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Showing 49–71 of 71 matching trials from the live ClinicalTrials.gov search.
Conditions
Eosinophilic Esophagitis
Interventions
Cortisol level
Diagnostic Test
Lead sponsor
University of Colorado, Denver
Other
Eligibility
18 Years to 85 Years
Enrollment
300 participants
Timeline
2022 – 2023
U.S. locations
1
States / cities
Aurora, Colorado
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
Obesity, Addison's Disease
Interventions
Hydrocortisone, Isocaloric Diet
Drug · Dietary Supplement
Lead sponsor
Oregon Health and Science University
Other
Eligibility
18 Years and older
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2004
U.S. locations
1
States / cities
Portland, Oregon
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
Bone Marrow Failure Syndrome, Thalassemia, Sickle Cell Disease, Diamond Blackfan Anemia, Acquired Neutropenia in Newborn, Acquired Anemia Hemolytic, Acquired Thrombocytopenia, Hemophagocytic Lymphohistiocytoses, Wiskott-Aldrich Syndrome, Chronic Granulomatous Disease, Common Variable Immunodeficiency, X-linked Lymphoproliferative Disease, Severe Combined Immunodeficiency, Hurler Syndrome, Mannosidosis, Adrenoleukodystrophy
Interventions
Thiotepa--single daily dose, Thiotepa--escalated dose
Drug
Lead sponsor
University of Florida
Other
Eligibility
3 Months to 39 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2023
U.S. locations
1
States / cities
Gainesville, Florida
Conditions
Leukodystrophy, White Matter Disease, Leukoencephalopathies, 4H Syndrome, Adrenoleukodystrophy, AMN, ALD, ALD Gene Mutation, ALD (Adrenoleukodystrophy), X-linked Adrenoleukodystrophy, X-ALD, Adrenomyeloneuropathy, Aicardi Goutieres Syndrome, AGS, Alexander Disease, Alexanders Leukodystrophy, AxD, ADLD, Canavan Disease, CTX, Cerebrotendinous Xanthomatoses, Krabbe Disease, GALC Deficiency, Globoid Leukodystrophy, TUBB4A-Related Leukodystrophy, H-ABC - Hypomyelination, Atrophy of Basal Ganglia and Cerebellum, HBSL, HBSL - Hypomyelination, Brain Stem, Spinal Cord, Leg Spasticity, LBSL, Leukoencephalopathy With Brain Stem and Spinal Cord Involvement and High Lactate Syndrome (Disorder), Leukoencephalopathy With Brainstem and Spinal Cord Involvement and Lactate Elevation, ALSP, CSF1R Gene Mutation, HCC - Hypomyelination and Congenital Cataract, MLC1, Megalencephalic Leukoencephalopathy With Subcortical Cysts, MLD, Metachromatic Leukodystrophy, PMD, Pelizaeus-Merzbacher Disease, PLP1 Null Syndrome, PLP1 Gene Duplication | Blood or Tissue | Mutations, Pelizaeus Merzbacher Like Disease, Peroxisomal Biogenesis Disorder, Zellweger Syndrome, Refsum Disease, Salla Disease, Sialic Storage Disease, Sjögren, Sjogren-Larsson Syndrome, Van Der Knapp Disease, Vanishing White Matter Disease, Charcot-Marie-Tooth, CMT, Mct8 (Slc16A2)-Specific Thyroid Hormone Cell Transporter Deficiency, Allan-Herndon-Dudley Syndrome, Cadasil, Cockayne Syndrome, Multiple Sulfatase Deficiency, Gangliosidoses, GM2 Gangliosidosis, BPAN, Labrune Syndrome, LCC, Mucopolysaccharidoses, TBCK-Related Intellectual Disability Syndrome
Interventions
Not listed
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
Not listed
Enrollment
12,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2030
U.S. locations
23
States / cities
Los Angeles, California • Orange, California • Palo Alto, California + 18 more
Conditions
Sphingolipidoses, Enzyme Deficiency
Interventions
no intervention
Other
Lead sponsor
University of California, San Francisco
Other
Eligibility
Not listed
Enrollment
120 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2025 – 2028
U.S. locations
1
States / cities
San Francisco, California
Conditions
Adrenal Insufficiency, Pituitary Neoplasm, Obesity
Interventions
Not listed
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
18 Years to 100 Years
Enrollment
1,588 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2019
U.S. locations
1
States / cities
Bethesda, Maryland
Conditions
Adrenal Insufficiency Neonatal
Interventions
No pre-operative steroids
Other
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
1 Day to 60 Days
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
1
States / cities
Birmingham, Alabama
Conditions
Adrenoleukodystrophy
Interventions
MIN-102, Placebos
Drug
Lead sponsor
Minoryx Therapeutics, S.L.
