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NCT06148311 | ENROLLING BY INVITATION | Familial Dysautonomia

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

NYU Langone Health

Brief 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

Condition or disease

Families

Intervention/treatment

Matching placebo

Dexmedetomidine

Phase

PHASE2

Study Type : INTERVENTIONAL
Estimated Enrollment : 15 participants
Masking : TRIPLE
Primary Purpose : TREATMENT
Official Title : Dexmedetomidine Sublingual Film for the Ambulatory Treatment of Hyperadrenergic Autonomic Crisis in Patients With Familial Dysautonomia
Actual Study Start Date : 2024-07-01
Estimated Primary Completion Date : 2027-01-01
Estimated Study Completion Date : 2027-09-01

Information not available for Arms and Intervention/treatment

Ages Eligible for Study: 16 Years to 80 Years
Sexes Eligible for Study: ALL
Accepts Healthy Volunteers:
Criteria
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.
Dexmedetomidine Sublingual Film for the Ambulatory Treatment of Hyperadrenergic Autonomic Crisis in Patients With Familial Dysautonomia

Location Details

NCT06148311

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How to Participate

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Locations

Not yet recruiting

United States, New York

NYU Langone Health

New York, New York, United States, 10016