Assessment of the safety and effectiveness of vedolizumab induction therapy compared to standard infliximab therapy in pediatric patients with ulcerative colitis VEDI-UC” - VEDI-UC

Start Date20 Nov 2024

Sponsor / Collaborator

$Instytut \"Pomnik - Centrum Zdrowia Dziecka\"$ Instytut \"Pomnik - Centrum Zdrowia Dziecka\"

NCT06614387

/ Not yet recruitingPhase 3IIT

Assessment of Safety and Efficacy of Vedolizumab in Pediatric Uclerative Colitis in Comparison With Standard Biological Therapy With Infliximab

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.

Start Date01 Oct 2024

Sponsor / Collaborator

$Instytut \"Pomnik - Centrum Zdrowia Dziecka\"$ Instytut \"Pomnik - Centrum Zdrowia Dziecka\"

NCT05534672

/ RecruitingPhase 3IIT

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).

Start Date23 Jan 2023

Sponsor / Collaborator

$Instytut \"Pomnik - Centrum Zdrowia Dziecka\"$ Instytut \"Pomnik - Centrum Zdrowia Dziecka\"

100 Clinical Results associated with Instytut \"Pomnik - Centrum Zdrowia Dziecka\"

0 Patents (Medical) associated with Instytut \"Pomnik - Centrum Zdrowia Dziecka\"

1,847

Literatures (Medical) associated with Instytut \"Pomnik - Centrum Zdrowia Dziecka\"

01 Dec 2025·Child's Nervous System

Meningeal melanocytoma of the central nervous system in children

Review

Author: Grajkowska, Wieslawa ; Trubicka, Joanna ; Antkowiak, Lukasz ; Luszawski, Jerzy ; Mandera, Marek

PURPOSEThis study aimed to summarize the existing English-language literature on central nervous system (CNS) meningeal melanocytomas in children, and additionally describe our institutional case report.METHODSPubMed database was screened on September 2, 2024, for English-language papers reporting on pediatric patients with CNS meningeal melanocytoma.RESULTSA total of 17 papers reporting on 18 patients with 19 CNS meningeal melanocytomas were found in the literature. Additionally, we reported on a 15-year-old male patient with C2-C6 meningeal melanocytoma. Pediatric cohort analysis showed nearly equal sex distribution and a mean age at diagnosis of 11.9 years. There were fifteen intracranial (75%) and five spinal tumors (25%). Four lesions (20%) were diagnosed as intermediate-grade melanocytomas, while the remaining sixteen (80%) were benign meningeal melanocytomas. Most tumors were hyperintense on T1-weighted imaging (85%) and hypointense on T2-weighted imaging (73%). All tumors showed positivity for S100 and Melan-A. Most tumors were characterized by a lack of CNS invasion (91%). Gross-total resection (GTR) was performed in 61% of tumors. Adjuvant radiotherapy (RT) was applied in 50% of patients with incomplete tumor resection. Postoperatively, 62% of patients achieved a favorable outcome. We found 1, 2, 3, and 4-year overall survival of 80%, 71%, 71%, and 50%, respectively. The recurrence rate was 15% after a mean time of 10 months.CONCLUSIONSMeningeal melanocytomas constitute a rare subgroup of CNS tumors. Surgical tumor removal aiming at maximally safe GTR remains a standard approach, resulting in favorable postoperative outcomes. Considering high recurrence rate, long-term follow-up is needed.

01 Feb 2025·Journal of Neurology

Ocrelizumab dose selection for treatment of pediatric relapsing–remitting multiple sclerosis: results of the OPERETTA I study

Article

Author: Valeriani, Massimiliano ; Banwell, Brenda ; Schreiner, Teri ; Kotulska, Katarzyna ; Mar, Soe ; Kletzl, Heidemarie ; Manlius, Corinne ; El Azzouzi, Bouchra ; Steinborn, Barbara ; Bonati, Ulrike ; Lin, Chien-Ju ; Evershed, Joanna ; Filippi, Massimo ; Mazurkiewicz-Beldzinska, Maria ; Shen, Yun-An ; Waubant, Emmanuelle ; Hogea, Alexandra

AbstractBackgroundThe presented study identified the appropriate ocrelizumab dosing regimen for patients with pediatric-onset multiple sclerosis (POMS).MethodsPatients with POMS aged 10–17 years were enrolled into cohort 1 (body weight [BW] < 40 kg, ocrelizumab 300 mg) and cohort 2 (BW ≥ 40 kg, ocrelizumab 600 mg) during a 24-week dose-exploration period (DEP), followed by an optional ocrelizumab (given every 24 weeks) extension period. Primary endpoints: pharmacokinetics, pharmacodynamics (CD19+ B-cell count); secondary endpoint: safety; exploratory endpoints: MRI activity, protocol-defined relapses, Expanded Disability Status Scale (EDSS) score change.ResultsA total of 23 patients (cohort 1: n = 6, age 10–12 years, BW 27.0–39.0 kg; cohort 2: n = 17, age 11–17 years, BW 42.1–108.4 kg) were enrolled. Median treatment duration was 120 (range, 24–193) weeks at the primary analysis cutoff (October 5, 2023). Overall, the pharmacokinetic data were within the range observed at 600 mg in adult patients with MS; however, the exposure at 300 mg in patients < 40 kg was lower than with 600 mg in patients ≥ 40 kg. Shifting the cutoff to 35 kg would provide better exposure to patients with 35–40 kg body weight. Sustained, near-complete B-cell depletion was observed. The safety profile was consistent with that in adults. EDSS scores remained stable; no clinical relapses were observed.ConclusionA dosing regimen of 300 mg ocrelizumab for patients < 35 kg, and 600 mg for patients ≥ 35 kg (every 24 weeks), was selected for the phase 3 OPERETTA II trial (NCT05123703).Trial registrationClinicalTrials.gov: NCT04075266.

