Vitoria Gasteiz

Researchers are studying children with Hypochondroplasia, a genetic condition affecting growth, to observe how their growth changes over time and to understand the clinical course of this condition. The study gathers detailed growth measurements and other important health variables to track development. This is an observational study conducted across multiple centers and countries, focusing on children up to 15 years old with confirmed genetic diagnosis of Hypochondroplasia. The study does not involve active treatments but monitors participants regularly over a long period, up to 15 years. Every six months, various growth measurements are taken, including annualized growth velocity, height, body mass index (BMI), and ratios of different body segments such as upper to lower body, leg length ratios, and arm span to height. These detailed assessments help track changes in physical growth and body proportions over time. Participants will attend scheduled visits every six months for up to 15 years, during which researchers will measure and record growth parameters and body ratios. The study aims to capture changes in growth patterns and body measurements continuously throughout the study period. No specific interventions are given, but the study carefully monitors participant growth and development to better understand Hypochondroplasia and its effects on children.

Researchers are evaluating the effectiveness and safety of a combination treatment called triple therapy, which includes bempedoic acid, ezetimibe, and either atorvastatin or rosuvastatin. This study focuses on patients with primary hypercholesterolemia or mixed dyslipidemia who are at high or very high cardiovascular risk. The goal is to understand how well this combination lowers LDL cholesterol (LDL-C) in a real-world clinical setting. The study observes patients who have already started triple therapy within the last four weeks. No drugs are administered as part of this study; instead, it monitors the ongoing treatment with bempedoic acid combined with ezetimibe and either rosuvastatin or atorvastatin. The study measures LDL-C changes from baseline to eight weeks after starting triple therapy and continues follow-up for one year to assess lipid goal achievement, adherence to therapy, treatment changes, laboratory value shifts, and occurrence of cardiovascular events. Participants will have their LDL-C levels and other lab values assessed at baseline, eight weeks, and one year after starting triple therapy. Researchers will collect data on adverse events, adherence to treatment, and cardiovascular outcomes such as heart attack, stroke, death from cardiovascular causes, and coronary procedures during the follow-up year. The study also tracks treatment pathways and changes over this period to better understand real-world use and effectiveness of this triple therapy approach.

Researchers are studying the safety and effectiveness of brenipatide, given alongside standard treatment, compared to a placebo with standard treatment, to see if it can delay the return of symptoms in adults with major depressive disorder. This is a Phase 3, randomized, double-blind study involving adult participants aged 18 to 75 years. The trial is designed to assess how long it takes for depression symptoms to relapse after starting the adjunctive treatment. Participants will receive either brenipatide or placebo, both administered by subcutaneous injection, in addition to their stable standard of care medication. The study has three main periods: a screening period lasting about one month, followed by a treatment phase of at least 12 months where participants receive the assigned injections, and finally a follow-up period of roughly two months. The total time in the study can be shorter if symptoms worsen or if a participant withdraws. During the trial, participants will need to attend scheduled visits, self-inject the study drug, maintain study diaries, and complete questionnaires. Researchers will monitor participants closely to determine the time until relapse of major depressive disorder symptoms occurs. Safety and adherence to study procedures will be tracked throughout the trial, with the primary outcome measuring the number of days from randomization until relapse.

Narcolepsy Type 1 (NT1), Narcolepsy Type 2 (NT2), and Idiopathic Hypersomnia (IH) are rare sleep disorders causing excessive daytime sleepiness, making it difficult for people to stay alert during daily activities like school, work, or driving. NT1 often includes sudden muscle weakness called cataplexy triggered by strong emotions, while NT2 does not have cataplexy. People with IH feel tired even after long sleep and may have trouble waking up. This study aims to evaluate the safety, tolerability, and effects of ORX750, a drug designed to mimic orexin, a brain protein that helps keep people awake, in individuals with NT1, NT2, and IH. Participants will be randomly assigned to receive either ORX750 capsules or matching placebo capsules in this Phase 2a trial. The study will compare the drug against placebo to learn about its safety, how the body processes it, and its potential to reduce sleepiness and improve symptoms in these conditions. Participants must stop all other narcolepsy or hypersomnia medications and follow study requirements throughout the trial. During the study, researchers will monitor participants for treatment-related side effects, changes in laboratory tests, vital signs, heart electrical activity (ECG), and any suicidal thoughts or behaviors up to day 35. The main outcomes focus on safety and tolerability of ORX750. The study includes adults aged 18 to 65 years with specific diagnoses of NT1, NT2, or IH and tracks their response and health closely during the trial period.

