This is a multicenter, double-blind, randomized study to compare the safety, effectiveness and immunogenicity (antibodies against drug) of two biological drugs administered intravenously - vedolizumab (a drug not registered in pediatric patients) and infliximab (used as standard therapy), in pediatric patients with UC aged 6-18 years.
Before enrollment to the study, informed consent must be obtained from the participant's legal guardian and, additionally, from the participant aged >16 years before any procedures are performed.

开始日期2024-10-01

申办/合作机构

Instytut Pomnik Centrum Zdrowia Dziecka

NCT05534672 / Recruiting临床3期IIT

Multicenter, Randomized, Double-blind, Placebo Controlled Study to Assess the Efficacy and Safety of Rapamycin in Drug Resistant Epilepsy Associated With TSC

The purpose of the RaRE-TS study is to determine safety, tolerability and efficacy of rapamycin versus placebo in a drug resistant epilepsy associated with tuberous sclerosis complex (TSC).

开始日期2023-01-23

申办/合作机构

Instytut Pomnik Centrum Zdrowia Dziecka

NCT05909800 / Recruiting临床2期IIT

Randomized, Double-blind, Multicenter, Parallel-group, Placebo-controlled Study to Evaluate the Efficacy of Phenofibrate Treatment on the Functions of Beta Cells in Children and Adolescents With Newly Diagnosed of Type 1 Diabetes.

The goal of this clinical trial is to evaluate of the effect of phenofibrate on the functions of beta cells in children with new diagnosis of type 1 diabetes. The main question it aims to answer is: whether phenofibrate may prolong residual beta-cell function therefore own insulin secretion. Participants will be asked to take a phenofibrate or identically appearing placebo (a neutral substance), orally, once daily, for 12 months with no knowledge what is administred to them. They will be invited for follow-up visits including blood tests every 3 months. Researchers will be monitoring the two groups for the safety of the phenofibrate, and at the trial end they compare the residual insulin secretion results in two groups.

开始日期2022-09-29

申办/合作机构

Warszawski Uniwersytet Medyczny

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1,756

项与 Instytut Pomnik Centrum Zdrowia Dziecka 相关的文献（医药）

2024-12-31·Future Rare Diseases

Plain language summary of a study looking at the long-term benefits of enzyme replacement therapy in children and teenagers with Gaucher disease type 3

作者: El-Beshlawy, Amal ; Mistry, Pramod K ; Belmatoug, Nadia ; Tylki-Szymanska, Anna

What is this study about? Gaucher disease is a rare genetic condition. There are three types of Gaucher disease: type 1, type 2, and type 3 (GD3). Symptoms of GD3 include problems with the brain and spinal cord, bones, blood, enlarged liver and spleen, and slow growth. Symptoms have a great impact on the quality of life of people with GD3 and are known to cause loss of life in childhood. In Gaucher disease, people have two non-working copies of a gene called GBA, which tells the body how to make an enzyme called beta-glucosidase (which breaks down excess fats called sphingolipids). In Gaucher disease, people do not make enough beta-glucosidase enzyme, meaning sphingolipids build up inside cells, affecting many organs and systems of the body. Enzyme replacement therapy (ERT) is a treatment for Gaucher disease. Previous studies looking at ERT showed that treatment can greatly improve most symptoms and quality of life in people with Gaucher disease. How ERT may help people with GD3 is only available in small studies. What was the aim of this study? Researchers looked at long-term changes in the blood, spleen, liver, and growth in children and teenagers living with GD3 and how long they survived once they had started an ERT called imiglucerase. Researchers used data from a large global database called the International Collaborative Gaucher Group (ICGG) Gaucher Registry. The aim was to investigate if imiglucerase could improve long-term symptoms and prevent early loss of life. What were the results of this study? Low red blood cell counts (causing a condition called anaemia), low platelet (specialised blood cells that stop bleeding and bruising) counts, enlarged liver and spleen, and slow growth were the most common symptoms in people with GD3 before treatment. People with GD3 who had received imiglucerase had improved symptoms after 5 years of treatment. They also had a greater chance of living longer, with 92% of people alive after 5 years of treatment. These results show that the ERT imiglucerase helps to improve blood, spleen, liver, and growth symptoms, and most importantly that it is a life-prolonging treatment.

