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NCT ID: NCT06150716 Recruiting - Clinical trials for Pelizaeus-Merzbacher Disease

Orbit Study: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356 in Participants With Pelizaeus Merzbacher Disease (PMD)

Start date: April 10, 2024
Phase: Phase 1
Study type: Interventional

The primary purpose of this study is to evaluate the safety and tolerability of ION356.

NCT ID: NCT06150482 Recruiting - Liver Cirrhosis Clinical Trials

Healthcare Utilization in Liver Cirrhosis Patients, a Multicenter Retrospective Cohort Study

Start date: September 1, 2022
Phase:
Study type: Observational

Multicenter, retrospective cohort study investigating healthcare utilization in compensated and decompensated liver cirrhosis patients, including disease course and characteristics associated with healthcare utilization and, for example psychosocial factors.

NCT ID: NCT06149520 Recruiting - Thrombolysis Clinical Trials

A Study to Learn More About How Well BAY3018250 Works and How Safe it is for People With Proximal Deep Vein Thrombosis

SIRIUS
Start date: January 15, 2024
Phase: Phase 2
Study type: Interventional

Researchers are looking for a better way to treat people who have deep vein thrombosis (DVT). DVT is a condition that occurs when a blood clot forms in a deep vein in the leg. DVT is called 'proximal' when the clot is formed in the veins of the hip, thigh, and knee. DVT can cause serious health problems. The blood clots in the veins can break loose and can then travel through the bloodstream and get stuck in the lungs, blocking blood flow to the lungs. Symptoms of DVT include swelling, pain, and tenderness in the affected leg, as well as redness and warmth in the area. Currently, DVT is usually treated using blood thinners to prevent the clot from getting bigger or breaking off and traveling to the lungs. However, blood thinners may not be able to remove a blood clot quickly and may not be suitable for everyone who has DVT. BAY3018250 is a drug that works by dissolving blood clots. In this study, researchers will compare BAY3018250 with placebo to learn how well it works and how safe it is in participants with proximal DVT. A placebo looks like the study drug but does not have any medicine in it. Using a placebo helps researchers to confirm that the results observed during the study were caused by the study drug and not by other factors. The main purposes of this study are to learn: - How well BAY3018250 works in dissolving blood clots in participants with proximal DVT and - How safe is BAY3018250 as a treatment for participants with proximal DVT? For this, the researchers will use ultrasound tests to measure blood clots in participants before and at various times after study treatment. They call these measurements a clot burden score. They will compare the clot burden score before and after treatment and will calculate a complex measure called AUC. This tells researchers how the clots have changed over time. And researchers will collect the number of bleeding events that require medical attention. The study participants will be randomly (by chance) assigned to one of 3 treatment groups. Dependent on the group, they will receive a single dose of high dose or low dose of BAY3018250 or placebo. Researchers will closely monitor participants for 90 days after receiving the study treatment. During the study, the doctors and their study team will: - take blood samples - do physical examinations - examine heart health using electrocardiogram (ECG) - check vital signs such as blood pressure, heart rate - undergo ultrasound tests to measure the blood clots - ask the participants questions about how they are feeling and what adverse events they are having. An adverse event is any medical problem that a participant has during a study. Doctors keep track of all adverse events, irrespective if they think it is related or not to the study treatments.

NCT ID: NCT06149403 Recruiting - Clinical trials for MPS-IH (Hurler Syndrome)

A Study to Investigate the Efficacy and Safety of OTL-203 in Subjects With MPS-IH Compared With Standard of Care With Allogeneic HSCT

HURCULES
Start date: December 11, 2023
Phase: Phase 3
Study type: Interventional

A multi-center randomized clinical trial to compare OTL-203 (gene therapy) with stem cell transplant (standard of care) in patients with MPS-IH (Hurler syndrome).

NCT ID: NCT06147635 Recruiting - Acute Pancreatitis Clinical Trials

Prophylactic Tributyrin Supplementation in Acute Pancreatitis

PARROT
Start date: February 12, 2024
Phase: Phase 2
Study type: Interventional

The goal of this clinical trial is to investigate the possible effects of tributyrin supplementation in patients with a first episode of acute pancreatitis. The main question it aims to answer is: • The effect of oral tributyrin supplementation on the plasma endotoxin level Participants will be randomized between two groups: intervention and control group. They will receive: - three times daily 4grams of micro-encapsulated granules of tributyrin, and the control group three times daily 4 grams of micro-encapsulated sunflower oil (i.e. placebo), for a total of 14 days In total 92 adult patients with a first episode of acute pancreatitis will be included.

