Clinical trials: sponsored by Ionis Pharmaceuticals, Inc.
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.
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.
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.
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.
Hereditary Transthyretin-Mediated Amyloid Polyneuropathy
NEURO-TTRansform: A Study to Evaluate the Efficacy and Safety of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in Participants With Hereditary Transthyretin-Mediated Amyloid Polyneuropathy
The main objective of this study was to evaluate the efficacy of eplontersen as compared with the historical control of the placebo cohort in the NEURO-TTR trial (NCT01737398/2012-001831-30), in subjects with hereditary…
- Trial name
- NCT04136184
- Testing
- Inotersen, Eplontersen
- Where
- 45 sites in United States, Argentina and 14 more
- Volunteers
- 168 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Acromegaly
A Study to Assess the Safety, Tolerability, and Efficacy of IONIS-GHR-LRx Administered in Patients With Acromegaly
The purpose of this study was to determine the safety, tolerability, and efficacy of IONIS-GHR-LRx subcutaneous (SC) injection as monotherapy in patients with acromegaly.
- Trial name
- NCT04522180
- Testing
- GHR-LRX
- Where
- 22 sites in United States, Estonia and 8 more
- Volunteers
- 34 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
FAP, Familial Amyloid Polyneuropathy
Extension Study Assessing Long Term Safety and Efficacy of IONIS-TTR Rx in Familial Amyloid Polyneuropathy (FAP)
This study evaluates the safety and tolerability of extended dosing with IONIS-TTR Rx in patients with Familial Amyloid Polyneuropathy.
- Conditions
- FAP, Familial Amyloid Polyneuropathy, TTR, Transthyretin
- Trial name
- NCT02175004
- Testing
- Inotersen
- Where
- 22 sites in United States, Argentina and 7 more
- Volunteers
- 135 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Hereditary Angioedema
A Study to Assess the Clinical Efficacy of Donidalorsen (Also Known as IONIS-PKK-LRx and ISIS 721744) in Participants With Hereditary Angioedema
The purpose of this study was to evaluate the clinical efficacy, safety, and tolerability of donidalorsen in participants with hereditary angioedema (HAE) type 1 (HAE-1), HAE type 2 (HAE-2), or HAE with normal C1-inhibitor…
- Trial name
- NCT04030598
- Testing
- Donidalorsen
- Where
- 8 sites in United States, Netherlands
- Volunteers
- 23 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
End-stage Renal Disease (ESRD)
A Study of ISIS 416858 Administered Subcutaneously to Participants With End-Stage Renal Disease (ESRD) on Hemodialysis
Evaluation of safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of ISIS 416858 for up to 204 participants with ESRD receiving chronic hemodialysis as assessed by FXI activity reduction.
- Trial name
- EMERALD (NCT03358030)
- Testing
- ISIS 416858
- Where
- 2 sites in Spain
- Volunteers
- 213 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Hypertension
A Study to Assess the Safety, Tolerability and Efficacy of IONIS-AGT-LRx
This study evaluated the effect of ISIS 757456 (IONIS-AGT-LRx) on plasma angiotensinogen (AGT) and systolic blood pressure (SBP) in uncontrolled hypertensive participants who were on two to three antihypertensive medications.
- Trial name
- NCT04083222
- Testing
- ISIS 757456
- Where
- 9 sites in United States
- Volunteers
- 26 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Mild Hypertension
A Study to Assess the Safety, Tolerability and Efficacy of IONIS-AGT-LRx, an Antisense Inhibitor Administered Subcutaneously to Hypertensive Participants With Controlled Blood Pressure
This was a Phase 2, double-blind, randomized, placebo-controlled study of IONIS-AGT-LRx conducted in mild hypertensive participants.
- Trial name
- NCT03714776
- Testing
- ISIS 757456
- Where
- 6 sites in United States
- Volunteers
- 25 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Chronic Bronchitis, Chronic Obstructive Pulmonary Disease
A Study to Assess the Safety, Tolerability, and Efficacy of ION-827359 in Participants With Mild to Moderate Chronic Obstructive Pulmonary Disease (COPD) With Chronic Bronchitis (CB)
The purpose of this study was to evaluate the effect of ION-827359 on forced expiratory volume in 1 second (FEV1) in participants with mild to moderate COPD with CB.
- Trial name
- NCT04441788
- Testing
- ION-827359
- Where
- 15 sites in Czechia, Germany and 2 more
- Volunteers
- 60 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Healthy Volunteers, hATTR Amyloidosis
Evaluate the Safety and Tolerability, as Well as the Pharmacokinetic and Pharmacodynamic Profiles of Single and Multiple Doses of Eplontersen Administered Subcutaneously to Healthy Volunteers and Patients With Hereditary Transthyretin-Mediated Amyloidosis (hATTR ).
To evaluate the safety and tolerability, as well as the pharmacokinetic and pharmacodynamic profiles of single and multiple doses of Eplontersen administered subcutaneously to healthy volunteers and patients with Hereditary…
- Trial name
- NCT03728634
- Testing
- ION-682884, Vitamin A
- Where
- Canada
- Volunteers
- 47 people, healthy volunteers welcome
- Sponsor
- Ionis Pharmaceuticals, Inc.
Acromegaly
Safety, Tolerability, and Efficacy of IONIS-GHR-LRx in Participants With Acromegaly Being Treated With Long-acting Somatostatin Receptor Ligands
The purpose of this study was to assess the safety, tolerability, and efficacy of IONIS-GHR-LRx in up to 60 participants with acromegaly.
- Trial name
- NCT03548415
- Testing
- IONIS-GHR-LRx
- Where
- 33 sites in United States, Hungary and 5 more
- Volunteers
- 43 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Hypertriglyceridemia
The COMPASS Study: A Study of Volanesorsen (Formally ISIS-APOCIIIRx) in Patients With Hypertriglyceridemia
The purpose of this study is to evaluate the efficacy and safety of volanesorsen given for 26 weeks in participants with Hypertriglyceridemia.
- Trial name
- NCT02300233
- Testing
- Volanesorsen
- Where
- 45 sites in United States, Canada and 4 more
- Volunteers
- 114 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Familial Chylomicronemia Syndrome
The APPROACH Study: A Study of Volanesorsen (Formerly IONIS-APOCIIIRx) in Patients With Familial Chylomicronemia Syndrome
The purpose of this study is to evaluate the efficacy and safety of volanesorsen given for 52 weeks in participants with Familial Chylomicronemia Syndrome
- Trial name
- NCT02211209
- Testing
- Volanesorsen
- Where
- 40 sites in United States, Brazil and 10 more
- Volunteers
- 67 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Hepatic Steatosis
Safety, Tolerability, and Pharmacodynamics of IONIS-DGAT2Rx in Adult Patients With Type 2 Diabetes
The purpose is to assess the Safety, Tolerability, and Pharmacodynamics effect of IONIS DGAT2Rx in up to 45 Adult Patients with Type 2 Diabetes.
- Trial name
- NCT03334214
- Testing
- IONIS DGAT2Rx
- Where
- 22 sites in Canada, Hungary and 2 more
- Volunteers
- 44 people
- Sponsor
- Ionis Pharmaceuticals, Inc.