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NCT ID: NCT04860960 Active, not recruiting - Clinical trials for Niemann-Pick Disease, Type C1

Phase 3 Study to Evaluate Intravenous Trappsol(R) Cyclo(TM) in Pediatric and Adult Patients With Niemann-Pick Disease Type C1

TransportNPC
Start date: July 20, 2021
Phase: Phase 3
Study type: Interventional

A prospective, randomized, double-blind, placebo controlled, multi-center therapeutic study for patients age 3 and older with confirmed diagnosis of Niemann Pick disease type C1 (NPC1). The objective of this study is to evaluate the safety, tolerability and efficacy of 2000 mg/kg dose of Trappsol Cyclo (hydroxypropyl betacyclodextrin) administered intravenously compared to standard of care. An open-label sub-study in countries following European Medicines Agency (EMA) guidance will enroll asymptomatic or symptomatic patients from infancy up to age 3 to evaluate safety in that population.

NCT ID: NCT04860830 Active, not recruiting - Schizophrenia Clinical Trials

CONNEX-3: A Study to Test Whether Iclepertin Improves Learning and Memory in People With Schizophrenia

Start date: June 18, 2021
Phase: Phase 3
Study type: Interventional

This study is open to adults with schizophrenia. Schizophrenia can affect the way a person thinks, their memory and their mental functioning. Examples include struggling to remember things, or to read a book or pay attention to a movie. Some people have difficulty calculating the right change or planning a trip so that they arrive on time. The purpose of this study is to find out whether a medicine called iclepertin improves learning and memory in people with schizophrenia. Participants are put into two groups randomly, which means by chance. One group takes iclepertin tablets and the other group takes placebo tablets. Placebo tablets look like iclepertin tablets but do not contain any medicine. Participants take a tablet once a day for 26 weeks. In addition, all participants take their normal medication for schizophrenia. During this time, doctors regularly test learning and memory of the participants by use of questionnaires, interviews, and computer tests. The results of the mental ability tests are compared between the groups. Participants are in the study for about 8 months and visit the study site about 14 times. During this time, doctors regularly check participants' health and take note of any unwanted effects.

NCT ID: NCT04860752 Active, not recruiting - Clinical trials for Aortic Valve Stenosis

SAPIEN 3 Ultra EU PMS

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

This study will evaluate real-world outcomes for the SAPIEN 3 Ultra Transcatheter Heart Valve System in transcatheter aortic valve implantation centres that are implementing minimalist periprocedural practices and facilitating early discharge home.

NCT ID: NCT04860011 Active, not recruiting - Heart Failure Clinical Trials

DAPAgliflozin Versus Thiazide Diuretic in Patients With Heart Failure and Diuretic RESISTance

DAPARESIST
Start date: April 27, 2021
Phase: Phase 3
Study type: Interventional

To assess the effect of dapagliflozin compared with metolazone, added to furosemide, on diuresis and decongestion in hospitalised heart failure patients with diuretic resistance, and renal impairment. The primary analysis will be in patients with HFrEF but patients with HFpEF will also be recruited in an ancillary study and included in supplementary analyses.

NCT ID: NCT04857476 Active, not recruiting - Psoriatic Arthritis Clinical Trials

Do Patients With Skin Psoriasis Have Sub-clinical Features of Active Psoriatic Arthritis on Musculoskeletal UltraSound?

PsACT-US
Start date: June 8, 2021
Phase: N/A
Study type: Interventional

Psoriatic Arthritis is an inflammatory condition that typically affects joints and soft tissues such as tendons. Poorly controlled or untreated psoriatic arthritis can lead to joint damage, disability and poor physical and mental wellbeing. Evidence suggests that early diagnosis and treatment of psoriatic arthritis can minimise adverse health outcomes. Musculoskeletal ultrasound has become an extremely useful tool in aiding rheumatologists to diagnose inflammatory joint conditions particularly at an early stage in the course of a disease. Psoriatic arthritis is known to affect up to 30% of patients with skin psoriasis. Therefore, national dermatology guidelines advise that patients with skin psoriasis should be asked about any joint symptoms at least every year. This study investigates whether skin psoriasis patients who are not on biologic treatment are indeed being asked about any joint symptoms and we subsequently invite patients for a musculoskeletal ultrasound scan to see if they have features of early psoriatic arthritis on ultrasound. Those who do are then invited for a thorough rheumatology clinical assessment.

NCT ID: NCT04857229 Active, not recruiting - Breast Cancer Clinical Trials

Histolog Comparison to Standard Histology

HiCoSH
Start date: June 4, 2021
Phase:
Study type: Observational

This is a clinical device trial to assess the accuracy of margin assessment for a confocal scanning device (Histolog Scanner) at assessing the margins of breast tissue following wide local excision surgery.

NCT ID: NCT04854499 Active, not recruiting - Clinical trials for Head and Neck Squamous Cell Carcinoma

Study of Magrolimab Combination Therapy in Patients With Head and Neck Squamous Cell Carcinoma

ELEVATE HNSCC
Start date: September 7, 2021
Phase: Phase 2
Study type: Interventional

The goals of this clinical study are to learn about the safety, tolerability, dosing and effectiveness of the study drug, magrolimab in combination with other anticancer therapies in patients with head and neck squamous cell carcinoma (HNSCC).

NCT ID: NCT04854070 Active, not recruiting - Clinical trials for Complex Coronary Lesions

Intravascular Ultrasound Guidance for Complex High-risk Indicated Procedures

IVUS-CHIP
Start date: November 2, 2021
Phase: N/A
Study type: Interventional

The IVUS CHIP trial is a post-marketing strategy study in which patients with complex coronary lesions, undergoing percutaneous coronary intervention (PCI), are treated either with intravascular ultrasound (IVUS) guided PCI or angiographic guided PCI . The IVUS-guided PCI approach is indicated to reduce the frequency of target-lesion failure (cardiac death, target-vessel myocardial infarction, and clinically indicated target-lesion revascularization) in patients with complex coronary lesions undergoing PCI. The objective of this study is to assess the superiority of an IVUS-guided approach versus an angio-guided approach in patients with complex coronary lesions undergoing PCI.

NCT ID: NCT04853420 Active, not recruiting - Cancer Clinical Trials

Minimal Residual Disease: A Trial Using Liquid Biopsies in Solid Malignancies.

MARTINI
Start date: February 16, 2021
Phase:
Study type: Observational

The NHS' Genomic Medicine Service offers whole genome sequencing as part of the drive to improve cancer outcomes. It is recognised that actionable mutations (current and emerging), will ultimately improve outcomes across multiple disease sites by identifying which treatments may benefit individual patients the most, and by providing earlier and more accurate diagnoses. However, testing in the cancer setting is currently limited to haematological malignancies and sarcoma. The majority of patients with solid tumours do not yet have access to this platform and the benefits that it may bring. Therefore, expanding genomic testing capacity within a research setting has potential to benefit those patients that would otherwise not be able to access testing. In this study we will be using tissue derived from patients undergoing surgery for cancer to validate an in-house genomic testing platform against Roche's Foundation Medicine genomic profile service, which is an FDA- approved commercial platform. In addition, two blood samples will be taken in order that we can test whether markers present in the tissue may also be seen in blood. We hope that this will help us monitor minimal residual disease in patients, allowing earlier detection of relapse/recurrence than radiology currently allows. Patients may also agree to donate optional excess fresh tissue from their surgery. This will be integrated with other laboratory platforms which may offer information on prospective drug response based on genotypic profiles (e.g., patient-derived explants).

NCT ID: NCT04852965 Active, not recruiting - Clinical trials for Anthracycline Induced Cardiotoxicity

Late Anthracycline Induced Cardiotoxicity- Childhood Cancer Survivors

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

Anthracyclines treat up to 60% of childhood malignancies with remarkable improvements survival rates. Unfortunately anthracyclines are associated with an increased cardiomyopathy risk. One study showed an almost six-fold greater risk of developing cardiomyopathy compared to sibling controls. A retrospective pilot study showed evidence of subclinical dysfunction (including impaired global longitudinal strain) in 42/52 childhood cancer survivors. There is limited research in this area, therefore current guidelines are based on expert opinion alone and lack consensus. Current methods of detection diagnose cardiomyopathy at an irreversible stage i.e. when the compensatory mechanisms are exhausted and the left ventricular ejection fraction impaired. Small trials have shown that early treatment with standard heart failure therapy may reverse damage, further validation is however required in this cohort. Newer techniques such as tissue doppler and strain rate imaging have shown promise for early prediction of cardiomyopathy in adult studies. Biomarkers such as troponin and NT-proBNP have also shown a correlation with cardiomyopathy. This study (n=208) aims to use echocardiography, strain imaging, holter monitoring and MRI for early detection of cardiomyopathy. Biomarkers, both currently used (for example, troponin and NTproBNP,) and more novel (for example, IL6, MPO, and sST2) will be assessed to see if early cardiomyopathy can be predicted. This study will explore biomarker discovery by analysing an age/gender matched subgroup for the top differentially expressed microRNA and protein biomarkers. Selected biomarkers will then be validated in a larger cohort.