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NCT ID: NCT05565092 Active, not recruiting - Clinical trials for Sickle Cell Disease (SCD)

Safety, Efficacy, Pharmacokinetic, and Pharmacodynamic Study of ALXN1820 in Adult Participants With Sickle Cell Disease

PHOENIX
Start date: February 22, 2023
Phase: Phase 2
Study type: Interventional

The primary objective of this study is to assess the safety and tolerability of ALXN1820 SC (subcutaneous) in participants with SCD (Sickle Cell Disease).

NCT ID: NCT05564936 Recruiting - Myasthenia Gravis Clinical Trials

The ME&MG Digital Solution for Autonomous Assessment of Myasthenia Gravis

Start date: January 24, 2024
Phase: N/A
Study type: Interventional

ME&MG is a standalone software (digital solution) running on patients smartphones, connected to a web portal for physicians. It is intended to be used as an unsupervised digital self-assessment tool for the monitoring of disabilities in patients living with MG. ME&MG contains digital active tests for the assessment of ptosis, breathing, dysarthria, upper- and lower-limb (arms and legs) weakness, treatment follow-up, and validated e-questionnaires related to daily activities, pain, fatigue, sleep, and depression disorders. The objectives of this study are to validate the accuracy, reliability and reproducibility of the unsupervised at-home self-assessment of symptoms on the patient's smartphone with ME&MG versus the standard in-clinic testing, as well as to evaluate the safety of the solution, its usability and satisfaction.

NCT ID: NCT05564546 Completed - Clinical trials for Obstructive Ventilatory Disorders

Study of Chronic Exposure of the Population Living on the Atlantic Coast and on the Caribbean Coast, in Martinique, During the Stranding Period and Outside the Stranding Period of Sargassum Seaweeds

SÉSAR
Start date: August 18, 2022
Phase: N/A
Study type: Interventional

Sargassum are pelagic brown sea algae. Two species grow on the surface of the ocean and drift freely with ocean currents in the form of slicks: the pelagic Sargassum fluitans and Sargassum natans. Since 2011, massive strandings of Sargassum seaweed in the French West Indies are to be deplored. In recent years, strandings have increased. In Martinique, sargassum stranding occurs mainly on the Atlantic coast. The duration of stranding from one year to another is variable, but the period during which the stranding is maximum is between April and October. The accumulation of strandings causes the compaction of algae and limits access to oxygen, which leads to anaerobic degradation which quickly results in the release of various gases, in particular hydrogen sulphide (H2S). Acute H2S poisoning is well characterized clinically according to exposure (cutaneous, respiratory, ophthalmological, digestive, neurological symptoms, etc.). On the other hand, there are very few data available on the clinical effects of prolonged, repeated exposure and most often at low doses. Among the toxic effects for which there are still uncertainties regarding long-term exposure to H2S are lung function, measured by spirometry, and airway inflammation, appreciable by measurement of FeNO (fractional exhaled nitric oxide). The investigators consider it relevant to focus on the clinical and biological characteristics of the population exposed to sargassum in Martinique, in the field, assuming that exposure to sargassum H2S causes pulmonary effects measurable by spirometry and by FeNO, during the stranding period and outside this period.

NCT ID: NCT05564520 Recruiting - Lipin-1 Deficiency Clinical Trials

International Multicenter Study on Lipin-1 Deficiency

LIPIN1
Start date: November 24, 2023
Phase:
Study type: Observational

LIPIN1 is an international registry that allows the collection of data on the follow-up, management and treatment of patients with Lipin-1 deficiency.

NCT ID: NCT05564507 Completed - Clinical trials for Cardiovascular Diseases

Effectiveness of Simulation-based Training on Transoesophageal Echocardiography Learning

SIMULATOR
Start date: November 1, 2020
Phase: N/A
Study type: Interventional

Background: Simulation-based training in transesophageal echocardiography (TEE) seems promising. However, data are limited to non-randomized or single-center studies. Objectives: This large multicenter randomized, parallel-group, unblinded, study will assess the impact of simulation-based versus traditional teaching on TEE knowledge and performance for medical fellows in cardiology. Eligibility: - All consecutive fellows in cardiology of all training levels (year 1-4) - who have never performed a TEE alone Design: - Multicenter, parallel-group, unblinded, randomized study with a prospective enrollment of all consecutive fellows in cardiology of all training level (year 1 to 4) who were recruited in 42 centers throughout France. - Randomization with stratification by center will be performed at the individual (fellow) level in 1:1 ratio to assign all the fellows to the traditional group or to the TEE simulation-based training group. - Each participant will complete two different tests during the study: 1) a pre-training test before starting the educational program; and 2) a final test performed 3 months after the end of the educational program. Each of these tests will include a theoretical test and a practical test on a TEE simulator. - The coprimary outcomes of the study to compare the two groups will be the scores in the final theoretical and practical tests after the training will be completed.

