- 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
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 109
matching trials from the live ClinicalTrials.gov search.
- Conditions
- Healthy
- Interventions
- warfarin sodium, mipomersen sodium; warfarin sodium
- Drug
- Lead sponsor
- Kastle Therapeutics, LLC
- Industry
- Eligibility
- 18 Years to 45 Years
- Enrollment
- 18 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2010
- U.S. locations
- 1
- States / cities
- Austin, Texas
- Conditions
- Chronic Migraine
- Interventions
- IONIS-PKKRx (ISIS 546254), Placebo
- Drug · Other
- Lead sponsor
- Smith, Timothy R., M.D.
- Individual
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 30 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2019
- U.S. locations
- 2
- States / cities
- City of Saint Peters, Missouri • Springfield, Missouri
- Conditions
- Acromegaly
- Interventions
- GHR-LRX
- Drug
- Lead sponsor
- Ionis Pharmaceuticals, Inc.
- Industry
- Eligibility
- 18 Years to 75 Years
- Enrollment
- 34 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2023
- U.S. locations
- 7
- States / cities
- Los Angeles, California • Boston, Massachusetts • Las Vegas, Nevada + 4 more
- Conditions
- Chronic Heart Failure With Reduced Ejection Fraction
- Interventions
- IONIS-AGT-LRx, Placebo
- Drug
- Lead sponsor
- Ionis Pharmaceuticals, Inc.
- Industry
- Eligibility
- 18 Years to 80 Years
- Enrollment
- 72 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2023
- U.S. locations
- 10
- States / cities
- Little Rock, Arkansas • Crystal River, Florida • Hialeah, Florida + 6 more
- Conditions
- Healthy Participants
- Interventions
- Olezarsen
- Drug
- Lead sponsor
- Ionis Pharmaceuticals, Inc.
- Industry
- Eligibility
- 18 Years to 64 Years
- Enrollment
- 104 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2022 – 2023
- U.S. locations
- 1
- States / cities
- Cincinnati, Ohio
- Conditions
- Healthy Volunteer
- Interventions
- mipomersen, Placebo
- Drug
- Lead sponsor
- Kastle Therapeutics, LLC
- Industry
- Eligibility
- 18 Years to 75 Years
- Enrollment
- 20 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2011 – 2012
- U.S. locations
- 1
- States / cities
- New York, New York
- Conditions
- Severe Hypertriglyceridemia
- Interventions
- Olezarsen, Placebo
- Drug
- Lead sponsor
- Ionis Pharmaceuticals, Inc.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 617 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2025
- U.S. locations
- 56
- States / cities
- Birmingham, Alabama • Scottsdale, Arizona • Chula Vista, California + 51 more
- 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
- 21 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2007 – 2011
- U.S. locations
- 5
- States / cities
- Chicago, Illinois • Auburn, Maine • Biddeford, Maine + 2 more
- Conditions
- Hypertension
- Interventions
- ION904, Placebo
- Drug
- Lead sponsor
- Ionis Pharmaceuticals, Inc.
- Industry
- Eligibility
- 18 Years to 75 Years
- Enrollment
- 48 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2023
- U.S. locations
- 8
- States / cities
- Cypress, California • Los Angeles, California • Sacramento, California + 5 more
- Conditions
- Familial Chylomicronemia Syndrome, Lipoprotein Lipase Deficiency, Hyperlipoproteinemia Type 1
- Interventions
- Volanesorsen
- Drug
- Lead sponsor
- Akcea Therapeutics
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 68 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2020
- U.S. locations
- 8
- States / cities
- Huntington Beach, California • San Francisco, California • Boca Raton, Florida + 5 more
- Conditions
- Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)
- Interventions
- Eplontersen, Placebo
- Drug
- Lead sponsor
- Ionis Pharmaceuticals, Inc.
- Industry
- Eligibility
- 18 Years to 90 Years
- Enrollment
- 1,438 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2026
- U.S. locations
- 51
- States / cities
- Phoenix, Arizona • La Jolla, California • Los Angeles, California + 39 more
- Conditions
- Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM)
- Interventions
- Scintigraphy scan
- Diagnostic Test
- Lead sponsor
- Ionis Pharmaceuticals, Inc.
