Clinical trials: Autonomic Neuropathy
Burns
Determination of Nerve Growth Factor (NGF) Expression in Pediatric Patients With Severe Burns: a Pilot Study
The goal of this observational study is to determine the blood values of NGF in severely burned patients, to describe any changes over time and finally to correlate the expression of NGF to the severity of the burn.
- Trial name
- NCT05751174
- Testing
- pediatric patients affected by severe burns
- Where
- Italy
- Volunteers
- 12 people
- Sponsor
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
16P11.2 Deletion Syndrome, 16p11.2 Duplications
Online Study of People Who Have Genetic Changes and Features of Autism: Simons Searchlight
Simons Searchlight is an observational, online, international research program for families with rare genetic variants that cause neurodevelopmental disorders and may be associated with autism.
- Conditions
- 16P11.2 Deletion Syndrome, 16p11.2 Duplications, 1Q21.1 Deletion, 1Q21.1 Microduplication Syndrome (Disorder)
- Trial name
- NCT01238250
- Where
- 2 sites in United States
- Volunteers
- 100,000 people
- Sponsor
- Simons Searchlight
Prostatic Neoplasms
Prevalence of HRR-related Genes Mutations and Prognosis in Metastatic Castration Resistant Prostate Cancer (mCRPC) Patients in Real World Setting
The purpose of this study is to investigate the prevalence of tissue homologous recombination repair (HRR)-related gene mutations (positive/negative/Variant of uncertain significance (VUS)), clinical outcome such as…
- Trial name
- ZENSHIN (NCT04425200)
- Where
- 26 sites in Japan
- Volunteers
- 205 people
- Sponsor
- AstraZeneca
Port Wine Stain
The Role of Angiogenesis-related Pathways in the Development of Port Wine Stains
1. Port wine stain (PWS) is a congenital, progressive vascular malformation of human skin involving the superficial vascular plexus that occurs in estimated 3-5 children per 1,000 live births.
- Trial name
- NCT03948997
- Testing
- Pulsed dye laser (PDL)
- Volunteers
- 80 people
- Sponsor
- xjpfW
PIK3CA-Related Overgrowth Spectrum (PROS)
Retrospective Chart Review Study of Patients With PIK3CA-Related Overgrowth Spectrum Who Have Received Alpelisib
The study was a site-based retrospective non-interventional medical chart review of pediatric and adult male and female patients with PIK3CA-Related Overgrowth Spectrum (PROS) who initiated alpelisib at least 24 weeks before the…
- Trial name
- EPIK-P1 (NCT04285723)
- Testing
- alpelisib
- Where
- 7 sites in United States, Australia and 3 more
- Volunteers
- 57 people
- Sponsor
- Novartis Pharmaceuticals
PIK3CA-Related Overgrowth Spectrum
A Study of the Epidemiology and Hospital Management of Patients With PROS in France
The purpose of this study was to estimate the incidence and prevalence of a group of genetic disorders known as PIK3CA-Related Overgrowth Spectrum (PROS) in France.
- Trial name
- EpiPROS (NCT07222423)
- Where
- United States
- Volunteers
- 3,605 people
- Sponsor
- Novartis Pharmaceuticals
PIK3CA-Related Overgrowth Spectrum (PROS)/Proteus Syndrome
Study of Miransertib (MK-7075) in Participants With PIK3CA-related Overgrowth Spectrum and Proteus Syndrome (MOSAIC) (MK-7075-002)
This is an open label, Phase 1/2 study of oral miransertib (MK-7075) administered to participants at least 2 years of age with phosphatidylinositol-4,5-bisphosphate 3-kinase, catalytic subunit alpha (PIK3CA)-related Overgrowth…
- Trial name
- MOSAIC (NCT03094832)
- Testing
- Miransertib
- Where
- 11 sites in United States, Australia and 2 more
- Volunteers
- 50 people
- Sponsor
- ArQule, Inc., a subsidiary of Merck Sharp & Dohme LLC, a subsidiary of Merck & Co., Inc. (Rahway, NJ USA)
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
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
PIK3CA-related Overgrowth Spectrum, CLOVES Syndrome
National Evaluation of Patients With PIK3CA-Related Overgrowth Spectrum (PROS)
Overgrowth syndromes are rare genetic disorders defined by tissue hypertrophy that can be either localized or generalized, affecting both latitudinal and longitudinal growth.