NCT ID: NCT05564208 Completed - Clinical trials for Healthy Participants

A Study to Evaluate the Effect of Food on the Level of Circulating Elafibranor in Healthy Participants After Intake of a Single Tablet

Start date: October 27, 2022
Phase: Phase 1
Study type: Interventional

This study will evaluate the effects of food on how much test drug is able to access the circulation and reach the target area (known as bioavailability). The test drug, elafibranor (IPN60190), is in development for the treatment of primary biliary cholangitis (PBC). PBC is a rare, long-term autoimmune disease of the liver. An autoimmune disease occurs when the immune (defence) system treats healthy parts of the body as if they were foreign and attacks them. This study will be in healthy volunteers, so this trial is not to test if the drug helps to improve health.

NCT ID: NCT05564169 Not yet recruiting - Alzheimer Disease Clinical Trials

Masitinib in Patients With Mild to Moderate Alzheimer's Disease

Start date: January 2024
Phase: Phase 3
Study type: Interventional

Masitinib is an orally administered tyrosine kinase inhibitor that targets activated cells of the neuroimmune system (mast cells and microglia). Study AB21004 will evaluate masitinib as an adjunct to cholinesterase inhibitor and/or memantine in patients with mild-to-moderate Alzheimer's disease.

NCT ID: NCT05563883 Active, not recruiting - Cancer Clinical Trials

Atrial Fibrillation and Cancer: a Nationwide French Cohort Study

Start date: September 22, 2022
Phase:
Study type: Observational

Atrial fibrillation (AF) is a common complication associated with cancer but the risk of AF according to the cancer localization and status as well as the risk of thromboembolisms, bleedings and mortality are poorly known. The objective of this study is to use a very large French nationwide cohort to adress thèses questions.

NCT ID: NCT05563831 Recruiting - Clinical trials for Vascular Malformations

National Evaluation of Patients With PIK3CA-Related Overgrowth Spectrum (PROS)

COSY
Start date: February 21, 2023
Phase:
Study type: Observational

Overgrowth syndromes are rare genetic disorders defined by tissue hypertrophy that can be either localized or generalized, affecting both latitudinal and longitudinal growth. The genes involved in overgrowth syndromes are not well characterized but mostly concern the PIK3CA/AKT/mTOR pathway, a major actor of cell growth and proliferation. The mutations are not inherited but occurs during embryogenesis leading to somatic mosaicism. Owing to the variability of the clinical presentation, their exact prevalence is yet unknown. In order to answer this question, the investigators team create here the first French national registry on overgrowth syndromes.

NCT ID: NCT05563688 Recruiting - Hypoxia Clinical Trials

Effect of Acute Sleep Restriction on Responses to Hypoxia

HypSom
Start date: October 26, 2022
Phase:
Study type: Observational

At altitude, humans are exposed to environmental hypoxia induced by the decrease in barometric pressure. On duty or in training, mountain troops, paratroopers or aircrew are regularly exposed to altitude. The effects of altitude on humans occur gradually from 1500 m and depend on both the duration of exposure and the altitude level. Cognitive disorders can occur from 3500 m (threshold of disorders) but there is a very large inter-individual variability. The countermeasure to altitude hypoxia is oxygen but its use is not systematic between 3000 and 4000 m. Its use depends on the duration of exposure, without clearly established standards. Incapacitating effects on the operational capacity and health of soldiers can therefore occur as early as 3500 m. In operations or during training, altitude exposure is often associated with a significant sleep debt (particularly during night or early morning missions), jet lag or precarious rest conditions in overseas operations. These sleep restrictions promote the degradation of mental performance with effects similar to those observed in hypoxia. The combination of these constraints induces a physiological stress which can favour alterations in mental performance, an increase in incapacity, intolerance to altitude or the occurrence of altitude-related pathologies in military personnel. This could occur in particular in the operational zone around the threshold of disorders (3500 m) where the indication of oxygen is discussed. The objective of this study is to assess the impact of acute sleep restriction on hypoxia tolerance.