In short
Clinical trials are studying ZOREVUNERSEN SODIUM in people with Dravet syndrome. These studies aim to evaluate safety, tolerability, and whether the treatment can reduce major motor seizures. The main trial listed here includes patients with this rare seizure disorder.
Key points
- ZOREVUNERSEN SODIUM is being studied in clinical trials for Dravet syndrome, a rare seizure disorder. The listed study is a Phase 3, double-blind, interventional trial with 185 planned participants. It is designed to compare ZOREVUNERSEN SODIUM with sham treatment and measure changes in major motor seizure frequency. The main outcome is the percent change from baseline in major motor seizures from Week 16 to Week 28. The trial also looks at safety, efficacy, and tolerability in patients with Dravet syndrome.
Clinical trial overview
The listed study is a Phase 3 clinical trial of ZOREVUNERSEN SODIUM in patients with Dravet syndrome, a rare seizure disorder. The trial is designed to assess efficacy, safety, and tolerability while looking at whether the treatment can reduce major motor seizures.
Study design and treatment groups
This is an interventional study, which means the researchers give a treatment and measure the results. The study is described as double-blind, so the source indicates a design meant to reduce bias in the results. The trial compares ZOREVUNERSEN SODIUM with sham treatment, which is an inactive comparison used to help show whether the study drug has a real effect.
The trial lists two study doses: 45 mg and 70 mg, both given by intrathecal use, meaning treatment is delivered into the fluid around the spinal cord.
Who can join the study
The source data says the trial is for patients with Dravet syndrome. It does not provide more details about age limits, seizure history rules, or other entry criteria, so those parts cannot be confirmed from the source.
What the trial measures
The main endpoint is the percent change from baseline in major motor seizure frequency. Baseline means the starting point before treatment begins. The study measures seizures per 28 days in patients receiving ZOREVUNERSEN SODIUM compared with sham from Week 16 to Week 28, using three 4-week periods.
The trial focuses on major motor seizures, which the source defines as hemiclonic, focal with motor signs, focal to bilateral tonic-clonic, generalized tonic-clonic, tonic, tonic/atonic, and bilateral clonic seizures. In simple terms, these are seizure types that involve strong body movement and may cause falls or whole-body shaking.
Trial status and size
The trial status is listed as Authorised. The planned enrollment is 185 participants, which means the study expects to include 185 people. This size suggests the trial is meant to collect enough data to better judge how the treatment performs in a larger group.
Patient-friendly terms
Efficacy means how well the treatment works. Safety means whether the treatment causes harmful effects. Tolerability means how well patients can receive the treatment without too much trouble or discomfort.
Sham treatment is used as a comparison and does not contain the active study drug. Double-blind means the people in the study and the study team do not know who is getting which treatment, which helps make the results more reliable.