RNA
Total Trials
154
As Lead Sponsor
114
As Collaborator
40
Total Enrollment
28,019
NCT00005594
ISIS 2503 in Treating Patients With Advanced Cancer of the Pancreas
Phase: Phase 2
Role: Collaborator
Start: Jul 31, 2000
Completion: Not specified
NCT00017407
Carboplatin and Paclitaxel With or Without ISIS 3521 in Treating Patients With Non-Small Cell Lung Cancer
Phase: Phase 3
Role: Lead Sponsor
Start: Oct 13, 2000
Completion: Sep 9, 2002
NCT00048113
Alicaforsen (ISIS 2302) in Patients With Active Crohn's Disease
Start: Jan 10, 2001
Completion: Apr 27, 2002
NCT00035945
ISIS 14803-CS2, Treatment With ISIS 14803, Administered IV in Patients With Chronic Hepatitis C Virus Infections
Start: Oct 1, 2001
Completion: May 1, 2004
NCT00048321
ISIS 104838, an Inhibitor of Tumor Necrosis Factor, for Active Rheumatoid Arthritis
Start: Jan 1, 2002
Completion: Feb 1, 2003
NCT00048295
Start: May 31, 2002
NCT00063414
ISIS 2302-CS22, A 6-Week, Active-Controlled Clinical Study to Evaluate the Effectiveness of Alicaforsen (ISIS 2302) in Patients With Mild to Moderate Active Ulcerative Colitis
Start: Nov 20, 2002
Completion: Mar 24, 2005
NCT00330330
Safety, Tolerability, and Activity of ISIS 113715 in People With Type 2 Diabetes Mellitus Who Have Not Received Prior Treatment
Start: Feb 28, 2003
Completion: Aug 31, 2006
NCT00063830
ISIS 2302-CS27, A 6-Week, Placebo-Controlled Clinical Study to Evaluate the Effectiveness of Alicaforsen (ISIS 2302) in Patients With Mild to Moderate Active Ulcerative Colitis.
Start: Apr 3, 2003
Completion: Oct 11, 2004
NCT00062816
Addition of ISIS 14803 to Therapy With Peginterferon and Ribavirin for Chronic Hepatitis C (HCV) Patients Not Responding Adequately to the Two Drugs
Phase: Phase 1/2
Start: Jun 30, 2003
Completion: Jan 31, 2004
NCT00129194
Study of KP-1461 for the Treatment of HIV Positive Patients Who Have Failed Multiple HAART Regimens
Phase: Phase 1
Start: Aug 31, 2005
NCT00216463
Safety and Tolerability of Varying Load and Dose of ISIS 301012 in People With Elevated LDL-cholesterol Levels
Completion: Aug 31, 2007
NCT00231569
Dose-escalating Safety Study in Subjects on Stable Statin Therapy
Start: Sep 30, 2005
Completion: Dec 31, 2007
NCT00330200
Effects of ISIS 113715 on Insulin Sensitivity, Glucose, and Lipid Metabolism and Energy Expenditure in Type 2 Diabetics
Start: Nov 1, 2005
Completion: Mar 1, 2007
NCT00280995
Dose-escalating Safety Study of ISIS 301012 in Homozygous Familial Hypercholesterolemia Subjects on Lipid Lowering Therapy
Start: Jan 31, 2006
NCT00281008
Study of ISIS 301012 (Mipomersen) in Heterozygous Familial Hypercholesterolemia Subjects on Lipid Lowering Therapy
Start: Feb 28, 2006
NCT00327626
Safety, Tolerability, and Activity of ISIS 113715 in People With Type 2 Diabetes Mellitus Being Treated With Sulfonylurea
Start: May 1, 2006
NCT00362180
Measure Liver Fat Content After ISIS 301012 (Mipomersen) Administration
Start: Jul 31, 2006
Completion: Sep 30, 2010
NCT00365781
Examination of Dermatologic Effects From Subcutaneous Injections of ISIS 113715
Start: Aug 31, 2006
Completion: Sep 30, 2006
NCT00455598
Placebo Controlled, Dose Escalation Study in Subjects With Type 2 Diabetes Mellitus Being Treated With Sulfonylurea
Start: Feb 28, 2007
Completion: May 31, 2009
NCT00477594
Open Label Extension of ISIS 301012 (Mipomersen) to Treat Familial Hypercholesterolemia
