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NCT ID: NCT05181839 Completed - Clinical trials for X-Linked Hypophosphatemia

A Study to Describe the Lived Experience of XLH for Adolescents at End of Skeletal Growth

Start date: November 24, 2021
Phase:
Study type: Observational [Patient Registry]

An observational, prospective, mixed-methods study involving the integration of quantitative and qualitative data exploring the lived experience of burosumab-treated adolescents with XLH at the end of skeletal growth.

NCT ID: NCT05181735 Recruiting - Clinical trials for Myelodysplastic Syndromes

Study Evaluating Combination of Luspatercept in LR-MDS Without RS Having Failed or Being Ineligible to ESA

Start date: May 18, 2022
Phase: Phase 1/Phase 2
Study type: Interventional

Study of the combination of luspatercept in low-risk myelodysplastic syndrom (LR-MDS) without ring sideroblasts (RS) having failed or being ineligible to ESA

NCT ID: NCT05181345 Recruiting - Sleep Disorder Clinical Trials

Effect of Sleep Debt on Neurophysiological Responses to Heat Exposure

ChaSPerf
Start date: January 14, 2022
Phase:
Study type: Observational

Many people are required to work in stressful situations combining sleep debt and hot environmental conditions. If the effect of sleep debt on cognitive performance is proven, this effect could be increased, during heat exposure, through the deleterious effects of sleep debt on thermoregulatory abilities. These alterations may favour the occurrence of accidents. The changes in cognitive performance induced by hyperthermia are also poorly characterised and often not dissociated from the effects of dehydration. Little is known about the effects of the combination of sleep debt and heat exposure on mental performance. Describing and understanding the alterations induced by this combined situation could provide a better understanding of the mechanisms explaining the deterioration of performance in hot conditions and promote the development of appropriate countermeasures.

NCT ID: NCT05180825 Recruiting - Clinical trials for Pleomorphic Xanthoastrocytoma

Pediatric Low Grade Glioma - MEKinhibitor TRIal vs Chemotherapy

PLGG - MEKTRIC
Start date: May 5, 2022
Phase: Phase 2
Study type: Interventional

Pediatric low-grade glioma (PLGG) is a heterogeneous group of WHO grade I and II brain tumors, associated with a 10-year overall survival of 90%. It is the most common form of primary central nervous system (CNS) tumor arising during childhood, adolescence and young adulthood, accounting for over 30% of CNS tumors in this age group. A large group of PLGG patients will benefit from a complete resection of their tumor. Nevertheless, PLGG can occur anywhere and can be in some locations associated with neurological symptoms, unresectable or radiological progressive tumors that need medical treatments rapidly to avoid long-term sequelae. The current problem during this first line therapy is to improve tumor response, overall survival rate, as well as progression free survival. In our study, we will focus on a specific group of PLGGs without any congenital NF1 mutation and with a wild-type BRAF gene in the tumor. In this subgroup, for instance, the PFS is not increasing anymore above 50% at 3 years independently from the chemotherapeutic scheme. The two current standard therapies are carboplatin plus vincristine during 81 weeks or a weekly IV administration of vinblastine during 70 weeks. The most recent Canadian approach with vinblastine seems to have the same PFS rate, but with a better daily tolerance and less toxicities than the carboplatin/vincristine combination. Therefore, it is becoming the new standard approach in those patients. Nevertheless, we need to improve more their outcome with less recurs and a better first-line tumor response. The recent molecular discoveries involving the Ras/mitogen-activated protein kinase pathway in those PLGG is opening a new era with specific targeted therapies that might be the key to improve their survivals and giving hope to less treatment lines and a better tumor response. Therefore, we designed a prospective open randomized phase II study, named PLGG-MEKTRIC, comparing the experimental arm (a daily MEK inhibitor, Trametinib, Mekinist©) to a standard arm comprising weekly vinblastine during 18 courses of 4 weeks each. The study will enroll 134 patients with a PLGG during childhood, adolescence or young adulthood with no NF1-related disease and without any BRAFv600 mutation located in brain or spine. 67 patients, in each treatment arm, are planned to be enrolled to answer our primary objective. This primary objective will be to determine in the experimental arm a 20% superiority of the 3-year PFS rate in comparison with the standard treatment administered during 18 courses (e.g. 72 weeks). A stratification of the patients will be done in both arms based on molecular tumor results and brain/spine locations to obtain two equivalent arms to be analyzed. The recruitment time will be 36 months and the complete follow-up of each patient will last 3 years. The secondary objectives will be in both arms: the tumor response rate at 24 and 72 weeks of treatment, the 3-year PFS and OS rates and the frequency of AE/SAE/SUSAR (Adverse Event/Serious Adverse Event) based on CTCAE criteria during the 3 years after the first administration. A Quality of Life (QoL) assessment, based on PEDsQL questionnaires, at 24 weeks, at the end of treatment and 3 years after 1st treatment administration in both arms will be part of this study. Finally, 3-year PFS and OS will be analyzed according to molecular biomarkers and visual assessment (LogMar scale) in each arm. An economic analysis is also planned as an ancillary study to determine a cost effectiveness of the best arm and complementary ancillary molecular studies are already organized. In the future, we hope to push forward this new-targeted therapy as a referenced first line treatment of pediatric PLGG to obtain the best tolerance and positive long-term impact and to extend our knowledge of MEK inhibitor impact in molecular subgroups and in optical pathway locations. We also plan to do a "switch" strategy in patients relapsing in standard arm and we will propose systematically to those patients the experimental treatment (MEK inhibitor ).

