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

Familial Dysautonomia Clinical Trial

Official title:

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

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT06148311
• Other study ID #: 22-01332
• Status: Not yet recruiting
• Phase: Phase 2
• Start date: April 1, 2024
• Est. completion date: September 1, 2027

Study information

• Verified date: February 2024
• Source: NYU Langone Health
• Contact: Alejandra Gonzalez-Duarte, MD
• Phone: 2122637225
• Email: Alejandra.gonzalez-duarte[@]nyulangone.org
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 15
• Est. completion date: September 1, 2027
• Est. primary completion date: January 1, 2027
• Accepts healthy volunteers: No
• Gender: All
• Age group: 16 Years to 80 Years

Eligibility

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.

Related Conditions & MeSH terms

• Autonomic Nervous System Diseases
• Dysautonomia, Familial
• Familial Dysautonomia
• Primary Dysautonomias

Intervention

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

Locations

Country	Name	City	State
United States	NYU Langone Health	New York	New York

Sponsors (1)

Lead Sponsor	Collaborator
NYU Langone Health

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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).	Up to 2 hours post administration
Primary	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.	Up to 2 hours post administration
Primary	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.	Up to 2 hours post administration
Primary	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.	Up to 2 hours post administration
Secondary	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.	Up to 48 months
Secondary	Percentage of patients with = 20% reduction in hospital stay duration	Study team will compare historical data to number of days during hospital stay that occurred after taking one or more doses.	Up to 48 months
Secondary	Percentage of patients with = 30% reduction in ICU stay duration	Study team will compare historical data to number of days at the ICU that occurred after taking one or more doses.	Up to 48 months
Secondary	Change in number of medical complications	Study team will compare historical data to number of medical complications of hospitalizations occurred after taking one or more doses.	Baseline, up to 48 months