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EMPEROR: A Double-blind Study Evaluating the Efficacy, Safety, and Tolerability of Zorevunersen (STK-001) in Patients with Dravet Syndrome
EU CTIS ID: 2024-519555-28-00
What this study is testing
To assess the effect of zorevunersen to reduce the frequency of major motor seizures in patients with DS. Major motor seizures are defined as the following seizure types: hemiclonic, focal with motor signs, focal to bilateral tonic-clonic, generalized tonic-clonic, tonic, tonic/atonic (drop attacks with fall or risk of fall), and bilateral clonic.
- Therapeutic confirmatory (Phase III)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Patient and/or authorized representative must be willing and able to give informed consent/assent and any authorizations required by local law for participation in the study.
- Patient and their caregiver must be willing and able (in the Investigator’s opinion) to comply with all protocol requirements.
- At signing of consent/assent, patient must be ≥2 and <18 years of age.
- Patient must have a clinical diagnosis of DS confirmed by the Epilepsy Study Consortium, Inc. (ESCI) and as defined by: Onset, prior to 12 months of age, of recurrent focal with motor signs, hemiclonic, or generalized tonic-clonic seizures, which are often prolonged and triggered by hyperthermia. No past history of causal magnetic resonance imaging (MRI) lesion (past MRI or study-associated MRI not required to confirm absence of lesion). No other known etiology causing clinical DS manifestations. Normal development at seizure onset.
- Patient must have a documented pathogenic, likely pathogenic variant, or variant of uncertain significance in the sodium voltage-gated channel type 1 alpha subunit (SCN1A) gene. Patients who have SCN1A testing results of Negative (no variants identified) cannot be randomized.
- Experiences the required number of major motor seizures during the 6-week Observation Period. Seizure types included in counts are Hemiclonic, Focal with Motor Signs, Focal to Bilateral Tonic-Clonic, Generalized Tonic-Clonic, Tonic, Tonic/Atonic (Drop Attacks with fall or risk of fall), and Bilateral Clonic.
You likely can't join if
- Patient has documented variant in the SCN1A gene associated with gain-of-function potentially including but not limited to: Ala23Glu, Thr162Ile, Arg1636Gln, Thr226Met, Ile236Val, Val250Leu, Leu263Val, Thr398Met, Ala420Val, Val422Leu, Thr1174Ser, Trp1204Arg, Pro1345Ser, Ile1483Met, Gln1489Lys, Ile1498Thr, Ile1498Met, Phe1499Leu, Met1500Val, Arg1575Cys, Val1611Phe, Leu1624Pro, Arg1636Gln, Arg1648Cys, Leu1649Gln, Leu1660Ile, Phe1661Leu, Leu1670Trp, Gly1674Arg, Phe1774Ser, or Asp1866Tyr.
- Patient has a diagnostic result in a gene other than SCN1A on the epilepsy gene screening panel. For genes associated with dominant inheritance, one heterozygous pathogenic or likely pathogenic variant is exclusionary. For genes associated with recessive inheritance, biallelic pathogenic or likely pathogenic variant(s) are exclusionary. This includes homozygous variants and compound heterozygous variants. Results consistent with carrier status are not exclusionary.
- Patient is currently treated with a maintenance ASM acting primarily as a sodium channel blocker, including but not limited to phenytoin, carbamazepine, oxcarbazepine, lamotrigine, lacosamide, rufinamide, or cenobamate, given the mechanism of action of zorevunersen.
- Patient is currently treated with neuromodulation techniques (e.g., responsive neurostimulation, deep brain stimulation, or transcranial magnetic stimulation), with the exception of VNS.
- Patient has emergence of a new seizure type or reemergence of a past seizure type (seizure types that last occurred more than 12 months before Screening Visit A) during the Baseline Period, or has more than 1 hospitalization for seizures during the Baseline Period.
- Patient has a spinal deformity or other condition that may alter the free flow of CSF or has an implanted CSF drainage shunt. This does not exclude patients with scoliosis or spinal rods if, in the judgement of the Investigator, lumbar puncture can be performed and there is free flow of CSF.
See the full eligibility criteria
Who can join
- Patient and/or authorized representative must be willing and able to give informed consent/assent and any authorizations required by local law for participation in the study.
- Patient and their caregiver must be willing and able (in the Investigator’s opinion) to comply with all protocol requirements.