Start: May 31, 2007
Completion: Jul 31, 2011
NCT00504452
Safety and Efficacy Study of KP-1461 to Treat ART-Experienced HIV+ Patients
Start: Jul 31, 2007
Completion: Jun 30, 2009
NCT00607373
Study to Assess the Safety and Efficacy of ISIS 301012 (Mipomersen) in Homozygous Familial Hypercholesterolemia
Completion: Mar 31, 2009
NCT00519727
Safety Study of ISIS 325568 in Healthy Volunteers
Start: Aug 31, 2007
Completion: Jun 30, 2008
NCT00694109
An Open-label Extension Study to Assess the Long-term Safety and Efficacy of ISIS 301012 (Mipomersen) in Patients With Familial Hypercholesterolemia or Severe-Hypercholesterolemia
Start: Apr 30, 2008
Completion: Sep 30, 2014
NCT00734240
Safety and Tolerability of Single and Multiple Doses of ISIS 353512 in Healthy Volunteers
Start: Jul 31, 2008
Completion: Mar 31, 2010
NCT00706849
Efficacy and Safety Study of ISIS 301012 (Mipomersen) as Add-on in Familial Hypercholesterolemic Patients With Coronary Artery Disease
Completion: May 31, 2010
NCT00707746
Safety and Efficacy Study of ISIS 301012 (Mipomersen) Administration in High Risk Statin Intolerant Subjects
Start: Oct 31, 2008
Completion: Jan 31, 2011
NCT00770146
Safety and Efficacy of Mipomersen (ISIS 301012) As Add-on Therapy in High Risk Hypercholesterolemic Patients
Start: Nov 30, 2008
Completion: Oct 31, 2010
NCT00836225
Safety, Tolerability and Activity Study of Multiple Doses of ISIS-SGLT2Rx in Healthy Volunteers
Start: Jan 31, 2009
Completion: Jan 31, 2012
NCT00794664
Safety and Efficacy of Mipomersen in Patients With Severe Hypercholesterolemia on a Maximally Tolerated Lipid-Lowering Regimen and Who Are Not on Apheresis
NCT01041222
Safety, Tolerability, and Activity Study of ISIS SOD1Rx to Treat Familial Amyotrophic Lateral Sclerosis (ALS) Caused by SOD1 Gene Mutations
Start: Jan 31, 2010
NCT01061814
A Study to Evaluate 3 Different Dosing Regimens of Mipomersen Administered Via Subcutaneous Injections to Healthy Volunteers
Completion: Jun 30, 2010
NCT01090661
A Study to Evaluate the Effect of Mipomersen on Cardiac Repolarization Conducted in Healthy Subjects
Start: Mar 31, 2010
NCT01133366
A Study to Determine the Effects of Multiple Doses of Mipomersen (200 mg SC) on the Pharmacodynamics and Pharmacokinetics of Single-dose Warfarin
Start: May 31, 2010
Completion: Jul 31, 2010
NCT01234025
Safety and Tolerability Study of ISIS EIF4E Rx in Combination With Docetaxel and Prednisone (CRPC)
Start: Nov 30, 2010
Completion: Dec 31, 2013
NCT01234038
Safety and Tolerability Study of ISIS EIF4E Rx in Combination With Carboplatin and Paclitaxel
Completion: Jan 31, 2013
NCT01299298
A Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of Single Doses of Mipomersen in Japanese Healthy Volunteers
Start: Jan 31, 2011
Completion: Apr 30, 2011
NCT01414101
Safety, Tolerability, and Pharmacodynamic Study of ISIS CRP Rx in Rheumatoid Arthritis
Start: Aug 31, 2011
Completion: May 31, 2013
NCT01414881
Study to Assess the Effects of Mipomersen on Lipid and Lipoprotein Metabolism in Healthy Subjects
Start: Sep 30, 2011
Completion: Nov 30, 2012
NCT01563302
Phase 1/2, Open-label, Dose-escalation Study of IONIS-STAT3Rx, Administered to Patients With Advanced Cancers
Start: Feb 27, 2012
Completion: Mar 23, 2016
NCT01529424
Safety, Tolerability, and Pharmacokinetic Study of ISIS ApoC-III Rx in Hypertriglyceridemia
