Clinical trials: Congenital Diseases
Lactose Intolerance
The Effect of Probiotics on Lactose Intolerance
Lactose intolerance (LI), also known as lactose malabsorption is the most common type of carbohydrate malabsorption. It is associated with the inability to digest lactose into its constituents, glucose and galactose, due to low…
- Trial name
- PLI (NCT01593800)
- Testing
- Bio-25
- Volunteers
- 60 people
- Sponsor
- Rabin Medical Center
Cystic Fibrosis, Portal Hypertension
Portal Hypertension and Systemic Endothelial Function
Cystic fibrosis can affect organs other than the lungs. Liver disease affects about 30% of patients: its main manifestation is the development of portal hypertension (PHT).
- Trial name
- ENDOTH-MUCO (NCT02850692)
- Testing
- measure of endothelial function, Blood sample, Hepatic elastography
- Where
- France
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Hopital Foch
Gout Arthritis
The Relationship Between Cold Water Immersion and the Progression in Gout Arthritis
Gouty arthritis is a type of autoinflammatory arthritis that generates higher levels of pain with only minimum movement in the joint.
- Trial name
- NCT04587544
- Testing
- Cold Water Immersion
- Where
- Indonesia
- Volunteers
- 76 people
- Sponsor
- Taipei Medical University
Cancer
Ketamine Hydrochloride and Best Pain Management in Treating Cancer Patients With Neuropathic Pain
RATIONALE: Ketamine hydrochloride may lessen neuropathic pain in patients with cancer. It is not yet known whether ketamine hydrochloride given together with the best pain management is more effective than a placebo given…
- Trial name
- NCT01316744
- Testing
- ketamine hydrochloride, pharmacogenomic studies, questionnaire administration
- Where
- 3 sites in United Kingdom
- Volunteers
- 214 people
- Sponsor
- University of Glasgow
Molar-Incisor Hypomineralization (MIH)
Nano Silver Fluoride and Photobiomodulation Therapy for Hypersensitivity and Pain in MIH-Affected Molars
Molar-incisor hypomineralization (MIH) is a developmental condition affecting primarily the first permanent molars and may be associated with post-eruptive enamel breakdown, hypersensitivity, and difficulties in achieving…
- Trial name
- NCT07826130
- Testing
- Nano Silver Fluoride, Photobiomodulation Therapy, Pre-appointment analgesics
- Volunteers
- 45 people
- Sponsor
- Ain Shams University
Neurodevelopmental Disorders, Congenital Cardiomyopathy
KIDSHEART AND BRAIN : Early EEG Surgery Congenital Heart Disease Predict Onset of Neurodevelopmental Disorders
Congenital cardiopathy are frequent malformations (1/100 birth). The progress of surgery permit a survival rate at the adult age of more than 90%.
- Trial name
- KHB (NCT05658965)
- Where
- France
- Volunteers
- 50 people
- Sponsor
- University Hospital, Lille
Down Syndrome
Effect of the Sensory Integration Approach on Balance and Motor Coordination in Children With Down Syndrome
Down syndrome can be characterized by global mental and physical dysfunction or isolated gait, cognition, growth, or sensory disturbances.
- Trial name
- NCT05583565
- Testing
- sensory integration approach, traditional physical therapy program
- Where
- Egypt
- Volunteers
- 30 people
- Sponsor
- Cairo University
Mucopolysaccharidosis I, Hurler Syndrome
Study of Aldurazyme® Replacement Therapy in Patients With Mucopolysaccharidosis I (MPS I) Disease
This is a multi-center, open label, study conducted to evaluate the safety of laronidase administered by intravenous drip infusion in Japanese patients with MPS I disease.
- Conditions
- Mucopolysaccharidosis I, Hurler Syndrome, Hurler-Scheie Syndrome, Scheie Syndrome
- Trial name
- NCT00258011
- Testing
- Aldurazyme (Recombinant Human Alpha-L-Iduronidase)
- Where
- 2 sites in Japan
- Volunteers
- 3 people
- Sponsor
- Genzyme, a Sanofi Company
Non Malignant Disorders, Immunodeficiencies
Reduced Intensity Conditioning for Umbilical Cord Blood Transplant in Pediatric Patients With Non-Malignant Disorders
The primary objective is to determine the feasibility of attaining acceptable rates of donor cell engraftment (>25% donor chimerism at 180 days) following reduced intensity conditioning (RIC) regimens in pediatric patients < 21…
- Conditions
- Non Malignant Disorders, Immunodeficiencies, Congenital Marrow Failures, Hemoglobinopathies
- Trial name
- NCT00744692
- Testing
- Unrelated Umbilical Cord Blood Transplant, Reduced Intensity Conditioning
- Where
- United States
- Volunteers
- 22 people
- Sponsor
- Duke University
Craniosynostosis, Bleeding
Tranexamic Acid for Craniofacial Surgery
This is a randomized, blinded, prospective study that will investigate the potential benefit of tranexamic acid to reduce the intraoperative bleeding and blood transfusions in pediatric patients undergoing craniofacial surgeries.
