Clinical trials: Genetic Conditions
Dry Eye Disease, Dry Eye
Neurological Dry Eye Study Stimulating Both Lacrimal and Vagus Nerves
This is a study on neurological dry eye disease, focusing on a patented over-the-counter supplement supporting tear production neurologically, including the lacrimal and vagus nerves.
- Trial name
- NCT06271005
- Testing
- NeuroTears
- Volunteers
- 75 people
- Sponsor
- Genetic Disease Investigators
Atopic Dermatitis, Effects of Immunotherapy
Effect of Sublingual Immunotherapy in Patients With Atopic Dermatitis
Atopic dermatitis (AD) is a chronic and recurrent inflammatory disease, prevalent between 1 and 20% in the world population, with a predominance of childhood, but which may be present in adult life.
- Trial name
- NCT03388866
- Testing
- Mite extract sublingual immunotherapy (SLIT)
- Where
- Brazil
- Volunteers
- 91 people
- Sponsor
- Casa Espirita Terra de Ismael
Rett Syndrome
Nutritional Aspects of Rett Syndrome
OBJECTIVES: I. Determine dietary macronutrient intake in children with Rett syndrome and in healthy controls. II. Measure sleeping and awake metabolic rates in various positions, i.e., reclining, sitting, and standing, by…
- Trial name
- NCT00004656
- Testing
- Metabolic assessment with body composition evaluation
- Volunteers
- healthy volunteers welcome
- Sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Childhood Absence Epilepsy, Epilepsy
Search for Genes Influencing Childhood Absence Epilepsy (CAE) Study
The purpose of our study is to identify gene(s) involved in the cause of childhood absence epilepsy (CAE).
- Conditions
- Childhood Absence Epilepsy, Epilepsy, Seizures
- Trial name
- NCT00041951
- Where
- United States
- Volunteers
- 185 people, healthy volunteers welcome
- Sponsor
- Icahn School of Medicine at Mount Sinai
Sudden Cardiac Death, Acute Coronary Event
Finnish Genetic Study for Arrhythmic Events
Finnish Genetic Study for Arrhythmic Events (FinGesture is a prospective case-control study assessing the characteristics and genetic background of consecutive series of autopsy verified out-of-hospital victims of SCD vs.
- Trial name
- FinGesture (NCT02075866)
- Where
- Finland
- Volunteers
- 8,000 people
- Sponsor
- University of Oulu
Aggressive Behavior in Fragile X Syndome
Inhibition of Aggressive Behavior in Participants With Fragile X Syndrome
The goal of this clinical trial is to learn if drug SRX246 works to treat irritability, agitation, aggression and self-injury (IAAS) behaviors in adult males with Fragile X Syndrome (FXS).
- Trial name
- NCT07328529
- Testing
- SRX246
- Where
- 3 sites in United States
- Volunteers
- 80 people
- Sponsor
- Azevan Pharmaceuticals
Obesity
Dopamine Action on Metabolism in Relation to Genotype
Obesity is a widespread disease with increasing prevalence and associated with serious secondary complications. So far, the origin of the disease, regardless of an existing positive energy balance, is not fully understood.
- Trial name
- NCT03525002
- Testing
- Bromocriptine
- Where
- 3 sites in Germany
- Volunteers
- 120 people
- Sponsor
- University Hospital Tuebingen
Chagas Disease, Heart Diseases
Genetic Architecture of Heart Disease in Rural Brazil
To examine the genetics of human susceptibility to Chagas' disease, a leading cause of heart disease throughout Latin America.
- Conditions
- Chagas Disease, Heart Diseases, Arrhythmia, Heart Failure, Congestive
- Trial name
- NCT00023556
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3
An Active Treatment Study of SRK-015 in Patients With Type 2 or Type 3 Spinal Muscular Atrophy
The TOPAZ study will assess the safety and efficacy of SRK-015 in later-onset Spinal Muscular Atrophy (SMA Type 2 and Type 3) in pediatric and adult patients.
- Conditions
- Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3, Spinal Muscular Atrophy Type 2, SMA
- Trial name
- TOPAZ (NCT03921528)
- Testing
- SRK-015
- Where
- 16 sites in United States, Italy and 2 more
- Volunteers
- 58 people
- Sponsor
- Scholar Rock, Inc.
Cystic Fibrosis (CF)
Autogenic Drainage Versus Mechanical Percussion in Children With Cystic Fibrosis
The goal of this clinical trial is to compare the effects of autogenic drainage and mechanical percussion on lung function, dyspnea, and quality of life in children with cystic fibrosis.
- Trial name
- ADMP-CF (NCT07729787)
- Testing
- Autogenic Drainage, Mechanical Percussion
- Where
- Pakistan
- Volunteers
- 32 people
- Sponsor
- Riphah International University
Bardet-Biedl Syndrome, Orphan Diseases
Bardet-Biedl Syndrome Study: Clinical and Genetic Epidemiology Study in Adults
This study is based on the study of the natural history of a rare disorder: the Bardet-Biedl syndrome (BBS) (which is associated with retinitis pigmentosa, polydactyly, cognitive impairment, obesity, and kidney failure).
