Clinical trials: Congenital Malformation
Beta-Thalassemia
To Document the Burden of Illness on the Quality of Life and the Impact on Healthcare Utilization in (Beta) β-thalassemia Subjects Who Are Transfusion Dependent (TD) and Non-transfusion Dependent (NTD) Receiving Standard of Care
This is a multi-site, prospective, observational study implemented in β-thalassemia treatment centers from 5 countries (Italy, Turkey, Greece, Lebanon, and Thailand).
- Trial name
- NCT02626689
- Testing
- Quality of Life (QOL) questionnaires, Healthcare Resource Utilization
- Where
- 5 sites in Greece, Italy and 3 more
- Volunteers
- 100 people
- Sponsor
- Celgene
Ventral Hernia, Umbilical Hernia
Prospective Study Of A Size Tailored Approach To Repair Umbilical And Epigastric Primary Ventral Hernias
Primary ventral hernias, such as umbilical and epigastric hernias, are best repaired with abdominal wall reinforcement by mesh implantation.
- Conditions
- Ventral Hernia, Umbilical Hernia, Epigastric Hernia
- Trial name
- SITUP (NCT01639118)
- Where
- Belgium
- Volunteers
- 11 people, healthy volunteers welcome
- Sponsor
- Algemeen Ziekenhuis Maria Middelares
Hypospadias
Hypospadias and Exome: Identification of New Genes for Familial Hypospadias
Hypospadias is the second most common malformation of the male genitalia and consists in a congenital hypoplasia of its ventral face.
- Trial name
- NCT02495090
- Testing
- Exome sequencing
- Where
- France
- Volunteers
- 60 people
- Sponsor
- University Hospital, Montpellier
Behçet's Syndrome
A Phase 3 Randomized, Double-blind Study to Evaluate the Efficacy and Safety of Apremilast (CC-10004) in Subjects With Active Behçet's Disease
The main objective of this study is to evaluate the efficacy and safety of apremilast in the treatment of oral ulcers in adults with active Behçet's disease (BD).
- Trial name
- NCT02307513
- Testing
- Apremilast
- Where
- 63 sites in United States, France and 8 more
- Volunteers
- 207 people
- Sponsor
- Amgen
Retinitis Pigmentosa
Safety and Tolerability of Intravitreal Administration of VG901 in Patients With Retinitis Pigmentosa Due to Mutations in the CNGA1 Gene
The goal of this phase 1 clinical trial is to learn about the safety and efficacy of a gene therapy, VG901, in patients with a rare disorder of the eye called Retinitis Pigmentosa.
- Trial name
- NCT06291935
- Testing
- VG901
- Where
- Germany
- Volunteers
- 6 people
- Sponsor
- VeonGen Therapeutics GmbH
Psuedomas Infection, Cystic Fibrosis
The Role of Bacteria and Genetic Variations in Cystic Fibrosis
This study will examine 1) the role of hereditary factors in cystic fibrosis; i.e., the relationship of the disease to specific gene variations, and 2) the role of bacterial products involved in lung infections substances…
- Trial name
- NCT00043225
- Where
- 4 sites in United States
- Volunteers
- 76 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Central Core Disease, Multi-Minicore Disease
The Natural History and Muscle Fatigability of Patients With Congenital Myopathies.
Core myopathies (CCD/MmD), nemaline myopathies (NEM) and centronuclear myopathies (CNM) are three types of rare congenital myopathies.
- Conditions
- Central Core Disease, Multi-Minicore Disease, Nemaline Myopathy, Centronuclear Myopathy
- Trial name
- READYCOM (NCT06157268)
- Testing
- Natural history and non therapeutical therapy
- Where
- 2 sites in Netherlands
- Volunteers
- 100 people
- Sponsor
- Radboud University Medical Center
Hemochromatosis
Treatment of Hemochromatosis
This study will evaluate the effectiveness of a test called MCV in guiding phlebotomy (blood drawing) therapy in patients with hemochromatosis an inherited disorder that causes too much iron to be absorbed by the intestine.
- Trial name
- NCT00007150
- Testing
- Phlebotomy
- Where
- United States
- Volunteers
- 622 people
- Sponsor
- National Institutes of Health Clinical Center (CC)
Fetal Alcohol Spectrum Disorders, Hyperactivity Disorder
Monitoring and Evaluation of a Cognitive Behavioral Therapy Program Focused on Attention and Emotion Regulation for Children With Fetal Alcohol Spectrum Disorder
This is a multi-center, prospective, randomized, open-label, controlled trial with two parallel arms and blinded endpoint assessment.
- Conditions
- Fetal Alcohol Spectrum Disorders, Hyperactivity Disorder, Attention Deficit
- Trial name
- SEPAGE (NCT05273918)
- Testing
- cognitivo-comportmental therapy, body mediation
- Where
- 3 sites in France, Reunion
- Sponsor
- Centre Hospitalier Universitaire de la Réunion
Retinitis Pigmentosa
Electro-acupuncture and Transcorneal Electrical Stimulation (TES) for Retinitis Pigmentosa
Measures of vision in RP patients receiving promising therapy using transcorneal electrical stimulation.
