Clinical trials: Congenital Malformation
Esophageal Atresia With Tracheo-esophageal Fistula, Congenital Diaphragmatic Hernia
Open or Keyhole Surgery Through the Chest for Newborn Babies: Effect on Blood Gases
This is a pilot randomised controlled trial comparing open versus thoracoscopic surgery for repair of oesophageal atresia with tracheo-oesophageal fistula or congenital diaphragmatic hernia in neonates.
- Trial name
- CO2 (NCT01467245)
- Testing
- Hypercapnia during thoracoscopy, Open surgery
- Where
- United Kingdom
- Volunteers
- 20 people
- Sponsor
- Great Ormond Street Hospital for Children NHS Foundation Trust
Hemophilia A
Study to Learn More About the Safety of Drug Jivi Over a Long Period of Time in Previously Treated Patients With Hemophilia A (Bleeding Disorder Resulting From a Lack of FVIII) Who Are Receiving Jivi Regularly at Their Treating Doctors to Prevent Bleeding
In this observational study researchers want to learn more about the safety of drug Jivi over a long period of time. Jivi (generic name: Damoctocog alfa pegol) is an approved blood clotting Factor VIII (FVIII) medication for the…
- Trial name
- HA-SAFE (NCT04461639)
- Testing
- Damoctocog alfa pegol (Jivi, BAY94-9027)
- Where
- 6 sites in Austria, Germany and 4 more
- Volunteers
- 62 people
- Sponsor
- Bayer
Duchenne Muscular Dystrophy (DMD)
Virtual Reality and Whole Body Vibration on Muscle Strength and Balance in Children With DMD
we measure balance and quadriceps strength in Duchenne muscular dystrophy children after applying virtual reality and whole body vibration
- Trial name
- NCT07338812
- Testing
- virtual reality, whole body vibration
- Where
- 2 sites in Egypt
- Volunteers
- 50 people
- Sponsor
- Cairo University
Hemophilia A
Long-term Study Evaluating Joint Health in People With Haemophilia A Receiving Real-world Prophylactic Treatment With Efanesoctocog Alfa
The rationale for this study is to further understand and describe the long-term prophylactic effectiveness of efanesoctocog alfa in preventing joint bleeds in a real-life setting.
- Trial name
- NCT06940830
- Testing
- Efanesoctocog alfa
- Where
- 31 sites in Croatia, Czechia and 6 more
- Volunteers
- 252 people
- Sponsor
- Swedish Orphan Biovitrum
LGMD2B, Miyoshi Myopathy
Deflazacort in Dysferlinopathies
The present study is designed to assess the natural history in a one year pre-phase of the trial and evaluate therapeutic efficacy and side effects of deflazacort in LGMD2B/MM patients in a placebo-controlled trial.
- Conditions
- LGMD2B, Miyoshi Myopathy, Dysferlinopathy
- Trial name
- NCT00527228
- Testing
- deflazacort
- Where
- Germany
- Volunteers
- 25 people
- Sponsor
- Ludwig-Maximilians - University of Munich
Tourette Syndrome
Safety and Efficacy Study of NBI-98854 in Adults With Tourette Syndrome
Phase 2, double-blind, placebo-controlled study to assess the safety and efficacy of NBI-98854 administered once daily (qd) for a total of 8 weeks of treatment.
- Trial name
- NCT02581865
- Testing
- NBI-98854
- Where
- 30 sites in United States
- Volunteers
- 124 people
- Sponsor
- Neurocrine Biosciences
Inborn Errors of Metabolism
Growth and Development of Children With Inborn Errors of Metabolism in Assiut Governorate
Assessment of growth and development of children with inborn errors of metabolism in Assiut Governorate
- Trial name
- NCT06654765
- Where
- Egypt
- Volunteers
- 201 people
- Sponsor
- Assiut University
Hemophilia A
Biomarkers Serum Collection Methodology Pilot Study
The purpose of this study is to compare the standard collection of whole blood in tiger top tubes for serum collection versus a collection method of added FVIII to whole blood samples in the hemophilia population for routine…
- Trial name
- NCT01318707
- Where
- United States
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Georgetown University
Gout
The the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of XNW3009 in Health Subject
XNW3009 is a small molecule hURAT1 inhibitor developed independently by Sinovent Pty Ltd., and is intended to treat gout-related hyperuricemia.
- Trial name
- NCT04040907
- Testing
- XNW3009
- Where
- Australia
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Evopoint Biosciences Inc.
Jaundice, Neonatal
Research of Diagnostic Value for BMJ Infants
Breast milk jaundice (BMJ) is the main cause of neonatal hyperbilirubinemia. Excessive serum unconjugated bilirubin level will not only cause the interruption or early termination of breastfeeding, but also cause kernicterus.
- Trial name
- NCT04527536
- Testing
- Neonatal hyperbilirubinemia
- Volunteers
- 300 people
- Sponsor
- Shanghai First Maternity and Infant Hospital
Hereditary Angioedema (HAE)
FIRAZYR General Drug Use-Results Survey (Japan)
The objectives of this survey are to collect data to report the safety and efficacy of Firazyr (Icatibant acetate) in the post-marketing phase in participants diagnosed with Hereditary Angioedema (HAE).