Industry
Eligibility
18 Years to 65 Years · Male only
Enrollment
105 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2025
U.S. locations
3
States / cities
Stanford, California • Baltimore, Maryland • Boston, Massachusetts
Conditions
Mucopolysaccharidosis, Hurler Syndrome, Hunter Syndrome, Maroteaux-Lamy Syndrome, Sly Syndrome, Alpha Mannosidosis, Fucosidosis, Aspartylglucosaminuria, Adrenoleukodystrophy (ALD), Krabbe Disease, Metachromatic Leukodystrophy (MLD), Sphingolipidoses, Peroxisomal Disorders
Interventions
Campath-1H, Cyclophosphamide, Busulfan, Allogeneic stem cell transplantation, Cyclosporine A, Mycophenolate Mofetil
Drug · Procedure
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 21 Years
Enrollment
46 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2017
U.S. locations
1
States / cities
Minneapolis, Minnesota
Conditions
Generalized Myasthenia Gravis, gMG
Interventions
Oral Corticosteroid Tapering Schedule, Ravulizumab, Prednisone/Prednisolone
Procedure · Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older
Enrollment
75 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2027
U.S. locations
5
States / cities
Chicago, Illinois • Schaumburg, Illinois • Neptune City, New Jersey + 2 more
Conditions
SCAD, Addison Disease, Ankylosing Spondylitis, Antiphospholipid Antibody Syndrome, Celiac Disease, Crohn Disease, Dermatomyositis, Polymyositis, Guillain-Barre Syndrome, Hepatitis, Autoimmune, Graves Disease, Hashimoto Thyroiditis, Multiple Sclerosis, Myasthenia Gravis, Pernicious Anemia, Polymyalgia Rheumatica, Primary Biliary Cirrhosis, Psoriasis, Rheumatoid Arthritis, Systemic Sclerosis, Sjögren Syndrome, Systemic Lupus Erythematosus, Takayasu Arteritis, Type 1 Diabetes Mellitus, Ulcerative Colitis, Uveitis, Vasculitis, Vitiligo, Raynaud
Interventions
Not listed
Lead sponsor
Mayo Clinic
Other
Eligibility
18 Years to 110 Years
Enrollment
114 participants
Timeline
1995 – 2020
U.S. locations
1
States / cities
Rochester, Minnesota
Conditions
Central Adrenal Insufficiency, Mifepristone
Interventions
Mifepristone
Drug
Lead sponsor
Tobias Else
Other
Eligibility
18 Years and older
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021
U.S. locations
1
States / cities
Ann Arbor, Michigan
Conditions
Cerebral Adrenoleukodystrophy (CALD), Adrenoleukodystrophy (ALD), X-Linked Adrenoleukodystrophy (X-ALD)
Interventions
No interventional drug product utilized in this follow-up study
Genetic
Lead sponsor
Genetix Biotherapeutics Inc.
Industry
Eligibility
Up to 19 Years · Male only
Enrollment
64 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2038
U.S. locations
4
States / cities
Los Angeles, California • Palo Alto, California • Boston, Massachusetts + 1 more
Conditions
Infantile Refsum's Disease, Zellweger Syndrome, Adrenoleukodystrophy, Peroxisomal Disorders, Cholestasis
Interventions
Cholic Acids
Drug
Lead sponsor
Mirum Pharmaceuticals, Inc.
