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NCT06148311

Dexmedetomidine Sublingual Film for the Ambulatory Treatment of Hyperadrenergic Autonomic Crisis in Patients With Familial Dysautonomia

Enrolling by Invitation
PHASE2Ages 16–80InterventionalTreatment
NYU Langone Health
~15 participants
Updated 2026-04-13 on ClinicalTrials.gov
What's tested:Matching placeboDexmedetomidine

At a glance

Recruiting sites
0 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Percentage of patients with reduction of Autonomic crisis severity assessment scores (ACSAS) to ≤ 5 points
Measured over Up to 2 hours post administration
+3 more outcomes measured

Conditions

Familial Dysautonomia

Explore related trials

Familial Dysautonomia

Where it's being run

1 sites across 1 states
New York1

Study leadership

  • Alejandra Gonzalez-Duarte, MD · PRINCIPAL_INVESTIGATOR · NYU Langone Health, NYU Dysautonomia Center

Who to contact

This trial hasn't published a contact. View it on ClinicalTrials.gov

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Eligibility criteria

Inclusion

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.

Exclusion

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:
The patient is a female and has a positive pregnancy test.
The Montreal Cognitive Exam (MoCA) is below 25 points.

What this trial measures

  • Percentage of patients with reduction of Autonomic crisis severity assessment scores (ACSAS) to ≤ 5 pointsUp to 2 hours post administration

    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).

  • Percentage of patients with 25% reduction in blood pressureUp to 2 hours post administration

    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.

  • Percentage of patients with >20% reduction in heart rateUp to 2 hours post administration

    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.

  • Percentage of patients with >50% reduction in vomiting/retching episodesUp to 2 hours post administration

    Patients will be monitored by their care givers for of episodes of vomiting/ retching before taking each dose and up to 2 hours after.