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 Familial Dysautonomia at home. The primary aims are to examine the feasibility of performing a clinical trial using dexmedetomidine at home to terminate autonomic crisis, and refine the interventions and assessments used to evaluate autonomic crisis termination.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
5
Dexmedetomidine 120 mcg on two thin dissolvable films for sublingual administration
NYU Langone Health
New York, New York, United States
Number of subjects screened per month
Time frame: Month 6
Number of subjects enrolled per month
Time frame: Month 6
Number of completed crises treated at home within the protocol
Time frame: Month 6
Number of subjects that underwent an autonomic crisis per month
Time frame: Month 6
Average duration of the study from visit 1 to the last visit of the last patient
Time frame: From enrollment to end of treatment (up to 6 months)
Length of time from autonomic crisis onset to the initiation of the video recording
Time frame: Up to 2 hours
Length of time from autonomic crisis onset to administration of the medication
Time frame: Up to 2 hours
Length of time from autonomic crisis onset to crisis resolution
Time frame: Up to 24 hours
Length of time it took to complete assessments from start of autonomic crisis
Time frame: Up to 24 hours
Percentage of completion of all rating scales
The scales required to be completed is the Autonomic Crisis Symptom Assessment Scale (ACSAS), the study safety assessments and the Richmond Agitation-Sedation Scale.
Time frame: Month 6
Change in blood pressure
Time frame: Pre-dose, up to 2 hours post-dose
Change in heart rate
Time frame: Pre-dose, up to 2 hours post-dose
Change in number of vomiting episodes
Time frame: Pre-dose, up to 2 hours post-dose
Change in number of retching episodes
Time frame: Pre-dose, up to 2 hours post-dose
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