Status:
ENROLLING_BY_INVITATION
Dexmedetomidine Sublingual Film for the Ambulatory Treatment of Hyperadrenergic Autonomic Crisis in Patients With Familial Dysautonomia
Lead Sponsor:
NYU Langone Health
Collaborating Sponsors:
Food and Drug Administration (FDA)
Conditions:
Familial Dysautonomia
Eligibility:
All Genders
16-80 years
Phase:
PHASE2
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 auto...
Eligibility Criteria
Inclusion
- 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
- 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.
Key Trial Info
Start Date :
July 1 2024
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
September 1 2027
Estimated Enrollment :
15 Patients enrolled
Trial Details
Trial ID
NCT06148311
Start Date
July 1 2024
End Date
September 1 2027
Last Update
June 4 2025
Active Locations (1)
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1
NYU Langone Health
New York, New York, United States, 10016