A study to test the safety and tolerability of Staccato alprazolam in study participants 12 years of age and older with epilepsy
- Conditions
- MedDRA version: 20.0Level: LLTClassification code 10076333Term: Prolonged seizureSystem Organ Class: 100000004852Treatment of stereotypical prolonged seizureTherapeutic area: Diseases [C] - Nervous System Diseases [C10]
- Registration Number
- EUCTR2021-002637-42-ES
- Lead Sponsor
- CB Biopharma SR
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Authorised-recruitment may be ongoing or finished
- 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 caregiver =18 years of age at the time of signing the informed consent; the 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 myoclonic seizure 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
- Male and female participants:
a) A male participant must agree to use contraception during the Treatment Period and for at least 7 days after investigational medicinal product (IMP) administration
b) A female participant is eligible to participate if she is not pregnant, not breastfeeding, and:
i) Not a woman of childbearing potential (WOCBP)
OR
ii) A WOCBP who agrees to follow the contraceptive guidance during the Treatment Period and for at least 30 days after IMP administration
- Participant is capable of giving signed informed consent (or giving assent, where required).
The informed consent form (ICF) or a specific assent form, where required, will be signed and dated by minors
- The participant’s caregiver(s) must be capable of giving signed informed, which includes compliance with the requirements and restrictions listed in the ICF, the protocol, and the individualized participant management plan (iPMP)
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 history of convulsive 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 (eg, asthma, chronic obstructive pulmonary disease, restrictive lung diseases [including idiopathic pulmonary fibrosis]) and/or recent history or presence of hemoptysis or pneumothorax
- Participant has a condition for which oral alprazolam is contraindicated (eg, myasthenia gravis, severe respiratory insufficiency, and sleep apnea syndrome)
- 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 (eg, propranolol, nadolol, and timolol) on a chronic basis
- Participant has an FEV1 <80 % of predicted forced expiratory volume in 1 second (FEV1) as measured via spirometry at the Screening Visit
- Participant has an oxygen saturation <95 %
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 6 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 6 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 6 months<br>2. Treatment success after IMP administration with no recurrence after 2 hours for seizures occurring within first 6 months<br>3. Frequency of respiratory TEAEs;Timepoint(s) of evaluation of this end point: 1&2: From start of IMP treatment up to 6 months<br>3: From Baseline up to the End of Study Visit (up to 48 months)