A Study Evaluating NPT 2042 Versus Placebo in Subjects Aged 16-75 Years With Genetic Generalized Epilepsy (GGE) and Absence Seizures

Официальное название

A Single-center, Double-blind, Placebo-controlled Crossover Study Evaluating NPT 2042 Versus Placebo in Subjects Aged 16-75 Years With Genetic Generalized Epilepsy (GGE) and Absence Seizures

Обзор

This study will compare the effect of NPT 2042 and placebo in subjects with GGE on the frequency and duration of electroencephalographic absence seizures, separated by a 14-day washout period. The study will be a single-center, double-blind, crossover study with subjects receiving either NPT 2042 BID orally or matching placebo BID in each of two treatment periods. Two doses of NPT 2042 will be evaluated.

Вмешательства

• Препарат NPT 2042
  NPT 2042 is a new drug being developed as an anti-seizure treatment
• Другое Placebo
  Placebo Comparator

Первичные конечные точки

• Mean and median across subjects of the within subject difference in percent change from treatment period baseline of the frequency of absence seizures [Срок оценки: Up to 12 weeks]

Вторичные конечные точки (6)

• Mean and median across subjects of the within subject difference between NPT 2042 and placebo on absence seizures [Срок оценки: Up to 12 weeks]
• Mean and median across subjects of the within subject categorical difference in percent change between NPT 2042 and placebo of the frequency of absence seizure [Срок оценки: Up to 12 weeks]
• The proportion of NPT 2042- vs. placebo-treated subjects meeting the study specific response criteria [Срок оценки: Up to 12 weeks]
• The mean change from Baseline across subjects of the within subject difference between NPT 2042 and placebo on the Quality of Life in Epilepsy Questionnaire (QOLIE-31-P) [Срок оценки: Up to 12 weeks]
• The mean change from Baseline in the Digit Symbol Substitution Test [Срок оценки: Up to 12 weeks]
• The mean change from Baseline in the Epworth Sleepiness Scale [Срок оценки: Up to 12 weeks]

Критерии участия

Критерии включения

• Subject is capable of and provides consent/assent, and the study participant's parent/legal representative/caregiver provides signed informed consent for minor study participants, which includes compliance with the requirements and restrictions listed in the informed consent form (ICF)
• Subject is aged 16-75 years at the time of consent/assent
• Subject is diagnosed with genetic generalized epilepsy with absence seizures (consistent with the International League Against Epilepsy (ILAE) Classification of Seizures (2017))
• Subject has electroencephalogram (EEG) evidence of bilateral synchronous generalized paroxysmal spike-wave (2.5 Hz to 6 Hz) bursts lasting 3 seconds or more at least 4 times on the screening 72-hour ambulatory EEG.
• Subject has been on a stable dose of at least one antiseizure medication (ASM) for at least 30 days. Vagal nerve stimulation at stable settings (for at least 30 days before screening), without use of the magnet, is also acceptable.
• Subject has normal cognition and no clinically significant abnormalities on neurological examination at screening in the opinion of the Investigator
• Subject is in otherwise good health (with the exception of epilepsy), as determined by the investigator, and as documented in the medical history, physical examination, and screening laboratory investigations
• Subject has a body mass index (BMI) between 18 and 40 kg/m2 inclusive, at screening
• Female subjects of child-bearing potential and all men agree to use of highly effective methods of contraception during the study and for 28 days after last dose of study drug
• Subject (and parent/caregiver, if applicable) is able to communicate with the investigator and to understand and comply with all study requirements, including the clinic visit schedule

Критерии исключения

• Subject has metabolic or mitochondrial encephalopathies, seizures associated with structural abnormalities, or infection-related seizures.
• Subject has a developmental epileptic encephalopathy (e.g. Lennox-Gastaut syndrome)
• Subject has a history of convulsive status epilepticus within the past year.
• Subject has a history of surgical intervention for treatment of epilepsy
• Subject has a history of nonepileptic seizures (e.g., metabolic, structural, or paroxysmal non epileptic seizures)
• Subject has severe intellectual disability, severe autism spectrum disorder, or severe developmental disorder such that the subject cannot consent or assent to participate or cannot cooperate with the study procedures
• Female subject who is pregnant or lactating
• Subject has any clinically significant laboratory abnormality which, in the opinion of the investigator, will exclude the subject from the study
• Subject has an active CNS infection, demyelinating disease, degenerative neurological disease, or any CNS disease deemed to be progressive during the course of the study that may confound the interpretation of the study results
• Subject has any clinically significant psychiatric illness, psychological or behavioral problems which, in the opinion of the investigator, would interfere with the subject's ability to participate in the study, including but not limited to the following:

• Subject has active suicidal ideation prior to study entry as indicated by a positive response ("yes") to either Question 4 or Question 5 of the Columbia Suicide Severity Rating Scale (C-SSRS)
• Study participant has a lifetime history of suicide attempt (including an active attempt, interrupted attempt, or aborted attempt)
• Subject is suffering from clinically significant active liver disease, porphyria or has a family history of severe hepatic dysfunction indicated by abnormal liver function tests greater than three times the upper limit of normal (AST and ALT)
• Subject has a DSM-V diagnosis of alcohol or drug abuse, or drug addiction within the past 12 months
• Subject has participated in any other trials involving an investigational product or device within 30 days of screening or longer, as required by local regulations
• Subject is currently using prohibited medications or products
• Subject is unable to complete ingestion of four placebo SGCs with a minimum of eight ounces of water at screening
• Subject (and parent/caregiver, if applicable) has daily commitments during the study duration that would interfere with attending all study visits
• Positive urine drug test for substance of abuse or illegal recreational substances at screening

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Рандомизированное

Модель

Перекрёстный дизайн

Маскирование

Четверное слепое

Основная цель

Лечение

Центры проведения

США · 1 центр

• Clinical Trials, Inc. (CTI) — Little Rock

Идентификаторы

NCT: NCT06769659 · NPT 2042 CL-200

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

Открыть это исследование на ClinicalTrials.gov ↗