- Conditions
- Adrenoleukodystrophy, Restless Legs Syndrome
- Interventions
- Pramipexole, Placebo
- Drug
- Lead sponsor
- Massachusetts General Hospital
- Other
- Eligibility
- 18 Years to 75 Years · Female only
- Enrollment
- 24 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2026
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
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Showing 1–24
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matching trials from the live ClinicalTrials.gov search.
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- Conditions
- Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias, Inflammatory Conditions, Systemic Juvenile Idiopathic Arthritis (sJIA), Juvenile Rheumatoid Arthritis (JRA)
- Interventions
- Hydroxyurea, Alemtuzumab, Fludarabine, Melphalan, Thiotepa
- Drug
- Lead sponsor
- Paul Szabolcs
- Other
- Eligibility
- 2 Months to 55 Years
- Enrollment
- 100 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2027
- U.S. locations
- 1
- States / cities
- Pittsburgh, Pennsylvania
- Conditions
- Adrenal Insufficiency
- Interventions
- Not listed
- Lead sponsor
- Tripler Army Medical Center
- Federal
- Eligibility
- 3 Months to 18 Years
- Enrollment
- 149 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2011
- U.S. locations
- 1
- States / cities
- Honolulu, Hawaii
- Conditions
- X-linked Adrenoleukodystrophy
- Interventions
- Lorenzo's Oil
- Drug
- Lead sponsor
- University of Minnesota
- Other
- Eligibility
- 18 Months to 18 Years · Male only
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota
- 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
- 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
- Adrenal Gland Hyperfunction, Adrenal Gland Hypofunction, Cushing's Syndrome, Healthy
- Interventions
- Ovine Corticotropin-Releasing Hormone (oCRH)
- Drug
- Lead sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- NIH
- Eligibility
- Not listed
- Enrollment
- 2,250 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 1982 – 2005
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Adrenoleukodystrophy
- Interventions
- glyceryl trierucate/glyceryl trioleate
- Drug
- Lead sponsor
- Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
- Other
- Eligibility
- 18 Months to 8 Years · Male only
- Enrollment
- 126 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 1998 – 2014
- U.S. locations
- 2
- States / cities
- Baltimore, Maryland
- Conditions
- Cerebral Adrenoleukodystrophy (CALD)
- Interventions
- Lenti-D Drug Product (eli-cel)
- Genetic
- Lead sponsor
- Genetix Biotherapeutics Inc.
- Industry
- Eligibility
- Up to 17 Years · Male only
- Enrollment
- 32 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2021
- U.S. locations
- 3
- States / cities
- Los Angeles, California • Boston, Massachusetts • Minneapolis, Minnesota
- Conditions
- Adrenal Insufficiency, Cirrhosis, Spur Cell Anemia, Lecithin Acyltransferase Deficiency
- Interventions
- Cosyntropin
- Drug
- Lead sponsor
- University of Virginia
- Other
- Eligibility
- 18 Years to 110 Years
- Enrollment
- 100 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2019
- U.S. locations
- 1
- States / cities
- Charlottesville, Virginia
- Conditions
- AMN, AMN Gene Mutation, X-ALD
- Interventions
- Natural History Observation
- Other
- Lead sponsor
- SwanBio Therapeutics, Inc.
- Industry
- Eligibility
- 18 Years and older · Male only
- Enrollment
- 65 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2025
- U.S. locations
- 4
- States / cities
- Stanford, California • Boston, Massachusetts • New York, New York + 1 more
- Conditions
- Adrenal Insufficiency, Osteopenia, Osteoporosis, Steroid Suppression of ACTH Secretion
- Interventions
- Low Dose Cosyntropin (ACTH) stimulation test, High Dose Cosyntropin (ACTH) stimulation test
- Drug
- Lead sponsor
- Nationwide Children's Hospital
- Other
- Eligibility
- 9 Years to 17 Years
- Enrollment
- 10 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2017 – 2019
- U.S. locations
- 1
- States / cities
- Columbus, Ohio
- 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
- 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
- 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
- Adrenal Insufficiency, Growth Disorder, Endocrine Diseases, Metabolic Disease, Bone Diseases, Metabolic
- Interventions
- Not listed
- Lead sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- NIH
- Eligibility
- 3 Months to 100 Years
- Enrollment
- 15,000 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2030
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Type 1 Diabetes, Hashimoto Disease, Graves Disease, Addison Disease, Autoimmune Polyglandular Syndrome Type III, Depression, Endocrine System Diseases
- Interventions
- Telehealth Resource Oriented Music Therapy
- Behavioral
- Lead sponsor
- Appalachian State University
- Other
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026
- U.S. locations
- 1
- States / cities
- Boone, North Carolina
- 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
- 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
- X-linked Adrenoleukodystrophy
- Interventions
- exercise training
- Behavioral
- Lead sponsor
- Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
- Other
- Eligibility
- 21 Years to 70 Years · Female only
- Enrollment
- 31 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2012 – 2014
- U.S. locations
- 1
- States / cities
- Baltimore, Maryland
- 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
- 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
- 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
- Adrenal Insufficiency, Excess Androgen, Congenital Adrenal Hyperplasia (CAH)
- Interventions
- Hydrocortisone (Solucortef), Insulin pump (Medtronic)
- Drug · Device
- Lead sponsor
- National Institutes of Health Clinical Center (CC)
- NIH
- Eligibility
- 18 Years to 99 Years
- Enrollment
- 8 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2016
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland