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

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

Interventions

• Drug Matching placebo
  One matching placebo will be given under the tongue on a thin dissolvable film.
• Drug Dexmedetomidine
  Dexmedetomidine 120 mcg will be given under the tongue on a thin dissolvable film.

Primary outcome measures

• Percentage of patients with reduction of Autonomic crisis severity assessment scores (ACSAS) to ≤ 5 points [Time frame: Up to 2 hours post administration]
• Percentage of patients with 25% reduction in blood pressure [Time frame: Up to 2 hours post administration]
• Percentage of patients with >20% reduction in heart rate [Time frame: Up to 2 hours post administration]
• Percentage of patients with >50% reduction in vomiting/retching episodes [Time frame: Up to 2 hours post administration]

Secondary outcome measures (4)

• Percentage of patients with ≥ 20% reduction in hospitalizations [Time frame: Up to 48 months]
• Percentage of patients with ≥ 20% reduction in hospital stay duration [Time frame: Up to 48 months]
• Percentage of patients with ≥ 30% reduction in ICU stay duration [Time frame: Up to 48 months]
• Change in number of medical complications [Time frame: Baseline, up to 48 months]

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:

• Oxygen saturation less than 92% on room air or baseline need for oxygen, change from baseline oxygen dependency.
• Respiratory rate >25 breaths per minute.
• Supine blood pressure ≤ 90/860mmHg
• Febrile illness with temperature >100.3 F.
• 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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation

Randomized

Model

Parallel assignment

Masking

Triple blind

Primary purpose

Treatment

Study locations

United States · 1 center

• NYU Langone Health — New York

Identifiers

NCT: NCT06148311 · 22-01332 · 1R01FD007826-01

Primary sources (government registries)

• ClinicalTrials.gov (US NIH)

View this study on ClinicalTrials.gov ↗