Ionis Pharmaceuticals, Inc.
Ionis Pharmaceuticals, Inc.
Explore linked clinical trials.22 results · Page 1 of 1
Clinical trials
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| Trial | Conditions | Status | Phase | Updated | More info | NCT ID |
|---|---|---|---|---|---|---|
| A Study of Olezarsen for the Treatment of Familial Chylomicronemia Syndrome (FCS) in Pediatric Participants | familial chylomicronemia syndrome, familial lipoprotein lipase deficiency | Recruiting | Phase 3 | 2026-10-08 | NCT07727538 | |
| 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) | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Neurobehavioral Manifestations, Neurologic Manifestations, Rett syndrome, X-linked disease, hereditary disease, inherited neurodegenerative disorder, intellectual disability, nervous system disorder | Recruiting | Phase 1 / Phase 2 | 2026-10-08 | NCT06430385 | |
| Rocket Study: A Study to Characterize Biomarkers and Disease Progression in Participants With Pelizaeus-Merzbacher Disease | Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hereditary Central Nervous System Demyelinating Diseases, Leukoencephalopathies, Nutritional and Metabolic Diseases, Pelizaeus-Merzbacher spectrum disorder, X-linked disease, brain disorder, central nervous system disorder, demyelinating disease, hereditary disease, inborn errors of metabolism, metabolic disease, nervous system disorder | Recruiting | Not stated | 2026-10-02 | NCT05659901 | |
| REVEAL: A Phase 3 Study of Obudanersen (ION582) in Angelman Syndrome | Abnormalities, Multiple, Angelman syndrome, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Imprinting Disorders, central nervous system disorder, chromosomal disorder, hereditary disease, movement disorder, nervous system disorder | Active, not recruiting | Phase 3 | 2026-09-29 | NCT06914609 | |
| CHAMPION: A Study to Evaluate the Efficacy and Safety of Obudanersen (ION582) in Children and Adults With Angelman Syndrome (AS) | Abnormalities, Multiple, Angelman syndrome, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Imprinting Disorders, central nervous system disorder, chromosomal disorder, hereditary disease, movement disorder, nervous system disorder | Recruiting | Phase 3 | 2026-09-28 | NCT07782827 | |
| PrProfile: A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION717 | Infections, central nervous system disorder, central nervous system infectious disorder, nervous system disorder, neurodegenerative disease, prion disease | Active, not recruiting | Phase 1 / Phase 2 | 2026-09-18 | NCT06153966 | |
| A Study to Assess the Safety, Tolerability and Efficacy of ION775 | Dyslipidemias, Lipid Metabolism Disorders, Nutritional and Metabolic Diseases, hyperlipidemia, hypertriglyceridemia, metabolic disease | Recruiting | Phase 2 | 2026-09-03 | NCT07604974 | |
| ASCEND: Safety and Tolerability of ION337 for the Treatment of Dravet Syndrome | Epilepsies, Myoclonic, Epilepsy, Generalized, Epileptic Syndromes, brain disorder, central nervous system disorder, epilepsy, nervous system disorder | Recruiting | Phase 1 / Phase 2 | 2026-09-03 | NCT07531745 | |
| Donidalorsen Treatment in Children With Hereditary Angioedema | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hereditary Complement Deficiency Diseases, Hypersensitivity, Immediate, Primary Immunodeficiency Diseases, Skin and Connective Tissue Diseases, angioedema, cardiovascular disorder, hereditary angioedema, hereditary disease, immune system disorder, inborn error of immunity, skin disorder, skin vascular disease, urticaria, vascular disorder | Recruiting | Phase 3 | 2026-09-02 | NCT07298447 | |
| A Study to Evaluate the Safety and Efficacy of Zilganersen (ION373) in Patients With Alexander Disease (AxD) | Alexander disease, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hereditary Central Nervous System Demyelinating Diseases, Leukoencephalopathies, Nutritional and Metabolic Diseases, brain disorder, central nervous system disorder, demyelinating disease, hereditary disease, inborn errors of metabolism, inherited neurodegenerative disorder, metabolic disease, nervous system disorder, neurodegenerative disease | Active, not recruiting | Phase 3 | 2026-08-21 | NCT04849741 | |
| HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple Ascending Doses of ION582 in Participants With Angelman Syndrome | Abnormalities, Multiple, Angelman syndrome, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Imprinting Disorders, central nervous system disorder, chromosomal disorder, hereditary disease, movement disorder, nervous system disorder | Active, not recruiting | Phase 1 / Phase 2 | 2026-08-20 | NCT05127226 | |
| FUSION: A Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of ION363 in Amyotrophic Lateral Sclerosis Participants With Fused in Sarcoma Mutations (FUS-ALS) | Nutritional and Metabolic Diseases, TDP-43 proteinopathy, amyotrophic lateral sclerosis, central nervous system disorder, metabolic disease, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, proteostasis deficiencies, spinal cord disorder | Active, not recruiting | Phase 3 | 2026-07-21 | NCT04768972 | |
| Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of ION464 Administered to Adults With Multiple System Atrophy (HORIZON) | autonomic nervous system disorder, basal ganglia disorder, brain disorder, central nervous system disorder, movement disorder, multiple system atrophy, nervous system disorder, neurodegenerative disease, primary dysautonomia, synucleinopathy | Recruiting | Phase 1 | 2026-04-27 | NCT04165486 | |
| An Extension Study to Assess Long-Term Safety of Eplontersen in Adults With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM) | Nutritional and Metabolic Diseases, amyloidosis, metabolic disease, proteostasis deficiencies | Enrolling by invitation | Phase 3 | 2026-04-17 | NCT05667493 | |
| Evaluation of Outcome Metrics in Alexander Disease | Alexander disease, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hereditary Central Nervous System Demyelinating Diseases, Leukoencephalopathies, Nutritional and Metabolic Diseases, brain disorder, central nervous system disorder, demyelinating disease, hereditary disease, inborn errors of metabolism, inherited neurodegenerative disorder, metabolic disease, nervous system disorder, neurodegenerative disease | Recruiting | Not stated | 2026-01-15 | NCT02714764 | |
| Orbit Study: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356 in Participants With Pelizaeus Merzbacher Disease (PMD) | Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hereditary Central Nervous System Demyelinating Diseases, Leukoencephalopathies, Nutritional and Metabolic Diseases, Pelizaeus-Merzbacher spectrum disorder, X-linked disease, brain disorder, central nervous system disorder, demyelinating disease, hereditary disease, inborn errors of metabolism, metabolic disease, nervous system disorder | Recruiting | Phase 1 | 2025-12-12 | NCT06150716 | |
| A Study to Assess the Long-Term Safety and Efficacy of Donidalorsen in the Prophylactic Treatment of Hereditary Angioedema (HAE) | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hereditary Complement Deficiency Diseases, Hypersensitivity, Immediate, Primary Immunodeficiency Diseases, Skin and Connective Tissue Diseases, angioedema, cardiovascular disorder, hereditary angioedema, hereditary disease, immune system disorder, inborn error of immunity, skin disorder, skin vascular disease, urticaria, vascular disorder | Active, not recruiting | Phase 3 | 2025-12-12 | NCT05392114 | |
| A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRX) Administered to Adults With Familial Chylomicronemia Syndrome (FCS) Previously Treated With Volanesorsen | familial chylomicronemia syndrome, familial lipoprotein lipase deficiency | Active, not recruiting | Phase 3 | 2025-12-12 | NCT05185843 | |
| A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) in Participants With Familial Chylomicronemia Syndrome (FCS) | familial chylomicronemia syndrome, familial lipoprotein lipase deficiency | Active, not recruiting | Phase 3 | 2025-12-12 | NCT05130450 | |
