- 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
Search Results
Search by objective public record fields.
Search by query text, NCT ID, condition, intervention, sponsor, city, state, recruitment status, phase, study type, healthy volunteer eligibility, sex, or age. Results come from ClinicalTrials.gov. When the live registry is unavailable, a clearly labeled stored copy may be shown.
These results were retrieved from the live ClinicalTrials.gov registry. Filters and sort order apply to this result list.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Showing 25–48
of 71
matching trials from the live ClinicalTrials.gov search.
- 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
- 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
- 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
- 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
- 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
- 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
- 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
- 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
- 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
- Prader Willi Syndrome, Adrenal Insufficiency
- Interventions
- Low dose (1 mcg) ACTH stimulation test, Overnight metyrapone test
- Other
- Lead sponsor
- Nationwide Children's Hospital
- Other
- Eligibility
- 2 Years and older
- Enrollment
- 23 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2014 – 2016
- U.S. locations
- 1
- States / cities
- Columbus, Ohio
- 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
- 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
- X-Linked Adrenoleukodystrophy (X-ALD), Cerebral Adrenoleukodystrophy (CALD), Adrenoleukodystrophy (ALD)
- Interventions
- Allo-HSCT
- Genetic
- Lead sponsor
- Genetix Biotherapeutics Inc.
- Industry
- Eligibility
- Up to 17 Years · Male only
- Enrollment
- 59 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2019
- U.S. locations
- 6
- States / cities
- Los Angeles, California • Palo Alto, California • Boston, Massachusetts + 3 more
- 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
- 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
- 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
- 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
- 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
- Adrenoleukodystrophy, Metachromatic Leukodystrophy, Globoid Cell Leukodystrophy, Tay Sachs Disease, Sandhoffs Disease, Wolman Disease, I-Cell Disease, Sanfilippo Syndrome, GM1 Gangliosidosis
- Interventions
- Clofarabine, Total body Irradiation, Melphalan, Hematopoietic Stem Cell Transplantation, Alemtuzumab, mycophenylate mofetil, Cyclosporine A, Hydroxyurea
- Drug · Procedure · Biological + 1 more
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Other
- Eligibility
- Up to 70 Years
- Enrollment
- 38 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2006 – 2014
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota
- 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
- 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
- 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