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

Inclusion criteria

• 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.

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:
• 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.
• 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.