01 Jan 2025·Nature Genetics

ZIC1 is a context-dependent medulloblastoma driver in the rhombic lip

Article

Author: Mack, Stephen C ; Mount, Christopher W ; Fong, Vernon ; Pollack, Ian F ; Northcott, Paul A ; Hendriske, Liam D ; Phillips, Joanna J ; Hamilton, Ronald L ; Tominaga, Teiji ; Richman, Cory M ; Daniels, Craig ; Garzia, Livia ; Morrissy, Sorana ; Saad, Ali G ; Cho, Byung-Kyu ; Dai, Shizhong ; Farooq, Hamza ; Abeysundara, Namal ; Lee, Ji-Yeoun ; Singh, Sheila K ; Lee, John J Y ; Palotta, Jonelle ; Leary, Sarah E S ; Gallo, Marco ; Lach, Boleslaw ; Rasnitsyn, Alex ; Loukides, James ; Bognár, László ; Chico Ponce de León, Fernando ; Das Gupta, Neha ; Visvanathan, Abhi ; Kool, Marcel ; Thompson, Reid C ; Klekner, Almos ; Grajkowska, Wieslawa A ; Luu, Betty ; Weiss, William A ; Batts, Melissa ; Bendel, Anne ; Millen, Kathleen J ; You, Zhen ; Liu, Miao ; Livingston, Bryn G ; Kros, Johan M ; Cooper, Michael K ; Wang, Evan Y ; Wang, Kyu-Chang ; Jabado, Nada ; Wu, Xiaochong ; Huang, L Frank ; Lupien, Mathieu ; Bailey, Swneke ; Suzuki, Hiromichi ; Haldipur, Parthiv ; Tao, Ran ; Kim, Seung-Ki ; Korshunov, Andrey ; Perezpeña-Diazconti, Mario ; Zadeh, Gelareh ; Perek-Polnik, Marta ; Cavalli, Florence M G ; Ra, Young-Shin ; Hadley, Jennifer ; Erickson, Anders W ; Suarez, Raul ; Ramaswamy, Vijay ; Kenney, Anna M ; Michealraj, Kulandaimanuvel Antony ; French, Pim J ; Suva, Mario L ; Taylor, Michael D

AbstractTranscription factors are frequent cancer driver genes, exhibiting noted specificity based on the precise cell of origin. We demonstrate that ZIC1 exhibits loss-of-function (LOF) somatic events in group 4 (G4) medulloblastoma through recurrent point mutations, subchromosomal deletions and mono-allelic epigenetic repression (60% of G4 medulloblastoma). In contrast, highly similar SHH medulloblastoma exhibits distinct and diametrically opposed gain-of-function mutations and copy number gains (20% of SHH medulloblastoma). Overexpression of ZIC1 suppresses the growth of group 3 medulloblastoma models, whereas it promotes the proliferation of SHH medulloblastoma precursor cells. SHH medulloblastoma ZIC1 mutants show increased activity versus wild-type ZIC1, whereas G4 medulloblastoma ZIC1 mutants exhibit LOF phenotypes. Distinct ZIC1 mutations affect cells of the rhombic lip in diametrically opposed ways, suggesting that ZIC1 is a critical developmental transcriptional regulator in both the normal and transformed rhombic lip and identifying ZIC1 as an exquisitely context-dependent driver gene in medulloblastoma.

100 Deals associated with Instytut \"Pomnik - Centrum Zdrowia Dziecka\"

100 Translational Medicine associated with Instytut \"Pomnik - Centrum Zdrowia Dziecka\"

Corporation Tree

Boost your research with our corporation tree data.

view full example data

Pipeline

Pipeline Snapshot as of 23 Jan 2025

No data posted

Deal

Boost your decision using our deal data.

view full example data

Translational Medicine

Boost your research with our translational medicine data.

view full example data

Profit

Explore the financial positions of over 360K organizations with Synapse.

view full example data

Grant & Funding(NIH)

Access more than 2 million grant and funding information to elevate your research journey.

view full example data

Investment

Gain insights on the latest company investments from start-ups to established corporations.

view full example data

Financing

Unearth financing trends to validate and advance investment opportunities.

view full example data

Chat with Hiro

Get started for free today!

Accelerate Strategic R&D decision making with Synapse, PatSnap’s AI-powered Connected Innovation Intelligence Platform Built for Life Sciences Professionals.

Start your data trial now!

Synapse data is also accessible to external entities via APIs or data packages. Empower better decisions with the latest in pharmaceutical intelligence.

Bio

Bio Sequences Search & Analysis

Chemical

Chemical Structures Search & Analysis

Instytut \"Pomnik - Centrum Zdrowia Dziecka\"

Overview

Related

Corporation Tree

Pipeline

Pipeline Snapshot as of 23 Jan 2025

Deal

Translational Medicine

Profit

Grant & Funding(NIH)

Investment

Financing