Clinical trials: sponsored by Ionis Pharmaceuticals, Inc.
Hypertriglyceridemia, Atherosclerotic Cardiovascular Disease
A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) in Adults With Hypertriglyceridemia and Atherosclerotic Cardiovascular Disease (Established or at Increased Risk for), and/or With Severe Hypertriglyceridemia
The purpose of the study was to evaluate the effect of olezarsen on percent change in fasting triglyceride (TG) levels compared to placebo at Months 6 and 12 and the proportion of participants who achieve different thresholds in…
- Conditions
- Hypertriglyceridemia, Atherosclerotic Cardiovascular Disease, Severe Hypertriglyceridemia
- Trial name
- NCT05355402
- Testing
- Olezarsen
- Where
- 24 sites in United States, Canada
- Volunteers
- 154 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)
An Extension Study to Assess Long-Term Safety of Eplontersen in Adults With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM)
The purpose of this study is to evaluate the safety and tolerability of extended dosing with eplontersen in participants with ATTR-CM.
- Trial name
- NCT05667493
- Testing
- Eplontersen
- Where
- 134 sites in United States, Argentina and 18 more
- Volunteers
- 1,400 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome
Observational Study to Characterize Biomarkers and Disease Progression in Participants With Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome
The purpose of the study is to prospectively assess longitudinal changes in biomarkers (MECP2, potential biomarkers of target engagement and disease activity) in cerebrospinal fluid (CSF) and blood; characterize longitudinal…
- Trial name
- NCT06014541
- Where
- 5 sites in United States
- Volunteers
- 29 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Alzheimer Disease, Down Syndrome
Hero: A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics (PK) and Pharmacodynamics (PD) of ION269 in Participants With Down Syndrome (DS) at Risk for Alzheimer's Disease (AD)
The primary purpose of this study is to evaluate the safety and tolerability of ION269 in adults with Down syndrome with evidence of brain amyloid positivity.
- Trial name
- NCT06673069
- Testing
- ION269
- Where
- 6 sites in United States, Spain
- Volunteers
- 1 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Severe Hypertriglyceridemia
A Study of Olezarsen (ISIS 678354) Administered to Participants With Severe Hypertriglyceridemia
The purpose of the study is to evaluate the efficacy of olezarsen as compared to placebo on the percent change in fasting triglycerides (TG) from baseline.
- Trial name
- NCT05079919
- Testing
- Olezarsen
- Where
- 209 sites in United States, Australia and 21 more
- Volunteers
- 617 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Hypertriglyceridemia, Cardiovascular Diseases
A Study of Olezarsen (ISIS 678354) in Participants With Hypertriglyceridemia and Atherosclerotic Cardiovascular Disease, or With Severe Hypertriglyceridemia
The purpose of the study is to evaluate the effect of olezarsen on percent change in fasting triglyceride (TG) levels compared to placebo in participants with hypertriglyceridemia and atherosclerotic cardiovascular disease, or…
- Conditions
- Hypertriglyceridemia, Cardiovascular Diseases, Atherosclerosis
- Trial name
- NCT05610280
- Testing
- Olezarsen
- Where
- 168 sites in United States, Bulgaria and 12 more
- Volunteers
- 1,478 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Familial Chylomicronemia Syndrome
A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRX) Administered to Adults With Familial Chylomicronemia Syndrome (FCS) Previously Treated With Volanesorsen
The purpose of the study is to evaluate the safety, tolerability, pharmacokinetic (PK) and pharmacodynamic (PD) effects of olezarsen (formerly known as AKCEA -APOCIII-LRX) in participants with FCS previously treated with…
- Trial name
- NCT05185843
- Testing
- Olezarsen
- Where
- 11 sites in United States, Canada and 1 more
- Volunteers
- 24 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Hereditary Angioedema
A Study to Assess the Long-Term Safety and Efficacy of Donidalorsen in the Prophylactic Treatment of Hereditary Angioedema (HAE)
The purpose of this study is to evaluate the long-term safety and efficacy of donidalorsen in people with HAE and the effects of donidalorsen on the number of HAE attacks and their impact on quality of life (QoL).
- Trial name
- NCT05392114
- Testing
- Donidalorsen
- Where
- 50 sites in United States, Belgium and 12 more
- Volunteers
- 154 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Familial Chylomicronemia Syndrome
A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) in Participants With Familial Chylomicronemia Syndrome (FCS)
The purpose of this study is to evaluate the effect of olezarsen (formerly known as AKCEA-APOCIII-LRx) on the percent change in fasting triglycerides (TG) from baseline.
- Trial name
- NCT05130450
- Testing
- Olezarsen
- Where
- 27 sites in United States, Canada and 9 more
- Volunteers
- 60 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Pelizaeus-Merzbacher Disease
Orbit Study: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356 in Participants With Pelizaeus Merzbacher Disease (PMD)
The primary purpose of this study is to evaluate the safety and tolerability of ION356.
