AgitationPhase 1NCT04010305
Sub-Lingual Dexmedetomidine in Agitation Associated With Schizophrenia
A Phase 1 study testing Sublingual film containing BXCL501 (Dexmedetomidine) in people with Agitation and Schizophrenia, run by BioXcel Therapeutics Inc at 12 sites in United States.
Trial summary
This is a two-stage adaptive Phase Ib trial design, that will identify two doses (lowest dose with clinical benefit and highest safe dose) in a first stage and better evaluate safety, tolerability and variability of effect in the second stage. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Agitation, Schizophrenia
- What is being tested?
- Sublingual film containing BXCL501 (Dexmedetomidine)
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 135 participants
- Where
- 12 sitesUnited States
- Study dates
- 22 May 2019to 31 Jul 2019
- Phase
- Phase 1
- Are there results?
- None posted in the registry
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 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Male and female patients between the ages of 18 to 65 years, inclusive.
- Patients who have met Diagnostic and Statistical Manual (DSM) -5 criteria for schizophrenia, schizoaffective, or schizophreniform disorder.
- Patients who are judged to be clinically agitated at 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).
- Patients who have a score of ≥ 4 on at least 1 of the 5 items on the PEC.
- Patients who read, understand and provide written informed consent.
- Patients who are in good general health prior to study participation as determined by a detailed medical history, physical examination, 12-lead ECG, blood chemistry profile, hematology, urinalysis and in the opinion of the Principal Investigator.
- Female participants, if of child-bearing potential and sexually active, and male participants, if sexually active with a partner of child-bearing potential, who agree to use a medically acceptable and effective birth control method throughout the study and for one 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 (IUD), 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.
Who cannot join
- Patients with agitation caused by acute intoxication, including positive identification of alcohol by breathalyzer or non-prescription drugs (with the exception of THC) during urine screening.
- Patients treated within 4 hours prior to study drug administration with benzodiazepines, other hypnotics or oral or short-acting intramuscular antipsychotics.
- Treatment with alpha-1 noradrenergic blockers (terazosin, doxazosin, tamsulosin, and alfuzosin, and prazosin) or other prohibited medications.
- Patients with significant risk of suicide or homicide per the investigator's assessment, or any suicidal behavior in last 6 months prior to screening.
- Female patients who have a positive pregnancy test at screening or are breastfeeding.
- Patients who have hydrocephalus, seizure disorder, or history of significant head trauma, stroke, transient ischemic attack, subarachnoid bleeding, brain tumor, encephalopathy, meningitis, Parkinson's disease or focal neurological findings.
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- History of syncope or other syncopal attacks, current evidence of hypovolemia, orthostatic hypotension, a screening heart rate of < 55 beats per minutes or systolic blood pressure <110 mmHg or diastolic BP <70 mmHg.
- Patients with laboratory or ECG abnormalities considered clinically significant by the investigator or qualified designee [Advanced heart block (second-degree or above atrioventricular block without pacemaker), diagnosis of Sick sinus syndrome] that would have clinical implications for the patient's participation in the study.
- Patients with serious or unstable medical illnesses. These include current hepatic (moderate-severe hepatic impairment), renal, gastroenterologic, respiratory, cardiovascular (including ischemic heart disease, congestive heart failure), endocrinologic, or hematologic disease.
- Patients who have received an investigational drug within 30 days prior to the current agitation episode.
- Patients who are unable to use the sublingual film or considered by the investigator, for any reason, to be an unsuitable candidate for receiving dexmedetomidine; e.g. patients with a history of allergic reactions to dexmedetomidine.
Trial locations and contacts
12 sites in 1 country.
12 sites in 1 country
United States12 sites
BioXcel Clinical Research Site
Atlanta, Georgia, 30331
BioXcel Clinical Research Site
Austin, Texas, 78754
BioXcel Clinical Research Site
Berlin, New Jersey, 08009
BioXcel Clinical Research Site
Cerritos, California, 90703
BioXcel Clinical Research Site
Flowood, Mississippi, 39232
BioXcel Clinical Research Site
Gaithersburg, Maryland, 20877
BioXcel Clinical Research Site
Lemon Grove, California, 91945
BioXcel Clinical Research Site
Little Rock, Arkansas, 72211
BioXcel Clinical Research Site
Long Beach, California, 90806
BioXcel Clinical Research Site
Miami Lakes, Florida, 33016
BioXcel Clinical Research Site
Orange, California, 92868
BioXcel Clinical Research Site
Richardson, Texas, 75080
About this study
Official title: A Phase Ib Multicenter, Randomized, Double-blind, Placebo-controlled, Multiple Ascending Dose Study to Determine Efficacy, Pharmacokinetics and Safety of BXCL501 in Agitation Associated With Schizophrenia
This is a two-stage adaptive Phase Ib trial design, that will identify two doses (lowest dose with clinical benefit and highest safe dose) in a first stage and better evaluate safety, tolerability and variability of effect in the second stage.
