IONIS PHARMACEUTICALS, INC.
- Country
- 🇺🇸United States
- Ownership
- Public, Private, Subsidiary
- Established
- 1989-01-01
- Employees
- 927
- Market Cap
- -
- Website
- http://www.ionis.fr
REVEAL: A Phase 3 Study of ION582 in Angelman Syndrome
- First Posted Date
- 2025-04-06
- Last Posted Date
- 2025-05-06
- Lead Sponsor
- Ionis Pharmaceuticals, Inc.
- Target Recruit Count
- 210
- Registration Number
- NCT06914609
- Locations
- 🇺🇸
University of North Carolina at Chapel Hill School of Medicine, Carrboro, North Carolina, United States
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)
- First Posted Date
- 2024-11-04
- Last Posted Date
- 2025-05-01
- Lead Sponsor
- Ionis Pharmaceuticals, Inc.
- Target Recruit Count
- 30
- Registration Number
- NCT06673069
- Locations
- 🇪🇸
Ionis Investigative Site, Barcelona, Spain
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)
- Conditions
- Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome
- Interventions
- Procedure: Sham procedure
- First Posted Date
- 2024-05-28
- Last Posted Date
- 2025-05-14
- Lead Sponsor
- Ionis Pharmaceuticals, Inc.
- Target Recruit Count
- 48
- Registration Number
- NCT06430385
- Locations
- 🇺🇸
Rady Children's Hospital, San Diego, California, United States
🇺🇸Gillette Children's Specialty Healthcare, Saint Paul, Minnesota, United States
🇺🇸Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, 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
- 2024-12-27
- 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
- First Posted Date
- 2023-12-01
- Last Posted Date
- 2024-12-27
- Lead Sponsor
- Ionis Pharmaceuticals, Inc.
- Target Recruit Count
- 56
- Registration Number
- NCT06153966
- Locations
- 🇺🇸
UCLA Neurology Clinic, Los Angeles, California, United States
🇺🇸University of Colorado Hospital, Denver, Colorado, United States
🇺🇸Massachusetts General Hospital, Boston, Massachusetts, United States
Orbit Study: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356 in Participants With Pelizaeus Merzbacher Disease (PMD)
- First Posted Date
- 2023-11-29
- Last Posted Date
- 2025-05-01
- Lead Sponsor
- Ionis Pharmaceuticals, Inc.
- Target Recruit Count
- 24
- Registration Number
- NCT06150716
- Locations
- 🇳🇱
Ionis Investigative Site, Amsterdam, Netherlands
The CARDIO-TTRansform Scintigraphy Sub-study
- 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
CARDIO-TTRansform Magnetic Resonance Imaging (MRI) Sub-study
- 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
Observational Study to Characterize Biomarkers and Disease Progression in Participants With Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome
- Conditions
- Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome
- First Posted Date
- 2023-08-28
- Last Posted Date
- 2025-02-04
- 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