- Conditions
- Gaucher Disease
- Interventions
- Not listed
- Lead sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
- NIH
- Eligibility
- Not listed
- Enrollment
- 50 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2007 – 2008
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
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 625–648
of 1,608
matching trials from the live ClinicalTrials.gov search.
- Conditions
- G6PD Deficiency
- Interventions
- SD Biosensor STANDARD G6PD Test, Pointe Scientific Test Kit, HemoCue System
- Diagnostic Test
- Lead sponsor
- PATH
- Other
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 229 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2019 – 2020
- U.S. locations
- 1
- States / cities
- Reading, Pennsylvania
- Conditions
- Mucopolysaccharidosis IVA, Morquio A Syndrome, MPS IVA
- Interventions
- BMN 110
- Drug
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- 7 Years and older
- Enrollment
- 25 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2014
- U.S. locations
- 4
- States / cities
- Oakland, California • Chicago, Illinois • New York, New York + 1 more
- Conditions
- Niemann-Pick Disease, Type C
- Interventions
- N-Acetyl Cysteine
- Drug
- Lead sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- NIH
- Eligibility
- 12 Months and older
- Enrollment
- 35 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2010
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Krabbe Disease
- Interventions
- FBX-101
- Biological
- Lead sponsor
- Forge Biologics, Inc
- Industry
- Eligibility
- Not listed
- Enrollment
- 2 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2025
- U.S. locations
- 1
- States / cities
- Ann Arbor, Michigan
- Conditions
- Phenylketonuria
- Interventions
- BMN 165
- Drug
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- 18 Years to 70 Years
- Enrollment
- 261 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2015
- U.S. locations
- 31
- States / cities
- La Jolla, California • Oakland, California • Aurora, Colorado + 28 more
- Conditions
- Gaucher Disease
- Interventions
- Not listed
- Lead sponsor
- Yale University
- Other
- Eligibility
- 3 Months to 75 Years
- Enrollment
- 30 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2030
- U.S. locations
- 1
- States / cities
- New Haven, Connecticut
- Conditions
- Fabry Disease
- Interventions
- Not listed
- Lead sponsor
- Shire
- Industry
- Eligibility
- Not listed
- Enrollment
- 4,000 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2001 – 2021
- U.S. locations
- 1
- States / cities
- Lexington, Massachusetts
- Conditions
- Familial Hypercholesterolemia
- Interventions
- Cascade screening
- Behavioral
- Lead sponsor
- Northwestern University
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 800 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2027
- U.S. locations
- 1
- States / cities
- Philadelphia, Pennsylvania
- Conditions
- Adrenal Insufficiency, Excess Androgen, Congenital Adrenal Hyperplasia (CAH)
- Interventions
- Hydrocortisone (Solucortef), Insulin pump (Medtronic)
- Drug · Device
- Lead sponsor
- National Institutes of Health Clinical Center (CC)
- NIH
- Eligibility
- 18 Years to 99 Years
- Enrollment
- 8 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2016
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Hypercholesterolemia
- Interventions
- Evolocumab, Low-density Lipoprotein Cholesterol (LDL-C) Apheresis
- Biological · Procedure
- Lead sponsor
- Amgen
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 39 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2017
- U.S. locations
- 4
- States / cities
- Boca Raton, Florida • Kansas City, Kansas • Grandville, Michigan + 1 more
- 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
- Hypertension
- Interventions
- 6R-BH4 (sapropterin dihydrochloride)
- Drug
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 84 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2006 – 2008
- U.S. locations
- 24
- States / cities
- Hoover, Alabama • Huntsville, Alabama • Mobile, Alabama + 21 more
- Conditions
- Hyperuricemia, Gout, Chronic Kidney Diseases
- Interventions
- ALLN-346, Placebo
- Drug
- Lead sponsor
