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NCT ID: NCT04848779 Recruiting - Clinical trials for Glycogen Storage Disease Type II

A Prospective Study to Observe & Describe Clinical Outcomes of Alglucosidase Alfa Treatment in Patients ≤6 Months of Age With Infantile-onset Pompe Disease (IOPD)

Start date: June 10, 2021
Phase:
Study type: Observational [Patient Registry]

Primary Objective: To describe the effect of routine practice with alglucosidase alfa in patients with IOPD ≤6 months of age, on invasive ventilation-free survival after 52 weeks of treatment. Secondary Objectives: - To describe the effect of routine practice with alglucosidase alfa on invasive ventilation-free survival and survival at 12 and 18 months of age, as well as on change in left ventricular mass (LVM) Z score, Alberta Infant Motor Scale (AIMS) score, body weight, body length, and head circumference Z scores, and urinary glucose tetrasaccharide (Hex4), at Week 52 of treatment. - To describe the safety, tolerability, and immunogenicity of alglucosidase alfa in the routine practice of IOPD treatment.

NCT ID: NCT04848506 Enrolling by invitation - Clinical trials for Symptomatic Hypertrophic Cardiomyopathy (HCM)

Open-label Extension Study to Evaluate the Long-term Safety and Tolerability of Aficamten in Adults With HCM

FOREST-HCM
Start date: May 6, 2021
Phase: Phase 2
Study type: Interventional

The purpose of this study is to collect long-term safety and tolerability data for aficamten (CK-3773274)

NCT ID: NCT04848389 Completed - Cancer Clinical Trials

Optimization of the Nursing Time After the Use of Tissue Adhesives During the Chest Port Placement on Patients Treated With Chemotherapy Versus Sutures

PAC_COLLE
Start date: April 1, 2021
Phase: N/A
Study type: Interventional

Skin adhesive (Octyl cyanoacrylate - LIQUID BAND©) is increasingly used instead of suture for sutures on the superficial plane of the skin. It is frequently used in children, in traumatology and in plastic surgery. Several randomized trials have shown satisfactory results of the glue compared to suture in terms of infections and scar dehiscence, and aesthetics after abdominoplasty and mammoplasty. In the field of port-a-cath® placement in oncology: - The use of skin adhesive can save substantial nursing time, which makes it possible to consider the transition of this technique into routine patient management. Potential benefits include nurses managing large numbers of patients in day hospitals, and patients no longer needing a nurse for suture removal. - Cancer and chemotherapy toxicity can alter patients' body image and view of themselves. The presence of a scar can be traumatic for some patients. The second goal of this study is to test whether the use of glue can improve the appearance of the scar following port-a-cath® placement, an area in which there are divergent results.

NCT ID: NCT04848194 Completed - Psoriasis Clinical Trials

Pleiotropic Role of TRPV1 in Psoriasis Inflammation

TRIP
Start date: December 22, 2021
Phase: N/A
Study type: Interventional

Widely expressed in the sensory nerve endings of the skin, Transient Receptor Potential Vanilloid 1 (TRPV1) is a receptor that plays an important role in the perception of pain and pruritus but also in skin inflammation, primarily by inducing the local release of several neuropeptides. Although the mechanisms by which TRPV1-sensitizing inflammatory mediators in damaged skin have received considerable attention, the role of TRPV1 in psoriasis has so far been little explored. However, two studies have reported that ablation of sensory nerves expressing TRPV1 reduced psoriasiform skin inflammation, demonstrating the neuronal contribution to inflammation in psoriasis. However, the expression of TRPV1 is not limited to neurons alone. TRPV1 is also expressed by epidermal keratinocytes and skin microvessels. For example, in 2018, transcriptomic analysis of psoriatic patient skins (by definition devoid of neuron nuclei) revealed that TRPV1 expression was increased in the skin of psoriatic patients suffering from itching (pruritus). Regarding human keratinocytes, it is recognized that the activation of TRPV1 present on their surface induces the release of pro-inflammatory factors such as cyclooxygenase-2. In addition, the investigators have demonstrated that TRPV1 has a pivotal role in the keratinocyte production of inflammatory mediators, which is mediated by the protease-activated receptor-2 (PAR-2). However, the role of vascular TRPV1 in inflammation is not described. The investigators hypothesize that in addition to neuronal TRPV1, non-neuronal TRPV1 receptors of non-neuronal cells (keratinocytes and endothelial cells) may be involved in the vicious circle of the inflammatory process characteristic of psoriasis. Putting TRPV1 at the center of the deregulation of the homeostatic balance including epithelial, neuronal and vascular inflammation in psoriasis is totally innovative.

