Merthyr Tydfil

Researchers are evaluating SYX-5219 in a multi-part, first-in-human Phase 1 study involving healthy volunteers and people with moderate to severe atopic dermatitis AD. The study aims to determine the safety, tolerability, and levels of SYX-5219 in blood and urine when given in different dosing schedules. This includes single ascending doses, multiple ascending doses, and multiple dose administrations in participants with AD. The study has three parts Part 1 involves single ascending doses and a food effect evaluation in healthy volunteers, with doses given once on Day 1 and again after a washout period. Part 2 involves multiple ascending doses given once or twice daily to healthy volunteers over a defined period. Part 3 enrolls participants with AD who receive SYX-5219 or placebo twice daily for up to 42 days. Each part includes randomization and placebo control to compare effects. Participants will undergo safety and exploratory efficacy assessments during and after treatment, including monitoring adverse events up to 10 days after dosing in Part 1, 14 days after the last dose in Part 2, and up to Day 56 in Part 3. Blood and urine samples are collected at multiple timepoints to measure SYX-5219 levels. The study includes follow-up visits to monitor participant health and drug effects for several weeks after treatment ends.

Researchers are evaluating the safety, tolerability, pharmacokinetics, and pharmacodynamics of increasing doses of WVE-007, a stereopure siRNA oligonucleotide, in adults living with overweight or obesity. The study includes two parts Part A focuses on single ascending doses in adults with overweight or obesity, and Part B involves repeated doses in two groups of adults with obesity who have pre-Type 2 diabetes or Type 2 diabetes. This Phase 12a study is randomized, double-blind, and placebo-controlled to carefully assess the investigational treatment.

Researchers are studying MTX325 as a potential treatment to modify the progression of Parkinsons Disease PD. This Phase 1 trial includes healthy volunteers and patients with mild to moderate, early-stage Parkinsons Disease. The study aims to evaluate safety, tolerability, pharmacokinetics, brain biodistribution, and the effect of food on the drug. Parts 1 to 4 of the study, involving single and multiple ascending doses as well as elderly participants and PET biodistribution, have been completed. Part 5, which assesses multiple doses in PD patients, is currently ongoing. Participants receive MTX325 in different doses as oral capsules or placebo capsules depending on the study part. In some parts, a radiolabelled MTX325 solution is used for PET imaging in healthy volunteers to assess central nervous system penetration and biodistribution. The study uses a randomized, parallel design with several parts examining single ascending doses, multiple ascending doses, elderly subjects, and patients with Parkinsons Disease. PET scans and MRI are used during screening and on dosing days to study brain biodistribution. During the trial, participants undergo safety and tolerability assessments up to 28 days after dosing. Screening includes MRI, PET scans, clinical evaluations, and laboratory tests such as HIV, hepatitis, and drug abuse screening. Participants are monitored for vital signs and adverse events, and cognitive assessments are performed to exclude dementia. The study involves scheduled visits for dosing, imaging, and follow-up to measure safety, drug distribution in the brain, and pharmacokinetics. Participation duration varies by study part, with some completed and others ongoing until late 2027.

Researchers are evaluating IMVT-1402 in a global, randomized, double-blind, placebo-controlled Phase 2b study for adults with Graves disease GD who remain hyperthyroid despite antithyroid drug ATD treatment. The study aims to assess the efficacy, safety, and tolerability of IMVT-1402 in this population. Participants will receive one of two doses of IMVT-1402 or a placebo for 26 weeks. The study includes two experimental groups with different doses of IMVT-1402 and a placebo comparator group. Treatments are given over the same 26-week period to evaluate their effects. During the study, participants will be monitored to see if they become euthyroid and are able to stop ATD by Week 26. Researchers will measure thyroid hormone levels such as triiodothyronine T3 and free thyroxine FT4 at various time points, including Weeks 2, 4, and 26. Safety and tolerability will also be assessed throughout the trial, which is expected to complete in May 2027.

This research aims to identify if a new biomarker related to blood clot risk, called endothelial derived extracellular vesicles, along with changes in blood clotting time, can predict which patients with a Transient Ischemic Attack TIA are most likely to have a stroke. The study focuses on TIA patients, as over 25% of ischemic stroke cases occur in people who previously had a TIA. Stroke is a leading cause of disability, and preventing strokes in high-risk patients is a national health priority. The study will follow 300 participants over 12 months, divided into four groups healthy individuals without stroke or TIA history people referred to TIA clinics but found not to have TIA patients confirmed with TIA who did not have a stroke within 12 months and patients with TIA who did have a stroke within 12 months. Researchers will monitor and compare changes in extracellular vesicle levels and prothrombin time in these groups to assess stroke risk. Participants will undergo blood tests to measure the biomarker and clotting time, with follow-up over three years to observe changes. The study collects clinical information and imaging results to confirm stroke or TIA diagnosis. Researchers will also track patient diet status and medication use. The main outcomes are changes in the extracellular vesicles and prothrombin time, which may help identify those at highest risk for stroke after TIA.