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 withoutsignificant side effects, and registered medical data within the year preceding thestudy 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 responsiblecaregiver.

• Able to state willingness to comply with all study procedures and availability forthe duration of the study

• For males and females of reproductive potential: use condoms for contraception ifsexually active.

Exclusion Criteria:

• At the consideration of the principal investigator, the caregiver cannot fullyunderstand the protocol or communicate during the crisis with the Center.

• The patient during the crisis, before taking the medication, has any of thefollowing:

• a. Oxygen saturation less than 92% on room air or baseline need for oxygen, changefrom 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, orabove 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.