Clinical trials

Nerandomilast is being developed to help people with systemic sclerosis by potentially improving symptoms and slowing disease progression. This study is open to adults who are at least 18 years old and have systemic sclerosis (SSc). People can join the study if they have limited or diffuse cutaneous SSc with disease onset within 7 years of the first non-Raynaud's symptom. The purpose of this study is to find out whether a medicine called nerandomilast helps people with systemic sclerosis. This study also aims to find out how well nerandomilast is tolerated in people with systemic sclerosis. Participants are put into 2 groups randomly, which means by chance. One group takes nerandomilast tablets and the other group takes placebo tablets. Placebo tablets look like nerandomilast tablets but do not contain any medicine. Participants take the tablets twice a day. Participants are in the study for 1 to about 4 years. During this time, they visit the study site regularly and get phone calls from the site staff. During study visits participants regularly have blood samples taken and doctors check changes in skin thickening, lung function, and internal organs, overall health and the safety and tolerability of study treatment in people with SSc. The results are compared between the groups to see whether the treatment works. The doctors also regularly check participants' health and take note of any unwanted effects.

This study is open to adults with systemic lupus erythematosus (SLE). The purpose of this study is to find out whether a medicine called BI 3000202 helps people with SLE. The study tests different doses of BI 3000202 and aims to find the best dose for people with this condition. Participants are put into 5 groups randomly, which means by chance. 4 groups get different doses of BI 3000202, and 1 group gets a placebo. Placebo tablets look like BI 3000202 tablets but do not contain any medicine. Participants take the tablets for 1 year. All participants also continue their regular treatment for SLE. Participants are in the study for a bit longer than 1 year. During this time, they visit the study site regularly. Doctors check the participants' health and take note of any unwanted effects. They also compare the results between the groups to see if the treatment works.

PODOMOUNT-pFSGS This study is open to adults and adolescents with a kidney condition called focal segmental glomerulosclerosis (FSGS). The purpose of this study is to find out whether a medicine called BI 764198 helps people with FSGS. Participants are put into 2 groups randomly, which means by chance. Every participant has an equal chance of being in each group. One group takes BI 764198 tablets, and the other group takes placebo tablets. Placebo tablets look like BI 764198 tablets but do not contain any medicine. Participants take a tablet once a day for up to 2 years. All participants also continue their standard medication for FSGS. Participants are in the study for up to 2 years. During this time, they visit the study site about every 3 months. Participants regularly collect urine samples. This is done to check their kidneys. The results are compared between the two groups to see whether the treatment works. The doctors also regularly check participants' health and take note of any unwanted effects.

This study is open to adults with type 2 diabetes, high blood pressure, and cardiovascular disease. People can join the study if they have these conditions and do not have a history of heart failure. The purpose of this study is to find out if a medicine called vicadrostat, when taken with empagliflozin, helps reduce cardiovascular risk in people with these conditions. The study will compare this combination to a placebo version of vicadrostat with empagliflozin. Participants are put into 2 groups randomly, which means by chance. One group takes vicadrostat and empagliflozin tablets, and the other group takes placebo tablets with empagliflozin. Placebo tablets look like vicadrostat tablets but do not contain any medicine. Participants take a tablet once per day for 2 and a half years and up to 4 years and 3 months. All participants also continue their medication for type 2 diabetes, high blood pressure, and cardiovascular disease. Participants have an equal chance of receiving the study medicine or placebo. Participants are in the study for up to 4 years and 3 months. During this time, they visit the study site regularly. During these visits, doctors collect information about participants' health and take blood samples. The doctors document when participants experience cardiovascular events. The doctors also regularly check participants' health and take note of any unwanted effects.

In this study, researchers will learn more about the use of felzartamab in participants with immunoglobulin A nephropathy (IgAN). IgAN is a kidney disease caused by the buildup of an antibody called IgA in the kidneys over time. In people with IgAN, abnormal IgA and other antibodies form clusters that build up in the small filters of the kidneys, which leads to inflammation and damage. Felzartamab is designed to target certain immune cells that produce these abnormal antibodies. This study will focus on participants who have protein in their urine (proteinuria) as a result of damaged kidneys. The main goal of the study is to learn about the effect felzartamab has on proteinuria. The main question that researchers want to answer is: • How much does the amount of protein in the urine change from the start of the study to Week 36? Researchers will learn about the effect felzartamab has on the kidneys' ability to filter blood. They will also learn more about the safety of felzartamab and how it is processed by the body. The study will be done as follows: * Participants will be screened to check if they can join the study. * Participants will be randomized to receive either felzartamab or a placebo. A placebo looks like the study drug but contains no real medicine. * Neither the researchers nor the participants will know what the participants will receive. * Participants will receive felzartamab or placebo as intravenous (IV) infusions. The treatment period will last 24 weeks. * Afterwards, participants will enter a follow-up period which will last 80 weeks. * In total, participants will have 17 study visits. Participants will stay in the study for about 2 years.

