Clinical trials: Congenital Diseases
Congenital Heart Disease, Single-ventricle
Ventripoint Single Ventricle Study
This study will validate a coordinate-based 3-dimensional echocardiographic technique for the assessment of cardiac size and function in children and young adults with functional single ventricles.
- Trial name
- NCT05262907
- Testing
- Transthoracic Echocardiogram or Cardiac MRI, Ventripoint Echocardiogram
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- Duke University
Leukemia
Biomarkers in Samples of Bone Marrow From Patients With Acute Myeloid Leukemia
RATIONALE: Studying samples of bone marrow from patients with cancer in the laboratory may help doctors learn more about changes that occur in DNA and identify biomarkers related to cancer.
- Trial name
- NCT01290107
- Testing
- DNA analysis, gene rearrangement analysis, microarray analysis
- Volunteers
- 9 people
- Sponsor
- Children's Oncology Group
Bicuspid Aortic Valve
Genetic Test Based Risk Prediction of Early Calcific Aortic Valve Disease in Patients With Bicuspid Aortic Valve
This study is to elucidate the impact of germline mutations and clonal hematopoiesis (CHIP) on the progression of early aortic valve calcification in patients with bicuspid aortic valves.
- Trial name
- NCT06153407
- Where
- South Korea
- Volunteers
- 240 people
- Sponsor
- Yonsei University
Multiple Endocrine Neoplasia Type 1, Pancreatic Neuroendocrine Tumor
EUS-FNI for MEN1-related Pancreatic Neuroendocrine Tumors
The present study aims to evaluate the feasibility, safety and efficacy of EUS-FNI for MEN1-related pNETs
- Trial name
- NCT05554744
- Testing
- EUS-guided fine-needle injection
- Where
- China
- Volunteers
- 20 people
- Sponsor
- Guangxi Medical University
Premature Retinopathy, Visual Impairment
Evaluation of Visual - Motor Development in Children With Retinopathy of Prematurity
The aim of this study was to investigate the eye structure, visual impairment, visual motor development and motor coordination of children between 2-6 ages that had applied to diod laser photocoagulation and intravitreal anti …
- Conditions
- Premature Retinopathy, Visual Impairment, Motor Delay
- Trial name
- NCT03865134
- Testing
- Snellen Test, Autorefractometer, Titmus Stereo Fly Test
- Where
- Turkey (Türkiye)
- Volunteers
- 69 people, healthy volunteers welcome
- Sponsor
- Istanbul Medipol University Hospital
Mucopolysaccharidosis Type III A, Sanfilippo Disease Type A
Long-term Follow-up of Sanfilippo Type A Patients Treated by Intracerebral SAF-301 Gene Therapy
P2-SAF-301 is an open-label interventional study without administration of investigational product, evaluating the long-term safety and tolerability of intracerebral SAF-301 previously administered to 4 patients with Sanfilippo…
- Trial name
- NCT02053064
- Testing
- SAF-301
- Where
- France
- Volunteers
- 4 people
- Sponsor
- LYSOGENE
Infantile Colic
Preliminary Efficacy and Safety Study of Oral Nepadutant in Infant With Colic Not Responding to Conventional Treatment
This phase IIa study is designed as a multi-centre, single country, randomised, double-blind, placebo controlled study in three parallel groups, with the aim to evaluate the efficacy and safety of Nepadutant given at two oral…
- Trial name
- nocry-a (NCT01309009)
- Testing
- Nepadutant oral solution
- Sponsor
- Menarini Group
Hemophilia B
RIXUBIS Drug Use-Result Survey (Japan)
The purpose of this survey is to understand the following items observed in the actual clinical use of RIXUBIS. 1. Unexpected adverse drug reactions 2. Occurrence of adverse drug reactions in the actual clinical use 3.
- Trial name
- NCT02937831
- Testing
- RIXUBIS
- Where
- 5 sites in Japan
- Volunteers
- 6 people
- Sponsor
- Baxalta now part of Shire
Hailey-Hailey Disease, Darier Disease
Evaluation of the Improvement of Quality of Life of Patients Suffering From Hailey Hailey or Darier Disease After Injections of Botulism Toxin Into Large Folds.
Hailey Hailey and Darier disease are rare genetic dermatoses. Mutations of 2 genes (ATP2C1 or ATP2A2 respectively) are responsible for the diseases.
- Trial name
- ToxHD (NCT02782702)
- Testing
- Botulism Toxin Treatment
- Where
- France
- Volunteers
- 30 people
- Sponsor
- University Hospital, Toulouse
Cystic Fibrosis
Nasal Potential Difference (NPD) for the Diagnosis of Cystic Fibrosis
The purpose of this research study is to provide a novel method for the diagnosis of Cystic Fibrosis (CF). This protocol is designed to test the ability of the cells to regulate the movement of salt and water in people with…
- Trial name
- NCT00589745
- Testing
- Nasal potential difference measurement
- Where
- United States
- Volunteers
- 27 people
- Sponsor
- Children's Hospital of Philadelphia
Epidermolysis Bullosa
MT2015-20: Biochemical Correction of Severe EB by Allo HSCT and Serial Donor MSCs
This is a single-institution, phase II study to determine the event-free survival at 1 year post allogeneic transplant and serial mesenchymal stem cell (MSC) infusions from a related donor (HLA identical, mismatched or…
- Trial name
- NCT02582775
- Testing
- Thymoglobulin, Cyclophosphamide, Fludarabine
- Where
- United States
- Volunteers
- 17 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Type 2 Diabetes
Influence of Endothelial Function on Central and Peripheral Causes Of Exercise Impairment in Type 2 Diabetes
This study will evaluate the effects of impaired blood flow regulation on exercise. It will also determine whether the effects are more important in the heart or in the skeletal muscle tissue during exercise.
