Study of zorevunersen to reduce seizures in patients with Dravet syndrome

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What is this study about?

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.

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)

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

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

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

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

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 Study?

  • 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 Study?

  • Age below 2 years old
  • Lack of confirmed genetic diagnosis of Dravet syndrome
  • 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

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Other Sites

Site Name City Country Status
ASST Fatebenefratelli Sacco Milan Italy
IRCCS Istituto Giannina Gaslini Genoa Italy
Azienda Ospedaliera Universitaria Meyer IRCCS Florence Italy
Hospital Ruber Internacional Madrid Spain
Queen Silvia Childrens Hospital – Sahlgrenska University Hospital – Vaestra Goetalandsregionen Gothenburg Sweden
Instytut Centrum Zdrowia Matki Polki Lodz Poland
Gesellschaft Fuer Epilepsieforschung Bielefeld Germany
Region Skane Skanes Universitetssjukhus Lund Sweden
Charite Universitaetsmedizin Berlin KöR Berlin Germany
Karolinska University Hospital Solna Sweden
Ludwig Maximilian University Of Munich Munich Germany
Rheinische Friedrich-Wilhelms-Universitaet Bonn Bonn Germany
Medical Center – University Of Freiburg Freiburg Im Breisgau Germany
Uniwersytecki Szpital Kliniczny W Poznaniu Poznan Poland
Centre Hospitalier Lyon Sud Pierre Benite France
Hopital Beaujon Clichy France
Ospedale Pediatrico Bambino Gesu’ Rome Italy
Acbefyerch Pgkhoeun Hpdxbaux Dq Mfagwigji Marseille France
Cxjesg Hsbeftpthst Rpilvblu Udavfrmqxiurc Da Tfill Tours France
Gllbxi Urjcpvrhqe Fngrpmlku Frankfurt Germany
Uxuebwzwmziltr Cqawtmj Kmbfuzjku Gdansk Poland
Huuqifau Dc Ls Saqpy Cpuc I Sqia Phj Barcelona Spain
Haawnvbc Ukakqeidsgdsys Stcvalhbjn &dfajnq Hhkuaai dt Hnyzqghzdva STRASBOURG, Alsace France

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
France France
Recruiting
01.04.2026
Germany Germany
Recruiting
01.04.2026
Italy Italy
Recruiting
01.04.2026
Poland Poland
Not recruiting
01.04.2026
Spain Spain
Recruiting
01.04.2026
Sweden Sweden
Not recruiting
01.04.2026

Trial locations

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.

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.

Trial ID:
2024-519555-28-00
Protocol code:
STK-001-DS-301
NCT ID:
NCT06872125
Trial Phase:
Therapeutic confirmatory (Phase III)

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