Huddinge

Researchers are conducting a Phase 3, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of rilzabrutinib in adults with active Immunoglobulin G4-related disease IgG4-RD. The study aims to measure the time to the first adjudicated disease flare and assess other important outcomes such as flare-free rates, disease activity control, glucocorticoid use, and safety parameters including adverse events, laboratory tests, and electrocardiograms ECG. Participants will be assigned to one of two groups one receiving rilzabrutinib tablets and the other receiving placebo tablets, both administered orally. The treatment period lasts 52 weeks in a double-blind manner, preceded by a 4 to 6 week screening period. After treatment, there is a 2-week follow-up, with an optional open-label extension lasting up to 108 weeks. The study includes a total of 16 visits during the main period and up to 9 additional visits during the optional extension. During their participation, adults diagnosed with IgG4-RD will undergo repeated imaging procedures such as CT, MRI, PET, or ultrasound to assess disease status. Researchers will monitor disease flares, remission status, glucocorticoid dosage, clinical activity scores, laboratory values, vital signs, and ECG results. Safety monitoring continues up to week 160 to capture treatment-emergent adverse events. Overall, participation lasts up to 60 weeks, with possible extension for those continuing in the optional phase.

Researchers are evaluating CPV-104, a new medicine designed to regulate the complement system, which can be overactive in diseases such as C3 glomerulopathy C3G, a very rare kidney disorder. This phase 1 trial is the first time CPV-104 is being tested in people, including both healthy adults and adults with C3G, to assess its safety, tolerability, how the body processes the medicine, and whether the immune system reacts to it. The study has two parts Part 1 involves healthy volunteers receiving a single intravenous dose of CPV-104 or a placebo in a randomized, double-blind manner across several dose levels. Part 2 includes patients with C3G receiving four weekly intravenous doses of CPV-104 without placebo. Doses are escalated if the medicine is tolerated, and a Safety Review Committee regularly reviews results to ensure safety before progressing. Participants will undergo close monitoring throughout the study, including side-effect checks, 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. The primary outcome is the incidence of severe and serious adverse drug reactions up to Day 29 for healthy volunteers and Day 50 for C3G patients. The total study duration varies by part, with detailed safety assessments conducted throughout.

Researchers are evaluating a lifestyle intervention aimed at improving health habits and reducing cardiometabolic risk factors in adults with obsessive-compulsive disorder OCD. This condition is linked to higher risks of heart and metabolic diseases, partly due to unhealthy lifestyle behaviors. This trial is the first randomized controlled study to assess the effectiveness and cost-efficiency of such an intervention for people with OCD. Participants are randomly assigned to one of two groups. The first group receives a 13-week lifestyle program including one personal session to set goals, followed by 12 weekly group sessions combining education on healthy habits and physical exercise. After these sessions, they get access to a digital booster module to maintain changes. The second group receives one individual session with feedback on their health and written advice on healthy lifestyle habits based on national guidelines. During the study, participants will wear accelerometers to measure physical activity, and assessments will take place at the start, after 14 weeks, and at 12 months to track changes in activity, diet, mental health, and cardiometabolic markers. Questionnaires and physical measurements will be used to evaluate outcomes such as stress, sleep, OCD symptoms, quality of life, and various blood tests. Safety and cost-effectiveness will also be monitored throughout the trial period.

Researchers are evaluating whether retatrutide and tirzepatide can prevent major adverse liver outcomes in adults with metabolic dysfunction-associated steatotic liver disease MASLD who are at high risk based on non-invasive tests. This Phase 3 randomized controlled trial aims to assess these treatments compared to placebo in about 4,500 adults over approximately 224 weeks. The study is sponsored by Eli Lilly and Company and focuses on liver disease progression and related health measures. Participants will be randomly assigned to receive retatrutide, tirzepatide, or placebo, all administered by subcutaneous injection. The trial includes two placebo groups corresponding to each experimental drug. After completing the main study, eligible participants may join a 2-year extension where all will receive either retatrutide or tirzepatide regardless of their initial assignment. During the study, participants may attend around 25 to 30 clinic visits for health monitoring, study procedures, and assessments of liver function and disease status. Researchers will measure the time to major adverse liver outcomes, changes in liver fibrosis scores, liver stiffness, liver fat content, liver enzyme levels, body weight, and cardiovascular events. Monitoring will continue from baseline through study completion, with detailed evaluations at multiple timepoints including week 104.

Researchers are evaluating the combination of baxdrostat and dapagliflozin in people with chronic kidney disease CKD and high blood pressure hypertension. This Phase III, double-blind, placebo-controlled study aims to assess whether this combination reduces the risk of serious kidney damage, heart failure events, or cardiovascular death compared to dapagliflozin alone. The study includes participants with CKD and hypertension who meet specific kidney function and blood pressure criteria. Participants who are not already taking SGLT2 inhibitors will first complete a 4-week dapagliflozin run-in period. Then, they will be randomly assigned to receive either baxdrostat plus dapagliflozin or a placebo plus dapagliflozin. Baxdrostat dosing may start low and be increased if needed. Study visits will occur at 2, 4, 8, 16, 34, and 52 weeks after randomization, and then approximately every four months until the study ends, which is based on the number of key kidney or heart-related events. Throughout the study, participants will have regular assessments including blood tests to monitor kidney function and potassium levels, blood pressure measurements, and evaluations of heart and kidney health. If participants stop the blinded study drug early, they will continue dapagliflozin if possible and remain in the study for ongoing visits and monitoring. The main outcome is whether the combination treatment reduces the risk of a 50% sustained decline in kidney function, kidney failure, heart failure events, or cardiovascular death over up to 37 months.