Start: Feb 29, 2012
Completion: Jan 31, 2014
NCT01647308
Mechanistic Study of ISIS ISIS-APOCIIIRX in Type 2 Diabetes
Start: Jul 31, 2012
Completion: Mar 31, 2014
NCT01710852
Antiarrhythmic and Symptomatic Effect of ISIS CRP Rx Targeting CRP in Paroxysmal Atrial Fibrillation
Start: Oct 31, 2012
Completion: Apr 30, 2014
NCT01713361
Active Comparator-Controlled Study to Assess Safety and Efficacy of ISIS-FXIRx in Total Knee Arthroplasty
Completion: Aug 31, 2014
NCT01737398
Efficacy and Safety of Inotersen in Familial Amyloid Polyneuropathy
Phase: Phase 2/3
Start: Mar 15, 2013
Completion: Nov 7, 2017
NCT01839604
A Phase I/Ib Study of AZD9150 (ISIS-STAT3Rx) in Patients With Advanced/Metastatic Hepatocellular Carcinoma
Start: May 31, 2013
Completion: Feb 28, 2015
NCT01885260
Safety, Tolerability and Efficacy of ISIS-GCGRRx in Type 2 Diabetes
Start: Jul 31, 2013
Completion: Nov 30, 2014
NCT01918865
Safety, Tolerability, and Efficacy of ISIS-PTP1BRx in Type 2 Diabetes
Start: Aug 31, 2013
NCT01968265
Safety, Tolerability and Efficacy of ISIS-GCCRRx in Type 2 Diabetes
Start: Oct 31, 2013
Completion: May 31, 2015
NCT02175004
Extension Study Assessing Long Term Safety and Efficacy of IONIS-TTR Rx in Familial Amyloid Polyneuropathy (FAP)
Start: Jun 26, 2014
Completion: Jan 7, 2021
NCT02160899
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ISIS-APO(a)Rx in Participants With High Lipoprotein(a)
Start: Jun 30, 2014
Completion: Nov 30, 2015
NCT02211209
The APPROACH Study: A Study of Volanesorsen (Formerly IONIS-APOCIIIRx) in Patients With Familial Chylomicronemia Syndrome
Start: Dec 31, 2014
Completion: Mar 28, 2017
NCT02312011
A Safety andTolerability Study of Multiple Doses of ISIS-DMPKRx in Adults With Myotonic Dystrophy Type 1
Completion: Aug 31, 2016
NCT02300233
The COMPASS Study: A Study of Volanesorsen (Formally ISIS-APOCIIIRx) in Patients With Hypertriglyceridemia
Start: Feb 5, 2015
Completion: Jan 24, 2017
NCT02414594
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of IONIS APO(a)-LRx in Healthy Volunteers With Elevated Lipoprotein(a)
Start: Apr 30, 2015
Completion: Feb 29, 2016
NCT02476019
A Safety, Tolerability, PK, and PD Study of Once Weekly ISIS-FGFR4RX SC in Obese Patients
Start: Jun 30, 2015
Completion: Sep 30, 2016
NCT02519036
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ISIS 443139 in Participants With Early Manifest Huntington's Disease
Start: Aug 6, 2015
Completion: Nov 8, 2017
NCT02583919
Safety, Tolerability and Efficacy of ISIS-GCGRRx in Patients With Type 2 Diabetes
Start: Sep 30, 2015
Completion: May 11, 2017
NCT02553889
A Study of Safety, PK, & PD of ISIS 416858 Administered Subcutaneously to Patients With End-Stage Renal Disease on Hemodialysis
Start: Oct 31, 2015
Completion: Nov 30, 2016
NCT02709850
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of IONIS ANGPTL3-LRx in Healthy Volunteers With Elevated Triglycerides and Participants With Familial Hypercholesterolemia
Start: Nov 30, 2015
Completion: Jun 26, 2017
NCT02658175
The Approach Open Label Study: A Study of Volanesorsen (Formerly IONIS-APOCIIIRx) in Participants With Familial Chylomicronemia Syndrome
Start: Dec 23, 2015
Completion: Jan 15, 2020
NCT02527343
The BROADEN Study: A Study of Volanesorsen (Formerly IONIS-APOCIIIRx) in Participants With Familial Partial Lipodystrophy