- Industry
- Eligibility
- 18 Years to 90 Years
- Enrollment
- 163 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2026
- U.S. locations
- 4
- States / cities
- New York, New York • Durham, North Carolina • Cleveland, Ohio + 1 more
- Conditions
- Amyotrophic Lateral Sclerosis
- Interventions
- ION363, Placebo
- Drug
- Lead sponsor
- Ionis Pharmaceuticals, Inc.
- Industry
- Eligibility
- 10 Years and older
- Enrollment
- 89 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2028
- U.S. locations
- 8
- States / cities
- La Jolla, California • Palo Alto, California • Baltimore, Maryland + 5 more
- Conditions
- Myotonic Dystrophy Type 1
- Interventions
- IONIS-DMPKRx, Placebo
- Drug
- Lead sponsor
- Ionis Pharmaceuticals, Inc.
- Industry
- Eligibility
- 20 Years to 55 Years
- Enrollment
- 48 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2016
- U.S. locations
- 8
- States / cities
- Stanford, California • Gainesville, Florida • Kansas City, Kansas + 5 more
- Conditions
- Castrate-Resistant Prostate Cancer
- Interventions
- ISIS EIF4E Rx, Prednisone, Docetaxel
- Drug
- Lead sponsor
- Ionis Pharmaceuticals, Inc.
- Industry
- Eligibility
- 18 Years and older · Male only
- Enrollment
- 113 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2013
- U.S. locations
- 11
- States / cities
- Fayetteville, Arkansas • San Bernardino, California • Fort Collins, Colorado + 8 more
- Conditions
- Hypercholesterolemia, Coronary Heart Disease
- Interventions
- Mipomersen sodium, Placebo
- Drug
- Lead sponsor
- Kastle Therapeutics, LLC
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 158 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2010
- U.S. locations
- 62
- States / cities
- Birmingham, Alabama • Huntsville, Alabama • Muscle Shoals, Alabama + 51 more
- Conditions
- Cytomegalovirus Retinitis, HIV Infections
- Interventions
- Fomivirsen sodium
- Drug
- Lead sponsor
- Ionis Pharmaceuticals, Inc.
- Industry
- Eligibility
- 18 Years and older
- Healthy volunteers
- Healthy volunteers not accepted
- U.S. locations
- 17
- States / cities
- Los Angeles, California • Pasadena, California • San Francisco, California + 11 more
- Conditions
- Obesity
- Interventions
- ISIS-FGFR4RX, Placebo
- Drug
- Lead sponsor
- Ionis Pharmaceuticals, Inc.
- Industry
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 13 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2016
- U.S. locations
- 1
- States / cities
- Orlando, Florida
- Conditions
- Healthy Participants
- Interventions
- Placebo, Pelacarsen
- Drug
- Lead sponsor
- Ionis Pharmaceuticals, Inc.
- Industry
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 29 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2018 – 2019
- U.S. locations
- 1
- States / cities
- Glendale, California
- Conditions
- Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM)
- Interventions
- Cardiac MRI
- Diagnostic Test
- Lead sponsor
- Ionis Pharmaceuticals, Inc.
- Industry
- Eligibility
- 18 Years to 90 Years
- Enrollment
- 160 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2026
- U.S. locations
- 2
- States / cities
- Boston, Massachusetts • Portland, Oregon
- Conditions
- Healthy Participants
- Interventions
- Eplontersen, Placebo
- Drug
- Lead sponsor
- Ionis Pharmaceuticals, Inc.
- Industry
- Eligibility
- 20 Years to 65 Years
- Enrollment
- 24 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2020
- U.S. locations
- 1
- States / cities
- Cypress, California
- Conditions
- Familial Partial Lipodystrophy
- Interventions
- AKCEA-ANGPTL3-LRx
- Drug
- Lead sponsor
- Akcea Therapeutics
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 4 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2019
- U.S. locations
- 1
- States / cities
- Ann Arbor, Michigan
- Conditions
- Hypertriglyceridemia, Cardiovascular Diseases, Atherosclerosis
- Interventions
- Olezarsen, Placebo
- Drug
- Lead sponsor
- Ionis Pharmaceuticals, Inc.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 1,478 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2025
- U.S. locations
- 68
- States / cities
- Birmingham, Alabama • Huntsville, Alabama • Beverly Hills, California + 54 more