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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 weeks3 visits

Treatment ~ Varies

Follow Up ~ month 6

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.

0 / 5Eligibility criteria met