- Trial name
- NCT00722436
- Testing
- Tranexamic acid, saline
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- University of Pittsburgh
Congenital Diaphragmatic Hernia
Early FETO for Severe Congenital Diaphragmatic Hernia
Congenital diaphragmatic hernia (CDH) is associated high mortality and morbidity, mainly in those cases with severe forms where there are extremely reduced lung volumes, liver herniation and decreased abnormal pulmonary…
- Trial name
- NCT01731509
- Testing
- Fetal endoscopic tracheal occlusion
- Where
- Brazil
- Volunteers
- 70 people
- Sponsor
- University of Sao Paulo General Hospital
Children Born After Assisted Reproductive Technologies, Health Status
Health Status of Children Born After Assisted Reproductive Technologies
The goal of this observational study is to study the features of psychophysical development and the morbidity patterns of children born after assisted pregnancy, and to identify the connection with the health status of mothers…
- Conditions
- Children Born After Assisted Reproductive Technologies, Health Status, Child Development
- Trial name
- NCT06094998
- Testing
- hemogram examination
- Where
- Kazakhstan
- Volunteers
- 252 people, healthy volunteers welcome
- Sponsor
- Kazakhstan's Medical University "KSPH"
Acute Mountain Sickness, High Altitude
CO2 Supplement for Treatment of Acute Mountain Sickness
It was reported that up to 76% people who rapidly ascend to the altitude higher than 4500 m without sufficient acclimatization will have AMS which often develops 6-8 hours after reaching at high altitude.
- Conditions
- Acute Mountain Sickness, High Altitude, Inhalation; Gas
- Trial name
- NCT06393998
- Testing
- Breathing with CO2 during daytime and night at high altitude, Sleep and doing exercise test under inhalation of air
- Where
- China
- Volunteers
- 46 people, healthy volunteers welcome
- Sponsor
- State Key Laboratory of Respiratory Disease
Hemophilia A
Efficacy and Safety Study of a Recombinant Protein-Free Manufactured Factor VIII (rAHF-PFM) in Previously Untreated Hemophilia A Patients
The purpose of this study is to evaluate whether Antihemophilic factor, recombinant, manufactured protein-free (rAHF-PFM) is effective and safe in the treatment of hemophilia A patients who have not been treated with factor VIII…
- Trial name
- NCT00157157
- Testing
- Recombinant Antihemophilic Factor Manufactured and Formulated without Added Human or Animal Proteins (rAHF-PFM)
- Where
- 35 sites in United States, Austria and 8 more
- Volunteers
- 66 people
- Sponsor
- Baxalta now part of Shire
Plagiocephaly, Cranial Defect
Associations of Cranial Outcomes and Parental Expectations and Satisfaction
This study aims to investigate the association between objective cranial morphological changes and parental expectations and satisfaction in infants undergoing treatment with a cranial remolding orthosis (CRO).
- Conditions
- Plagiocephaly, Cranial Defect, Orthosis, Expectancy
- Trial name
- NCT07356401
- Testing
- Cranial orthosis
- Where
- Turkey (Türkiye)
- Volunteers
- 10 people
- Sponsor
- Gulhane School of Medicine
Methylmalonic Acidemia, Propionic Acidemia
"The MaP Study": Mapping the Patient Journey in MMA and PA
Longitudinal, exploratory, natural history study of patients with MMA due to mut deficiency and PA to characterize the changes in blood disease biomarkers over time and the frequency and severity of clinical events related to…
- Trial name
- NCT03484767
- Where
- 17 sites in United States, France and 2 more
- Volunteers
- 97 people
- Sponsor
- ModernaTX, Inc.
Severe Congenital Diaphragmatic Hernia
FETO With Long Tail Balloon for a Treatment of Severe CDH
Congenital diaphragmatic hernia (CDH) has an incidence of 1:2200 to 1:4000 newborns. The survival rate depends on the extent of the lung hypoplasia and pulmonary hypertension.
- Trial name
- LongTailFETO (NCT03431792)
- Testing
- Long Tail Balloon
- Where
- Germany
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Martin-Luther-Universität Halle-Wittenberg
Cystic Fibrosis, Colorectal Cancer
Colorectal Cancer Screening in Cystic Fibrosis
This multi-center study will compare multi-target DNA and quantitative FIT stool-based testing to colonoscopy in individuals with Cystic Fibrosis (CF) undergoing colon cancer screening with colonoscopy.
- Conditions
- Cystic Fibrosis, Colorectal Cancer, Adenoma
- Trial name
- NICE-CF (NCT05362344)
- Testing
- Stool tests
- Where
- 26 sites in United States
- Volunteers
- 350 people
- Sponsor
- University of Washington, the Collaborative Health Studies Coordinating Center
Bronchomalacia, Asthma
Mapping Sound Propagation Through the Human Lung for Better Diagnosis
This study investigates the propagation of sound from a source in the chest to the chest wall. The methodology of the study will be to place a sound source at a known location in the chest and measure the acoustic response on the…
- Conditions
- Bronchomalacia, Asthma, Lung Neoplasms, Lung Diseases, Interstitial
- Trial name
- NCT03043898
- Testing
- Lung sound transmission measurement, Lung sound recording
- Where
- United Kingdom
- Volunteers
- 62 people, healthy volunteers welcome
- Sponsor
- Maximilian Nussbaumer
Atopic Dermatitis
A Phase I Study of HY209 Gel in Healthy Male Volunteers for Atopic Dermatitis
A randomized, double-blind, placebo-controlled single and multiple dosing, dose escalation phase I clinical trial to investigate the safety/tolerability and pharmacokinetics of HY209 gel after transdermal administration in…
- Trial name
- Shaperon (NCT03492398)
- Testing
- HY209
- Where
- South Korea
- Volunteers
- 56 people, healthy volunteers welcome
- Sponsor
- Shaperon
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