Read the detailed description
This is a two-stage adaptive Phase Ib trial design. It is a randomized, double-blind, placebo-controlled, multiple ascending dose study assessing efficacy, pharmacokinetics, safety and tolerability of BXCL501 dosing in adult (18-65 years old) males and females with acute agitation associated with schizophrenia, schizoaffective disorder, or schizophreniform disorder.
The first stage will characterize a safe and tolerable dose range that results in a calming effect as measured using the PEC total score. The lowest dose with clear clinical benefit, and the highest safe and well-tolerated dose that demonstrates efficacy in a large proportion of subjects will be selected over the course of testing multiple escalating dose cohorts. The second stage will comprise a total of 40 subjects per dose group in a three-arm placebo-controlled design in order to better characterize the broader range of safety and tolerability as well as better estimate variability (effect size) which may be observed in later phase placebo-controlled trials.
- Conditions
- Agitation, Schizophrenia
- Being tested
- Sublingual film containing BXCL501 (Dexmedetomidine), Placebo film
- Medical subject headings
- Psychomotor Agitation, Schizophrenia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Sublingual film containing BXCL501 (Dexmedetomidine)Drug
Also called Dexmedetomidine
Sublingual film containing BXCL501 (Dexmedetomidine) for the treatment of agitation associated with Schizophrenia
Placebo filmDrug
Also called Placebo
Placebo film for BXCL501
| Study group | Role | What they receive |
|---|---|---|
| Placebo | Placebo comparator | Sublingual Film with no active drug; single administration with the possibility of repeating dose after 1 hour in case of lack of significant efficacy Receives: Drug: Placebo film |
| 20 micrograms | Experimental | Sublingual Film containing 20 micrograms BXCL501; single administration with the possibility of repeating dose after 1 hour in case of lack of significant efficacy Receives: Drug: Sublingual film containing BXCL501 (Dexmedetomidine) |
| 60 micrograms | Experimental | Sublingual Film containing 60 micrograms BXCL501; single administration with the possibility of repeating dose after 1 hour in case of lack of significant efficacy Receives: Drug: Sublingual film containing BXCL501 (Dexmedetomidine) |
| 120 micrograms | Experimental | 2 Sublingual Films, each containing 60 micrograms BXCL501; single administration of 2 films with the possibility of repeating dose after 1 hour in case of lack of significant efficacy Receives: Drug: Sublingual film containing BXCL501 (Dexmedetomidine) |
| 180 micrograms | Experimental | 2 Sublingual Films, each containing 60 micrograms BXCL501. Receives: Drug: Sublingual film containing BXCL501 (Dexmedetomidine) |
What is being measured
The primary outcome is the main question the study is designed to answer.
PANSS-EC Change From Baseline
Positive and Negative Syndrome Scale - Excited Component (PEC). The 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 to 35.
Measured: 120 minutes
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Jul 2019
- Study start22 May 2019
- Primary completion31 Jul 2019
- Study completion31 Jul 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for AgitationStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- This is a two-stage adaptive Phase Ib trial design. It is a randomized, double-blind, placebo-controlled, multiple ascending dose study.
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 135 participants (actual)
Sponsor and investigators
- Lead sponsor
- BioXcel Therapeutics Inc
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Cognitive Research Corporation
- Responsible party
- Sponsor
- Investigators
- BioXcel MM, MD, Study chair, BioXcel Therapeutics
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04010305
- Sponsor's protocol ID
- BXCL501-102
- Status last verified
- Aug 2026
- First submitted
- 3 Jul 2019
- Last updated
- 11 Aug 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 11 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase Ib Multicenter, Randomized, Double-blind, Placebo-controlled, Multiple Ascending Dose Study to Determine Efficacy, Pharmacokinetics and Safety of BXCL501 in Agitation Associated With Schizophrenia
- ClinicalTrials.gov ID
- NCT04010305
- Acronym / sponsor ID
- BXCL501-102
- Conditions
- Agitation, Schizophrenia
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 135 participants (actual)
- Lead sponsor
- BioXcel Therapeutics IncIndustry (pharma, biotech, devices)
- Collaborators
- Cognitive Research Corporation
- Study start
- 22 May 2019 (actual)
- Primary completion
- 31 Jul 2019 (actual)
- Study completion
- 31 Jul 2019 (actual)
- First posted
- 8 Jul 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a two-stage adaptive Phase Ib trial design, that will identify two doses (lowest dose with clinical benefit and highest safe dose) in a first stage and better evaluate safety, tolerability and variability of effect in the second stage.
