Clinical trial NCT06148311: Dexmedetomidine Sublingual Film for the Ambulatory Treatment of Hyperadrenergic Autonomic Crisis in Patients With Familial Dysautonomia
Official title: 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
Keywords: Familial Dysautonomia
Hereditary Sensory and Autonomic Neuropathy type 3
HSAN
autonomic sympathetic crises
Lead sponsor: NYU Langone Health
Sponsor class: OTHER
Collaborators: name: Food and Drug Administration (FDA)
First posted: 2023-11-28
Enrollment: 15
Sex: ALL
Minimum age (years): 16
Maximum age (years): 80
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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


Eligibility 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.

Primary outcomes:
measure: Percentage of patients with reduction of Autonomic crisis severity assessment scores (ACSAS) to ≤ 5 points
timeFrame: Up to 2 hours post administration
measure: Percentage of patients with 25% reduction in blood pressure
timeFrame: Up to 2 hours post administration
measure: Percentage of patients with >20% reduction in heart rate
timeFrame: Up to 2 hours post administration
measure: Percentage of patients with >50% reduction in vomiting/retching episodes
timeFrame: Up to 2 hours post administration
Secondary outcomes:
measure: Percentage of patients with ≥ 20% reduction in hospitalizations
timeFrame: Up to 48 months
measure: Percentage of patients with ≥ 20% reduction in hospital stay duration
timeFrame: Up to 48 months
measure: Percentage of patients with ≥ 30% reduction in ICU stay duration
timeFrame: Up to 48 months
measure: Change in number of medical complications
timeFrame: Baseline, up to 48 months
Other outcomes:
Locations:
city: New York
country: United States
facility: NYU Langone Health
state: New York
Study officials:
affiliation: NYU Langone Health, NYU Dysautonomia Center
name: Alejandra Gonzalez-Duarte, MD
role: PRINCIPAL_INVESTIGATOR
References: