Status:
ENROLLING_BY_INVITATION
Pilot Study of Dexmedetomidine Sublingual Film for the Ambulatory Treatment of Hyperadrenergic Autonomic Crisis in Patients With Familial Dysautonomia
Lead Sponsor:
NYU Langone Health
Conditions:
Familial Dysautonomia
Eligibility:
All Genders
18-80 years
Phase:
PHASE2
Brief Summary
This is a pilot open-label study to evaluate the feasibility of conducting a clinical trial using sublingual dexmedetomidine sublingual film to treat hyperadrenergic autonomic crises in patients with ...
Eligibility Criteria
Inclusion
- 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
- 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.
Key Trial Info
Start Date :
June 1 2024
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
December 1 2028
Estimated Enrollment :
5 Patients enrolled
Trial Details
Trial ID
NCT06128356
Start Date
June 1 2024
End Date
December 1 2028
Last Update
May 1 2025
Active Locations (1)
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1
NYU Langone Health
New York, New York, United States, 10016