By InvitationPhase 2

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

United States15 participantsStarted 2024-07-01

Plain-language summary

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

Who can participate

Age range16 Years – 80 Years

SexALL

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Exclusion criteria

✕. Oxygen saturation less than 92% on room air or baseline need for oxygen, change from baseline oxygen dependency.
✕. Respiratory rate \>25 breaths per minute.
✕. Supine blood pressure ≤ 90/860mmHg
✕. Febrile illness with temperature \>100.3 F.
✕. 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.

What they're measuring

Percentage of patients with reduction of Autonomic crisis severity assessment scores (ACSAS) to ≤ 5 points

Timeframe: Up to 2 hours post administration

Percentage of patients with 25% reduction in blood pressure

Timeframe: Up to 2 hours post administration

Percentage of patients with >20% reduction in heart rate

Timeframe: Up to 2 hours post administration

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

Timeframe: Up to 2 hours post administration

Trial details

NCT IDNCT06148311

SponsorNYU Langone Health

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2027-01-01

View on ClinicalTrials.gov More Familial Dysautonomia trials