Salford

Researchers are investigating the safety and tolerability of an investigational drug called PGN-EDODM1 in adults with myotonic dystrophy type 1 (DM1), a genetic condition characterized by muscle weakness and myotonia. This Phase 2 study compares multiple doses of PGN-EDODM1 to a placebo, aiming to understand how well the drug is tolerated and its potential effects in people with this condition. Participants will receive PGN-EDODM1 or placebo through intravenous (IV) infusions. The study is randomized, double-blind, and placebo-controlled, with multiple ascending dosing to evaluate safety across different dose levels. Both treatments are administered by IV infusion, and the study includes adult participants aged 16 to 65 years with confirmed DM1. During the study, researchers will monitor participants closely for any adverse events from the start of treatment through Day 112 to assess safety and tolerability. This includes physical exams, muscle biopsies, laboratory tests, and other clinical assessments to track participant health and response to treatment. The total duration of participation covers the dosing period and follow-up assessments to ensure comprehensive safety monitoring.

Researchers are studying the dose-response effects of galvokimig compared with a placebo in adults with moderate-to-severe atopic dermatitis, a chronic skin condition lasting at least one year. The study focuses on adults aged 18 years and older who have significant disease activity as measured by specific clinical scores and a history of inadequate response to topical treatments or contraindications to them. This phase 2 trial aims to evaluate the safety, effectiveness, and how the drug behaves in the body. Participants will receive either galvokimig or a placebo as an injection. The study uses a randomized, double-blind, placebo-controlled design with multiple doses tested in parallel groups. Treatments are given as solutions for injection, and the study monitors participants over a defined period to assess how the drug works and its safety profile. During the study, participants will undergo assessments including clinical scoring of their skin condition such as the Eczema Area and Severity Index at week 16 to measure response. Researchers will also monitor safety through physical exams, laboratory tests, and medical history reviews. The study requires stopping other systemic or topical treatments before starting and tracks participant adherence and outcomes carefully throughout the study duration.

Researchers are conducting a global, multicenter, prospective observational registry to study patients with Pompe disease, including those with late-onset Pompe disease (LOPD) and infantile-onset Pompe disease (IOPD). The study includes both patients who are untreated and those receiving approved Pompe disease therapies. The main goals are to assess the long-term safety and real-world effectiveness of these treatments, understand their impact on quality of life and patient-reported outcomes, and describe the natural history of untreated Pompe disease. Participants may be treated with various therapies including enzyme replacement therapies such as cipaglucosidase alfa delivered by intravenous infusion, alglucosidase alfa or avalglucosidase alfa once approved locally, and miglustat co-administered with ATB200. Patients not receiving any medical therapy for Pompe disease are also included. The study gathers data from both treated and untreated patients as they are managed in routine clinical practice. Throughout the study, participant data will be collected to monitor the frequency of adverse events and serious adverse events over a period of five years. Researchers will also evaluate treatment effectiveness, quality of life, and patient-reported outcomes during this time. This observational approach allows for long-term safety monitoring and understanding of Pompe disease progression in a real-world setting.

Researchers are conducting a multi-center, multi-country observational study to evaluate the safety and effectiveness of pegunigalsidase alfa (Elfabrio®) in people with Fabry disease. This hybrid study includes both retrospective and prospective data collection from participants treated in routine clinical care. The study focuses on adults with genetically confirmed Fabry disease who are either currently receiving or planning to receive pegunigalsidase alfa. Participants receive pegunigalsidase alfa through intravenous infusion as part of their standard care. The study includes different participant groups such as a cardiac cohort with evidence of Fabry-related heart disease, a naïve cohort with no prior Fabry therapy, and a long-term cohort previously enrolled in a related open-label study. Cardiac magnetic resonance imaging (cMRI) and other assessments are part of standard care and are used to monitor participants. During the 4-year study period, researchers will assess kidney function through estimated glomerular filtration rate (eGFR), measure plasma globotriaosylsphingosine (LysoGb3) levels, evaluate heart structure with left ventricular mass index (LVMI), and monitor heart injury markers like high sensitivity troponin (hs-cTnT). Safety assessments will be conducted throughout. Participants provide informed consent and complete electronic patient-reported outcomes to help understand treatment effects and safety over time.

This research aims to study the safety, tolerability, pharmacokinetics, and pharmacodynamics of the drug VX-670 in adults who have Myotonic Dystrophy Type 1 (DM1). The trial involves participants aged 18 to 64 years with a confirmed diagnosis of DM1, including a genetic test showing a specific CTG repeat count. The study is a Phase 1/2 trial designed to assess how the drug behaves and how well it is tolerated in this population. Participants will receive VX-670 or a placebo, both administered intravenously, in single and multiple dose escalations. The study is randomized, double-blind, and placebo-controlled to compare the effects of the drug against a non-active treatment. The treatment periods include initial dosing and extended follow-up to evaluate responses over time. During the study, researchers will monitor participants closely for any adverse events from the start up to 42 days in the initial phase and up to 168 days in the extended phase. Safety and tolerability will be the main focus, alongside collecting data on how the drug is processed by the body and its biological effects. Participants will undergo assessments to track these outcomes throughout their involvement in the trial.