Start: Dec 28, 2015
Completion: Nov 13, 2019
NCT02623699
An Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of BIIB067 (Tofersen) in Adults With Inherited Amyotrophic Lateral Sclerosis (ALS)
Start: Jan 20, 2016
Completion: Jul 16, 2021
NCT02714764
Evaluation of Outcome Metrics in Alexander Disease
Phase: N/A
Start: Jan 26, 2016
Completion: Dec 31, 2030
NCT02627820
The Effect of an Antisense Oligonucleotide to Lower Transthyretin (TTR) Levels on the Progression of -Wild-type TTR Involving the Heart
Start: Jan 31, 2016
Completion: Dec 31, 2018
NCT02824003
Study to Evaluate the Effect of ISIS GCGRRx on Hepatic Lipid and Glycogen Content in Type 2 Diabetes
Start: Apr 30, 2016
Completion: May 22, 2017
NCT02910635
A Study to Evaluate the Effect of Volanesorsen on Cardiac Repolarization Conducted in Healthy Volunteers
Start: Sep 19, 2016
Completion: Dec 20, 2016
NCT02900027
Safety, Tolerability, PK, and Pharmacodynamics(PD) of IONIS APOCIII-LRx in Healthy Volunteers With Elevated Triglycerides
Start: Sep 30, 2016
Completion: Apr 30, 2018
NCT03047369
The Myelin Disorders Biorepository Project
Start: Dec 8, 2016
Completion: Dec 8, 2030
NCT02981602
Safety, Tolerability, Pharmacokinetics and Antiviral Activity of IONIS-HBVRx in Treatment-Naïve Patients With Chronic HBV Infection
Start: Feb 24, 2017
Completion: Dec 26, 2019
NCT03070782
Phase 2 Study of ISIS 681257 (AKCEA-APO(a)-LRx) in Participants With Hyperlipoproteinemia(a) and Cardiovascular Disease
Start: Mar 7, 2017
Completion: Nov 13, 2018
NCT03070119
Long-Term Evaluation of BIIB067 (Tofersen)
Start: Mar 8, 2017
Completion: Aug 12, 2024
NCT03101878
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Ionis AGT-LRx in Healthy Volunteers
Start: Apr 5, 2017
Completion: Aug 1, 2018
NCT03165864
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ISIS 702843 Administered Subcutaneously to Healthy Volunteers
Start: May 9, 2017
Completion: Sep 25, 2018
NCT03108469
Efficacy and Safety of IONIS-PKKRx for Preventive Treatment of Chronic Migraine
Start: Aug 1, 2017
Completion: Apr 26, 2019
NCT03263507
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Donidalorsen (IONIS-PKK-LRx) Administered Subcutaneously to Healthy Volunteers
Start: Aug 28, 2017
Completion: Oct 9, 2018
NCT03186989
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of IONIS-MAPTRx in Patients With Mild Alzheimer's Disease
Start: Oct 12, 2017
Completion: May 12, 2022
NCT03334214
Safety, Tolerability, and Pharmacodynamics of IONIS-DGAT2Rx in Adult Patients With Type 2 Diabetes
Start: Nov 3, 2017
Completion: Nov 28, 2018
NCT03360747
Phase 2 Study of AKCEA-ANGPTL3-LRx (ISIS 703802) in Participants With Familial Chylomicronemia Syndrome (FCS)
Start: Dec 21, 2017
Completion: Sep 4, 2018
NCT03371355
Study of ISIS 703802 in Participants With Hypertriglyceridemia, Type 2 Diabetes Mellitus, and Nonalcoholic Fatty Liver Disease
Completion: Feb 24, 2020
NCT03358030
A Study of ISIS 416858 Administered Subcutaneously to Participants With End-Stage Renal Disease (ESRD) on Hemodialysis
Start: Dec 26, 2017
Completion: Jul 10, 2019
NCT03385239
Study of ISIS 678354 (AKCEA-APOCIII-LRx) in Participants With Hypertriglyceridemia and Established Cardiovascular Disease (CVD)
Start: Jan 30, 2018
Completion: Feb 25, 2020
NCT03446144
