Clinical trials: Functional Deformities
Dilated Cardiomyopathy
Bone Marrow Derived Adult Stem Cells for Dilated Cardiomyopathy
A randomised, double-blind, placebo-controlled trial to evaluate the role of intracoronary injection of progenitor cells compared to placebo injection in patients with Dilated Cardiomyopathy who have been pre-treated with G-CSF…
- Trial name
- REGEN-DCM (NCT01302171)
- Testing
- granulocyte colony stimulating factor (GCSF), bone marrow mononuclear cells
- Where
- United Kingdom
- Volunteers
- 60 people
- Sponsor
- Barts & The London NHS Trust
Fragile X Syndrome
Optical Imaging as a Tool for Monitoring Brain Function in Fragile X Syndrome
The objective of this project is to explore the potential of functional near- infrared spectroscopy (fNIRS) as innovative functional biomarker for clinical trial readiness in Fragile X Syndrome (FXS) that is still without cure.
- Trial name
- NCT06293027
- Where
- Italy
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- IRCCS Fondazione Stella Maris
Type II Diabetes Mellitus
Effects of a Spiced Meat Patty on Inflammation in Men With Type 2 Diabetes
Polyphenols belong to the largest group of secondary metabolites produced by plants, mainly, in response to biotic or abiotic stresses such as infections, wounding, UV irradiation, exposure to ozone, pollutants, and other hostile…
- Trial name
- NCT01076829
- Testing
- Spice polyphenols
- Where
- United States
- Volunteers
- 10 people, healthy volunteers welcome
- Sponsor
- University of California, Los Angeles
Duchenne Muscular Dystrophy
Investigation of the Effects of Functional Inspiratory Muscle Training Applied With Tele-Rehabilitation on Respiratory and Functional Parameters and Quality of Life in Children With Duchenne Muscular Dystrophy: Randomized Controlled Study
Dushenne muscular dystrophy (DMD), which is characterized by the deficiency of dystrophin protein, prevents the muscle from maintaining its normal activity, causing progressive damage to the heart, respiratory muscles and…
- Trial name
- NCT07172737
- Testing
- Respiratory Muscle Training
- Where
- Turkey (Türkiye)
- Volunteers
- 40 people
- Sponsor
- Izmir Katip Celebi University
Hemophilia, Upper Extremity
Comparison of Upper Extremity Functional Performance in Hemophilic and Healthy Children
The goal of this observational study is to evaluate the functional performance of the upper extremity joints (scapula, shoulder, elbow, and wrist) in haemophilic children and compare muscle strength and ADL limitations with…
- Conditions
- Hemophilia, Upper Extremity, Strength, Performance
- Trial name
- NCT06826209
- Where
- Turkey (Türkiye)
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Hacettepe University
Cystic Fibrosis
Sildenafil Exercise: Role of PDE5 Inhibition
Exercise intolerance is an understudied phenomenon in people with CF. The investigators hypothesized that vascular dysfunction plays a significant role, and can be partially reversed by administration of the phosphodiesterase…
- Trial name
- NCT04039087
- Testing
- Sildenafil 40mg oral capsule
- Where
- 2 sites in United States
- Volunteers
- 31 people
- Sponsor
- National Jewish Health
Cystic Fibrosis
Development Of An Innovative Panel of Methods To Measure Intestinal Macronutrient Digestion, Absorption, and Function
Malnutrition is a significant problem in children and adults with Cystic fibrosis (CF). An impaired intestinal digestion and absorption capacity is one of the main factors responsible for the malnutrition in CF.
- Trial name
- NCT01494909
- Testing
- Ensure plus
- Where
- United States
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Texas A&M University
Hereditary Angioedema
Social Evaluated Cold Pressor Test in Hereditary Angioedema Patients
This study aims to evaluate the differences in objective and subjective stress responses between patients with hereditary angioedema and healthy individuals to a stress-induced challenge like socially-evaluated cold pressure…
- Trial name
- FROSEN (NCT06414252)
- Where
- Italy
- Volunteers
- 40 people
- Sponsor
- Istituti Clinici Scientifici Maugeri SpA
Cardiac Amyloidosis
Mitochondrial Function in Transthyretin Amyloidosis
Hereditary (familial) amyloidosis arising from the misfolding of a mutated or variant transthyretin, is the most frequent form of amyloid cardiomyopathy in the Caribbean basin.
- Trial name
- MIT-Amylose (NCT03328338)
- Where
- Martinique
- Volunteers
- 12 people
- Sponsor
- University Hospital Center of Martinique
Aplasia Cutis Congenita
Genetic and Functional Analysis of Aplasia Cutis Congenital (ACC)
The goal of this research study is to identify genes and regulatory elements on chromosomes that cause ACC. The investigators also study tissue samples from patients to learn about the processes that lead to this disorder.
- Trial name
- ACC (NCT01630421)
- Where
- United States
- Volunteers
- 600 people
- Sponsor
- UConn Health
Cystic Fibrosis
A Study Evaluating Efficacy and Safety of Elexacaftor/Tezacaftor/Ivacaftor in Subjects 6 Through 11 Years of Age With Cystic Fibrosis and F/MF Genotypes
This study will evaluate the efficacy and safety of elexacaftor (ELX) / tezacaftor (TEZ) / ivacaftor (IVA) triple combination (TC) in subjects 6 through 11 years of age with cystic fibrosis (CF) who are heterozygous for F508del…
- Trial name
- NCT04353817
- Testing
- ELX/TEZ/IVA, IVA
- Where
- 34 sites in Australia, Canada and 8 more
- Volunteers
- 121 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Dilated Cardiomyopathy
Exercise Stress MRI to Evaluate Aortic Function (Compliance, Distensibility, Pulse Wave Velocity) and Left Ventricular Function : Validation in Healthy Volunteers and in Selected Patients. A Pilot Study.
