Clinical trials: Autonomic Neuropathy
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
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)
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
Overactive Bladder
Urinary Neurotrophin Levels in Healthy Children
The aim of this study is to analyse urinary neurotrophin levels in healthy children. Urine samples will be collected at 3 different times of day (morning, noon, and evening) at urge sensation from all age groups of children who…
- Trial name
- NCT02389270
- Volunteers
- 150 people, healthy volunteers welcome
- Sponsor
- Children's Hospital Zagreb
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
Hypoxic-Ischemic Encephalopathy
Study of Cerebrolysin for Treatment of Infants With History of Neonatal Hypoxic Ischemic Encephalopathy
The purpose of this study is to determine whether nerve growth factor (cerebrolysin®) therapy will improve the psychomotor outcome in infants with moderate and severe hypoxic ischemic encephalopathy after hospital discharge.
- Trial name
- CerebroHIE (NCT01059461)
- Testing
- Cerebrolysin®
- Where
- 2 sites in Egypt
- Volunteers
- 40 people
- Sponsor
- Sahar M.A. Hassanein, MD
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
Advanced Stage Solid Tumors
ATRi Transition Rollover Study
The main purpose of the study was to monitor the safety of participant receiving long-term treatment of Berzosertib as monotherapy.
- Trial name
- NCT03309150
- Testing
- Berzosertib
- Where
- United Kingdom
- Volunteers
- 1 people
- Sponsor
- Merck KGaA, Darmstadt, Germany
Cardiac Autonomic Neuropathy, Diabete Type 1
Evaluation of Cardiac Autonomic Neuropathy in Children and Adolescents With Type 1 Diabetes Mellitus
study is to evaluate the prevalence of cardiac autonomic neuropathy in children with Type 1 DM and correlation with duration of DM and glycemic control.
- Conditions
- Cardiac Autonomic Neuropathy, Diabete Type 1, Pediatrics
- Trial name
- NCT07613372
- Testing
- Holter
- Where
- Egypt
- Volunteers
- 75 people
- Sponsor
- Marwa Abouelela Abd Elsalam
Megalencephaly-capillary Malformation Polymicrogyria Syndrome (MCAP)
Assessment of the Efficacy and Safety of Alpelisib (BYL719) in Pediatric and Adult Patients With Megalencephaly-CApillary Malformation Polymicrogyria Syndrome (MCAP)
This study is a two periods multi-center Phase II trial, with a 6 months double-blind, placebo-controlled period followed by open label period, to assess the efficacy and safety of alpelisib (BYL719) in pediatric and adult…
- Trial name
- SESAM (NCT05577754)
- Testing
- Alpelisib (BYL719), Optional lumbar puncture + blood sample
- Where
- 13 sites in France
- Volunteers
- 20 people
- Sponsor
- Centre Hospitalier Universitaire Dijon
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
Same-day Surgery
Is Partial Knee Replacement as Same-day Surgery Generalizable?
Fast-track/rapid recovery/Enhanced Recovery After Surgery (ERAS) programs have proven both safe and effective in joint replacement surgery, to the degree where same-day surgery have been tested in selected cases.
- Trial name
- NCT04790591
- Testing
- Same-day surgery
- Where
- 2 sites in Sweden
- Volunteers
- 33 people
- Sponsor
- Region Skane
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
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
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
Peutz-Jeghers Syndrome
Efficacy of Rapamycin (Sirolimus) in the Treatment of Peutz-Jeghers Syndrome
A prospective non-randomized open label single arm clinical trial to examine the efficacy and safety of sirolimus in patients with Peutz-Jeghers Syndrome.
- Trial name
- NCT03781050
- Testing
- Rapamycin
- Where
- China
- Volunteers
- 10 people
- Sponsor
- Peking Union Medical College Hospital
Tumor, Tumor, Solid
Phase 2 Basket Trial of Nab-sirolimus in Patients With Malignant Solid Tumors With Pathogenic Alterations in TSC1/TSC2 Genes (PRECISION 1)
A Phase 2 multi-center open-label basket trial of nab-sirolimus for adult and adolescent patients with malignant solid tumors harboring pathogenic inactivating alterations in TSC1 or TSC2 genes
- Conditions
- Tumor, Tumor, Solid, Metastasis, Metastatic Cancer
- Trial name
- NCT05103358
- Testing
- nab-sirolimus
- Where
- 166 sites in United States, Puerto Rico and 1 more
- Volunteers
- 128 people
- Sponsor
- Aadi Bioscience, Inc.
Vascular Anomalies, Vascular Anomaly
24VA022; VATCH Alpelisib for TIE2/PIK3CA Pathway VAs
The study will enroll participants 2 months of age up to 30 years of age. The purpose of this study is to assess the effectiveness and safety of Alpelisib (the "Study Drug") in patients with PIK3CA/TIE-2/TEK pathway driven…
- Conditions
- Vascular Anomalies, Vascular Anomaly, TIE2/PIK3CA Pathway Driven Vascular Anomalies
- Trial name
- VATCH (NCT07543822)
- Testing
- alpelisib (BYL719)
- Where
- United States
- Volunteers
- 61 people
- Sponsor
- Children's Hospital of Philadelphia
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
Diabetic Autonomic Neuropathy
Efficacy of Administration of ACE-Inhibition on Autonomic and Peripheral Neuropathy in Patients With Diabetes Mellitus
Patients with diabetes mellitus and definite autonomic and peripheral neuropathy were randomized to receive quinapril or no treatment for 2 years.
- Trial name
- NCT03031834
- Testing
- Quinapril
- Sponsor
- Aristotle University Of Thessaloniki