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.
Hereditary Amyloidosis, Transthyretin-Related
Early Detection of Neuropathy in ATTRv
The purpose of the study is to evaluate and compare different tools that are used to detect evidence of peripheral neuropathy in patients with TTRv.
- Trial name
- EDONA (NCT05311488)
- Testing
- neurofilament light chain, In-vivo Meissner Corpuscle imaging, Nerve conduction study
- Where
- United States
- Volunteers
- 47 people, healthy volunteers welcome
- Sponsor
- University of Pennsylvania
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.
Alexander Disease
Evaluation of Outcome Metrics in Alexander Disease
The purpose of this study is to define the natural history of Alexander Disease, a leukodystrophy that causes neurological dysfunction.
- Trial name
- AxD Outcomes (NCT02714764)
- Where
- United States
- Volunteers
- 200 people
- Sponsor
- Children's Hospital of Philadelphia
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.
Spinocerebellar Ataxia Type 2, Spinocerebellar Ataxia Type 7
Integrated Functional Evaluation of the Cerebellum
One of the main objectives of this project is to validate potential biological, clinical and/or imaging biomarkers in SCA patients through a multimodal assessment, for future ASOs trials.
- Trial name
- CERMOI (NCT04288128)
- Testing
- Lumbar puncture, Magnetic Resonance Imaging (MRI)
- Where
- France
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Institut National de la Santé Et de la Recherche Médicale, France
Leukodystrophy, White Matter Disease
The Myelin Disorders Biorepository Project
The Myelin Disorders Biorepository Project (MDBP) seeks to collect and analyze clinical data and biological samples from leukodystrophy patients worldwide to support ongoing and future research projects.
- Conditions
- Leukodystrophy, White Matter Disease, Leukoencephalopathies, 4H Syndrome
- Trial name
- MDBP (NCT03047369)
- Where
- 23 sites in United States
- Volunteers
- 12,000 people
- Sponsor
- Children's Hospital of Philadelphia
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.
ALS Caused by Superoxide Dismutase 1 (SOD1) Mutation
Long-Term Evaluation of BIIB067 (Tofersen)
The primary objective of the study is to evaluate the long-term safety and tolerability of BIIB067 (tofersen) in participants with amyotrophic lateral sclerosis (ALS) and confirmed superoxide dismutase 1 (SOD1) mutation.
- Trial name
- NCT03070119
- Testing
- Tofersen
- Where
- 30 sites in United States, Belgium and 7 more
- Volunteers
- 139 people
- Sponsor
- Biogen
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.