- Conditions
- Lysosomal Acid Lipase Deficiency
- Interventions
- Sebelipase Alfa
- Drug
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Industry
- Eligibility
- 8 Months and older
- Enrollment
- 31 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2017
- U.S. locations
- 3
- States / cities
- Chicago, Illinois • Shreveport, Louisiana • Cincinnati, Ohio
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 97–120
of 501
matching trials from the live ClinicalTrials.gov search.
- Conditions
- LCAT Deficiency
- Interventions
- Demographics, diagnosis type, genotype, lipid profile, renal function profile, treatment allocation, ophthalmology exam, country of residence.
- Other
- Lead sponsor
- University of Pennsylvania
- Other
- Eligibility
- Not listed
- Enrollment
- 40 participants
- Timeline
- 2022 – 2028
- U.S. locations
- 1
- States / cities
- Philadelphia, Pennsylvania
- Conditions
- Fabry Disease
- Interventions
- Not listed
- Lead sponsor
- University of Washington
- Other
- Eligibility
- 1 Day to 90 Years
- Enrollment
- 58 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2019
- U.S. locations
- 3
- States / cities
- Decatur, Georgia • Minneapolis, Minnesota • Seattle, Washington
- Conditions
- Niemann-Pick Type C Disease
- Interventions
- Adrabetadex
- Drug
- Lead sponsor
- Mandos LLC
- Industry
- Eligibility
- 4 Years and older
- Enrollment
- 27 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2021
- U.S. locations
- 17
- States / cities
- Little Rock, Arkansas • Loma Linda, California • Los Angeles, California + 13 more
- Conditions
- Fabry Disease
- Interventions
- agalsidase beta
- Biological
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- 16 Years and older
- Enrollment
- 67 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2004 – 2005
- U.S. locations
- 18
- States / cities
- Birmingham, Alabama • Los Angeles, California • San Francisco, California + 15 more
- Conditions
- Fabry Disease
- Interventions
- Iohexol
- Drug
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- 5 Years to 25 Years · Male only
- Enrollment
- 39 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2016
- U.S. locations
- 5
- States / cities
- La Jolla, California • Decatur, Georgia • Cincinnati, Ohio + 2 more
- Conditions
- Type 2 Gaucher Disease
- Interventions
- Not listed
- Lead sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
- NIH
- Eligibility
- Up to 5 Years · Male only
- Enrollment
- 1 participant
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2005 – 2006
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Parkinson Disease, GBA Gene Mutation, Gaucher Disease
- Interventions
- PET scan, neuroQWERTY
- Diagnostic Test
- Lead sponsor
- Pacific Parkinson's Research Centre
- Other
- Eligibility
- 18 Years to 80 Years
- Enrollment
- 30 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2026
- U.S. locations
- 2
- States / cities
- Portland, Oregon • Seattle, Washington
- Conditions
- Gaucher Disease, Type 1 Gaucher Disease, Gaucher Disease, Type 1
- Interventions
- afegostat tartrate
- Drug
- Lead sponsor
- Amicus Therapeutics
- Industry
- Eligibility
- 18 Years to 74 Years
- Enrollment
- 19 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2009
- U.S. locations
- 7
- States / cities
- Beverly Hills, California • Coral Springs, Florida • Decatur, Georgia + 4 more
- Conditions
- Cholesterol Ester Storage Disease(CESD), Lysosomal Acid Lipase Deficiency
- Interventions
- Not listed
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Industry
- Eligibility
- 5 Years and older
- Enrollment
- 49 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2013
- U.S. locations
- 6
- States / cities
- Stanford, California • Chicago, Illinois • Minneapolis, Minnesota + 3 more
- Conditions
- Gaucher Disease, Bone Diseases
- Interventions
- Not listed
- Lead sponsor
- Lysosomal and Rare Disorders Research and Treatment Center, Inc.
