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

Conditions

• Familial Dysautonomia

Eligibility Criteria

Sex

ALL

Age

16 Years - 80 Years

Healthy Volunteers

Not accepted

Interventions

• Matching placebo — DRUG
  One matching placebo will be given under the tongue on a thin dissolvable film.
• Dexmedetomidine — DRUG
  Dexmedetomidine 120 mcg will be given under the tongue on a thin dissolvable film.

Study Details

The purpose of this placebo controlled interventional study is to collect preliminary data on administering dexmedetomidine in patients with Familial Dysautonomia (FD) during a rapid cessation of autonomic crisis. The primary aims are to assess the feasibility and evaluate if measurements of heart rate, blood pressure and oxygen saturation can predict the start of an autonomic crisis. Funding Source- FDA OOPD

Key Dates

Start date

Jul 1, 2024

Status verified

Apr 2026

Primary completion

Jan 1, 2027

Completion

Sep 1, 2027

Study Design

Enrollment

15 participants (estimated)

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Arms

• Experimental: Sublingual dexmedetomidine
  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.
• Placebo Comparator: Matching Sublingual Placebo
  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.

Primary Outcome Measure

Percentage of patients with reduction of Autonomic crisis severity assessment scores (ACSAS) to ≤ 5 points [ Time Frame: Up to 2 hours post administration ]

Locations (1)

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