An Open-Label, Multicenter, Outpatient Extension Study to Evaluate the Safety and Tolerability of Staccato Alprazolam in Study Participants 12 Years of Age and Older With Stereotypical Prolonged Seizures
试验速览
- 阶段
- 3 期
- 状态
- Enrolling By Invitation
- 入组人数
- 300
- 试验地点
- 346
- 主要终点
- Frequency of treatment-emergent adverse events (TEAEs)
研究概览
简要总结
The purpose of the study is to evaluate the long-term safety and tolerability of Staccato alprazolam.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 12 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participant must be ≥12 years of age at the time of signing informed consent
- •Participant must have a study caregiver ≥18 years of age at the time of signing the informed consent; the study caregiver(s) must be able to recognize and observe the participant's seizures
- •Participants with an established diagnosis of focal or generalized epilepsy or combined focal and generalized epilepsy with a documented history of stereotypical episodes of prolonged seizures that includes at least 1 of the following:
- •Generalized seizure episodes starting with a flurry of absence seizures or myoclonic seizures with a minimum total duration of 5 minutes
- •Episodes of a focal seizure with a minimum duration of 3 minutes
- •Episodes of a focal seizure or a flurry of myoclonic seizures for at least 90 seconds followed by a generalized/bilateral tonic-clonic seizure with a minimum total duration of 3 minutes
- •Prior to the Screening Visit, participant completed a study using Staccato alprazolam (such as EP0162 (NCT05077904), ENGAGE-E-001 (NCT03478982), or UP0100 (NCT04857307))
排除标准
- •Participant has a current history of alcohol or drug use disorder, as defined in the Diagnostic and Statistical Manual of Mental Disorders 5, within the previous 1 year
- •Participant has a known hypersensitivity to any components of the investigational medicinal product (IMP) or comparable drugs (and/or an investigational device) as stated in this protocol or to albuterol (or similar bronchospasm rescue medication if needed to meet country-specific requirements)
- •Participant has a history of convulsive (generalized tonic-clonic) status epilepticus in the 8 weeks prior to the Screening Visit
- •Participant has a history or presence of known nonepileptic seizures which cannot be distinguished from qualifying epileptic seizures
- •Participant has a clinically significant known airway hypersensitivity (eg, bronchospasm to known allergens, such as pollen, animals, or food) and/or acute respiratory signs/symptoms (eg, shortness of breath, wheezing on lung auscultation). NOTE: Participants with mild asthma who qualify for inclusion in the are allowed to be enrolled even though they have known airway hypersensitivity
- •Participant has a clinically significant chronic pulmonary disorder other than mild asthma (eg, chronic obstructive pulmonary disease, restrictive lung diseases [including idiopathic pulmonary fibrosis]) and/or recent history or presence of hemoptysis or pneumothorax
- •Participant has had a positive antigen test for severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) and experienced moderate to severe signs/symptoms of respiratory distress necessitating hospitalization or outpatient treatment such as ambulatory oxygen, extensive treatment with inhaler medications, and/or oral medications for a duration of 4 weeks or more, unless full resolution occurred at least 6 months prior to Screening
- •Participant has experienced a severe upper respiratory tract infection within 4 weeks or severe bronchitis/pneumonia within 3 months before the Screening Visit
- •Participant has a history or presence of acute narrow-angle glaucoma
- •Participant has a condition for which oral alprazolam is contraindicated as per the regional labeling
- •Participant is taking any drug that is a strong CYP3A4 inhibitor, including azole antifungal agents (ketoconazole and itraconazole) and nefazodone
- •Participant is taking any opioids or sedative hypnotics on a chronic basis
- •Participant is taking nonselective beta blockers on a chronic basis
研究组 & 干预措施
Staccato alprazolam
Participants will receive Staccato alprazolam by inhalation.
干预措施: Staccato alprazolam (Drug)
结局指标
主要结局
Frequency of treatment-emergent adverse events (TEAEs)
时间窗: From Baseline up to the End of Study Visit (up to 78 months)
An Adverse event (AE) is defined as any untoward medical occurrence, unintended disease or injury, or untoward clinical signs (including abnormal laboratory finding) in study participants, users, or other persons, whether or not related to the investigational medical product (IMP) and whether anticipated or unanticipated. A treatment-emergent adverse event (TEAE) is defined as any AE with a start date/time on or after the first IMP administration.
Frequency of TEAEs leading to withdrawal from study
时间窗: From Baseline up to the End of Study Visit (up to 78 months)
An Adverse event (AE) is defined as any untoward medical occurrence, unintended disease or injury, or untoward clinical signs (including abnormal laboratory finding) in study participants, users, or other persons, whether or not related to the investigational medical product (IMP) and whether anticipated or unanticipated. A treatment-emergent adverse event (TEAE) is defined as any AE with a start date/time on or after the first IMP administration.
Frequency of serious TEAEs
时间窗: From Baseline up to the End of Study Visit (up to 78 months)
A serious adverse event (SAE) is any untoward medical occurrence that at any dose: * Results in death * Is life-threatening * Requires inpatient hospitalisation or prolongation of existing hospitalisation * Results in persistent or significant disability/incapacity, or * Is a congenital anomaly/birth defect * Other important medical events which based on medical or scientific judgement may jeopardize the patients, or may require medical or surgical intervention to prevent any of the above
次要结局
- Treatment success after investigational medicinal product (IMP) administration for seizures occurring within the first 12 months(From start of IMP treatment up to 12 months)
- Treatment success after IMP administration with no recurrence after 2 hours for seizures during the first 12 months(From start of IMP treatment up to 12 months)
- Treatment success after investigational medicinal product (IMP) administration for seizures occurring within the first 12 months(From start of IMP treatment up to 12 months)
- Treatment success after IMP administration with no recurrence after 2 hours for seizures during the first 12 months(From start of IMP treatment up to 12 months)
- Frequency of respiratory TEAEs(From Baseline up to the End of Study Visit (up to 78 months))