- Trial name
- NCT02086890
- Testing
- Electro-acupuncture, Laser Acupuncture, Transcorneal Electrical Stimulation
- Where
- United States
- Volunteers
- 21 people
- Sponsor
- Nova Southeastern University
Granulomatous and Lymphocytic Interstitial Lung Disease
Treatment of Granulomatous and Lymphocytic Interstitial Lung Disease in Patients With Common Variable Immunodeficiency
This phase II study will assess the effect of a treatment combination of Rituximab and azathioprine in patients with Granulomatous and Lymphocytic Interstitial Lung Disease (GLILD) compared to placebo, based on change in lung…
- Trial name
- NCT02789397
- Testing
- Rituximab (RTX) and Azathioprine (AZA)
- Sponsor
- Medical College of Wisconsin
Hypercholesterolemia, Hypercholesterolemia, Familial
PET Imaging of Inflammation and Lipid Lowering Study
While 18F-fluorodeoxyglucose (FDG) positron emission tomography (PET) imaging has been used as an early marker of drug efficacy in numerous clinical cardiovascular drug trials, as a glucose analog, its signal in the vasculature…
- Conditions
- Hypercholesterolemia, Hypercholesterolemia, Familial, Atherosclerosis, Carotid Artery Plaque
- Trial name
- PIILL (NCT04073797)
- Testing
- Inclisiran, 68Ga-DOTATATE PET-MRI, Colchicine
- Where
- United Kingdom
- Volunteers
- 60 people
- Sponsor
- University of Cambridge
Congenital Heart Disease
Outcome of Patients With Congenital Heart Disease
Advanced surgical and medical healthcare systems have resulted in an increased prevalence of children and adults with congenital heart disease in Western countries. These patients often necessitate non-cardiac interventions.
- Trial name
- OUTCARDIOP (NCT03913520)
- Testing
- Outcome research
- Where
- Belgium
- Volunteers
- 150 people
- Sponsor
- Cliniques universitaires Saint-Luc- Université Catholique de Louvain
Hemophilia A
Characterization of Laboratory Response to DDAVP in Adult Hemophilia A Carriers
The purpose of this study is to determine how female hemophilia A carriers respond to a medication called DDAVP (Desmopressin).
- Trial name
- NCT02506023
- Testing
- Desmopressin
- Where
- 2 sites in United States
- Volunteers
- 2 people
- Sponsor
- Emory University
Achondroplasia
Radiological Analysis on Patients With Achondroplasia Disorder
Achondroplasia is a genetic disorder characterized by disproportionate short stature. It affects about 1 in 2500 live births in the world.
- Trial name
- Achondroplasia (NCT04184817)
- Testing
- Collection of clinical data registered in Easily software
- Where
- France
- Volunteers
- 60 people
- Sponsor
- Hospices Civils de Lyon
Cystic Fibrosis, Pulmonary Hypertension
Impact of Availability of Showerpatch for Patients With Intravenous Catheters
Venous catheters are necessary for the treatment of many patients. Showering with a venous catheter is often prohibited due to the infection risk when the insertion site becomes wet.
- Conditions
- Cystic Fibrosis, Pulmonary Hypertension, Neoplasms
- Trial name
- ISIC (NCT02324868)
- Testing
- Shower patch IV catheter protection, Conventional IV catheter protection
- Where
- Belgium
- Volunteers
- 18 people
- Sponsor
- Universitaire Ziekenhuizen KU Leuven
Neonatal Infection, Neonatal SEPSIS
Reduction of Intravenous Antibiotics In Neonates
Randomized controlled open-label non-inferiority trial comparing complete intravenous antibiotic treatment with a short iv. course followed by oral antibiotics in neonates (0-28 days) with probable bacterial infection.
- Trial name
- RAIN (NCT03247920)
- Testing
- Amoxicillin Clavulanate, Antibiotics
- Where
- 18 sites in Netherlands
- Volunteers
- 510 people
- Sponsor
- Franciscus Gasthuis
Down Syndrome (DS)
Awareness of Families With Down Syndrome Children About the Role of Physical Therapy in Egypt
This study explores how much families with children who have Down Syndrome in Egypt know about the benefits and role of physical therapy.
- Trial name
- DS-AWARENESS (NCT06993324)
- Where
- Egypt
- Volunteers
- 240 people
- Sponsor
- Cairo University
Fetal Growth Restriction
Sildenafil Citrate for Treatment of Growth-restricted Fetuses
The aim of this study is to assess the effect of Sildenafil citrate therapy on neonatal outcomes in women with fetal growth restriction.
- Trial name
- NCT03177824
- Testing
- Sildenafil Citrate 25Mg Tab
- Where
- Egypt
- Volunteers
- 60 people
- Sponsor
- Ain Shams University
Elevated LDL-Cholesterol (LDL-C)
Trial to Evaluate Safety and Tolerability of ALN-PCS02 in Subjects With Elevated LDL-Cholesterol (LDL-C)
The purpose of this study is to determine the safety, tolerability, pharmacokinetics and pharmacodynamics of a single dose of ALN-PCS02 in subjects with Elevated LDL-Cholesterol (LDL-C).
- Trial name
- NCT01437059
- Testing
- ALN-PCS02, Sterile Normal Saline (0.9% NaCl)
- Where
- 2 sites in United Kingdom
- Volunteers
- 32 people, healthy volunteers welcome
- Sponsor
- Alnylam Pharmaceuticals
The first 1,020 results can be paged through. Add a filter or change the sort order to reach the rest.