Clinical trials: Congenital Diseases
Hemophilia A
An Open Label Study to Determine the Safety and Efficacy of Replacement Factor VIII Protein (Known as rFVIIIFc) in Previously Untreated Males With Severe Hemophilia A
The primary objective of the study was to evaluate the safety of rFVIIIFc (BIIB031) in previously untreated participants (PUPs) with severe hemophilia A.
- Trial name
- NCT02234323
- Testing
- rFVIIIFc
- Where
- 71 sites in United States, Australia and 12 more
- Volunteers
- 108 people
- Sponsor
- Bioverativ, a Sanofi company
Brain and Central Nervous System Tumors, Childhood Germ Cell Tumor
ABT-751 in Treating Young Patients With Refractory Solid Tumors
RATIONALE: Drugs used in chemotherapy, such as ABT-751, work in different ways to stop tumor cells from dividing so they stop growing or die.
- Conditions
- Brain and Central Nervous System Tumors, Childhood Germ Cell Tumor, Extragonadal Germ Cell Tumor, Kidney Cancer
- Trial name
- NCT00036959
- Testing
- ABT-751
- Where
- 3 sites in United States
- Volunteers
- 90 people
- Sponsor
- National Institutes of Health Clinical Center (CC)
Hyperuricemia or Gout
Clinical Efficacy Trial of Burdock Root Extract in Patients With Asymptomatic Hyperuricaemia
Multicentre, double-blind, randomized, placebo-controlled trial of the clinical efficacy of burdock root extract in patients with asymptomatic hyperuricemia, with the primary objective to assess the efficacy of the use of burdock…
- Trial name
- NCT07002762
- Testing
- Burdock ( Arctium lappa L.) root extract in a form of a nutritional additive
- Where
- Ukraine
- Volunteers
- 100 people
- Sponsor
- OMNIFARMA LLC
Heart Defects, Congenital
Mixed Venous Oxygen Saturation From Central Venous Line, Venous Side of Heart Lung Machine and the Pulmonary Artery
This study was designed to examine if venous saturations from the central venous line and from the venous side of the heart lung machine are interchangeable or not with mixed venous saturation from the pulmonary artery in…
- Trial name
- NCT02498626
- Testing
- venous saturations
- Where
- Egypt
- Volunteers
- 40 people
- Sponsor
- Assiut University
Von Willebrand Diseases, Glanzmann Thrombasthenia
Videomicroscopy for the Prediction of Bleeding in Constitutional Haemorrhagic Diseases
In Willebrand disease, there is currently no test available to identify non-invasively patients with a high risk of bleeding from angiodysplasias The study propose to use a sublingual capillary bed analysis by video-microscopy, a…
- Trial name
- VIDEO-BLEED (NCT04119908)
- Testing
- Sublingual videomicroscopy, blood sample
- Where
- France
- Volunteers
- 400 people
- Sponsor
- University Hospital, Lille
Cystic Fibrosis
Sit-to-stand Test in Cystic Fibrosis Children and Adolescents
Cystic fibrosis (CF) is a chronic hereditary respiratory disease. Exercise testing is part of CF patients regular assessment. Cardio-Pulmonary Exercise Testing (CPET) is currently considered as the gold standard to assess…
- Trial name
- NCT03069625
- Testing
- Sit-to-stand test, 6-Minute Walk Test
- Where
- 3 sites in France
- Volunteers
- 36 people
- Sponsor
- Groupe Hospitalier du Havre
Acute Myeloid Leukemia, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities
Clofarabine, Cytarabine, and G-CSF in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia
RATIONALE: Drugs used in chemotherapy, such as clofarabine and cytarabine, work in different ways to stop the growth of cancer cells, either by killing the cells or stopping them from dividing.
- Conditions
- Acute Myeloid Leukemia, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Inv(16)(p13;q22), Adult Acute Myeloid Leukemia With t(15;17)(q22;q12)
- Trial name
- NCT00602225
- Testing
- clofarabine, cytarabine, filgrastim
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- University of Washington
Undescended Testis
Comparison Between the Clinical Assessment of the Undescended Testis and Its' Ultrasonographic Size
In addition to the manual preoperative examination, the undescended testicle will be measured ultrasonographically. The compared data will help us to evaluate the routine need for preoperative ultrasonographic evaluation of the…
- Trial name
- NCT02731989
- Where
- Israel
- Volunteers
- 60 people
- Sponsor
- Meir Medical Center
Bronchopulmonary Dysplasia
Safety of Sildenafil in Premature Infants
Describe the safety of sildenafil in premature infants at risk of bronchopulmonary dysplasia and determine preliminary effectiveness and pharmacokinetics (PK) of sildenafil.
- Trial name
- SIL02 (NCT03142568)
- Testing
- Sildenafil
- Where
- 15 sites in United States, Canada
- Volunteers
- 109 people
- Sponsor
- University of North Carolina, Chapel Hill
Atopic Dermatitis
A Study of Lebrikizumab (LY3650150) on Vaccine Response in Adults With Atopic Dermatitis (ADopt-VA)
The reason for this study is to assess the impact of lebrikizumab on vaccine immune response in adult participants with moderate to severe atopic dermatitis (AD).
