Familial DysautonomiaPhase 2NCT06128356
Pilot Study of Dexmedetomidine Sublingual Film for the Ambulatory Treatment of Hyperadrenergic Autonomic Crisis in Patients With Familial Dysautonomia
A Phase 2 study testing Dexmedetomidine Sublingual in people with Familial Dysautonomia, run by NYU Langone Health at 1 site in United States.
Trial summary
This is a pilot open-label study to evaluate the feasibility of conducting a clinical trial using sublingual dexmedetomidine sublingual film to treat hyperadrenergic autonomic crises in patients with Familial Dysautonomia at home. Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Familial Dysautonomia
- What is being tested?
- Dexmedetomidine Sublingual
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 5 participants
- Where
- 1 siteUnited States
- Study dates
- 1 Jun 2024to Dec 2028
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
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 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Genetically confirmed diagnosis of Familial Dysautonomia.
- Evidence of autonomic crisis, previous treatment with IV dexmedetomidine without significant side effects, and registered medical data within the year preceding the study in our database.
- One or more autonomic crises during the last year.
- Age above 18 years.
- The patient has a responsible caretaker to communicate with the medical providers.
- Provision of signed and dated informed consent form from the patient and responsible caregiver.
- Able to state willingness to comply with all study procedures and availability for the duration of the study
- For males and females of reproductive potential: use condoms for contraception if sexually active.
Who cannot join
- At the consideration of the principal investigator, the caregiver cannot fully understand the protocol or communicate during the crisis with the Center.
- The patient during the crisis, before taking the medication, has any of the following:
- a. Oxygen saturation less than 92% on room air or baseline need for oxygen, change from baseline oxygen dependency.
- b. Respiratory rate >20 breaths per minute.
- c. Supine blood pressure ≤ 90/60mmHg
- d. Febrile illness with temperature >100.3 F.
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- e. Serological signs of infection (WBC count >10 g/dL, or CRP >10 mg/L or ESR>20, or above their steady historical baseline levels) in recent (less than one month) studies.
- The patient is a female and has a positive pregnancy test.
- MoCA score <25 points.
Trial locations and contacts
1 site in 1 country.
About this study
This is a pilot open-label study to evaluate the feasibility of conducting a clinical trial using sublingual dexmedetomidine sublingual film to treat hyperadrenergic autonomic crises in patients with Familial Dysautonomia at home. The primary aims are to examine the feasibility of performing a clinical trial using dexmedetomidine at home to terminate autonomic crisis, and refine the interventions and assessments used to evaluate autonomic crisis termination.
- Conditions
- Familial Dysautonomia
- Being tested
- Dexmedetomidine Sublingual
- Medical subject headings
- Dysautonomia, Familial
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Dexmedetomidine SublingualDrug
Also called IGALMI
Dexmedetomidine 120 mcg on two thin dissolvable films for sublingual administration
| Study group | Role | What they receive |
|---|---|---|
| Sublingual dexmedetomidine | Experimental | Participants will be administered two 60 micrograms sublingual films of IGLMI following start of an autonomic crises. The maximum amount for the study is four oral films of 60 micrograms in 24 hours, two at the beginning of the crises and if needed, two additional within two hours. Receives: Drug: Dexmedetomidine Sublingual |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of subjects screened per month
Measured: Month 6
Number of subjects enrolled per month
Measured: Month 6
Number of completed crises treated at home within the protocol
Measured: Month 6
Number of subjects that underwent an autonomic crisis per month
Measured: Month 6
Average duration of the study from visit 1 to the last visit of the last patient
Measured: From enrollment to end of treatment (up to 6 months)
Secondary outcomes (9)
Length of time from autonomic crisis onset to the initiation of the video recording
Measured: Up to 2 hours
Length of time from autonomic crisis onset to administration of the medication
Measured: Up to 2 hours
Length of time from autonomic crisis onset to crisis resolution
Measured: Up to 24 hours
Length of time it took to complete assessments from start of autonomic crisis
Measured: Up to 24 hours
Percentage of completion of all rating scales
The scales required to be completed is the Autonomic Crisis Symptom Assessment Scale (ACSAS), the study safety assessments and the Richmond Agitation-Sedation Scale.
Measured: Month 6
Change in blood pressure
Measured: Pre-dose, up to 2 hours post-dose
Change in heart rate
Measured: Pre-dose, up to 2 hours post-dose
Change in number of vomiting episodes
Measured: Pre-dose, up to 2 hours post-dose
Change in number of retching episodes
Measured: Pre-dose, up to 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.
- First posted13 Nov 2023
- Study start1 Jun 2024
- Primary completionSep 2028 (estimated)
- Study completionDec 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Familial DysautonomiaStudies of the same condition, open and under way.
- More trials run by NYU Langone HealthThe sponsor's other registered studies.
