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

NCT ID: NCT06128356

Last Updated: 2025-05-01

Basic Information

• Recruitment Status: ENROLLING_BY_INVITATION
• Clinical Phase: PHASE2
• Total Enrollment: 5 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2024-06-01
• Study Completion Date: 2028-12-31

Brief Summary

This is a pilot open-label study to evaluate the feasibility of conducting a clinical trial using sublingual dexmedetomidine sublingual film to treat hyperadrenergic autonomic crises in patients with Familial Dysautonomia at home. The primary aims are to examine the feasibility of performing a clinical trial using dexmedetomidine at home to terminate autonomic crisis, and refine the interventions and assessments used to evaluate autonomic crisis termination.

Conditions

Familial Dysautonomia

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Sublingual dexmedetomidine

Participants will be administered two 60 micrograms sublingual films of IGLMI following start of an autonomic crises. The maximum amount for the study is four oral films of 60 micrograms in 24 hours, two at the beginning of the crises and if needed, two additional within two hours.

Group Type: EXPERIMENTAL

Dexmedetomidine Sublingual

Intervention Type: DRUG

Dexmedetomidine 120 mcg on two thin dissolvable films for sublingual administration

Interventions

Dexmedetomidine Sublingual

Dexmedetomidine 120 mcg on two thin dissolvable films for sublingual administration

Intervention Type: DRUG

Other Intervention Names

IGALMI

Eligibility Criteria

Inclusion Criteria

• Genetically confirmed diagnosis of Familial Dysautonomia.
• Evidence of autonomic crisis, previous treatment with IV dexmedetomidine without significant side effects, and registered medical data within the year preceding the study in our database.
• One or more autonomic crises during the last year.
• Age above 18 years.
• 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 potential: 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:
• a. Oxygen saturation less than 92% on room air or baseline need for oxygen, change from baseline oxygen dependency.
• b. Respiratory rate >20 breaths per minute.
• c. Supine blood pressure ≤ 90/60mmHg
• d. Febrile illness with temperature >100.3 F.
• e. 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.
• MoCA score <25 points.

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 80 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

NYU Langone Health

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Alejandra Gonzalez-Duarte, MD

Role: PRINCIPAL_INVESTIGATOR

NYU Langone Health

Locations

NYU Langone Health

New York, New York, United States

Countries

United States

Other Identifiers

23-00174

Identifier Type: -

Identifier Source: org_study_id