Huddinge

Researchers are evaluating the safety and effectiveness of rilzabrutinib compared to placebo in adults with active Immunoglobulin G4 Related Disease (IgG4-RD). This Phase 3, randomized, double-blind study aims to measure the time until the first IgG4-RD clinical disease flare during a 52-week treatment period. Additional goals include assessing disease control, flare-free rates, use of glucocorticoid rescue, and monitoring safety through adverse events, laboratory tests, and electrocardiograms. Participants will be randomly assigned to receive either oral rilzabrutinib tablets or placebo for 52 weeks. Glucocorticoids may be used as rescue medication if needed. The study includes a screening period lasting 4 to 6 weeks before treatment begins, followed by the 52-week double-blind treatment phase, and a 2-week follow-up after treatment. An optional open-label extension lasting up to 108 weeks is also available for participants. During the study, participants will attend 16 visits for assessments, which may include clinical evaluations, imaging tests such as CT, MRI, PET, or ultrasound to monitor disease activity, and laboratory tests. Researchers will track time to disease flare and collect data on flare-free rates, safety parameters, and medication use. Participants' vaccination status and contraceptive use will be monitored according to local guidelines, and overall study participation could last up to 60 weeks or longer if joining the extension phase.

Researchers are evaluating CPV-104, a new medicine designed to regulate the complement system, which can be overactive in rare kidney diseases like C3 glomerulopathy (C3G). This is the first time CPV-104 is being tested in people. The study includes both healthy adults and adult patients with C3G to assess safety, tolerability, how the body processes the medicine, and immune system reactions. The study is a phase 1, first-in-human trial with two parts: Part 1 involves healthy volunteers receiving a single dose, while Part 2 involves C3G patients receiving multiple doses. The trial has two parts: Part 1 (Single Ascending Dose with healthy volunteers) is double-blind, randomized, and placebo-controlled, where healthy adults receive a single intravenous (IV) dose of CPV-104 or placebo. Part 2 (Multiple Ascending Dose with C3G patients) is open-label and single-arm, with patients receiving four weekly IV doses of CPV-104. Safety data is regularly reviewed by a Safety Review Committee before advancing to higher doses or new groups. All doses are given by healthcare professionals. Participants will undergo close monitoring throughout the study, including checks for side effects, blood and urine tests, ECGs, vital signs, and blood samples to measure drug levels and antibodies. For C3G patients, kidney function will also be observed, although the main focus is safety rather than effectiveness. The study tracks serious and severe drug reactions up to Day 29 for healthy volunteers and Day 50 for C3G patients to ensure safety. The total study length varies by part and cohort.

This research aims to assess the effectiveness and cost-efficiency of a lifestyle intervention designed to improve lifestyle habits and reduce cardiometabolic risks in adults with obsessive-compulsive disorder (OCD). OCD is a common condition linked to higher risks of heart and metabolic diseases, partly due to unhealthy lifestyle behaviors like inactivity and poor diet. This is the first randomized controlled trial to study how a lifestyle program affects health and well-being in people with OCD. The study compares two groups: one receiving a 13-week group-based lifestyle intervention including an individual session to set personalized goals, 12 group sessions combining education on lifestyle habits and physical exercise, and digital homework assignments; the other group receiving one individual session with a clinical psychologist providing feedback and written advice on healthy lifestyle habits based on national guidelines. After the intervention, participants in the lifestyle group get access to a digital booster module to help maintain changes. Participants will be monitored for physical activity using an accelerometer at baseline, week 14, and at 3, 6, and 12 months afterward. The study will also evaluate changes in cardiometabolic risk factors, mental health, daily functioning, quality of life, and the cost-effectiveness of the intervention from a healthcare perspective. Inclusion involves assessments through interviews, clinical evaluations, and physiological measurements, ensuring participants meet criteria and can attend sessions consistently.

This is a long-term follow-up trial to the post-authorisation efficacy and safety (PAES) trial (trial 20-HMedIdeS-19). The trial will include patients who participated in the PAES trial and were transplanted with a new kidney after treatment with imlifidase (trial drug) or standard of care medication. Imlifidase is a medicine used to prevent the body from rejecting a newly transplanted kidney and is used before transplantation in adults who have antibodies against the donor kidney and are considered 'highly sensitised' based on a positive crossmatch test. The purpose of this follow-up trial is to fulfil requirements from the European Medicines Agency (EMA) to continue to evaluate efficacy (kidney function) and safety (side effects) over time, for the patients who were transplanted with a new kidney in the PAES trial. The patients will be followed for up to 5 years after transplantation in the PAES trial to collect valuable long-term data.

