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Agitation,PsychomotorPhase 1/2TRANQUILITYNCT04251910

Sub-Lingual Dexmedetomidine in Agitation Associated With Dementia

A Phase 1/2 study testing Sublingual film containing Dexmedetomidine in people with Agitation,Psychomotor and Dementia, run by BioXcel Therapeutics Inc at 5 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

This is an adaptive Phase 1b/2 trial design. It is randomized, double-blind, placebo-controlled, multiple ascending dose study assessing efficacy, pharmacokinetics, safety and tolerability of BXCL-501 dosing in adult (65 years and older) males and females with acute agitation associated with dementia. Read more about this study

Who is it for?
Ages 65 years and older
Conditions the study is for
Agitation,Psychomotor, Dementia
What is being tested?
Sublingual film containing Dexmedetomidine
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
100 participants
Where
5 sitesUnited States
Study dates
27 Dec 2019to 24 Jan 2022
Phase
Phase 1/2

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 65 years and older
  • All sexes
  • No healthy volunteers
  • Older adult

Who may be able to join

  • Male and female patients 65 years and older.
  • Patients who have dementia and a history of acute agitation.
  • History of agitation that requires intervention or impairs social or daily activities
  • Patients who meet International Psychogeriatric Association (IPA) diagnostic criterion for agitation.
  • Patients with a total score of ≥ 8 on the Pittsburgh Agitation Scale (PAS).
  • Patients who have a score of ≥ 2 on at least 1 of the 4 items on the Pittsburgh Agitation Scale (PAS).
  • Patients who read, understand and provide written informed consent, or who have a Legally Authorized Representative (LAR).
  • Patients who are in good general health.

Who cannot join

  • For Part B: Patients with dementia associated with Parkinson's disease and/or Lewy Body Disease, if etiology of dementia is known.
  • Patients with agitation caused by acute intoxication.
  • Patients treated within 4 hours prior to study drug administration with benzodiazepines, other sedatives, hypnotics or oral or short-acting intramuscular antipsychotics must be excluded.
  • Treatment with alpha-1 noradrenergic blockers, alpha adrenergic antagonists within 8 hours prior to dosing.
  • No new chronic medications initiated in the past 14 days prior to screening excluding over-the-counter products taken sporadically.
  • Patients at significant risk of harm to themselves or others
Show all 13Show fewer
  • Patients considered medically unstable or in recovery
  • Patients with history of clinically significant syncope or syncopal attacks, orthostatic hypotension within the past 2 years, current evidence of hypovolemia, orthostatic hypotension.
  • Cohort 3 only: Patients who are taking nitrates or beta blockers shall be excluded. Any other anti-hypertensives should be maintained in the course of the study.
  • Patients who have received an investigational drug within 30 days prior to the current agitation episode must be excluded.
  • Patients experiencing clinically significant pain, per Investigator.
  • Cohort 3 only: Patients who are a high fall risk assessed via the Johns Hopkins Fall Risk Assessment (total score >13) or during the 1-week safety observation period
  • Pregnancy

Trial locations and contacts

5 sites in 1 country.

5 sites in 1 country

United States5 sites
  • BioXcel Clinical Research Site

    Caro, Michigan, 48723

  • BioXcel Clinical Research Site

    Homestead, Florida, 33032

  • BioXcel Clinical Research Site

    Miami Lakes, Florida, 33016

  • BioXcel Clinical Research Site

    Springfield, Massachusetts, 01103

  • BioXcel Clinical Research Site

    Toms River, New Jersey, 08755

Other trials in United States

About this study

Official title: A Phase Ib/II, Multicenter, Randomized, Double Blind, Placebo Controlled, Ascending Dose Finding, Efficacy, Pharmacokinetic and Safety Study of BXCL501 In Agitation Associated With Dementia

This is an adaptive Phase 1b/2 trial design. It is randomized, double-blind, placebo-controlled, multiple ascending dose study assessing efficacy, pharmacokinetics, safety and tolerability of BXCL-501 dosing in adult (65 years and older) males and females with acute agitation associated with dementia. Evaluation of 3 doses are planned.

Read the detailed description

This is a Phase 1b/2 randomized, double-blind, placebo controlled, ascending dose finding study assessing efficacy, pharmacokinetics, safety, and tolerability of BXCL501 with 3 dosing groups in adult (65 years and older) males and females with acute agitation associated with all forms of dementia. Evaluation of three (3) doses of sublingual BXCL 501 are planned. Cohort 1, Cohort 2 and Cohort 3 will be given 30µg, 60µg and 90µg dose respectively of BXCL501 or placebo. Subjects assigned to Cohort 3 will participate in a 1-week safety observation before being enrolled.

This is an adaptive design as doses selected for testing may be different from these, based upon safety reviews. Doses lower or higher may be chosen to test, up to 180µg, and additional subjects may be added to a cohort. BXCL501 films may be divided in half if needed to deliver half-dose strengths. At least 30 subjects (10 per cohort) will be enrolled at up to 3 study sites in the United States. In Part B a total of 46 subjects will receive BXCL501 40 μg or matching placebo film.

