View clinical trials related to Dysautonomia, Familial.
Filter by: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 depression and/or obsessive compulsive or related disorders. The trial will enroll 20 adult patients each with FD who have anxiety and/or depression and/or obsessive compulsive or related disorders by the DSM V criteria. Enrolled participants will be allocated to receive, in a non-randomized fashion, weekly 5-10 min talking sessions (i.e., placebo) for 8 weeks, followed by weekly 30-60 min CBT sessions during 8 weeks. Although investigators will be un-blinded to the intervention, participants will be blinded to the expected effects of each intervention. Both the talking sessions (i.e., placebo) and CBT sessions will be performed via telemedicine either via a HIPAA secure telemedicine platform or the telephone based on the preference of the individual patient. If a patient specifically requests talking or CBT sessions to be performed in person, this will be accommodated. The use of telemedicine is to accommodate disability and potential physical limitations of this unique patient population. The CBT sessions will be supervised by Lily Armstrong, certified mental health therapist and Dr. Thomas Boes, NYU Clinical Assistant Professor in the Departments of Psychiatric and Neurology.
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). FD is a rare autosomal recessive disease in which the growth and development of selective nerves is impaired. Patients with FD suffer recurrent uncontrollable nausea and vomiting crises accompanied by skin flushing, tachycardia and arterial hypertension. Current treatments of nausea are ineffective or have intolerable side sides. Our long-term goal is to treat nausea effectively and without side effects, a therapeutic intervention that would markedly improve the quality of life of patients with FD.