Florencio Varela

Researchers are studying adults with confirmed Primary Biliary Cholangitis (PBC) and cirrhosis, a scarring of the liver caused by damage to bile ducts. PBC is a slowly progressing disease that causes bile acid buildup and further liver damage, which can lead to cirrhosis. This study aims to evaluate if elafibranor, a daily medication, can prevent worsening clinical outcomes such as the need for liver transplant or death, compared to a placebo. It also looks at the safety of long-term elafibranor use and its effect on symptoms like itching and tiredness. Participants will take either an 80 mg tablet of elafibranor or a matching placebo once daily for up to 3.5 years in a double-blind setup, meaning neither the participants nor researchers know who receives which treatment. This long-term treatment period is designed to monitor the drug's impact over time. The study includes two groups: one receiving elafibranor and the other receiving placebo, with treatment lasting up to approximately 42 months. During the study, participants will be regularly assessed from the start until 4 weeks after treatment ends, with a maximum involvement of 3.5 years. Researchers will measure event-free survival, tracking if participants avoid clinical events indicating disease worsening. Safety monitoring will include tracking side effects and overall health, while symptom impact will be evaluated. Participants will provide informed consent and follow the study protocol throughout this extended observation period.

Researchers are evaluating the efficacy and safety of benralizumab, given as a subcutaneous injection, in children and adolescents aged 6 to under 18 years who have severe eosinophilic asthma. These patients have a history of asthma exacerbations and uncontrolled symptoms despite treatment with high-dose inhaled corticosteroids plus at least one other controller medication. This Phase III study aims to compare benralizumab to placebo in reducing the time to the first asthma exacerbation. The study includes a screening period lasting from 4 to 12 weeks to confirm eligibility. After screening, patients are randomly assigned in a 1:1 ratio to receive either benralizumab or placebo via subcutaneous injections during a double-blind treatment period lasting a minimum of 16 weeks. This period continues until the patient experiences an asthma exacerbation or a set number of events occur. Patients who exacerbate can enter an open-label extension where all receive benralizumab for at least 48 weeks. An end-of-treatment visit occurs 8 weeks after the last dose in the extension phase. Participants will be monitored through visits and assessments including confirmation of severe eosinophilic asthma, asthma control questionnaires, and symptom diaries. Researchers will measure the time to first asthma exacerbation as the primary outcome. Medication adherence is tracked during screening, and safety is monitored throughout both the double-blind and extension periods. Total participation may span over a year, considering screening, treatment, extension, and follow-up visits.

Researchers are evaluating the effectiveness and safety of a single dose of nexiguran ziclumeran (NTLA-2001) compared to a placebo in adults aged 18 to 85 with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN). This multinational, multicenter, phase 3 study includes approximately 60 participants and uses a double-blind, randomized, placebo-controlled design to provide reliable results. The study aims to better understand treatment effects on nerve function and disease progression in this genetic neurodegenerative condition. Participants will be randomly assigned to receive either a single intravenous infusion of 55 mg nexiguran ziclumeran or a placebo consisting of normal saline. To give all participants a chance to receive the investigational treatment, those initially on placebo may cross over to the treatment group after 12 or 18 months, depending on study criteria. This crossover ensures further assessment of long-term outcomes with the study drug. During the 18-month study, researchers will monitor participants through clinical evaluations, including the Modified Neuropathy Impairment Score plus 7 (mNIS+7) and blood tests to measure serum transthyretin levels at specified intervals. Safety and efficacy will be carefully tracked, and participants will undergo regular assessments to understand the impact of treatment on their neuropathy and overall health throughout the study period.

Researchers are investigating the effects of two different doses of Glycopyrronium (GP) metered dose inhaler (MDI) compared to a placebo MDI when added to background treatment with Budesonide and Formoterol Fumarate (BFF) MDI. This study focuses on children aged 4 to less than 12 years who have asthma. The goal is to assess how these treatments affect lung function in this pediatric population during a Phase II clinical trial. The study is designed as a multi-center, randomized, double-blind, 3-period, 6-sequence crossover trial. It begins with a 3-week run-in period, followed by three separate 3-week treatment periods during which participants receive one of the three treatments: BFF MDI plus GP MDI Dose A, BFF MDI plus GP MDI Dose B, or BFF MDI plus placebo MDI. All inhalers are taken twice daily via oral inhalation. After completing the treatment periods, participants attend a safety follow-up visit 12 to 16 days after their last dose. Participants will undergo regular assessments including lung function tests to measure Forced Expiratory Volume in one second (FEV1) one hour after dosing at the end of treatment. Researchers will monitor safety through clinical exams and follow-up visits. The total participation duration includes the run-in, treatment periods, and safety follow-up, providing a comprehensive evaluation of the treatments' effects on asthma control in children.

Researchers are studying the effectiveness and safety of a combination inhaler containing fluticasone propionate and albuterol sulfate delivered through a multidose dry powder inhaler with an electronic module (Fp/ABS eMDPI). This Phase 3 trial focuses on people aged 12 years and older who have asthma. The study also looks at the safety and tolerability of this inhaler when used four times daily over four weeks, as well as the pharmacokinetics of the combination and its individual components after a single dose. Participants will be randomly assigned to receive either the Fp/ABS combination inhaler, fluticasone propionate alone, albuterol sulfate alone, or a placebo inhaler. All treatments are given as inhalation powders. The main treatment period lasts four weeks, during which the inhalers are taken four times a day. The total study duration for each participant is about 10 weeks, not counting an optional prescreening visit. Throughout the study, researchers will measure lung function changes, specifically forced expiratory volume in one second (FEV1), from baseline to week 4. Participants will undergo assessments including lung function tests and safety evaluations. The study monitors how the inhaler affects breathing over time and checks for any side effects or tolerability issues during the treatment period.

