Clinical trials: Neonatal Disorder, open to volunteers
Transfusion Dependent Beta-Thalassemia
A Study Evaluating the Safety and Efficacy of LentiRed Drug Product in Transfusion-dependent β-Thalassemia [TDT]
This is a single-arm, open label, single-dose study in subjects with transfusion dependent β-thalassaemia. The study will evaluate the safety and efficacy of autologous CD34+ Human Hematopoietic Stem Cells that was transduced…
- Trial name
- NCT05762510
- Testing
- GMCN-508B (LentiRed)
- Where
- China
- Volunteers
- 5 people
- Sponsor
- First Affiliated Hospital of Guangxi Medical University
Macrosomia, Fetal, Gestational Diabetes Mellitus in Pregnancy
New Markers of Glycation to Predict Gestational Diabetes Mellitus and Macrosomia.
Gestational diabetes mellitus (GDM) increases the risk of macrosomia and other adverse pregnancy outcomes. Screening strategies are debated: universal vs.
- Trial name
- GLYCAGEST (NCT06048510)
- Testing
- Pregnant women
- Where
- France
- Volunteers
- 800 people
- Sponsor
- University Hospital, Bordeaux
Bowel Dysfunction, Dysmotility
Comparison of a New Non-invasive Device With the Invasive Gold-standard Investigation to Assess Function of the Large Bowel in Children
The goal of this clinical trial is to learn if the new non-invasive device (Body Surface Colonic Mapping) works to accurately assess colonic movement and function in children.The main questions it aims to answer are: Are Body…
- Conditions
- Bowel Dysfunction, Dysmotility, Hirschsprung Disease, Anorectal Malformations
- Trial name
- NCT07789782
- Testing
- Body Surface Colonic Mapping
- Where
- Australia
- Volunteers
- 50 people
- Sponsor
- Murdoch Childrens Research Institute
Familial Mediterranean Fever, Non-celiac Gluten Sensitivity
Can Gluten/Wheat or Other Foods be Responsible for FMF Attacks
Familial Mediterranean Fever (FMF) is a chronic hereditary autoinflammatory disease caused by mutations in the MEditerranean FeVer (MEFV) gene which codes for pyrin.
- Trial name
- NCT06338891
- Testing
- Main questionnaire, Secondary questionnaire
- Where
- Italy
- Volunteers
- 60 people
- Sponsor
- University of Palermo
Congenital Heart Disease, Heart Diseases
IPSC Repository of Pediatric Cardiovascular Disease
Obtain blood samples for generation and maintenance of induced pluripotent stem cells (iPSCs) and genomic/DNA sequencing for biomedical research that will improve the understanding and treatment of pediatric cardiovascular disease
- Trial name
- NCT05550324
- Testing
- Blood draw
- Where
- United States
- Volunteers
- 300 people, healthy volunteers welcome
- Sponsor
- Mingtao Zhao
Dermatitis, Atopic
A First-in-human Study to Investigate Single Doses of DCY636 in Healthy Volunteers and Multiple Doses in Participants With Moderate to Severe Atopic Dermatitis
The purpose of this first-in-human (FIH) study is to assess the safety and tolerability, pharmacokinetics (PK), immunogenicity (IG) and pharmacodynamics (PD) of DCY636.
- Trial name
- NCT07604324
- Testing
- DCY636
- Where
- Japan
- Volunteers
- 63 people, healthy volunteers welcome
- Sponsor
- Novartis Pharmaceuticals
Brugada Syndrome
UZ Brussel HRMC Registry of Brugada Syndrome
The monocentric UZB registry for Brugada registry is intended to collect all data on patients affected by Brugada syndrome at UZ Brussel hospital (UZB).
- Trial name
- HRMCBrS (NCT05283759)
- Testing
- No intervention because it is a patient registry
- Where
- Belgium
- Volunteers
- 2,000 people
- Sponsor
- Universitair Ziekenhuis Brussel
Vitiligo Vulgaris, Psoriasis
PALLAS Laser for Skin Diseases
The primary objective of this study is to get clinical experiences with the PALLAS laser in the treatment of skin conditions that respond well to UV light (vitiligo, psoriasis, alopecia areata, atopic dermatitis).
- Conditions
- Vitiligo Vulgaris, Psoriasis, Alopecia Areata, Atopic Dermatitis
- Trial name
- PALLAS-UV (NCT07250997)
- Testing
- UVB laser treatment
- Where
- Hungary
- Volunteers
- 50 people
- Sponsor
- Szeged University
Congenital Adrenal Hyperplasia (CAH), Familial Male-Limited Precocious Puberty (FMPP)
Natural History Study of Patients With Excess Androgen
This study will evaluate and gather information in patients with genetic causes of too much androgen (male-like hormone) in order to better understand the effects of too much androgen and describe problems associated with it.
- Trial name
- NCT00250159
- Where
- 2 sites in United States
- Volunteers
- 3,000 people
- Sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Hemophilia A
Low Dose Emicizumab vs Low Dose Factor VIII in Prophylaxis in Hemophilia A Patients
Low dose factor VIII prophylaxis is practiced around the world. Role of standard dose Emicizumab prophylaxis is well established. Emicizumab is an expensive drug.