Industry
Eligibility
Not listed
Enrollment
85 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1992 – 2009
U.S. locations
1
States / cities
Cincinnati, Ohio
Conditions
Leukodystrophy, White Matter Disease, 4H Syndrome, Adrenoleukodystrophy, AMN, ALD, ALD (Adrenoleukodystrophy), X-linked Adrenoleukodystrophy, X-ALD, Adrenomyeloneuropathy, Aicardi Goutieres Syndrome, AGS, Alexander Disease, Alexanders Leukodystrophy, AxD, ADLD, Canavan Disease, CTX, Cerebrotendinous Xanthomatoses, Krabbe Disease, GALC Deficiency, Globoid Leukodystrophy, TUBB4A-Related Leukodystrophy, H-ABC - Hypomyelination, Atrophy of Basal Ganglia and Cerebellum, HBSL, HBSL - Hypomyelination, Brain Stem, Spinal Cord, Leg Spasticity, LBSL, Leukoencephalopathy With Brain Stem and Spinal Cord Involvement and High Lactate Syndrome (Disorder), Leukoencephalopathy With Brainstem and Spinal Cord Involvement and Lactate Elevation, ALSP, CSF1R Gene Mutation, HCC - Hypomyelination and Congenital Cataract, MLC1, Megalencephalic Leukoencephalopathy With Subcortical Cysts 1, MLD, Metachromatic Leukodystrophy, PMD, Pelizaeus-Merzbacher Disease, PLP1 Null Syndrome, PLP1 Gene Duplication | Blood or Tissue | Mutations, Pelizaeus-Merzbacher-Like Disease, 1, Peroxisomal Biogenesis Disorder, Zellweger Syndrome, Refsum Disease, Salla Disease, Sialic Storage Disease, Sjögren, Sjogren-Larsson Syndrome, Van Der Knapp Disease, Vanishing White Matter Disease, Charcot-Marie-Tooth, CMT, Mct8 (Slc16A2)-Specific Thyroid Hormone Cell Transporter Deficiency, Allan-Herndon-Dudley Syndrome, Cadasil, Cockayne Syndrome, Multiple Sulfatase Deficiency, Gangliosidoses, GM2 Gangliosidosis, BPAN, Labrune Syndrome, LCC, Mucopolysaccharidoses, TBCK-Related Intellectual Disability Syndrome
Interventions
Not listed
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
Up to 18 Years
Enrollment
236 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2024
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Conditions
Congenital Adrenal Hyperplasia (CAH), Familial Male-Limited Precocious Puberty (FMPP)
Interventions
Not listed
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
1 Day to 99 Years
Enrollment
3,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2006
U.S. locations
2
States / cities
Washington D.C., District of Columbia • Bethesda, Maryland
Conditions
Inherited Metabolic Diseases, Lysosomal Storage Disorders, Peroxisomal Storage Diseases, Inborn Errors of Metabolism, Mucopolysaccharidosis
Interventions
ALD-101
Biological
Lead sponsor
Aldagen
Industry
Eligibility
Up to 16 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2011
U.S. locations
3
States / cities
Los Angeles, California • New York, New York • Durham, North Carolina
Conditions
X-Linked Adrenoleukodystrophy, Adrenomyeloneuropathy
Interventions
Sobetirome
Drug
Lead sponsor
Thomas S. Scanlan
Other
Eligibility
18 Years to 64 Years · Male only
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2014
U.S. locations
1
States / cities
Portland, Oregon
Conditions
ALD (Adrenoleukodystrophy), Adrenoleukodystrophy, Cerebral Adrenoleukodystrophy
Interventions
Medical Record Abstraction, Biospecimen Sample Collection
Other
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Not listed
Enrollment
1,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2030
U.S. locations
1
States / cities
Minneapolis, Minnesota
Conditions
Sepsis, Relative Adrenal Insufficiency
Interventions
Hydrocortisone Administration (Standard of Care Therapy)
Drug
Lead sponsor
Stanford University
Other
Eligibility
18 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2006
U.S. locations
1
States / cities
Stanford, California