Phase 2 Epilepsy Trial, Recruiting

Written by an AI model from the ClinicalTrials.gov eligibility criteria and checked on a sample basis. Please confirm against the original criteria.

Who might be able to join this trial:

• Children aged 2 to 12 years old (at the time of screening) who have been diagnosed with a specific type of severe epilepsy called Developmental Epileptic Encephalopathy (DEE), caused by a confirmed fault in a gene called KCNT1, verified through central genetic testing.
• Children whose other regular medications, special diets (such as the ketogenic diet), or other seizure treatments (such as vagal nerve stimulation) have been at a stable, unchanged dose or level for a period of time.

Who may not be able to join:

• Children whose KCNT1 gene fault causes a type of epilepsy other than "Epilepsy of Infancy with Migrating Focal Seizures" or "Early-Onset Epileptic Encephalopathy."
• Children who have been found through previous genetic testing to have a confirmed fault in any other gene known to cause epilepsy (cases where the genetic finding is uncertain may be discussed with the trial sponsor).
• Children who have significant health conditions or physical examination findings beyond their epilepsy that the trial doctor considers would make participation unsafe or difficult to complete, including any significant health events within the 30 days before screening.
• Children who have an abnormal heart tracing (ECG) at the screening visit, as judged by the trial doctor.
• Children whose blood or urine tests at screening show that their kidneys are not working well enough (below a specific threshold of kidney function), or that their liver is showing signs of significant stress (certain liver-related values above defined limits).
• Children who have tested positive for Hepatitis B, Hepatitis C, or HIV within the 6 months before the screening visit, or at the time of screening blood tests.
• Children who have bone, spine, or bleeding disorders (such as haemophilia or Von Willebrand's disease), or other conditions that could make a lumbar puncture (spinal tap) unsafe or unlikely to succeed.
• Children for whom MRI scans, lumbar punctures, or injections into the spinal fluid are not safe or suitable for any reason.
• Children who have a history of tumours or cancers affecting the brain or spinal cord, including cancer that has spread to the brain or spinal cord.
• Children who need ongoing breathing support (such as continuous oxygen or a breathing machine like CPAP) during their waking hours. Note: devices used only to help clear the airways do not apply here.
• Children who require a breathing tube or have a tracheostomy (a surgical opening in the throat for breathing).
• Children who have taken a medicine called quinidine within 30 days before the screening visit.
• Children who are currently taking, or expected to take during the trial, medicines that thin the blood or prevent clotting (antiplatelet or anticoagulant therapy).
• Children who have taken part in another clinical trial involving an experimental treatment within 30 days before screening (or within 5 half-lives of that experimental treatment, whichever period is longer).
• Children who have an implanted device in their brain or spinal cord that could interfere with delivering the trial medicine via lumbar puncture.
• Children who have a known allergic reaction to similar types of medicines (called oligonucleotides), shown by a serious body-wide reaction, or who have any other known allergy that the trial doctor believes would make participation unsafe.

Important: Always verify eligibility with the trial site directly before applying.