Phase 2 Epilepsy Trial, Recruiting

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

Who might be able to join this trial:

• You must be willing and able to sign a consent form (and an assent form if required for your age) and follow all study requirements.
• You must be between 10 and 18 years old (inclusive) at the time of signing the consent form.
• Female participants must not be pregnant or breastfeeding.
• All male participants and females who could become pregnant must avoid donating sperm or eggs from the time of consent until at least 12 weeks after receiving the study drug.
• If you are sexually active and pregnancy is possible, you must use highly effective birth control from the time of consent until at least 12 weeks after receiving the study drug.
• You must have a genetically confirmed diagnosis of Lafora disease, shown by a known gene mutation in EPM2A or EPM2B.
• You must score 9 or higher on the Lafora Disease Problem Scale (LDPS), including a motor score of at least 2, meaning you can walk at least 10 steps on your own.

Who may not be able to join:

• You have significant health problems in your medical history, such as a stroke in the past 6 months or major surgery in the past 3 months.
• Your platelet count (a blood measure related to clotting) is below 80,000/mm³, or you have other significant abnormal blood test results.
• You have a history of bleeding disorders or problems with blood clotting.
• You currently have an active infection requiring antiviral or antibiotic treatment that will not be finished before the first day of the study.
• You are unwilling to follow the study procedures or cooperate with the research team.
• You are unable or unwilling to have a lumbar puncture (a procedure to collect fluid from around the spine).
• You have a known history of, or have tested positive for, HIV, hepatitis C, or chronic hepatitis B.
• You have moderate to severe liver or kidney problems.
• You have had cancer in the past 5 years (some exceptions may apply — confirm with trial site).
• You have uncontrolled high blood pressure (the specific blood pressure limits depend on your age — confirm with trial site).
• You have received a similar type of drug (called an oligonucleotide or siRNA) within the past 4–12 months, or you have had an allergic reaction to this type of drug in the past (note: COVID-19 mRNA vaccines are not included in this restriction).
• You have a history of alcohol or drug misuse within the past 12 months, or are currently misusing alcohol or drugs.
• You have taken part in another clinical trial or used an experimental drug or device within the past 30 days (or longer, depending on the drug — confirm with trial site).
• You have taken blood-thinning or anti-clotting medications (such as warfarin, clopidogrel, or rivaroxaban) within the 14 days before screening, or expect to need them during the study (low-dose aspirin may be an exception — confirm with trial site).

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