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

NCT06128356 | Active Not Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: 23-00174
• Study Type: interventional
• Target: 5
• Locations: 1 country
• Sites: 1

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

Outcome Measures

Primary Outcomes (5)

• Number of subjects screened per month

  Month 6

• Number of subjects enrolled per month

  Month 6

• Number of completed crises treated at home within the protocol

  Month 6

• Number of subjects that underwent an autonomic crisis per month

  Month 6

• Average duration of the study from visit 1 to the last visit of the last patient

  From enrollment to end of treatment (up to 6 months)

Secondary Outcomes (9)

• Length of time from autonomic crisis onset to the initiation of the video recording

  Up to 2 hours

• Length of time from autonomic crisis onset to administration of the medication

  Up to 2 hours

• Length of time from autonomic crisis onset to crisis resolution

  Up to 24 hours

• Length of time it took to complete assessments from start of autonomic crisis

  Up to 24 hours

• Percentage of completion of all rating scales

  Month 6

Study Arms (1)

Sublingual dexmedetomidine

EXPERIMENTAL

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.

Drug: Dexmedetomidine Sublingual

Interventions

Dexmedetomidine Sublingual DRUG

Dexmedetomidine 120 mcg on two thin dissolvable films for sublingual administration

Also known as: IGALMI

Sublingual dexmedetomidine

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• NYU Langone Health: lead

Study Sites (1)

NYU Langone Health

New York, New York, 10016, United States

Location

MeSH Terms

Conditions

Dysautonomia, Familial

Interventions

Dexmedetomidine

Condition Hierarchy (Ancestors)

Primary Dysautonomias; Autonomic Nervous System Diseases; Nervous System Diseases; Hereditary Sensory and Autonomic Neuropathies; Nervous System Malformations; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Polyneuropathies; Peripheral Nervous System Diseases; Neuromuscular Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn

Intervention Hierarchy (Ancestors)

Imidazoles; Azoles; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds

Study Officials

• Alejandra Gonzalez-Duarte, MD

  NYU Langone Health

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 7, 2023
• First Posted: November 13, 2023
• Study Start: June 1, 2024
• Primary Completion (Estimated): September 1, 2028
• Study Completion (Estimated): December 1, 2028
• Last Updated: March 4, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will share
• Time Frame: Beginning 9 months and ending 36 months following article publication or as required by a condition of awards and agreements supporting the research.
• Access Criteria: The investigator who proposed to use the data will be provided access upon reasonable request. Requests should be directed to Alejandra.gonzalez-duarte@nyulangone.org. To gain access, data requestors will need to sign a data access agreement.

Locations

US (1)