This study is open to adults and adolescents aged 12 to under 18 with bronchiectasis. People can participate in this study if they produce sputum and have had flare-ups (also called exacerbations). The purpose of this study is to find out whether a medicine called BI 1291583 helps people with bronchiectasis. Participants are put into 2 groups randomly, which means by chance. One group takes BI 1291583 tablets and the other group takes placebo tablets. A placebo tablet looks like the BI 1291583 tablet but does not contain any medicine. Participants take 1 tablet once a day for up to 1 year and 6 months. Participants are in the study for up to 1 year and 8 months. During this time, participants visit the study site up to 10 times and get about 13 phone calls from the site staff. Participants regularly complete a diary on a smartphone about their bronchiectasis symptoms and study doctors regularly check for any changes. The study doctors document when participants experience flare-ups. The number of flare-ups is compared between the participants who receive BI 1291583 and those who receive the placebo. The study doctors also regularly check participants' health and take note of any unwanted effects.

This study is open to adults with certain kidney conditions, including secondary focal segmental glomerulosclerosis (sFSGS), treatment-resistant primary minimal change disease (TR-pMCD), Alport Syndrome (AS), and treatment-resistant primary membranous nephropathy (TR-pMN). Adolescents with treatment-resistant primary MCD can also participate in this study. The purpose of this study is to find out whether a medicine called BI 764198 helps people with these kidney conditions. Participants are put into 2 groups randomly, which means by chance. One group takes BI 764198 tablets, and the other group takes placebo tablets. Placebo tablets look like BI 764198 tablets but do not contain any medicine. Participants take a tablet once a day for 20 weeks. All participants also continue their standard medication for their kidney condition during the study. Participants have twice the chance of being placed in the BI 764198 group than in the placebo group. Participants are in the study for about 7 months. During this time, they visit the study site 6 times and have 3 phone calls. Doctors regularly test the protein levels in participants' urine by collecting urine samples. They also check kidney function by taking blood samples. The results are compared between the two groups to see whether the treatment works. The doctors also regularly check participants' health and take note of any unwanted effects.

This is a trial to evaluate the efficacy, safety, and tolerability of adagrasib plus pembrolizumab plus platinum-doublet chemotherapy versus placebo plus pembrolizumab plus platinum-doublet chemotherapy in participants with previously untreated, locally advanced or metastatic NSCLC with KRAS G12C mutation

The Phase 2 portion of this study evaluates the efficacy and safety of MRTX849 monotherapy and in combination with pembrolizumab in cohorts of patients with advanced NSCLC with KRAS G12C mutation and any PD-L1 TPS and who are candidates for first-line treatment. The Phase 3 portion of the study compares the efficacy of adagrasib in combination with pembrolizumab versus pembrolizumab in patients with unresectable, locally advanced or metastatic squamous or nonsquamous NSCLC with KRAS G12C mutation and PD-L1 TPS \>=50% and who are candidates for first line treatment.

The ICGG Gaucher Registry is an ongoing, international multi-center, strictly observational program that tracks the routine clinical outcomes for patients with Gaucher disease, irrespective of treatment status. No experimental intervention is involved; patients in the Registry undergo clinical assessments and receive care as determined by the patient's treating physician. The objectives of the Registry are: * To enhance understanding of the variability, progression, identification, and natural history of Gaucher disease, with the ultimate goal of better guiding and assessing therapeutic intervention. * To assist the Gaucher medical community with the development of recommendations for monitoring patients, and to provide reports on patient outcomes, to optimize patient care. * To characterize the Gaucher disease population. * To evaluate the long-term effectiveness of imiglucerase and of eliglustat. Gaucher Pregnancy Sub-registry: The primary objective of this Sub-registry is to track pregnancy outcomes, including complications and infant growth, in all women with Gaucher disease during pregnancy, regardless of whether they receive disease-specific therapy. No experimental intervention is given; thus a patient will undergo clinical assessments and receive standard of care treatment as determined by the patient's physician.If a patient consents to this Sub-registry, information about the patient's medical and obstetric history, pregnancy, and birth will be collected, and, if a patient consents to data collection for her infant, data on infant growth through month 36 postpartum will be collected.