Researchers are evaluating whether the drugs retatrutide and tirzepatide can prevent major adverse liver outcomes (MALO) in adults with metabolic dysfunction-associated steatotic liver disease (MASLD) who are at high risk. This Phase 3 trial enrolls about 4,500 adults with MASLD identified by non-invasive tests indicating an increased likelihood of developing serious liver problems. The study aims to understand how these treatments might affect liver health over time compared to a placebo. Participants will be randomly assigned to receive either retatrutide, tirzepatide, or a placebo, all given by subcutaneous injection. The study will last approximately 224 weeks, during which participants may attend 25 to 30 clinic visits for monitoring and assessment. After the main study, eligible participants can join an optional 2-year extension where all will receive either retatrutide or tirzepatide regardless of their original group. Throughout the trial, participants’ liver function and disease progression will be closely monitored through various health assessments. Researchers will track the time to the first major adverse liver event as the main outcome. Safety and health status will be evaluated regularly during clinic visits, ensuring thorough observation over the long study period.

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 evaluating AZD0780, an oral PCSK9 inhibitor, in a phase 3, randomized, placebo-controlled study to see if it can reduce the risk of major adverse cardiovascular events (MACE-PLUS) in adults with established atherosclerotic cardiovascular disease (ASCVD) or those at high risk for a first ASCVD event. The study compares AZD0780 to a placebo and monitors participants from randomization until the primary analysis censoring date, followed by a final study closure visit. Participants will be randomly assigned to receive either oral AZD0780 or an oral placebo once daily. The treatment period lasts until the primary analysis censoring date, after which a study closure visit will occur. The study is event-driven and designed to assess the time to the first major cardiovascular event during treatment. During the study, participants will be closely monitored with various assessments to evaluate cardiovascular outcomes and safety over approximately 54 months. Researchers will track the time to first event of any component of MACE-PLUS and collect data to assess the effect of AZD0780 compared to placebo. The study includes regular visits and evaluations to ensure participant safety and adherence to treatment.

Researchers are evaluating the safety and tolerability of Karonudib for treating patients with blood cancers such as Acute Myeloid Leukemia (AML), Acute Lymphoblastic Leukemia (ALL), Diffuse Large B-Cell Lymphoma, Multiple Myeloma, and high-risk Myelodysplastic Syndrome. This phase 1 study aims to find the recommended dose and schedule for Karonudib, understand how the drug behaves in the body, and look for signs of treatment effectiveness. Overall survival of participants will also be tracked. The study includes two parts: the first part tests escalating doses of Karonudib alone in four different dose groups. The extension part combines Karonudib taken twice a day with the standard treatment Idarubicin given on days 1 to 3. Patients receive Karonudib at these doses and schedules to assess safety and tolerability. Participants will undergo regular assessments including blood tests, bone marrow or imaging exams to measure their disease, and heart function tests. Researchers will monitor patients for side effects, drug levels, and response to treatment throughout the study. The study lasts at least 28 days for the first treatment cycle, with careful safety and tolerability evaluations during this time.

Researchers are evaluating the safety and effectiveness of bomedemstat (MK-3543) compared with the best available therapy (BAT) in adults with essential thrombocythemia (ET) who have not responded well to or cannot tolerate hydroxyurea. This phase 3 clinical trial aims to determine if bomedemstat provides a better durable clinicohematologic response in these participants. Participants will receive either bomedemstat as an oral capsule or one of the best available therapies, including anagrelide (oral capsule), busulfan (oral tablet), interferon alfa or its pegylated forms (subcutaneous solution), or ruxolitinib (oral tablet). The study involves a randomized, open-label design where treatments are compared directly. Throughout the study, participants will be monitored for their hematologic response up to about 52 weeks. Assessments include platelet and neutrophil counts before starting treatment to ensure eligibility. Safety and efficacy are tracked to evaluate the long-term impact of the treatments on ET.

Researchers are evaluating how well elritercept (TAK-226, KER-050) works in reducing the need for red blood cell (RBC) transfusions in adults with very low, low, or intermediate risk myelodysplastic syndromes (MDS) who require regular blood transfusions. The study is a Phase 3, double-blind, randomized, placebo-controlled trial that also aims to assess the safety and tolerability of elritercept over both short and longer periods, including in adults with high transfusion needs. Participants will be randomly assigned in a 2:1 ratio to receive either elritercept or a matching placebo by subcutaneous injection every 4 weeks. The study includes a Primary Phase lasting 24 weeks and a Secondary Phase lasting an additional 24 weeks, during which participants continue the same treatment. Following these phases, an Extension Phase allows eligible participants to continue treatment until discontinuation or study unblinding. Study visits occur every 2 weeks during the first 6 cycles and every 4 weeks thereafter. Treatment continuation depends on meeting disease assessment criteria every 24 weeks. Participants will undergo various assessments including bone marrow aspirates, transfusion evaluations, and disease status checks throughout the study. Safety follow-up lasts for 8 weeks after the last dose, with visits every 4 weeks during this time. Afterward, long-term follow-up occurs quarterly for up to 5 years or until withdrawal, death, loss to follow-up, or study closure. The main outcome measured is the percentage of participants achieving transfusion independence for at least 8 weeks during the first 24 weeks of treatment.