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

Enrolling by invitation · Phase 2 · Has a placebo group

Conditions studied: Familial Dysautonomia

In brief

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

Key facts

Study ID

NCT06148311

Run by

NYU Langone Health

People needed

15

Starts

2024-07-01

Expected to finish

2027-09-01

Last updated by the study team

2026-04-13

Who can join

Age: 16 and older, up to 80. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Genetically confirmed diagnosis of Familial Dysautonomia.
• One or more autonomic crises during the last year.
• Our database shows evidence of autonomic crisis, previous treatment with IV dexmedetomidine, and registered medical data within the year preceding the study.
• Age 16 years or older
• 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 age: use condoms for contraception if sexually active.

You may not qualify if…

• 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:
• Oxygen saturation less than 92% on room air or baseline need for oxygen, change from baseline oxygen dependency.
• Respiratory rate >25 breaths per minute.
• Supine blood pressure ≤ 90/860mmHg
• Febrile illness with temperature >100.3 F.
• 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.
• The Montreal Cognitive Exam (MoCA) is below 25 points.

Where it is running

• NYU Langone Health — New York, New York, United States

Full record on ClinicalTrials.gov

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