Dexmedetomidine for Familial Dysautonomia

Recruiting in Palo Alto (17 mi)

Overseen byAlejandra Gonzalez-Duarte, MD

Age: Any Age

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Waitlist Available

Sponsor: NYU Langone Health

Prior Safety Data

Approved in 4 Jurisdictions

Trial Summary

What is the purpose of this trial?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

Eligibility Criteria

This trial is for individuals aged 16 or older with a genetic diagnosis of Familial Dysautonomia who've had an autonomic crisis in the past year. They must be willing to follow study procedures, use condoms if sexually active, have a responsible caretaker, and provide informed consent. Excluded are those with low oxygen levels, high respiratory rates, very low blood pressure, fever/infection signs recently or pregnant women.

Inclusion Criteria

I have a genetic diagnosis of Familial Dysautonomia.

I am 16 years old or older.

I use condoms for birth control if I am sexually active.

+5 more

Exclusion Criteria

The Montreal Cognitive Exam (MoCA) is below 25 points

My oxygen levels are below 92% without extra oxygen or I need more oxygen than before.

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

+5 more

Participant Groups

The study tests dexmedetomidine as a sublingual film against a placebo to manage hyperadrenergic autonomic crises in patients with Familial Dysautonomia. It aims to see if it's feasible and whether heart rate, blood pressure and oxygen saturation can predict the onset of these crises.

2Treatment groups

Experimental Treatment

Placebo Group

Group I: Sublingual dexmedetomidineExperimental Treatment1 Intervention

Participants will be administered 120 micrograms sublingual film following start of an autonomic crisis. In case the crisis has not subsided after 2 hours, the patient will be instructed to take the second dose. The maximum amount for the study is two oral films within two hours for each episode but not more than 24 hours apart, one at the beginning of the crisis and if needed, one additional within two hours.

Group II: Matching Sublingual PlaceboPlacebo Group1 Intervention

Participants will be administered the matching placebo sublingual film following start of an autonomic crisis. In case the crisis has not subsided after 2 hours, the patient will be instructed to take the second dose. The maximum amount for the study is two oral films within two hours for each episode but not more than 24 hours apart, one at the beginning of the crisis and if needed, one additional within two hours.

Dexmedetomidine is already approved in European Union, United States, Canada, Japan for the following indications:

🇪🇺 Approved in European Union as Precedex for: