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

NCT ID: NCT06148311

Last Updated: 2025-06-04

Basic Information

• Recruitment Status: ENROLLING_BY_INVITATION
• Clinical Phase: PHASE2
• Total Enrollment: 15 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2024-07-01
• Study Completion Date: 2027-09-01

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

Conditions

Familial Dysautonomia

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: TRIPLE

Study Groups

Sublingual dexmedetomidine

Participants will be administered 120 micrograms sublingual film following start of an autonomic crisis. In case the crisis has not subsided after 2 hours, the patient will be instructed to take the second dose. The maximum amount for the study is two oral films within two hours for each episode but not more than 24 hours apart, one at the beginning of the crisis and if needed, one additional within two hours.

Group Type: EXPERIMENTAL

Dexmedetomidine

Intervention Type: DRUG

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

Matching Sublingual Placebo

Participants will be administered the matching placebo sublingual film following start of an autonomic crisis. In case the crisis has not subsided after 2 hours, the patient will be instructed to take the second dose. The maximum amount for the study is two oral films within two hours for each episode but not more than 24 hours apart, one at the beginning of the crisis and if needed, one additional within two hours.

Group Type: PLACEBO_COMPARATOR

Matching placebo

Intervention Type: DRUG

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

Interventions

Matching placebo

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

Intervention Type: DRUG

Dexmedetomidine

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

Intervention Type: DRUG

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.

• Minimum Eligible Age: 16 Years
• Maximum Eligible Age: 80 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Food and Drug Administration (FDA)

FED

Sponsor Role: collaborator

NYU Langone Health

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Alejandra Gonzalez-Duarte, MD

Role: PRINCIPAL_INVESTIGATOR

NYU Langone Health, NYU Dysautonomia Center

Locations

NYU Langone Health

New York, New York, United States

Countries

United States

Other Identifiers

1R01FD007826-01

Identifier Type: FDA

Identifier Source: secondary_id

View Link

22-01332

Identifier Type: -

Identifier Source: org_study_id