Clinical trial NCT06128356: Pilot Study of Dexmedetomidine Sublingual Film for the Ambulatory Treatment of Hyperadrenergic Autonomic Crisis in Patients With Familial Dysautonomia
Official title: Pilot Study of Dexmedetomidine Sublingual Film for the Ambulatory Treatment of Hyperadrenergic Autonomic Crisis in Patients With Familial Dysautonomia
Study type: INTERVENTIONAL
Overall status: ENROLLING_BY_INVITATION
Phase: PHASE2
Conditions: Familial Dysautonomia
Lead sponsor: NYU Langone Health
Sponsor class: OTHER
First posted: 2023-11-13
Enrollment: 5
Sex: ALL
Minimum age (years): 18
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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 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.


Eligibility criteria: 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.

Primary outcomes:
measure: Number of subjects screened per month
timeFrame: Month 6
measure: Number of subjects enrolled per month
timeFrame: Month 6
measure: Number of completed crises treated at home within the protocol
timeFrame: Month 6
measure: Number of subjects that underwent an autonomic crisis per month
timeFrame: Month 6
measure: Average duration of the study from visit 1 to the last visit of the last patient
timeFrame: From enrollment to end of treatment (up to 6 months)
Secondary outcomes:
measure: Length of time from autonomic crisis onset to the initiation of the video recording
timeFrame: Up to 2 hours
measure: Length of time from autonomic crisis onset to administration of the medication
timeFrame: Up to 2 hours
measure: Length of time from autonomic crisis onset to crisis resolution
timeFrame: Up to 24 hours
measure: Length of time it took to complete assessments from start of autonomic crisis
timeFrame: Up to 24 hours
measure: Percentage of completion of all rating scales
timeFrame: Month 6
measure: Change in blood pressure
timeFrame: Pre-dose, up to 2 hours post-dose
measure: Change in heart rate
timeFrame: Pre-dose, up to 2 hours post-dose
measure: Change in number of vomiting episodes
timeFrame: Pre-dose, up to 2 hours post-dose
measure: Change in number of retching episodes
timeFrame: Pre-dose, up to 2 hours post-dose
Other outcomes:
Locations:
city: New York
country: United States
facility: NYU Langone Health
state: New York
Study officials:
affiliation: NYU Langone Health
name: Alejandra Gonzalez-Duarte, MD
role: PRINCIPAL_INVESTIGATOR
References: