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NCT ID: NCT06082999 Recruiting - Epilepsy Clinical Trials

Gene-STEPS: Shortening Time of Evaluation in Paediatric Epilepsy Services

Gene-STEPS
Start date: September 1, 2021
Phase:
Study type: Observational

Overall, this observational cohort study aims too: 1. Implement rapid trio WGS for all children presenting to our health systems with epilepsy onset under 12 months of age. 2. Utilize electronic healthcare records and research databases to unite phenotypic and genomic data and to create a "virtual" registry across all institutions that will promote ongoing discovery. 3. Assess the impact of early genetic diagnosis on epilepsy, developmental, and health economic outcomes through formal longitudinal assessments of all children enrolled.

NCT ID: NCT06081452 Recruiting - Cancer Clinical Trials

Public Survey on Breath Testing for Health and Disease

Start date: June 17, 2023
Phase:
Study type: Observational

A brief survey for the public to determine interest in health surveillance and disease detection

NCT ID: NCT06079372 Recruiting - Hypophosphatasia Clinical Trials

Phase 3 Study of ALXN1850 in Pediatric Participants With HPP Previously Treated With Asfotase Alfa

CHESTNUT
Start date: April 2, 2024
Phase: Phase 3
Study type: Interventional

The primary purpose of this study is to assess the safety and tolerability of ALXN1850 versus asfotase alfa in pediatric participants with HPP previously treated with asfotase alfa.

NCT ID: NCT06079359 Recruiting - Hypophosphatasia Clinical Trials

Phase 3 Study of ALXN1850 in Treatment-Naïve Pediatric Participants With HPP

MULBERRY
Start date: May 14, 2024
Phase: Phase 3
Study type: Interventional

The primary purpose of this study is to evaluate the efficacy of ALXN1850 versus placebo on radiographic outcomes in pediatric participants with HPP who have not previously been treated with asfotase alfa.

NCT ID: NCT06079281 Recruiting - Hypophosphatasia Clinical Trials

Phase 3 Study of ALXN1850 Versus Placebo in Adolescent and Adult Participants With HPP Who Have Not Previously Been Treated With Asfotase Alfa

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

The primary objective of this study is to assess the efficacy of ALXN1850 versus placebo on functional outcomes in adolescent and adult participants with HPP who have not previously been treated with asfotase alfa.

NCT ID: NCT06079190 Recruiting - Alzheimer's Disease Clinical Trials

Efficacy and Safety of GSK4527226 [AL101] in Participants With Early Alzheimer's Disease

PROGRESS-AD
Start date: October 20, 2023
Phase: Phase 2
Study type: Interventional

The aim of this study is to assess the efficacy and safety of GSK4527226 in participants with early Alzheimer's Disease (AD) (including mild cognitive impairment [MCI] and mild dementia due to AD) of 2 dose levels of GSK4527226 compared to placebo.

NCT ID: NCT06078553 Recruiting - Clinical trials for Congenital Myasthenic Syndrome

A Natural History Study in Participants With DOK7 Congenital Myasthenic Syndromes (CMS)

Start date: February 13, 2024
Phase:
Study type: Observational

Participants will attend up to 3 study visits to collect clinical assessments. The assessments will evaluate participants' symptoms and quality of life to understand disease activity in patients with DOK7-CMS better and may inform future study design.

NCT ID: NCT06078020 Recruiting - Cerebral Hemorrhage Clinical Trials

PLatform Study for INTracerebral Haemorrhage (PLINTH): Community-based Feasibility Study

PLINTH
Start date: October 1, 2023
Phase:
Study type: Observational

The goal of this observational study is to answer three uncertainties about the design of a platform study for adults with stroke due to intracerebral haemorrhage. The main things the investigators aim to find out are: - The acceptability of a platform trial and its comparisons to brain haemorrhage survivors or their carers, and their clinicians. - Estimates of eligibility, willingness to participate, diversity, representativeness, adherence, retention, data completion, and event rates of intermediate and clinical outcomes over time for each comparison. - The most efficient platform trial design considering the findings of this feasibility study. Participants, or their welfare guardian, welfare attorney, or nearest relative will: - Watch a video informing them about the study - Read written information about the study - Provide informed consent - Permit collection of demographic and clinical details - Provide information in interviews at ~3 and ~14 days after the onset of the stroke

NCT ID: NCT06077864 Recruiting - Obesity Clinical Trials

A Study to Test the Effect of Survodutide (BI 456906) on Cardiovascular Safety in People With Overweight or Obesity (SYNCHRONIZE™ - CVOT)

Start date: November 17, 2023
Phase: Phase 3
Study type: Interventional

This study is open to adults who are at least 18 years old and have a body mass index (BMI)bof 27 kg/m2 or more. People can take part if they have cardiovascular or chronic kidney disease. People who have at least 2 health problems related to their weight or risks of cardiovascular disease can participate. Participants must have previously tried to lose weight by changing their diet. The purpose of this study is to find out whether people with overweight or obesity who take a medicine called survodutide (BI 456906) are less or more likely to develop serious cardiovascular problems. It also aims to find out whether health parameters like blood pressure improve. Overweight and obesity are linked to cardiovascular disease. Survodutide is a medicine that is developed to help people with obesity or overweight to lose weight. Participants are divided into 3 groups of almost equal size. 2 groups get different doses of survodutide and 1 group gets placebo. Placebo looks like survodutide but does not contain any medicine. Every participant has a 2 in 3 chance of getting survodutide. Participants inject survodutide or placebo under the skin once a week. All participants also receive counselling on diet and physical activity. Participants are in the study for up to 2 years and 3 months. During this time, it is planned that participants visit the study site up to 21 times and attend remote visits by video calls. During these visits, the doctors check participants' cardiovascular and overall health. The results are compared between survodutide and placebo groups. The study staff also takes note of any unwanted effects.

NCT ID: NCT06076473 Recruiting - Clinical trials for Primary Aldosteronism

Untargeted Metabolomics for Primary Aldosteronism

PUMPAP
Start date: January 19, 2023
Phase:
Study type: Observational

Primary aldosteronism (PA) is a common and likely under-diagnosed cause of secondary hypertension with associated cardiovascular morbidity and mortality. Current diagnosis comprises screening, confirmatory testing and sub-type classification (lateralisation) to distinguish unilateral disease (requiring surgery) from bilateral disease (requiring medical management). This multi-step process is complex and variable with a lack of uniformity in diagnostic protocols, standardised/reference assay methodologies, and diagnostic thresholds. There is evidence in the literature that targeted serum steroid panels may have a role in diagnosis of PA, and both targeted steroid panels and untargeted metabolomics in serum and urine are a promising area of research. This study aims to identify and recruit participants (n=40; 20 with confirmed PA and 20 with other causes of hypertension) willing to donate lithium heparin plasma for a metabolomics pilot study. This plasma will be interrogated through untargeted metabolomics using gas/liquid chromatography-mass-spectrometric methods and computational data processing to allow power calculations and inform experimental design for future studies. The utility of metabolites from the metabolomics dataset will be evaluated by comparison against current biomarkers for screening, diagnosis and lateralisation as well as radiology and histology acquired through routine diagnostic work-up. The long-term aim for larger studies is to identify suitable candidate analytes in plasma for future development into targeted, clinically-useful analyte panels.