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Ionis Pharmaceuticals clinical trials

Source: ClinicalTrials.gov, U.S. National Library of Medicine and the EU Clinical Trials Information System (CTIS) — current trials in our USA, UK, Ireland, India, China and EU (CTIS) registers with Ionis Pharmaceuticals as lead sponsor. Synced 26 Sep 2026
20Current trials
9Recruiting now
4Drugs in these trials
6Trial registers
Maintained by Sreepriya Prasannan
MSc Digital Transformation of Life Sciences (Innopharma Education / Griffith College); MSc & BSc Botany HSE Spark Ignite 2026 — Top 14 finalist
Compiled from ClinicalTrials.gov and CTIS registry records · Last verified · Editorial standards
At a glance

Ionis Pharmaceuticals is the lead sponsor of 20 current clinical trials in our USA, UK, Ireland, India, China and EU (CTIS) registers. 9 are recruiting patients now. The most common phase is Phase 3. The drugs named most often in its trials are Olezarsen, Donidalorsen and Eplontersen. The most-studied condition is Familial Chylomicronemia Syndrome.

ClinicalTrials.gov trials by status

  • Recruiting947%
  • Active, closed947%
  • By invitation15%

Trials by phase

  • Phase 31158%
  • Phase 1/2421%
  • Phase 1211%
  • Observational15%
  • Phase 215%
01

Clinical trials sponsored by Ionis Pharmaceuticals

19 on ClinicalTrials.gov
StudyPhaseStatusStartRegisters
Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of ION464 Administered to Adults With Multiple System Atrophy (HORIZON)
NCT04165486 · Multiple System Atrophy
Phase 1 Recruiting 2022-07 UK
Rocket Study: A Study to Characterize Biomarkers and Disease Progression in Participants With Pelizaeus-Merzbacher Disease
NCT05659901 · ClinicalTrials.gov ↗ · Pelizaeus-Merzbacher Disease
Observational Recruiting 2022-10 UK, USA
Donidalorsen Treatment in Children With Hereditary Angioedema
NCT07298447 · Hereditary Angioedema (HAE)
Phase 3 Recruiting 2026-04 USA
ASCEND: Safety and Tolerability of ION337 for the Treatment of Dravet Syndrome
NCT07531745 · Dravet Syndrome
Phase 1/2 Recruiting 2026-05 USA
REVEAL: A Phase 3 Study of Obudanersen (ION582) in Angelman Syndrome
NCT06914609 · ClinicalTrials.gov ↗ · Angelman Syndrome
Phase 3 Recruiting 2025-06 UK, USA
A Study of Olezarsen for the Treatment of Familial Chylomicronemia Syndrome (FCS) in Pediatric Participants
NCT07727538 · ClinicalTrials.gov ↗ · Familial Chylomicronemia Syndrome
Drug: Olezarsen
Phase 3 Recruiting 2026-07 USA
A Study to Assess the Safety, Tolerability and Efficacy of ION775
NCT07604974 · ClinicalTrials.gov ↗ · Hypertriglyceridemia, Severe Hypertriglyceridemia
Phase 2 Recruiting 2026-05 USA
Orbit Study: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356 in Participants With Pelizaeus Merzbacher Disease (PMD)
NCT06150716 · ClinicalTrials.gov ↗ · Pelizaeus-Merzbacher Disease
Phase 1 Recruiting 2024-04 USA
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)
NCT06430385 · ClinicalTrials.gov ↗ · Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome
Phase 1/2 Recruiting 2024-10 USA
An Extension Study to Assess Long-Term Safety of Eplontersen in Adults With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM)
NCT05667493 · Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)
Phase 3 By invitation 2022-11 UK, USA
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)
NCT04768972 · Amyotrophic Lateral Sclerosis
Phase 3 Active, closed 2021-06 Ireland, UK, USA
A Study to Evaluate the Safety and Efficacy of Zilganersen (ION373) in Patients With Alexander Disease (AxD)
NCT04849741 · Alexander Disease
Phase 3 Active, closed 2021-06 UK, USA
CORE-OLE: A Study of Olezarsen (ISIS 678354) Administered Subcutaneously to Participants With Severe Hypertriglyceridemia (SHTG)
NCT05681351 · ClinicalTrials.gov ↗ · Severe Hypertriglyceridemia
Drug: Olezarsen
Phase 3 Active, closed 2022-12 UK, USA, India
HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple Ascending Doses of ION582 in Participants With Angelman Syndrome
NCT05127226 · ClinicalTrials.gov ↗ · Angelman Syndrome
Phase 1/2 Active, closed 2021-12 UK, USA
A Study to Assess the Long-Term Safety and Efficacy of Donidalorsen in the Prophylactic Treatment of Hereditary Angioedema (HAE)
NCT05392114 · ClinicalTrials.gov ↗ · Hereditary Angioedema
Phase 3 Active, closed 2022-07 UK, USA
A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) in Participants With Familial Chylomicronemia Syndrome (FCS)
NCT05130450 · ClinicalTrials.gov ↗ · Familial Chylomicronemia Syndrome
Drug: Olezarsen
Phase 3 Active, closed 2021-11 UK, USA
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
NCT05071300 · ClinicalTrials.gov ↗ · Hereditary Transthyretin-Mediated Amyloid Polyneuropathy
Phase 3 Active, closed 2022-01 USA, China
A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRX) Administered to Adults With Familial Chylomicronemia Syndrome (FCS) Previously Treated With Volanesorsen
NCT05185843 · ClinicalTrials.gov ↗ · Familial Chylomicronemia Syndrome
Drug: Olezarsen
Phase 3 Active, closed 2022-02 USA
PrProfile: A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION717
NCT06153966 · ClinicalTrials.gov ↗ · Prion Disease
Phase 1/2 Active, closed 2024-01 USA