Researchers are evaluating AZD0780, an oral PCSK9 inhibitor, in a phase 3, randomized, placebo-controlled study. This trial focuses on patients with established atherosclerotic cardiovascular disease ASCVD or those at high risk for a first ASCVD event. The study aims to assess how AZD0780 compares to placebo in reducing the risk of major adverse cardiovascular events, also known as MACE-PLUS, over the course of the trial. Participants are randomly assigned to receive either oral AZD0780 once daily or a matching placebo once daily. The study continues until a primary analysis censoring date, which may be up to approximately 54 months from randomization. After this, a study closure visit will be conducted as the final visit for each participant. During the study, participants will be regularly monitored for cardiovascular events including heart attacks, strokes, urgent coronary revascularizations, and other related outcomes. Researchers will track the time to first occurrence of these events as the primary outcome. Safety and other secondary outcomes like all-cause mortality will also be assessed. The total participation time can last up to about 54 months, with ongoing evaluations throughout this period.

Researchers are evaluating the safety and tolerability of Karonudib, an oral drug, for treating patients with advanced blood cancers including Acute Myeloid Leukemia AML, Acute Lymphoblastic Leukemia ALL, Diffuse Large B-Cell Lymphoma DLBCL, Multiple Myeloma MM, high-risk Myelodysplastic Syndrome MDS, and Burkitts lymphoma. The study also aims to find a recommended dose and schedule for further development, understand how the drug is processed in the body, and look for early signs of treatment effect. Overall survival of patients will also be recorded during the trial. The study includes a dose escalation phase where Karonudib is given as an oral solution every other day in three planned dose groups. An extension phase involves Karonudib taken twice a week together with standard care treatment Idarubicin on days 1 to 3. Patients will receive the drug orally and the study is designed sequentially to evaluate safety and tolerability first before further phases. This is a Phase 1 interventional study focused on treatment. Participants will be monitored closely for safety and tolerability during the first 28-day treatment cycle, which is the primary outcome period. Researchers will collect blood and bone marrow samples or imaging to measure disease and treatment response. Additional assessments include heart function, liver and kidney tests, and pregnancy tests for women of childbearing potential. The trial lasts until April 2027 and includes repeated assessments to understand the drugs effects and patient survival.

Researchers are evaluating AZD0120, a CD19BCMA dual targeting CAR T-cell therapy, in adults with autoimmune diseases including systemic sclerosis SSc, idiopathic inflammatory myopathies IIM, and difficult-to-treat rheumatoid arthritis D2T RA. This Phase 1b, open-label, multi-center study aims to assess the safety and tolerability of AZD0120 and to determine the recommended dose for each disease group. Approximately 9 to 12 participants will be studied in each disease category. Participants will receive an infusion of one of two AZD0120 regimens. The study is designed as a multi-cohort trial with parallel groups, where each participant receives the assigned CAR T-cell therapy infusion. The main focus is on monitoring dose-limiting toxicities and treatment-emergent adverse events over one year. Secondary outcomes include various cellular kinetics measures, antibody development, viral replication presence, and changes in disease activity and skin scores over the same period. During the study, participants will be closely monitored with clinical evaluations and laboratory tests to assess safety and therapeutic effects. Measurements such as Disease Activity Score DAS 28-CRP, modified Rodnan Skin Score mRSS, and total improvement scores will be tracked. Participants must comply with study requirements including medication washout periods and use of effective birth control. The overall participation involves intensive follow-up for one year to evaluate safety and disease response after receiving the AZD0120 infusion.

Researchers are evaluating elritercept TAK-226, KER-050, an investigational drug, for treating anemia in adults with very low, low, or intermediate risk myelodysplastic syndromes MDS who need regular red blood cell RBC transfusions. The study is a Phase 3, randomized, double-blind, placebo-controlled trial designed to assess how well elritercept reduces the need for RBC transfusions and to evaluate its safety and tolerability over time. Participants will be randomly assigned in a 21 ratio to receive either elritercept or a matching placebo, both given as subcutaneous injections 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 their assigned treatments. Eligible participants may also enter an Extension Phase to continue treatment until individual discontinuation or study unblinding. After treatment ends, a Safety Follow-Up Period of 8 weeks and a long-term follow-up lasting up to 5 years will monitor participants. During the study, participants will have visits approximately every 2 weeks initially, then every 4 weeks, to assess treatment effects and safety. Researchers will evaluate the percentage of participants achieving transfusion independence and monitor adverse events, laboratory values, vital signs, and heart tests. Long-term follow-up will continue through regular check-ins for up to five years or until the participant withdraws or the study ends.

Primary immune thrombocytopenia ITP is a condition where the immune system mistakenly destroys platelets, the cells responsible for blood clotting, resulting in increased bleeding and bruising. This trial evaluates whether mezagitamab given as a subcutaneous injection can effectively maintain stable platelet counts in adults with chronic ITP compared to a placebo. The study is a phase 3, randomized, double-blind, placebo-controlled trial designed to assess both the efficacy and safety of mezagitamab in this population. Participants are randomly assigned to receive either mezagitamab or a matching placebo by subcutaneous injection once weekly. The treatment schedule consists of 8 weekly doses followed by an 8-week break, then another 8 weekly doses, totaling up to 6 months of treatment. Those who finish this trial or do not respond to treatment by week 16 may have the option to join a continuation study to receive open-label mezagitamab if eligible and if the site is open. During the study, participants will visit the clinic multiple times for treatment and monitoring. Researchers will measure platelet responses, symptom changes using patient questionnaires, bleeding events, and blood concentrations of mezagitamab. Safety assessments include monitoring for anti-drug antibodies and adverse events. The primary outcome is the percentage of participants with a durable platelet response up to week 24. The trial is expected to complete in March 2028.