- Conditions
- PIK3CA-related Overgrowth Spectrum, CLOVES Syndrome, Klippel Trenaunay Syndrome, Megalencephaly
- Trial name
- COSY (NCT05563831)
- Testing
- national registry
- Where
- France
- Volunteers
- 28 people
- Sponsor
- Institut National de la Santé Et de la Recherche Médicale, France
Optic Pathway Glioma
Safety and Efficacy of the PAINLESS Nerve Growth Factor CHF6467 in Optic Pathway Glioma (OPG)
Infantile optic pathway glioma (OPG) is generally benign and slow-growing, but due to infiltration and compression of sensitive neuronal structures in the optical pathways, progressive visual loss is a frequent and highly…
- Trial name
- NGF-OPG (NCT05733572)
- Testing
- CHF6467
- Where
- Italy
- Volunteers
- 36 people
- Sponsor
- Benedetto Falsini
Coronary Heart Disease, Diabetes
The Effect of Autonomic Neuropathy Status on the Endpoint Events in Diabetes Patients With Coronary Heart Disease and Sleep Apnea.
Background: diabetes combined with cardiac autonomic neuropathy (CAN) can denervate the heart, which may prevent patients with sleep apnea syndrome from bradycardia in the early stage, and directly show an increased risk of…
- Conditions
- Coronary Heart Disease, Diabetes, Sleep Apnea Syndrome, Cardiovascular Autonomic Neuropathy (CAN)
- Trial name
- NCT07723222
- Testing
- The total score of Ewing test (i.e. cardiovascular autonomic reflex test and heart rate variability) was ≥1, which was divided into cardiac autonomic neuropathy group (CAN grou
- Volunteers
- 326 people
- Sponsor
- Xiangtan Central Hospital
Neuropathy; Peripheral, Autonomic, in Diabetes Mellitus (Manifestation)
The Relationship Between Vascular Adhesion Protein-1 and Diabetic Cardiovascular Autonomic Neuropathy
Exploring the relationship between serum VAP-1 and cardiovascular autonomic neuropathy in subjects with type 2 diabetes mellitus
- Trial name
- NCT01706289
- Where
- Taiwan
- Volunteers
- 150 people, healthy volunteers welcome
- Sponsor
- National Taiwan University Hospital
Rare Disorders, Undiagnosed Disorders
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
The Coordination of Rare Diseases at Sanford (CoRDS) is an international patient registry that serves as a centralized resource for rare disease research.
- Conditions
- Rare Disorders, Undiagnosed Disorders, Disorders of Unknown Prevalence, Cornelia De Lange Syndrome
- Trial name
- CoRDS (NCT01793168)
- Where
- United States
- Volunteers
- 20,000 people
- Sponsor
- Sanford Health
Diabetic Autonomic Neuropathy, Diabetic Neuropathy
High Diagnostic Performance of the Indicator Plaster Neuropad for the Detection of Diabetic Autonomic Neuropathy
Diabetic autonomic neuropathy (DAN) is a prevalent and severe complication of Diabetes Mellitus (DM). When the autonomic regulation of the cardiovascular system is affected, it is referred to as cardiovascular autonomic…
- Trial name
- NCT06734143
- Testing
- Neuropad
- Where
- Greece
- Volunteers
- 174 people
- Sponsor
- Aristotle University Of Thessaloniki
Epilepsy, Tuberous Sclerosis Complex
Everolimus (RAD001) Therapy for Epilepsy in Patients With Tuberous Sclerosis Complex (TSC)
The goal of this study is to learn if the study drug RAD001 can reduced the number of epileptic seizures, and can be taken safety by people who have epilepsy associated with Tuberous Sclerosis Complex.
- Trial name
- NCT01070316
- Testing
- Everolimus
- Where
- 2 sites in United States
- Volunteers
- 20 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Rare Unresectable or Metastatic BRAF V600E Mutation-positive Solid Tumors
Clinical Study To Further Evaluate The Efficacy Of Dabrafenib Plus Trametinib In Patients With Rare BRAF V600E Mutation-Positive Unresectable or Metastatic Solid Tumors
This study is a phase IV, pragmatic single-arm prospective, open label study in pediatric (1 years or older) and adult study participants with rare BRAF V600E mutation-positive unresectable or metastatic solid tumors for whom a…
- Trial name
- NCT05868629
- Testing
- Non-investigational
- Where
- 8 sites in United States
- Volunteers
- 40 people
- Sponsor
- Novartis Pharmaceuticals
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
Giant Axonal Neuropathy (GAN)
Intraneural Administration of scAAV9/JeT-GAN Into the Vagus Nerve for Patients With Giant Axonal Neuropathy (GAN)
Giant axonal neuropathy (GAN) is a rare pediatric disorder caused by autosomal recessive mutations in the GAN gene. GAN is a multisystem, neurodegenerative disorder affecting the peripheral nervous system (PNS), central nervous…
- Trial name
- NCT07543991
- Testing
- scAAV9/JeT-GAN
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- University of Texas Southwestern Medical Center
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