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Enrolling by Invitation

NCT Number: NCT06148311

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

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

Enrolling by Invitation

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Key information

Conditions

Familial Dysautonomia Autonomic Nervous System Diseases Congenital Abnormalities Congenital, Hereditary, and Neonatal Diseases and Abnormalities Dysautonomia, Familial Genetic Diseases, Inborn Hereditary Sensory and Autonomic Neuropathies Heredodegenerative Disorders, Nervous System Nervous System Diseases Nervous System Malformations Neurodegenerative Diseases Neuromuscular Diseases Peripheral Nervous System Diseases Polyneuropathies Primary Dysautonomias

Age range

16 year–80 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

NYU Langone Health

New York, 10016, United States

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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 criteria

  • 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.
  • 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.

Treatment and study plan

Matching Placebo

Drug

One matching placebo will be given under the tongue on a thin dissolvable film.

Dexmedetomidine

Drug

Dexmedetomidine 120 mcg will be given under the tongue on a thin dissolvable film.

Primary outcomes

  1. Percentage of patients with reduction of Autonomic crisis severity assessment scores (ACSAS) to ≤ 5 points

    Time frame: Up 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).

  2. Percentage of patients with 25% reduction in blood pressure

    Time frame: Up 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.

  3. Percentage of patients with >20% reduction in heart rate

    Time frame: Up 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.

  4. Percentage of patients with >50% reduction in vomiting/retching episodes

    Time frame: Up 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.

Secondary outcomes

  1. Percentage of patients with ≥ 20% reduction in hospitalizations

    Time frame: Up to 48 months

    Study team will compare historical data to number of hospitalizations occurred after taking one or more doses.

  2. Percentage of patients with ≥ 20% reduction in hospital stay duration

    Time frame: Up to 48 months

    Study team will compare historical data to number of days during hospital stay that occurred after taking one or more doses.

  3. Percentage of patients with ≥ 30% reduction in ICU stay duration

    Time frame: Up to 48 months

    Study team will compare historical data to number of days at the ICU that occurred after taking one or more doses.

  4. Change in number of medical complications

    Time frame: Baseline, up to 48 months

    Study team will compare historical data to number of medical complications of hospitalizations occurred after taking one or more doses.

Sponsors and collaborators

Lead sponsor

NYU Langone Health

Other

Collaborators

  • Food and Drug Administration (FDA)

Registry information

Important dates

Study start
2024
Primary completion
2027
Study completion
2027
First posted
Nov 28, 2023
Registry last updated
Apr 13, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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