Determining Efficacy and Safety of BXCL501 in Agitation Associated With Pediatric Schizophrenia and Bipolar Disorder

Study Overview
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Brief Summary
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This is a study of the efficacy and safety of BXCL501 in children and adolescents with acute agitation and either bipolar disorder or schizophrenia.

Detailed Description
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The study will enroll approximately 140 subjects randomized to dose regimens of 80 µg or 120 µg BCXL501 or placebo (Group one), or 60 µg BCXL501 or placebo (Group two; Europe only). Subjects with acute agitation will include male and female children and adolescents who are either newly admitted to a hospital setting or already admitted and experiencing acute agitation. Subjects will be domiciled in a clinical research setting or hospitalized to remain under medical supervision while undergoing screening procedures to assess eligibility. Efficacy and safety assessments will be conducted periodically before and after dosing.

Official Title
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A Randomized, Double-blind, Placebo-controlled Study to Determine Efficacy and Safety of BXCL501 in Agitation Associated With Pediatric Schizophrenia and Bipolar Disorder

Conditions
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Schizophrenia, Schizo-Affective Disorder, Schizophreniform; Schizophrenic, Bipolar Disorder I, Bipolar Disorder II

Intervention / Treatment
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* Drug: BXCL501 80 Micrograms
* Drug: Placebo Film
* Drug: BXCL501 120 Micrograms
* Drug: BXCL501 60 Micrograms


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Male and female subjects between the ages of 10-17 years, inclusive, with bipolar disorder (DSM-5 criteria) and 13-17 years, inclusive, in subjects with schizophrenia (DSM-5 criteria). Patients who are judged to be clinically agitated at Screening and Baseline with a total score of ≥14 on the 5 items comprising the PANSS Excited Component (PEC). Patients who have a score of ≥ 4 on at least 1 of the 5 items on the PEC at Baseline. Participants who agree to use a medically acceptable and effective birth control method Exclusion Criteria: Patients with agitation caused by acute intoxication, including alcohol or drugs of abuse (with the exception of THC) during urine screening. Use of benzodiazepines or other hypnotics or oral or short-acting intramuscular antipsychotic drugs in the 4 hours before study treatment. Patients who are judged to be at significant risk of suicide. Patients with serious or unstable medical illnesses. Patients who have received an investigational drug within 30 days prior to the current agitation episode. Patients who are considered by the investigator, for any reason, to be an unsuitable candidate for receiving the study drug.

Ages Eligible for Study
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Minimum Age: 10 Years
Maximum Age: 17 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Interventional Model Description: Study will randomize subjects in Group one 1:1:1 to receive BXCL501 80 μg, BXCL501 120 μg, or matching placebo film; Group two 2:1 to receive BXCL501 60 μg or matching placebo film
Masking: Double


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: 80 Micrograms<br>Sublingual film containing 80 micrograms Dexmedetomidine | Drug: BXCL501 80 Micrograms<br>* Sublingual film containing 80 Micrograms BXCL501<br>* Other names: Dexmedetomidine;|
| Experimental: 120 Micrograms<br>Sublingual film containing 120 micrograms Dexmedetomidine | Drug: BXCL501 120 Micrograms<br>* Sublingual film containing 120 Micrograms BXCL501<br>* Other names: Dexmedetomidine;|
| Placebo Comparator: Placebo<br>Sublingual Placebo film | Drug: Placebo Film<br>* Matching Sublingual Placebo film<br>* Other names: Placebo;|
| Experimental: 60 Micrograms<br>Sublingual film containing 60 micrograms Dexmedetomidine Europe Only | Drug: BXCL501 60 Micrograms<br>* Sublingual film containing 60 Micrograms BXCL501 Europe Only<br>* Other names: Dexmedetomidine;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Absolute change from baseline in the Positive and Negative Syndrome Scale - Excited Component (PEC) total score | The Positive and Negative Syndrome Scale - Excited Component (PEC) comprises 5 items associated with agitation: poor impulse control, tension, hostility, uncooperativeness, and excitement; each scored 1 (minimum) to 7 (maximum). The PEC, the sum of these 5 subscales, thus ranges from 5 (absence of agitation) to 35 (extremely severe) | 120 minutes - Group one only | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Absolute change from baseline in the Positive and Negative Syndrome Scale - Excited Component (PEC) total score | The Positive and Negative Syndrome Scale - Excited Component (PEC) comprises 5 items associated with agitation: poor impulse control, tension, hostility, uncooperativeness, and excitement; each scored 1 (minimum) to 7 (maximum). The PEC, the sum of these 5 subscales, thus ranges from 5 (absence of agitation) to 35 (extremely severe) | 90, 60, 45, 30, 20,10 minutes - Group one only |