New under-the-tongue film may stop dangerous attacks in rare nerve disease
NCT ID NCT06128356
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This small pilot study is testing whether a medicated film placed under the tongue can safely and effectively stop autonomic crises in people with familial dysautonomia at home. Five adults who have had previous crises and tolerated a similar IV drug are enrolled. The goal is to see if running a full trial is possible and to refine how we measure crisis relief.
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Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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5 people
The number who actually took part.
- Started
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Jun 2024
- Expected to finish
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Dec 2028
An estimate. End dates often move.
- Lead sponsor
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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
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18 to 80 years
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
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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. * 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.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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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.