Safety and Efficacy of IONIS-FB-Lrx in up to 120 Patients 55 and Older With Geographic Atrophy (GA) Secondary to Age-Related Macular Degeneration (AMD)
Start: Mar 16, 2018
Completion: Oct 10, 2018
NCT03506854
Study of ISIS 681257 in Patients With Renal Impairment Compared to Healthy Patients
Start: Mar 20, 2018
Completion: Oct 11, 2018
NCT03455777
Study of AKCEA-ANGPTL3-LRX (ISIS 703802) in Patients With Homozygous Familial Hypercholesterolemia (HoFH)
Start: Apr 12, 2018
NCT03514420
Study of AKCEA-ANGPTL3-LRx (ISIS 703802) in Participants With Familial Partial Lipodystrophy (FPL)
Start: Jun 15, 2018
Completion: Aug 21, 2019
NCT03582462
A Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Single and Multiple Doses of IONIS FXI-LRx in up to 84 Healthy Volunteers
Start: Jul 9, 2018
NCT03548415
Safety, Tolerability, and Efficacy of IONIS-GHR-LRx in Participants With Acromegaly Being Treated With Long-acting Somatostatin Receptor Ligands
Start: Sep 13, 2018
Completion: Apr 2, 2021
NCT05337878
A Study to Assess Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Pelacarsen (ISIS 681257) in Healthy Japanese Participants
Start: Oct 15, 2018
Completion: Dec 18, 2019
NCT03647228
A Phase 1/2a Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single and Multiple Doses of IONIS-ENaCRx in Healthy Volunteers and Patients With Cystic Fibrosis
Start: Dec 13, 2018
Completion: Oct 13, 2020
NCT03728634
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 ).
Start: Dec 21, 2018
Completion: Feb 20, 2020
NCT03714776
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
Start: Jan 3, 2019
NCT03876522
Natural History and Functional Status Study of Patients With Lafora Disease
Start: Jan 9, 2019
Completion: Apr 1, 2022
NCT03815825
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)
Start: Mar 4, 2019
Completion: Jun 12, 2024
NCT03967249
Extension Study of IONIS-GHR-LRx Administered to Participants With Acromegaly Being Treated With Long-acting Somatostatin Receptor Ligands
Start: Jul 25, 2019
Completion: Jul 7, 2022
NCT03976349
A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of BIIB094 in Adults With Parkinson's Disease
Start: Aug 12, 2019
NCT04083222
A Study to Assess the Safety, Tolerability and Efficacy of IONIS-AGT-LRx
Start: Nov 13, 2019
Completion: Jul 20, 2020
NCT04014335
A Study to Evaluate the Effectiveness and Safety of IONIS-FB-LRx, an Antisense Inhibitor of Complement Factor B, in Adult Participants With Primary IgA Nephropathy
Start: Dec 4, 2019
Completion: Apr 11, 2024
NCT04136184
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
Start: Dec 11, 2019
Completion: Jul 12, 2023
NCT04030598
A Study to Assess the Clinical Efficacy of Donidalorsen (Also Known as IONIS-PKK-LRx and ISIS 721744) in Participants With Hereditary Angioedema
Start: Jan 7, 2020
Completion: Mar 1, 2021
NCT04136171
CARDIO-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 Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)
Start: Mar 13, 2020
Completion: Aug 31, 2026
NCT04307381
An Extension Study of Donidalorsen (IONIS-PKK-LRx) in Participants With Hereditary Angioedema
Start: Apr 1, 2020
Completion: Jan 24, 2025
NCT04302064
A Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Single-Ascending and Multiple Doses of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in Healthy Japanese Participants