- Trial name
- InsITE (NCT00786019)
- Testing
- Exercise program, Ascorbic Acid (Vitamin C)
- Where
- United States
- Volunteers
- 47 people, healthy volunteers welcome
- Sponsor
- University of Colorado, Denver
Neonatal Hyperbilirubinemia
Modified Diagnosis and Treatment of Neonatal Hemolysis With ETCOc in sNH
The goal of this clinical trial is to learn if modified diagnosis and treatment (MDT) of neonatal hemolysis (a common cause to newborn jaundice) incorporated with ETCOc measurement (a non-invasive measurement of exhaled gas)…
- Trial name
- MDTinsNH (NCT06832800)
- Testing
- MDT, control (current management)
- Where
- China
- Volunteers
- 250 people
- Sponsor
- Women's Hospital School Of Medicine Zhejiang University
Down Syndrome
Proactive Speech and Language Intervention for Infants With Down Syndrome
Children with Down syndrome (DS) face life-long struggles with verbal communication. Babble and speech sound development is delayed, and speech can be difficult to understand.
- Trial name
- BBC-DS (NCT06450509)
- Testing
- Babble Boot Camp
- Where
- United States
- Volunteers
- 60 people
- Sponsor
- Arizona State University
Sickle Cell Disease, Umbilical Cord Blood
US Phase I Study of ECT-001-CB in Patients With Sickle-Cell Disease
The application of experimental hematopoietic cell transplantation (HCT) therapy in sickle-cell disease (SCD) must strike a balance between the underlying disease severity and the possibility of a direct benefit of the treatment…
- Conditions
- Sickle Cell Disease, Umbilical Cord Blood, Hematopoietic Cell Proliferation
- Trial name
- NCT04594031
- Testing
- ECT-001-CB (UM171-Expanded Cord Blood Transplant)
- Where
- 2 sites in United States
- Sponsor
- ExCellThera inc.
Down Syndrome
Role of Sleep Apnea in the Neuropsychological Function in Down Syndrome People
This is the first study about Neuropsychological function and OSAS in Taiwan. Although the correlation between OSAS and neuropsychological abnormalities had been reported, it is worth to understand more about the detailed domains…
- Trial name
- NCT03267602
- Testing
- CPAP
- Volunteers
- 45 people
- Sponsor
- National Taiwan University Hospital
Lung Injury, Preterm Birth
Positive End-Expiratory Pressure (PEEP) Levels During Resuscitation of Preterm Infants at Birth (The POLAR Trial).
Premature babies often need help immediately after birth to open their lungs to air, start breathing and keep their hearts beating.
- Trial name
- POLAR (NCT04372953)
- Testing
- Positive End-Expiratory Pressure (PEEP)
- Where
- 27 sites in United States, Australia and 5 more
- Volunteers
- 906 people
- Sponsor
- Murdoch Childrens Research Institute
Recurrent Solid Tumor, Recurrent Ewing Sarcoma
Study of Onivyde With Talazoparib or Temozolomide in Children With Recurrent Solid Tumors and Ewing Sarcoma
The phase I portion of this study is designed for children or adolescents and young adults (AYA) with a diagnosis of a solid tumor that has recurred (come back after treatment) or is refractory (never completely went away).
- Conditions
- Recurrent Solid Tumor, Recurrent Ewing Sarcoma, Recurrent Hepatoblastoma, Recurrent Malignant Germ Cell Tumor
- Trial name
- NCT04901702
- Testing
- Onivyde, Talazoparib, Temozolomide
- Where
- 10 sites in United States, Canada
- Volunteers
- 90 people
- Sponsor
- St. Jude Children's Research Hospital
Urea Cycle Disorders
To Evaluate the Safety of Long-term Use of HPN-100 in the Management of Urea Cycle Disorders (UCDs)
This was an open-label, long-term safety study of HPN-100 (RAVICTI; glycerol phenylbutyrate) in participants with a urea cycle disorder (UCD) who completed the safety extensions of HPN-100-005 (NCT00947544; HPN-100-005SE)…
- Trial name
- NCT01257737
- Testing
- HPN-100
- Where
- 16 sites in United States, Canada
- Volunteers
- 88 people
- Sponsor
- Amgen
Missing Teeth
A Proof of Concept for All-ceramic Zirconia Resin Bonded Bridges for Canine, Premolar and Short Span Posterior Teeth
Trial Design - Objectives and Purpose The aim of this study is to perform a proof of concept for the successful retention and outcome measures of zirconia RBBs for the replacement of missing canine or posterior teeth (6-8mm span)…
- Trial name
- NCT02793037
- Testing
- Zirconia resin bonded bridge
- Where
- China
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- The University of Hong Kong
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