NCT ID: NCT04848025 Recruiting - Airway Disease Clinical Trials

Treatment of Central Airway Stenoses Using Computer-Assisted Customized 3d Stents TATUM

TATUM
Start date: June 17, 2021
Phase: N/A
Study type: Interventional

Airway stenting is usually efficient to treat central airway obstruction but can lead to severe and potentially lethal complications (stent migration, obstructive granuloma, perforation, and hemoptysis). 3D customized airway stents (computer-aided conception using CT-scan data), perfectly suited to airway anatomy, should improve the tolerance and safety of airway stenting. We demonstrated in a previous pilot study, dedicated to patients with anatomically complex stenoses the safety of 3D patient-specific stents. These appealing results in highly complex situations suggest the need to evaluate these devices in more common situations. In this study we aim to test our new generation stents in all patients planned for airway stenting. After computer-assisted segmentation of airways (VGStudioMAX 3.0 software), the stenosis is virtually corrected, leading to the conception of a virtual stent (AnatomikModeling, Toulouse), allowing for the fabrication of a mold using a 3D printer. The stent is then built and sterilized (NOVATECH, Berlin) and inserted during rigid bronchoscopy. Close follow up is then conducted, including clinical evaluation after one week, three, six and 12 months; spirometry at one week, 3 months, 6 months and 12 months and chest CT-scan at one week.

NCT ID: NCT04847882 Completed - Cystic Fibrosis Clinical Trials

Case-control Study of Sleep Disorders in Children With Cystic Fibrosis

Start date: May 20, 2021
Phase:
Study type: Observational

Our primary purpose is to compare the prevalence of sleep disorders in children aged 6 to 17 with cystic fibrosis versus controls with a Sleep disorder screening score, the SDSC. Our hypothesis is that patients aged 6 to 17 with cystic fibrosis have a higher prevalence of sleep disturbances than the general population of the same age group. Our secondary hypothesis is that these sleep disorders are mixed and that there are non-respiratory causes, sometimes modifiable by simple non-medical treatment and that's why our secondary purpose is to identify the responsible factors, in particular non-respiratory factors in the 2 groups and to compare them.

NCT ID: NCT04847622 Recruiting - Covid19 Clinical Trials

Study to Evaluate the Clinical Outcomes in Adults With Covid-19 Who Have Been Treated With Remdesivir.

909REM
Start date: February 1, 2021
Phase:
Study type: Observational

This is a multi-centre, multi-country retrospective cohort study. At least 450COVID-19 cases from up to 20 participating study sites who meet all eligibility criteria will be included in the analysis. Deidentified data will be extracted from electronic medical record (EMR) databases, clinical registries, case series or additional sources from participating sites and countries, and then entered into a structured e-CRF system. addition, each site/country will be surveyed to determine the local standard of care therapy for COVID-19 infection and to determine if standard protocols were/are in place for the use of Remdesivir and if/how the protocols changed over time.

NCT ID: NCT04847531 Completed - Clinical trials for Chronic Kidney Disease

REVEAL-CKD: Prevalence and Consequences of Undiagnosed Chronic Kidney Disease

REVEAL-CKD
Start date: December 15, 2020
Phase:
Study type: Observational

This is a retrospective, multinational, non-interventional, observational study. A series of cohort studies will be conducted to assess the prevalence of undiagnosed stage 3 CKD in each region. The study will also assess the current state of CKD management in patients with undiagnosed CKD

NCT ID: NCT04847180 Recruiting - Shoulder Capsulitis Clinical Trials

Study of the Antipodal Capsular Fold and Its Potential Role in Antero-inferior Glenohumeral Instability

INSTABIO
Start date: August 26, 2020
Phase: N/A
Study type: Interventional

In schoulders instabilty, the soft tissue has not been the subject of histological studies, as has the "bare area" or zone devoid of cartilage, the exact role of which in glenohumeral biomechanics is unknown. This research is based on the hypothesis that the antipodal lesions are constant, underestimated and that an architectural disorganization at the capsulo-ligament level could contribute to the instability of the shoulder. The aim of this study is therefore to better characterize these lesions which could be the subject of a complementary stabilization procedure even in the absence of a humeral notch

NCT ID: NCT04847037 Active, not recruiting - Clinical trials for Bariatric Surgery Candidate

Place of Connected Tools in Bariatric Patients Follow-up.

Start date: March 5, 2020
Phase: N/A
Study type: Interventional

The aim of this study is to assess the safety (in terms of post-operative complications) of the use of connected devices for the monitoring of patients operated on by longitudinal laparoscopic gastrectomy (LSG) and discharged 24 hours after surgery according to the protocol Enhanced Recovery After Surgery (ERAS).