The effects of BXCL 501 on acute agitation will be assessed by the following scales: Pittsburgh Agitation Scale (PAS), the PANSS-EC (PEC), Cohen-Mansfield Agitation Inventory (CMAI), CGI-Severity for Agitation and CGI Improvement for Agitation. Adverse Events (AEs), clinical laboratory tests, ECG, and vital signs will be monitored, and all observed.

Being tested
Sublingual film containing Dexmedetomidine, Sublingual Placebo Film
Medical subject headings
Psychomotor Agitation, Dementia

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Sublingual film containing DexmedetomidineDrug

    Also called BXCL501

    Sublingual film containing Dexmedetomidine

  • Sublingual Placebo FilmDrug

    Sublingual placebo film that matches BXCL501

Study groups in NCT04251910
Study groupRoleWhat they receive
Cohort 1- 30 MicrogramsActive comparatorCohort 1 consists of 10 patients out of whom 8 patients receive 30 Micrograms film and the remaining 2 patients receive a placebo
Receives: Drug: Sublingual film containing Dexmedetomidine; Drug: Sublingual Placebo Film
Cohort 2- 60 MicrogramsActive comparatorCohort 2 consists of 10 patients out of whom 8 patients receive 60 Micrograms film and the remaining 2 patients receive a placebo. Additional 20 subjects receive 60 Micrograms or placebo.
Receives: Drug: Sublingual film containing Dexmedetomidine; Drug: Sublingual Placebo Film
Cohort 3- 90 MicrogramsActive comparatorCohort 3 consists of 10 patients out of whom 8 patients receive 90 Micrograms film and the remaining 2 patients receive a placebo
Receives: Drug: Sublingual film containing Dexmedetomidine; Drug: Sublingual Placebo Film
Part B CohortActive comparatorPart B cohort consists 46 subjects receiving 40 Micrograms or placebo
Receives: Drug: Sublingual film containing Dexmedetomidine; Drug: Sublingual Placebo Film

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Mean Change From Baseline in Positive and Negative Syndrome Scale-Excited Component (PEC) Total Score

    The change in PEC score was evaluated at 2 hours following the administration of the BXCL501 60 mcg, and BXCL501 30 mcg (for Part A) and BXCL501 40 mcg (for Part B) versus placebo. PEC is the sum of 5 subscales (poor impulse control, tension, hostility, uncooperativeness, and excitement, each subscale ranging from 1 to 7) and thus ranges from 5 to 35. Change from baseline (pre-dose) PEC total score, with negative values is in favor of improvement.

    Measured: Baseline and 2 hours post-dose

  2. Number of Patients With Adverse Events

    The safety and tolerability of single doses of BXCL501 was determined in treatment of acute agitation associated with dementia.

    Measured: Day 7 post dose

Secondary outcomes (10)
  1. Change in Pittsburgh Agitation Scale (PAS) Total Score From Baseline

    The onset and magnitude of calming effects of different doses of BXCL501 on symptoms of acute agitation associated with dementia was described as measured by the PAS. The PAS is an instrument that measured 4 behaviors namely: aberrant vocalization, motor agitation, aggressiveness and resisting to care. The patients are evaluated on a scale of 0 to 4, where 0 indicated no agitation and 4 indicated highest form of agitation. The PAS total score ranges from 0 to 16. Higher scores mean a worse outcome. Change in value of PAS total score, with negative value indicated the improvement in condition of the patients.

    Measured: Baseline and at 30 minutes, 1 hour, 2 hours, 4 hours, 8 hours, 24 hours, Day 3, Day 7 post-dose

  2. Changes in Agitation-Calmness Evaluation Scale (ACES) Score From Baseline

    To evaluate the change in the total score of ACES from baseline to 8 hours post administration of 30 mcg, 60 mcg and 40 mcg compared to placebo. The ACES is a single item measure rating overall agitation and sedation which ranges from 1 to 9, where 1 indicates marked agitation, 2 - moderate agitation; 3 - mild agitation; 4 - normal behavior; 5 - mild calmness; 6 - moderate calmness; 7 - marked calmness; 8 - deep sleep; and 9 - unarousable. Change from baseline (pre-dose) ACES total score, with negative values in favor of improvement.

    Measured: Baseline and 1 hour, 2 hours, 4 hours, 8 hours post-dose

  3. Changes in Positive and Negative Syndrome Scale (PANSS) Excited Component (PEC) Total Score From Baseline

    The change in PEC score was evaluated following the administration of the BXCL501 60 mcg, and BXCL501 30 mcg (for Part A) and BXCL501 40 mcg (for Part B) versus placebo. PEC is the sum of 5 subscales (poor impulse control, tension, hostility, uncooperativeness, and excitement, each subscale ranging from 1 to 7) and thus ranges from 5 to 35. Change from baseline (pre-dose) PEC total score, with negative values is in favor of improvement.

