Riyadh

This research aims to evaluate the effects of litifilimab (BIIB059), a monoclonal antibody, in adults with active subacute or chronic cutaneous lupus erythematosus (CLE), with or without systemic lupus erythematosus (SLE). Participants have active skin symptoms of CLE that have not improved with antimalarial therapy or had difficulties continuing that treatment. The study focuses on reducing skin disease activity using several scores including CLA-IGA-R and CLASI, while also assessing safety, immune response, and quality of life. Participants will be randomly assigned to receive either litifilimab or a placebo injection under the skin every four weeks during a 24-week double-blind period where neither participants nor researchers know which treatment is given. After this, all participants will receive litifilimab injections every four weeks for an additional 28 weeks. Those who complete the treatment may join a long-term extension study or enter a follow-up safety period lasting up to 24 weeks. Total participation may last up to 80 weeks. Throughout the study, researchers will monitor skin disease activity using the CLA-IGA-R erythema score and the CLASI-A activity score to see how many participants improve. They will also assess safety, tolerability, immune system effects, and participants' quality of life using questionnaires. These evaluations occur regularly during both treatment periods and follow-up to understand the impact of litifilimab on CLE symptoms and overall health.

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 the long-term safety and effects of nerandomilast in people with idiopathic pulmonary fibrosis (IPF) or progressive pulmonary fibrosis (PPF) who have previously completed treatment with nerandomilast in earlier studies. The study aims to understand how well participants tolerate nerandomilast over time, and whether it helps improve lung function, delays symptom worsening, reduces hospital visits, or impacts survival. This is a Phase 3 open-label extension trial. Participants take nerandomilast tablets daily for up to 1 year and 10 months while continuing their usual pulmonary fibrosis treatments. The study follows an open-label design where all participants receive nerandomilast. There are no placebo or comparator groups in this extension phase. Throughout the study, participants regularly visit their doctors for health assessments and lung function tests. Doctors monitor any health problems or side effects experienced during treatment. The main outcome measured is whether participants experience any adverse events up to the final follow-up visit, which occurs at week 99. This close monitoring helps evaluate the long-term safety and potential benefits of nerandomilast in this patient group.

Researchers are evaluating the safety and effectiveness of zodasiran injections in adolescents and adults aged 12 years and older who have genetically or clinically diagnosed Homozygous Familial Hypercholesterolemia (HoFH). This phase 3, randomized, placebo-controlled study aims to understand how the treatment affects levels of LDL cholesterol, a harmful type of cholesterol, in the blood over time. Participants receive either zodasiran or a placebo through subcutaneous injections during the double-blind treatment period. After completing this phase, those who wish can enter an optional open-label extension where all placebo participants have the chance to switch to the active drug. The study involves ongoing monitoring to compare the effects of zodasiran versus placebo. Throughout the study, participants will have their LDL cholesterol levels measured at the start and after 12 months to assess changes. Researchers will also monitor safety through regular clinical assessments and laboratory tests. The total duration includes the double-blind period plus the optional extension, providing detailed information on treatment impact and participant health over time.

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 studying the real-world effectiveness of pegcetacoplan in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH). This long-term, multicenter observational study aims to fill knowledge gaps about how pegcetacoplan works in routine medical practice. It also seeks to provide important information on red blood cell transfusions and healthcare resource use before and after starting pegcetacoplan treatment. Patients who have started pegcetacoplan treatment within the last 12 months or are prescribed it at enrollment will be followed for approximately 36 months. The study will collect both retrospective data from up to 12 months before treatment start and prospective data during treatment, with a total data collection period of up to about 48 months. After stopping pegcetacoplan, patients will remain in the study for 8 weeks to monitor for any adverse events. Participants will attend their usual medical visits, and data from these visits will be collected, including effectiveness measures, safety reports, patient- and clinician-reported outcomes, and healthcare use. The primary outcome includes changes in hemoglobin levels over 6 months from the start of pegcetacoplan treatment. The study plans to enroll about 200 patients across multiple countries, and data collection includes both retrospective and prospective periods, capturing comprehensive information on pegcetacoplan use in real-world settings.

