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NCT ID: NCT05882357 Recruiting - Cystic Fibrosis Clinical Trials

Evaluation of Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) in Cystic Fibrosis (CF) Participants 12 to Less Than 24 Months of Age

Start date: June 27, 2023
Phase: Phase 3
Study type: Interventional

This study will evaluate the pharmacokinetics (PK), safety, tolerability, pharmacodynamics (PD), and efficacy of ELX/TEZ/IVA in CF subjects 12 to less than (<) 24 months of age.

NCT ID: NCT05882058 Recruiting - Clinical trials for Small Cell Lung Carcinoma

DAREON™-5: A Study to Test Whether Different Doses of BI 764532 Help People With Small Cell Lung Cancer or Other Neuroendocrine Cancers

Start date: September 25, 2023
Phase: Phase 2
Study type: Interventional

This study is open to adults with small cell lung cancer and other neuroendocrine tumours. The study is in people with advanced cancer for whom previous treatment was not successful or no standard treatment exists. The purpose of this study is to find a suitable dose of BI 764532 that people with advanced cancer can tolerate when taken alone. 2 different doses of BI 764532 are tested in this study. Another purpose is to check whether BI 764532 can make tumours shrink. BI 764532 is an antibody-like molecule (DLL3/CD3 bispecific) that may help the immune system fight cancer. Participants are put into 2 groups randomly, which means by chance. One group gets dose 1 of BI 764532 and the other group gets dose 2 of BI 764532. Participants get BI 764532 infusions into a vein when starting treatment. If there is benefit for the participants and if they can tolerate it, the treatment is given up to the maximum duration of the study. During this time, participants visit the study site regularly. The total number of visits depends on how they respond to and tolerate the treatment. The first study visits include an over-night stay to monitor participants' safety. Doctors record any unwanted effects and regularly check the general health of the participants.

NCT ID: NCT05881408 Recruiting - Clinical trials for Duchenne Muscular Dystrophy

A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Non-Ambulatory and Ambulatory Participants With Duchenne Muscular Dystrophy (DMD)

ENVISION
Start date: May 31, 2023
Phase: Phase 3
Study type: Interventional

The study will evaluate the safety and efficacy of delandistrogene moxeparvovec gene transfer therapy in non-ambulatory and ambulatory males with DMD. This is a randomized, double-blind, placebo-controlled 2-part study. Participants will be in the study for approximately 128 weeks. All participants will have the opportunity to receive intravenous (IV) delandistrogene moxeparvovec in either Part 1 or Part 2.

NCT ID: NCT05880160 Recruiting - Heart Failure Clinical Trials

Safety of Withdrawal of Pharmacological Treatment for Recovered HER2 Targeted Therapy Related Cardiac Dysfunction

HER-SAFE
Start date: July 14, 2023
Phase: N/A
Study type: Interventional

Breast cancer is the most common cancer in the United Kingdom (UK), but improvements in treatment mean 3 in 4 people survive for more than 10 years. Many people receive treatments called human epidermal growth factor receptor 2 (HER2) targeted therapies for their breast cancer, however these can affect heart function. This 'cardiotoxicity' is generally temporary and mild, but patients receive drugs to help their heart recover. Currently it is not known how long patients should receive these treatments. Patients with other types of heart failure are treated lifelong, but this may not be necessary here as the damaging cancer drugs have stopped. Taking drugs for many years can have an impact on people's quality of life, particularly for young patients. It is therefore important to understand the best treatment length. The investigators will study people whose heart function has recovered after HER2 therapy heart problems and are not at high risk for heart disease. The investigators will carefully stop their heart drugs whilst monitoring them closely with special heart scans and blood tests to detect problems early. The investigators will also study how patients are currently treated using national data. The results of this study will help doctors better guide breast cancer survivors about treatment of heart damage from HER2 cancer therapies.

NCT ID: NCT05878912 Recruiting - Obesity Clinical Trials

The Effect of Obesity and Weight Loss in Heart Failure With Preserved Ejection Fraction

OWL-HFPEF
Start date: January 20, 2023
Phase: N/A
Study type: Interventional

This study looks at the effects of weight loss in people who have heart failure with preserved ejection fraction (HFpEF) and are overweight or obese. The main questions it aims to answer are whether weight loss in this group of people improves: 1. The heart's shape, how well it pumps blood and how well it uses fuels 2. The person's quality of life and how much they can exercise Participants will attend 2 study visits, separated by 3-6 months. The intervention period takes place in between the 2 study visits. Each study visit will involve measurements including: - Symptom and quality of life questionnaires - Body measurements such as height and weight - Blood tests - Ultrasound scans of the heart (echocardiogram) - Magnetic Resonance Imaging (MRI) scans of the heart - Exercise components during the scans - 6 minute walk test Participants are randomly allocated (in other words, by lottery) to either 'diet' or 'control' groups. The control group will continue standard care. The diet group will be enrolled in a weight loss program supervised by the study team. Participants in the diet group will replace their usual meals with meal replacement products specifically designed to deliver a low calorie diet for weight loss. The products will consist of formula soups, shakes and porridges. This diet would last 8 weeks, followed by a guided period of food reintroduction and maintenance.

