Clinical trials: Familial Dysautonomia (Riley-Day Syndrome)
Nausea, Vomiting
The Safety, Tolerability and Efficacy of Dronabinol, for the Treatment of Nausea and Vomiting in Familial Dysautonomia
This is a pilot clinical trial of dronabinol to treat disabling attacks of nausea and vomiting in patients with familial dysautonomia (FD, also known as Riley Day syndrome or hereditary sensory and autonomic neuropathy type III).
- Conditions
- Nausea, Vomiting, Familial Dysautonomia
- Trial name
- NCT02608931
- Testing
- Dronabinol
- Where
- United States
- Sponsor
- NYU Langone Health
Neurodegenerative Disorders
Development of IPS from Donated Somatic Cells of Patients with Neurological Diseases
Human fibroblasts and possibly other human somatic cells may be reprogrammed into induced pluripotent stem (iPS) cells by the forced expression of transcription factors (1-5).
- Trial name
- NCT00874783
- Where
- Israel
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- Hadassah Medical Organization
Familial Dysautonomia
Pilot Study of Dexmedetomidine Sublingual Film for the Ambulatory Treatment of Hyperadrenergic Autonomic Crisis in Patients With Familial Dysautonomia
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…
- Trial name
- NCT06128356
- Testing
- Dexmedetomidine Sublingual
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- NYU Langone Health
Tay Sachs Disease, Canavan Disease
Efficacy Study of an Online Educational Module Before Carrier Genetic Screening in Persons of Ashkenazi Jewish Descent.
The investigators have developed a new website to educate persons of Ashkenazi Jewish ancestry about their increased risk for having children with certain genetic conditions, and the genetic testing the investigators offer.
- Conditions
- Tay Sachs Disease, Canavan Disease, Familial Dysautonomia
- Trial name
- NCT01999257
- Testing
- Online pre-test genetic education tool
- Where
- Canada
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- McGill University Health Centre/Research Institute of the McGill University Health Centre
Familial Dysautonomia
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.
- Trial name
- NCT06148311
- Testing
- Dexmedetomidine
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- NYU Langone Health
Familial Dysautonomia
Telemedicine Clinical Trial for Cognitive Behavioral Therapy in Familial Dysautonomia
This is a single-blind, placebo-controlled, telemedicine clinical trial to assess the efficacy of cognitive behavioral therapy (CBT) in adult patients 18 years and older with familial dysautonomia (FD) and anxiety and/or…
- Trial name
- NCT03911063
- Testing
- CBT sessions, Talking Sessions
- Where
- United States
- Volunteers
- healthy volunteers welcome
- Sponsor
- NYU Langone Health
Familial Dysautonomia
Carbidopa for the Treatment of Nausea and Vomiting in Familial Dysautonomia
This is a pilot clinical trial of carbidopa to treat disabling attacks of nausea and vomiting in patients with familial dysautonomia (FD, also known as Riley Day syndrome or hereditary sensory and autonomic neuropathy type III).
- Trial name
- NCT01212484
- Testing
- Carbidopa
- Volunteers
- 12 people
- Sponsor
- NYU Langone Health
Atunomic Neuropathy
Proprioception and Sensorimotor Control in Hereditary Sensory and Autonomic Neuropathy
This project comprises three sets of physiological studies - testing eight specific hypotheses - that will contribute new knowledge on proprioception and motor control in a genetic disorder that affects specific components of the…
- Trial name
- NCT02876939
- Testing
- kinesiology tape around the hip, knee and ankle joints, No kinesiology tape around the hip, knee and ankle joints
- Where
- United States
- Volunteers
- 16 people
- Sponsor
- NYU Langone Health
Familial Dysautonomia
The Safety and Tolerability of Kinetin, in Patients With Familial Dysautonomia
This is a study of kinetin, a nutritional supplement that corrects the mRNA splicing defect in patients with familial dysautonomia (FD, also known as Riley Day syndrome or hereditary sensory and autonomic neuropathy type III).
- Trial name
- NCT02274051
- Testing
- Kinetin
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- NYU Langone Health
Familial Dysautonomia (Riley-Day Syndrome), Hereditary Sensory and Autonomic Neuropathies
The Natural History of Familial Dysautonomia
The study will collect clinical information from patients with FD and allow them to give blood to help develop biological markers of the disease to aid diagnosis and treatment.
- Conditions
- Familial Dysautonomia (Riley-Day Syndrome), Hereditary Sensory and Autonomic Neuropathies, Hereditary Sensory and Autonomic Neuropathy 3
- Trial name
- NCT03920774
- Where
- 2 sites in United States, Israel
- Volunteers
- 400 people
- Sponsor
- NYU Langone Health
Dysthymia, Anxiety Disorders
A Trial of Cognitive Behavioral Therapy in Familial Dysautonomia
To determine the effect of cognitive behavioral therapy (CBT) in the severity of anxiety and depression in adult patients with familial dysautonomia. Patients will be enrolled in an 8-week CBT program.
- Conditions
- Dysthymia, Anxiety Disorders, Familial Dysautonomia, Paroxysmal Hypertension
- Trial name
- NCT03013777
- Testing
- 8-week CBT Program
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- NYU Langone Health
Familial Dysautonomia
The Effects Of Bronchodilator Therapy On Respiratory And Autonomic Function In Patients With Familial Dysautonomia
Evaluate the effects of bronchodilator therapy on respiratory function. Our overall goal is to determine whether, in patients with familial dysautonomia (FD), there is a component of airway obstruction that is reversible.
- Trial name
- NCT01987219
- Testing
- Albuterol-sulphate, Ipratropium-bromide
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- NYU Langone Health
Familial Dysautonomia
The Nutritional Supplement Phosphatidylserine in Patients With Familial Dysautonomia
Familial dysautonomia (FD) is a devastating hereditary disease in which the development of selective neuronal populations is impaired because of a deficiency of the protein IKAP (Slaugenhaupt, 2002). There is no known cure.
- Trial name
- NCT02276716
- Testing
- Phosphatidylserine
- Where
- United States
- Volunteers
- 26 people
- Sponsor
- NYU Langone Health
Dysautonomia, Familial, Baroreflex Failure Syndrome
Carbidopa for the Treatment of Excessive Blood Pressure Variability
The overall study objectives are to determine whether carbidopa (Lodosyn®) is safe and well tolerated and to assess whether it can inhibit catecholamine-induced paroxysmal hypertension and normalize or reduce the exaggerated…
- Trial name
- CarbiFD (NCT02553265)
- Testing
- Carbidopa Low-Dose, Carbidopa High-Dose
- Where
- 2 sites in United States
- Volunteers
- 22 people
- Sponsor
- NYU Langone Health