Detecting abnormalities in the left ventricular mechanical and hemodynamic response to the stress of exercise may offer early diagnostic indicators in patients suffering from valvular disease such as mitral regurgitation.
- Trial name
- NCT02018835
- Testing
- MRI, cardiac echography transthoracic
- Where
- France
- Volunteers
- 57 people, healthy volunteers welcome
- Sponsor
- Assistance Publique Hopitaux De Marseille
Infantile Colics, Constipation
Randomized, Blinded, Placebo-Controlled Evaluation of BioAmicus Complete
This is a randomized, single-blind, placebo-controlled study to evaluate the efficacy of the dietary supplement BioAmicus Complete (oral drops; a mixture of 10 strains of lacto- and bifidobacteria) in managing functional…
- Conditions
- Infantile Colics, Constipation, Infantile Diarrhea, Atopic Dermatitis
- Trial name
- NCT07444008
- Testing
- BioAmicus Complete
- Where
- Russia
- Volunteers
- 140 people
- Sponsor
- NovoNatum Ltd
Spinal Muscular Atrophy Type 3
Whole Body Vibration Therapy in Children With Spinal Muscular Atrophy
Spinal muscular atrophy (SMA) are one of the common physical disabilities in childhood. For SMA, progressive muscle weakness and early fatigue hamper the mobility of the sufferers.
- Trial name
- NCT03056144
- Testing
- whole body vibration therapy
- Where
- Hong Kong
- Volunteers
- 1 people
- Sponsor
- The Hong Kong Polytechnic University
Chronic Kidney Disease, Nephrolithiasis
Kidney Sodium Functional Imaging
The corticomedullary gradient is largely responsible for developing the gradients that are needed to concentrate urine (more solutes and less water).
- Conditions
- Chronic Kidney Disease, Nephrolithiasis, ADPKD
- Trial name
- NCT05014178
- Testing
- Sodium-23 MRI
- Where
- Canada
- Volunteers
- 200 people, healthy volunteers welcome
- Sponsor
- London Health Sciences Centre Research Institute OR Lawson Research Institute of St. Joseph's
Long QT Syndrome, Channelopathies
Physical Activity and Functional Capacity in Patients With Congenital Long QT Syndrome
Patients with congenital long QT syndrome (LQTS) have traditionally been discouraged from physical activity and competitive sports, although the rate of exercise-related arrhythmic events is considered low.
- Trial name
- CANMOVE (NCT07862491)
- Testing
- Questionnaires, Cardiopulmonary exercise testing, 24-hour Holter monitoring
- Where
- Brazil
- Volunteers
- 120 people
- Sponsor
- University of Sao Paulo
Growth Hormone Deficiency, Idiopathic Short Stature
Skeletal Muscle Effects of GH in Boys
The purpose of the study is to measure the functional effects of recombinant GH in skeletal muscle, in addition to growth promotion, in short prepubertal boys with either growth hormone deficiency or idiopathic short stature.
- Trial name
- NCT04020913
- Testing
- Somatropin injection
- Where
- United States
- Volunteers
- 45 people, healthy volunteers welcome
- Sponsor
- Nemours Children's Clinic
Functional Pancreatic Neuroendocrine Tumor, Malignant Somatostatinoma
Veliparib, Capecitabine, and Temozolomide in Patients With Advanced, Metastatic, and Recurrent Neuroendocrine Tumor
This phase I trial studies the side effects and best dose of veliparib when given together with capecitabine and temozolomide in treating patients with neuroendocrine tumor that has spread to other places in the body and usually…
- Conditions
- Functional Pancreatic Neuroendocrine Tumor, Malignant Somatostatinoma, Merkel Cell Carcinoma, Metastatic Adrenal Gland Pheochromocytoma
- Trial name
- NCT02831179
- Testing
- Capecitabine, Temozolomide, Veliparib
- Sponsor
- Vanderbilt-Ingram Cancer Center
Retinitis Pigmentosa
Retina Implant Pilot Trial to Evaluate Safety & Efficacy in Blind Patients Having Degenerated Photo-receptors
The outcome of this trial will reveal the possibilities of the retinal implant to improve the situation of patients with hereditary retinal blindness caused by degenerations of the outer retina.
- Trial name
- NCT00515814
- Testing
- Retina implant is surgically placed into subretinal position
- Where
- 2 sites in Germany
- Volunteers
- 15 people
- Sponsor
- Retina Implant AG
Glycogen Storage Disease Type 2, Dystrophia Myotonica
An MRI Study on Muscular Diseases -Pompe Disease and Dystrophia Myotonica-
The aim of the project is to develop new Magnetic Resonance (MR) imaging techniques for better diagnosis and monitoring of patients with muscular disorders.
- Trial name
- NCT02708784
- Where
- 2 sites in Denmark
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- University of Aarhus
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