| A Study to Assess the Long-Term Safety and Efficacy of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in Patients With Hereditary Transthyretin-Mediated Amyloid Polyneuropathy | Hereditary Transthyretin-Mediated Amyloid Polyneuropathy | Active, not recruiting | Phase 3 | 2025-12-04 | NCT05071300 | |
| CORE-OLE: A Study of Olezarsen (ISIS 678354) Administered Subcutaneously to Participants With Severe Hypertriglyceridemia (SHTG) | Dyslipidemias, Lipid Metabolism Disorders, Nutritional and Metabolic Diseases, hyperlipidemia, hypertriglyceridemia, metabolic disease | Active, not recruiting | Phase 3 | 2025-12-01 | NCT05681351 | |
| The Myelin Disorders Biorepository Project | Abnormalities, Multiple, Aicardi-Goutieres syndrome, Alexander disease, Alexanders leukodystrophy, Allan-Herndon-Dudley syndrome, Bone Diseases, Developmental, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Canavan disease, Cerebral Small Vessel Diseases, Charcot-Marie-Tooth disease, Cockayne syndrome, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, DNA repair disease, Digestive System Diseases, Dwarfism, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, GM2 gangliosidosis, Gangliosidoses, Hereditary Central Nervous System Demyelinating Diseases, Infant, Newborn, Diseases, Infarction, Krabbe disease, Leukoencephalopathies, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Male Urogenital Diseases, Necrosis, Neurobehavioral Manifestations, Neurologic Manifestations, Nutritional and Metabolic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Pelizaeus-Merzbacher spectrum disorder, Peroxisomal Disorders, Salla disease, Sjogren syndrome, Sjogren-Larsson syndrome, Skin Abnormalities, Skin and Connective Tissue Diseases, Stomatognathic Diseases, Sulfatidosis, Urogenital Diseases, X-linked disease, Xerostomia, Zellweger spectrum disorders, adrenal gland disorder, adrenocortical insufficiency, adrenoleukodystrophy, adrenomyeloneuropathy, adult Refsum disease, adult-onset autosomal dominant demyelinating leukodystrophy, alpha-gal syndrome, arthritic joint disease, arthropathy, attention deficit hyperactivity disorder, inattentive type, autoimmune disease, bone disorder, brain disorder, brain infarction, brain ischemia, cardiovascular disorder, central nervous system disorder, central nervous system malformation, cerebral arterial disease, cerebral infarction, cerebrotendinous xanthomatosis, cerebrovascular disorder, connective tissue disorder, cutaneous mucinosis, dementia, demyelinating disease, dry eye syndrome, endocrine system disorder, eye disorder, hereditary disease, hereditary motor and sensory neuropathy, hereditary skin disorder, hypomyelination with brain stem and spinal cord involvement and leg spasticity, ichthyosis, immune system disorder, inborn carbohydrate metabolic disorder, inborn errors of metabolism, inherited neurodegenerative disorder, intellectual disability, intracranial arterial disease, ischemic disease, keratosis, kidney disorder, lacrimal apparatus disorder, leukodystrophy, leukodystrophy, hypomyelinating, 5, leukodystrophy, hypomyelinating, 6, leukodystrophy, hypomyelinating, 7, with or without oligodontia and/or hypogonadotropic hypogonadism, leukoencephalopathy with brain stem and spinal cord involvement-high lactate syndrome, leukoencephalopathy with calcifications and cysts, leukoencephalopathy with vanishing white matter 1, leukoencephalopathy, diffuse hereditary, with spheroids 1, liver disorder, lysosomal lipid storage disorder, lysosomal storage disease, megalencephalic leukoencephalopathy with subcortical cysts, metabolic disease, metachromatic leukodystrophy, mouth disorder, mucopolysaccharidosis, mucosulfatidosis, musculoskeletal system disorder, nervous system disorder, neurodegeneration with brain iron accumulation 5, neurodegenerative disease, neurodevelopmental disorder, neuromuscular disease, null syndrome, oppositional defiant disorder, peripheral nervous system disorder, polyneuropathy, psychiatric disorder, rheumatic disorder, rheumatoid arthritis, salivary gland disorder, skin disorder, sphingolipidosis, stroke disorder, urinary system disorder, vascular dementia, vascular disorder, xanthomatosis | Recruiting | Not stated | 2025-10-23 | NCT03047369 |