Salford

Researchers are evaluating the effectiveness of delgocitinib cream 20 mgg applied twice daily compared to a cream vehicle in treating adults with mild to severe palmoplantar pustulosis PPP. This trial is a phase 2a, double-blind study focusing on this skin condition characterized by pustules on the palms and soles. The main goal is to see how well delgocitinib cream improves symptoms over a 16-week treatment period. Participants will be randomly assigned to one of two groups one group will apply delgocitinib cream twice a day for 16 weeks, while the other group will use a matching vehicle cream twice daily for the same duration. The study includes a total of approximately 18 weeks for each participant, involving around 9 visits. The trial compares the active cream to the placebo cream to assess efficacy and safety. During the study, participants will attend scheduled visits to monitor their condition and collect data on skin symptoms, pustule counts, and quality of life measures. Researchers will assess the improvement in PPP severity using specific scales such as the PPP Area and Severity Index PPPASI. Safety is monitored through reports of any treatment-emergent side effects. Overall participation lasts about 18 weeks, including screening and treatment phases.

Researchers are evaluating the effects of an investigational medicine called PGN-EDODM1 to understand how safe and tolerable multiple doses are for adults with myotonic dystrophy type 1 DM1. This phase 2 study compares PGN-EDODM1 to a placebo to assess its safety and tolerability in people with this condition. Participants will be randomly assigned to receive either PGN-EDODM1 or a placebo. The treatment is given by intravenous infusion once every 4 weeks for a total of 12 weeks. Those in the PGN-EDODM1 group will receive ascending doses, while the placebo group will receive saline infusions on the same schedule. During the study, participants will be monitored for safety by tracking adverse events from the start through Day 112. Researchers will measure drug levels in the blood, changes in muscle tissue, myotonia severity, hand grip strength, and mobility using specific tests. The total participation lasts through about 16 weeks, including treatment and follow-up assessments.

Researchers are studying the safety and effects of VHB937 in people with early Alzheimers disease, including those with Mild Cognitive Impairment due to Alzheimers or mild Alzheimers itself. This randomized, double-blind, placebo-controlled Phase II trial aims to evaluate whether VHB937 can benefit memory, thinking abilities, daily functioning, and brain changes. The study also looks at how the body processes VHB937 and responds to it. Participants receive intravenous infusions of either a low dose or high dose of VHB937, or a placebo, over a 72-week double-blind period. After this, an extension phase follows for further observation. The treatments are given through infusions, and participants are randomly assigned to one of the three groups in parallel. Throughout the study, participants and their study partners attend regular visits for assessments including clinical dementia rating scales, cognitive tests, daily living activities evaluation, and brain imaging biomarkers. Safety is monitored by tracking adverse events and serious adverse events. Blood samples are collected to measure VHB937 levels and immune responses. The total study duration includes the 72-week treatment period plus additional time in the extension phase.

Researchers are evaluating the dose-response relationship of galvokimig compared with placebo in adults with moderate-to-severe atopic dermatitis AtD. The study focuses on participants who have had chronic AtD for at least one year and aims to assess how different doses of galvokimig impact the condition. This phase 2 trial is designed to better understand the drugs effects on symptoms and safety in this population. Participants are randomly assigned to one of several groups receiving different predefined doses of galvokimig or a matching placebo during an initial 16-week intervention period. After week 16, participants continue treatment with the same or a modified dose of galvokimig. The study uses a double-blind design to compare the effects of these doses on atopic dermatitis. During the study, participants will undergo regular assessments including the Eczema Area and Severity Index EASI, Investigator Global Assessment vIGA, and Peak Pruritus Numerical Rating Scale PP-NRS. Safety is monitored through reported adverse events up to week 58. The primary outcome is the percentage of participants achieving a significant improvement in EASI score at week 16. The total study duration extends beyond 16 weeks to include ongoing safety and response evaluations.

Researchers are evaluating the gene therapy FLT201 in adults with Gaucher disease Type 1 who have been on stable enzyme replacement therapy ERT or substrate reduction therapy SRT for at least two years. This Phase 3, non-randomized, multicenter study aims to confirm the efficacy and safety of FLT201 after participants stop their current treatments. FLT201 is an advanced therapy designed to address this rare genetic condition by delivering a gene using a recombinant adeno-associated virus vector. Participants receive a single intravenous infusion of FLT201 during the study. This gene therapy uses a replication-incompetent viral vector to introduce the therapeutic gene. The study includes only one treatment group and does not involve placebo or comparison arms. The primary treatment period focuses on evaluating FLT201s impact over one year after stopping prior therapies. Throughout the study, participants undergo regular assessments to monitor their health and treatment effects. Researchers evaluate the therapys efficacy by measuring clinical outcomes at one year. Safety is closely observed to identify any adverse effects. The total study duration extends to 2032, allowing long-term follow-up of participants responses and overall well-being.

