Clinical trials: Familial Dysautonomia
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
Parkinson's Disease, Parkinsonian Disorders
Gait Analysis in Neurological Disease
The purpose of this study is to investigate whether speed-dependent measures of gait (so called gait signatures) can be identified in patients with neurological conditions that affect gait, particularly in subjects with…
- Conditions
- Parkinson's Disease, Parkinsonian Disorders, Atypical Parkinson Disease, Progressive Supranuclear Palsy
- Trial name
- NCT02994719
- Testing
- Anti-Parkinson medication, Deep Brain Stimulation
- Where
- United States
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- Beth Israel Deaconess Medical Center
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
Rare Disorders, Undiagnosed Disorders
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
CoRDS, or the Coordination of Rare Diseases at Sanford, is based at Sanford Research in Sioux Falls, South Dakota. It provides researchers with a centralized, international patient registry for all rare diseases.
- Conditions
- Rare Disorders, Undiagnosed Disorders, Disorders of Unknown Prevalence, Cornelia De Lange Syndrome
- Trial name
- CoRDS (NCT01793168)
- Where
- 2 sites in United States, Australia
- Volunteers
- 20,000 people
- Sponsor
- Sanford Health
Parkinson Disease Dementia, Parkinson Disease
Community Outreach for Palliative Engagement -- Parkinson Disease
The purpose of this study is to learn more about the effectiveness of palliative care training for community physicians and telemedicine support services for patients and carepartners with Parkinson's disease and Lewy Body…
- Conditions
- Parkinson Disease Dementia, Parkinson Disease, Parkinson's Disease and Parkinsonism, Lewy Body Parkinsonism
- Trial name
- COPE-PD (NCT05222386)
- Testing
- Parkinson Disease Standard Care, Online Community-Supported Palliative Care
- Where
- United States
- Volunteers
- 632 people, healthy volunteers welcome
- Sponsor
- University of Rochester
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
Functional Gastrointestinal Disorders (FGIDs), Cyclic Vomiting Syndrome
Autonomic Reactivity and Personalized Neurostimulation
Disorders of gut-brain interaction (DGBI) affect up to 25% of U.S. children. Patients often suffer from disabling, multisystem comorbidities that suggest a common root (sleep disturbances, fatigue, anxiety, etc).
- Conditions
- Functional Gastrointestinal Disorders (FGIDs), Cyclic Vomiting Syndrome, Functional Dyspepsia, Dysautonomia
- Trial name
- NCT06863207
- Testing
- Percutaneous electrical nerve field stimulation, Hypnotherapy
- Where
- United States
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- Medical College of Wisconsin
Polyneuropathy, Amyloid, Neuropathic Pain
Screening for the Transthyretin-Related Familial Amyloidotic Polyneuropathy (TTR FAP)
An International, multicenter, epidemiological observational study investigating the prevalence of Transthyretin-Related Familial Amyloidotic Polyneuropathy (TTR-FAP) in participants with small fiber polyneuropathy of no obvious…
- Conditions
- Polyneuropathy, Amyloid, Neuropathic Pain, Cardiac Failure, Orthostatic Hypotension
- Trial name
- TRAP2-1 (NCT01705626)
- Where
- 11 sites in Austria, Hungary and 4 more
- Volunteers
- 500 people
- Sponsor
- CENTOGENE GmbH Rostock
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
Chronic Pain, Musculoskeletal Pain
Empowered Relief for Caregivers
This study is testing a new program called Empowered Relief for Caregivers (ER-CY), designed to support caregivers of children and teens who live with chronic pain. ER-CY is a single two-hour class delivered online.
- Conditions
- Chronic Pain, Musculoskeletal Pain, Fibromyalgia, Neuropathic Pain
- Trial name
- ER-CY (NCT07636980)
- Testing
- Empowered Relief for Caregivers (ER-CY)
- Where
- United States
- Volunteers
- 80 people
- Sponsor
- Stanford University
Progressive Supranuclear Palsy, Dementia With Lewy Bodies
In-Home Care for Patients With PSP and Related Disorders
Progressive Supranuclear Palsy and related disorders (PRD) are debilitating, costly, and understudied conditions. Improving access to comprehensive, specialized, in-home patient care offers the potential to minimize the downward…
- Conditions
- Progressive Supranuclear Palsy, Dementia With Lewy Bodies, Multiple System Atrophy, Corticobasal Syndrome
- Trial name
- NCT03552484
- Testing
- Home Visit Program, Usual Care/Online Survey
- Where
- United States
- Volunteers
- 56 people, healthy volunteers welcome
- Sponsor
- Rush University Medical Center
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 Mediterranean Fever, Autonomic Dysfunction
Investigation of Autonomic Dysfunction in Patients With Familial Mediterranean Fever
Familial Mediterranean Fever (FMF) is the most common inherited autoinflammatory disease affecting 150,000 patients worldwide. Periodic febrile exacerbations, peritonitis, and pleuritis are characteristic disease features.
- Trial name
- NCT05596643
- Testing
- Composite Autonomic Symptom Score-31, r-r interval variability with normal breathing, r-r interval variability with deep breathing
- Where
- Turkey (Türkiye)
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Marmara University
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