The Fabry Registry is an ongoing, international multi-center, strictly observational program that tracks the routine clinical outcomes for patients with Fabry disease, irrespective of treatment status. No experimental intervention is involved; patients in the Registry undergo clinical assessments and receive care as determined by the patient's treating physician. The primary objectives of the Registry are: * To enhance the understanding of the variability, progression, and natural history of Fabry disease, including heterozygous females with the disease; * To assist the Fabry medical community with the development of recommendations for monitoring patients and reports on patient outcomes to help optimize patient care; * To characterize and describe the Fabry population as a whole; * To evaluate the long-term safety and effectiveness of Fabrazyme® Fabry Pregnancy Sub-registry: This Sub-registry is a multicenter, international, longitudinal, observational, and voluntary program designed to track pregnancy outcomes for any pregnant woman enrolled in the Fabry Registry, regardless of whether she is receiving disease-specific therapy (such as enzyme replacement therapy with agalsidase beta) and irrespective of the commercial product with which she may be treated. Data from the Sub-registry are also used to fulfill various global regulatory requirements, to support product development/reimbursement, and for other research and non-research-related purposes. No experimental intervention is given; thus a patient will undergo clinical assessments and receive standard of care treatment as determined by the patient's physician. If a patient consents to this Sub-registry, information about the patient's medical and obstetric history, pregnancy, and birth will be collected, and, if a patient consents to data collection for her infant, data on infant growth through month 36 postpartum will be collected.

The purpose of this study is to provide efficacy and safety data for remibrutinib in patients with secondary progressive multiple sclerosis (SPMS)

Orelabrutinib is a CNS-penetrable BTK inhibitor. This is a phase 3, randomized, double-blind, parallel-group, multicenter study to evaluate the efficacy and safety of orelabrutinib compared with placebo in patients with PPMS. Patients will be treated for approximately 30 to 60 months, with a minimum treatment duration of 12 months. The study will enroll approximately 705 subjects in a 2:1 randomization (orelabrutinib: placebo), globally.

This observational, multicentric prospective study aims to understand the prevalence and burden of antimicrobial resistance (AMR) in people with chronic lung diseases (CLD) across the globe, understanding also geographical variability and the risk factors for acquiring AMR. The primary objective is to understand the prevalence and burden of AMR in CLD both at a local and gloval level. Secondary objectives include: 1) understanding susceptibilities models of high priority pathogens in people with CLD; 2) understanding geographic variability in the prevalence of AMR in CLD; 3) analysing risk factors for AMR in CLD Participants will be recruited

The OPTION-EMEA post-market study is intended to gather real world clinical data for patients undergoing AF ablation with the FARAPULSE™ Pulsed Field Ablation (PFA) System and subsequent left atrial appendage closure (LAAC) with a WATCHMAN FLX™ Pro or WATCHMAN FLX device during one interventional case. The study will include patients that are clinically indicated for atrial fibrillation (AF) ablation using the FARAPULSE PFA System and for left atrial appendage closure (LAAC) treatment using a WATCHMAN FLX Pro or WATCHMAN FLX device as part of the patient's standard of care determined by their doctor's medical judgement.

This trial is to investigate if in patients with clinically node positive breast cancer undergoing upfront surgery, treatment with TAS and ART is superior to ALND in terms of arm-related Quality of Life (QoL) and occurrence of lymphedema two years after randomization.

Psoriatic arthritis (PsA) is a chronic inflammatory disease that affects the joints and skin in people who have psoriasis (PsO). The main aim of the study is to know how well zasocitinib (TAK-279) works in participants with active PsA who have not previously been treated with biologic disease-modifying antirheumatic drugs. The participants will be treated with either zasocitinib, active comparator, or placebo. Participants will be in the study for up to 60 weeks.

The purpose of this protocol is to evaluate the efficacy and safety of tulisokibart in participants with moderately to severely active Crohn's disease. Study 1's primary hypotheses are that at least 1 tulisokibart dose level is superior to placebo in the proportion of participants achieving clinical remission per Crohn's Disease Activity Index score (\<150, US/FDA) or per stool frequency and abdominal pain score (EU/EMA) and in the proportion of participants achieving endoscopic response at Week 52 (US/FDA and EU/EMA), and that at least 1 tulisokibart dose level is superior to placebo in the proportion of participants achieving clinical remission per Crohn's Disease Activity Index score (\<150, US/FDA) or per stool frequency and abdominal pain score (EU/EMA) and in the proportion of participants achieving endoscopic response at Week 12 (US/FDA and EU/EMA). Study 2's primary hypothesis is that at least 1 tulisokibart dose level is superior to placebo in the proportion of participants achieving clinical remission per Crohn's Disease Activity Index score (\<150, US/FDA) or stool frequency and abdominal pain score (EU/EMA) and in the proportion of participants achieving endoscopic response at Week 12 (US/FDA and EU/EMA).