- Other
- Eligibility
- 5 Years to 21 Years
- Enrollment
- 20 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2023 – 2025
- U.S. locations
- 1
- States / cities
- Fairfax, Virginia
- 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
- Sjogren-Larsson Syndrome
- Interventions
- ADX-629
- Drug
- Lead sponsor
- University of Nebraska
- Other
- Eligibility
- 5 Years to 50 Years
- Enrollment
- 8 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2026
- U.S. locations
- 1
- States / cities
- Omaha, Nebraska
- Conditions
- Mucopolysaccharidosis I, Mucopolysaccharidosis II, Mucopolysaccharidosis IV A, Mucopolysaccharidosis VI, Mucopolysaccharidosis VII, Pompe Disease Infantile-Onset, Neuronopathic Gaucher Disease, Wolman Disease
- Interventions
- There is no intervention
- Other
- Lead sponsor
- University of California, San Francisco
- Other
- Eligibility
- Up to 64 Years
- Enrollment
- 250 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2050
- U.S. locations
- 1
- States / cities
- San Francisco, California
- Conditions
- Lysosomal Storage Diseases, Inborn Errors of Metabolism
- Interventions
- ALD-601
- Biological
- Lead sponsor
- Joanne Kurtzberg, MD
- Other
- Eligibility
- 18 Years and older · Female only
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2017
- U.S. locations
- 2
- States / cities
- Durham, North Carolina
- Conditions
- MLD, Krabbe Disease, ALD, MPS I, MPS II, MPS III, Vanishing White Matter Disease, GM3 Gangliosidosis, PKAN, Tay-Sachs Disease, NP Deficiency, Osteopetrosis, Alpha-Mannosidosis, Sandhoff Disease, Niemann-Pick Diseases, MPS IV, Gaucher Disease, GAN, GM1 Gangliosidoses, Morquio Disease, S-Adenosylhomocysteine Hydrolase Deficiency, Batten Disease, Pelizaeus-Merzbacher Disease, Leukodystrophy, Lysosomal Storage Diseases, Purine Nucleoside Phosphorylase Deficiency, Multiple Sulfatase Deficiency Disease
- Interventions
- Palliative Care, Hematopoetic Stem Cell Transplantation
- Other · Biological
- Lead sponsor
- University of Pittsburgh
- Other
- Eligibility
- Not listed
- Enrollment
- 1,500 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2035
- U.S. locations
- 1
- States / cities
- Pittsburgh, Pennsylvania
- Conditions
- Fabry Disease
- Interventions
- Not listed
- Lead sponsor
- Baylor Research Institute
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 2,724 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2009 – 2017
- U.S. locations
- 1
- States / cities
- Dallas, Texas
- Conditions
- G(M2) Ganglioside, Tay-Sachs Disease Ganglioside, Sandhoff Disease Ganglioside
- Interventions
- Pyrimethamine
- Drug
- Lead sponsor
- Exsar Corporation
- Industry
- Eligibility
- 18 Years and older
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Started 2008
- U.S. locations
- 2
- States / cities
- New York, New York • Cleveland, Ohio
- Conditions
- Smith-Lemli-Opitz Syndrome
- Interventions
- Simvastatin, Lactose
- Drug · Dietary Supplement
- Lead sponsor
- Oregon Health and Science University
- Other
- Eligibility
- 1 Year to 89 Years
- Enrollment
- 1 participant
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2014
- U.S. locations
- 1
- States / cities
- Portland, Oregon
- Conditions
- Fabry Disease, Alpha Galactosidase A Deficiency
- Interventions
- agalsidase beta
- Drug
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- Not listed
- Enrollment
- 7 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2006 – 2024
- U.S. locations
- 3
- States / cities
- Decatur, Georgia • Fairfax, Virginia • Milwaukee, Wisconsin
- Conditions
- Lipid Metabolism, Inborn Errors, Hypercholesterolemia, Autosomal Dominant, Hyperlipidemias, Metabolic Diseases, Hyperlipoproteinemia Type II, Metabolism, Inborn Errors, Genetic Diseases, Inborn, Infant, Newborn, Diseases, Metabolic Disorder, Congenital Abnormalities, Hypercholesterolemia, Hyperlipoproteinemias, Dyslipidemias, Lipid Metabolism Disorders
- Interventions
- Mipomersen Sodium
- Drug
- Lead sponsor
- Kastle Therapeutics, LLC
- Industry
- Eligibility
- 12 Years and older
- Enrollment
- 144 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2014
- U.S. locations
- 20
- States / cities
- Mission Viejo, California • Newport Beach, California • Bridgeport, Connecticut + 17 more
- Conditions
- Fabry Disease
- Interventions
- Not listed
- Lead sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
- NIH
- Eligibility
- 18 Years and older · Male only
- Enrollment
- 35 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2005 – 2008
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Heterozygous or Homozygous Familial Hypercholesterolemia
- Interventions
- Inclisiran
- Drug
- Lead sponsor
- Novartis Pharmaceuticals
- Industry
- Eligibility
- 2 Years to 100 Years
- Enrollment
- 195 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2032
- U.S. locations
- 5
- States / cities
- Boca Raton, Florida • New York, New York • Cincinnati, Ohio + 2 more
- Conditions
- Fabry Disease
- Interventions
- Lucerastat
- Drug
- Lead sponsor
- Idorsia Pharmaceuticals Ltd.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 107 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2029
- U.S. locations
- 14
- States / cities
- Birmingham, Alabama • Irvine, California • Oakland, California + 10 more