Clinical trials: Congenital Diseases
Ductus Arteriosus, Patent, Very Low Birth Weight Infants
Comparing Ibuprofen And Indomethacin For The Treatment Of The Patent Ductus Arteriosus in Very Premature Babies
The purpose of the study is to determine the safety and efficacy of ibuprofen, compared with indomethacin, in the treatment for the closure of the patent ductus arteriosus in premature babies born under 29 weeks gestation
- Trial name
- NCT00470743
- Testing
- Ibuprofen, Indomethacin
- Where
- Singapore
- Sponsor
- KK Women's and Children's Hospital
Maxillary Deficiency
Paranasal Sinus Volumes of Maxillary Deficiency Patients Requiring Le Fort Osteotomy
The present study aims to compare ethmoid, sphenoid and maxillary sinus volumes of patients with maxillary deficiency requiring Le Fort osteotomy with healthy patients, by employing computed tomography imaging.
- Trial name
- NCT04637568
- Testing
- Computerized tomography
- Where
- Turkey (Türkiye)
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- Ankara University
Accelerated Phase Chronic Myelogenous Leukemia, Acute Undifferentiated Leukemia
Sunitinib in Treating Patients With Idiopathic Myelofibrosis
This phase II trial is studying how well sunitinib works in treating patients with idiopathic myelofibrosis. Sunitinib may stop the growth of abnormal cells by blocking some of the enzymes needed for cell growth and by blocking…
- Conditions
- Accelerated Phase Chronic Myelogenous Leukemia, Acute Undifferentiated Leukemia, Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Myeloid Leukemia in Remission
- Trial name
- NCT00387426
- Testing
- sunitinib malate
- Where
- United States
- Volunteers
- 14 people
- Sponsor
- National Cancer Institute (NCI)
Stargardt Disease
Safety and Effects of a Single Intravitreal Injection of vMCO-010 Optogenetic Therapy in Subjects With Stargardt Disease
The purpose of the study is to evaluate the safety and effects of a single intravitreal injection of virally-carried Multi-Characteristic Opsin (vMCO-010) in Subjects with Stargardt Disease
- Trial name
- STARLIGHT (NCT05417126)
- Testing
- Gene Therapy-vMCO-010
- Where
- 2 sites in United States
- Volunteers
- 6 people
- Sponsor
- Nanoscope Therapeutics Inc.
Li-Fraumeni Syndrome, Hereditary Cancer Syndromes
Biomarker Monitoring in TP53 Mutation Carriers
Purpose This study is an 'N-of-one' observational study focusing on individuals with a hereditary predisposition to cancer due to a genetic mutation in the TP53 gene.
- Conditions
- Li-Fraumeni Syndrome, Hereditary Cancer Syndromes, TP53 Gene Germline Mutation Carrier
- Trial name
- NCT02289326
- Where
- United States
- Volunteers
- 6 people, healthy volunteers welcome
- Sponsor
- Scripps Health
Atopic Dermatitis, Food Allergy
Skin Barrier Abnormalities and Oxidative Stress Response
This is a prospective, single-site controlled observational study designed to comprehensively determine whether children and adults with atopic dermatitis (AD) and food allergy (FA) have skin abnormalities which distinguish them…
- Trial name
- NCT04606615
- Where
- United States
- Volunteers
- 160 people, healthy volunteers welcome
- Sponsor
- National Jewish Health
Primary Ciliary Dyskinesia, Primary Immune Deficiency
Defining the Genetic Etiology of Suppurative Lung Disease in Children and Adults
The investigators will utilize a systematic approach for the diagnostic evaluation of patients to identify characteristics which may distinguish between Primary Immunodeficiency (PID) disorders versus Primary Ciliary Dyskinesia…
- Conditions
- Primary Ciliary Dyskinesia, Primary Immune Deficiency, Kartagener Syndrome
- Trial name
- NCT04702243
- Testing
- Genetic Testing for PCD or PID, Unaffected Family Member Genetic Testing
- Where
- 8 sites in United States, Canada
- Volunteers
- 436 people
- Sponsor
- University of North Carolina, Chapel Hill
Down Syndrome
A 4-Week Safety Study of Oral ELND005 in Young Adults With Down Syndrome Without Dementia
This is a prospective, randomized, double-blind, placebo-controlled, parallel-group, three-arm, multicenter study of the safety and PK of ELND005 administered orally for 4 weeks.
- Trial name
- DS201 (NCT01791725)
- Testing
- ELND005
- Where
- 3 sites in United States
- Volunteers
- 23 people
- Sponsor
- OPKO Health, Inc.
Heart Disease, Congenital, Single Ventricle
Single Ventricle Outcome
The objective of this research proposal is to perform an ongoing single institution prospective clinical trial examining the overall clinical outcome of children with single ventricle physiology.
- Conditions
- Heart Disease, Congenital, Single Ventricle, Hypoplastic Left Heart Syndrome
- Trial name
- NCT00308217
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Emory University
Cystic Fibrosis
Microbiome Acquisition and Progression of Inflammation and Airway Disease in Infants and Children With Cystic Fibrosis
Cystic Fibrosis (CF) is a fatal, recessive genetic disorder characterized by progressive inflammation and lung damage. It is unclear whether current treatment strategies, which focus on detection and eradication of pathogenic…
- Trial name
- NCT01661491
- Where
- United States
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Yale University
Milk Intolerance
Low-grade Inflammation and Gut Symptoms From A2 and Hydrolyzed A1 Milk
The aim of this study is to reveal how different forms of milk casein, lactose and moderate breakdown of proteins affect the symptoms that may arise from milk and markers of inflammation in volunteers receiving symptoms from…
- Trial name
- NCT05305391
- Testing
- Dietary intervention
- Where
- Finland
- Volunteers
- 37 people, healthy volunteers welcome
- Sponsor
- University of Turku
Hemoglobinopathies, Thalassemia
Hemoglobinopathy Nursing Program and Pediatric Nursing Students
Hemoglobinopathies are the most common life threatening, monogenic disorders in the world. The most common causes of hemoglobinopathies are sickle cell disease and thalassemia.