This research is a global, multicenter, prospective observational registry studying patients with Pompe disease, including both late-onset LOPD and infantile-onset IOPD forms. It enrolls both untreated patients and those receiving approved therapies to better understand the long-term safety, real-world effectiveness, and quality of life impacts of treatments for Pompe disease. The study also aims to describe the natural history of untreated Pompe disease. Participants include groups receiving different enzyme replacement therapies such as Cipaglucosidase alfa with Miglustat, other approved ERTs like Alglucosidase alfa or Avalglucosidase alfa, as well as untreated individuals who are not on any medical therapy for Pompe disease. No experimental treatments are given as this is an observational study tracking real-world treatment use and outcomes. During the study, participant data on adverse events, treatment effectiveness, quality of life, and patient-reported outcomes will be collected over a period of at least five years. Researchers will monitor safety through the frequency of adverse events and serious adverse events. Participants health and treatment impacts will be regularly evaluated to provide long-term insights into Pompe disease management and outcomes.

Researchers are conducting an observational study to better understand fucosidosis, a rare disease, its symptoms, and how it changes over time. The study aims to gather detailed information about the diseases natural history and progression, including patients who have or have not undergone stem cell transplant. There is currently no approved treatment for fucosidosis, so this study focuses on observing the condition without introducing new medications. The study is divided into two parts Part A involves collecting past medical data from up to 57 participants, and Part B involves following up with up to 31 participants prospectively, many of whom may also be in Part A. Participants will continue to receive their usual medical care throughout the study, with no additional treatments or interventions provided by the study team. Participants will provide medical history and health information that will be reviewed over time. The main focus is to track disease progression in individuals untreated by investigational products for up to four years. This includes gathering retrospective data and ongoing prospective observations to aid future research and potential treatment development. The study is expected to run until January 2031.

Researchers are conducting a large, multi-country observational study to assess the safety and effectiveness of pegunigalsidase alfa Elfabrio in patients with Fabry disease. This study includes patients who are currently treated with pegunigalsidase alfa in routine care and gathers data both retrospectively and prospectively. The study considers different patient groups such as those with Fabry-related heart disease, treatment-nave patients, and those transitioning from an earlier open-label extension study. The treatment being observed is pegunigalsidase alfa, administered through intravenous infusion as part of routine clinical care. Participants are grouped into cohorts based on their disease status and treatment history, including a cardiac cohort with Fabry-related heart involvement, a nave cohort who have not received prior Fabry treatment, and a long-term cohort of patients continuing from a previous clinical study. The study does not intervene in treatment but observes patients receiving this drug in real-world settings. Participants will be followed for up to four years, during which researchers will collect various clinical measurements such as kidney function estimated glomerular filtration rate, plasma lysoGb3 levels, heart structure and function via left ventricular mass index and cardiac biomarkers, and overall safety assessments. Data will be collected through routine clinical visits, imaging studies, and patient-reported outcomes. The study aims to provide comprehensive long-term information on the effects and safety of pegunigalsidase alfa in everyday clinical practice.

Researchers are evaluating the safety, tolerability, and how the body processes and responds to VX-670 in adults with Myotonic Dystrophy Type 1 DM1. This study is a Phase 12 trial that compares different doses of VX-670 to placebo in people diagnosed with DM1 who have a confirmed genetic test showing a specific repeat in their DNA. Participants will be randomly assigned to receive either single or multiple doses of VX-670 or matching placebo. The study has two parts Part A focuses on single ascending doses, while Part B includes both single and multiple ascending doses. The dosing levels in Part B will be based on results from Part A. Both VX-670 and placebo are given as intravenous solutions. During the study, participants will be monitored for adverse events up to 42 days in Part A and 168 days in Part B to assess safety and tolerability. Researchers will measure drug concentrations in blood plasma and muscle at various time points, including baseline, days 15, and 120. Muscle biopsies will be used to analyze changes in gene splicing. The total participation duration varies by study part, with detailed monitoring of drug effects and safety throughout.

Researchers are evaluating the pharmacodynamic effect, safety, and tolerability of Povetacicept in adults with generalized myasthenia gravis gMG. This phase 2 study focuses on participants aged 18 to 80 years who have generalized muscle weakness and fall within the MGFA clinical classification II-IV. The study aims to better understand how Povetacicept affects immunoglobulin levels and the safety profile in this population. Participants will be randomly assigned to receive one of two doses of Povetacicept or a placebo via subcutaneous injection for the initial 12 weeks. Those who are eligible may continue treatment with Povetacicept for an additional 96 weeks in a long-term extension phase. This study uses a double-blind, placebo-controlled design to evaluate the treatment over a total period of up to 108 weeks. During the study, participants will undergo evaluations of immunoglobulin G levels at baseline and week 12. Safety and tolerability will be monitored throughout the entire treatment period by tracking adverse and serious adverse events. The study includes regular assessments to measure outcomes and monitor participant health, with the goal of completing by March 2029.