Clinical trials: Congenital Malformation
Congenital Disorders
Hypoxemia in Infants After Palliative Surgery
Congenital heart disease is a major cause of morbidity and mortality in infants. Many cardiac defects require surgical palliation or repair in the newborn period.
- Trial name
- NCT00327977
- Where
- United States
- Volunteers
- 375 people
- Sponsor
- Children's Healthcare of Atlanta
Basal Cell Carcinoma, Locally Advanced Basal Cell Carcinoma
Oral Hedgehog Inhibitors in the Treatment of Basal Cell Carcinoma in the Netherlands: a Prospective Registration Study
Background: Oral hedgehog inhibitors vismodegib and sonidegib have been used for the treatment of locally advanced (laBCC), metastatic basal cell carcinoma (mBCC) and in basal cell nevus syndrome (BCNS) patients.
- Conditions
- Basal Cell Carcinoma, Locally Advanced Basal Cell Carcinoma, Metastatic Basal Cell Carcinoma, Gorlin Syndrome
- Trial name
- NCT05463757
- Testing
- Vismodegib, Sonidegib
- Where
- 8 sites in Netherlands
- Volunteers
- 80 people
- Sponsor
- Maastricht University Medical Center
Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Del(5q)
Decitabine in Treating Patients With Previously Untreated Acute Myeloid Leukemia
This phase II trial is studying how well decitabine works in treating patients with previously untreated acute myeloid leukemia.
- Conditions
- Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Del(5q), Adult Acute Myeloid Leukemia With Inv(16)(p13;q22), Adult Acute Myeloid Leukemia With t(15;17)(q22;q12)
- Trial name
- NCT00492401
- Testing
- decitabine, laboratory biomarker analysis, pharmacological study
- Where
- United States
- Volunteers
- 55 people
- Sponsor
- National Cancer Institute (NCI)
Nevus Flammeus
Treatment of Nevus Flammeus With Alexandrite Laser
Nevus flammeus is a congenital vascular malformation. Nevus flammeus is traditionally treated with pulsed dye lasers (PDL); however, around 20 percent of patients are poor responders and do not get satisfactory results from…
- Trial name
- NCT01968681
- Testing
- Alexandrite laser
- Where
- Denmark
- Volunteers
- 19 people
- Sponsor
- Bispebjerg Hospital
Telangiectasia, Hereditary Hemorrhagic, Epistaxis
North American Study of Epistaxis in Hereditary Hemorrhagic Telangiectasia (HHT)
The purpose of the NOSE Study is to carefully examine the efficacy and safety of 3 nasal sprays (bevacizumab, estriol, and tranexamic acid), compared to placebo, for the treatment of HHT related nosebleeds.
- Trial name
- NOSE (NCT01408030)
- Testing
- Sterile saline, Bevacizumab, Estriol
- Where
- 6 sites in United States
- Volunteers
- 123 people
- Sponsor
- James Gossage
Congenital Diaphragmatic Hernia
Fetal Endoscopic Tracheal Occlusion (FETO) Trial for Congenital Diaphragmatic Hernia (CDH)
This is a single site pilot trial to assess the feasibility and safety of treating severe CDH with Fetal Endoscopic Tracheal Occlusion with the Goldballoon Detachable Balloon (GOLDBAL2) along with the Delivery Microcatheter…
- Trial name
- FETO (NCT04052828)
- Testing
- FETO with Goldballoon Detachable Balloon (GOLDBAL2) along with the Delivery Microcatheter (BALTACCI-BDPE100)
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- Dr Erin Perrone
Dermatitis, Atopic, Eczema, Atopic
Safety and Efficacy of Quadriderme® in the Treatment of Impetiginous Eczema (Study P05134AM4)
This is a parallel-group, randomized, active-controlled, double-blind, Phase 4 trial comparing three creams in the treatment of impetiginous eczema: Arm A: QUADRIDERME® cream (betamethasone diproprionate, clotrimazole, and…
- Conditions
- Dermatitis, Atopic, Eczema, Atopic, Skin Diseases, Eczematous
- Trial name
- NCT00671528
- Testing
- Cream (betamethasone diproprionate, clotrimazole, and gentamicin sulfate), Cream (betamethasone diproprionate and gentamicin), Cream (betamethasone diproprionate)
- Volunteers
- 3 people
- Sponsor
- Organon and Co
Spinocerebellar Ataxia Type 3
Efficacy Of Oral Trehalose In Spinocerebellar Ataxia 3
This study evaluates the effectiveness of oral trehalose in alleviating the neuropathological and motor behaviour deficits among patients with SCA3.
- Trial name
- NCT04399265
- Testing
- Trehalose, Maltose
- Where
- Malaysia
- Volunteers
- 20 people
- Sponsor
- National University of Malaysia
Deformity, Genu Valgus
Stapling Versus 8plate for Correction of Genu Valgus
In children with excessive knock knees it may be necessary to use guided growth (small surgical procedure) so the child outgrows the condition before maturity.