2024-12-01·Journal of Pathology Informatics

Quantitative digital pathology enables automated and quantitative assessment of inflammatory activity in patients with autoimmune hepatitis

Article

作者: Pronicki, Maciej ; Shumbayawonda, Elizabeth ; Chełstowska, Sylwia ; Fleming, Ken ; Socha, Piotr ; Langford, Caitlin ; Aljabar, Paul ; Roy, Abhishek ; Janowski, Kamil ; Grajkowska, Wieslawa ; Jurkiewicz, Elzbieta ; Banerjee, Rajarshi ; Wozniak, Malgorzata

BackgroundChronic liver disease diagnoses depend on liver biopsy histopathological assessment. However, due to the limitations associated with biopsy, there is growing interest in the use of quantitative digital pathology to support pathologists. We evaluated the performance of computational algorithms in the assessment of hepatic inflammation in an autoimmune hepatitis in which inflammation is a major component.MethodsWhole-slide digital image analysis was used to quantitatively characterize the area of tissue covered by inflammation [Inflammation Density (ID)] and number of inflammatory foci per unit area [Focal Density (FD)] on tissue obtained from 50 patients with autoimmune hepatitis undergoing routine liver biopsy. Correlations between digital pathology outputs and traditional categorical histology scores, biochemical, and imaging markers were assessed. The ability of ID and FD to stratify between low-moderate (both portal and lobular inflammation ≤1) and moderate-severe disease activity was estimated using the area under the receiver operating characteristic curve (AUC).ResultsID and FD scores increased significantly and linearly with both portal and lobular inflammation grading. Both ID and FD correlated moderately-to-strongly and significantly with histology (portal and lobular inflammation; 0.36≤R≤0.69) and biochemical markers (ALT, AST, GGT, IgG, and gamma globulins; 0.43≤R≤0.57). ID (AUC: 0.85) and FD (AUC: 0.79) had good performance for stratifying between low-moderate and moderate-severe inflammation.ConclusionQuantitative assessment of liver biopsy using quantitative digital pathology metrics correlates well with traditional pathology scores and key biochemical markers. Whole-slide quantification of disease can support stratification and identification of patients with more advanced inflammatory disease activity.

2024-12-01·Pediatric Nephrology

Ambulatory isolated diastolic hypertension and risk of left ventricular hypertrophy in children with primary and secondary hypertension

Article

作者: Myette, Robert L ; Litwin, Mieczysław ; Obrycki, Łukasz ; Seeman, Tomáš ; Feber, Janusz ; Šuláková, Terezie

AbstractBackgroundPediatric blood pressure (BP) assessment and management is increasingly important. Uncontrolled systolic and combined hypertension leads to hypertension-mediated organ damage. The impact of isolated diastolic hypertension is less clearly understood.MethodsWe analyzed the prevalence of ambulatory isolated diastolic hypertension (IDH) in primary (PH) and secondary (SH) hypertension, and associations with BMI Z-score (BMIz) and left ventricular mass index adjusted to the 95th percentile (aLVMI) in a large, multicenter cohort of hypertensive children. Hypertensive children were divided and analyzed in three ambulatory hypertension subgroups: 24-h, daytime, and nighttime. Specifically, we sought to determine the prevalence of ambulatory 24-h, daytime, or nighttime IDH.ResultsPrevalence of IDH varied based on ambulatory phenotypes, ranging from 6 to 12%, and was highest in children with SH. Children with IDH tended to be more likely female and, in some cases, were leaner than those with isolated systolic hypertension (ISH). Despite previous pediatric studies suggesting no strong association between diastolic blood pressure and left ventricular hypertrophy (LVH), we observed that children with IDH were equally likely to have LVH and had comparable aLVMI to those with ISH and combined systolic-diastolic hypertension.ConclusionsIn summary, ambulatory IDH appears to be a unique phenotype with a female sex, and younger age predilection, but equal risk for LVH in children with either PH or SH.Graphical abstract

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