Start: Apr 23, 2020
Completion: Sep 10, 2020
NCT04288128
Integrated Functional Evaluation of the Cerebellum
Start: May 28, 2020
Completion: Jun 1, 2022
NCT04059406
Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Sapablursen (Formerly ISIS 702843, IONIS-TMPRSS6-LRx)
Start: Sep 24, 2020
Completion: Mar 28, 2023
NCT04549922
Antisense Therapy to Block the Kallikrein-kinin Pathway in COVID-19
Start: Oct 19, 2020
Completion: Dec 9, 2021
NCT04568434
A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) Administered to Patients With Familial Chylomicronemia Syndrome (FCS)
Start: Nov 18, 2020
Completion: Oct 17, 2023
NCT04441788
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)
Start: Dec 22, 2020
Completion: Aug 9, 2021
NCT04522180
A Study to Assess the Safety, Tolerability, and Efficacy of IONIS-GHR-LRx Administered in Patients With Acromegaly
Start: Jan 4, 2021
Completion: May 4, 2023
NCT04659096
A Study of ION537 in Patients With Molecularly Selected Advanced Solid Tumors
Start: Jan 5, 2021
Completion: Oct 19, 2022
NCT04398485
A Study of ION251 Administered to Patients With Relapsed/Refractory Multiple Myeloma
Start: Jan 20, 2021
Completion: Sep 30, 2024
NCT04731623
A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION904
Start: Feb 26, 2021
Completion: Sep 11, 2021
NCT04934891
A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single and Multiple Doses of ION547
Start: Mar 24, 2021
Completion: Sep 14, 2022
NCT06073574
CARDIO-TTRansform Magnetic Resonance Imaging (MRI) Sub-study
Start: Mar 31, 2021
Completion: Apr 30, 2026
NCT04714320
A Study to Assess the Safety, Tolerability and Efficacy of IONIS-AGT-LRx in Hypertensive Participants With Uncontrolled Blood Pressure
Start: Apr 28, 2021
Completion: Oct 3, 2022
NCT04849741
A Study to Evaluate the Safety and Efficacy of Zilganersen (ION373) in Patients With Alexander Disease (AxD)
Start: Jun 1, 2021
Completion: Sep 30, 2029
NCT04836182
A Study to Assess the Safety, Tolerability and Efficacy of IONIS-AGT-LRx in Participants With Chronic Heart Failure With Reduced Ejection Fraction
Start: Jun 8, 2021
Completion: Jan 11, 2023
NCT04768972
FUSION: A Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of ION363 in Amyotrophic Lateral Sclerosis Participants With Fused in Sarcoma Mutations (FUS-ALS)
Start: Jun 14, 2021
Completion: Mar 31, 2028
NCT04932512
A Study to Assess the Safety, Efficacy, and Pharmacokinetics of Multiple Doses of ION224
Start: Jun 17, 2021
Completion: Feb 28, 2024
NCT05637697
Angelman Syndrome Video Assessment (ASVA) Source Material Study
Start: Aug 31, 2021
Completion: Aug 1, 2023
NCT05079919
A Study of Olezarsen (ISIS 678354) Administered to Participants With Severe Hypertriglyceridemia
Start: Oct 25, 2021
Completion: Jul 8, 2025
NCT05130450
A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) in Participants With Familial Chylomicronemia Syndrome (FCS)
Start: Nov 18, 2021
Completion: Feb 29, 2028
NCT05139810
OASIS-HAE: A Study to Evaluate the Safety and Efficacy of Donidalorsen (ISIS 721744 or IONIS-PKK-LRx) in Participants With Hereditary Angioedema (HAE)
Start: Dec 3, 2021
Completion: Nov 9, 2023
NCT05127226
HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple Ascending Doses of ION582 in Participants With Angelman Syndrome
Start: Dec 22, 2021
Completion: Mar 31, 2029
NCT05143957
