Staccato Alprazolam for Seizures

Enrolling by invitation at 189 trial locations

Overseen By UCB Cares

Age: Any Age

Sex: Any

Trial Phase: Phase 3

Sponsor: UCB Biopharma SRL

Must be taking: Staccato alprazolam

Must not be taking: CYP3A4 inhibitors, Opioids, Sedatives, Beta blockers

Disqualifiers: Alcohol/drug use disorder, Airway hypersensitivity, Chronic pulmonary disorder, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Approved in 2 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: 48 months

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial examines the long-term safety of Staccato alprazolam, an inhaled medication for managing seizures. It targets individuals with epilepsy who have experienced specific seizures, such as prolonged focal seizures or a series of myoclonic seizures followed by a generalized seizure. Candidates should have previously participated in a study using Staccato alprazolam and must have a caregiver who can recognize their seizures. Participants must not have a recent history of certain respiratory issues or a sensitivity to the medication's components. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants a chance to contribute to the potential availability of a new treatment option.

Will I have to stop taking my current medications?

You may need to stop taking certain medications to join this trial. Specifically, you cannot take strong CYP3A4 inhibitors (like some antifungal medications), opioids, sedative hypnotics, or nonselective beta blockers on a regular basis.

Is there any evidence suggesting that Staccato alprazolam is likely to be safe for humans?

Research has shown that Staccato alprazolam is generally safe for people. In a previous study, doses of 1.0 mg and 2.0 mg were tested in patients with seizures. These doses stopped seizures quickly and were well-tolerated by participants. Another study found that a single dose of Staccato alprazolam reduced seizure activity in just two minutes. While these studies focused on the treatment's effectiveness, they also demonstrated its safety, with few negative effects reported. The use of Staccato alprazolam in past trials suggests it is safe for people, though ongoing studies continue to assess its long-term safety. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Most treatments for seizures involve oral medications or injectables that can take time to work. But Staccato alprazolam is unique because it is delivered through inhalation, offering a rapid onset of action. This delivery method allows the medication to be absorbed quickly through the lungs, potentially providing faster relief from seizures compared to traditional options. Researchers are excited about this treatment because it could offer a new, fast-acting option for people who need immediate seizure control.

What evidence suggests that Staccato alprazolam might be an effective treatment for seizures?

Research has shown that Staccato alprazolam can quickly stop seizures. Studies found that both 1.0 mg and 2.0 mg doses ended seizures in just a few minutes and were well tolerated by patients. Participants in this trial will receive Staccato alprazolam by inhalation. The device for Staccato alprazolam ensures rapid action, which is crucial during a seizure. These findings suggest that Staccato alprazolam could be a promising option for managing seizures due to its quick action. ^{1 4 6 7 8}

Who Is on the Research Team?

UCB Cares

Principal Investigator

001 844 599 2273 (UCB)

Are You a Good Fit for This Trial?

This trial is for people aged 12 and older with focal or generalized epilepsy who have had prolonged seizures. They must have a caregiver to monitor them, no recent severe respiratory issues, no drug abuse in the past year, not be allergic to alprazolam or similar drugs, and should not take certain medications like strong CYP3A4 inhibitors.

Inclusion Criteria

I have been diagnosed with epilepsy and have a history of long-lasting seizures.

I have completed a study with Staccato alprazolam.

I am 12 years old or older.

Exclusion Criteria

I have or had acute narrow-angle glaucoma.

I have mild asthma or other allergies affecting my breathing but can still participate.

I have had a major seizure episode in the last 8 weeks.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive Staccato alprazolam by inhalation to evaluate long-term safety and tolerability

48 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Open-label extension

Participants continue to receive Staccato alprazolam to assess long-term safety and tolerability

Long-term

What Are the Treatments Tested in This Trial?

Interventions

• Staccato alprazolam

Trial Overview The study tests Staccato alprazolam's long-term safety and tolerability in patients with epilepsy experiencing prolonged seizures. Participants previously involved in specific Staccato alprazolam studies are eligible.

How Is the Trial Designed?

Group I: Staccato alprazolam Experimental Treatment 1 Intervention

Participants will receive Staccato alprazolam by inhalation.

Staccato alprazolam is already approved in United States, European Union for the following indications:

Approved in United States as Alprazolam for:

• Anxiety disorder
• Panic disorder

Approved in European Union as Alprazolam for:

• Anxiety disorder
• Panic disorder

Who Is Running the Clinical Trial?

