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| ID | Type | Description | Link |
|---|---|---|---|
| 1R01FD007826-01 | U.S. FDA Grant/Contract | View source |
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| Name | Class |
|---|---|
| Food and Drug Administration (FDA) | FED |
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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
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Sublingual dexmedetomidine | Experimental | 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. |
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| Matching Sublingual Placebo | Placebo Comparator | 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. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Matching placebo | Drug | One matching placebo will be given under the tongue on a thin dissolvable film. |
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| Measure | Description | Time Frame |
|---|---|---|
| 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 |
| 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 |
| 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 |
| 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 |
| Measure | Description | Time Frame |
|---|---|---|
| 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 |
| Percentage of patients with ≥ 20% reduction in hospital stay duration |
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Inclusion Criteria:
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:
The patient is a female and has a positive pregnancy test.
The Montreal Cognitive Exam (MoCA) is below 25 points.
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| Name | Affiliation | Role |
|---|---|---|
| Alejandra Gonzalez-Duarte, MD | NYU Langone Health, NYU Dysautonomia Center | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| NYU Langone Health | New York | New York | 10016 | United States |
The de-identified participant data from the final research dataset used in the published manuscript will be shared upon reasonable request beginning 9 months and ending 36 months following article publication or as required by a condition of awards and agreements supporting the research provided the investigator who proposes to use the data executes a data use agreement with NYU Langone Health. Requests may be directed to: Alejandra.gonzalez-duarte@nyulangone.org. The protocol and statistical analysis plan will be made available on Clinicaltrials.gov only as required by federal regulation or as a condition of awards and agreements supporting the research.
Beginning 9 months and ending 36 months following article publication or as required by a condition of awards and agreements supporting the research.
The investigator who proposed to use the data will be provided access upon reasonable request. Requests should be directed to Alejandra.gonzalez-duarte@nyulangone.org. To gain access, data requestors will need to sign a data access agreement.
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| Dexmedetomidine | Drug | Dexmedetomidine 120 mcg will be given under the tongue on a thin dissolvable film. |
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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 |
| 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 |
| 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 |
| ID | Term |
|---|---|
| D004402 | Dysautonomia, Familial |
| D009477 | Hereditary Sensory and Autonomic Neuropathies |
| ID | Term |
|---|---|
| D054969 | Primary Dysautonomias |
| D001342 | Autonomic Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D009421 | Nervous System Malformations |
| D020271 | Heredodegenerative Disorders, Nervous System |
| D019636 | Neurodegenerative Diseases |
| D011115 | Polyneuropathies |
| D010523 | Peripheral Nervous System Diseases |
| D009468 | Neuromuscular Diseases |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D030342 | Genetic Diseases, Inborn |
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| ID | Term |
|---|---|
| D020927 | Dexmedetomidine |
| ID | Term |
|---|---|
| D007093 | Imidazoles |
| D001393 | Azoles |
| D006573 | Heterocyclic Compounds, 1-Ring |
| D006571 | Heterocyclic Compounds |
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