrosinemia, Type I, Fructose 1,6 Bisphosphatase Deficiency, Glycogen Storage Disease Type I, G6PD Deficiency, Glycogen Storage Disease II, Galactokinase Deficiency, Mucopolysaccharidosis Type IV A, Galactosemias, Guanidinoacetate Methyltransferase Deficiency, Agat Deficiency, Glutaryl-CoA Dehydrogenase Deficiency, Gtp Cyclohydrolase I Deficiency, Hyperinsulinism-Hyperammonemia Syndrome, Primary Hyperoxaluria Type 2, 3-Hydroxyacyl-CoA Dehydrogenase Deficiency, Long-chain 3-hydroxyacyl-CoA Dehydrogenase Deficiency, Mitochondrial Trifunctional Protein Deficiency, Sickle Cell Disease, Beta-Thalassemia, Holocarboxylase Synthetase Deficiency, 3-Hydroxy-3-Methylglutaric Aciduria, Primary Hyperoxaluria Type 3, Hermansky-Pudlak Syndrome 1, Hermansky-Pudlak Syndrome 4, Apparent Mineralocorticoid Excess, HSDB, CBAS1, Mucopolysaccharidosis Type 2, Mucopolysaccharidosis Type 1, Severe Combined Immunodeficiency, X Linked, Severe Combined Immunodeficiency Due to IL-7Ralpha Deficiency, Diabetes Mellitus, Permanent Neonatal, Isovaleric Acidemia, Severe Combined Immunodeficiency T-Cell Negative B-Cell Positive Due to Janus Kinase-3 Deficiency (Disorder), Jervell and Lange-Nielsen Syndrome 2, Hyperinsulinemic Hypoglycemia, Familial, 2, Diabetes Mellitus, Permanent Neonatal, With Neurologic Features, Jervell and Lange-Nielsen Syndrome 1, Lysosomal Acid Lipase Deficiency, CblF, 3-Methylcrotonyl CoA Carboxylase 1 Deficiency, 3-Methylcrotonyl CoA Carboxylase 2 Deficiency, Waardenburg Syndrome Type 2A, Methylmalonic Aciduria cblA Type, Methylmalonic Aciduria cblB Type, Methylmalonic Aciduria and Homocystinuria Type cblC, MAHCD, Methylmalonic Aciduria Due to Methylmalonyl-CoA Mutase Deficiency, Congenital Disorder of Glycosylation Type 1B, Mthfr Deficiency, Methylcobalamin Deficiency Type Cbl G (Disorder), Methylcobalamin Deficiency Type cblE, Usher Syndrome, Type 1B, N-acetylglutamate Synthase Deficiency, Ornithine Transcarbamylase Deficiency, Phenylketonurias, Waardenburg Syndrome Type 1, Congenital Hypothyroidism, Propionic Acidemia, Usher Syndrome, Type 1F, Pancreatic Agenesis 1, Hereditary Hypophosphatemic Rickets, Glycogen Storage Disease IXB, Glycogen Storage Disease IXC, MOWS, Epilepsy, Early-Onset, Vitamin B6-Dependent, Pyridoxal Phosphate-Responsive Seizures, Pituitary Hormone Deficiency, Combined, 1, Ptsd, Dihydropteridine Reductase Deficiency, Severe Combined Immunodeficiency Due to RAG1 Deficiency, Severe Combined Immunodeficiency Due to RAG2 Deficiency, Retinoblastoma, Multiple Endocrine Neoplasia Type 2B, Pseudohypoaldosteronism, Type I, Liddle Syndrome, Biotin-Responsive Basal Ganglia Disease, SCD, DIAR1, GSD1C, Acrodermatitis Enteropathica, Thyroid Dyshormonogenesis 1, Riboflavin Transporter Deficiency, Waardenburg Syndrome, Type 2E, SRD, Congenital Lipoid Adrenal Hyperplasia Due to STAR Deficiency, Barth Syndrome, Adrenocorticotropic Hormone Deficiency, Transcobalamin II Deficiency, Thyroid Dyshormonogenesis 3, Segawa Syndrome, Autosomal Recessive, Autosomal Recessive Nonsyndromic Hearing Loss, Thyroid Dyshormonogenesis 2A, Congenital Isolated Thyroid Stimulating Hormone Deficiency, Hypothyroidism Due to TSH Receptor Mutations, Usher Syndrome Type 1C, Usher Syndrome Type 1G (Diagnosis), Von Willebrand Disease, Type 3, Combined Immunodeficiency Due to ZAP70 Deficiency, Adenine Phosphoribosyltransferase Deficiency, Metachromatic Leukodystrophy, Canavan Disease, Menkes Disease, Carbonic Anhydrase VA Deficiency, Developmental and Epileptic Encephalopathy 2, 17 Alpha-Hydroxylase Deficiency, Smith-Lemli-Opitz Syndrome, Krabbe Disease, Glutathione Synthetase Deficiency, Mucopolysaccharidosis Type 7, Rett Syndrome, Molybdenum Cofactor Deficiency, Type A, Niemann-Pick Disease, Type C1, Niemann-Pick Disease Type C2, Ornithine Aminotransferase Deficiency, 3-Phosphoglycerate Dehydrogenase Deficiency, Leber Congenital Amaurosis 2, Dravet Syndrome, Mucopolysaccharidosis Type 3 A, Ornithine Translocase Deficiency, Carnitine-acylcarnitine Translocase Deficiency, Glucose Transporter Type 1 Deficiency Syndrome, Creatine Transporter Deficiency, Niemann-Pick Disease Type A, Pitt Hopkins Syndrome, Tuberous Sclerosis 1, Tuberous Sclerosis 2, Ataxia With Isolated Vitamin E Deficiency, Angelman Syndrome, Prader-Willi Syndrome, Homocystinuria, Permanent Neonatal Diabetes Mellitus, Transient Neonatal Diabetes Mellitus, Factor VII Deficiency, Glycogen Storage Disease Type IXA1, Glycogen Storage Disease, Type IXA2, Glycogen Storage Disease IC, Glycogen Storage Disease Type IB, Central Hypoventilation Syndrome With or Without Hirschsprung Disease
- Interventions
- Confirmatory Testing
- Diagnostic Test
- Lead sponsor
- RTI International
- Other
- Eligibility
- 1 Day to 31 Days
- Enrollment
- 30,000 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2018 – 2026
- U.S. locations
- 1
- States / cities
- Research Triangle Park, 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
- 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
- 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 – 2027
- U.S. locations
- 1
- States / cities
- Durham, North Carolina
- 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
- Allan-Herndon-Dudley Syndrome
- Interventions
- Tiratricol
- Drug
- Lead sponsor
- Rare Thyroid Therapeutics International AB
- Industry
- Eligibility
- Up to 30 Months · Male only
- Enrollment
- 22 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2027
- U.S. locations
- 2
- States / cities
- Portland, Oregon • Philadelphia, Pennsylvania