Registers list studies that are recruiting, about to open, active but closed to enrolment, or enrolling by invitation. The UK and USA registers cover industry-sponsored studies only. Titles and statuses are copied from the registry as published.

02

Ionis Pharmaceuticals in the EU trials register (CTIS)

1 trial

Authorised or ongoing trials in the EU Clinical Trials Information System with Ionis Pharmaceuticals as sponsor. Some are the same study as a ClinicalTrials.gov record above; the two registries share no common ID. Trials with a site in Ireland are listed first.

StudyPhaseStatusCountries
Phase 1-2, Open-Label, Single and Multiple Ascending Dose Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intrathecally-Administered ION337 in Patients with Dravet Syndrome
2025-523835-20-00 · CTIS ↗ · Nervous System Diseases
Phase 1 Authorised Spain, France, Belgium + 2 more
03

Drugs in Ionis Pharmaceuticals trials

4 compounds

Drugs and biologicals named as interventions in these trials, with the number of this sponsor's trials that name each one. Placebos and generic comparators are excluded.

04

Conditions in Ionis Pharmaceuticals trials

15 conditions
Familial Chylomicronemia Syndrome3Pelizaeus-Merzbacher Disease2Angelman Syndrome2Severe Hypertriglyceridemia2Amyotrophic Lateral Sclerosis1Multiple System Atrophy1Alexander Disease1Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)1Hereditary Angioedema1Hereditary Angioedema (HAE)1Dravet Syndrome1Hereditary Transthyretin-Mediated Amyloid Polyneuropathy1Hypertriglyceridemia1Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome1Prion Disease1
05

By trial register

RegisterTrialsRecruiting
Clinical trials in USA188
Clinical trials in UK103
Clinical trials in Ireland10
Clinical trials in India10
Clinical trials in China10
EU register (CTIS)1—

A trial with sites in more than one country appears in each of those registers.

06

Questions about Ionis Pharmaceuticals trials

How many clinical trials does Ionis Pharmaceuticals sponsor?

Ionis Pharmaceuticals is the lead sponsor of 19 current trials on ClinicalTrials.gov in our USA, UK, Ireland, India and China registers, and 1 authorised or ongoing trial in the EU register (CTIS), as of the registry sync on 26 September 2026. Completed and withdrawn studies are not counted.

Are Ionis Pharmaceuticals trials recruiting?

Yes. 9 of its 19 ClinicalTrials.gov trials are recruiting. Eligibility and site contacts are on each trial's record. Only the study team can confirm whether a patient is eligible.

Which drugs is Ionis Pharmaceuticals testing?

Its current trials name 4 drugs and biologicals as interventions. The most frequent are Olezarsen (4), Donidalorsen (2), Eplontersen (2) and Obudanersen (2). The number in brackets is how many of its trials name each drug.

Does Ionis Pharmaceuticals have clinical trials in Ireland?

Yes. 1 trial in our Ireland register (ClinicalTrials.gov). Some may be the same study registered in both. See the Irish clinical trials register for sites and contacts.

Where does this information come from?

Every trial, status, phase and condition on this page is copied from public ClinicalTrials.gov and EU CTIS records by a daily sync, grouped by the sponsor name each registry records. It says nothing about the organisation beyond its registered trials, and it is not medical advice.

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How this page is built

Everything on this page is copied from public registry records — it is not written from memory, and nothing here describes the organisation beyond its registered trials. Each trial links to its own record so you can check it yourself.

This page is built automatically: a daily sync job reads the registry records behind our trial registers and groups every current trial by its lead sponsor name, ignoring legal-form suffixes such as Inc. or Ltd. A small, hand-checked list joins the same organisation's different registry names (for example its EU legal entity). There is no AI drafting and no per-sponsor editorial pass. The “last verified” date above is the timestamp of that sync, not a review date. Our general editorial approach is described in our editorial standards and corrections policy. The the registries data on this page was last retrieved on 26 Sep 2026.

Please verify before you rely on this. This page is general information for life-science and pharmaceutical professionals. It is not medical advice, and it has not been reviewed by a clinician — our editorial team holds life-science qualifications, not clinical ones. It is not exhaustive and may not reflect the most recent label change. Always check the official prescribing information (US Prescribing Information or EU SmPC) and speak to your doctor or pharmacist before acting on anything here. Drugs in the same class are not automatically interchangeable, and approvals, brand names and indications differ between the US, the EU/Ireland (EMA/HPRA) and other regions. Spotted an error? Tell us — we correct promptly and log it.