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Our clinical studies span a variety of medical conditions. Please see below and select an option to view the currently enrolling trials for that condition.

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Therapeutic Conditions

Cardio-Renal

Neurological

Cancer

Cardio-Renal

Condition

Status

Phase

Primary IgA Nephropathy

A Study to Evaluate the Effectiveness and Safety of IONIS-FB-LRx, an Antisense Inhibitor of Complement Factor B, in Adult Participants With Primary IgA Nephropathy
NCT04014335
ISIS 696844-CS4
The purpose of this study is to evaluate the effectiveness and safety of IONIS-FB-LRx, an antisense inhibitor of complement factor B messenger ribonucleic acid (CFB mRNA), and to evaluate the effect of IONIS-FB-LRx on plasma factor B (FB) levels and serum AH50, CH50 activity in participants with primary immunoglobulin A (IgA) nephropathy.
Phase 2
IONIS-FB-LRx
This is a Phase 2, single arm open-label clinical study in up to 10 participants that will consist of screening period (which may include a titration of maximum dose/maximally tolerated dose of angiotensin converting enzyme (ACE) and/or angiotensin II receptor blocker (ARB)), a 24-week treatment period and a 12-week post-treatment follow-up evaluation period.

Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)

CARDIO-TTRansform: A Study to Evaluate the Efficacy and Safety of AKCEA-TTR-LRx in Participants With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)
NCT04136171
ION-682884-CS2
To evaluate the efficacy of eplontersen compared to placebo in participants with ATTR-CM receiving available standard of care (SoC). For more information, please visit https://www.cardio-ttransform.com.
Phase 3
Eplontersen
This is a multicenter, double-blind study in up to 750 participants, who will be randomized to receive subcutaneous (SC) injections of either AKCEA-TTR-LRx or placebo once every 4 weeks. Participants will also receive daily supplemental doses of the recommended daily allowance of vitamin A.

Hereditary Angioedema

OASIS-HAE: A Study to Evaluate the Safety and Efficacy of Donidalorsen (ISIS 721744 or IONIS-PKK-LRx) in Participants With Hereditary Angioedema (HAE)
NCT05139810
ISIS 721744-CS5
The purpose of this study is to evaluate the safety and efficacy of donidalorsen in participants with HAE and effect of donidalorsen on the quality and pattern of HAE attacks and their impact on quality of life (QoL).
Phase 3
Donidalorsen: Cohort A
This is a Phase 3, multi-center, double-blind, randomized, placebo-controlled study of donidalorsenin up to 84 participants. Participants will be randomized in a 2:1 ratio to Cohort A (donidalorsenor placebo every 4 weeks) or Cohort B (donidalorsenor placebo every 8 weeks), respectively. Within each Cohort, participants will be randomized in a 3:1 ratio to receive donidalorsenor matching-placebo. The length of participation in the study is approximately 11 months, which includes an up to 8-week Screening Period, a 25-week Treatment Period, and an up to 13-week Post-treatment Period.

Neurological

Condition

Status

Phase

Parkinson's Disease

A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of BIIB094 in Adults With Parkinson’s Disease
NCT03976349
254PD101
The primary objective of this study is to evaluate the safety and tolerability of single and multiple doses of BIIB094 administered via intrathecal (IT) injection to participants with Parkinson's Disease (PD). The secondary objective of this study is to evaluate the pharmacokinetic (PK) profile of BIIB094.The study is open for PD patients with verified presence or absence of variations in the leucine-rich repeated kinase 2 (LRRK2) gene, but also for patients without any verified PD-related genetic variant.
Phase 1
Part A (SAD): BIIB094 Dose 1

Amyotrophic Lateral Sclerosis

Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of ION363 in Amyotrophic Lateral Sclerosis Participants With Fused in Sarcoma Mutations (FUS-ALS)
NCT04768972
ION363-CS1
The primary purpose of this study is to evaluate the clinical efficacy of ION363 on clinical function and survival in carriers of fused in sarcoma mutations with amyotrophic lateral sclerosis (FUS-ALS).
Phase 3
ION363
This is a multi-center, two-part study of ION363 in up to 64 participants. Part 1 will consist of participants that will be randomized in a 2:1 ratio to receive a multi-dose regimen of ION363 or placebo for a period of 29 weeks, followed by Part 2, which will be an open-label period where all participants will receive ION363 for a period of 73 weeks.

Alexander Disease

A Study to Evaluate the Safety and Efficacy of ION373 in Patients With Alexander Disease (AxD)
NCT04849741
ION373-CS1
The purpose of this study is to evaluate the safety and efficacy of ION373 in improving or stabilizing gross motor function across the full range of affected domains in patients with AxD.
Phase 3
ION373
This is a Phase 1-3, multi-center, double-blind, placebo-controlled, multiple-ascending dose (MAD) study in up to 58 patients with AxD. Participants will be randomized in a 2:1 ratio to receive ION373 or matching placebo for a 60-week double-blind treatment period; then all participants will receive ION373 for a 60-week open-label treatment period.

Angelman Syndrome

HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple Ascending Doses of ION582 in Participants With Angelman Syndrome
NCT05127226
ION582-CS1
The purpose of this study is to evaluate the safety and tolerability of ascending doses of ION582 administered intrathecally in participants with Angelman syndrome.
Phase 1
Part 1 MAD: Cohort A
This is a Phase 1-2a, open-label dose-escalation study of ION582 enrolling up to approximately 44 participants. Following a screening period of up to 4 weeks, eligible participants will receive intrathecal (IT) injections of ION582. Participants will be followed for up to 32 weeks after dosing.

Cancer

Condition

Status

Phase

Advanced Solid Tumors

A Study of ION537 in Patients With Molecularly Selected Advanced Solid Tumors
NCT04659096
ION537-CS1
The purpose of this study is to evaluate the safety and establish the maximum tolerated dose (MTD) and recommended Phase 2 dose (RP2D) of ION537 when administered by intravenous infusion in patients with advanced solid tumors.
Phase 1
ION537
This is a single center, open label, non-randomized, Phase 1, two-part study of ION537 in up to 102 participants. Part 1 of the study consists of sequential cohort, dose escalation in patients with advanced solid tumors. Part 2 is dose expansion in patients with molecularly selected advanced solid tumors. In total, the study includes up to 102 participants. The study will consist of a 30-day screening period, a treatment period consisting of sequential consecutive treatment cycles (each cycle will be of 28 days) and a post-treatment follow-up period of at least 28 days following the last dose of study drug.

Relapsed Multiple Myeloma, Refractory Multiple Myeloma

A Study of ION251 Administered to Patients With Relapsed/Refractory Multiple Myeloma
NCT04398485
ION251-CS1
The purpose of this study is to determine the maximum-tolerated dose (MTD) and recommended Phase 2 dose (RP2D) of ION251 in patients with relapsed/refractory multiple myeloma.
Phase 1
ION251
This is a two-part, multi-center first in human study of ION251 in up to 80 participants. Part 1 will use a 3+3 dose-escalation scheme in sequential cohorts to determine the MTD and RP2D during repeated 28-day treatment cycles. MTD will be determined by the number of participants with AEs meeting the dose-limiting toxicity (DLT) criteria during Cycle 1. The MTD determined in Part 1 will be used with other variables to inform a RP2D for participants proceeding to Part 2 for further assessments in the safety, tolerability and anti-myeloma activity.