Researchers are investigating the effectiveness, safety, and tolerability of combining baxdrostat with dapagliflozin compared to dapagliflozin alone in people with chronic kidney disease (CKD) and high blood pressure. This Phase III, international, multicenter, double-blind, placebo-controlled study aims to see if this combination reduces risks such as significant kidney function decline, kidney failure, heart failure events, or cardiovascular death. The study includes a 4-week run-in period where participants not previously treated with SGLT2 inhibitors receive dapagliflozin alone. After this, participants are randomly assigned to receive either baxdrostat plus dapagliflozin or placebo plus dapagliflozin in a double-blinded manner. Study visits occur frequently initially (at 2, 4, 8, 16, 34, and 52 weeks after randomization) and then approximately every 4 months. If participants stop the blinded treatment early, they continue dapagliflozin alone unless specific criteria require its discontinuation. Participants will undergo regular assessments including blood pressure monitoring and laboratory tests related to kidney function and cardiovascular health. The primary outcome measures the reduction in risk of major kidney and heart events over up to 37 months. Even if participants stop the study treatment, they will continue follow-up visits and data collection to ensure comprehensive safety and efficacy evaluation throughout the study duration.

Researchers are investigating the long-term safety and tolerability of open-label iptacopan in adults with primary IgA nephropathy who have previously completed specific clinical trials (CLNP023X2203 or CLNP023A2301). This extension study is designed to allow participants continued access to iptacopan until certain conditions are met, such as reaching three years from the last patient first visit, loss of treatment benefit, negative benefit-risk profile, initiation of dialysis or kidney transplant, or commercial availability of the drug. The study will also assess the drug's effects on disease progression every six months. Participants who completed the prior trials and meet inclusion criteria may receive oral iptacopan capsules at a dose of 200 mg twice daily. The study is open-label and non-randomized and will continue treatment under this regimen until one of the study-defined stopping points is reached. Supportive care with ACE inhibitors or ARBs is maintained as per clinical guidelines, and vaccination against certain infections is required before enrollment. During the study, participants will be monitored for safety, including serious adverse events, adverse events of special interest, vital sign abnormalities, ECG changes, and laboratory test abnormalities from the first day of treatment until seven days after the last dose. Efficacy assessments occur every six months to evaluate clinical effects on disease progression. The study aims to collect long-term safety and tolerability data while providing ongoing treatment access until the drug becomes commercially available or other stopping criteria apply.

Researchers are evaluating the safety and effectiveness of brenipatide compared to a placebo for adults with moderate-to-severe Alcohol Use Disorder (AUD). This phase 3 study aims to better understand if brenipatide can help reduce drinking in this population. Participants will be followed for about 56 weeks to gather comprehensive information. Participants will receive either brenipatide (LY3537031) or a placebo, both given by subcutaneous injection. The study involves a randomized, double-blind design, meaning neither the participants nor the researchers know who receives which treatment during the trial. This method helps provide reliable results about the effects and safety of brenipatide. During the study, participants will attend scheduled visits, self-inject the study drug, and complete electronic and paper diaries as well as questionnaires. Researchers will monitor changes in drinking patterns using the Timeline Followback Method for up to 56 weeks. Safety monitoring and regular assessments will be performed throughout the study to track participants' health and adherence.

Researchers are evaluating the safety and effectiveness of brenipatide compared to placebo for people with opioid use disorder. This study focuses on participants who are also using buprenorphine, with or without naloxone, as part of their treatment. The trial includes two parts, each with separate groups of participants, to better understand how brenipatide works alongside current therapies in early recovery from opioid use disorder. The study has two parts: Part A involves a double-blind treatment phase followed by an open-label extension, while Part B offers an open-label treatment only. Brenipatide and placebo are given as subcutaneous injections, and buprenorphine is administered either sublingually or buccally. Participants will be enrolled in only one part of the study, with treatment durations potentially lasting up to 144 weeks in Part A and 116 weeks in Part B, depending on enrollment timing and study progress. Participants will regularly attend study visits where they will be assessed through urine drug screens and self-reports to measure abstinence from opioid use. They will also maintain study diaries and complete questionnaires to track adherence and effects. The main outcomes measured include the percentage of weeks participants remain abstinent from opioids between weeks 13 and 24, verified by negative drug tests and no self-reported opioid use. Safety and long-term effectiveness will be monitored throughout the study duration.

Researchers are evaluating how well LY4005130 works in adults with severe alopecia areata, a condition causing significant hair loss. This Phase 2 study compares LY4005130 with a placebo to assess its effectiveness, safety, and side effects. Blood tests will be conducted to understand how the body processes the drug and how the drug affects the body. The study drug, LY4005130, and placebo are both given intravenously into a vein in the arm. The treatment period includes administration of these study drugs under controlled conditions. The study lasts about 48 weeks in total, which includes a screening period before treatment. Participants will be involved in various assessments such as blood tests and evaluations of hair loss severity using the Severity of Alopecia Tool (SALT). The main outcome measured is the percentage of participants who achieve a SALT score of 20 or less by week 24. Safety and tolerability will be monitored throughout the study, with follow-up visits scheduled during the 48-week period.