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NCT ID: NCT06468527 Recruiting - Cystic Fibrosis Clinical Trials

Clinical Trial to Evaluate the Efficacy and Safety of Dirocaftor/Posenacaftor/Nesolicaftor in Adults With CF

CHOICES
Start date: June 3, 2024
Phase: Phase 2
Study type: Interventional

CF is caused by mutations in the gene that encodes the 'Cystic Fibrosis Transmembrane Conductance Regulator (CFTR)' channel. To re-establish the function of this complex chloride channel, typically two to three drug modes of action are needed. To date, clinical studies of CFTR modulators have focused on patients carrying the F508del CFTR mutation, which is present in approximately 80% of CF patients, or gating mutations which are present in 5% of CF patients (gating mutations result in a reduced opening of the CFTR-channel at the cell surface which limits the flow of chloride ions through the CFTR channel). Although CF is a monogenetic disease, the 15% remaining patients represent more than 2000 different rare and mostly uncharacterized CFTR mutations. Multiple pharma companies have one or more CF drugs in their developmental pipeline. However, it is not known which patients may respond to the drugs in the pipeline. It is hypothesized that by using individual patient's intestinal organoids to screen for drug response, a subset of patients with rare CFTR mutations can be identified who will clinically respond to drugs in the developmental pipeline. The Human Individualized Therapy of CF (HIT-CF) project has been designed to further evaluate this hypothesis. The project has received funding from the European Union's Horizon 2020 research and innovation program under grant agreement No 755021. The core of the project consists of a two-step approach to identify patients outside the existing drug label who may also benefit from CFTR-modulator treatment. In the first step of the project (HIT-CF Organoid Study, NTR7520), novel CFTR modulators and their combinations were tested on organoids from over 500 European and Israeli CF patients with rare CFTR mutations to identify patients who are predicted to clinically benefit from these treatments. The second step will evaluate the predicted clinical effect of the CFTR modulators in subjects identified by their organoid response to investigational products. CFTR modulators from the HIT-CF participating pharmaceutical company, FAIR Therapeutics, will be evaluated in the CHOICES clinical study described in this protocol. Data from this clinical study will be compared with the HIT-CF Organoid Study results to validate the organoid model.

NCT ID: NCT06464302 Recruiting - Rib Fractures Clinical Trials

Investigation of the Effect of Age and Injury Severity on Short-term Intra-hospital Outcomes After Surgical Stabilized Rib Fractures

Start date: May 1, 2024
Phase:
Study type: Observational

The study aims to investigate the effect of surgical stabilisation of rib fractures (SSRF) on clinical outcomes measured during the hospital stay (mortality, days on a mechanical ventilator, intensive care unit and hospital length of stay, rate of complications). Furthermore, the effect of the patients age and overall injury severity on the outcomes after SSRF will be investigated. We hypothesise that the combination of high age and high injury severity will lead to worse outcomes after SSRF.

NCT ID: NCT06461481 Recruiting - Multiple Sclerosis Clinical Trials

Strategies for Adaptive Follow-up and Evaluation - Guiding Use of Indicators for De-Escalation in Multiple Sclerosis

SAFEGUIDE-MS
Start date: June 15, 2024
Phase:
Study type: Observational

The study will attempt to closely analyze Multiple Sclerosis (MS) patients after de-escalating or discontinuation of immunotherapy using clinical monitoring as well as digital and serological biomarkers in order to detect clinical progression or disease activity. As this is an observational study, it aims to closely follow-up on patients where the clinical decision to de-escalate or end treatment has been independently made. Specifically, we want to find out to what extent patients will show increased disease activity after de-escalation/discontinuation from high-efficacy treatment (HET) and which measurement method (clinical, digital, serological) retrospectively reflects the disease activity most closely or detects it most sensitively.

NCT ID: NCT06460142 Recruiting - Clinical trials for Giant Cell Arteritis

Assessing Biomarker in Giant Cell Arteritis and Polymyalgia Rheumatic

GCAIO
Start date: September 1, 2023
Phase:
Study type: Observational

The GCAIO study is an innovative, multimodal research initiative designed to enhance the understanding, diagnosis, and management of giant cell arteritis (GCA) and frequently associated polymyalgia rheumatica (PMR). This longitudinal study aims to dissect the complex immunological landscape and systemic manifestations of these conditions through a combination of diagnostic imaging and detailed immunological profiling. The study focuses on three primary objectives: (1) Identifying and analyzing cytokine profiles and immune cell phenotypes, employing techniques like flow cytometry, enzyme-linked immunosorbent assays (ELISA), and next-generation sequencing to predict disease activity and therapeutic responses. (2) Advancing diagnostic and monitoring capabilities through the application of novel and established imaging technologies, including MRI, optical coherence tomography angiography (OCTA), and ultrasound. These modalities aim to improve the detection of neuro-ophthalmological, cardiac, and aortic complications in GCA, potentially offering more precise monitoring and earlier diagnosis. (3) Enhancing the understanding of PMR within the context of GCA by exploring specific biomarkers and advanced imaging to refine diagnostic accuracy and treatment strategies, thus improving patient outcomes.