- At signing of consent/assent, patient must be ≥2 and <18 years of age.
- Patient must have a clinical diagnosis of DS confirmed by the Epilepsy Study Consortium, Inc. (ESCI) and as defined by: Onset, prior to 12 months of age, of recurrent focal with motor signs, hemiclonic, or generalized tonic-clonic seizures, which are often prolonged and triggered by hyperthermia. No past history of causal magnetic resonance imaging (MRI) lesion (past MRI or study-associated MRI not required to confirm absence of lesion). No other known etiology causing clinical DS manifestations. Normal development at seizure onset.
- Patient must have a documented pathogenic, likely pathogenic variant, or variant of uncertain significance in the sodium voltage-gated channel type 1 alpha subunit (SCN1A) gene. Patients who have SCN1A testing results of Negative (no variants identified) cannot be randomized.
- Experiences the required number of major motor seizures during the 6-week Observation Period. Seizure types included in counts are Hemiclonic, Focal with Motor Signs, Focal to Bilateral Tonic-Clonic, Generalized Tonic-Clonic, Tonic, Tonic/Atonic (Drop Attacks with fall or risk of fall), and Bilateral Clonic.
- Patient must have used at least 2 prior interventions for seizures. These can include anti-seizure medications (ASMs), ketogenic diet and/or vagus nerve stimulation (VNS) with either lack of adequate seizure control or discontinued due to an AE(s). These interventions can be ongoing therapies.
- Patient must be taking at least one ASM. Benzodiazepines or ASMs used on a standing basis (i.e., not as needed [PRN]) for any indication will be considered an ASM.
- All maintenance ASMs and interventions for seizures (i.e., ketogenic diet or VNS), as well as any marijuana- or cannabinoid-based products, must have been stable (unless adjusted for weight) during the Baseline Period. Felbamate must have been stable (unless adjusted for weight) for at least 6 months prior to Screening Visit A and during the Baseline Period. VNS implantation must have occurred at least 6 months prior to Screening Visit A. Rescue ASMs used on an as needed basis do not need to be stable.
What rules you out
- Patient has documented variant in the SCN1A gene associated with gain-of-function potentially including but not limited to: Ala23Glu, Thr162Ile, Arg1636Gln, Thr226Met, Ile236Val, Val250Leu, Leu263Val, Thr398Met, Ala420Val, Val422Leu, Thr1174Ser, Trp1204Arg, Pro1345Ser, Ile1483Met, Gln1489Lys, Ile1498Thr, Ile1498Met, Phe1499Leu, Met1500Val, Arg1575Cys, Val1611Phe, Leu1624Pro, Arg1636Gln, Arg1648Cys, Leu1649Gln, Leu1660Ile, Phe1661Leu, Leu1670Trp, Gly1674Arg, Phe1774Ser, or Asp1866Tyr.
- Patient has a diagnostic result in a gene other than SCN1A on the epilepsy gene screening panel. For genes associated with dominant inheritance, one heterozygous pathogenic or likely pathogenic variant is exclusionary. For genes associated with recessive inheritance, biallelic pathogenic or likely pathogenic variant(s) are exclusionary. This includes homozygous variants and compound heterozygous variants. Results consistent with carrier status are not exclusionary.
- Patient is currently treated with a maintenance ASM acting primarily as a sodium channel blocker, including but not limited to phenytoin, carbamazepine, oxcarbazepine, lamotrigine, lacosamide, rufinamide, or cenobamate, given the mechanism of action of zorevunersen.
- Patient is currently treated with neuromodulation techniques (e.g., responsive neurostimulation, deep brain stimulation, or transcranial magnetic stimulation), with the exception of VNS.
- Patient has emergence of a new seizure type or reemergence of a past seizure type (seizure types that last occurred more than 12 months before Screening Visit A) during the Baseline Period, or has more than 1 hospitalization for seizures during the Baseline Period.
- Patient has a spinal deformity or other condition that may alter the free flow of CSF or has an implanted CSF drainage shunt. This does not exclude patients with scoliosis or spinal rods if, in the judgement of the Investigator, lumbar puncture can be performed and there is free flow of CSF.
The study team makes the final eligibility decision.
Where it's taking place
- United States
- United Kingdom
- China
- Japan
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 0-17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include United States; United Kingdom; China; Japan. Enter your location above to see the nearest site and check your eligibility.
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BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.