Familial Dysautonomia Clinical Trial
Official title:
Dexmedetomidine Sublingual Film for the Ambulatory Treatment of Hyperadrenergic Autonomic Crisis in Patients With Familial Dysautonomia
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.
Status | Not yet recruiting |
Enrollment | 15 |
Est. completion date | September 1, 2027 |
Est. primary completion date | January 1, 2027 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 16 Years to 80 Years |
Eligibility | Inclusion Criteria: - Genetically confirmed diagnosis of Familial Dysautonomia. - One or more autonomic crises during the last year. - Our database shows evidence of autonomic crisis, previous treatment with IV dexmedetomidine, and registered medical data within the year preceding the study. - Age 16 years or older - 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 age: 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: 1. Oxygen saturation less than 92% on room air or baseline need for oxygen, change from baseline oxygen dependency. 2. Respiratory rate >25 breaths per minute. 3. Supine blood pressure = 90/860mmHg 4. Febrile illness with temperature >100.3 F. 5. 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. - The Montreal Cognitive Exam (MoCA) is below 25 points. |
Country | Name | City | State |
---|---|---|---|
United States | NYU Langone Health | New York | New York |
Lead Sponsor | Collaborator |
---|---|
NYU Langone Health |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Percentage of patients with reduction of Autonomic crisis severity assessment scores (ACSAS) to = 5 points | ACSAS tool will be used to measure objectively and record signs and symptoms of autonomic crisis, which includes documentation of blood pressure, heart rate, skin flushing and blotching, sweating, and behavioral changes. Scores are totaled at each timepoint (0 minutes, 15 min., 45 min., 1.5 hours, 2hr.) ranging from 0 (Normal) to 16 (most severe symptoms occurred). | Up to 2 hours post administration | |
Primary | Percentage of patients with 25% reduction in blood pressure | Patients will be monitored with a hospital-at-home (H@H) device to allow visualization of vital signs in real life. The H@H monitor (Biobeat™) is an FDA approved device and will be attached to the patient´s chest by an adhesive patch which will transmit blood pressure. Blood pressure will be monitored before taking each dose and up to 2 hours after. | Up to 2 hours post administration | |
Primary | Percentage of patients with >20% reduction in heart rate | Patients will be monitored with a hospital-at-home (H@H) device to allow visualization of vital signs in real life. The H@H monitor (Biobeat™) is an FDA approved device and will be attached to the patient´s chest by an adhesive patch which will transmit heart rate. Heart rate will be monitored before taking each dose and up to 2 hours after. | Up to 2 hours post administration | |
Primary | Percentage of patients with >50% reduction in vomiting/retching episodes | Patients will be monitored by their care givers for of episodes of vomiting/ retching before taking each dose and up to 2 hours after. | Up to 2 hours post administration | |
Secondary | Percentage of patients with = 20% reduction in hospitalizations | Study team will compare historical data to number of hospitalizations occurred after taking one or more doses. | Up to 48 months | |
Secondary | Percentage of patients with = 20% reduction in hospital stay duration | Study team will compare historical data to number of days during hospital stay that occurred after taking one or more doses. | Up to 48 months | |
Secondary | Percentage of patients with = 30% reduction in ICU stay duration | Study team will compare historical data to number of days at the ICU that occurred after taking one or more doses. | Up to 48 months | |
Secondary | Change in number of medical complications | Study team will compare historical data to number of medical complications of hospitalizations occurred after taking one or more doses. | Baseline, up to 48 months |
Status | Clinical Trial | Phase | |
---|---|---|---|
Withdrawn |
NCT02608931 -
The Safety, Tolerability and Efficacy of Dronabinol, for the Treatment of Nausea and Vomiting in Familial Dysautonomia
|
Phase 2 | |
Completed |
NCT02276716 -
The Nutritional Supplement Phosphatidylserine in Patients With Familial Dysautonomia
|
Phase 2 | |
Completed |
NCT02274051 -
The Safety and Tolerability of Kinetin, in Patients With Familial Dysautonomia
|
Phase 1 | |
Completed |
NCT01987219 -
The Effects Of Bronchodilator Therapy On Respiratory And Autonomic Function In Patients With Familial Dysautonomia
|
Phase 3 | |
Completed |
NCT01999257 -
Efficacy Study of an Online Educational Module Before Carrier Genetic Screening in Persons of Ashkenazi Jewish Descent.
|
N/A | |
Completed |
NCT03013777 -
A Trial of Cognitive Behavioral Therapy in Familial Dysautonomia
|
N/A | |
Completed |
NCT01212484 -
Carbidopa for the Treatment of Nausea and Vomiting in Familial Dysautonomia
|
Phase 3 | |
Not yet recruiting |
NCT06128356 -
Pilot Study of Dexmedetomidine Sublingual Film for the Ambulatory Treatment of Hyperadrenergic Autonomic Crisis in Patients With Familial Dysautonomia
|
Phase 2 | |
Withdrawn |
NCT03911063 -
Telemedicine Clinical Trial for Cognitive Behavioral Therapy in Familial Dysautonomia
|
N/A |