NCT ID: NCT05180695 Recruiting - Clinical trials for Advanced Soft-tissue Sarcoma

HDM201 and Pazopanib in Patients With P53 Wild-type Advanced/Metastatic Soft Tissue Sarcomas

AMPHISARC
Start date: April 15, 2022
Phase: Phase 1/Phase 2
Study type: Interventional

This trial is a two-step Phase I/II study comprising: Part 1: A dose escalation part with the aim to assess the safety of the proposed combination (N= up to 30 patients). In the dose escalation part, eligible patients will be treated with a fixed dose of pazopanib and escalating doses of HDM201. Part 2: An extension part to collect preliminary data about the clinical activity of the proposed combination according to the 6M-PFR.

NCT ID: NCT05180643 Completed - Parkinson Disease Clinical Trials

Mindfulness Meditation (MBSR) and Parkinson's Disease (PD)

M-PARK
Start date: February 17, 2020
Phase: N/A
Study type: Interventional

Non-pharmacological therapies become more important in the management of Parkinson's disease (PD). Among these, mindfulness meditation is the subject of high expectations. This intervention, such as the Mindfulness-Based Stress Reduction-based (MBSR) stress reduction program, have shown effects on psychological distress, motor and non-motor disorders, and quality of life. However, the data is still very frail and the conditions for practical use are still very uncertain. The objective of the study is to determine the feasibility of a standardized MBSR program in Parkinsonians patients.

NCT ID: NCT05180565 Completed - Colorectal Cancer Clinical Trials

Safe Anastomosis Feasibility Study

SAFE2019
Start date: December 18, 2019
Phase: N/A
Study type: Interventional

A clinical trial to assess the efficacy, mechanism of action and safety of the Colovac+ Colorectal Anastomosis Protection Device in providing temporary protection of the anastomosis in subjects undergoing lower anterior resection for colorectal cancer

NCT ID: NCT05180474 Recruiting - Clinical trials for Endometrial Cancer, Endometrial Neoplasm

GEN1047 for Solid Tumors - First in Human (FIH) Trial

Start date: December 13, 2021
Phase: Phase 1/Phase 2
Study type: Interventional

The drug investigated in the study is an antibody, GEN1047. Since this is the first study of GEN1047 in humans, the main purpose is to evaluate safety. Besides safety, the study will determine the recommended GEN1047 dose to be tested in a larger group of participants and assess preliminary clinical activity of GEN1047. GEN1047 will be studied in a broad group of cancer participants, having different kinds of solid tumors. All participants will get GEN1047. The study consists of two parts: Part 1 tests increasing doses of GEN1047 ("escalation"), followed by Part 2 ("expansion") which tests the recommended GEN1047 dose from Part 1.

NCT ID: NCT05180357 Completed - Nasal Polyps Clinical Trials

RANS. Study in Patients With Severe Eosinophilic Asthma and Nasal Polyps.

RANS
Start date: November 23, 2021
Phase:
Study type: Observational

The purpose of this observational study is to describe the population of patients with SEA + NP who have been prescribed FASENRA and assess available clinical outcomes for both NP and asthma.

NCT ID: NCT05180240 Recruiting - Acute Myocarditis Clinical Trials

Impact of CardiolRx on Myocardial Recovery in Patients With Acute Myocarditis

ARCHER
Start date: June 22, 2022
Phase: Phase 2
Study type: Interventional

Multi-center, double-blind, placebo-controlled, parallel group design. Patients with myocarditis will be screened and, if eligible, randomized within 10 days of the diagnostic CMR to CardiolRx or placebo. CardiolRx is pharmaceutically produced Cannabidiol and is free of tetrahydrocannabinol (THC<5 ppm). The treatment period is 12 weeks; a last follow-up visit is scheduled one week after the last treatment, 13 weeks after randomization. Study assessments include Cardiac Magnetic Resonance imaging (CMR), ECG monitoring, the Kansas City Cardiomyopathy Questionnaire (KCCQ), the Columbia-Suicide Severity Rating Scale (C-SSRS) as well as physical exams and laboratory tests. The primary and secondary outcome parameters are measured by CMR. Additional outcomes include clinical endpoints and changes in inflammatory and biomarkers.