- Trial name
- NCT04057131
- Testing
- Firazyr
- Where
- 18 sites in Japan
- Volunteers
- 179 people
- Sponsor
- Shire
Skin Condition, Cleft Lip and Palate
Online Psychosocial Support for Young People With a Visible Difference: A Randomised Control Study
A visible difference can have a profound impact in a society with a massive emphasis on appearance and "looks". A vulnerable group is adolescents with a condition affecting their appearance as a result of injuries (burns…
- Conditions
- Skin Condition, Cleft Lip and Palate, Burns, Other Conditions Leading to a Visible Difference
- Trial name
- UNGFaceIT (NCT03165331)
- Testing
- Ung Face IT
- Where
- Norway
- Volunteers
- 130 people, healthy volunteers welcome
- Sponsor
- Oslo University Hospital
Antepartum Hemorrhage, Postpartum Hemorrhage
Strengthening Maternal Neonatal and Child Health Services in a Rural District of Pakistan
The Maternal Neonatal and Child health indicators in District Dadu of Pakistan portrays a dismal pictures and after the floods of 2010-2011 the health infrastructure of this district was badly affected.
- Conditions
- Antepartum Hemorrhage, Postpartum Hemorrhage, Anemia, Eclampsia
- Trial name
- SRC (NCT02797353)
- Testing
- Maternal Neonatal and Child Health Interventions
- Volunteers
- 15,000 people, healthy volunteers welcome
- Sponsor
- Aga Khan University
Familial Adenomatous Polyposis
Safety Study for the Use of Rapamycin in Children With Familial Adenomatous Polyposis
The hypothesize of this research is that rapamycin is effective and well-tolerated in teenagers with familial adenomatous polyposis (FAP).
- Trial name
- RAPA-4-PAF (NCT06308445)
- Testing
- Rapamycin
- Where
- 4 sites in France
- Volunteers
- 25 people
- Sponsor
- University Hospital, Toulouse
Hereditary Tyrosinemia, Type I
Bio Equivalency 20 Mgm Orfadin and 20 Mgm of Nitisonine
he purpose of this study is to determine whether Nitisinone 10 mg Tablets (Test Product are bioequivalent to the reference product Orfadin 10 mg
- Trial name
- Orfadin (NCT04113772)
- Testing
- Nitisinone, Orfadin
- Where
- India
- Volunteers
- 4 people
- Sponsor
- Sutphin Drugs
Beta Thalassemia Major
Effect of Spirulina Compared to Amlodipine on Cardiac Iron Overload in Children With Beta Thalassemia
the aim of this study is to evaluate the effect of Spirulina compared to Amlodipine on cardiac iron overload and cardiac functions in multi-transfused children with beta thalassemia major
- Trial name
- NCT02671695
- Testing
- Spirulina, Amlodipine
- Where
- Egypt
- Volunteers
- 40 people
- Sponsor
- Tanta University
Cytomegalovirus Infections, HIV Infections
A Randomized, Double-Blind Study of the Efficacy and Safety of Oral Ganciclovir for the Prevention of CMV Disease in People Infected With the Human Immunodeficiency Virus
To evaluate the efficacy of oral ganciclovir in preventing progression to cytomegalovirus (CMV) disease (e.g., CMV retinitis, gastrointestinal CMV disease) in CMV-infected people with HIV infection and CD4 lymphocyte counts <=…
- Trial name
- NCT00002095
- Testing
- Ganciclovir
- Where
- 19 sites in United States
- Volunteers
- 700 people
- Sponsor
- Hoffmann-La Roche
Asthma, COPD
Blood Collection From Individuals With Lung Disease for Genetic Studies
This is a research study where researchers are collecting blood to evaluate the genetic characteristics of individuals with chronic lung diseases, including asthma, chronic obstructive pulmonary disease (COPD), interstitial lung…
- Conditions
- Asthma, COPD, Interstitial Lung Disease, Cystic Fibrosis
- Trial name
- NCT00362739
- Where
- United States
- Volunteers
- 3,087 people, healthy volunteers welcome
- Sponsor
- Weill Medical College of Cornell University
Cystic Fibrosis
Oral Glucose Tolerance Testing Using Candy for Cystic Fibrosis
The study aims to determine if a popular dextrose candy alternative yields a similar glycemic curve compared to the standard oral Dextrose solution used in the Oral Glucose Tolerance Test.
- Trial name
- NCT04579939
- Testing
- Candy Glucose Tolerance Test
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Mayo Clinic
Polycystic Kidney Diseases
Everolimus on CKD Progression in ADPKD Patients
The study will evaluate whether the administration of everolimus (1.5 mg/day) can slow down the progression of CKD in ADPKD patients.
- Trial name
- NCT01009957
- Testing
- Everolimus
- Where
- 7 sites in Italy
- Volunteers
- 71 people
- Sponsor
- A. Manzoni Hospital
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