    Measured: Baseline and at 30 minutes, 1 hour, 4 hours, 8 hours, 24 hours, Day 3 and Day 7 post-dose

  4. Number of Patients at Each Dose Who Achieve a 40% Reduction From Baseline in PEC Total Score at 2 Hours Post-dose ("Responders")

    The Number of patients who achieved a 40%reduction in total PEC score from baseline at 2 hours following administration of BXCL501 30 mcg, 60 mcg (for Part A) and BXCL501 40 mcg (for Part B) compared to placebo were evaluated. Responder defined as achieving >= 40% reduction in PEC from baseline (pre-dose). The change from baseline in (pre-dose) PEC total score is presented for the Primary Outcome above.

    Measured: Baseline and 2 hours post-dose

  5. Change in Clinician Global Impression of Severity (CGI-S) Agitation Score From Baseline

    The CGI-S was based upon the severity of agitation. It was assessed based on the following scale: 0 = Not assessed; 1 = Normal not at all symptomatic; 2 = Minimally symptomatic- few or mild symptoms -little interference with patients functioning; 3 = Mildly symptomatic-low level of symptoms-little interference in social functioning; 4 = Moderately symptomatic-some prominent symptoms-some interference in functioning; 5 = Markedly symptomatic-significant symptoms with very substantial interference in functioning; 6 = Severely symptomatic- very marked symptoms make it difficult for patients to engage with others; 7 = Among the most extremely symptomatic patients-extreme symptoms -patient is incapacitated or highly dangerous to self or others requires extra care and supervision. The higher the score, the higher is the severity of the agitation and lesser the score, the lower the agitation. Change from baseline CGIS total score, with negative values in favor of improvement.

    Measured: Baseline and at 2 hours, and 24 hours post-dose

  6. Clinical Global Impression - Improvement (CGI-I) Agitation Score

    To evaluate the CGI-I agitation score at 30 minutes, 1 hour, 2 hours, and 8 hours after administration of 30 mcg, 60 mcg and 40 mcg of BXCL501 compared to placebo. The CGI-I scores range from 1 to 7 comprise of 0 = Not assessed (missing), 1 = Very much improved, 2 = Much improved, 3 = Minimally improved, 4 = No change, 5 = Minimally worse, 6 = Much worse, 7 = Very much worse. The lower score (1) indicated the improvement in the condition of patient and higher score (7) indicates the worsening of the condition. Straight CGI-I total score, with lower values in favor of improvement.

    Measured: 30 minutes, 1 hour, 2 hours, 4 hours, 8 hours post-dose

  7. Change in Cohen Mansfield Agitation Inventory (CMAI) Total Score From Baseline

    To evaluate the change in Cohen Mansfield Agitation Inventory (CMAI) total score from baseline after 2 hour and on Day 7 post administration of 30 mcg, 60 mcg and 40 mcg BXCL501 compared to placebo. The CMAI is a rating which is comprised of 29 behaviors each rated on a 7-point scale of frequency. A total CMAI score is obtained by summing all the individual items, giving a range from 29 to 203. Change from baseline (pre-dose) CMAI total score, with negative values in favor of improvement in the condition of the patients.

    Measured: Baseline and at 2 Hours and Day 7 post-dose

  8. Number of Patients With Event "Time Taken for Medication to Dissolve"

    To evaluate the time taken for BXCL501 30 mcg, 60mcg, 90 mcg and 40 mcg compared to placebo to dissolve which was measured after 30 minutes of administration.

    Measured: At 30 minutes post-dose

  9. Number of Patients Showing Negative Reaction to Sublingual Film in the Examiner's Opinion

    To evaluate the number patient showing negative reactions to sublingual film by assessing the buccal at 30 minutes, 2 hours, 4 hours and 24 hours post administration of 30mcg, 60 mcg, 40 mcg and 90 mcg v/s placebo. The larger number of patients reporting negative reaction, the lesser is the reliability of the drug.

    Measured: At 30 minutes, 2 hours, 4 hours, 24 hours post dose

  10. Part B: Change From Baseline in the Total Score of 3 Supplementary Items of Positive and Negative Syndrome Scale (PANSS)

    To evaluate the change in PANSS Supplementary Items Total Score from Baseline to 2 hours post administration of 40 mcg of BXCL501 compared to placebo. PANSS Supplementary Items: The total score (sum score of anger, difficulty in delaying gratification, and affective lability) ranges from 3 to 21. The higher score indicates the worsening of the condition and lower score indicates the improvement of the condition of the patient. Change from baseline (pre-dose) PANSS Supplementary Items total score, with negative values in favor of improvement.

    Measured: Baseline and at 2 hours post-dose

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted5 Feb 2020
  2. Study start27 Dec 2019
  3. Primary completion24 Jan 2022
  4. Study completion24 Jan 2022

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1/2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
100 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04251910
Sponsor's protocol ID
BXCL501-103
Acronym
TRANQUILITY
Status last verified
Aug 2026
First submitted
28 Jan 2020
Last updated
11 Aug 2026
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No

Publications

  • Persson NDA, Uusalo P, Nedergaard M, Lohela TJ, Lilius TO. Could dexmedetomidine be repurposed as a glymphatic enhancer? Trends Pharmacol Sci. 2022 Dec;43(12):1030-1040. doi: 10.1016/j.tips.2022.09.007. Epub 2022 Oct 21.

    PubMed 36280451

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