A Study to Evaluate Sapablursen (Formerly ISIS 702843, IONIS-TMPRSS6-LRx) in Patients With Polycythemia Vera
Start: Dec 30, 2021
Completion: Jun 30, 2026
NCT05071300
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
Start: Jan 4, 2022
Completion: Aug 31, 2029
NCT05259072
carDIo-ttranSSfOrm nucLEar Imaging Study
Start: Jan 19, 2022
Completion: Dec 30, 2023
NCT05311488
Early Detection of Neuropathy in ATTRv
Start: Feb 14, 2022
Completion: Feb 14, 2029
NCT05185843
A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRX) Administered to Adults With Familial Chylomicronemia Syndrome (FCS) Previously Treated With Volanesorsen
Start: Feb 25, 2022
Completion: Jun 30, 2027
NCT05314439
A Study to Assess the Safety, Tolerability, and Efficacy of Monthly Subcutaneous Administration of ION904 in Participants With Uncontrolled Hypertension
Start: Apr 29, 2022
Completion: Feb 15, 2023
NCT05355402
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
Start: May 9, 2022
Completion: Dec 21, 2023
NCT05392114
A Study to Assess the Long-Term Safety and Efficacy of Donidalorsen in the Prophylactic Treatment of Hereditary Angioedema (HAE)
Start: Jul 13, 2022
Completion: Mar 31, 2027
NCT04165486
Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of ION464 Administered to Adults With Multiple System Atrophy (HORIZON)
Start: Jul 21, 2022
Completion: Sep 30, 2027
NCT05552326
A Study of Olezarsen Administered Subcutaneously to Participants With Severe Hypertriglyceridemia
Start: Aug 31, 2022
Completion: Sep 12, 2025
NCT05659901
Rocket Study: A Study to Characterize Biomarkers and Disease Progression in Participants With Pelizaeus-Merzbacher Disease
Start: Oct 3, 2022
NCT05579860
A Study Comparing Two Subcutaneous Formulations: Vial and Autoinjector (AI) With Olezarsen, at Two Dose Levels, in Healthy Adult Participants
Start: Oct 5, 2022
Completion: May 22, 2023
NCT05610280
A Study of Olezarsen (ISIS 678354) in Participants With Hypertriglyceridemia and Atherosclerotic Cardiovascular Disease, or With Severe Hypertriglyceridemia
Start: Nov 21, 2022
Completion: Jun 9, 2025
NCT05667493
An Extension Study to Assess Long-Term Safety of Eplontersen in Adults With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM)
Start: Nov 30, 2022
NCT05681351
CORE-OLE: A Study of Olezarsen (ISIS 678354) Administered Subcutaneously to Participants With Severe Hypertriglyceridemia (SHTG)
Start: Dec 13, 2022
NCT06073587
The CARDIO-TTRansform Scintigraphy Sub-study
Start: Apr 4, 2023
Completion: Mar 31, 2026
NCT06014541
Observational Study to Characterize Biomarkers and Disease Progression in Participants With Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome
Start: Oct 3, 2023
NCT06153966
PrProfile: A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION717
Start: Jan 4, 2024
Completion: Jul 31, 2027
NCT06150716
Orbit Study: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356 in Participants With Pelizaeus Merzbacher Disease (PMD)
Start: Apr 10, 2024
Completion: Jun 30, 2028
NCT06430385
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)
Start: Oct 21, 2024
Completion: Apr 30, 2030
NCT06673069
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)
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REVEAL: A Phase 3 Study of ION582 in Angelman Syndrome
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