Dexmedetomidine Sublingual Film for the Ambulatory Treatment of Hyperadrenergic Autonomic Crisis in Patients With Familial Dysautonomia

Phase 2Enrolling By InvitationNCT06148311

NYU Langone Health15 enrolled

Overview

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

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

TRIPLE

Enrollment

Conditions

Familial Dysautonomia

Interventions

Matching placeboDRUG

One matching placebo will be given under the tongue on a thin dissolvable film.

DexmedetomidineDRUG

Dexmedetomidine 120 mcg will be given under the tongue on a thin dissolvable film.

Eligibility

Sex: ALLMin age: 16 YearsMax age: 80 Years

Medical Language ↔ Plain English

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.

Locations (1)

NYU Langone Health

New York, New York, United States

Outcomes

Primary Outcomes

Percentage of patients with reduction of Autonomic crisis severity assessment scores (ACSAS) to ≤ 5 points

ACSAS tool will be used to measure objectively and record signs and symptoms of autonomic crisis, which includes documentation of blood pressure, heart rate, skin flushing and blotching, sweating, and behavioral changes. Scores are totaled at each timepoint (0 minutes, 15 min., 45 min., 1.5 hours, 2hr.) ranging from 0 (Normal) to 16 (most severe symptoms occurred).

Time frame: Up to 2 hours post administration

Percentage of patients with 25% reduction in blood pressure

Patients will be monitored with a hospital-at-home (H@H) device to allow visualization of vital signs in real life. The H@H monitor (Biobeat™) is an FDA approved device and will be attached to the patient´s chest by an adhesive patch which will transmit blood pressure. Blood pressure will be monitored before taking each dose and up to 2 hours after.

Time frame: Up to 2 hours post administration

Percentage of patients with >20% reduction in heart rate

Patients will be monitored with a hospital-at-home (H@H) device to allow visualization of vital signs in real life. The H@H monitor (Biobeat™) is an FDA approved device and will be attached to the patient´s chest by an adhesive patch which will transmit heart rate. Heart rate will be monitored before taking each dose and up to 2 hours after.

Time frame: Up to 2 hours post administration

Percentage of patients with >50% reduction in vomiting/retching episodes

Patients will be monitored by their care givers for of episodes of vomiting/ retching before taking each dose and up to 2 hours after.

Time frame: Up to 2 hours post administration

Secondary Outcomes

Percentage of patients with ≥ 20% reduction in hospitalizations

Study team will compare historical data to number of hospitalizations occurred after taking one or more doses.

Data from ClinicalTrials.gov

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