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.

Research Team

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).