Clinical trials: sponsored by 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
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
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.
Angelman Syndrome
Angelman Syndrome Video Assessment (ASVA) Source Material Study
This is a longitudinal, observational, nonrandomized, fully remote study enrolling approximately 55 participant-caregiver dyads.
- Trial name
- ASVA SMS (NCT05637697)
- Where
- United States
- Volunteers
- 73 people, healthy volunteers welcome
- Sponsor
- The Emmes Company, LLC
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.
Healthy Volunteers
A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single and Multiple Doses of ION547
The purpose of this study is to evaluate the safety and tolerability of single and multiple doses of the antisense inhibitor ION547 administered subcutaneously (SC) in healthy participants.
- Trial name
- NCT04934891
- Testing
- ION547
- Where
- Canada
- Volunteers
- 48 people, healthy volunteers welcome
- Sponsor
- Ionis Pharmaceuticals, Inc.
Healthy Volunteers
A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION904
The purpose of this study is to assess the safety and tolerability of ION904.
- Trial name
- NCT04731623
- Testing
- ION904
- Where
- Canada
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Ionis Pharmaceuticals, Inc.
Healthy Volunteers
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Donidalorsen (IONIS-PKK-LRx) Administered Subcutaneously to Healthy Volunteers
The purpose is to assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Donidalorsen administered subcutaneously to Healthy Volunteers
- Trial name
- NCT03263507
- Testing
- Donidalorsen
- Where
- Canada
- Volunteers
- 32 people, healthy volunteers welcome
- Sponsor
- Ionis Pharmaceuticals, Inc.
Healthy Participants
A Study to Assess Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Pelacarsen (ISIS 681257) in Healthy Japanese Participants
The purpose of the study is to assess the safety, tolerability, and pharmacokinetics (PK) of single and multiple subcutaneous (SC) doses of Pelacarsen (ISIS 681257) in healthy Japanese participants.
- Trial name
- NCT05337878
- Testing
- Pelacarsen
- Where
- United States
- Volunteers
- 29 people, healthy volunteers welcome
- Sponsor
- Ionis Pharmaceuticals, Inc.
Healthy Participants
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
To evaluate the safety and tolerability of single and potentially multiple doses of Eplontersen administered subcutaneously (SC) to healthy Japanese participants.
- Trial name
- NCT04302064
- Testing
- Eplontersen
- Where
- United States
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Ionis Pharmaceuticals, Inc.
Abnormalities, Cardiovascular
A Study to Evaluate the Effect of Volanesorsen on Cardiac Repolarization Conducted in Healthy Volunteers
The primary objective of this study is to assess the corrected QT interval (QTc) effect of volanesorsen (ISIS 304801) administered as a 300 mg subcutaneous (SC) therapeutic and a 300 mg intravenous (IV; 2-hour infusion)…
- Trial name
- NCT02910635
- Testing
- Volanesorsen, Moxifloxacin
- Where
- United States
- Volunteers
- 52 people, healthy volunteers welcome
- Sponsor
- Ionis Pharmaceuticals, Inc.
Healthy Participants
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
To evaluate the safety and tolerability, pharmacokinetics, and pharmacodynamics, including Factor XI activity and antigen levels, activated partial thromboplastin time, prothrombin time and international normalized ratio of…
- Trial name
- NCT03582462
- Testing
- IONIS FXI-LRx
- Where
- Canada
- Volunteers
- 66 people, healthy volunteers welcome
- Sponsor
- Ionis Pharmaceuticals, Inc.
Healthy Subjects, Cystic Fibrosis
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
This Phase 1/2a study is a double-blinded (subject and Investigator), randomized, placebo-controlled, dose-escalation study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of single and multiple…
- Trial name
- NCT03647228
- Testing
- IONIS-ENaCRx
- Where
- 8 sites in Germany, United Kingdom
- Volunteers
- 98 people, healthy volunteers welcome
- Sponsor
- Ionis Pharmaceuticals, Inc.
Hypertriglyceridemia, Familial Hypercholesterolemia
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of IONIS ANGPTL3-LRx in Healthy Volunteers With Elevated Triglycerides and Participants With Familial Hypercholesterolemia
The purpose is to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of IONIS ANGPTL3-LRx (ISIS 703802) given to healthy volunteer subjects with elevated triglycerides and subjects with familial…
- Trial name
- NCT02709850
- Testing
- IONIS ANGPTL3-LRx
- Where
- Canada
- Volunteers
- 48 people, healthy volunteers welcome
- Sponsor
- Ionis Pharmaceuticals, Inc.
Renal Impairment
Study of ISIS 681257 in Patients With Renal Impairment Compared to Healthy Patients
This is a Phase 1, Multicenter, Open-Label, Parallel Group Adaptive Pharmacokinetic Single Dose Study of ISIS 681257 Subcutaneous Injections in Male And Female Subjects with Normal and Impaired Renal Function
- Trial name
- NCT03506854
- Testing
- ISIS 681257
- Where
- 3 sites in United States, Canada
- Volunteers
- 16 people, healthy volunteers welcome
- Sponsor
- Akcea Therapeutics
Thalassemia
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ISIS 702843 Administered Subcutaneously to Healthy Volunteers
The purpose is to assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of IONIS TMPRSS6-Lrx Administered Subcutaneously for up to 44 Healthy Volunteers
- Trial name
- NCT03165864
- Testing
- IONIS TMPRSS6-Lrx
- Where
- Australia
- Volunteers
- 36 people, healthy volunteers welcome
- Sponsor
- Ionis Pharmaceuticals, Inc.
Elevated Lipoprotein(a)
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of IONIS APO(a)-LRx in Healthy Volunteers With Elevated Lipoprotein(a)
The purpose is to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of IONIS APO(a)-LRx (ISIS 681257) given to healthy volunteer subjects.
- Trial name
- NCT02414594
- Testing
- IONIS-APO(a)-LRx, Sterile Normal Saline (0.9% NaCl)
- Where
- Canada
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Ionis Pharmaceuticals, Inc.
Healthy Volunteers
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Ionis AGT-LRx in Healthy Volunteers
The purpose is to assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of IONIS AGT-LRx in up to 82 Healthy Volunteers
- Trial name
- NCT03101878
- Testing
- Ionis AGT-LRx
- Where
- Canada
- Volunteers
- 62 people, healthy volunteers welcome
- Sponsor
- Ionis Pharmaceuticals, Inc.
Elevated Triglycerides (TG)
Safety, Tolerability, PK, and Pharmacodynamics(PD) of IONIS APOCIII-LRx in Healthy Volunteers With Elevated Triglycerides
The purpose is to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of IONIS APOC-III-LRx given to healthy volunteer subjects.
- Trial name
- NCT02900027
- Testing
- APOC-III-L-Rx
- Where
- Canada
- Volunteers
- 56 people, healthy volunteers welcome
- Sponsor
- Ionis Pharmaceuticals, Inc.
Lipid Metabolism, Inborn Errors, Hyperlipidemias
Measure Liver Fat Content After ISIS 301012 (Mipomersen) Administration
This study will assess what, if any, effect that ISIS 301012 (mipomersen) has on liver triglyceride content in multiple groups of subjects with varying degrees of risk for hepatic steatosis.
- Conditions
- Lipid Metabolism, Inborn Errors, Hyperlipidemias, Metabolic Diseases, Hypolipoproteinemia
- Trial name
- NCT00362180
- Testing
- mipomersen
- Where
- Netherlands
- Volunteers
- 38 people, healthy volunteers welcome
- Sponsor
- Kastle Therapeutics, LLC