Pharmaceutical sponsor
Ionis Pharmaceuticals, Inc. Clinical Trials
Studies where Ionis Pharmaceuticals, Inc. is the lead sponsor: its pipeline by phase, what is enrolling now and where. Studies it supports as a collaborator are in the search.
In numbers
Open to volunteers: recruiting, not yet recruiting or enrolling by invitation. Active adds studies under way that have stopped recruiting.
Open to volunteers
All 10Angelman Syndrome
CHAMPION: A Study to Evaluate the Efficacy and Safety of Obudanersen (ION582) in Children and Adults With Angelman Syndrome (AS)
The primary purpose of the study is to evaluate efficacy of obudanersen in participants with AS due to uniparental disomy or imprinting defects (UPD/ID) as measured through expressive communication.
- Trial name
- NCT07782827
- Testing
- obudanersen
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Hypertriglyceridemia, Severe Hypertriglyceridemia
A Study to Assess the Safety, Tolerability and Efficacy of ION775
The main objective of this study is to evaluate the effect of ION775 on fasting triglyceride (TG) levels in participants with hypertriglyceridemia (HTG) and severe hypertriglyceridemia (sHTG).
- Trial name
- NCT07604974
- Testing
- ION775
- Where
- 17 sites in United States, Canada
- Volunteers
- 72 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome
ATTUNE: A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intrathecally-Administered ION440 in Participants With Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome (MDS)
The primary purpose of this study is to evaluate the safety and tolerability of ION440.
- Trial name
- NCT06430385
- Testing
- ION440
- Where
- 12 sites in United States, Austria and 3 more
- Volunteers
- 48 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Familial Chylomicronemia Syndrome
A Study of Olezarsen for the Treatment of Familial Chylomicronemia Syndrome (FCS) in Pediatric Participants
The primary purpose of the study is to evaluate the efficacy of olezarsen administered by subcutaneous injection to pediatric participants with FCS.
- Trial name
- NCT07727538
- Testing
- Olezarsen
- Where
- 5 sites in United States
- Volunteers
- 12 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Pelizaeus-Merzbacher Disease
Rocket Study: A Study to Characterize Biomarkers and Disease Progression in Participants With Pelizaeus-Merzbacher Disease
The purpose of the study is to prospectively assess longitudinal changes in proteolipid protein 1 (PLP1) protein, disease-related biomarkers in cerebral spinal fluid (CSF) and blood, neuroimaging parameters relevant to…
- Trial name
- NCT05659901
- Where
- 10 sites in United States, France and 5 more
- Volunteers
- 32 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Dravet Syndrome
ASCEND: Safety and Tolerability of ION337 for the Treatment of Dravet Syndrome
The primary purpose of this study is to evaluate the safety and tolerability of ION337 in participants with Dravet syndrome (DS).
- Trial name
- NCT07531745
- Testing
- ION337
- Where
- 6 sites in United States, Canada
- Volunteers
- 32 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Hereditary Angioedema (HAE)
Donidalorsen Treatment in Children With Hereditary Angioedema
The purpose of this study is to evaluate the safety, tolerability and pharmacokinetics of donidalorsen in pediatric participants with hereditary angioedema (HAE) Type I (HAE-1) or Type II (HAE-2).
- Trial name
- NCT07298447
- Testing
- Donidalorsen
- Where
- 16 sites in United States, Australia and 3 more
- Volunteers
- 20 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Multiple System Atrophy
Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of ION464 Administered to Adults With Multiple System Atrophy (HORIZON)
The primary objectives are to evaluate the safety and tolerability of multiple doses of ION464 administered via intrathecal (IT) injection (Part 1) and to evaluate the long-term safety and tolerability of ION464 (Part 2) in…
- Trial name
- HORIZON (NCT04165486)
- Testing
- ION464
- Where
- 15 sites in Austria, France and 3 more
- Volunteers
- 40 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.
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.
Completed, with results posted
All –Hereditary Angioedema
An Extension Study of Donidalorsen (IONIS-PKK-LRx) in Participants With Hereditary Angioedema
The purpose of this study was to evaluate the safety and efficacy of extended dosing of donidalorsen administered subcutaneously (SC), with alternative dosing and/or dose frequency with donidalorsen in participants with…
- Trial name
- NCT04307381
- Testing
- Donidalorsen
- Where
- 7 sites in United States, Netherlands
- Volunteers
- 20 people
- Sponsor
- 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.
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.
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.