Clinical trials: Congenital Diseases
Brain and Central Nervous System Tumors, Childhood Germ Cell Tumor
Dasatinib, Ifosfamide, Carboplatin, and Etoposide in Treating Young Patients With Metastatic or Recurrent Malignant Solid Tumors
RATIONALE: Dasatinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Drugs in chemotherapy, such as ifosfamide, carboplatin, and etoposide, work in different ways to stop the growth of…
- Conditions
- Brain and Central Nervous System Tumors, Childhood Germ Cell Tumor, Extragonadal Germ Cell Tumor, Kidney Cancer
- Trial name
- NCT00788125
- Testing
- carboplatin, dasatinib, etoposide phosphate
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- City of Hope Medical Center
Sickle Cell Disease
A Stratified Sickle Event Randomized Trial (ASSERT)
The purpose of this study is to compare the effects of ICA-17043 to placebo with or without hydroxyurea (an oral drug used for treatment of sickle cell disease) in patients with sickle cell disease who have had 2 or more acute…
- Trial name
- ASSERT (NCT00102791)
- Testing
- ICA-17043
- Where
- 49 sites in United States, Brazil and 4 more
- Volunteers
- 297 people
- Sponsor
- Icagen
Sepsis, Neonatal
Comparison Between Split Septum and Mechanical Valve Needleless Connector in Preterm Babies
The goal of this clinical trial study is to compare the effectiveness between split septum and mechanical valve needleless connector in very preterm babies (or under 1500 grams) The main questions it aims to answer are: What is…
- Trial name
- NCT06414174
- Testing
- Split septum needleless connector, Mechanical valve needleless connector
- Where
- Indonesia
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Universitas Indonesia
Thyroid Eye Disease, TED
Psychological Burden of Thyroid Eye Disease
This interview study aims to look in depth at the quality of life of people living with TED and their caregivers, with emphasis on impact on mood, relationships, work/education, and leisure.
- Trial name
- NCT07444190
- Testing
- focus groups and individual interviews as well as filling out questionnaires
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Thomas Jefferson University
Congenital Hemophilia A
Pharmacokinetic, Efficacy, and Safety Study of Recombinant Factor VIII Single Chain (rVIII-SingleChain) in Children With Severe Hemophilia A
This is an international, multicenter, open-label study to assess the efficacy, safety, and pharmacokinetic (PK) profile of rVIII-SingleChain in pediatric patients with severe hemophilia A.
- Trial name
- NCT02093897
- Testing
- rVIII-SingleChain
- Where
- 37 sites in United States, Australia and 17 more
- Volunteers
- 84 people
- Sponsor
- CSL Behring
Carpal Tunnel Syndrome, Median Nerve Entrapment
Hydrodissection in Carpal Tunnel Syndrome
The aim of this study was to compare the effectiveness of perineural hydrodissection methods performed with 5 mL D5W solution and three different injection techniques [ultrasound-guided in-plane, ultrasound-guided out-of-plane…
- Trial name
- KTS (NCT07207460)
- Testing
- Comparison of three different injection techniques using hydrodissection with 5% dextrose in the treatment of CTS
- Where
- Turkey (Türkiye)
- Volunteers
- 74 people
- Sponsor
- Ankara City Hospital Bilkent
HIV Infections
Anti-HIV Drugs for Treating Infants Who Acquired HIV Infection at Birth
The purpose of this study is to compare the effects of anti-HIV drug courses of different lengths in infants who became HIV infected at birth.
- Trial name
- NCT00102960
- Testing
- Abacavir sulfate, Didanosine, Efavirenz
- Volunteers
- 377 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Haemophilia
Quality of Life in Adult Patients With Severe Haemophilia in Turkey
The primary aim of this study is to evaluate the overall quality of life in adult patients with severe haemophilia (an inherited bleeding disorder affecting mainly men) in Turkey and to assess the impact of certain disease and…
- Trial name
- TurkHaemQoL (NCT01623960)
- Volunteers
- 500 people
- Sponsor
- Turkish Society of Hematology
Atopic Dermatitis
Effect of Pimecrolimus Cream on Cathelicidin Levels in Subjects With Eczema
The purpose of this study is to determine the effect of topical pimecrolimus on the immune system by assessing the levels of antimicrobial peptides in the skin of patients with eczema.
- Trial name
- NCT00946478
- Testing
- Pimecrolimus, Vehicle cream
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- University of California, San Diego
Obesity Associated With Defects in Leptin-melanocortin Pathway
Open-Label Extension Study of Setmelanotide
This is an open-label extension study designed to evaluate the long-term safety and tolerability of continued setmelanotide treatment in male and female patients ≥2 years of age who have completed or transitioned from a previous…
- Trial name
- NCT06596135
- Testing
- Setmelanotide
- Where
- 13 sites in United States
- Volunteers
- 28 people
- Sponsor
- Rhythm Pharmaceuticals, Inc.
Glaucoma, Suspect, Optic Nerve Hypoplasia and Abnormalities of the Central Nervous System
A More Engaging Visual Field Test to Increase Use and Reliability in Pediatrics
The majority of young children do not think that visual field (VF) testing of peripheral vision is similar to a game; therefore, it is not surprising that they have difficulty maintaining attention during VF testing and thus the…
- Conditions
- Glaucoma, Suspect, Optic Nerve Hypoplasia and Abnormalities of the Central Nervous System, Congenital Coloboma of the Optic Nerve, Optic Nerve Head Pits, Bilateral Congenital
- Trial name
- NCT02157025
- Testing
- Cartoon video fixation target and cartoon character voice audio instructions during Humphrey perimetry, Usual Care procedures during Humphrey perimetry for children
- Where
- United States
- Volunteers
- 11 people, healthy volunteers welcome
- Sponsor
- Nova Southeastern University
Thalassemia Major
Perceptions of Thalassemia Major in Singapore: An Exploratory Study of Stigma
Background: Thalassemia major (TM) is a chronic disorder that affects a person s ability to produce hemoglobin, resulting in anemia.