- Trial name
- NCT00213811
- Testing
- clinical, biological, and radiological
- Where
- France
- Volunteers
- 40 people
- Sponsor
- University Hospital, Strasbourg, France
Myocardial Infarction, Stroke
My Research Legacy Pilot Study
The My Research Legacy Pilot Study will establish a participant registry that collects self-reported health data and answers to online survey questions about individual daily choices, diets, and exercise; data from wearable…
- Conditions
- Myocardial Infarction, Stroke, Cardiomyopathy, Dilated, Systolic Heart Failure
- Trial name
- NCT02958098
- Volunteers
- 2,267 people
- Sponsor
- American Heart Association
Hemophilia A
Joint Status in Subjects With Severe Hemophilia A in Relation to Different Treatment Regimens
The joint status (knees, ankles) of patients suffering from severe Hemophilia A (too little blood clotting factor VIII in blood) is evaluated in a single magnetic resonance imaging session. No study medication is given.
- Trial name
- NCT00927667
- Testing
- No Drug
- Where
- 15 sites in Germany, Greece and 4 more
- Volunteers
- 156 people
- Sponsor
- Bayer
Cardiac Glycogenosis, Cardiomyopathy
ATR 1072 in Participants With PRKAG2 Syndrome
ATR 1072 is an investigational medicine being studied in adults with PRKAG2 Syndrome. This study will evaluate the safety of ATR 1072 and how the body processes the drug.
- Conditions
- Cardiac Glycogenosis, Cardiomyopathy, Hypertrophic, Wolff-Parkinson-White Syndrome
- Trial name
- NCT07738107
- Testing
- ATR 1072
- Where
- United States
- Volunteers
- 37 people
- Sponsor
- Atrium Therapeutics
Cesarean Section Complications
The Effect of Paula Method Exercises on Post-cesarean Section Distension
Aim:To examine the effect of "Paula Method Exercises" after cesarean section on the prevention of postoperative distention and pain due to distension.
- Trial name
- NCT05283707
- Testing
- Paula Method Exercises
- Where
- Turkey (Türkiye)
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- Akdeniz University
Myocardial Infarction
Genetic Variation in Platelet Aggregation
The aim of the study is to test whether genetic variation in the alpha 2A adrenergic receptor affects diurnal variation in platelet aggregation.
- Trial name
- NCT01576536
- Where
- United States
- Volunteers
- 117 people, healthy volunteers welcome
- Sponsor
- Vanderbilt University
Lesch-Nyhan Syndrome
Phase II Pilot Study of Aminoimidazole Carboxamide Riboside (AICAR), a Precursor of Purine Synthesis, for Lesch-Nyhan Disease
OBJECTIVES: I. Determine the effects of aminoimidazole carboxamide riboside (AICAR) on hematologic manifestations of Lesch-Nyhan disease. II.
- Trial name
- NCT00004314
- Testing
- aminomidazole carboxamide riboside
- Volunteers
- 2 people
- Sponsor
- National Center for Research Resources (NCRR)
Familial Lymphohistiocytosis, Lymphohistiocytosis
Impact of Ultra-fast Genetic Diagnosis of Familial Lymphohistiocytosis on the Time to Bone Marrow Transplantation and Overall Survival
Familial lymphohistiocytosis (FHL) is a group of rare genetic diseases (around fifteen cases per year in France). The defect in T lymphocyte cytotoxicity resulting from this disease is responsible for hemophagocytic…
- Trial name
- LongRead-HLH (NCT07741747)
- Testing
- Third-generation sequencing
- Where
- France
- Volunteers
- 240 people
- Sponsor
- Assistance Publique Hopitaux De Marseille
Nephrocalcinosis, Renal Calculi
Randall's Plaque Study: Pathogenesis and Relationship to Nephrolithiasis
Kidney stones are very common. They affect 3-5% of the population in the United States. Many people are hospitalized for the treatment of kidney stones and some may die.
- Conditions
- Nephrocalcinosis, Renal Calculi, Hypercalciuria, Hyperparathyroidism
- Trial name
- NCT00169806
- Testing
- videotape for mapping of renal anatomy and papillary biopsy
- Where
- United States
- Volunteers
- 600 people
- Sponsor
- Indiana Kidney Stone Institute
Cystinosis
Open-Label, Safety and Superior Effectiveness Study of Cysteamine Bitartrate Delayed-Release Capsules (RP103) in Cystinosis
The purpose of this study is to gather information about the effectiveness (how well it works to treat cystinosis) and safety of a new form of cysteamine bitartrate called RP103, compared to the already-approved drug cystinosis…
- Trial name
- NCT01733316
- Testing
- RP103, Cystagon®
- Where
- 14 sites in United States, Belgium and 4 more
- Volunteers
- 41 people
- Sponsor
- Amgen
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