UCB Biopharma SRL

Lead Sponsor

Trials

118

Recruited

23,200+

Jean-Christophe Tellier

UCB Biopharma SRL

Chief Executive Officer since 2015

MD from University of Reims Champagne-Ardenne, Rheumatology specialization from University of Paris V, Executive business programs at Harvard and INSEAD

Dr. Iris Loew-Friedrich

UCB Biopharma SRL

Chief Medical Officer since 2014

MD from University of Leuven, PhD in Medical Sciences from University of Leuven

Published Research Related to This Trial

Clobazam, a 1,5-benzodiazepine, has been shown to significantly reduce seizure frequency by 65% in patients with refractory epilepsy, making it an effective adjunctive therapy.

While clobazam is generally well tolerated with minimal cognitive impairment, it can cause sedation in some patients, and about 36% may develop tolerance over time, necessitating careful management of dosage and withdrawal.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Current status of the 1,4- and 1,5-benzodiazepines in the treatment of epilepsy: the place of clobazam. Robertson, MM.[2022]

In a study involving 116 patients with epilepsy, both 1.0 mg and 2.0 mg doses of Staccato alprazolam effectively terminated seizures within 2 minutes in 65.8% of cases, compared to 42.5% for the placebo, indicating its potential as a rapid treatment for seizures.

Staccato alprazolam was well tolerated, with mild to moderate side effects like cough and somnolence, and no serious adverse events reported, suggesting it is a safe option for seizure management.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A randomized phase 2b efficacy study in patients with seizure episodes with a predictable pattern using Staccato® alprazolam for rapid seizure termination. French, J., Biton, V., Dave, H., et al.[2023]

Three approved rescue therapies for treating seizure clusters in the U.S. are diazepam rectal gel, midazolam nasal spray, and diazepam nasal spray, with diazepam rectal gel being effective but potentially socially uncomfortable for adults and adolescents.

Midazolam and diazepam nasal sprays offer a rapid and socially acceptable alternative to rectal administration, allowing for easier self-administration during seizure clusters.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Rescue Treatments for Seizure Clusters. Kotloski, RJ., Gidal, BE.[2022]

Midazolam nasal spray (MDZ-NS) has been shown to be effective in treating seizure clusters, with a significant success rate in terminating seizures within 10 minutes and maintaining seizure-free status for at least six hours, compared to placebo.

The 5 mg dose of MDZ-NS was found to be as safe as a placebo, indicating that it is a well-tolerated option for patients aged 12 years and older experiencing seizures.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

The Evaluation of the Efficacy and Safety of Midazolam Nasal Spray in Patients With Seizure Clusters: A Systematic Review and Meta-Analysis. Niraj, N., Mahajan, S., Prakash, A., et al.[2023]

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10107237/

A randomized phase 2b efficacy study in patients with seizure ... Both 1.0 mg and 2.0 mg doses of Staccato alprazolam demonstrated efficacy in rapidly terminating seizures in an inpatient setting and were well tolerated.

2. neurologylive.com neurologylive.com/view/advancing-rest-in-epilepsy-with-staccato-alprazolam

Advancing REST in Epilepsy with Staccato Alprazolam Key Findings: · The Staccato alprazolam device showed rapid onset of action, with most patients achieving seizure termination within minutes.

3. clinicaltrials.gov clinicaltrials.gov/study/NCT03478982

NCT03478982 | Inpatient, Dose-Ranging Study of Staccato ... This is a multi-center, double-blind, randomized, parallel group, dose-ranging study to investigate the efficacy and clinical usability of STAP-001 in adult ...

4. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/36268811/

A randomized phase 2b efficacy study in patients with ... Both 1.0 mg and 2.0 mg doses of Staccato alprazolam demonstrated efficacy in rapidly terminating seizures in an inpatient setting and were well tolerated.

5. ucb-usa.com ucb-usa.com/stories-media/UCB-U-S-News/detail/article/ucb-presents-new-data-on-impact-and-burden-of-epilepsy-at-2024-american-epilepsy-society-aes-meeting

UCB Presents New Data on Impact and Burden of Epilepsy ... We are excited to present new data at AES on Staccato alprazolam, an investigational drug under study in a phase 3 trial for prolonged seizures, ...

6. clinicaltrials.gov clinicaltrials.gov/study/NCT05077904

NCT05077904 | A Study to Test the Efficacy and Safety of ... The purpose of the study is to assess the success of a single administration of Staccato alprazolam compared with placebo both in rapidly terminating a ...

7. onlinelibrary.wiley.com onlinelibrary.wiley.com/doi/10.1111/epi.18643?af=R

Pharmacokinetics and tolerability of single‐dose Staccato ... Results from a Phase 2a study showed that Staccato alprazolam rapidly suppressed epileptiform activity within 2 min in patients with ...

8. mayo.edu mayo.edu/research/clinical-trials/cls-20535684

A Study to Test the Effectiveness and Safety of Staccato ... The purpose of the study is to assess the success of a single administration of Staccato alprazolam compared with placebo both in rapidly terminating a seizure ...