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Ionis Pharmaceuticals Inc.
Sponsor

Ionis Pharmaceuticals Inc.

United States 2855 Gazelle Court, 92010-6670, United States

About the sponsor

Research activity includes amyotrophic lateral sclerosis with FUS mutations, Alexander disease, Angelman syndrome, Pelizaeus-Merzbacher disease, multiple system atrophy, and prion disease. The portfolio also includes studies in MECP2 duplication syndrome and Alzheimer’s disease in individuals with Down syndrome.

  • FUS-ALS
  • Angelman syndrome
  • Multiple system atrophy
  • Prion disease
  • MECP2 duplication syndrome

These programmes reflect sustained interest in rare inherited and progressive conditions affecting the central nervous system.

About Specializations

Ionis Pharmaceuticals Inc. runs clinical trials in conditions such as Familial hypertriglyceridaemia, Hyperchylomicronaemia, Hereditary angioedema, Angelman's syndrome, among others.

Recruiting now

Ongoing trials

Clinical trials run by this sponsor – those enrolling participants first.

Condition
Country
City
Type of trial
Recruitment status
Quick filters

20 clinical trials

Angelman's syndrome

Recruiting

A Phase 3 study to evaluate the efficacy and safety of ION582 in children and adults with Angelman syndrome

InvestigationalNo placebo
Countries:GermanyGermany
  • Participants:0–17 years · 18–64 years
  • Substances:2'-O-(2-Methoxyethyl) Modified Antisense Oligonucleotide Targeting Ube3A Antisense Transcript Rna
  • Sponsor:Ionis Pharmaceuticals Inc.

Hereditary angioedema

Recruiting

A study of donidalorsen for children with hereditary angioedema

InvestigationalNo placebo
Countries:ItalyItaly
  • Participants:0–17 years
  • Substances:Donidalorsen
  • Sponsor:Ionis Pharmaceuticals Inc.

Myelodysplastic syndrome

Recruiting

Study on the Safety and Effects of ION440 for Patients with MECP2 Duplication Syndrome

InvestigationalNo placebo
Countries:AustriaAustria
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:ION440
  • Sponsor:Ionis Pharmaceuticals Inc.

Hereditary neuropathic amyloidosis

Recruiting

Long-Term Safety and Efficacy Study of Eplontersen in Patients with Hereditary Transthyretin-Mediated Amyloid Polyneuropathy

InvestigationalNo placebo
Countries:CyprusCyprus
  • Participants:18–64 years · 65+ years
  • Substances:Eplontersen
  • Sponsor:Ionis Pharmaceuticals Inc.

Angelman's syndrome

Recruiting

Study on the Safety and Effects of ION582 for Patients with Angelman Syndrome

InvestigationalNo placebo
Countries:FranceFrance
  • Participants:0–17 years · 18–64 years
  • Substances:2'-O-(2-Methoxyethyl) Modified Antisense Oligonucleotide Targeting Ube3A Antisense Transcript Rna
  • Sponsor:Ionis Pharmaceuticals Inc.

Pelizaeus-Merzbacher disease

Recruiting

Study on the Safety and Effects of ION356 for Patients with Pelizaeus-Merzbacher Disease

InvestigationalNo placebo
Countries:FranceFrance
  • Participants:0–17 years
  • Substances:2'-O-(2-Methoxyethyl) Modified Antisense Oligonucleotide Targeting Plp1 Pre-Mrna
  • Sponsor:Ionis Pharmaceuticals Inc.

Familial hypertriglyceridaemia+1

Recruiting

Study of ISIS 678354 for Patients with Familial Chylomicronemia Syndrome Previously Treated with Volanesorsen

InvestigationalNo placebo
Countries:SwedenSweden
  • Participants:18–64 years · 65+ years
  • Substances:ISIS 678354 sodium salt
  • Sponsor:Ionis Pharmaceuticals Inc.

Severe myoclonic epilepsy of infancy

Not yet recruiting

Safety and Tolerability of Intrathecal ION337 in Patients with Dravet Syndrome

InvestigationalNo placebo
Countries:BelgiumBelgium
  • Participants:0–17 years
  • Sponsor:Ionis Pharmaceuticals Inc.

Familial hypertriglyceridaemia+1

Not recruiting

Study of ISIS 678354 for Patients with Severe High Triglycerides

Investigational
Countries:BelgiumBelgium
  • Participants:18–64 years · 65+ years
  • Substances:ISIS 678354 sodium salt
  • Sponsor:Ionis Pharmaceuticals Inc.

Amyotrophic lateral sclerosis

Not recruiting

Study on the Effects of ION363 for Patients with Amyotrophic Lateral Sclerosis with Fused in Sarcoma Mutations

Investigational
Countries:BelgiumBelgium
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Ulefnersen
  • Sponsor:Ionis Pharmaceuticals Inc.

Familial hypertriglyceridaemia+1

Not recruiting

Study of Olezarsen (ISIS 678354) in Patients with Severe Hypertriglyceridemia: Testing Effects on Triglyceride Levels

Investigational
Countries:BulgariaBulgaria
  • Participants:18–64 years · 65+ years
  • Substances:ISIS 678354 sodium salt
  • Sponsor:Ionis Pharmaceuticals Inc.

Hypertriglyceridaemia+1

Not recruiting

Study of Olezarsen (ISIS 678354) for Patients with High Triglycerides and Heart Disease or Severe High Triglycerides

Investigational
Countries:BulgariaBulgaria
  • Participants:18–64 years · 65+ years
  • Substances:ISIS 678354 sodium salt
  • Sponsor:Ionis Pharmaceuticals Inc.

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