Only 21 spots left

~21 spots leftby Jun 2026

ION363 for ALS

Recruiting in Palo Alto (17 mi)

+27 other locations

Age: Any Age

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Recruiting

Sponsor: Ionis Pharmaceuticals, Inc.

Must be taking: Edaravone, Riluzole, Relyvrio, others

Must not be taking: Antiplatelets, Anticoagulants, others

Disqualifiers: Ventilation, HIV, Hepatitis, others

Pivotal Trial (Near Approval)

Prior Safety Data

Trial Summary

What is the purpose of this trial?

This trial is testing a new drug called ION363 to help people with a specific genetic form of ALS (FUS-ALS). The study will see if the drug can help these patients live longer and maintain their abilities better. Participants will receive the drug for a period of time, then all will get the drug for an extended duration.

Will I have to stop taking my current medications?

The trial requires that participants taking edaravone, riluzole, Relyvrio, sodium phenylbutyrate, or tauroursodeoxycholic acid must be on a stable dose for at least 28 days before starting the study and continue on that dose during the study, unless a doctor advises otherwise. For other medications, they should be stable for at least 1 month before the study, but changes may be allowed with approval.

Eligibility Criteria

This trial is for ALS patients with FUS mutations. Adults over 30 and children aged 12-65 can join if they meet certain disease progression rates and breathing capacity criteria. Participants must have stable medication use, a caregiver to report on their condition, and no recent participation in other trials or treatments like gene therapy.

Inclusion Criteria

I am 12-65 years old with ALS symptoms, and if I'm 30-65, my ALS is progressing quickly.

I am over 30, show ALS symptoms, and my ALS condition is progressing slowly.

I have been on a stable dose of edaravone and/or riluzole for at least 28 days and can continue it during the study.

See 4 more

Exclusion Criteria

I have never had gene therapy, cell transplantation, or experimental brain surgery.

I haven't taken any experimental drugs or used new medical devices recently.

My blood pressure is not higher than 160/100 mm Hg.

See 10 more

Trial Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment (Part 1)

Participants receive a multi-dose regimen of ION363 or placebo for 60 weeks

60 weeks

Every 12 weeks, with an additional loading dose at 4 weeks

Open-label Extension (Part 2)

Participants receive open-label ION363 for 84 weeks

84 weeks

Every 12 weeks, with an additional loading dose at 4 weeks

Long-term Extension (Part 3)

Participants may continue to receive open-label ION363 for up to 3 additional years or until ION363 becomes commercially available

Up to 3 years

Every 12 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

ION363 (Antisense Oligonucleotide)

Trial OverviewThe study tests ION363's effect on ALS symptoms and survival compared to a placebo. It aims to understand how the drug works in the body (pharmacokinetics) and its impact on the disease process (pharmacodynamics).

Participant Groups

2Treatment groups

Experimental Treatment

Placebo Group

Group I: ION363Experimental Treatment1 Intervention

ION363 will be administered by lumbar intrathecal (IT) bolus injection every 12 weeks, with an additional loading dose at 4 weeks, over a 60-week double-blind treatment period in Part 1; every 12 weeks for 84 weeks in the open-label extension treatment period (Part 2), with an additional loading dose administered 4 weeks after the first dose. Patients may continue to receive open-label ION363 every 12 weeks in Part 3 for up to 3 additional years or until ION363 becomes commercially available in the patient's country or until the Sponsor discontinues the development program, whichever occurs earlier.

Group II: PlaceboPlacebo Group1 Intervention

Placebo will be administered by lumbar IT bolus injection every12 weeks, with an additional loading dose at 4 weeks, over a 60-week double-blind treatment period (Part 1).