NCT ID: NCT06459765 Recruiting - Clinical trials for Hearing Loss, Sensorineural

Mi2000 Totally Implantable Cochlear Implant (Mi2000 TICI)

Start date: June 2024
Phase: N/A
Study type: Interventional

The goal of this clinical trial is to test the Mi2000 Totally Implantable Cochlear Implant in a population of candidates for a cochlear implant. The main question it aims to answer is, whether the device is able to improve speech perception compared to the pre-operative score. Participants will undergo cochlear implantation and fitting, and will be asked to perform the following tests pre- and post-operatively: - Word test in quiet - Sentence test in noise - Audiograms - Health Utilities Index 2 and 3 (HUI2&3), a generic quality-of-life questionnaire - Nijmengen Cochlear Implant Questionnaire (NCIQ), a disease specific quality-of life questionnaire - Speech, Spatial and Qualities of Hearing Scale (SSQ12), a disease specific questionnaire - Hearing Implant Sound Quality Index (HISQUI19), a sound quality questionnaire

NCT ID: NCT06459115 Recruiting - Clinical trials for Acute Decompensated Heart Failure

Adherence to Medication in Patients With Acute Decompensated Heart Failure

ADHF-ED
Start date: February 1, 2023
Phase:
Study type: Observational [Patient Registry]

Every day, patients present to emergency department due to acute heart failure. There are many causes for decompensation. One possible cause is a lack of adherence to heart failure medication (prognosis-improving medications and diuretics). The aim of this study is to directly measure adherence in patients with acute heart failure (gold standard of adherence measurement using liquid chromatography coupled to high-resolution mass spectrometry= LC-HRMS/MS) at the emergency department. Questionnaires are used to investigate possible factors influencing adherence.

NCT ID: NCT06453577 Recruiting - Clinical trials for Acute Decompensated Heart Failure

Pharmacokinetics of Bisoprolol and SGLT2i in Acutely Decompensated Heart Failure

BISO-ADHF
Start date: May 1, 2023
Phase:
Study type: Observational

The pharmacokinetics (PK) and pharmacodynamics (PD) of bisoprolol and sodium-glucose co-transporter-2 inhibitors (SGLT2i, dapagliflozin and empagliflozin) in patients with acutely decompensated heart failure (ADHF), compared to the recompensated state, is unknown. If not in cardiogenic shock (no need of vasopressor (catechoalmines) therapy or other inotropic support), established oral betablocker therapy should de continued. Whether this holds true for SGLT2i in ADHF is less clear but current evidence suggest safety and potentially beneficial effects in doing so. To the best of our knowledge, no data regarding PK/PD are available for the most widely used beta blocker bisoprolol and the newly approved/in Germany available SGLT2i Dapagliflozin and Empagliflozin. This study shall provide first evidence on the PK/PD-profile of p.o. bisoprolol and SGLT2i (dapaglifozin or empagliflozin) regarding acute (hemodynamic) effects and safety as well as to provide data on dose recommendations eventually in patients with ADHF.

NCT ID: NCT06452654 Recruiting - Clinical trials for Diabetes Mellitus, Type 1

Anti-viral Action Against Type 1 Diabetes Autoimmunity

Start date: May 8, 2024
Phase: Phase 4
Study type: Interventional

The study GPPAD-05 AVAnT1A is a phase 4 clinical trial intending to enroll 2252 children, who will be randomly assigned to receive COVID-19 vaccination (Comirnaty® 3 μg Omicron XBB.1.5 or new variant Comirnaty vaccines ) or placebo from age 6 months. The study is an investigator initiated, randomized, controlled, multicentre, multinational, primary prevention trial for children at increased risk of type 1 diabetes. The primary objective is to determine whether vaccination of children with elevated genetic risk for type 1 diabetes against COVID-19 from 6 months of age reduces the cumulative incidence of islet autoantibodies or type 1 diabetes in childhood. Secondary objectives are: 1. to determine whether vaccination against COVID-19 similarly reduces the cumulative incidence of multiple islet autoantibodies in childhood. 2. to determine whether vaccination against COVID-19 similarly reduces the cumulative incidence of type 1 diabetes in childhood and 3. to determine whether vaccination against COVID-19 similarly reduces the cumulative incidence of celiac disease-associated transglutaminase autoantibodies in childhood. Further exploratory objectives are described in the study protocol. Study participants will be identified through an ongoing study screening for genetic risk of type 1 diabetes using a polygenic risk score (NCT03316261). Eligible participants will be enrolled at age 3.00 to 4.00 months (baseline visit). Randomization to vaccine or placebo will occur at age 6.00 to 7.00 months at visit 2. Consent will be obtained by the custodial parents prior to enrollment.

NCT ID: NCT06446856 Recruiting - Schizophrenia Clinical Trials

EVEN - Effects of VR on Empathy for Nature in Patients With Psychosis and Depressive Disorders

EVEN
Start date: May 27, 2024
Phase:
Study type: Observational

Study group: Experimental study to evaluate empathy, compassion, and nature connectedness before and after an immersive virtual reality experience in patients with depressive disorder, patients with psychotic disorder and healthy control subjects (subjects between 18 and 65 years of age). Primary hypothesis: The increase in nature connectedness explored by virtual body ownership of a tree in VR differs depending on the health condition (schizophrenia, depression, healthy controls).

NCT ID: NCT06445153 Recruiting - Clinical trials for Postoperative Delirium

Digitalized Clinical Decision Support for the Prevention of Postoperative Delirium (POD)

Start date: June 3, 2024
Phase: N/A
Study type: Interventional

The project aims to improve patient safety, reduce barriers to the implementation of current guideline recommendations, reduce workload in clinics, increase efficiency in work processes and close gaps in care.