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ClinicalTrials.gov public records Last synced Oct 6, 2026, 6:42 PM EDT

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Showing 25–48 of 109 matching trials from the live ClinicalTrials.gov search.
Conditions
Severe Hypertriglyceridemia
Interventions
Olezarsen
Drug
Lead sponsor
Ionis Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older
Enrollment
885 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
65
States / cities
Los Angeles, California • San Diego, California • Tarzana, California + 53 more
Conditions
Hypercholesterolemia, Familial
Interventions
ISIS 301012
Drug
Lead sponsor
Kastle Therapeutics, LLC
Industry
Eligibility
18 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2007
U.S. locations
5
States / cities
Chicago, Illinois • New York, New York • Winston-Salem, North Carolina + 2 more
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
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
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
Pelizaeus-Merzbacher Disease
Interventions
Not listed
Lead sponsor
Ionis Pharmaceuticals, Inc.
Industry
Eligibility
6 Months to 17 Years · Male only
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2029
U.S. locations
3
States / cities
Atlanta, Georgia • Baltimore, Maryland • Philadelphia, Pennsylvania
Conditions
Familial Chylomicronemia Syndrome
Interventions
Olezarsen
Drug
Lead sponsor
Ionis Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2028
U.S. locations
7
States / cities
Huntington Beach, California • Boca Raton, Florida • Park Ridge, Illinois + 3 more
Completed Phase 3 Interventional Results available

Long-Term Evaluation of BIIB067 (Tofersen)

NCT03070119
Conditions
ALS Caused by Superoxide Dismutase 1 (SOD1) Mutation
Interventions
Tofersen
Drug
Lead sponsor
Biogen
Industry
Eligibility
18 Years and older
Enrollment
139 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2024
U.S. locations
16
States / cities
Phoenix, Arizona • La Jolla, California • San Francisco, California + 13 more
Conditions
Familial Chylomicronemia Syndrome
Interventions
Olezarsen
Drug
Lead sponsor
Ionis Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older
U.S. locations
1
States / cities
Carlsbad, California
Conditions
Abnormalities, Cardiovascular
Interventions
Volanesorsen, Moxifloxacin, Placebo
Drug
Lead sponsor
Ionis Pharmaceuticals, Inc.
Industry
Eligibility
18 Years to 55 Years
Enrollment
52 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2016
U.S. locations
1
States / cities
Dallas, Texas
Conditions
Amyloidosis, Transthyretin Amyloid Cardiomyopathy
Interventions
99m-technetium pyrophosphate scintigraphy
Diagnostic Test
Lead sponsor
Oregon Health and Science University
Other
Eligibility
60 Years to 90 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2023
U.S. locations
1
States / cities
Portland, Oregon
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
Prion Disease
Interventions
ION717, Placebo
Drug
Lead sponsor
Ionis Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older
Enrollment
85 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2030
U.S. locations
3
States / cities
Boston, Massachusetts • New York, New York • Cleveland, Ohio
Conditions
NAFLD, Diabetes Mellitus, Type 2, Hypertriglyceridemia, Fatty Liver, Nonalcoholic
Interventions
Placebo, ISIS 703802 40 mg, ISIS 703802 80 mg, ISIS 703802 20 mg
Drug
Lead sponsor
Akcea Therapeutics
Industry
Eligibility
18 Years to 70 Years
Enrollment
105 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2020
U.S. locations
38
States / cities
Chandler, Arizona • Fountain Hills, Arizona • Glendale, Arizona + 30 more
Conditions
Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome
Interventions
ION440, Sham procedure
Drug · Procedure
Lead sponsor
Ionis Pharmaceuticals, Inc.
Industry
Eligibility
2 Years to 65 Years · Male only
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2030
U.S. locations
8
States / cities
San Diego, California • Aurora, Colorado • Baltimore, Maryland + 5 more
Conditions
Lafora Disease
Interventions
Not listed
Lead sponsor
Ionis Pharmaceuticals, Inc.
Industry
Eligibility
5 Years and older
Enrollment
33 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
2
States / cities
Los Angeles, California • Dallas, Texas
Completed No phase listed Observational Accepts healthy volunteers

Early Detection of Neuropathy in ATTRv

NCT05311488
Conditions
Hereditary Amyloidosis, Transthyretin-Related
Interventions
neurofilament light chain, In-vivo Meissner Corpuscle imaging, Nerve conduction study
Diagnostic Test
Lead sponsor
University of Pennsylvania
Other
Eligibility
30 Years to 90 Years
Enrollment
47 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2026
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Conditions
Hypertriglyceridemia
Interventions
ISIS apoC-III Rx, Placebo
Drug
Lead sponsor
Ionis Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older
Enrollment
89 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2014
U.S. locations
6
States / cities
Farmville, North Carolina • Greenville, North Carolina • Kinston, North Carolina + 1 more
Conditions
Crohn's Disease
Interventions
Alicaforsen
Drug
Lead sponsor
Ionis Pharmaceuticals, Inc.
Industry
Eligibility
12 Years and older
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2002
U.S. locations
27
States / cities
Little Rock, Arkansas • Orange, California • San Diego, California + 24 more
Conditions
Type 2 Diabetes Mellitus
Interventions
ISIS 113715
Drug
Lead sponsor
Ionis Pharmaceuticals, Inc.
Industry
Eligibility
18 Years to 70 Years
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2007
U.S. locations
1
States / cities
New Haven, Connecticut
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
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
Hereditary Angioedema
Interventions
Donidalorsen
Drug
Lead sponsor
Ionis Pharmaceuticals, Inc.
Industry
Eligibility
12 Years and older
Enrollment
154 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
16
States / cities
Paradise Valley, Arizona • La Jolla, California • Santa Monica, California + 13 more
Conditions
Hereditary Transthyretin-Mediated Amyloid Polyneuropathy
Interventions
Eplontersen
Drug
Lead sponsor
Ionis Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older
Enrollment
151 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2029
U.S. locations
8
States / cities
Scottsdale, Arizona • Indianapolis, Indiana • Baltimore, Maryland + 5 more