Clinical trial NCT05025605: Determining Efficacy and Safety of BXCL501 in Agitation Associated With Pediatric Schizophrenia and Bipolar Disorder
Official title: A Randomized, Double-blind, Placebo-controlled Study to Determine Efficacy and Safety of BXCL501 in Agitation Associated With Pediatric Schizophrenia and Bipolar Disorder
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE1
Conditions: Schizophrenia
Schizo-Affective Disorder
Schizophreniform; Schizophrenic
Bipolar Disorder I
Bipolar Disorder II
Lead sponsor: BioXcel Therapeutics Inc
Sponsor class: INDUSTRY
Collaborators: name: Cognitive Research Corporation
First posted: 2021-08-27
Enrollment: 140
Sex: ALL
Minimum age (years): 10
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention 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
Primary purpose: TREATMENT

Brief summary: 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: 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.

Eligibility criteria: Inclusion Criteria:

1. 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).
2. 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).
3. Patients who have a score of ≥ 4 on at least 1 of the 5 items on the PEC at Baseline.
4. Participants who agree to use a medically acceptable and effective birth control method

Exclusion Criteria:

1. Patients with agitation caused by acute intoxication, including alcohol or drugs of abuse (with the exception of THC) during urine screening.
2. Use of benzodiazepines or other hypnotics or oral or short-acting intramuscular antipsychotic drugs in the 4 hours before study treatment.
3. Patients who are judged to be at significant risk of suicide.
4. Patients with serious or unstable medical illnesses.
5. Patients who have received an investigational drug within 30 days prior to the current agitation episode.
6. Patients who are considered by the investigator, for any reason, to be an unsuitable candidate for receiving the study drug.

Primary outcomes:
measure: Absolute change from baseline in the Positive and Negative Syndrome Scale - Excited Component (PEC) total score
timeFrame: 120 minutes - Group one only
Secondary outcomes:
measure: Absolute change from baseline in the Positive and Negative Syndrome Scale - Excited Component (PEC) total score
timeFrame: 90, 60, 45, 30, 20,10 minutes - Group one only
Other outcomes:
Locations:
city: Anaheim
country: United States
facility: BioXcel Clinical Research Site
state: California
city: Hialeah
country: United States
facility: Bioxcel Clinical Research Site
state: Florida
city: Atlanta
country: United States
facility: BioXcel Clinical Research Site
state: Georgia
city: Decatur
country: United States
facility: Bioxcel Clinical Research Site
state: Georgia
city: DeSoto
country: United States
facility: BioXcel Clinical Research Site
state: Texas
Study officials:
affiliation: BioXcel Therapeutics
name: Robert Risinger, MD
role: STUDY_CHAIR
References: