Coimbra

Heart failure is a condition with rising cases that causes significant health problems and costs, despite treatment advances. A crucial issue in heart failure is myocardial fibrosis, which occurs in different heart failure types, including non-ischemic dilated cardiomyopathy (HFrEF) and hypertrophic cardiomyopathy (HFpEF). Since fibrosis may be reversible with certain drugs, researchers are interested in non-invasive ways to monitor this process and treatment effects. Cardiac magnetic resonance imaging (CMR) is currently the best tool to detect fibrosis but has limits in identifying active or early fibrosis, which 68Ga-FAPI PET/CT might improve. The study evaluates the use of 68Ga-FAPI PET/CT, a special imaging test, to assess myocardial fibrosis in patients with heart failure types HFrEF and HFpEF. This is a single-center, prospective, observational pilot study comparing 68Ga-FAPI PET/CT to standard CMR. Researchers aim to develop methods for measuring 68Ga-FAPI uptake to support future anti-fibrotic treatments and explore links between fibrosis, blood biomarkers, and heart events. Participants will undergo 68Ga-FAPI PET/CT scans along with recent echocardiograms and CMR within 3 months. They must have a normal coronary angiogram or CT coronary angiography within 6 months. The main outcome measured is the uptake of 68Ga-FAPI at baseline to evaluate fibrosis. The study monitors heart function, biomarkers, and cardiovascular events, aiming to improve assessment and treatment of heart failure related to fibrosis.

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 effectiveness, safety, and tolerability of subcutaneous ianalumab in adults with diffuse cutaneous systemic sclerosis. This Phase 2 study compares ianalumab with a placebo in participants diagnosed according to established classification criteria, focusing on those with active disease and specific autoantibodies. The goal is to better understand ianalumab's impact on this condition over a long treatment period. The study includes several phases: up to 6 weeks for screening, followed by a 52-week initial treatment period where participants receive either ianalumab or placebo by subcutaneous injection. After this, there is a second 52-week open-label treatment period where all participants receive ianalumab. Finally, a post-treatment follow-up period lasts at least 20 weeks and can extend up to 2 years after the last dose. Participants will undergo various assessments throughout the study, including evaluations of their skin condition using the rCRISS25 response at week 52. Safety and tolerability will also be closely monitored. The study involves regular visits for clinical evaluations, laboratory tests, and monitoring of disease activity and antibody status, with the total participation potentially lasting over two years including follow-up.

Researchers are evaluating the safety, tolerability, and therapeutic effects of a combination treatment using BNT113 and pembrolizumab compared to pembrolizumab alone for patients with unresectable recurrent or metastatic head and neck squamous cell carcinoma (HNSCC) that is positive for human papillomavirus 16 (HPV16+) and expresses the PD-L1 protein with a combined positive score of 1 or higher. This Phase II/III trial includes patients whose cancer cannot be treated with local therapies and who have not received prior systemic anticancer therapy for their current disease condition. The trial consists of two parts. Part A is a non-randomized Safety Run-In Phase to confirm the safety and tolerability of BNT113 combined with pembrolizumab at the selected dose. Part B is a randomized phase that compares BNT113 plus pembrolizumab against pembrolizumab alone as first-line treatment. Patients in Part A continue their treatment without randomization. Treatments are given by intravenous injection or infusion, and patients may receive either combination therapy or monotherapy for up to 24 months. There is also an optional pre-screening phase to test tumor samples for HPV16 DNA and PD-L1 expression before entering the main trial. Participants undergo regular assessments including tumor measurements based on RECIST 1.1 criteria confirmed by independent review. Researchers monitor treatment-emergent adverse events for up to 27 months in Part A and evaluate overall survival and progression-free survival for up to 48 months in Part B. Tumor tissue samples are collected before treatment to confirm eligibility. The study involves ongoing safety monitoring and efficacy evaluations throughout the treatment and follow-up periods.

Researchers are evaluating the safety, tolerability, and effectiveness of Navepegritide (TransCon CNP) in infants diagnosed with achondroplasia, a genetic condition affecting growth. This Phase 2, multicenter, double-blind, randomized, placebo-controlled trial involves infants aged from birth up to but not including 2 years, with genetically confirmed heterozygous achondroplasia. The study aims to monitor the growth impact and safety of weekly doses of Navepegritide over one year. Participants receive either 100 micrograms per kilogram of Navepegritide or a placebo, both administered as subcutaneous injections once per week for 52 weeks. This treatment period is followed by an open-label extension phase where participants may continue receiving the study drug. The trial compares the effects of the investigational drug against placebo to assess its tolerability and growth outcomes. Throughout the 52 weeks, infants will undergo regular medical evaluations including physical examinations, vital signs monitoring, ECGs, imaging, and laboratory tests. Researchers will track adherence to weekly injections and daily vitamin D supplementation where applicable. The primary outcomes focus on safety and growth effects of Navepegritide, with continuous monitoring to ensure participant well-being during the trial period.

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 efficacy and safety of rilvegostomig compared to pembrolizumab as first-line treatments for patients with metastatic non-small cell lung cancer (mNSCLC) whose tumors have high PD-L1 expression. This Phase III, randomized, double-blind, and global study focuses on participants with stage IV mNSCLC who do not have certain genetic mutations or rearrangements and are eligible for systemic therapy. Participants receive either rilvegostomig or pembrolizumab intravenously on Day 1 of each 21-day cycle. The study compares these two biological treatments given as monotherapy. Both groups will be monitored over time to assess treatment impact and safety. Throughout the study, participants undergo evaluations including tumor measurements by CT or MRI, performance status assessments, and organ function tests. Researchers will measure overall survival and progression-free survival for up to approximately five years. Tumor samples are collected before treatment for central testing, and participants’ health and treatment responses are closely followed during the trial period.

The purpose of this study is to assess the long-term safety and tolerability after an intravitreal injection (a shot of medicine into the eye) of JNJ-81201887 administered in parent clinical studies.

Researchers are evaluating the effects of pelacarsen (TQJ230), given as a monthly injection under the skin, in people with mild to moderate calcific aortic valve stenosis. This study aims to see if pelacarsen can safely slow the progression of this heart valve condition compared to a placebo. The trial is a phase 2, randomized, double-blind, placebo-controlled study conducted at multiple centers. Participants will receive either pelacarsen 80 mg or a matching placebo once a month. Before starting the treatment, they must have elevated lipoprotein(a) levels and be optimally treated for existing cardiovascular risk factors. The study focuses on those aged 50 to under 80 years with mild or moderate calcific aortic valve stenosis. During the 36 months of participation, researchers will monitor changes in peak aortic jet velocity and aortic valve calcium score to assess disease progression. Safety, tolerability, and the impact of the treatment will be evaluated. Participants will undergo regular assessments, including laboratory tests and clinical evaluations, to track heart valve condition and overall health throughout the study.

Researchers are evaluating the pharmacokinetics (PK), pharmacodynamics (PD), safety, and tolerability of fenebrutinib, an oral drug, in children and adolescents aged 10 to under 18 years with relapsing multiple sclerosis (RMS). This open-label, single-arm Phase 2 study aims to understand how fenebrutinib behaves in the body and its effects in this younger population with RMS, diagnosed according to established pediatric MS criteria and showing active disease signs. The study includes two main periods: a Dose Exploration Period where participants receive fenebrutinib orally and are monitored for drug levels and effects, followed by an Optional Extension Period where eligible participants may continue treatment. Fenebrutinib dosing details and adjustments are guided during the Dose Exploration Period, with ongoing safety and tolerability assessments throughout both periods. Participants will undergo various assessments including blood tests to measure fenebrutinib plasma concentration up to week 96, and MRI scans at week 12 to count new brain lesions enhanced by gadolinium. Safety, tolerability, and disease activity will be closely monitored through clinical evaluations and imaging. The study duration may extend based on participant continuation into the Optional Extension Period, with regular follow-ups and data collection to assess long-term effects and treatment impact.