A study to test the safety and tolerability of Staccato alprazolam in study participants 12 years of age and older with epilepsy
- Conditions
- Treatment of stereotypical prolonged seizureMedDRA version: 20.0Level: LLTClassification code 10076333Term: Prolonged seizureSystem Organ Class: 10029205 - Nervous system disordersTherapeutic area: Diseases [C] - Nervous System Diseases [C10]
- Registration Number
- EUCTR2021-002637-42-BG
- Lead Sponsor
- CB Biopharma SR
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 300
- Participant must be =12 years of age at the time of signing informed consent (or giving assent, where required)
- 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
- 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:
a) Generalized seizure episodes starting with a flurry of absence seizures or myoclonic seizures with a minimum total duration of 5 minutes
b) Episodes of a focal seizure with a minimum duration of 3 minutes
c) 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
Additional inclusion criteria for treatment arms 2 to 5:
Prior to the Screening Visit, the participant completed the Phase 3 efficacy study with Staccato alprazolam (EP0162) at a clinical site located in the EU, UK, and UA, and experienced an IMP-treated seizure during the Outpatient Treatment Period.
Are the trial subjects under 18? yes
Number of subjects for this age range: 49
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 236
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 15
- 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)
- 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 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 (eg, myasthenia gravis, severe respiratory insufficiency, and sleep apnea syndrome)
- Participant has a history or presence of long QT syndrome, a family history of sudden death due to long QT syndrome, or unexplained syncope
- 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 (eg, fentanyl, oxycodone, morphine) or sedative hypnotics on a chronic basis
- Participant is taking nonselective beta blockers on a chronic basis
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: Evaluation of the long-term safety and tolerability of Staccato alprazolam;Secondary Objective: - Evaluation of the probability of success of repeated treatment with Staccato alprazolam (for seizures occurring within the first 12 months [up to a maximum of 10 treated seizures])<br>- Evaluation of the probability of success of repeated treatment with Staccato alprazolam with no recurrence of seizure(s) up to 2 hours (for seizures occurring within the first 12 months [up to a maximum of 10 treated seizures])<br>- Evaluation of the long-term pulmonary safety of Staccato alprazolam;Primary end point(s): 1. Frequency of treatment-emergent adverse events (TEAEs)<br>2. Frequency of TEAEs leading to withdrawal from study<br>3. Frequency of serious TEAEs;Timepoint(s) of evaluation of this end point: 1&3: From Baseline up to the End of Study Visit (up to 48 months)
- Secondary Outcome Measures
Name Time Method Secondary end point(s): 1. Treatment success after investigational medicinal product (IMP) administration for seizures occurring within the first 12 months <br>2. Treatment success after IMP administration with no recurrence after 2 hours for seizures occurring within first 12 months <br>3. Frequency of respiratory TEAEs<br><br>;Timepoint(s) of evaluation of this end point: 1&2: From start of IMP treatment up to 12 months<br>3: From Baseline up to the End of Study Visit (up to 48 months)