- 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 5 participants (actual)
Sponsor and investigators
- Lead sponsor
- NYU Langone Health
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Alejandra Gonzalez-Duarte, MD, Principal investigator, NYU Langone Health
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06128356
- Sponsor's protocol ID
- 23-00174
- Status last verified
- Mar 2026
- First submitted
- 7 Nov 2023
- Last updated
- 4 Mar 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: The de-identified participant data from the final research dataset used in the published manuscript will be shared upon reasonable request beginning 9 months and ending 36 months following article publication or as required by a condition of awards and agreements supporting the research provided the investigator who proposes to use the data executes a data use agreement with NYU Langone Health. Requests may be directed to: Alejandra.gonzalez-duarte@nyulangone.org. The protocol and statistical analysis plan will be made available on Clinicaltrials.gov only as required by federal regulation or as a condition of awards and agreements supporting the research
Study at a glance
From the registry record, last updated by the sponsor on 4 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pilot Study of Dexmedetomidine Sublingual Film for the Ambulatory Treatment of Hyperadrenergic Autonomic Crisis in Patients With Familial Dysautonomia
- ClinicalTrials.gov ID
- NCT06128356
- Acronym / sponsor ID
- 23-00174
- Conditions
- Familial Dysautonomia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 5 participants (actual)
- Lead sponsor
- NYU Langone HealthUniversity, hospital or other
- Study start
- 1 Jun 2024 (actual)
- Primary completion
- Sep 2028 (estimated)
- Study completion
- Dec 2028 (estimated)
- First posted
- 13 Nov 2023 (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 pilot open-label study to evaluate the feasibility of conducting a clinical trial using sublingual dexmedetomidine sublingual film to treat hyperadrenergic autonomic crises in patients with Familial Dysautonomia at home. The primary aims are to examine the feasibility of performing a clinical trial using dexmedetomidine at home to terminate autonomic crisis, and refine the interventions and assessments used to evaluate autonomic crisis termination.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Sublingual dexmedetomidine | Experimental | Drug: Dexmedetomidine SublingualParticipants will be administered two 60 micrograms sublingual films of IGLMI following start of an autonomic crises. The maximum amount for the study is four oral films of 60 micrograms in 24 hours, two at the beginning of the crises and if needed, two additional within two hours. |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Genetically confirmed diagnosis of Familial Dysautonomia.
- Evidence of autonomic crisis, previous treatment with IV dexmedetomidine without significant side effects, and registered medical data within the year preceding the study in our database.
- One or more autonomic crises during the last year.
- Age above 18 years.
- The patient has a responsible caretaker to communicate with the medical providers.
Key exclusion criteria (9 listed)
- At the consideration of the principal investigator, the caregiver cannot fully understand the protocol or communicate during the crisis with the Center.
- The patient during the crisis, before taking the medication, has any of the following:
- a. Oxygen saturation less than 92% on room air or baseline need for oxygen, change from baseline oxygen dependency.
- b. Respiratory rate >20 breaths per minute.
- c. Supine blood pressure ≤ 90/60mmHg
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 |
|---|---|---|
| Dexmedetomidine SublingualAlso: IGALMIDexmedetomidine 120 mcg on two thin dissolvable films for sublingual administration | Drug | Sublingual dexmedetomidine |
Primary outcome
Number of subjects screened per month
Time frame: Month 6
Number of subjects enrolled per month
Time frame: Month 6
Number of completed crises treated at home within the protocol
Time frame: Month 6
Number of subjects that underwent an autonomic crisis per month
Time frame: Month 6
Average duration of the study from visit 1 to the last visit of the last patient
Time frame: From enrollment to end of treatment (up to 6 months)
9 secondary outcomes
Length of time from autonomic crisis onset to the initiation of the video recording
Time frame: Up to 2 hours
Length of time from autonomic crisis onset to administration of the medication
Time frame: Up to 2 hours
Length of time from autonomic crisis onset to crisis resolution
Time frame: Up to 24 hours
Length of time it took to complete assessments from start of autonomic crisis
Time frame: Up to 24 hours
Percentage of completion of all rating scales
Time frame: Month 6
Change in blood pressure
Time frame: Pre-dose, up to 2 hours post-dose
Change in heart rate
Time frame: Pre-dose, up to 2 hours post-dose
Change in number of vomiting episodes
Time frame: Pre-dose, up to 2 hours post-dose
Change in number of retching episodes
Time frame: Pre-dose, up to 2 hours post-dose
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Mar 2026.
- Individual participant dataSharing planned: Yes. The de-identified participant data from the final research dataset used in the published manuscript will be shared upon reasonable request beginning 9 months and ending 36 months following article publication or as required by a condition of awards and agreements supporting the research provided the investigator who proposes to use the data executes a data use agreement with NYU Langone Health. Requests may be directed to: Alejandra.gonzalez-duarte@nyulangone.org. The protocol and statistical analysis plan will be made available on Clinicaltrials.gov only as required by federal regulation or as a condition of awards and agreements supporting the research
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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.
Alejandra Gonzalez-Duarte, MD principal investigator
NYU Langone Health
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Dexmedetomidine Sublingual in people with Familial Dysautonomia, run by NYU Langone Health at 1 site 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 8 inclusion and 9 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: under way, not looking for participants, as of the sponsor's last update on 4 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
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?
Number of subjects screened per month (time frame: Month 6). The study lists 5 primary outcomes in all. 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 4 Mar 2026.