ION363 for ALS

Recruiting at 27 trial locations

Overseen By Ionis Pharmaceuticals

Age: Any Age

Sex: Any

Trial Phase: Phase 3

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) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 36

95 Participants Needed

What You Need to Know Before You Apply

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.

Are You a Good Fit for This Trial?

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.

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.

Timeline for a Trial Participant

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

What Are the Treatments Tested in This Trial?

Interventions

• ION363

Trial Overview The 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).

How Is the Trial Designed?

Group I: ION363 Experimental Treatment 1 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: Placebo Placebo Group 1 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).

Who Is Running the Clinical Trial?

Ionis Pharmaceuticals, Inc.

Lead Sponsor

Trials

151

Recruited

27,800+

Dr. Brett P. Monia

Ionis Pharmaceuticals, Inc.

Chief Executive Officer since 2020

PhD in Pharmacology from the University of Pennsylvania, BSc in Molecular Biology and Analytical Chemistry from Stockton State College

Dr. Eric Bastings

Ionis Pharmaceuticals, Inc.

Chief Medical Officer

MD