- Trial name
- NCT01528553
- Testing
- 8plate, Staples (Richards, Smith & Nephew)
- Where
- Denmark
- Volunteers
- 20 people
- Sponsor
- University of Aarhus
Pseudophakic Bullous Keratopathy, Fuchs' Endothelial Dystrophy
The OPTIMISE Study
Rationale: The cornea is the most transplanted tissue in the Netherlands, with more than 1,500 procedures performed each year. A minimally invasive technique called Descemet Membrane Endothelial Keratoplasty (DMEK) has become the…
- Conditions
- Pseudophakic Bullous Keratopathy, Fuchs' Endothelial Dystrophy, Intraocular Pressure
- Trial name
- NCT05716945
- Testing
- Fluorometholone 0.1% Oph Susp, Dexamethasone 0.1% ophthalmic suspension, Stop steroids
- Where
- 8 sites in Netherlands
- Volunteers
- 342 people
- Sponsor
- Maastricht University Medical Center
Inflammatory Bowel Diseases, Anorectal Malformations
StoMakker Mobile Application
The goal of this clinical trial is to investigate if access to the StoMakker application will significantly improve health-related quality of life in children receiving surgery resulting in an ileostomy, colostomy or continent…
- Conditions
- Inflammatory Bowel Diseases, Anorectal Malformations, Spina Bifida, Ostomy
- Trial name
- StoMakker (NCT06065995)
- Testing
- StoMakker
- Where
- Netherlands
- Volunteers
- 30 people
- Sponsor
- Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)
Cystic Fibrosis (CF)
Proof of Principle Study for an Efficacy Trial of Linaclotide for Cystic Fibrosis
Linaclotide is a medicine used to treat constipation and irritable bowel syndrome with constipation (IBS-C). It works by acting on the surface of the gut lining, where it increases the movement of salt and water into the bowel.
- Trial name
- MODEL (NCT07569419)
- Testing
- Linaclotide 290 micrograms
- Where
- United Kingdom
- Volunteers
- 26 people, healthy volunteers welcome
- Sponsor
- University of Nottingham
Myotonic Dystrophy Type 1
Children's Health Research Institute(CHRI), Stanford Lucile Packard Children Hospital (LPCH) Protocol on Myotonic Dystrophy
Study to focus on the defining and managing the neuropsychological abnormalities of myotonic dystrophy and to find out if the neuropsychological abnormalities have any correlation with changes seen on Magnetic Resonance Imaging.
- Trial name
- CHRI (NCT02269865)
- Where
- United States
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Stanford University
Leber Congenital Amaurosis (LCA), Retinitis Pigmentosa (RP)
Natural History Study in Inherited Retinal Disease Subjects Caused by Mutations in RPE65 or LRAT
To evaluate the natural history of visual function in subjects with IRD phenotypically diagnosed as Leber congenital amaurosis (LCA) or retinitis pigmentosa (RP) caused by RPE65 or LRAT gene mutations.
- Trial name
- NCT02575430
- Testing
- No treatment: retrospective chart review
- Where
- 9 sites in United States, Canada and 5 more
- Volunteers
- 59 people
- Sponsor
- QLT Inc.
Hypercholesterolemia, Familial, Lipid Metabolism, Inborn Errors
Investigational Drug in Patients With Hypercholesterolemia or in Patients With Sitosterolemia (0653-026)(COMPLETED)
The purpose of this study is to provide an investigational drug to patients with a specific type of hypercholesterolemia (high cholesterol) or sitosterolemia (unusually high absorption of non-cholesterol sterols) in a treatment…
- Trial name
- NCT00092833
- Testing
- Comparator: ezetimibe
- Volunteers
- 49 people
- Sponsor
- Organon and Co
Hyperbilirubinemia, Neonatal
The Efficacy of Oral Probiotics on Neonatal Hyperbilirubinemia
This trial will be carried out in two stages in the sick baby room of the Children's Hospital of China Medical University (CMUH).
- Trial name
- NCT03876678
- Testing
- Lactobacillus salivarius AP-32, Bifidobacterium animalis subspecies lactis CP-9
- Where
- Taiwan
- Volunteers
- 180 people
- Sponsor
- China Medical University Hospital
Sickle Cell Anemia, Sickle Cell Disease
Optimizing Hydroxyurea Therapy in Children With SCA In Malaria Endemic Areas
The Novel use Of Hydroxyurea in an African Region with Malaria (NOHARM) study is the first placebo-controlled randomized clinical trial of hydroxyurea treatment in a malaria endemic region.
- Conditions
- Sickle Cell Anemia, Sickle Cell Disease, Malaria
- Trial name
- NOHARM-MTD (NCT03128515)
- Testing
- Hydroxyurea
- Where
- Uganda
- Volunteers
- 187 people
- Sponsor
- Indiana University
Arteriovenous Fistula Occlusion
O-GlcNac Modified Proteomics Study of the Maturation of Hemodialysis Arteriovenous
We divided into three parts to carry out. Firstly, evidence the pre-existing fibrosis in vein of AVF failure patients. investigation of expression, the role, and the mechanism by which the identified O-GlcNAac proteins promote…
- Trial name
- NCT05832476
- Testing
- No intervention
- Where
- Taiwan
- Volunteers
- 126 people
- Sponsor
- National Taiwan University Hospital
Lynch Syndrome, Endometrial Neoplasms
Universal Screening for Lynch Syndrome in Women With Endometrial and Non-Serous Ovarian Cancer
This study will maximize identification of women with Lynch Syndrome using an enhanced screening strategy to identify those at risk.
- Conditions
- Lynch Syndrome, Endometrial Neoplasms, Ovarian Neoplasms, Colorectal Neoplasms
- Trial name
- LS2 (NCT02494791)
- Testing
- Questionnaire, Educational Material
- Where
- Canada
- Volunteers
- 886 people, healthy volunteers welcome
- Sponsor
- University Health Network, Toronto
Atopic Dermatitis
Climatotherapy At The Dead Sea For Atopic Dermatitis
The purpose of this study is to evaluate the improvement in severity of Atopic Dermatitis and quality of life after 4 weeks of treatment in the Dead Sea.
- Trial name
- NCT01307150
- Where
- Israel
- Volunteers
- 50 people
- Sponsor
- Hadassah Medical Organization
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