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

Обзор

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

Вмешательства

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

Первичные конечные точки

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

Вторичные конечные точки (4)

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

Критерии участия

Критерии включения

• 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.

Критерии исключения

• 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.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Рандомизированное

Модель

Параллельные группы

Маскирование

Тройное слепое

Основная цель

Лечение

Центры проведения

США · 1 центр

• NYU Langone Health — New York

Идентификаторы

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

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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