Phase 1-2, Open-Label, Single and Multiple Ascending Dose Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intrathecally-Administered ION337 in Patients with Dravet Syndrome
Dravet syndrome is a rare and severe form of epilepsy that begins in early childhood. It causes frequent, difficult-to-control seizures and other developmental challenges. This study is testing a new investigational drug called ION337. It is a Phase 1 and 2 trial, meaning it is the first time this medicine is being given to people. The main goal is to find out if ION337 is safe and well tolerated. Because it is given directly into the fluid around the spine, researchers will closely track any side effects. They will also measure how much drug stays in the blood and spinal fluid, and check if it helps lower the frequency of serious motor seizures. Taking part involves hospital visits, regular health checks, and careful monitoring by a specialist team.
At a glance
What is this study about?
Dravet syndrome is a lifelong, severe type of epilepsy that typically begins in a child's first year of life. People with this condition experience frequent, prolonged seizures that are often very hard to control with standard epilepsy medicines. Alongside seizures, it can cause problems with movement, speech, and learning. Finding new treatments that address the root cause of the condition is vital for families living with this condition.
This clinical study is looking at a new experimental medicine named ION337. The medicine is designed to act inside the nervous system. Because it needs to reach the brain, it is given as an injection directly into the spinal fluid, a procedure known as an intrathecal injection. The trial is 'open-label', which means both the doctors and the families will know that the patient is receiving the study drug, rather than a dummy treatment (placebo).
Because this is the first time ION337 is being tested in humans, doctors are moving forward very carefully. The study will test gradually increasing doses, starting with a single dose in the first group of patients, followed by repeated doses in a second group. Researchers want to understand the safest dose and see whether the medicine helps reduce the number of major seizures over time.
Key takeaways
- Tests a new medicine, ION337, for people diagnosed with Dravet syndrome.
- This is an early-stage study checking safety, dosage, and side effects.
- The medicine is delivered through an injection into the lower back (spinal fluid).
- Doctors will measure changes in the number of major motor seizures.
- Participation is entirely voluntary and includes close, expert medical monitoring.
Who may be eligible?
This trial is designed for individuals who have been diagnosed with Dravet syndrome. Both males and females of any age may be considered, but final inclusion depends on specific medical criteria set by the research team.
Because this is an early safety study, participants must be well enough to undergo spinal injections and routine hospital tests. The medical team will check each person's general health, heart function, and medical history before they can join.
People may not be able to take part if they have certain other severe medical conditions, current infections, or problems that make spinal injections unsafe. The study doctors will review every candidate's medications to ensure there are no unsafe drug interactions.
Could this study suit you?
Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.
- Confirmed diagnosis of Dravet syndrome
- Experiences regular major motor seizures
- Able to undergo an injection into the spinal fluid
- Willing to attend regular hospital visits and health checks
- Able and willing to keep a daily seizure diary
What does participation involve?
Taking part in this study involves receiving the study medicine, ION337, through a small needle placed into the fluid around the lower spine (similar to a lumbar puncture). In the first part of the study, participants receive a single dose. In the second part, participants receive multiple doses given over a planned schedule.
Participants will need to visit the hospital regularly for health checks. These visits will include blood and urine tests, physical and neurological examinations, heart traces (ECGs), and checks on mood and wellbeing. Doctors will also collect small samples of spinal fluid and blood to see how the body processes the drug.
Families will be asked to keep a daily seizure diary to record any motor seizures. The total study duration spans several months, including a preparation period before the first dose, the treatment phase, and a follow-up period to ensure the participant remains safe and well.
Potential risks and benefits
Locations (5)
- —UnverifiedSpain
- —UnverifiedFrance
- —UnverifiedBelgium
- —UnverifiedPortugal
- —UnverifiedGermany
Common questions
What does 'first administration to humans' mean?
It means this is the very first time the medicine is being tested in people. Before this, it was only tested in laboratory studies.
Will my child or I definitely receive the real medicine?
Yes. This is an open-label study, meaning there is no placebo (dummy medicine), so every participant receives ION337.
How is the medicine given?
It is given as an injection into the fluid around the spinal cord in the lower back, using a thin needle. This is done by a trained specialist doctor.
Can we stop taking part if we change our mind?
Yes. You can leave the trial at any point, for any reason, and your regular medical care will continue as normal.
Does taking part cost anything?
No. Clinical trial medications and all trial-related tests are provided free of charge, and travel expenses are often reimbursed.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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