Researchers are evaluating the long-term safety and effectiveness of etavopivat, a new oral medicine being developed to treat inherited blood disorders such as sickle cell disease and thalassemia. These disorders affect hemoglobin, the protein responsible for carrying oxygen in the body. This phase 3 study aims to monitor how well etavopivat works and its safety profile over an extended period. Participants will receive one of three forms of etavopivat (A, B, or C) as oral doses. The study is open-label and multicenter, involving adults, adolescents, and children who have previously completed treatment in an etavopivat parent study and continue to benefit clinically. The treatment period can last up to 264 weeks but may end earlier if etavopivat is approved in the participant's country. During the study, researchers will track the number of treatment-emergent adverse events and adverse reactions for each participant by indication and age group from baseline through the end of the study, which can last up to 316 weeks. Participants' safety and response to long-term treatment will be closely monitored throughout this period.

Researchers are evaluating the safety, tolerability, and effectiveness of VX-880 infusion in adults with Type 1 diabetes who have impaired awareness of hypoglycemia and experience severe hypoglycemia. This Phase 1/2/3 study focuses on participants with a history of insulin dependence for over 5 years and severe hypoglycemic episodes. The study aims to understand how well VX-880 works and how safe it is for these individuals. The investigational treatment VX-880 is a biological product infused directly into the hepatic portal vein. Participants receive this infusion and are then monitored over time to assess treatment impact. The study includes long-term follow-up of up to five years to evaluate safety and tolerability, as well as efficacy measures such as insulin independence and the absence of severe hypoglycemic events one year after achieving insulin independence. During the study, participants will be closely monitored for adverse events and use continuous glucose monitors (CGM) before and throughout the study to track blood sugar levels. Researchers will assess the number of adverse events from infusion through the study period and measure the proportion of participants who become insulin independent without severe hypoglycemia. The total participation time may extend up to five years to gather comprehensive safety and efficacy data.

Researchers are evaluating treatments for adults with relapsed or refractory multiple myeloma who have previously received an anti-CD38 antibody and lenalidomide. The study compares the effectiveness of talquetamab combined with pomalidomide (Tal-P), talquetamab combined with teclistamab (Tal-Tec), and investigator's choice between two standard regimens: elotuzumab with pomalidomide and dexamethasone (EPd), or pomalidomide with bortezomib and dexamethasone (PVd). This Phase 3 trial aims to understand which combination best controls the disease progression. Participants will receive talquetamab as a subcutaneous injection, pomalidomide orally, teclistamab as a subcutaneous injection, elotuzumab intravenously, dexamethasone either orally or intravenously, and bortezomib as a subcutaneous injection. The study involves comparing these combinations with varying administration routes. The trial includes multiple treatment arms to assess different drug combinations in patients who have undergone 1 to 4 prior therapies. During the study, participants will be monitored for progression-free survival up to 3 years and 5 months. Researchers will regularly assess disease status, treatment response, and safety. Participants' performance status will be evaluated, and adherence to treatment and potential side effects will be carefully tracked. This long-term observation will help determine how well each treatment combination controls the disease over time.

Researchers are evaluating the effectiveness, safety, and tolerability of a combination treatment including adagrasib, pembrolizumab, and platinum-doublet chemotherapy compared to a placebo combined with pembrolizumab and platinum-doublet chemotherapy. This study focuses on adults with previously untreated, locally advanced or metastatic non-squamous non-small cell lung cancer (NSCLC) that has a KRAS G12C mutation. The trial is a randomized, double-blind, phase 3 study designed to provide insights into treatment options for this specific lung cancer type. Participants receive either adagrasib plus pembrolizumab alongside platinum-doublet chemotherapy drugs such as carboplatin or cisplatin and pemetrexed, or they receive a placebo plus pembrolizumab and the same chemotherapy regimen. The dosages and schedules of these drugs are specified and administered on predetermined days. The trial compares these two treatment groups to understand better the impact of adding adagrasib to the existing pembrolizumab and chemotherapy treatment. Throughout the study, participants are closely monitored for progression-free survival and overall survival, assessed up to seven years using standardized criteria for tumor response. Regular imaging scans such as CT or MRI are used to measure disease status. Safety and tolerability are also evaluated during the study, with ongoing assessments to track adverse effects and treatment response. The total duration of follow-up allows for long-term observation of treatment outcomes and participant health.