Clinical Trials on Familial Dysautonomia

Total 1754 results

NYU Langone Health

Completed

The Safety and Tolerability of Kinetin, in Patients With Familial Dysautonomia

Familial Dysautonomia

United States
NYU Langone Health

Completed

The Effects Of Bronchodilator Therapy On Respiratory And Autonomic Function In Patients With Familial Dysautonomia

Familial Dysautonomia

United States
NYU Langone Health

Withdrawn

Telemedicine Clinical Trial for Cognitive Behavioral Therapy in Familial Dysautonomia

Familial Dysautonomia

United States
NYU Langone Health

Recruiting

The Natural History of Familial Dysautonomia

Hereditary Sensory and Autonomic Neuropathies | Familial Dysautonomia (Riley-Day Syndrome) | Hereditary Sensory and Autonomic Neuropathy 3

United States, Israel
NYU Langone Health

Not yet recruiting

Dexmedetomidine Sublingual Film for the Ambulatory Treatment of Hyperadrenergic Autonomic Crisis in Patients With Familial Dysautonomia

Familial Dysautonomia

United States
NYU Langone Health

Not yet recruiting

Pilot Study of Dexmedetomidine Sublingual Film for the Ambulatory Treatment of Hyperadrenergic Autonomic Crisis in Patients With Familial Dysautonomia

Familial Dysautonomia

United States
NYU Langone Health

Completed

The Nutritional Supplement Phosphatidylserine in Patients With Familial Dysautonomia

Familial Dysautonomia

United States
NYU Langone Health

Withdrawn

The Safety, Tolerability and Efficacy of Dronabinol, for the Treatment of Nausea and Vomiting in Familial Dysautonomia

Nausea | Vomiting | Familial Dysautonomia

United States
NYU Langone Health

Completed

Carbidopa for the Treatment of Nausea and Vomiting in Familial Dysautonomia

Familial Dysautonomia
NYU Langone Health

Completed

Carbidopa for the Treatment of Excessive Blood Pressure Variability (CarbiFD)

Dysautonomia, Familial | Baroreflex Failure Syndrome

United States
NYU Langone Health

Completed

A Trial of Cognitive Behavioral Therapy in Familial Dysautonomia

Anxiety Disorders | Familial Dysautonomia | Dysthymia | Paroxysmal Hypertension | Autosomal Recessive Disease

United States
St Carlos Hospital, Madrid, Spain

Recruiting

Renal Arterial Denervation in Sympathetic Dysautonomia (RANSOM)

Hypertension | Blood Pressure Disorders | Renal Denervation | Blood Pressure Variability | Increased Variability | Sympathetic Dysautonomia

Spain
McGill University Health Centre/Research Institute...

Completed

Efficacy Study of an Online Educational Module Before Carrier Genetic Screening in Persons of Ashkenazi Jewish Descent.

Familial Dysautonomia | Tay Sachs Disease | Canavan Disease

Canada
Instituto Nacional de Cardiologia Ignacio Chavez

Unknown

Autonomic Cardiovascular Neuropathy in Recently Diagnosed DM2 Patients (ACNDM2) (ACNDM2)

Dysautonomia

Mexico
National Institute of Neurological Disorders and...

Recruiting

Data Collection of Standard Care of Patients in the EMG Section

Neuropathy | Muscle Disorders | Dysautonomia

United States
Wake Forest University Health Sciences

Completed

MRI to Assess the Effects of Dysautonomia and Chronic Nausea on Brain Transmitters

Dysautonomia

United States
University of Colorado, Denver

Completed

Implementation of Transdx Group for POTS

Depression | Anxiety | Dysautonomia

United States
Centre Hospitalier Universitaire de Saint Etienne

Recruiting

Longitudinal Evolution of Biomarkers of Dysautonomia and Inflammation During Sepsis in Children (DysREAped)

Sepsis | Autonomic Nervous System

France
Johns Hopkins University

Completed

Temporary Celiac Ganglion Block as a Test Before Celiac Ganglion Resection for Dysautonomia-Related Bowel Dysmotility

Dysautonomia

United States
Johns Hopkins University

Withdrawn

Controlling Hyperadrenergic Activity in Neurologic Injury (CHAIN)

Traumatic Brain Injury | Dysautonomia

United States
Université de Sherbrooke
Centre de Recherche sur le Vieillissement (CdRV)

Recruiting

Telerehabilitation for Dysautonomia in Parkinson's Disease (PKT)

