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IONIS PHARMACEUTICALS, INC.

IONIS PHARMACEUTICALS, INC. logo
🇺🇸United States
Ownership
Public, Private, Subsidiary
Established
1989-01-01
Employees
927
Market Cap
-
Website
http://www.ionis.fr

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

Phase 1
Completed
Conditions
Healthy Participants
Interventions
Drug: Placebo
First Posted Date
2020-03-10
Last Posted Date
2022-03-31
Lead Sponsor
Ionis Pharmaceuticals, Inc.
Target Recruit Count
24
Registration Number
NCT04302064
Locations
🇺🇸

WCCT Global, Cypress, California, United States

Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of ION464 Administered to Adults With Multiple System Atrophy (HORIZON)

Phase 1
Recruiting
Conditions
Multiple System Atrophy
Interventions
Drug: Placebo
First Posted Date
2019-11-18
Last Posted Date
2024-11-12
Lead Sponsor
Ionis Pharmaceuticals, Inc.
Target Recruit Count
40
Registration Number
NCT04165486
Locations
🇫🇷

CHU de Lyon - Hospices Civils de Lyon-H6pital Pierre Wertheimer, Neurologique HCL, Lyon, France

🇩🇪

Klinikum der Universtiatet Muenchen -Campus Grosshadern, Muenchen, Germany

🇬🇧

Salford Royal Hospital, Salford, United Kingdom

and more 11 locations

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

Phase 3
Completed
Conditions
Hereditary Transthyretin-Mediated Amyloid Polyneuropathy
Interventions
First Posted Date
2019-10-23
Last Posted Date
2024-12-13
Lead Sponsor
Ionis Pharmaceuticals, Inc.
Target Recruit Count
168
Registration Number
NCT04136184
Locations
🇺🇸

Mayo Clinic - Arizona, Scottsdale, Arizona, United States

🇺🇸

Mayo Clinic - Jacksonville, Jacksonville, Florida, United States

🇺🇸

Indiana University School of Medicine - Indianapolis, Indianapolis, Indiana, United States

and more 42 locations

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)

Phase 3
Active, not recruiting
Conditions
Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)
Interventions
Drug: Placebo
First Posted Date
2019-10-23
Last Posted Date
2024-12-27
Lead Sponsor
Ionis Pharmaceuticals, Inc.
Target Recruit Count
1438
Registration Number
NCT04136171
Locations
🇺🇸

Penn Presbyterian Medical Center, Philadelphia, Pennsylvania, United States

🇺🇸

University of Texas Southwestern Medical Center, Dallas, Texas, United States

🇺🇸

University of Maryland Medical Center, Baltimore, Maryland, United States

and more 141 locations

A Study to Assess the Safety, Tolerability and Efficacy of IONIS-AGT-LRx

Phase 2
Completed
Conditions
Hypertension
Interventions
Drug: Placebo
First Posted Date
2019-09-10
Last Posted Date
2023-01-18
Lead Sponsor
Ionis Pharmaceuticals, Inc.
Target Recruit Count
26
Registration Number
NCT04083222
Locations
🇺🇸

National Research Institute - Wilshire, Los Angeles, California, United States

🇺🇸

Midwest Institute for Clinical Research, Indianapolis, Indiana, United States

🇺🇸

Central Alabama Research, Birmingham, Alabama, United States

and more 6 locations

Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Sapablursen (Formerly ISIS 702843, IONIS-TMPRSS6-LRx)

Phase 2
Terminated
Conditions
Beta Thalassemia Intermedia
Interventions
First Posted Date
2019-08-16
Last Posted Date
2025-02-18
Lead Sponsor
Ionis Pharmaceuticals, Inc.
Target Recruit Count
29
Registration Number
NCT04059406
Locations
🇬🇷

Aghia Sophia General Children's Hospital, Athens, Attica, Greece

🇦🇺

Royal Prince Alfred Hospital, Camperdown, New South Wales, Australia

🇦🇺

Monash Medical Centre, Clayton, Victoria, Australia

and more 16 locations

A Study to Assess the Clinical Efficacy of Donidalorsen (Also Known as IONIS-PKK-LRx and ISIS 721744) in Participants With Hereditary Angioedema

Phase 2
Completed
Conditions
Hereditary Angioedema
Interventions
Drug: Placebo
First Posted Date
2019-07-24
Last Posted Date
2023-04-03
Lead Sponsor
Ionis Pharmaceuticals, Inc.
Target Recruit Count
23
Registration Number
NCT04030598
Locations
🇺🇸

Bernstein Clinical Research Center, LLC, Cincinnati, Ohio, United States

🇺🇸

Medical Research of Arizona, Scottsdale, Arizona, United States

🇺🇸

Penn State Hershey Medical Center, Hershey, Pennsylvania, United States

and more 5 locations

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

Phase 2
Completed
Conditions
Primary IgA Nephropathy
Interventions
First Posted Date
2019-07-10
Last Posted Date
2025-01-23
Lead Sponsor
Ionis Pharmaceuticals, Inc.
Target Recruit Count
23
Registration Number
NCT04014335
Locations
🇸🇬

IONIS Investigative Site, Singapore, Singapore

Extension Study of IONIS-GHR-LRx Administered to Participants With Acromegaly Being Treated With Long-acting Somatostatin Receptor Ligands

Phase 2
Completed
Conditions
Acromegaly
Interventions
First Posted Date
2019-05-30
Last Posted Date
2023-03-22
Lead Sponsor
Ionis Pharmaceuticals, Inc.
Target Recruit Count
39
Registration Number
NCT03967249
Locations
🇵🇱

Mazowiecki Szpital Brodnowski, Warsaw, Poland

🇷🇺

Interregional Clinical Diagnostic Center, Kazan, Russian Federation

🇷🇺

Federal State Budget Institution "National Medical Research Center of Endocrinology" of the Ministry of Healthcare of the Russian Federation, Moscow, Russian Federation

and more 19 locations

Natural History and Functional Status Study of Patients With Lafora Disease

Completed
Conditions
Lafora Disease
First Posted Date
2019-03-15
Last Posted Date
2022-09-28
Lead Sponsor
Ionis Pharmaceuticals, Inc.
Target Recruit Count
33
Registration Number
NCT03876522
Locations
🇪🇸

IONIS Investigative Site, Madrid, Spain

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