Researchers are evaluating the effect of a triple therapy inhaler called BGF MDI containing budesonide, glycopyrronium, and formoterol fumarate compared with a dual therapy inhaler called GFF MDI containing glycopyrronium and formoterol fumarate in people with Chronic Obstructive Pulmonary Disease (COPD) who have a higher risk of heart and lung problems. This Phase III randomized, double-blind, parallel group study takes place at multiple centers and focuses on cardiopulmonary outcomes in these patients. Participants receive either the BGF MDI 320/14.4/9.6 micrograms twice daily or the GFF MDI 14.4/9.6 micrograms twice daily. The treatments are inhaled using metered dose inhalers. The study compares these two therapies over time to see how they affect the time until the first severe heart or lung event occurs. The study design ensures that neither participants nor researchers know which treatment is given to reduce bias. During the study, participants will have regular visits to the study site or virtual visits to complete assessments. Researchers will monitor lung function, symptoms, and blood tests, including blood eosinophil counts and COPD assessment test scores. The main outcome measured is the time to the first severe cardiac or COPD event, with follow-up lasting up to three years. Safety and adherence to treatment will also be closely observed throughout the study period.

Researchers are evaluating the safety, tolerability, pharmacokinetics, pharmacodynamics, and immune response to the drug GSK4527363. The study is a Phase 1 trial involving healthy participants, people with active Systemic Lupus Erythematosus (SLE), healthy individuals of Chinese and Japanese descent, and participants with interstitial lung disease linked to connective tissue disease. The trial aims to gather detailed safety and biological data across these groups. Participants will receive either GSK4527363, a matching placebo, or Belimumab. The study is divided into four parts: Part A includes healthy participants; Part B involves those with active SLE; Part C focuses on healthy participants of Chinese and Japanese ancestry; and Part D enrolls participants with connective tissue disease-associated interstitial lung disease. Treatments are administered according to group assignments, with monitoring continuing up to 52 weeks for Parts A and C and up to 68 weeks for Parts B and D. During the study, researchers will monitor participants closely through physical exams, laboratory tests, vital signs, and heart monitoring. Safety assessments include tracking adverse events and changes in mental health using specific rating scales. Participants are observed for up to 52 or 68 weeks depending on their group, with regular check-ins to evaluate the study drug's effects and ensure participant safety throughout the trial.

Researchers are evaluating the efficacy and safety of verekitug (UPB-101) in adults with moderate-to-severe Chronic Obstructive Pulmonary Disease (COPD), a long-term inflammatory lung condition. This global, multicenter Phase 2b study aims to understand how well verekitug works compared to a placebo, alongside participants' usual COPD medications. Participants must have a confirmed COPD diagnosis and meet specific lung function and symptom criteria to join the study. Participants will be randomly assigned to receive one of two doses of verekitug or a matching placebo, in addition to their regular COPD background treatments. The study includes a screening period of about 4 weeks, followed by treatment lasting between 60 and 108 weeks. After treatment, there is a 16-week follow-up period to monitor participants after their last dose. Throughout the study, participants will undergo various assessments including lung function tests and symptom evaluations. Researchers will track the annual rate of moderate or severe COPD flare-ups from the start of treatment through week 108. Safety and tolerability will be closely monitored during the treatment and follow-up periods to ensure participants' well-being over the course of the trial.

Researchers are evaluating the investigational drug volixibat to treat itching (pruritus) caused by Primary Biliary Cholangitis (PBC), a liver disease. This study aims to learn more about how volixibat affects itching symptoms and whether it has any impact on the progression of PBC. The trial is a Phase 2 randomized, double-blind, placebo-controlled study designed to assess the drug's safety and effectiveness. Participants will receive either volixibat oral capsules or placebo capsules that look identical but do not contain the active drug. Volixibat is taken twice daily and works as an Ileal Bile Acid Transporter (IBAT) inhibitor. The study compares volixibat to placebo over a treatment period to determine differences in itching severity and safety outcomes. During the study, participants will complete the Adult Itch Reported Outcome (Adult ItchRO) questionnaire to measure daily itch scores from the start of the trial to week 28. Researchers will monitor symptoms and adverse events throughout the study visits. Participants must comply with all scheduled visits and assessments until the study ends to provide data on the drug's effects and safety profile.

Researchers are evaluating the effects of three different dosing schedules of povorcitinib on lung function in adults with moderately to severely uncontrolled asthma. This Phase 2, randomized, double-blind, placebo-controlled study aims to assess both the safety and efficacy of povorcitinib in improving respiratory outcomes for this patient group. Participants have a history of asthma treatment with medium- to high-dose ICS-LABA therapy and meet specific lung function criteria before enrollment. Participants will receive one of three povorcitinib dosing regimens or a placebo while continuing their background ICS-LABA therapy. The study includes multiple treatment arms to compare the effects of different doses. The intervention period lasts 24 weeks, during which pulmonary function and other health measures will be monitored. During the study, participants will undergo regular assessments, including lung function tests measuring pre-bronchodilator forced expiratory volume in 1 second (pre-BD FEV1) at baseline and week 24. Researchers will also monitor safety and collect data on asthma exacerbations and symptom control. The total participation time covers the treatment duration and follow-up evaluations to assess changes in pulmonary function and overall health status.