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A Clinical Trial to Investigate the Efficacy, Safety and Pharmacokinetics of Elsunersen in Children
EU CTIS ID: 2024-515598-82-00
What this study is testing
To assess the efficacy of elsunersen on seizure frequency in participants with early-onset SCN2A-DEE
- 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
- Participant, or parent/legal guardian, is willing to sign an informed consent document indicating that he/she understands the purpose of the clinical trial; understands, and can perform, complete, and comply with all the procedures and assessments that are required during the clinical trial; and is willing to participate in the clinical trial.
- Has an eGFR ≥ 60 mL/min/1.73 m², calculated using the Schwartz formula (updated bedside version).
- Has a confirmed SCN2A variant based on genetic testing.
- Has onset of seizures prior to 3 months of age.
- Is between the ages of 0 (with a gestational age of at least 37 weeks +1 day) to ≤18 years at Screening.
- Seizure frequency of 4 or more countable motor seizures refractory to current treatment per 28-day during the Baseline Observation Period. Note: Countable motor seizures are defined as tonic seizures (bilateral; with or without fall or risk of fall); clonic seizures (bilateral); tonic-clonic seizures; atonic seizures (with fall or risk of fall) and atonic seizures (without fall or risk of fall); focal to bilateral tonic-clonic; focal or focal seizures with observable motor symptoms (including unilateral tonic or unilateral clonic).
You likely can't join if
- At screening, has any significant ongoing disease, disorder, laboratory abnormalities, environmental factor, or any ongoing or history of any psychiatric, medical, or surgical condition that, in the judgment of the investigator in consultation with the medical monitor and/or sponsor’s designee, might jeopardize the participant’s safety or interfere with the absorption, distribution, metabolism, or excretion of elsunersen; impact the clinical trial scientific objectives, or interfere with participation in the clinical trial.
- Is currently pregnant or breastfeeding or is planning to become pregnant during the clinical trial or within the timeframe specified in Section 5.5.3 of the protocol.
- Has any abnormal findings on brain MRI that may be contributing to the participant’s epilepsy, would put the participant at increased risk of ASO therapy (including but not limited to hydrocephalus), or any other finding that may be considered clinically significant as judged by the PI or ERC. Note: If a brain MRI has not been performed within 6 months of screening, the participant will need to have a brain MRI without gadolinium as part of Screening to assess ventricle size.
- Has any clinically significant or known pathogenic genetic variant other than in the SCN2A gene, or a genetic variant that may explain or contribute to the participant’s epilepsy and/or developmental disorder, in the opinion of the investigator or confirmed by the ERC.
- Has any other/additional etiology for epilepsy and/or DEE (e.g. encephalomalacia, etc) in the opinion of the investigator or confirmed by the ERC.
- Has bone, spine (eg, kyphosis, scoliosis), bleeding, or other disorder (including, but not limited to, intolerance to anesthesia, if applicable) that might expose the participant to risk of injury or unsuccessful lumbar puncture.
See the full eligibility criteria
Who can join
- Participant, or parent/legal guardian, is willing to sign an informed consent document indicating that he/she understands the purpose of the clinical trial; understands, and can perform, complete, and comply with all the procedures and assessments that are required during the clinical trial; and is willing to participate in the clinical trial.
- Has an eGFR ≥ 60 mL/min/1.73 m², calculated using the Schwartz formula (updated bedside version).
- Has a confirmed SCN2A variant based on genetic testing.
- Has onset of seizures prior to 3 months of age.
- Is between the ages of 0 (with a gestational age of at least 37 weeks +1 day) to ≤18 years at Screening.
- Seizure frequency of 4 or more countable motor seizures refractory to current treatment per 28-day during the Baseline Observation Period. Note: Countable motor seizures are defined as tonic seizures (bilateral; with or without fall or risk of fall); clonic seizures (bilateral); tonic-clonic seizures; atonic seizures (with fall or risk of fall) and atonic seizures (without fall or risk of fall); focal to bilateral tonic-clonic; focal or focal seizures with observable motor symptoms (including unilateral tonic or unilateral clonic).
- If prescribed any ASM or non-pharmacological intervention (including ketogenic diet and vagus nerve stimulation ) for the treatment of epilepsy or other symptoms of early-onset SCN2A-DEE (auxiliary medicinal products) is on a stable dose, settings, or parameters for 1 month prior to Screening (not to include weight-based dose changes of medications).
- Has been reviewed by the ERC prior to enrollment, including documentation of early onset SCN2A clinical phenotype , disease etiology, MRI results, and seizure frequency
- Seizure diary completion must occur on ≥80% days in the Screening/Observation
- Has a serum total bilirubin value <1.5× the ULN or a serum ALT or AST value <3×ULN.
What rules you out
- At screening, has any significant ongoing disease, disorder, laboratory abnormalities, environmental factor, or any ongoing or history of any psychiatric, medical, or surgical condition that, in the judgment of the investigator in consultation with the medical monitor and/or sponsor’s designee, might jeopardize the participant’s safety or interfere with the absorption, distribution, metabolism, or excretion of elsunersen; impact the clinical trial scientific objectives, or interfere with participation in the clinical trial.
- Is currently pregnant or breastfeeding or is planning to become pregnant during the clinical trial or within the timeframe specified in Section 5.5.3 of the protocol.
- Has any abnormal findings on brain MRI that may be contributing to the participant’s epilepsy, would put the participant at increased risk of ASO therapy (including but not limited to hydrocephalus), or any other finding that may be considered clinically significant as judged by the PI or ERC. Note: If a brain MRI has not been performed within 6 months of screening, the participant will need to have a brain MRI without gadolinium as part of Screening to assess ventricle size.
- Has any clinically significant or known pathogenic genetic variant other than in the SCN2A gene, or a genetic variant that may explain or contribute to the participant’s epilepsy and/or developmental disorder, in the opinion of the investigator or confirmed by the ERC.
- Has any other/additional etiology for epilepsy and/or DEE (e.g. encephalomalacia, etc) in the opinion of the investigator or confirmed by the ERC.
- Has bone, spine (eg, kyphosis, scoliosis), bleeding, or other disorder (including, but not limited to, intolerance to anesthesia, if applicable) that might expose the participant to risk of injury or unsuccessful lumbar puncture.
- Is unwilling or unable to discontinue medications that might increase the risk of bleeding (eg, heparin, low molecular weight heparin, platelet inhibitors) during the clinical trial (as defined in Section 6.4.1 of the protocol).
- Is required to take any excluded medication or is anticipated to require treatment with at least 1 excluded medication during the clinical trial (as defined in the relevant section of the protocol).
- Has laboratory test results at Screening outside the normal ranges for platelet count, prothrombin time (PT)/ partial thromboplastin time (PTT)/ international normalized ratio (INR), or renal function (serum creatinine [sCr] and urine protein [Uprot]), that are clinically meaningful as judged by the investigator.
- Has received any experimental or investigational drug, device, or other therapy within 30 days or 5 half-lives (whichever is longer) prior to Screening, including any prior use of gene therapy. Note: This restriction does not apply to participants from PRAX 222-111 clinical trial or to patients currently receiving elsunersen through Expanded or Emergency Access, who may enroll directly into the Extension Period.
- Has a known hypersensitivity to any component of the formulation of elsunersen.
The study team makes the final eligibility decision.
Where it's taking place
- United Kingdom
- United States
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, 18-64 years, 0-17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include United Kingdom; United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
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.