- Trial name
- NCT06150716
- Testing
- ION356
- Where
- 7 sites in United States, France and 3 more
- Volunteers
- 24 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Hereditary Transthyretin-Mediated Amyloid Polyneuropathy
A Study to Assess the Long-Term Safety and Efficacy of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in Patients With Hereditary Transthyretin-Mediated Amyloid Polyneuropathy
The purpose of this study is to evaluate the safety and tolerability of extended dosing with eplontersen in participants with hereditary transthyretin-mediated amyloid polyneuropathy (hATTR-PN).
- Trial name
- NCT05071300
- Testing
- Eplontersen
- Where
- 33 sites in United States, Argentina and 11 more
- Volunteers
- 151 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Severe Hypertriglyceridemia
CORE-OLE: A Study of Olezarsen (ISIS 678354) Administered Subcutaneously to Participants With Severe Hypertriglyceridemia (SHTG)
The purpose of this study is to evaluate the safety and tolerability of olezarsen in participants with SHTG.
- Trial name
- NCT05681351
- Testing
- Olezarsen
- Where
- 262 sites in United States, Argentina and 31 more
- Volunteers
- 885 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Severe Hypertriglyceridemia
A Study of Olezarsen Administered Subcutaneously to Participants With Severe Hypertriglyceridemia
The purpose of this study is to evaluate the efficacy of olezarsen as compared to placebo on the percent change in fasting triglycerides (TG) from baseline.
- Trial name
- NCT05552326
- Testing
- Olezarsen
- Where
- 205 sites in United States, Argentina and 21 more
- Volunteers
- 446 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Parkinson's Disease
A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of BIIB094 in Adults With Parkinson's Disease
The primary objective of this study is to evaluate the safety and tolerability of single and multiple doses of BIIB094 administered via intrathecal (IT) injection to participants with Parkinson's Disease (PD).
- Trial name
- REASON (NCT03976349)
- Testing
- BIIB094
- Where
- 18 sites in United States, Canada and 4 more
- Volunteers
- 62 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Mild Alzheimer's Disease
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of IONIS-MAPTRx in Patients With Mild Alzheimer's Disease
The purpose of this study was to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of IONIS-MAPTRx in patients with Mild Alzheimer's Disease.
- Trial name
- NCT03186989
- Testing
- IONIS MAPTRx
- Where
- 13 sites in Canada, Finland and 4 more
- Volunteers
- 46 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Macular Degeneration, Geographic Atrophy
GOLDEN STUDY: A Study to Assess Safety and Efficacy of Multiple Doses of IONIS-FB-LRx in Participants With Geographic Atrophy Secondary to Age-Related Macular Degeneration (AMD)
Evaluate the effect of IONIS-FB-LRx on the rate of change of the area of geographic atrophy (GA) secondary to age-related macular degeneration (AMD) measured by fundus autofluorescence (FAF).
- Trial name
- NCT03815825
- Testing
- IONIS-FB-LRx
- Where
- 88 sites in United States, Australia and 7 more
- Volunteers
- 332 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Familial Chylomicronemia Syndrome
A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) Administered to Patients With Familial Chylomicronemia Syndrome (FCS)
The purpose of the study was to evaluate the efficacy of olezarsen as compared to placebo on the percent change in fasting triglycerides (TG) from baseline.
- Trial name
- BALANCE (NCT04568434)
- Testing
- Olezarsen
- Where
- 47 sites in United States, Canada and 9 more
- Volunteers
- 66 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Hereditary Angioedema
OASIS-HAE: A Study to Evaluate the Safety and Efficacy of Donidalorsen (ISIS 721744 or IONIS-PKK-LRx) in Participants With Hereditary Angioedema (HAE)
The purpose of this study was to evaluate the safety and efficacy of donidalorsen in participants with HAE and effect of donidalorsen on the quality and pattern of HAE attacks and their impact on quality of life (QoL).
- Trial name
- NCT05139810
- Testing
- Donidalorsen
- Where
- 51 sites in United States, Belgium and 12 more
- Volunteers
- 91 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Beta Thalassemia Intermedia
Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Sapablursen (Formerly ISIS 702843, IONIS-TMPRSS6-LRx)
The purpose was to evaluate the efficacy, safety, tolerability, pharmacokinetics and pharmacodynamics of sapablursen administered subcutaneously to participants with non-transfusion dependent β-Thalassemia Intermedia.
- Trial name
- NCT04059406
- Testing
- sapablursen
- Where
- 19 sites in Australia, Greece and 3 more
- Volunteers
- 29 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Hypertension
A Study to Assess the Safety, Tolerability and Efficacy of IONIS-AGT-LRx in Hypertensive Participants With Uncontrolled Blood Pressure
The purpose of this study was to evaluate the effect of IONIS-AGT-LRx compared to placebo on seated automated office systolic blood pressure (SBP) from baseline to Study Day 85 in uncontrolled hypertensive participants on ≥ 3…
- Trial name
- ASTRAAS (NCT04714320)
- Testing
- IONIS-AGT-LRx
- Where
- 50 sites in United States, Canada
- Volunteers
- 160 people
- Sponsor
- Ionis Pharmaceuticals, Inc.