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New dissolving film may stop dangerous episodes in rare nerve disease

NCT ID NCT06148311

Symptom relief Sponsor: NYU Langone Health Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only This study
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tests a medicated film placed under the tongue to quickly ease severe episodes (autonomic crises) in people with familial dysautonomia. The film contains dexmedetomidine, a drug that calms the body's stress response. Researchers will check if it reduces symptoms like high blood pressure, rapid heart rate, and vomiting. The study involves 15 people aged 16 and older who have had at least one crisis in the past year.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

About 15 people

The number the study aims to enrol. It can still change while the study runs.

Started

Jul 2024

Expected to finish

Sep 2027

An estimate. End dates often move.

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

16 to 80 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * 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. 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: 1. Oxygen saturation less than 92% on room air or baseline need for oxygen, change from baseline oxygen dependency. 2. Respiratory rate \>25 breaths per minute. 3. Supine blood pressure ≤ 90/860mmHg 4. Febrile illness with temperature \>100.3 F. 5. 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.

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Conditions

The condition(s) this trial relates to.

hereditary sensory and autonomic neuropathy Riley-Day syndrome

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Familial dysautonomia

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    1 site. The list below names each one and where it is.

  2. The official record

    The full official record for this study. This one lists no contact details, but it is the first place any would appear.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • NYU Langone Health

    New York, New York, 10016, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.