Parkinson Disease

Canada
Karolinska University Hospital
Region Stockholm; Vinnova; Eurostars

Active, not recruiting

Dysautonomia and Systemic Interactions in Traumatic Brain Injury (DYSI-TBI)

Traumatic Brain Injury | Dysautonomia | Inflammation, Brain | Sympathetic; Imbalance

Sweden
Moens Maarten

Recruiting

Dysautonomia in Patients Post COVID-19 Infection (DYSCO)

Covid19

Belgium
Assistance Publique Hopitaux De Marseille

Not yet recruiting

Familial Aortopathies and Cellular Exploration (FACE)

Familial Aortopathies

France
Evangelismos Hospital
National and Kapodistrian University of Athens; LONG COVID GREECE; 414 Military...

Recruiting

Post Covid-19 Dysautonomia Rehabilitation Randomized Controlled Trial (LoCoDiRe-Dys)

Dysautonomia | Post-Acute COVID-19 Syndrome

Greece
National Medical Research Center for Therapy and...
Moscow State University of Medicine and Dentistry

Recruiting

Genetic Testing and Motivational Counseling for FH (GENMOTIV-FH)

Medication Adherence | Adherence, Medication | Treatment Adherence | Familial Hypercholesterolemia | Motivational Interviewing | Adherence, Patient | Treatment Adherence and Compliance | Patient Compliance | Adherence | Hypercholesterolemia, Familial | Patient Adherence | Hypercholesterolemia, Autosomal Dominant and other conditions

Russian Federation
University Hospital, Bordeaux
Ministry of Health, France

Terminated

Intrathecal Baclofen Therapy and Paroxysmal Dysautonomia in Severe Brain-Injured Patients

Traumatic Brain Injury | Coma | Dysautonomia | Cerebral Anoxia | Hypertonia

France
Centre Hospitalier Universitaire de Saint Etienne

Completed

Testing for Dysautonomia in Patients Hospitalized for SARS-CoV-2 Infection (COVID-19) : COVIDANS Study (COVIDANS)

SARS-CoV 2

France
University of California, Los Angeles

Recruiting

Mindfulness in Post Acute Sequelae of SARS-CoV-2 Infection (PASC) Dysautonomia

Dysautonomia | Long COVID

United States
University of California, San Diego
Beth Israel Deaconess Medical Center; Drexel University

Recruiting

Blood Pressure Effects on Cognition and Brain Blood Flow in PD

Parkinson Disease | Orthostatic Hypotension | Dysautonomia

United States
Alnylam Pharmaceuticals

Completed

The Study of an Investigational Drug, Revusiran (ALN-TTRSC), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Patients Whose Disease Has Continued to Worsen Following Liver Transplant

ATTR Amyloidosis | Transthyretin (TTR)-Mediated Amyloidosis | Familial Amyloidotic Polyneuropathy (FAP) | Familial Amyloid Neuropathies

France, Germany, Portugal, United Kingdom, Sweden, Spain
Leiden University Medical Center
Jacobijn Gussekloo, Prof. dr.; Simon P. Mooijaart, Prof. dr.

Recruiting

Longevity Families of the Netherlands (LOF-NL)

Aging | Extreme Longevity | Longevity | Familial Longevity

Netherlands
National Institute of Dental and Craniofacial Research...

Completed

Evaluation of the Role of the Autonomic Nervous System in Sj(SqrRoot)(Delta)Gren s Syndrome

Sjogren's Syndrome | Dysautonomia

United States
Boston University
National Institute of Neurological Disorders and Stroke (NINDS); Food and Drug...

Completed

The Effect of Diflunisal on Familial Amyloidosis

Familial Amyloid Polyneuropathy | Familial Amyloidosis

United States, Italy, Japan, Sweden, United Kingdom
Pfizer

Completed

The Effect Of Tafamidis For The Transthyretin Amyloid Polyneuropathy Patients With V30M Or Non-V30M Transthyretin

Transthyretin Familial Amyloid Polyneuropathy

Japan
CENTOGENE GmbH Rostock
Alnylam Pharmaceuticals

Recruiting

TRAMmoniTTR Study Genetic Screening of an At-risk Population for hATTR and Monitoring of TTR Positive Subjects (TRAMmoniTTR)

Cardiomyopathies | Polyneuropathies | Transthyretin Amyloidosis | Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy | Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy

