- 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
Search Results
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Showing 25–48
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matching trials from the live ClinicalTrials.gov search.
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- 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
- Zellweger Spectrum
- Interventions
- Not listed
- Lead sponsor
- University of South Florida
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 92 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2018
- U.S. locations
- 1
- States / cities
- Tampa, Florida
- 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
- Conditions
- Charcot-Marie-Tooth Disease, Hereditary Motor and Sensory Neuropathy IV
- Interventions
- Not listed
- Lead sponsor
- Neurogene Inc.
- Industry
- Eligibility
- Not listed
- Enrollment
- 21 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2022
- U.S. locations
- 2
- States / cities
- Iowa City, Iowa • Dallas, Texas
- Conditions
- Bipolar Disorder, Mania
- Interventions
- Not listed
- Lead sponsor
- University of Cincinnati
- Other
- Eligibility
- 10 Years to 18 Years
- Enrollment
- 80 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2010 – 2013
- U.S. locations
- 1
- States / cities
- Cincinnati, Ohio
- Conditions
- Mucopolysaccharidosis Disorders, Hurler Syndrome, Hunter Syndrome, Maroteaux Lamy Syndrome, Sly Syndrome, Alpha-Mannosidosis, Fucosidosis, Aspartylglucosaminuria, Glycoprotein Metabolic Disorders, Sphingolipidoses, Recessive Leukodystrophies, Globoid Cell Leukodystrophy, Metachromatic Leukodystrophy, Niemann-Pick B, Niemann-Pick C Subtype 2, Sphingomyelin Deficiency, Peroxisomal Disorders, Adrenoleukodystrophy With Cerebral Involvement, Zellweger Syndrome, Neonatal Adrenoleukodystrophy, Infantile Refsum Disease, Acyl-CoA Oxidase Deficiency, D-Bifunctional Enzyme Deficiency, Multifunctional Enzyme Deficiency, Alpha-methylacyl-CoA Racmase Deficiency, Mitochondrial Neurogastrointestingal Encephalopathy, Severe Osteopetrosis, Hereditary Leukoencephalopathy With Axonal Spheroids (HDLS; CSF1R Mutation), Inherited Metabolic Disorders
- Interventions
- Stem Cell Transplantation, IMD Preparative Regimen, Osteopetrosis Only Preparative Regimen, Osteopetrosis Haploidentical Only Preparative Regimen, cALD SR-A (Standard-Risk, Regimen A), cALD SR-B (Standard-Risk, Regimen B), cALD HR-D (High-Risk, Regimen C), cALD HR-D (High-Risk, Regimen D)
- Biological · Drug
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Other
- Eligibility
- Up to 55 Years
- Enrollment
- 149 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2029
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota
- 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
- Hyper IgD Syndrome, Mevalonate Kinase Deficiency
- Interventions
- Not listed
- Lead sponsor
- Michigan Technological University
- Other
- Eligibility
- 18 Years to 89 Years
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2016
- U.S. locations
- 3
- States / cities
- Houghton, Michigan • Omaha, Nebraska • 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
- 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
- Conditions
- Cerebral Adrenoleukodystrophy (CALD)
- Interventions
- Lenti-D
- Genetic
- Lead sponsor
- Genetix Biotherapeutics Inc.
- Industry
- Eligibility
- Up to 17 Years · Male only
- Enrollment
- 35 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2023
- U.S. locations
- 3
- States / cities
- Palo Alto, California • Boston, Massachusetts • Minneapolis, Minnesota
- Conditions
- Bile Acid Synthesis Disorders
- Interventions
- Cholbam
- Drug
- Lead sponsor
- Mirum Pharmaceuticals, Inc.
- Industry
- Eligibility
- Not listed
- Enrollment
- 55 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2039
- U.S. locations
- 24
- States / cities
- Birmingham, Alabama • Palo Alto, California • San Francisco, California + 18 more
- 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
- 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
- Eye Diseases Hereditary, Retinal Disease, Achromatopsia, Bardet-Biedl Syndrome, Bassen-Kornzweig Syndrome, Batten Disease, Best Disease, Choroidal Dystrophy, Choroideremia, Cone Dystrophy, Cone-Rod Dystrophy, Congenital Stationary Night Blindness, Enhanced S-Cone Syndrome, Fundus Albipunctatus, Goldmann-Favre Syndrome, Gyrate Atrophy, Juvenile Macular Degeneration, Kearns-Sayre Syndrome, Leber Congenital Amaurosis, Refsum Syndrome, Retinitis Pigmentosa, Retinitis Punctata Albescens, Retinoschisis, Rod-Cone Dystrophy, Rod Dystrophy, Rod Monochromacy, Stargardt Disease, Usher Syndrome
- Interventions
- Not listed
- Lead sponsor
- Foundation Fighting Blindness
- Other
- Eligibility
- Not listed
- Enrollment
- 20,000 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2014 – 2037
- U.S. locations
- 1
- States / cities
- Columbia, Maryland
- 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, 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
- Rhizomelic Chondrodysplasia Punctata
- Interventions
- Not listed
- Lead sponsor
- MED-LIFE DISCOVERIES LP
- Industry
- Eligibility
- 6 Months to 21 Years
- Enrollment
- 75 participants
- Timeline
- 2019 – 2021
- U.S. locations
- 1
- States / cities
- Wilmington, Delaware
- 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
- 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
- Mevalonic Aciduria, Mevalonate Kinase Deficiency, Immune System Diseases, Periodic Fever Syndromes, Hereditary, Lipid Metabolism, Inborn Errors
- Interventions
- Not listed
- Lead sponsor
- Oregon Health and Science University
- Other
- Eligibility
- Not listed
- Enrollment
- 1 participant
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2005 – 2016
- U.S. locations
- 1
- States / cities
- Portland, Oregon
- 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
- 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