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

• Status: Not yet recruiting
• Trial end date: 2028-12-01
• Target enrollment: 0
• Participant gender: All

Summary

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.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: NYU Langone Health
• Treatments: Dexmedetomidine

Criteria

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.