Empangeni

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 conducting a Phase 2 randomized, double-blind, placebo-controlled study to evaluate the safety and effectiveness of different doses of ELV001 in treating active rheumatoid arthritis (RA) in patients who have not responded well to methotrexate and tumor necrosis factor inhibitors. The trial plans to enroll around 180 to 220 adult participants aged 18 to 75 years with active RA meeting specific clinical criteria. The study aims to measure changes in disease activity using the DAS28-C-reactive protein score from baseline to week 12. The study includes four groups receiving placebo or ELV001 at doses of 25 mg, 75 mg, or 125 mg. Participants receive treatment for 24 weeks, starting with a 4-week screening period. The first 12 weeks are double-blind and placebo-controlled, followed by a treatment extension from weeks 12 to 24 where some participants may have their ELV001 dose adjusted. After treatment, a 4-week safety follow-up period monitors participants for any adverse effects. Participants will have regular assessments including blood tests, joint evaluations, and monitoring of disease activity scores throughout the study. Researchers will track medication adherence and safety through physical exams, laboratory tests, and ECGs. The primary outcome focuses on improvement in disease activity at week 12. The total study duration for each participant is about 32 weeks from screening to the end-of-study visit.

Researchers are evaluating the safety, effectiveness, and how the body processes obinutuzumab in adolescents aged 12 to less than 18 years who have biopsy-confirmed active Class III or IV lupus nephritis, a kidney inflammation caused by lupus. The study also includes an open-label safety and pharmacokinetics evaluation of obinutuzumab in younger children aged 5 to less than 12 years with lupus nephritis. This phase II, randomized, double-blind, placebo-controlled trial aims to gather important data to better understand treatment options for these age groups with lupus nephritis. Participants will receive obinutuzumab or a matching placebo through intravenous infusions on Day 1, Day 14, Week 24, Week 26, and Week 52. All participants will also take mycophenolate mofetil orally at home starting from Day 1 at a target dose based on body surface area. Before each infusion, participants will receive pre-medications including acetaminophen, diphenhydramine, and methylprednisolone to help prevent infusion reactions. Additionally, oral prednisone or an equivalent corticosteroid will be taken daily at home with a planned tapering schedule to reduce the dose by Week 24. Throughout the 76-week study, participants will be closely monitored for kidney response and any side effects. Researchers will assess the percentage of participants who achieve a complete renal response and track adverse events from the start to Week 76. These evaluations include various health assessments, laboratory tests, and safety monitoring to ensure participant well-being and gather detailed data on treatment effects over time. The study includes both adolescent and pediatric groups, with specific recruitment timelines for the younger children.

Pain relief in children with burn injuries can be difficult in settings with limited resources due to a lack of medication, monitoring tools, or trained staff. This research evaluates a simple, low-cost, non-electronic method using a kaleidoscope to distract and reduce acute pain during dressing changes in children aged 7 to 12 years with partial thickness burns. The study takes place in a resource-limited outpatient clinic in South Africa and aims to find an effective pain reduction tool that does not depend on expensive equipment or medication. Participants are randomly assigned to either a control group receiving standard care with paracetamol or NSAIDs or an intervention group receiving the same standard care plus distraction using a kaleidoscope during dressing changes. The kaleidoscope is introduced at the start of the dressing change and used until at least one minute after the procedure, encouraging distraction through smiles, laughter, or verbal response before removal of the dressing. Children will be assessed for changes in pain levels by measuring pain scores before and during dressing changes. Researchers will monitor pain relief effectiveness and safety, focusing on whether the kaleidoscope distraction reduces pain more than standard care alone. The study expects this tool to be safe, feasible, and helpful in improving psychological well-being and recovery in children undergoing painful procedures in low-resource environments.

Researchers are evaluating the effects of two inhalers, budesonide/albuterol metered-dose inhaler (BDA MDI) and albuterol sulfate metered-dose inhaler (AS MDI), both taken as needed, on reducing severe asthma attacks in adolescents aged 12 to under 18 years who have a clinical diagnosis of asthma and have experienced at least one severe asthma exacerbation in the past year. This is a Phase IIIb randomized, double-blind, multicenter study lasting 52 weeks with a safety follow-up period after treatment. Participants will be randomly assigned to receive either BDA MDI 160/180 micrograms (two puffs of 80/90 micrograms) or AS MDI 180 micrograms (two puffs of 90 micrograms) as needed, alongside their usual asthma maintenance therapy, for 52 weeks. The study includes a 7 to 28-day screening period before treatment and a safety follow-up visit 7 to 14 days after the end of treatment. Additionally, a pharmacokinetic sub-study involves a single dose of open-label BDA MDI administered after the safety follow-up. During the study, participants will be monitored for the annual rate of severe asthma exacerbations from randomization to week 52. Assessments include evaluating inhaler technique, peak expiratory flow measurements, and adherence to contraception methods for participants of childbearing potential. Safety will be monitored throughout the treatment and follow-up periods. The total study duration includes screening, 52 weeks of treatment, and safety follow-up.

Researchers are evaluating the effect of balcinrenone/dapagliflozin compared with dapagliflozin alone on cardiovascular death and heart failure events in patients with chronic heart failure and impaired kidney function who recently experienced a heart failure event. This is a Phase III, international, randomized, double-blind, parallel-group, active-controlled study involving approximately 700 sites in about 40 countries. Participants will be randomly assigned in a 1:1:1 ratio to receive one of three treatments once daily: a capsule of balcinrenone/dapagliflozin 15 mg/10 mg with a placebo tablet, a capsule of balcinrenone/dapagliflozin 40 mg/10 mg with a placebo tablet, or a dapagliflozin 10 mg tablet with a placebo capsule. The study is event-driven, with an estimated average duration of 22 months that includes a screening period, a 20-month blinded treatment phase, and a one-month follow-up on open-label dapagliflozin. During the study, participants will be monitored for the time to first occurrence of cardiovascular death, heart failure hospitalization, or heart failure events without hospitalization over approximately 38 months. Assessments include clinical evaluations, laboratory tests, and safety monitoring throughout the study and follow-up period to track treatment effects and patient outcomes.