NCT ID: NCT06147089 Recruiting - Clinical trials for Shift-Work Related Sleep Disturbance

Shift Work Intervention Strategies for Night Shift Workers

GRIP
Start date: February 9, 2023
Phase: N/A
Study type: Interventional

The goal of this intervention study is to test behavioral interventions in night shift workers. The main question it aims to answer is: • Can personalized sleep or nutrition intervention strategies help against the negative health effects of night shift work? Participants will be followed before and after the intervention. Participants will wear smart devices, do a alertness/performance test and complete questionnaires. Blood samples will be collected. Researchers will compare the intervention groups to the control group (which receives no intervention) to see if the sleep and nutrition interventions affected the health of night shift workers.

NCT ID: NCT06146738 Recruiting - Glioblastoma Clinical Trials

The PALSUR-study: Palliative Care Versus Surgery in High-grade Glioma Patients (ENCRAM 2203)

PALSUR
Start date: January 1, 2023
Phase:
Study type: Observational

There is no consensus on the optimal treatment of patients with high-grade glioma, especially when patients have limited functioning performance at presentation (KPS ≤70). Therefore, there are varied practice patterns around pursuing biopsy, resection, or palliation (best supportive care). This study aims to characterize the impact of palliative care versus biopsy versus resection on survival and quality of life in these patients. Also, it will aim to determine if there is a subset of patients that benefit the most from resection or biopsy, for which outcome, and how they could be identified preoperatively. This study is an international, multicenter, prospective, 3-arm cohort study of observational nature. Consecutive HGG patients will be treated with palliative care, biopsy, or resection at a 1:3:3 ratio. Primary endpoints are: 1) overall survival, and 2) quality of life at 6 weeks, 3 months and 6 months after initial presentation based on the EQ-5D, EORTC QLQ C30 and EORTC BN 20 questionnaires. Total duration of the study is 5 years. Patient inclusion is 4 years, follow-up is 1 year.

NCT ID: NCT06146725 Recruiting - Glioblastoma Clinical Trials

The RESBIOP-study: Resection Versus Biopsy in High-grade Glioma Patients (ENCRAM 2202)

RESBIOP
Start date: January 1, 2023
Phase:
Study type: Observational

There are no guidelines or prospective studies defining the optimal surgical treatment for gliomas of older patients (≥70 years) or those with limited functioning performance at presentation (KPS ≤70). Therefore, the decision between resection and biopsy is varied, amongst neurosurgeons internationally and at times even within an instiutition. This study aims to compare the effects of maximal tumor resection versus tissue biopsy on survival, functional, neurological, and quality of life outcomes in these patient subgroups. Furthermore, it evaluates which modality would maximize the potential to undergo adjuvant treatment. This study is an international, multicenter, prospective, 2-arm cohort study of observational nature. Consecutive HGG patients will be treated with resection or biopsy at a 3:1 ratio. Primary endpoints are: 1) overall survival (OS) and 2) proportion of patients that have received adjuvant treatment with chemotherapy and radiotherapy. Secondary endpoints are 1) proportion of patients with NIHSS (National Institute of Health Stroke Scale) deterioration at 6 weeks, 3 months and 6 months after surgery 2) progression-free survival (PFS); 3) quality of life at 6 weeks, 3 months and 6 months after surgery and 4) frequency and severity of Serious Adverse Events (SAEs). Total duration of the study is 5 years. Patient inclusion is 4 years, follow-up is 1 year.

NCT ID: NCT06145984 Recruiting - Clinical trials for Depression in Remission

Mechanisms of Change of Positive Interventions in Reducing Vulnerability for Depression

MINDCOG
Start date: June 19, 2020
Phase: N/A
Study type: Interventional

The purpose of this study is to understand the effects of mindfulness and fantasizing in reducing perseverative cognition underlying vulnerability for depression.

NCT ID: NCT06144840 Recruiting - Clinical trials for Erythropoietic Protoporphyria (EPP)

INcreased Sun Exposure Without Pain In Research Participants With EPP or XLP

INSPIRE
Start date: December 11, 2023
Phase: Phase 3
Study type: Interventional

To investigate the efficacy of MT-7117 on time to onset and severity of first prodromal symptoms (burning, tingling, itching, or stinging) associated with sunlight exposure in adults and adolescents with EPP or XLP.