- Trial name
- NCT04626297
- Testing
- Lebrikizumab
- Where
- 85 sites in United States
- Volunteers
- 254 people
- Sponsor
- Eli Lilly and Company
Hereditary Angioedema, HAE
Open-label, Long-term Safety Study of Avoralstat in Subjects With Hereditary Angioedema
This is an open-label study designed to evaluate the long-term safety of prophylactic avoralstat (500 mg three times daily) when given to approximately 150 patients with hereditary angioedema (HAE) for a duration of up to 72…
- Trial name
- OPuS-4 (NCT02670720)
- Testing
- avoralstat
- Where
- 3 sites in Belgium, France
- Volunteers
- 6 people
- Sponsor
- BioCryst Pharmaceuticals
Congenital Heart Disease, Dysbiosis
The Influence of Probiotics on Metabolome and Heart Rate Variability in Heart Failure of Structure Heart Disease
Poor body weight gain and failure to thrive is a very common condition in patients with congenital heart disease (CHD) with advanced HF and/or cyanosis, which are considered a predictor of morbidity and complicate the prognosis…
- Conditions
- Congenital Heart Disease, Dysbiosis, Malnutrition, Child, Probiotics
- Trial name
- NCT06872320
- Testing
- Lactobacillus Rhamnosus
- Where
- Taiwan
- Volunteers
- 120 people
- Sponsor
- Chang Gung Memorial Hospital
Myotonic Dystrophy 1, Muscular Dystrophies
Myotonic Dystrophy Type 1 Aerobic Exercise Study
Myotonic dystrophy type 1 (DM1) is a genetic disease that primarily targets skeletal muscle resulting in severe weakness and muscle loss.
- Trial name
- DM1ex (NCT04187482)
- Testing
- Exercise training
- Where
- Canada
- Volunteers
- 13 people, healthy volunteers welcome
- Sponsor
- McMaster University
Chronic Liver Disease, Thyroid Disease
The Development of Quantitative Ultrasound Imaging Software Platform
The goal of this observational study is to compare the image differences between conventional ultrasound and artificial intelligence-based ultrasound software in conscious adults.
- Conditions
- Chronic Liver Disease, Thyroid Disease, Benign Breast Disease, Malignant Breast Neoplasm
- Trial name
- NCT05836246
- Where
- South Korea
- Volunteers
- 196 people
- Sponsor
- Seoul National University Bundang Hospital
Dyspnea, Lung Diseases
Effects of Automated Oxygen Titration Alone or With High Flow Nasal Therapy on Dyspnea and Exercise Tolerance
The main purpose of this study is to evaluate the effects of automated oxygen administration (FreeO2 system) alone or with high-flow oxygen on dyspnea and exercise tolerance in people with desaturating chronic lung disease…
- Conditions
- Dyspnea, Lung Diseases, Pulmonary Hypertension, Chronic Obstructive Pulmonary Disease
- Trial name
- NCT05267418
- Testing
- Automated nasal O2 titration with FreeO2
- Where
- Canada
- Volunteers
- 30 people
- Sponsor
- Laval University
Gout
A Study of AC-201 Controlled-Release Tablet (CR Tablet) in Patients With Gout
The study is designed to test the urate-lowering effect, safety, and tolerability of AC-201CR in an initial dosing period, followed by the addition of a ULT to test the efficacy and safety of the combination and prophylaxis of…
- Trial name
- NCT02287818
- Testing
- AC-201, Febuxostat
- Where
- Taiwan
- Volunteers
- 127 people
- Sponsor
- TWi Biotechnology, Inc.
Maple Syrup Urine Disease
Phenylbutyrate Therapy for Maple Syrup Urine Disease
The investigators have learned in past research that the drug phenylbutyrate can decrease the amounts of branched chain amino acids and their byproducts in the bloodstreams of healthy volunteer patients and also patients with…
- Trial name
- MSUD (NCT01529060)
- Testing
- Phenylbutyrate
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Brendan Lee
Cystic Fibrosis, Cystic Fibrosis Gastrointestinal Disease
Phase 2 Combination Study With Escalating Doses of MS1819-SD on Top of a Stable Dose of PPEs
This is a Phase 2 study sponsored by AzurRx SAS and involves testing of a new medication for the compensation of exocrine pancreatic insufficiency (EPI) caused by cystic fibrosis (CF).
- Conditions
- Cystic Fibrosis, Cystic Fibrosis Gastrointestinal Disease, Cystic Fibrosis of Pancreas
- Trial name
- NCT04302662
- Testing
- MS1819-SD
- Where
- 10 sites in Hungary, Turkey (Türkiye)
- Volunteers
- 20 people
- Sponsor
- AzurRx SAS
Prader-Willi Syndrome
Dose Clinical Trial of Guanfacine Extended Release for the Reduction of Aggression and Self-injuries Behavior Associated With Prader-Willi Syndrome
This is a placebo-controlled clinical trial to assess the utility of Guanfacine Extended Release (GXR) in the management of patients with Prader Willi Syndrome (PWS) who have significant aggression or self-injury.
- Trial name
- NCT04066088
- Testing
- Guanfacine extended release (GXR)
- Where
- United States
- Sponsor
- NYU Langone Health
Retinitis Pigmentosa
Autologous Bone Marrow-Derived Stem Cells Transplantation For Retinitis Pigmentosa
The purpose of this study is to evaluate the short-term safety of a single intravitreal injection of autologous bone marrow stem cells in patients with retinitis pigmentosa.
- Trial name
- NCT01068561
- Testing
- intravitreal injection of autologous bone marrow stem cells
- Where
- Brazil
- Volunteers
- 5 people
- Sponsor
- University of Sao Paulo
The first 1,020 results can be paged through. Add a filter or change the sort order to reach the rest.