- Conditions
- Hemoglobinopathies, Thalassemia, Sickle Cell Disease, Nurse's Role
- Trial name
- NCT05389891
- Testing
- Hemoglobinopathy Nursing Program
- Where
- Egypt
- Volunteers
- 108 people, healthy volunteers welcome
- Sponsor
- Beni-Suef University
Cystic Fibrosis
Effects of an Antioxidant-Enriched Multivitamin Supplement on Inflammation and Oxidative Stress in Cystic Fibrosis
The purpose of this study will be to evaluate the effects of a modified formulation of AquADEKs (AquADEKs-2) on markers of inflammation, antioxidant levels and oxidative stress.
- Trial name
- AquADEKs-2 (NCT01859390)
- Testing
- AquADEKs-2, control multivitamin
- Where
- 15 sites in United States
- Volunteers
- 73 people
- Sponsor
- University of Colorado, Denver
Non Malignant Diseases, Immunodeficiencies
Reduced Intensity Conditioning (RIC) Regimen for Patients With Non-malignant Disorders
This Phase II pilot study evaluates the safety and effectiveness of reduced-intensity conditioning (RIC) regimens prior to allogeneic hematopoietic stem cell transplantation (HSCT) in pediatric and young adult patients with…
- Conditions
- Non Malignant Diseases, Immunodeficiencies, Hemoglobinopathies
- Trial name
- RIC (NCT01050855)
- Testing
- Reduced-Intensity Conditioning
- Where
- United States
- Volunteers
- 56 people
- Sponsor
- Children's Hospital of Philadelphia
Congenital Bleeding Disorder, Haemophilia A
Project to Update the Study of Congenital Haemophilia in Spain
This study is conducted in Europe. The aim of the study is to update the knowledge about the prevalence and severity of congenital haemophilia (A and B) in Spain.
- Conditions
- Congenital Bleeding Disorder, Haemophilia A, Haemophilia B
- Trial name
- PUCHS (NCT01959555)
- Testing
- No treatment given
- Where
- Spain
- Volunteers
- 1,157 people
- Sponsor
- Novo Nordisk A/S
Cystic Fibrosis
Canadian Observation Trial in CF Patients Undergoing Treatment With Ivacaftor
Specific aims: 1. To elucidate the biological mechanism that leads to pulmonary and nutritional improvement in CF patients following treatment with ivacaftor using advanced techniques to assess changes of the pulmonary and…
- Trial name
- G551D (NCT03390985)
- Volunteers
- 18 people
- Sponsor
- Tanja Gonska
Hemophilia B
Post Marketing Surveillance To Observe Safety and Efficacy Of BeneFIX In Patients With Hemophilia B
To provide safety and effectiveness information of BeneFIX during the post-marketing period as required by Korea FDA regulations, to identify any potential drug related treatment factors in Korean population including: 1) Unknown…
- Trial name
- NCT00484185
- Testing
- BeneFIX (coagulation factor IX (recombinant))
- Where
- South Korea
- Volunteers
- 183 people
- Sponsor
- Pfizer
Sickle Cell Disease
Risk Factors for Allo-immunization in Sickle Cell Disease
Sickle cell patients have a high prevalence of alloimmunization. This high rate of alloimmunization can be partially explained by the existence of an antigenic difference between the predominantly Caucasian donor population and…
- Trial name
- NCT03401125
- Testing
- Medical file data collection
- Where
- 2 sites in Belgium
- Sponsor
- Hanane EL KENZ
Chronic Myelomonocytic Leukemia, Chronic Myelomonocytic Leukemia-1
Azacitidine With or Without Lenalidomide or Vorinostat in Treating Patients With Higher-Risk Myelodysplastic Syndromes or Chronic Myelomonocytic Leukemia
This randomized phase II/III trial studies how well azacitidine works with or without lenalidomide or vorinostat in treating patients with higher-risk myelodysplastic syndromes or chronic myelomonocytic leukemia.
- Conditions
- Chronic Myelomonocytic Leukemia, Chronic Myelomonocytic Leukemia-1, Chronic Myelomonocytic Leukemia-2, Myelodysplastic Syndrome
- Trial name
- NCT01522976
- Testing
- Azacitidine, Laboratory Biomarker Analysis, Lenalidomide
- Where
- 294 sites in United States, Canada
- Volunteers
- 282 people
- Sponsor
- National Cancer Institute (NCI)
MELAS, LHON
A Dose-escalating Clinical Trial With KH176
Mitochondrial Diseases are rare progressive, multi-system, often early fatal disorders affecting both children and adults. KH176 is a novel chemical entity currently under development for the treatment of inherited mitochondrial…
- Conditions
- MELAS, LHON, Leigh Syndrome, Mitochondrial Disease
- Trial name
- NCT02544217
- Testing
- KH176
- Where
- Belgium
- Volunteers
- 32 people, healthy volunteers welcome
- Sponsor
- Khondrion BV
The first 1,020 results can be paged through. Add a filter or change the sort order to reach the rest.