Subject that meets DSM-5 criteria for schizophrenia, schizoaffective, or schizophreniform disorder, or other specified/unspecified schizophrenia spectrum and/or other psychotic disorders OR Subject meets DSM-5 criteria for bipolar disorder (bipolar I or II ). MedDRA version: 20.0 Level: HLGT Classification code 10039628 Term: Schizophrenia and other psychotic disorders System Organ Class: 10037175 - Psychiatric disorders MedDRA version: 20.0 Level: LLT Classification code 10039629 Term: Schiz
Conditions
Interventions
Product Name: BXCL501
Pharmaceutical Form: Buccal film
INN or Proposed INN: DEXMEDETOMIDINE HYDROCHLORIDE
CAS Number: 145108-58-3
Other descriptive name: DEXMEDETOMIDINE HYDROCHLORIDE
Concentration un
Sponsors
BioXcel Therapeutics, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1.Male and female subjects between the ages of 10-17 years, inclusive, in subjects with bipolar disorder and 13-17 years, inclusive, in subjects with schizophrenia. 2.Subject or legally acceptable representative (per local regulatory requirements for the legal age of consent for study participation) is able to sign and date written ICF prior to the start of any study-specific qualification procedures. 3.Subject meets DSM-5 criteria for schizophrenia, schizoaffective, or schizophreniform disorder, or other specified/unspecified schizophrenia spectrum and/or other psychotic disorders OR Subject meets DSM-5 criteria for bipolar disorder (bipolar I or II ). 4.Subject assessed to be clinically agitated at Screening and Baseline with a total score of =14 on the 5 items (poor impulse control, tension, hostility, uncooperativeness, and excitement) comprising the PANSS Excited Component (PEC). 5.Subject has a score of =4 on at least 1 of the 5 items on the PEC at Baseline. 6.Subject is in good general health prior to study participation as determined by a detailed medical history, physical examination, 12-lead ECG with rhythm strip, blood chemistry profile, hematology, urinalysis, and in the opinion of the investigator. 7.Female subjects, if of child-bearing potential and sexually active, and male subjects, if sexually active with a partner of child-bearing potential, agree to use a medically acceptable and effective birth control method throughout the study and for 1 week following the end of the study. Medically acceptable methods of contraception that may be used by the participant and/or his/her partner include abstinence, birth control pills or patches, diaphragm with spermicide, intrauterine device, condom with foam or spermicide, vaginal spermicidal suppository, surgical sterilization, and progestin implant or injection. Prohibited methods include the rhythm method, withdrawal, condoms alone, or diaphragm alone. Are the trial subjects under 18? yes Number of subjects for this age range: 120 F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Subjects with agitation caused by acute intoxication, including positive identification of alcohol by breathalyzer or drugs of abuse (except for THC) during urine screening. 2. Subjects with ADHD treated with an alpha2-adrenergic agonist (clonidine, guanfacine). 3. Subjects with agitation that cannot be attributed to schizophrenia or bipolar disorder (bipolar I or II) as diagnosed by DSM-5 criteria. 4. Use of benzodiazepines or other hypnotics or oral or short-acting intramuscular antipsychotic drugs in the 4 hours before study treatment. 5. Treatment with alpha-1 noradrenergic blockers (terazosin, doxazosin, tamsulosin, alfuzosin, or prazosin) or other prohibited medications. 6. Subjects with significant risk of suicide or homicide per the investigator’s assessment, or any subject with an answer of “yes” to item 4 or 5 on the C-SSRS. 7. Female subjects who have a positive pregnancy test at Screening. 8. Subjects who have hydrocephalus, seizure disorder, or history of significant head trauma, brain tumor, or meningitis. 9. History of syncope or other syncopal attacks, current evidence of hypovolemia, orthostatic hypotension, or a baseline heart rate of <55 beats per minutes (bpm) or a resting systolic/diastolic blood pressure (DBP) of <90/60 mmHg at Screening and before dosing. 10. Subjects with laboratory or ECG abnormalities considered clinically significant by the investigator or qualified designee that would have clinical implications for the subject’s participation in the study. 11. Subjects with serious or unstable medical illnesses. These include current hepatic impairment (moderate-severe), or renal, respiratory, endocrinologic, or hematologic disease. 12. Subjects who have received an investigational drug within 30 days prior to the current agitation episode. 13. Subjects who are considered by the investigator, for any reason, to be an unsuitable candidate for receiving dexmedetomidine, e.g., subjects with a history of allergic reactions to dexmedetomidine. 14. Subjects with a history of atrioventricular block.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To determine if a single dose of BXCL501, compared to placebo, can effectively reduce symptoms of acute agitation associated with pediatric schizophrenia and bipolar disorder.;Primary end point(s): The primary efficacy endpoint will be the absolute change from baseline in the Positive and Negative Syndrome Scale – Excited Component (PEC) total score at 2 hours.;Secondary Objective: Key Secondary Objective: •To determine the earliest time where an effect on agitation is apparent. Other Secondary Objectives: •To further determine the efficacy, safety, tolerability, and pharmacokinetics (PK) of BXCL501 for acute agitation associated with pediatric schizophrenia and bipolar disorder.;Timepoint(s) of evaluation of this end point: 2 hours after IMP administration. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): The key secondary efficacy endpoint will be the absolute change from baseline in the PEC total score at 90, 60, 45, 30, 20, and 10 minutes. Other Secondary Endpoints: 1. Overall clinical improvement after study drug administration as measured by the Clinical Global Impression-Improvement (CGI-I) score. 2. Duration of calming effect as described by the change from baseline in PEC total score, and Agitation-Calmness Evaluation Scale (ACES) score at 2, 4, and 8 hours after dosing. 3. The effect on overall psychotic symptoms and subscales (Positive and Negative Syndrome Scale [PANSS] total, positive, negative, and general psychopathology subscales). 4. Change from baseline in total PEC score over time measured from 10 minutes through 24 hours after dosing. 5. PEC responders and CGI-I responders at 2 hours following administration of BXCL501 compared with placebo: a. PEC responders will be defined as those who achieve at least a 40% reduction in PEC total score from baseline at or before 2 hours post-dose. b. CGI-I responders will be defined as subjects with a score of 1 or 2 on the CGI-I scale (CGI-I non-responders will be defined as subjects with scores from 3 to 7 at 2 hours post-dose). 6. Time to rescue medication during the entire 24-hour Post-treatment Evaluation Period for subjects receiving BXCL501 compared to placebo. 7. Number of subjects per treatment group who received rescue medication within 4 hours after dosing and within 24 hours after study drug administration. 8. The safety profile of BXCL501 as measured by reports of vital signs and treatment-emergent adverse events (TEAEs). 9. Characteristics of the patient population as assessed by the Young Mania Rating Scale (YMRS) (for bipolar disorder-only). 10. The overall tolerability in terms of TEAE reports and local site (sublingual/buccal) tolerability of oral film. 11. Descriptive PK of BXCL501 in the subject population. 12. Subject acceptability, taste, and likability of study med | — |
Countries
Spain, United States
Contacts
Public ContactFrancisco Cifuentes
Experior S.L.
Outcome results
None listed