- Conditions
- Pompe Disease (Late-onset)
- Interventions
- AT2221, ATB200
- Drug · Biological
- Lead sponsor
- Amicus Therapeutics
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 119 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2024
- U.S. locations
- 18
- States / cities
- Little Rock, Arkansas • Irvine, California • Gainesville, Florida + 14 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 49–72
of 1,608
matching trials from the live ClinicalTrials.gov search.
- Conditions
- Amyloidosis Cardiac, Healthy Adults
- Interventions
- Cardiac magnetic resonance image (CMR), Transthoracic Echocardiogram (TTE), Six-minute Walk Test
- Diagnostic Test
- Lead sponsor
- Mayo Clinic
- Other
- Eligibility
- 40 Years and older
- Enrollment
- 81 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2022 – 2026
- U.S. locations
- 1
- States / cities
- Rochester, Minnesota
- 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
- 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
- Barth Syndrome
- Interventions
- Not listed
- Lead sponsor
- Duke University
- Other
- Eligibility
- 8 Years to 35 Years · Male only
- Enrollment
- 64 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2012 – 2020
- U.S. locations
- 1
- States / cities
- St Louis, Missouri
- Conditions
- Methylmalonic Acidemia, Propionic Acidemia
- Interventions
- Not listed
- Lead sponsor
- ModernaTX, Inc.
- Industry
- Eligibility
- Not listed
- Enrollment
- 97 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2021
- U.S. locations
- 11
- States / cities
- Stanford, California • Atlanta, Georgia • Chicago, Illinois + 8 more
- Conditions
- Homozygous Familial Hypercholesterolemia
- Interventions
- AEGR-733
- Drug
- Lead sponsor
- Aegerion Pharmaceuticals, Inc.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 29 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2007 – 2011
- U.S. locations
- 2
- States / cities
- Los Angeles, California • Philadelphia, Pennsylvania
- Conditions
- Brain Diseases, Metabolic, Inborn, Urea Cycle Disorder, Ornithine Transcarbamylase Deficiency
- Interventions
- Not listed
- Lead sponsor
- Andrea Gropman
- Other
- Eligibility
- 18 Years to 60 Years
- Enrollment
- 46 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2007 – 2010
- U.S. locations
- 2
- States / cities
- Washington D.C., District of Columbia
- Conditions
- Gout
- Interventions
- RDEA3170
- Drug
- Lead sponsor
- Ardea Biosciences, Inc.
- Industry
- Eligibility
- 18 Years to 85 Years · Male only
- Enrollment
- 31 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2016
- U.S. locations
- 4
- States / cities
- Lakewood, Colorado • Orlando, Florida • Minneapolis, Minnesota + 1 more
- 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
- Methylmalonic Acidemia, Propionic Acidemia
- Interventions
- HST5040, Placebo
- Drug
- Lead sponsor
- HemoShear Therapeutics
- Industry
- Eligibility
- 2 Years and older
- Enrollment
- 26 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2023
- U.S. locations
- 14
- States / cities
- San Diego, California • New Haven, Connecticut • Washington D.C., District of Columbia + 11 more
- Conditions
- Primary Hyperoxaluria
- Interventions
- Oxalobacter formigenes, Placebo
- Biological · Drug
- Lead sponsor
- OxThera
- Industry
- Eligibility
- 2 Years and older
- Enrollment
- 36 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2011
- U.S. locations
- 1
- States / cities
- Rochester, Minnesota
- Conditions
- Neurological Regression, Myoclonus, Cherry Red Spot, Brain Atrophy
- Interventions
- Not listed
- Lead sponsor
- National Human Genome Research Institute (NHGRI)
- NIH
- Eligibility
- 1 Day to 100 Years
- Enrollment
- 200 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Started 2002
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Metachromatic Leukodystrophy
- Interventions
- Not listed
- Lead sponsor
- National Organization for Rare Disorders
- Other
- Eligibility
- Not listed
- Enrollment
- 21 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2025
- U.S. locations
- 1
- States / cities
- Danbury, Connecticut
- Conditions
- Gout
- Interventions
- lesinurad, febuxostat
- Drug
- Lead sponsor
- Ardea Biosciences, Inc.
- Industry
- Eligibility
- 18 Years to 85 Years
- Enrollment
- 196 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2016
- U.S. locations
- 55
- States / cities
- Birmingham, Alabama • Tempe, Arizona • Glendale, California + 50 more
- Conditions
- Cystinosis
- Interventions
- Not listed
- Lead sponsor
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
- NIH
- Eligibility
- 7 Years and older
- Enrollment
- 11 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2003 – 2014
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Glycogen Storage Disease VI, GLYCOGEN STORAGE DISEASE IXa1, GLYCOGEN STORAGE DISEASE IXa2, Glycogen Storage Disease IXB, Glycogen Storage Disease IXC, GSD 9 (All Subtypes), GSD 6
- Interventions
- No intervention
- Other
- Lead sponsor
- Duke University
- Other
- Eligibility
- 0 Years to 90 Years
- Enrollment
- 400 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2030
- U.S. locations
- 1
- States / cities
- Durham, North Carolina
- Conditions
- MPS IIIB (Sanfilippo B Syndrome)
- Interventions
- Not listed
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Industry
- Eligibility
- 1 Year and older
- Enrollment
- 30 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2017
- U.S. locations
- 2
- States / cities
- Minneapolis, Minnesota • Pittsburgh, Pennsylvania
- Conditions
- Cancers
- Interventions
- Laparoscopic surgery
- Procedure
- Lead sponsor
- Erin Rowell
- Other
- Eligibility
- Up to 11 Years · Female only
- Enrollment
- 250 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2035
- U.S. locations
- 1
- States / cities
- Chicago, Illinois
- Conditions
- Hypophosphatasia (HPP)
- Interventions
- Not listed
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Industry
- Eligibility
- Not listed
- Enrollment
- 1,571 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2031
- U.S. locations
- 20
- States / cities
- Centennial, Colorado • Hartford, Connecticut • Tampa, Florida + 16 more
- Conditions
- Urea Cycle Disorder, Inborn Errors of Metabolism
- Interventions
- UCD Anamix Infant
- Other
- Lead sponsor
- Nutricia Research
- Industry
- Eligibility
- Up to 30 Days
- Enrollment
- 3 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2019
- U.S. locations
- 1
- States / cities
- Philadelphia, Pennsylvania
- Conditions
- Disaster, Vulnerable Populations
- Interventions
- Nurses Taking On Readiness Measures (N- TORM)
- Behavioral
- Lead sponsor
- Hunter College of City University of New York
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 400 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2023 – 2024
- U.S. locations
- 1
- States / cities
- New York, New York
- Conditions
- Phenylketonuria
- Interventions
- Not listed
- Lead sponsor
- Boston Children's Hospital
- Other
- Eligibility
- 12 Years to 25 Years
- Enrollment
- 10 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2017 – 2018
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- Conditions
- Familial Hypercholesterolemia
- Interventions
- Rosuvastatin, Placebo
- Drug
- Lead sponsor
- AstraZeneca
- Industry
- Eligibility
- 10 Years to 17 Years
- Enrollment
- 173 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2006 – 2008
- U.S. locations
- 4
- States / cities
- Los Angeles, California • Hyde Park, New York • Cincinnati, Ohio + 1 more