Detailed description
This is a two-stage adaptive Phase Ib trial design. It is a randomized, double-blind, placebo-controlled, multiple ascending dose study assessing efficacy, pharmacokinetics, safety and tolerability of BXCL501 dosing in adult (18-65 years old) males and females with acute agitation associated with schizophrenia, schizoaffective disorder, or schizophreniform disorder.
The first stage will characterize a safe and tolerable dose range that results in a calming effect as measured using the PEC total score. The lowest dose with clear clinical benefit, and the highest safe and well-tolerated dose that demonstrates efficacy in a large proportion of subjects will be selected over the course of testing multiple escalating dose cohorts. The second stage will comprise a total of 40 subjects per dose group in a three-arm placebo-controlled design in order to better characterize the broader range of safety and tolerability as well as better estimate variability (effect size) which may be observed in later phase placebo-controlled trials.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 5
- Model description
- This is a two-stage adaptive Phase Ib trial design. It is a randomized, double-blind, placebo-controlled, multiple ascending dose study.
| Arm | Type | Receives |
|---|---|---|
| Placebo | Placebo comparator | Drug: Placebo filmSublingual Film with no active drug; single administration with the possibility of repeating dose after 1 hour in case of lack of significant efficacy |
| 20 micrograms | Experimental | Drug: Sublingual film containing BXCL501 (Dexmedetomidine)Sublingual Film containing 20 micrograms BXCL501; single administration with the possibility of repeating dose after 1 hour in case of lack of significant efficacy |
| 60 micrograms | Experimental | Drug: Sublingual film containing BXCL501 (Dexmedetomidine)Sublingual Film containing 60 micrograms BXCL501; single administration with the possibility of repeating dose after 1 hour in case of lack of significant efficacy |
| 120 micrograms | Experimental | Drug: Sublingual film containing BXCL501 (Dexmedetomidine)2 Sublingual Films, each containing 60 micrograms BXCL501; single administration of 2 films with the possibility of repeating dose after 1 hour in case of lack of significant efficacy |
| 180 micrograms | Experimental | Drug: Sublingual film containing BXCL501 (Dexmedetomidine)2 Sublingual Films, each containing 60 micrograms BXCL501. |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Male and female patients between the ages of 18 to 65 years, inclusive.
- Patients who have met Diagnostic and Statistical Manual (DSM) -5 criteria for schizophrenia, schizoaffective, or schizophreniform disorder.
- Patients who are judged to be clinically agitated at 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).
- Patients who have a score of ≥ 4 on at least 1 of the 5 items on the PEC.
- Patients who read, understand and provide written informed consent.
Key exclusion criteria (11 listed)
- Patients with agitation caused by acute intoxication, including positive identification of alcohol by breathalyzer or non-prescription drugs (with the exception of THC) during urine screening.
- Patients treated within 4 hours prior to study drug administration with benzodiazepines, other hypnotics or oral or short-acting intramuscular antipsychotics.
- Treatment with alpha-1 noradrenergic blockers (terazosin, doxazosin, tamsulosin, and alfuzosin, and prazosin) or other prohibited medications.
- Patients with significant risk of suicide or homicide per the investigator's assessment, or any suicidal behavior in last 6 months prior to screening.
- Female patients who have a positive pregnancy test at screening or are breastfeeding.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Sublingual film containing BXCL501 (Dexmedetomidine)Also: DexmedetomidineSublingual film containing BXCL501 (Dexmedetomidine) for the treatment of agitation associated with Schizophrenia | Drug | 120 micrograms, 180 micrograms, 20 micrograms, 60 micrograms |
| Placebo filmAlso: PlaceboPlacebo film for BXCL501 | Drug | Placebo |
Primary outcome
PANSS-EC Change From Baseline
Time frame: 120 minutes
Positive and Negative Syndrome Scale - Excited Component (PEC). The 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 to 35.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Aug 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
BioXcel MM, MD study chair
BioXcel Therapeutics
12 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Sublingual film containing BXCL501 (Dexmedetomidine) in people with Agitation and Schizophrenia, run by BioXcel Therapeutics Inc at 12 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 11 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 12 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
PANSS-EC Change From Baseline (time frame: 120 minutes). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 11 Aug 2026.