The main purpose of this study is to evaluate the safety and effectiveness of LY4268989 when compared to placebo in adult participants with moderately to severely active ulcerative colitis (UC). The study drug will be administered orally. The study will last up to approximately 108 weeks, excluding screening.

This is a Phase 2, randomized, double-blind, placebo-controlled, multicenter study to evaluate the efficacy and safety of 3 active dose regimens of MORF-057 in adult study participants with moderately to severely active Crohn's disease (CD).

Alopecia areata (AA) is a disease that happens when the immune system attacks hair follicles and causes hair loss. AA usually affects the head and face, but hair loss can happen on any part of the body. The purpose of this study is to assess how safe, effective, and tolerable upadacitinib is in adolescent and adult participants with severe AA. Upadacitinib is an approved drug being investigated for the treatment of AA. In Study 1 and Study 2 and Study 4 Period A, participants are placed in 1 of 3 groups, called treatment arms. Each group receives a different treatment. There is a 1 in 5 chance that participants will be assigned to placebo. In Study 1 and Study 2 and Study 4 Period B, participants originally randomized to upadacitinib dose group in Period A will continue their same treatment in Period B. Participants originally randomized to Placebo in Period A will either remain on placebo in Period B, or be randomized in 1 of 2 groups, based off of their Severity of Alopecia Tool (SALT) score. Participants who complete Study 1, Study 2 or Study 4, can join Study 3 and may be re-randomized to receive 1 of 2 doses of upadacitinib for up to 108 weeks. Around 1500 participants with severe AA will be enrolled in the study at approximately 280 sites worldwide. Participants will receive oral tablets of either upadacitinib or placebo once daily for up to 160 weeks with the potential of being re-randomized into a different treatment group at Weeks 24 and 52. Participants will be followed up for up to 30 days after last study drug dose. There may be higher treatment burden for participants in this trial compared to their standard of care. Participants will attend regular visits during the study at a hospital or clinic. The effect of the treatment will be checked by medical assessments, blood tests, checking for side effects and completing questionnaires.

The X-TOLE3 Phase 3 clinical trial is a randomized, double-blind, placebo-controlled study that will evaluate the clinical efficacy, safety and tolerability of XEN1101 administered as adjunctive therapy in focal-onset seizures.

This is a Phase 3, multicenter, randomized, double-blind, placebo-controlled study to evaluate the clinical efficacy, safety, and tolerability of XEN1101 administered as adjunctive treatment in primary generalized tonic-clonic seizures (PGTCS).

The purpose of the study is to assess the efficacy, safety and tolerability of PLENVU® by measuring its bowel cleansing success rate compared to sodium picosulfate in paediatric participants aged less than 18 years who are scheduled to undergo a colonoscopy. Colonoscopy is a crucial procedure that helps to diagnose and manage various gastrointestinal (GI) conditions in paediatric patients. An adequate level of bowel preparation and cleansing is essential for effective colonoscopy to allow proper visualisation of the bowel.

Neovascular age-related macular degeneration (nAMD), also known as "wet" AMD, is the abnormal growth of new blood vessels in the light-sensitive tissue at the back of the eye called the retina. The purpose of this study is to assess how safe and effective Surabgene Lomparvovec is in treating participants with Neovascular age-related macular degeneration (nAMD). Surabgene Lomparvovec (ABBV-RGX-314) is an investigational gene therapy being developed for the treatment of neovascular age-related macular degeneration (nAMD). Participants will be placed into 1 of 3 groups, called treatment arms. Each group receives different treatment. Adult participants aged 50 and older years with a diagnosis of previously treated nAMD will be enrolled. Around 561 participants will be enrolled in the study at approximately 150 sites worldwide. Participants in groups 1 and 2 will receive a single subretinal dose of ABBV-RGX-314. Participants in group 3 will receive Ranibizumab as needed throughout the study. Ranibizumab will be given as an intravitreal injection (injection into the jelly-like tissue that fills the eyeball injection), and ABBV-RGX-314 will be given as a subretinal (between the retina and the back of the eye) injection. The Assessment Period begins after randomization (1:1:1) to one of the ABBV-RGX-314 treatment groups or control at Week -2 and lasts up to 5 years. There may be higher treatment burden for participants in this trial compared to their standard of care. Participants will attend regular monthly visits during the study at a hospital or clinic. The effect of the treatment will be checked by medical assessments, blood tests, checking for side effects and completing questionnaires.