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

Familial Dysautonomia Clinical Trial

Official title:

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

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT06128356
• Other study ID #: 23-00174
• Status: Not yet recruiting
• Phase: Phase 2
• Start date: April 1, 2024
• Est. completion date: December 2028

Study information

• Verified date: March 2024
• Source: NYU Langone Health
• Contact: Alejandra Gonzalez-Duarte, MD
• Phone: 2122637225
• Email: Alejandra.gonzalez-duarte[@]nyulangone.org
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

This is a pilot open-label study to evaluate the feasibility of conducting a clinical trial using sublingual dexmedetomidine sublingual film to treat hyperadrenergic autonomic crises in patients with Familial Dysautonomia at home. The primary aims are to examine the feasibility of performing a clinical trial using dexmedetomidine at home to terminate autonomic crisis, and refine the interventions and assessments used to evaluate autonomic crisis termination.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 5
• Est. completion date: December 2028
• Est. primary completion date: September 2028
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 80 Years

Eligibility

Inclusion Criteria:

• Genetically confirmed diagnosis of Familial Dysautonomia.

• Evidence of autonomic crisis, previous treatment with IV dexmedetomidine without significant side effects, and registered medical data within the year preceding the study in our database.

• One or more autonomic crises during the last year.

• Age above 18 years.

• The patient has a responsible caretaker to communicate with the medical providers.

• Provision of signed and dated informed consent form from the patient and responsible caregiver.

• Able to state willingness to comply with all study procedures and availability for the duration of the study

• For males and females of reproductive potential: use condoms for contraception if sexually active.

Exclusion Criteria:

• At the consideration of the principal investigator, the caregiver cannot fully understand the protocol or communicate during the crisis with the Center.

• 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.

• MoCA score <25 points.

Related Conditions & MeSH terms

• Autonomic Nervous System Diseases
• Dysautonomia, Familial
• Familial Dysautonomia
• Primary Dysautonomias

Intervention

Drug:
• Dexmedetomidine Sublingual
Dexmedetomidine 120 mcg on two thin dissolvable films for sublingual administration

Locations

Country	Name	City	State
United States	NYU Langone Health	New York	New York

Sponsors (1)

Lead Sponsor	Collaborator
NYU Langone Health

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of subjects screened per month		Month 6
Primary	Number of subjects enrolled per month		Month 6
Primary	Number of completed crises treated at home within the protocol		Month 6
Primary	Number of subjects that underwent an autonomic crisis per month		Month 6
Primary	Average duration of the study from visit 1 to the last visit of the last patient		From enrollment to end of treatment (up to 6 months)
Secondary	Length of time from autonomic crisis onset to the initiation of the video recording		Up to 2 hours
Secondary	Length of time from autonomic crisis onset to administration of the medication		Up to 2 hours
Secondary	Length of time from autonomic crisis onset to crisis resolution		Up to 24 hours
Secondary	Length of time it took to complete assessments from start of autonomic crisis		Up to 24 hours
Secondary	Percentage of completion of all rating scales	The scales required to be completed is the Autonomic Crisis Symptom Assessment Scale (ACSAS), the study safety assessments and the Richmond Agitation-Sedation Scale.	Month 6
Secondary	Change in blood pressure		Pre-dose, up to 2 hours post-dose
Secondary	Change in heart rate		Pre-dose, up to 2 hours post-dose
Secondary	Change in number of vomiting episodes		Pre-dose, up to 2 hours post-dose
Secondary	Change in number of retching episodes		Pre-dose, up to 2 hours post-dose