IONIS PHARMACEUTICALS, INC.

🇺🇸United States

Country: 🇺🇸United States

Ownership: Public, Private, Subsidiary

Established: 1989-01-01

Employees: 927

Market Cap: -

Website: http://www.ionis.fr

Clinical Trials

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)

Phase 1

Recruiting

Conditions: Alzheimer Disease
Down Syndrome

Interventions: Drug: ION269

First Posted Date: 2024-11-04

Last Posted Date: 2024-12-06

Lead Sponsor: Ionis Pharmaceuticals, Inc.

Target Recruit Count: 30

Registration Number: NCT06673069

Locations: 🇺🇸
Ionis Investigative Site, Saint Louis, Missouri, United States

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)

Phase 1

Recruiting

Conditions: Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome

Interventions: Drug: ION440
Procedure: Sham procedure

First Posted Date: 2024-05-28

Last Posted Date: 2024-11-22

Lead Sponsor: Ionis Pharmaceuticals, Inc.

Target Recruit Count: 48

Registration Number: NCT06430385

Locations: 🇺🇸
Baylor College of Medicine, Houston, Texas, United States

Donidalorsen Expanded Access Program for Patients With Hereditary Angioedema

Conditions: Hereditary Angioedema

First Posted Date: 2024-05-16

Last Posted Date: 2024-05-16

Lead Sponsor: Ionis Pharmaceuticals, Inc.

Registration Number: NCT06415448

Olezarsen Early Access Program for Patients With Familial Chylomicronemia Syndrome (FCS)

Conditions: Familial Chylomicronemia Syndrome

First Posted Date: 2024-04-11

Last Posted Date: 2024-08-26

Lead Sponsor: Ionis Pharmaceuticals, Inc.

Registration Number: NCT06360237

Locations: 🇺🇸
Expanded Access Site, Carlsbad, California, United States

PrProfile: A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION717

Phase 1

Recruiting

Conditions: Prion Disease

Interventions: Drug: ION717
Drug: Placebo

First Posted Date: 2023-12-01

Last Posted Date: 2024-12-16

Lead Sponsor: Ionis Pharmaceuticals, Inc.

Target Recruit Count: 56

Registration Number: NCT06153966

Locations: 🇺🇸
Massachusetts General Hospital, Boston, Massachusetts, United States
🇯🇵
Mihara Memorial Hospital, Gunma, Isesaki-shi, Japan
🇯🇵
Neuromuscular Center Yoshimizu Hospital, Yamaguchi, Shimonoseki-shi, Japan

and more 13 locations

Orbit Study: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356 in Participants With Pelizaeus Merzbacher Disease (PMD)

Phase 1

Recruiting

Conditions: Pelizaeus-Merzbacher Disease

Interventions: Drug: ION356

First Posted Date: 2023-11-29

Last Posted Date: 2024-11-12

Lead Sponsor: Ionis Pharmaceuticals, Inc.

Target Recruit Count: 24

Registration Number: NCT06150716

Locations: 🇳🇱
Ionis Investigative Site, Amsterdam, Netherlands

The CARDIO-TTRansform Scintigraphy Sub-study

Active, not recruiting

Conditions: Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM)

Interventions: Diagnostic Test: Scintigraphy scan

First Posted Date: 2023-10-10

Last Posted Date: 2024-10-10

Lead Sponsor: Ionis Pharmaceuticals, Inc.

Target Recruit Count: 150

Registration Number: NCT06073587

Locations: 🇺🇸
Duke University Medical Center, Durham, North Carolina, United States
🇺🇸
Cleveland Clinic Main Campus, Cleveland, Ohio, United States
🇪🇸
Hospital Universitario Puerta de Hierro, Majadahonda, Spain

and more 2 locations

CARDIO-TTRansform Magnetic Resonance Imaging (MRI) Sub-study

Active, not recruiting

Conditions: Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM)

Interventions: Diagnostic Test: Cardiac MRI

First Posted Date: 2023-10-10

Last Posted Date: 2024-10-10

Lead Sponsor: Ionis Pharmaceuticals, Inc.

Target Recruit Count: 150

Registration Number: NCT06073574

Locations: 🇺🇸
Brigham and Women's Hospital, Boston, Massachusetts, United States
🇺🇸
Oregon Health and Science University, Portland, Oregon, United States
🇬🇧
Synexus - Scotland Clinical Research Centre, Bellshill, United Kingdom

and more 8 locations

Observational Study to Characterize Biomarkers and Disease Progression in Participants With Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome

Recruiting

Conditions: Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome

First Posted Date: 2023-08-28

Last Posted Date: 2024-07-24

Lead Sponsor: Ionis Pharmaceuticals, Inc.

Target Recruit Count: 40

Registration Number: NCT06014541

Locations: 🇺🇸
UCSD - Rady Children's Hospital, San Diego, California, United States
🇺🇸
Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States
🇺🇸
Baylor College of Medicine, Houston, Texas, United States

and more 2 locations

CORE-OLE: A Study of Olezarsen (ISIS 678354) Administered Subcutaneously to Participants With Severe Hypertriglyceridemia (SHTG)

Phase 3

Recruiting

Conditions: Severe Hypertriglyceridemia

Interventions: Drug: Olezarsen

First Posted Date: 2023-01-12

Last Posted Date: 2024-12-13

Lead Sponsor: Ionis Pharmaceuticals, Inc.

Target Recruit Count: 800

Registration Number: NCT05681351

Locations: 🇺🇸
The Presbyterian Hospital DBA Novant Health Heart and Vascular Institute, Charlotte, North Carolina, United States
🇺🇸
Kenneth Hilty, Greensboro, North Carolina, United States
🇺🇸
Clinical Trials of America, LLC, Lenoir, North Carolina, United States

and more 183 locations

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