ION337 for Dravet Syndrome

Recruiting at 1 trial location

Overseen By Ionis Pharmaceuticals, Inc.

Age: < 18

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Ionis Pharmaceuticals, Inc.

Must be taking: Anti-seizure medications

Must not be taking: Sodium channel blockers

Disqualifiers: Brain disease, Gene therapy, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

32 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores a new treatment called ION337 for children with Dravet syndrome, a severe form of epilepsy. The primary aim is to assess the safety and tolerability of this treatment. The study consists of two parts: initially testing different single doses, followed by administering multiple doses to participants who continue in the trial. Families with children diagnosed with Dravet syndrome and experiencing frequent major seizures may consider this trial. As a Phase 1/Phase 2 trial, the research focuses on understanding how the treatment works in people and measuring its effectiveness in an initial, smaller group, offering a chance to be among the first to benefit from this new treatment.

Is there any evidence suggesting that ION337 is likely to be safe for humans?

Researchers are conducting studies to determine if ION337 is safe and well-tolerated for people with Dravet syndrome, a severe form of epilepsy. This research is in the early stages, so data remains limited. However, reaching this stage indicates promise in lab and animal studies, allowing human testing. The treatment targets a specific gene linked to Dravet syndrome. Since this approach has limited testing in humans, monitoring for side effects and the body's response is crucial. Safety remains a top priority, and researchers will closely observe for any adverse effects. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for Dravet syndrome?

Unlike the standard treatments for Dravet Syndrome, which often include antiepileptic drugs like valproate and clobazam, ION337 offers a new approach by being directly administered into the spinal fluid through an intrathecal bolus injection. This method allows for potentially more effective delivery directly to the central nervous system. Researchers are excited about ION337 because it may offer a more targeted action on the neural pathways involved in this condition, potentially leading to better seizure control and fewer side effects compared to oral medications. The treatment's design to work at multiple dose levels also allows for personalized treatment adjustments, which could optimize effectiveness for each individual patient.

What evidence suggests that ION337 might be an effective treatment for Dravet syndrome?

Studies have shown that ION337 is designed to help manage Dravet syndrome, a severe form of epilepsy. It boosts the activity of the SCN1A gene, which often functions poorly in individuals with this condition. Early research suggests that this treatment can reduce the number of seizures patients experience. Specifically, some children and teenagers who received ION337 not only had fewer seizures but also improved their communication skills. These findings offer hope that ION337 could be an effective option for treating Dravet syndrome. ^{1 2 3 6 7}

Are You a Good Fit for This Trial?

Inclusion Criteria

I am between 2 and 12 years old.

I have been taking at least one anti-seizure medication at a stable dose for 4 weeks.

I meet a main requirement for this study.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Single Ascending Dose (SAD)

Participants receive a single intrathecal bolus injection of ION337 at ascending dose levels over 6 months

6 months

Multiple Ascending Dose (MAD)

Participants who complete Part 1 receive multiple doses of ION337 over 24 months

24 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

7 months

What Are the Treatments Tested in This Trial?

Interventions

• ION337

How Is the Trial Designed?

Group I: Part 2: Multiple Ascending Dose (MAD): Dose Level 1-4 Experimental Treatment 1 Intervention

Only participants who complete Part 1 will be eligible to participate in Part 2. Participants will receive multiple doses of ION337. Participants will begin treatment at the same dose level assigned in Part 1.

Group II: Part 1: Single Ascending Dose (SAD): Dose Level 1 Experimental Treatment 1 Intervention

Participants aged 2 to ≤ 12 years will receive a single intrathecal bolus (ITB) injection of ION337.

Group III: Part 1: SAD: Dose Level 4 Experimental Treatment 1 Intervention

Participants aged 2 to ≤ 12 will receive a single dose of ION337.

Group IV: Part 1: SAD: Dose Level 3 Experimental Treatment 1 Intervention

Participants aged 2 to ≤ 12 will receive a single dose of ION337.

Group V: Part 1: SAD: Dose Level 2 Experimental Treatment 1 Intervention

Participants aged 2 to ≤ 12 will receive a single dose of ION337.

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

Citations

1.

clinicaltrials.gov

clinicaltrials.gov/study/NCT07531745

ASCEND: Safety and Tolerability of ION337 for the ...

The primary purpose of this study is to evaluate the safety and tolerability of ION337 in participants with Dravet syndrome (DS). Detailed Description.

2.

investor.stoketherapeutics.com

investor.stoketherapeutics.com/news-releases/news-release-details/stoke-therapeutics-and-biogen-present-data-further-support/

Press Release Details

Compared with the general epilepsy population, people living with Dravet syndrome have a higher risk of sudden unexpected death in epilepsy, or ...

3.

ctv.veeva.com

ctv.veeva.com/study/ascend-safety-and-tolerability-of-ion337-for-the-treatment-of-dravet-syndrome

Safety and Tolerability of ION337 for the Treatment of Dravet ...

The primary purpose of this study is to evaluate the safety and tolerability of ION337 in participants with Dravet syndrome (DS).

4.

ir.ionis.com

ir.ionis.com/news-releases/news-release-details/ionis-showcases-transformational-medicines-and-industry-leading

Ionis showcases transformational medicines and industry ...

Ionis will share its next expected clinical-stage wholly owned neurology medicine, ION337 for Dravet syndrome, a serious, rare, genetic form of epilepsy with no ...

5.

facebook.com

facebook.com/DravetSyndromeFoundation/posts/were-excited-to-share-that-ionis-pharmaceuticals-has-announced-a-new-investigati/1252737953555186/

🧬We're excited to share that Ionis Pharmaceuticals has ...

Children and adolescents receiving the treatment not only had fewer seizures, but also showed significant improvements in expressive and ...

6.

dravetfoundation.org

dravetfoundation.org/dsf-funded-research/treatment-pipeline/

Current Clinical Trials & Treatment Pipeline

Investigational Medicine: ION337. Study Phase: Phase 1/2. Study Goal: To evaluate the safety and tolerability of ION337 in individuals with Dravet syndrome.

7.

adisinsight.springer.com

adisinsight.springer.com/drugs/800084943

ION 337 - AdisInsight

ION 337 is an antisense oligonucleotide therapy designed to upregulate SCN1A, being developed by Ionis Pharmaceuticals for the treatment of Dravet syndrome.