- Conditions
- Fabry Disease
- Interventions
- AMT-191
- Drug
- Lead sponsor
- UniQure Biopharma B.V.
- Industry
- Eligibility
- 18 Years to 65 Years · Male only
- Enrollment
- 20 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2031
- U.S. locations
- 10
- States / cities
- Birmingham, Alabama • Los Angeles, California • Atlanta, Georgia + 7 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 145–168
of 1,605
matching trials from the live ClinicalTrials.gov search.
- Conditions
- Mucopolysaccharidosis VII, MPS VII, MPS 7, Sly Syndrome
- Interventions
- No Intervention
- Other
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Industry
- Eligibility
- Not listed
- Enrollment
- 50 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2032
- U.S. locations
- 7
- States / cities
- Orange, California • Washington D.C., District of Columbia • Chicago, Illinois + 4 more
- Conditions
- Fructose Intolerance
- Interventions
- Placebo, Alanine
- Dietary Supplement
- Lead sponsor
- Augusta University
- Other
- Eligibility
- 18 Years to 70 Years
- Enrollment
- 100 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2007 – 2024
- U.S. locations
- 1
- States / cities
- Iowa City, Iowa
- Conditions
- Congenital Adrenal Hyperplasia
- Interventions
- Lu AG13909
- Drug
- Lead sponsor
- H. Lundbeck A/S
- Industry
- Eligibility
- 18 Years to 70 Years
- Enrollment
- 42 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2027
- U.S. locations
- 1
- States / cities
- Ann Arbor, Michigan
- Conditions
- TTR-mediated Amyloidosis, Amyloidosis, Hereditary, Amyloid Neuropathies, Familial, Familial Amyloid Polyneuropathies, Amyloid Neuropathies, Amyloidosis, Hereditary, Transthyretin-Related
- Interventions
- patisiran (ALN-TTR02), Sterile Normal Saline (0.9% NaCl)
- Drug
- Lead sponsor
- Alnylam Pharmaceuticals
- Industry
- Eligibility
- 18 Years to 85 Years
- Enrollment
- 225 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2017
- U.S. locations
- 13
- States / cities
- La Mesa, California • Orange, California • Denver, Colorado + 9 more
- 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
- Hypercholesterolemia, Familial
- Interventions
- Torcetrapib/atorvastatin
- Drug
- Lead sponsor
- Pfizer
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 30 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2005
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- Conditions
- Familial Partial Lipodystrophy, Hypertriglyceridemia, Fatty Liver, NASH - Nonalcoholic Steatohepatitis
- Interventions
- 300mg Gemcabene, 600mg Gemcabene
- Drug
- Lead sponsor
- Elif Oral
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 5 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2019
- U.S. locations
- 1
- States / cities
- Ann Arbor, Michigan
- Conditions
- Homozygous Familial Hypercholesterolemia
- Interventions
- REGN1500 250 mg SC/15 mg/kg IV/450 mg SC
- Drug
- Lead sponsor
- Regeneron Pharmaceuticals
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 9 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2018
- U.S. locations
- 3
- States / cities
- Los Angeles, California • Philadelphia, Pennsylvania • Dallas, Texas
- 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
- Fabry Disease
- Interventions
- PRX-102
- Drug
- Lead sponsor
- Protalix
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 18 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2016
- U.S. locations
- 9
- States / cities
- Sacramento, California • Atlanta, Georgia • Iowa City, Iowa + 6 more
- Conditions
- Pompe Disease
- Interventions
- Not listed
- Lead sponsor
- O & O Alpan LLC
- Other
- Eligibility
- 18 Years and older · Female only
- Enrollment
- 20 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2016
- U.S. locations
- 1
- States / cities
- Fairfax, Virginia
- 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
- X-Linked Hypophosphatemia
- Interventions
- KRN23
- Biological
- Lead sponsor
- Kyowa Kirin, Inc.
- Industry
- Eligibility
- Not listed
- Enrollment
- 20 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2018
- U.S. locations
- 5
- States / cities
- San Francisco, California • New Haven, Connecticut • Indianapolis, Indiana + 2 more
- Conditions
- Gaucher's Disease
- Interventions
- human glucocerebrosidase cDNA
- Genetic
- Lead sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
- NIH
- Eligibility
- Not listed
- Enrollment
- 120 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 1988 – 2002
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Hutchinson-Gilford Progeria Syndrome, Werner Syndrome
- Interventions
- Progerinin, Placebo
- Drug
- Lead sponsor
- PRG Science & Technology Co., Ltd.
- Industry
- Eligibility
- 18 Years to 45 Years
- Enrollment
- 64 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2020 – 2021
- U.S. locations
- 1
- States / cities
- Glendale, California
- Conditions
- Urea Cycle Disorders, Inborn
- Interventions
- Sodium Benzoate, Sodium Phenylbutyrate
- Drug
- Lead sponsor
- Baylor College of Medicine
- Other
- Eligibility
- 18 Years to 64 Years
- Enrollment
- 7 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2014 – 2015
- U.S. locations
- 1
- States / cities
- Houston, Texas
- Conditions
- Non Malignant Disorders, Immunodeficiencies, Congenital Marrow Failures, Hemoglobinopathies, Inborn Errors of Metabolism, Sickle Cell, Thalassemia, Lysosomal Storage Disease
- Interventions
- Unrelated Umbilical Cord Blood Transplant, Reduced Intensity Conditioning
- Biological · Drug
- Lead sponsor
- Duke University
- Other
- Eligibility
- Up to 21 Years
- Enrollment
- 22 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2014
- U.S. locations
- 1
- States / cities
- Durham, North Carolina
- Conditions
- Maple Syrup Urine Disease
- Interventions
- Neurocognitive testing
- Behavioral
- Lead sponsor
- Children's Hospital of Philadelphia
- Other
- Eligibility
- 21 Years to 80 Years
- Enrollment
- 44 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2021 – 2023
- U.S. locations
- 1
- States / cities
- Philadelphia, Pennsylvania
- Conditions
- Adrenal Hyperplasia, Congenital
- Interventions
- Not listed
- Lead sponsor
- Office of Rare Diseases (ORD)
- NIH
- Eligibility
- 12 Years and older
- Enrollment
- 233 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2008 – 2009
- U.S. locations
- 2
- States / cities
- New York, New York • Dallas, Texas
- 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
- Inherited Metabolic Disorders, Undiagnosed Diseases, Batten's Disease, Tay Sachs, Diabetes
- Interventions
- Not listed
- Lead sponsor
- National Human Genome Research Institute (NHGRI)
- NIH
- Eligibility
- 18 Years to 100 Years
- Enrollment
- 2,800 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2022 – 2030
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Homozygous Familial Hypercholesterolemia (HoFH)
- Interventions
- Single intravenous (IV) dose of human Low Density Lipoprotein Receptor (LDLR) Gene Therapy
- Drug
- Lead sponsor
- REGENXBIO Inc.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 8 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2025
- U.S. locations
- 4
- States / cities
- Columbus, Ohio • Portland, Oregon • Philadelphia, Pennsylvania + 1 more
- Conditions
- Alpha-Mannosidosis
- Interventions
- Not listed
- Lead sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- NIH
- Eligibility
- 5 Years to 60 Years
- Enrollment
- 11 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2019
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland