By InvitationPhase 2

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

United States15 participantsStarted 2024-07-01

Plain-language summary

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

Who can participate

Age range

16 Years – 80 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Exclusion criteria

. 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.

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Percentage of patients with reduction of Autonomic crisis severity assessment scores (ACSAS) to ≤ 5 points

Timeframe: Up to 2 hours post administration

Percentage of patients with 25% reduction in blood pressure

Timeframe: Up to 2 hours post administration

Percentage of patients with >20% reduction in heart rate

Timeframe: Up to 2 hours post administration

Percentage of patients with >50% reduction in vomiting/retching episodes

Timeframe: Up to 2 hours post administration

Trial details

NCT IDNCT06148311

SponsorNYU Langone Health

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2027-01-01

View on ClinicalTrials.gov More Familial Dysautonomia trials

Plain-language summary

Who can participate

Age range

16 Years – 80 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Exclusion criteria

. 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.

What they're measuring

Percentage of patients with reduction of Autonomic crisis severity assessment scores (ACSAS) to ≤ 5 points

Timeframe: Up to 2 hours post administration

Percentage of patients with 25% reduction in blood pressure

Timeframe: Up to 2 hours post administration

Percentage of patients with >20% reduction in heart rate

Timeframe: Up to 2 hours post administration

Percentage of patients with >50% reduction in vomiting/retching episodes

Timeframe: Up to 2 hours post administration