Independent directory Public ClinicalTrials.gov records United States
Clear filters
ClinicalTrials.gov public records Last synced Jul 22, 2026, 4:48 AM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Showing 1–24 of 70 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
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
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
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
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
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
X-Linked Adrenoleukodystrophy
Interventions
Not listed
Lead sponsor
NeuroVia, Inc.
Industry
Eligibility
2 Years to 13 Years · Male only
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
3
States / cities
Palo Alto, California • Boston, Massachusetts • Pittsburgh, Pennsylvania
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
Recruiting Not applicable Interventional Accepts healthy volunteers

Home Exercise for Individuals with Neurodegenerative Disease

NCT05443906
Conditions
Neurodegenerative Diseases, Leukodystrophy, Ataxia, LBSL, Leukoencephalopathy with Brain Stem and Spinal Cord Involvement and High Lactate Syndrome (Disorder), Leukoencephalopathy with Brainstem and Spinal Cord Involvement and Lactate Elevation, Cerebellar Ataxia, Adrenomyeloneuropathy
Interventions
Home exercise
Behavioral
Lead sponsor
Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Other
Eligibility
5 Years to 75 Years
Enrollment
30 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2023 – 2027
U.S. locations
1
States / cities
Baltimore, Maryland
Completed No phase listed Observational Results available

Pilot Study Assessing Oxidative Stress in Children

NCT01052207
Conditions
Adrenal Insufficiency, Critical Illness
Interventions
Not listed
Lead sponsor
Emory University
Other
Eligibility
1 Day to 18 Years
Enrollment
102 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2011
U.S. locations
1
States / cities
Atlanta, Georgia
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
Recruiting Not applicable Interventional Accepts healthy volunteers

Updated Diagnostic Cortisol Values for Adrenal Insufficiency

NCT05149638
Conditions
Adrenal Insufficiency
Interventions
Cosyntropin stimulation test
Diagnostic Test
Lead sponsor
Montefiore Medical Center
Other
Eligibility
18 Years and older
Enrollment
90 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2027
U.S. locations
1
States / cities
The Bronx, New York
Conditions
Adrenoleukodystrophy
Interventions
Not listed
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
1 Year to 5 Years · Male only
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2023
U.S. locations
1
States / cities
Minneapolis, Minnesota
Conditions
Adrenomyeloneuropathy, Adrenoleukodystrophy
Interventions
Lorenzo's oil
Drug
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
18 Years and older
Enrollment
240 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2007
U.S. locations
1
States / cities
Baltimore, Maryland
Completed Not applicable Interventional Results available

Evaluation of Etomidate on Adrenal Function in Trauma Patients

NCT00462644
Conditions
Adrenal Insufficiency
Interventions
RSI sedation with etomidate/succinylcholine, RSI sedation with fentanyl/midazolam/succinylcholine
Drug
Lead sponsor
University of Tennessee
Other
Eligibility
18 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006
U.S. locations
1
States / cities
Chattanooga, Tennessee
Completed Phase 2Phase 3 Interventional Results available

Stem Cell Transplant for Inborn Errors of Metabolism

NCT00176904
Conditions
Adrenoleukodystrophy, Metachromatic Leukodystrophy, Globoid Cell Leukodystrophy, Gaucher's Disease, Fucosidosis, Wolman Disease, Niemann-Pick Disease, Batten Disease, GM1 Gangliosidosis, Tay Sachs Disease, Sandhoff Disease
Interventions
Stem Cell Transplant, Busulfan, Cyclophosphamide, Antithymocyte Globulin
Procedure · Drug
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Not listed
Enrollment
135 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1995 – 2010
U.S. locations
1
States / cities
Minneapolis, Minnesota
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
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
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
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
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
Hematologic Malignancies, Inborn Errors of Metabolism Disorders, Immune Deficiencies
Interventions
CliniMACS CD34 Reagent System
Biological
Lead sponsor
Joanne Kurtzberg, MD
Other
Eligibility
Up to 65 Years
U.S. locations
1
States / cities
Durham, North Carolina
Conditions
Amenorrhea, Hypoaldosteronism, Hypogonadism, Infertility, Premature Ovarian Failure
Interventions
Not listed
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
18 Years to 42 Years · Female only
Enrollment
1,134 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1991 – 2017
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 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