Familial DysautonomiaPhase 2NCT06148311
Dexmedetomidine Sublingual Film for the Ambulatory Treatment of Hyperadrenergic Autonomic Crisis in Patients With Familial Dysautonomia
A Phase 2 study testing Dexmedetomidine in people with Familial Dysautonomia, run by NYU Langone Health at 1 site in United States.
Trial summary
The purpose of this placebo controlled interventional study is to collect preliminary data on administering dexmedetomidine in patients with Familial Dysautonomia (FD) during a rapid cessation of autonomic crisis. Read more about this study
- Who is it for?
- Ages 16 to 80 years
- Conditions the study is for
- Familial Dysautonomia
- What is being tested?
- Dexmedetomidine
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 15 participants
- Where
- 1 siteUnited States
- Study dates
- 1 Jul 2024to 1 Sep 2027
- Phase
- Phase 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 16 to 80 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Genetically confirmed diagnosis of Familial Dysautonomia.
- One or more autonomic crises during the last year.
- Our database shows evidence of autonomic crisis, previous treatment with IV dexmedetomidine, and registered medical data within the year preceding the study.
- Age 16 years or older
- 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 age: 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:
- Oxygen saturation less than 92% on room air or baseline need for oxygen, change from baseline oxygen dependency.
- Respiratory rate >25 breaths per minute.
- Supine blood pressure ≤ 90/860mmHg
- Febrile illness with temperature >100.3 F.
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- 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.
- The Montreal Cognitive Exam (MoCA) is below 25 points.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
About this study
The purpose of this placebo controlled interventional study is to collect preliminary data on administering dexmedetomidine in patients with Familial Dysautonomia (FD) during a rapid cessation of autonomic crisis. The primary aims are to assess the feasibility and evaluate if measurements of heart rate, blood pressure and oxygen saturation can predict the start of an autonomic crisis.
Funding Source- FDA OOPD
- Conditions
- Familial Dysautonomia
- Being tested
- Matching placebo, Dexmedetomidine
- Keywords
- Familial Dysautonomia, Hereditary Sensory and Autonomic Neuropathy type 3, HSAN, autonomic sympathetic crises
- Medical subject headings
- Dysautonomia, Familial, Hereditary Sensory and Autonomic Neuropathies
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Matching placeboDrug
One matching placebo will be given under the tongue on a thin dissolvable film.
DexmedetomidineDrug
Dexmedetomidine 120 mcg will be given under the tongue on a thin dissolvable film.
| Study group | Role | What they receive |
|---|---|---|
| Sublingual dexmedetomidine | Experimental | Participants will be administered 120 micrograms sublingual film following start of an autonomic crisis. In case the crisis has not subsided after 2 hours, the patient will be instructed to take the second dose. The maximum amount for the study is two oral films within two hours for each episode but not more than 24 hours apart, one at the beginning of the crisis and if needed, one additional within two hours. Receives: Drug: Dexmedetomidine |
| Matching Sublingual Placebo | Placebo comparator | Participants will be administered the matching placebo sublingual film following start of an autonomic crisis. In case the crisis has not subsided after 2 hours, the patient will be instructed to take the second dose. The maximum amount for the study is two oral films within two hours for each episode but not more than 24 hours apart, one at the beginning of the crisis and if needed, one additional within two hours. Receives: Drug: Matching placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of patients with reduction of Autonomic crisis severity assessment scores (ACSAS) to ≤ 5 points
ACSAS tool will be used to measure objectively and record signs and symptoms of autonomic crisis, which includes documentation of blood pressure, heart rate, skin flushing and blotching, sweating, and behavioral changes. Scores are totaled at each timepoint (0 minutes, 15 min., 45 min., 1.5 hours, 2hr.) ranging from 0 (Normal) to 16 (most severe symptoms occurred).
Measured: Up to 2 hours post administration
Percentage of patients with 25% reduction in blood pressure
Patients will be monitored with a hospital-at-home (H@H) device to allow visualization of vital signs in real life. The H@H monitor (Biobeat™) is an FDA approved device and will be attached to the patient´s chest by an adhesive patch which will transmit blood pressure. Blood pressure will be monitored before taking each dose and up to 2 hours after.
Measured: Up to 2 hours post administration
Percentage of patients with >20% reduction in heart rate
Patients will be monitored with a hospital-at-home (H@H) device to allow visualization of vital signs in real life. The H@H monitor (Biobeat™) is an FDA approved device and will be attached to the patient´s chest by an adhesive patch which will transmit heart rate. Heart rate will be monitored before taking each dose and up to 2 hours after.
Measured: Up to 2 hours post administration
Percentage of patients with >50% reduction in vomiting/retching episodes
Patients will be monitored by their care givers for of episodes of vomiting/ retching before taking each dose and up to 2 hours after.
Measured: Up to 2 hours post administration
Secondary outcomes (4)
Percentage of patients with ≥ 20% reduction in hospitalizations
Study team will compare historical data to number of hospitalizations occurred after taking one or more doses.
Measured: Up to 48 months
Percentage of patients with ≥ 20% reduction in hospital stay duration
Study team will compare historical data to number of days during hospital stay that occurred after taking one or more doses.
Measured: Up to 48 months
Percentage of patients with ≥ 30% reduction in ICU stay duration
Study team will compare historical data to number of days at the ICU that occurred after taking one or more doses.
Measured: Up to 48 months
Change in number of medical complications
Study team will compare historical data to number of medical complications of hospitalizations occurred after taking one or more doses.
Measured: Baseline, up to 48 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Nov 2023
- Study start1 Jul 2024
- Primary completion1 Jan 2027 (estimated)
- Study completion1 Sep 2027 (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
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 15 participants (estimated)
Sponsor and investigators
- Lead sponsor
- NYU Langone Health
- Sponsor type
- University, hospital or other
- Collaborators
- Food and Drug Administration (FDA)
- Responsible party
- Sponsor
- Investigators
- Alejandra Gonzalez-Duarte, MD, Principal investigator, NYU Langone Health, NYU Dysautonomia Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06148311
- Sponsor's protocol ID
- 22-01332
- Other IDs
- 1R01FD007826-01
- Status last verified
- Apr 2026
- First submitted
- 20 Nov 2023
- Last updated
- 13 Apr 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.