- Trial name
- AFEEL (NCT06938659)
- Testing
- Emicizumab Injection, Factor VIII (FVIII)
- Where
- Bangladesh
- Volunteers
- 20 people
- Sponsor
- Dhaka Medical College
Congenital Heart Disease
A Study of Hemodynamic Cardiopulmonary Exercise for CHD
The purpose of this study is to compare the difference in the ability to detect hemodynamic abnormalities between invasive hemodynamic assessments performed at rest versus exercise, to assess the correlation between invasive and…
- Trial name
- NCT06566196
- Testing
- Cardiac Cath
- Where
- United States
- Volunteers
- 200 people
- Sponsor
- Mayo Clinic
Metopic Synostosis
Comparing Conservative to Surgical Treatment of Trigonocephaly Craniofacial Surgery in Children With Trigonocephaly: an Observational Cohort Study on Clinical Outcomes, Psychosocial Wellbeing, and Costs
RESEARCH QUESTION Is the effectiveness of conservative policy comparable to that of surgery in children with trigonocephaly, with regard to clinical outcomes and psychosocial functioning, stress for parents, and costs? DESIGN…
- Trial name
- Treat Trigono (NCT06069479)
- Testing
- craniofacial surgery
- Where
- Netherlands
- Volunteers
- 440 people
- Sponsor
- Erasmus Medical Center
Down Syndrome (DS)
The Effect of the HEP Approach in Infants With Down Syndrome
This study evaluates the effect of the HEP (Homeostasis-Enrichment-Plasticity) approach on motor and sensory development in infants with Down syndrome.
- Trial name
- HEP-DS (NCT07163780)
- Testing
- HEP Intervention, Traditional Therapy
- Where
- Turkey (Türkiye)
- Volunteers
- 26 people
- Sponsor
- Izgi Miray Demirbag
Charcot-Marie-Tooth, CMT1A
Studying Nerve Function and Structure in Charcot-Marie-Tooth Disease, Anti-MAG Neuropathy and CIDP
The project aims to perform both conventional nerve-conduction studies and axonal-excitability assessments using the TRONDF protocol in patients with selected forms of Charcot-Marie-Tooth disease, with comparison to individuals…
- Conditions
- Charcot-Marie-Tooth, CMT1A, Chronic Inflammatory Demyelinating Polyneuropathy (CIDP), Polyneuropathy Associated With Anti-MAG Antibodies (Anti-MAG Polyneuropathy)
- Trial name
- CMT-NFS (NCT07461896)
- Where
- Italy
- Volunteers
- 39 people
- Sponsor
- Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta
Atopic Dermatitis, Nature, Human
Sand Play - the Effect of Biodiversity Exposure on Atopic Dermatitis
The prevalence of atopic dermatitis has increased along with urbanization and biodiversity loss. According to biodiversity hypothesis, the main reason is urban lifestyle and reduced contact to microbial diversity.
- Conditions
- Atopic Dermatitis, Nature, Human, Microbial Colonization, Immune System and Related Disorders
- Trial name
- BIWE (NCT06390696)
- Testing
- Microbial intervention
- Where
- 2 sites in Finland
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- Natural Resources Institute Finland
Pregnant Individuals, Aneuploidy
The ADVANCE (Assay Development and Validation for Pre-Natal and Obstetric Conditions) Study is the Largest U.S.-Based Prospective Study Demonstrating a Circulating Fetal Cell (CFC) Based Approach to Non-invasive Fetal Risk Assessment
The goal of the ADVANCE (Assay Development and Validation for Pre-Natal and Obstetric Conditions) study is to compare the concordance of results of a novel non-invasive circulating fetal cell (CFC) assay to the results of…
- Conditions
- Pregnant Individuals, Aneuploidy, Down Syndrome (Trisomy 21), 22q11.2 Deletion Syndrome
- Trial name
- ADVANCE (NCT07643896)
- Where
- 9 sites in United States
- Volunteers
- 1,000 people
- Sponsor
- BillionToOne Inc.
Hernia Surgery, Umbilical Hernia Repair
The PAPYRUS Study: Permanent vs. Absorbable Sutures in PrimarY Repair of Umbilical HerniaS: A Multicentre, Single-blind, Non-inferiority, Randomized Controlled Trial
Umbilical hernias can be found in over a quarter of the population and comprise of 15% of hernia repairs performed in Canada. While mesh repair is commonly used for larger hernias, smaller hernia defects are often closed with…
- Trial name
- PAPYRUS (NCT07156188)
- Testing
- Permanent Suture, Absorbable
- Where
- 5 sites in Canada
- Volunteers
- 914 people
- Sponsor
- University of British Columbia
Choledochal Cyst
Host-Microbe Interactions in Biliary Dilatation
This study is an observational cohort study aimed at continuously enrolling patients with biliary dilatation and healthy controls.
- Trial name
- NCT07152496
- Where
- China
- Volunteers
- 400 people, healthy volunteers welcome
- Sponsor
- Beijing Tsinghua Chang Gung Hospital
Sickle Cell Disease
Blood Collection for Research Related to Certain Diseases Involving Blood Vessels
The collection of human specimens from both patients and healthy volunteers is necessary for the development of laboratory assays to further basic and clinical research studies.
- Trial name
- NCT00047996
- Where
- 2 sites in United States
- Volunteers
- 2,000 people, healthy volunteers welcome
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Hemophilia
Oral Health Status and Oral Health Related Quality of Life in a Group pf Egyptian Hemophilic Children
aim of the study: 1. to assess, investigate, and compare the oral health status and oral health related quality of life of a group of hemophilic Egyptian children before and after an oral health educational program 2.
- Trial name
- NCT07161596
- Testing
- oral health educational program
- Where
- Egypt
- Volunteers
- 70 people
- Sponsor
- Cairo University
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