Clinical trials: sponsored by Ionis Pharmaceuticals, Inc.
Primary IgA Nephropathy
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
The purpose of this study is to evaluate the effectiveness and safety of IONIS-FB-LRx, an antisense inhibitor of complement factor B messenger ribonucleic acid (CFB mRNA), and to evaluate the effect of IONIS-FB-LRx on plasma…
- Trial name
- NCT04014335
- Testing
- IONIS-FB-LRx
- Where
- 7 sites in Australia, Canada and 2 more
- Volunteers
- 23 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Familial Chylomicronemia Syndrome
Olezarsen Early Access Program for Patients With Familial Chylomicronemia Syndrome (FCS)
The purpose of the Expanded Access Program is to provide pre-approval access of olezarsen to eligible patients with Familial Chylomicronemia Syndrome (FCS).
- Trial name
- NCT06360237
- Testing
- Olezarsen
- Where
- United States
- 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.
Steatohepatitis, Nonalcoholic
A Study to Assess the Safety, Efficacy, and Pharmacokinetics of Multiple Doses of ION224
The purpose of this study is to assess the effect of multiple ION224 doses when administered by subcutaneous (SC) injection for 49 weeks (end of the treatment [EOT]) on non-alcoholic steatohepatitis (NASH) histologic improvement…
- Trial name
- NCT04932512
- Testing
- ION224
- Where
- 51 sites in United States, Puerto Rico
- Volunteers
- 160 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Relapsed Multiple Myeloma, Refractory Multiple Myeloma
A Study of ION251 Administered to Patients With Relapsed/Refractory Multiple Myeloma
The purpose of this study is to determine the maximum-tolerated dose (MTD) and recommended Phase 2 dose (RP2D) of ION251 in patients with relapsed/refractory multiple myeloma.
- Trial name
- NCT04398485
- Testing
- ION251
- Where
- 6 sites in United States
- Volunteers
- 23 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.
Healthy Participants
A Study Comparing Two Subcutaneous Formulations: Vial and Autoinjector (AI) With Olezarsen, at Two Dose Levels, in Healthy Adult Participants
The purpose of the study is to assess the bioequivalence of olezarsen between 2 subcutaneous (SC) formulations [(autoinjector (AI) and vial] at 2 dose levels in healthy adult participants.
- Trial name
- NCT05579860
- Testing
- Olezarsen
- Where
- United States
- Volunteers
- 104 people, healthy volunteers welcome
- Sponsor
- Ionis Pharmaceuticals, Inc.
Hypertension
A Study to Assess the Safety, Tolerability, and Efficacy of Monthly Subcutaneous Administration of ION904 in Participants With Uncontrolled Hypertension
The purpose of this study is to evaluate the effect of ION904 on plasma angiotensinogen (AGT) in participants with uncontrolled hypertension.
- Trial name
- NCT05314439
- Testing
- ION904
- Where
- 8 sites in United States
- Volunteers
- 48 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.
Castrate-Resistant Prostate Cancer
Safety and Tolerability Study of ISIS EIF4E Rx in Combination With Docetaxel and Prednisone (CRPC)
The purpose of this study is to examine the safety, tolerability and progression-free survival of patients with Castrate-Resistant Prostate Cancer treated with ISIS EIF4E Rx in combination with docetaxel and prednisone.
- Trial name
- NCT01234025
- Testing
- ISIS EIF4E Rx, Prednisone, Docetaxel
- Where
- 38 sites in United States, Hungary and 4 more
- Volunteers
- 113 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Chronic Heart Failure With Reduced Ejection Fraction
A Study to Assess the Safety, Tolerability and Efficacy of IONIS-AGT-LRx in Participants With Chronic Heart Failure With Reduced Ejection Fraction
The purpose of this study is to evaluate the effect of IONIS-AGT-LRX weekly subcutaneous (SC) injection on plasma angiotensinogen (AGT) concentration from Baseline to Study Day 85 (Week 13) and to evaluate the effect of…
- Trial name
- ASTRAAS-HF (NCT04836182)
- Testing
- IONIS-AGT-LRx
- Where
- 19 sites in United States, Hungary and 1 more
- Volunteers
- 72 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.
Acromegaly
Extension Study of IONIS-GHR-LRx Administered to Participants With Acromegaly Being Treated With Long-acting Somatostatin Receptor Ligands
The purpose of this study is to assess the safety and efficacy of extended dosing with IONIS GHR-LRx in participants with acromegaly as add-on to somatostatin receptor ligands (SRL) therapy.
- Trial name
- NCT03967249
- Testing
- IONIS GHR-LRx, Somatostatin Receptor Ligand (SRL)
- Where
- 22 sites in United States, Hungary and 4 more
- Volunteers
- 39 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.
Advanced Solid Tumors
A Study of ION537 in Patients With Molecularly Selected Advanced Solid Tumors
The purpose of this study is to evaluate the safety and establish the maximum tolerated dose (MTD) and recommended Phase 2 dose (RP2D) of ION537 when administered by intravenous infusion in patients with advanced solid tumors.
- Trial name
- NCT04659096
- Testing
- ION537
- Where
- United States
- Volunteers
- 15 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.
Myotonic Dystrophy Type 1
A Safety andTolerability Study of Multiple Doses of ISIS-DMPKRx in Adults With Myotonic Dystrophy Type 1
This study will test the safety, tolerability, and pharmacokinetics of multiple escalating doses of ISIS-DMPKRx administered subcutaneously to adult patients with DM1.
- Trial name
- NCT02312011
- Testing
- IONIS-DMPKRx
- Where
- 8 sites in United States
- Volunteers
- 48 people
- Sponsor
- Ionis Pharmaceuticals, Inc.