A Phase 2 interventional study of Matching placebo and Dexmedetomidine in Familial Dysautonomia, sponsored by NYU Langone Health. Enrolling by invitation at 1 site in United States. Open to participants aged 16 Years to 80 Years. Per ClinicalTrials.gov, last updated 2026-04-13.
Sponsored by NYU Langone Health · Phase 2, Interventional, and Treatment
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
61 studies on the registry are indexed under Hereditary Sensory and Autonomic Neuropathies; 33 are open to participants now.
This study's planned enrollment of 15 is below the median of 61 across 46 interventional studies indexed under Hereditary Sensory and Autonomic Neuropathies.
Browse Hereditary Sensory and Autonomic Neuropathies studies →NYU Langone Health is the lead sponsor of 1,391 studies on the registry; 254 are open to participants now.
Of its 227 completed or terminated interventional studies of FDA-regulated products, 191 (84%) have results posted.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
The patient during the crisis, before taking the medication, has any of the following:
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.
Drug: Dexmedetomidine
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.
Drug: Matching placebo
One matching placebo will be given under the tongue on a thin dissolvable film.
Dexmedetomidine 120 mcg will be given under the tongue on a thin dissolvable film.
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).
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: Up to 2 hours post administration
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.
Time frame: Up to 48 months
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.
Time frame: 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.
Time frame: 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.
Time frame: Baseline, up to 48 months
Plan to share: Yes — 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.
Supporting information: Study protocol, Sap
No publications or documents are linked to this record.
Eligibility is decided by the study team. Share this record with your doctor or contact the team directly.
No contact was published for this record. The registry link below has the sponsor’s details.
Get an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Dysautonomia, Familial
NYU Langone Health