Germany, Austria, Switzerland
Regeneron Pharmaceuticals
Sanofi

Terminated

Long Term Safety Study of PRALUENT

Heterozygous Familial Hypercholesterolemia | Non-familial Hypercholesterolemia

United States, Bulgaria, Estonia, Russian Federation, South Africa, Ukraine
Merck Sharp & Dohme LLC

Terminated

A Study of Extended-release (ER) Niacin/Laropiprant in Adolescents With Heterozygous Familial Hypercholesterolemia (MK-0524A-158)

Hypercholesterolemia, Familial | Heterozygous Familial Hypercholesterolemia
Institut Investigacio Sanitaria Pere Virgili

Recruiting

Early Detection of Familial Hypercholesterolemia in Children (DECOPIN)

Familial Hypercholesterolemia | Familial Hypercholesterolemia - Homozygous | Familial Hypercholesterolemia - Heterozygous

Spain
Novartis Pharmaceuticals

Active, not recruiting

Study to Evaluate Efficacy and Safety of Inclisiran in Adolescents With Homozygous Familial Hypercholesterolemia (ORION-13)

Familial Hypercholesterolemia - Homozygous

Greece, Lebanon, Turkey, France, Canada, Malaysia, Netherlands, United States
Eidos Therapeutics, a BridgeBio company

Withdrawn

Efficacy and Safety of Acoramidis (AG10) in Subjects With Transthyretin Amyloid Polyneurophathy (ATTRibute-PN)

Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy
Eidos Therapeutics, a BridgeBio company

Withdrawn

Efficacy and Safety of AG10 in Subjects With Transthyretin Amyloid Polyneurophathy (ATTRibute-PN)

Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy

United States
SOM Innovation Biotech SA
Hospital Vall d'Hebron

Completed

Study of SOM0226 in Familial Amyloid Polyneuropathy

Familial Amyloid Polyneuropathy (FAP)

Spain
University Hospital, Brest

Unknown

Dysautonomic Phenotype in Male Patients With MECP2 Mutation (MECP2BOYS)

Dysautonomia | Masculinity | MECP2-Related Severe Neonatal Encephalopathy

France
Marcello Arca
Pfizer; Göteborg University; University of Helsinki

Not yet recruiting

In Vivo Metabolism of apoB-containing Lipoproteins in ANGPTL3 Deficient Subjects

Familial Hypobetalipoproteinemia | Familial Hypobetalipoproteinemia - Heterozygous Form
Novartis Pharmaceuticals

Recruiting

Long-term Safety and Tolerability of Inclisiran in Participants With HeFH or HoFH Who Have Completed the Adolescent ORION-16 or ORION-13 Studies (V-PEDS-OLE)

Heterozygous or Homozygous Familial Hypercholesterolemia

Netherlands, Israel, Hungary, Italy, Germany, Spain, France, Norway, South Africa, Turkey, United Kingdom, Canada, Switzerland, Brazil, Lebanon, Slovenia, United States, Russian Federation, Taiwan
Alnylam Pharmaceuticals

Completed

APOLLO: The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis

Amyloid Neuropathies | Amyloid Neuropathies, Familial | TTR-mediated Amyloidosis | Amyloidosis, Hereditary | Amyloidosis, Hereditary, Transthyretin-Related | Familial Amyloid Polyneuropathies

United States, Canada, Argentina, Australia, Bulgaria, Cyprus, Italy, Japan, Malaysia, Mexico, Netherlands, Portugal, Spain, Sweden, Germany, France, Brazil, Turkey, Korea, Republic of, Taiwan, United Kingdom
Intellia Therapeutics

Recruiting

Long-Term Follow-Up (LTFU) of Subjects Dosed With NTLA-2001

Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy | Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy | Wild-Type Transthyretin Cardiac Amyloidosis

United Kingdom, New Zealand, Sweden
Intellia Therapeutics

Active, not recruiting

Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NTLA-2001 in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy (ATTRv-PN) and Patients With Transthyretin Amyloidosis-Related Cardiomyopathy (ATTR-CM)

Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy | Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy | Wild-Type Transthyretin Cardiac Amyloidosis

France, United Kingdom, New Zealand, Sweden
Alnylam Pharmaceuticals

No longer available

Expanded Access Protocol of Patisiran for Patients With Hereditary ATTR Amyloidosis (hATTR)

Amyloid Neuropathies | Amyloid Neuropathies, Familial | TTR-mediated Amyloidosis | Amyloidosis, Hereditary | Amyloidosis, Hereditary, Transthyretin-Related | Familial Amyloid Polyneuropathies

Clinical Trials on Familial Dysautonomia

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