NCT ID: NCT05878847 Recruiting - Osteo-arthritis Clinical Trials

Oral Supplement in Older Adults to Support Physical Fitness and Mental Well-being

PQQA
Start date: April 22, 2023
Phase: Phase 1
Study type: Interventional

Cucumbers have been anecdotally claimed to have anti-inflammatory activity for a long time, but the active principle was not identified. idoBR1 is an iminosugar amino acid isolated from fruits of certain cucumbers, Cucumis sativus (Cucurbitaceae), which has been shown to have anti-inflammatory activity. IminoTech Inc in the USA has produced a quality-controlled cucumber extract containing measured idoBR1 (Q-actin™) that has given good results in osteo-arthritis from oral use. The investigators are aiming to recruit a cohort (n = 50) of middle aged and older adults (>50 years) who will be randomised into Q-actin (2 x 10 mg gummies daily) or placebo (2 x 10 mg gummies daily) supplementation for 12 weeks. Using hand grip strength and the Nine-Hole Peg Test (9HPT) the investigators will measure physical strength and finger dexterity respectively. The investigators will measure generic quality of life with the EuroQol 5 Dimension 5 (EQ-5D) questionnaire, sleep quality, diet choices and collect urine samples for the investigation into the chemical composition (metabolomics). Randomisation will be blinded, and the participant and the researcher will not know what group participants in until after the completion of the study, then it will be disclosed.

NCT ID: NCT05878769 Recruiting - Clinical trials for Chronic Obstructive Pulmonary Disease

A Study to Evaluate the Long-Term Safety of Astegolimab in Participants With Chronic Obstructive Pulmonary Disease (COPD)

Start date: June 28, 2023
Phase: Phase 3
Study type: Interventional

The purpose of this study is to assess the long-term safety and to explore the efficacy of astegolimab in participants with chronic obstructive pulmonary disease (COPD) who have completed the 52-week placebo-controlled treatment period in parent studies GB43311 or GB44332.

NCT ID: NCT05878717 Recruiting - Clinical trials for Scleroderma, Systemic

A Study of the Efficacy and Safety of Belimumab in Adults With Systemic Sclerosis Associated Interstitial Lung Disease

BLISSc-ILD
Start date: September 13, 2023
Phase: Phase 3
Study type: Interventional

This study investigates the efficacy and safety of belimumab compared to placebo, in addition to standard therapy, for the treatment of participants with systemic sclerosis associated interstitial lung disease (SSc-ILD). The study will evaluate the effect of belimumab treatment on lung function as well as on extra-pulmonary disease manifestations, including skin thickening and general symptoms, such as fatigue, that impact quality of life (QoL).

NCT ID: NCT05877547 Recruiting - Clinical trials for Non-alcoholic Fatty Liver Disease

A Clinical Study of Efinopegdutide in Participants With Precirrhotic Nonalcoholic Steatohepatitis (NASH) (MK-6024-013)

Start date: June 23, 2023
Phase: Phase 2
Study type: Interventional

The purpose of this study is to learn how well efinopegdutide works compared to placebo in people who have non-alcoholic steatohepatitis (NASH). Researchers will also learn about the safety and benefit of efinopegdutide and how well people tolerate the medicine. The main goal of the study is to compare how many people taking efinopegdutide or placebo stop showing evidence of NASH without liver scarring getting worse.

NCT ID: NCT05877157 Recruiting - Quality of Life Clinical Trials

Pain AND Opioids After Surgery

PANDOS
Start date: September 25, 2023
Phase:
Study type: Observational

The aim of this study is to document the perioperative opioid use and its safety in the UK and countries in Europe and to describe its association with surgical complications, persistent pain and quality of life. In an international, prospective, observational cohort study, data about the perioperative pain management will be collected in all eligible hospitalised adult patients who undergo surgery in a designated "study week" in as many hospitals as possible in Europe. Baseline data will be collected, and participants followed up at one week, and at three and twelve months post-operatively. The primary outcome will be opioid use at three months after surgery. Secondary outcomes will include opioid use during the preoperative month (preoperative use), during surgery and up to one week after surgery (or discharge, whichever is earlier), and up to the end of the postoperative year. Additional secondary outcomes are the incidence of preoperative pain, persistent pain with/without the presence of neuropathic components, quality of life and surgical complications. The potential association between opioid use and pain outcomes will be investigated. This study could help to develop strategies to improve quality of care, through pain management, for patients undergoing surgery.