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Alpha-2 Adrenergic Agonist

Dexmedetomidine for Familial Dysautonomia

Phase 2
Recruiting
Led By Alejandra Gonzalez-Duarte, MD
Research Sponsored by NYU Langone Health
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Genetically confirmed diagnosis of Familial Dysautonomia
Age above 18 years
Must not have
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
Timeline
Screening 3 weeks
Treatment Varies
Follow Up month 6
Awards & highlights
No Placebo-Only Group

Summary

This trial aims to see if dexmedetomidine can be used at home to stop autonomic crises in Familial Dysautonomia patients.

Who is the study for?
This trial is for adults over 18 with a genetic diagnosis of Familial Dysautonomia who've had at least one autonomic crisis in the past year and tolerated IV dexmedetomidine. They must have a caregiver, give informed consent, and use contraception if needed. Exclusions include certain health conditions during crises or inability to understand the protocol.
What is being tested?
The study tests if sublingual dexmedetomidine film can be used at home to stop hyperadrenergic autonomic crises in Familial Dysautonomia patients. It's an open-label pilot study aiming to see if such trials are feasible and how well the treatment works outside of a hospital setting.
What are the potential side effects?
While specific side effects aren't listed here, participants previously treated with IV dexmedetomidine without significant side effects can join, suggesting that similar mild reactions may occur with sublingual administration.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I have a genetic diagnosis of Familial Dysautonomia.
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I am over 18 years old.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I am a woman and my pregnancy test is positive.
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My cognitive test score is below 25.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~month 6
This trial's timeline: 3 weeks for screening, Varies for treatment, and month 6 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Secondary study objectives
Percentage of completion of all rating scales

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups
Experimental Treatment
Group I: Sublingual dexmedetomidineExperimental Treatment1 Intervention
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.

Find a Location

Who is running the clinical trial?

NYU Langone HealthLead Sponsor
1,407 Previous Clinical Trials
855,765 Total Patients Enrolled
8 Trials studying Familial Dysautonomia
98 Patients Enrolled for Familial Dysautonomia
Alejandra Gonzalez-Duarte, MDPrincipal InvestigatorNYU Langone Health
1 Previous Clinical Trials
15 Total Patients Enrolled
1 Trials studying Familial Dysautonomia
15 Patients Enrolled for Familial Dysautonomia
~3 spots leftby Sep 2028
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