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ClinicalTrials.gov public records Last synced May 21, 2026, 9:06 PM 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
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
Source: ClinicalTrials.gov public record
Updated Apr 24, 2022 · Synced May 21, 2026, 9:06 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers Results available
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
Source: ClinicalTrials.gov public record
Updated Aug 28, 2018 · Synced May 21, 2026, 9:06 PM EDT
Conditions
Endocrine Disease, Adrenal Insufficiency, Congenital Adrenal Hyperplasia
Interventions
Hydrocortisone Modified Release Capsules
Drug
Lead sponsor
Neurocrine UK Limited
Industry
Eligibility
18 Years and older
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2013
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated May 16, 2017 · Synced May 21, 2026, 9:06 PM EDT
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
Source: ClinicalTrials.gov public record
Updated May 6, 2026 · Synced May 21, 2026, 9:06 PM EDT
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
Source: ClinicalTrials.gov public record
Updated Dec 15, 2019 · Synced May 21, 2026, 9:06 PM EDT
Conditions
Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias, Inflammatory Conditions
Interventions
data collection
Drug
Lead sponsor
Paul Szabolcs
Other
Eligibility
2 Months to 60 Years
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2028
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jan 12, 2026 · Synced May 21, 2026, 9:06 PM EDT
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
Source: ClinicalTrials.gov public record
Updated Nov 19, 2025 · Synced May 21, 2026, 9:06 PM EDT
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
Source: ClinicalTrials.gov public record
Updated Oct 23, 2025 · Synced May 21, 2026, 9:06 PM EDT
Conditions
Adrenal Insufficiency
Interventions
Not listed
Lead sponsor
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
NIH
Eligibility
15 Years to 65 Years
Enrollment
225 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2004
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated May 6, 2026 · Synced May 21, 2026, 9:06 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers Results available
Conditions
Adrenal Insufficiency
Interventions
megestrol acetate
Drug
Lead sponsor
University of Arkansas
Other
Eligibility
65 Years to 80 Years
Enrollment
7 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2004
U.S. locations
1
States / cities
Little Rock, Arkansas
Source: ClinicalTrials.gov public record
Updated Aug 28, 2011 · Synced May 21, 2026, 9:06 PM EDT
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
Source: ClinicalTrials.gov public record
Updated Dec 21, 2017 · Synced May 21, 2026, 9:06 PM EDT
Conditions
Septic Shock, Acute Adrenal Insufficiency
Interventions
Corticosteroid
Drug
Lead sponsor
The Methodist Hospital Research Institute
Other
Eligibility
18 Years and older
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2011
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jun 28, 2011 · Synced May 21, 2026, 9:06 PM EDT
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
Source: ClinicalTrials.gov public record
Updated Jul 5, 2018 · Synced May 21, 2026, 9:06 PM EDT
Completed No phase listed Observational Results available
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
Source: ClinicalTrials.gov public record
Updated Mar 10, 2013 · Synced May 21, 2026, 9:06 PM EDT
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
Source: ClinicalTrials.gov public record
Updated Feb 20, 2019 · Synced May 21, 2026, 9:06 PM EDT
Conditions
Lysosomal Storage Disease, Peroxisomal Disorder
Interventions
Campath-1H, Clofarabine, Melphalan, Total Body Irradiation with Marrow Boosting, Hematopoietic stem cell transplantation, Cyclosporine A, Mycophenolate mofetil
Drug · Radiation · Biological
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 55 Years
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2013
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Dec 4, 2017 · Synced May 21, 2026, 9:06 PM EDT
Conditions
Cerebral Adrenoleukodystrophy (CALD)
Interventions
No Intervention
Other
Lead sponsor
Genetix Biotherapeutics Inc.
Industry
Eligibility
Male only
Enrollment
120 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2047
U.S. locations
3
States / cities
Boston, Massachusetts • Philadelphia, Pennsylvania • Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Jul 1, 2025 · Synced May 21, 2026, 9:06 PM EDT
Conditions
Sphingosine Phosphate Lyase Insufficiency Syndrome (SPLIS)
Interventions
no intervention
Other
Lead sponsor
University of California, San Francisco
Other
Eligibility
Not listed
Enrollment
28 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2030
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated May 12, 2025 · Synced May 21, 2026, 9:06 PM EDT
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
Source: ClinicalTrials.gov public record
Updated Oct 22, 2025 · Synced May 21, 2026, 9:06 PM EDT
Conditions
X-linked Adrenoleukodystrophy
Interventions
vitamin D3
Dietary Supplement
Lead sponsor
Stanford University
Other
Eligibility
18 Months to 25 Years · Male only
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2020
U.S. locations
1
States / cities
Palo Alto, California
Source: ClinicalTrials.gov public record
Updated Jun 8, 2022 · Synced May 21, 2026, 9:06 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers Results available
Conditions
Adrenal Insufficiency
Interventions
1 ug ACTH stimulation test, 250 ug ACTH stimulation test, 25 ug Cortrosyn stimulation test, Insulin tolerance test
Procedure
Lead sponsor
The Cleveland Clinic
Other
Eligibility
18 Years to 65 Years
Enrollment
22 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2011 – 2015
U.S. locations
1
States / cities
Cleveland, Ohio
Source: ClinicalTrials.gov public record
Updated Jul 24, 2017 · Synced May 21, 2026, 9:06 PM EDT
Recruiting Not applicable Interventional Accepts healthy volunteers
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
Source: ClinicalTrials.gov public record
Updated Feb 18, 2026 · Synced May 21, 2026, 9:06 PM EDT
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
Source: ClinicalTrials.gov public record
Updated Mar 1, 2012 · Synced May 21, 2026, 9:06 PM EDT
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
Source: ClinicalTrials.gov public record
Updated Apr 10, 2011 · Synced May 21, 2026, 9:06 PM EDT