- Trial name
- NCT01129076
- Where
- 2 sites in Singapore
- Volunteers
- 30 people
- Sponsor
- National Human Genome Research Institute (NHGRI)
Amelogenesis Imperfecta, Dental Fluoroses
Colorimetric, Ultra-structural and Elemental Comparison of Dental Enamel Defects
The study focuses on the analysis of enamel defects grouping together hypomineralization and hypoplasia. It focuses on 3 very characteristic enamel pathologies that are most often encountered in dental consultations: Molar…
- Conditions
- Amelogenesis Imperfecta, Dental Fluoroses, Hypomineralization Molar Incisor
- Trial name
- COLOEMAIL (NCT04704089)
- Testing
- spectrophotometer, Biological (protein analysis by western blot or mass spectrometry of matrix proteins), structural (electron microscopy) and physico-chemical analyses
- Where
- France
- Volunteers
- 80 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Aortic Stenosis
Role of Elastin Metabolites in Aortic Remodelling in AS
Around 1-2% of people are born with a 'bicuspid' aortic valve, with only two cusps instead of the common 'tri-leaflet' valve. People with this valve develop dysfunction of the valve (narrowing or leakage) at a much earlier age.
- Trial name
- ELASTIC-AS (NCT03883490)
- Testing
- ECG, Blood test and biobanking, Trans-thoracic Echocardiogram
- Where
- United Kingdom
- Volunteers
- 46 people
- Sponsor
- University of Leicester
Hypospadias, Caudal Block
Ultrasound Guided Sacral Erector Spinae Plane Block Versus Ultrasound Guided Caudal Block in Pediatric Undergoing Hypospadias Surgery
Randomized, parallel-group, assessor-blinded clinical trial at Tanta University Hospitals comparing ultrasound-guided sacral erector spinae plane block (sESPB) versus ultrasound-guided caudal epidural block for postoperative…
- Conditions
- Hypospadias, Caudal Block, Erector Spinae Plane Block, Pain, Postoperative
- Trial name
- NCT07099560
- Testing
- Ultrasound-guided sacral erector spinae plane block (sESPB), Ultrasound-guided caudal epidural block
- Where
- Egypt
- Volunteers
- 70 people
- Sponsor
- Tanta University
Hereditary Sensory Neuropathy Type I
Hereditary Sensory Neuropathy Serine Trial
This is a randomised double-blind placebo-controlled trial of L-serine in Hereditary Sensory Neuropathy type 1 (HSN1) due to variants in SPTLC1/2 gene.
- Trial name
- SENSE (NCT06113055)
- Testing
- L-serine
- Where
- United Kingdom
- Volunteers
- 50 people
- Sponsor
- University College, London
Pulmonary Hypertension, Valvular Heart Disease
Evaluating Right Ventricular (RV) Size and Function Using the Upper Valley RV Algorithm and Novel Imaging Modalities
The primary purpose of this study is to evaluate the diagnostic performance of three methods for measuring right ventricular size and function including the Philips Novel RV quantification technologies (RV Heart Model volumetric…
- Conditions
- Pulmonary Hypertension, Valvular Heart Disease, Congenital Heart Disease, Congestive Heart Failure
- Trial name
- UVRV (NCT03935178)
- Testing
- Measuring Right Ventricular Size and Function
- Where
- United States
- Volunteers
- 14 people
- Sponsor
- Dartmouth-Hitchcock Medical Center
Atrial Septal Defect
Cera™ ASD Occluder Post-Market Clinical Follow-Up Study
The objective of this post-market study is to: collect real-word clinical data and confirm the long-time safety and performance of the Lifetech Cera™ ASD Occluder identify previously unknown side-effects
- Trial name
- NCT06849635
- Where
- 6 sites in Poland
- Volunteers
- 139 people
- Sponsor
- Lifetech Scientific (Shenzhen) Co., Ltd.
Ectopia Lentis, Dislocation, Lens
Follow up Study on Visual Prognosis and Complications of Ectopia Lentis Lens
This is a single-centre, observational cohort study with the following objectives: Firstly, the investigators aim to evaluate the vision-related quality of life and mental health status in congenital ectopia lentis patients.
- Trial name
- NCT05654025
- Where
- China
- Volunteers
- 604 people, healthy volunteers welcome
- Sponsor
- Zhongshan Ophthalmic Center, Sun Yat-sen University
Atopic Dermatitis
Clobetasol Topical Oil for Children With Moderate to Severe Atopic Dermatitis
Open-Label Study designed to evaluate the HPA axis suppression potential of Clobetasol Topical Oil and pharmacokinetic safety / systemic exposure to clobetasol when Clobetasol Topical Oil is applied to pediatric subjects with…
- Trial name
- NCT03847389
- Testing
- Clobetasol propionate 0.05% Topical Oil
- Where
- 6 sites in United States
- Volunteers
- 8 people
- Sponsor
- Hill Dermaceuticals, Inc.
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