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Alpha-2 Adrenergic Agonist

Dexmedetomidine for Familial Dysautonomia

Phase 2

Waitlist Available

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 16 years or older

Must not have

Oxygen saturation less than 92% on room air or baseline need for oxygen, change from baseline oxygen dependency

Febrile illness with temperature >100.3 F

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 48 months

Summary

This trial tests if a drug can prevent autonomic crisis in people with Familial Dysautonomia, and if heart rate, BP, and oxygen can predict it.

Who is the study for?

This trial is for individuals aged 16 or older with a genetic diagnosis of Familial Dysautonomia who've had an autonomic crisis in the past year. They must be willing to follow study procedures, use condoms if sexually active, have a responsible caretaker, and provide informed consent. Excluded are those with low oxygen levels, high respiratory rates, very low blood pressure, fever/infection signs recently or pregnant women.

What is being tested?

The study tests dexmedetomidine as a sublingual film against a placebo to manage hyperadrenergic autonomic crises in patients with Familial Dysautonomia. It aims to see if it's feasible and whether heart rate, blood pressure and oxygen saturation can predict the onset of these crises.

What are the potential side effects?

While not explicitly listed here, dexmedetomidine may cause side effects such as dry mouth, low blood pressure upon standing (orthostatic hypotension), drowsiness or sedation. The severity varies from person to person.

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 16 years old or older.

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I have had one or more sudden episodes of severe autonomic symptoms in the last year.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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My oxygen levels are below 92% without extra oxygen or I need more oxygen than before.

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I have a fever higher than 100.3 F.

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I am pregnant.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 48 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 48 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 48 months for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Percentage of patients with 25% reduction in blood pressure

Percentage of patients with >20% reduction in heart rate

Percentage of patients with >50% reduction in vomiting/retching episodes

+1 more

Secondary study objectives

Change in number of medical complications

Percentage of patients with ≥ 20% reduction in hospital stay duration

Percentage of patients with ≥ 20% reduction in hospitalizations

+1 more

Side effects data

From 2020 Phase 4 trial • 798 Patients • NCT02004613

57%

Clinically important hypotension

Clinically important bradycardia

infection

stroke

Atelectasis

Hemorrhage

100%

80%

60%

40%

20%

Study treatment Arm

Placebo

Dexmedetomidine

Trial Design

2Treatment groups

Experimental Treatment

Placebo Group

Group I: Sublingual dexmedetomidineExperimental Treatment1 Intervention

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 II: Matching Sublingual PlaceboPlacebo Group1 Intervention

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.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Dexmedetomidine

2015

Completed Phase 4

~2050

0 / 6Eligibility criteria met