Not recruitingRare disease

Study of zorevunersen to reduce seizures in patients with Dravet syndrome

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial focuses on Dravet syndrome, a rare form of epilepsy that begins in early childhood and causes frequent, severe seizures. The study tests a new medication called zorevunersen (also known as STK-001), which is given as an injection into the spinal fluid. The purpose of the study is to determine if this medication can reduce the frequency of major seizures in people with Dravet syndrome.

The study examines how well zorevunersen works in controlling different types of seizures, including hemiclonic seizures, tonic seizures, tonic-clonic seizures, and other types of major motor seizures that can cause falling. The medication will be compared to a sham procedure to determine its effectiveness. During the study, participants will continue taking their regular anti-seizure medications while receiving either zorevunersen or the sham treatment.

The treatment period lasts for 52 weeks, during which participants will receive injections of either zorevunersen or sham treatment. The study will measure how much the frequency of seizures changes over time, particularly between weeks 16 and 28 of treatment. The researchers will also assess changes in the participants' daily living skills and overall functioning throughout the study period.

The research process

The trial runs in 6 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Initial observation period
A 6-week observation period will be conducted to monitor your current seizure frequency
During this time, you will need to maintain stable doses of your current anti-seizure medications (ASMs)
Step 2
Baseline period
Your current treatments must remain stable during this period
This includes anti-seizure medications, ketogenic diet, or vagus nerve stimulation (VNS) if applicable
Any marijuana or cannabinoid-based products must also remain unchanged
Step 3
Treatment initiation
You will receive either zorevunersen (STK-001) or a sham treatment
The medication will be administered through intrathecal injection (into the spinal fluid)
You will continue your current anti-seizure medications during the study
Step 4
Initial assessment period
From week 16 to week 28, your major motor seizure frequency will be monitored
Major motor seizures include: hemiclonic, focal with motor signs, focal to bilateral tonic-clonic, generalized tonic-clonic, tonic, and tonic/atonic seizures
Step 5
Extended assessment period
Monitoring will continue through week 52
Changes in seizure frequency will be tracked
Your daily functioning will be evaluated using the Vineland-3 assessment tool
Step 6
Study completion
The study will conclude after 52 weeks of treatment
Final assessments will measure changes in seizure frequency and daily functioning

Who can join the trial?

9 criteria

Patient or legal representative must be able to give informed consent for study participation
Patient and caregiver must be willing to follow all study requirements
Patient must be between 2 and 18 years old when signing consent
Patient must have a clinical diagnosis of Dravet syndrome confirmed by ESCI with: - Seizures starting before 12 months of age - Normal development when seizures first started - No other known cause for symptoms
Patient must have a specific genetic change (variant) in the SCN1A gene
Patient must have a required number of major motor seizures during the 6-week observation period
Patient must have tried at least 2 previous treatments for seizures, which can include: - Anti-seizure medications - Ketogenic diet (special high-fat diet) - Vagus nerve stimulation (device that helps control seizures)
Patient must be currently taking at least one anti-seizure medication
All current treatments must have been stable, including: - Regular anti-seizure medications - Ketogenic diet - Vagus nerve stimulation - Any marijuana or cannabinoid products

Who cannot join the trial?

14 criteria

Age below 2 years old
Lack of confirmed genetic diagnosis of Dravet syndrome
close
Not experiencing major motor seizures (these are seizures that include:
- hemiclonic seizures (affecting one side of the body)
- focal seizures with motor signs (seizures starting in one area with movement symptoms)
- tonic-clonic seizures (involving muscle stiffening and jerking)
- tonic seizures (involving muscle stiffening)
- drop attacks (sudden falls)
- Current participation in other clinical trials
- History of severe allergic reactions to similar medications
- Severe liver or kidney problems
- Pregnancy or breastfeeding
- Unable to follow study procedures or attend required visits
- Taking medications that could interfere with the study drug
- Having any medical condition that could make participation unsafe

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Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Verified
Assistance Publique Hopitaux De Marseille
Marseille, France
Responsive
Hospital Ruber Internacional
Madrid, Spain
Responsive
Ludwig Maximilian University Of Munich
Munich, Germany

Trial locations

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Investigated drugs

Zorevunersen (also known as STK-001) is a medication being studied for treating patients with Dravet syndrome, a rare and severe form of epilepsy. This medication is designed to reduce the frequency of major motor seizures, which include different types of seizures that affect body movement and muscle control. It works differently from traditional anti-seizure medications and is administered to help patients who experience frequent seizures. A sham procedure (similar to a placebo but for procedures rather than medications) is used as a control in this study to compare with the active treatment.

ZOREVUNERSEN SODIUM

What is already known about the treatment

Zorevunersen – An investigational antisense oligonucleotide medication administered through intrathecal injection (directly into the spinal fluid) being studied for the treatment of Dravet syndrome, a rare and severe form of epilepsy. The drug works at the molecular level by targeting and modulating the SCN1A gene expression, which is typically affected in Dravet syndrome patients, aiming to restore normal sodium channel function in brain cells. Currently undergoing Phase 3 clinical trials, zorevunersen (also known as STK-001) is being evaluated for its ability to reduce the frequency of major motor seizures, including various types such as tonic-clonic, hemiclonic, and focal seizures, representing a potential breakthrough in treating this challenging neurological condition.

Investigated diseases

Dravet syndrome - A rare genetic epileptic disorder that begins in the first year of life with frequent and prolonged seizures. The condition causes different types of seizures, including major motor seizures that can affect one side of the body or the entire body. Children with Dravet syndrome typically experience normal development in their first year, but then begin to show developmental delays. The condition is characterized by various seizure types, including hemiclonic, tonic-clonic, and focal seizures, which can occur multiple times per day. Dravet syndrome affects multiple aspects of a child's development, including mobility, language, and social skills.