Pilot Study of Dexmedetomidine Sublingual Film for the Ambulatory Treatment of Hyperadrenergic Autonomic Crisis in Patients With Familial Dysautonomia
This is a pilot open-label study to evaluate the feasibility of conducting a clinical trial using sublingual dexmedetomidine sublingual film to treat hyperadrenergic autonomic crises in patients with Familial Dysautonomia at home. The primary aims are to examine the feasibility of performing a clinical trial using dexmedetomidine at home to terminate autonomic crisis, and refine the interventions and assessments used to evaluate autonomic crisis termination.
• 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.