- Allena Pharmaceuticals
- Industry
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 16 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2022
- U.S. locations
- 1
- States / cities
- Tempe, Arizona
- Conditions
- Mitochondrial Diseases, Drug Resistant Epilepsy, Leigh Disease, Leigh Syndrome, Mitochondrial Encephalopathy (MELAS), Pontocerebellar Hypoplasia Type 6 (PCH6), Alpers Disease, Alpers Syndrome
- Interventions
- Vatiquinone, Placebo
- Drug · Other
- Lead sponsor
- PTC Therapeutics
- Industry
- Eligibility
- Up to 20 Years
- Enrollment
- 68 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2023
- U.S. locations
- 12
- States / cities
- San Diego, California • Stanford, California • New Haven, Connecticut + 7 more
- Conditions
- Homozygous Familial Hypercholesterolemia (HoFH)
- Interventions
- Not listed
- Lead sponsor
- REGENXBIO Inc.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 4 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2020
- U.S. locations
- 1
- States / cities
- Boca Raton, Florida
- Conditions
- Fabry Disease
- Interventions
- migalastat HCl
- Drug
- Lead sponsor
- Amicus Therapeutics
- Industry
- Eligibility
- 18 Years to 65 Years · Female only
- Enrollment
- 9 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2006 – 2008
- U.S. locations
- 1
- States / cities
- Decatur, Georgia
- Conditions
- Sickle Cell Disease
- Interventions
- Liver transient elastography (Fibroscan), Ferriscan, Liver biopsy in iron overloaded subjects.
- Other · Procedure
- Lead sponsor
- University of Miami
- Other
- Eligibility
- 10 Years to 65 Years
- Enrollment
- 26 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2017
- U.S. locations
- 1
- States / cities
- Miami, Florida
- Conditions
- Elevated LDL Cholesterol
- Interventions
- Cardiovascular fixed dose combination pill (acetylsalicylic acid, simvastatin and ramipril),, Simvastatin
- Drug
- Lead sponsor
- Ferrer Internacional S.A.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 36 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2011
- U.S. locations
- 1
- States / cities
- New York, New York
- Conditions
- Niemann-Pick Disease, Type C
- Interventions
- Not listed
- Lead sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- NIH
- Eligibility
- 1 Day to 120 Years
- Enrollment
- 900 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- Started 2006
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Epilepsy; Seizure, Neuromuscular Diseases, Brain Malformation, Intellectual Disability, Autism Spectrum Disorder, Hypotonia, Inborn Errors of Metabolism, Movement Disorders, Genetic Disease, Development Delay, Chromosome Abnormality, Hearing Loss, Dysmorphic Features, Skeletal Dysplasia, Congenital Abnormality, Microcephaly, Macrocephaly
- Interventions
- Pre-visit prep, usual care + exome seq
- Behavioral · Diagnostic Test
- Lead sponsor
- University of North Carolina, Chapel Hill
- Other
- Eligibility
- 0 Years and older
- Enrollment
- 548 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2024
- U.S. locations
- 3
- States / cities
- Asheville, North Carolina • Chapel Hill, North Carolina • Greenville, North Carolina
- Conditions
- Gaucher Disease Type I, Oxidative Stress, Inflammation
- Interventions
- Not listed
- Lead sponsor
- University of Minnesota
- Other
- Eligibility
- 18 Years to 75 Years
- Enrollment
- 34 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2026
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota
- Conditions
- Niemann-Pick Disease, Type C
- Interventions
- Parts A/B: Adrabetadex, Parts A/B: Sham Control
- Drug · Other
- Lead sponsor
- Mandos LLC
- Industry
- Eligibility
- 4 Years to 21 Years
- Enrollment
- 56 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2018
- U.S. locations
- 14
- States / cities
- Birmingham, Alabama • Orange, California • San Francisco, California + 11 more
- Conditions
- Congenital Disorders of Glycosylation
- Interventions
- D-Galactose
- Dietary Supplement
- Lead sponsor
- Tulane University
- Other
- Eligibility
- Up to 21 Years
- Enrollment
- 8 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2018
- U.S. locations
- 1
- States / cities
- New Orleans, Louisiana