Idiopathic Hypersomnia (IH) is a condition where adults feel very sleepy during the day, especially in the morning, even after getting plenty of sleep at night. They may find it hard to wake up and stay alert, affecting their ability to focus, think clearly, and carry out daily activities. This study is designed to evaluate the safety and tolerability of TAK-360, a drug that mimics orexin, a brain chemical that helps keep people awake, in adults with IH. Researchers also want to find out if TAK-360 helps improve wakefulness and determine the appropriate dose needed. Participants will be randomly assigned to receive either TAK-360 tablets or matching placebo tablets, which look the same but contain no active medicine. This study is a Phase 2 trial with a double-blind design, meaning neither the participants nor the researchers know who receives TAK-360 or placebo, to fairly assess the treatment's effects. The study focuses on finding the right dose of TAK-360 and monitoring its safety and tolerability. During the study, researchers will track treatment-emergent adverse events up to week 8 to evaluate safety. Participants will be monitored closely for how well they tolerate TAK-360 and any side effects. The study includes adults aged 18 to 70 years with a diagnosis of IH. Researchers will collect various health information to understand the drug's effects and ensure participant safety throughout the trial.

Researchers are evaluating the safety and tolerability of TAK-861 in people with narcolepsy type 1 (NT1) who have already been exposed to TAK-861 in earlier studies. The study also aims to observe improvements in symptoms such as excessive daytime sleepiness and the frequency of cataplexy episodes. This long-term extension trial continues from previous phase 2 and phase 3 trials and includes participants who completed those earlier studies. All participants in this trial will receive TAK-861 tablets. Those who were previously given a placebo in parent trials will be randomly assigned to a dose of TAK-861. The study plans to enroll up to 500 participants worldwide and will last approximately 5 years, or until the study is stopped or the drug is approved and launched. Participants will visit clinics multiple times, with some visits possibly done at home, and will have a follow-up check 4 weeks after their last dose. During the study, participants will be monitored for treatment-emergent adverse events from the time they consent until 4 weeks after their final dose, covering up to about 5 years. Researchers will assess safety and tolerability regularly through these visits and follow-ups. The focus is on identifying any side effects and understanding the long-term effects of TAK-861 in people with NT1.

Researchers are evaluating the safety, tolerability, and potentially effective doses of TYRA-300, a selective inhibitor targeting FGFR-3, in children aged 3 to 10 years with achondroplasia who have open growth plates. This Phase 2, multicenter, open-label study includes three groups: the Sentinel Safety Cohort, Cohort 1, and Cohort 2, to better understand treatment effects and appropriate dosing in this pediatric population. Participants will receive TYRA-300 starting at an initial dose of 0.125 mg/kg, with possible dose increases up to 0.50 mg/kg based on specific criteria in the study protocol. The study examines the effects of this treatment across different cohorts, including children naive to prior growth-accelerating therapies and those who have received such treatments before. The treatment is given orally and monitored closely for safety and effectiveness. During the study, children will be regularly assessed for treatment-related side effects up to 12 months using a standardized adverse event rating system. Growth velocity will be measured over 12 months for certain groups to evaluate changes from baseline. Safety evaluations, physical exams, and imaging to confirm growth plate status will be conducted. The study also tracks participants' ability to stand and walk independently and monitors medication adherence throughout the study period.

Psoriatic arthritis (PsA) is a chronic inflammatory condition that affects the joints and skin in people with psoriasis. This study aims to evaluate how well zasocitinib (TAK-279) works in adults with active PsA, considering their prior treatment experiences with specific medications. The study is a Phase 3 trial that compares zasocitinib to a placebo in participants who have or have not been treated with biologic medicines. Participants will receive either zasocitinib tablets or a matching placebo. The study is randomized, double-blind, and placebo-controlled. Treatment will continue with monitoring over a period of up to 60 weeks to assess the effects and safety of zasocitinib. During the study, participants will undergo assessments of joint and skin symptoms, including tender and swollen joint counts and evaluations of psoriatic skin lesions. Researchers will measure how many participants achieve a significant improvement in their arthritis symptoms by Week 16. Safety and response will be monitored throughout the study period, with detailed follow-up visits and evaluations to understand the treatment's impact over time.

Researchers are evaluating the ability of dapirolizumab pegol (DZP) added to standard care medications to improve moderate to severe systemic lupus erythematosus (SLE) symptoms over the long term. This Phase 3 trial focuses on participants aged 16 and older who have active SLE with specific disease activity and serological markers. The goal is to assess clinical improvement using the British Isles Lupus Assessment Group Disease Activity Index 2004 (BILAG 2004)-based Composite Lupus Assessment (BICLA) at Week 48. Participants will be randomly assigned to receive either dapirolizumab pegol (DZP) or placebo at scheduled times alongside their stable standard of care treatments. Standard medications include antimalarials combined with glucocorticoids and/or immunosuppressants or glucocorticoids and/or immunosuppressants alone if antimalarials are not suitable. The study is double-blind and placebo-controlled, ensuring unbiased comparison between the two groups. Throughout the study, participants will undergo regular assessments to monitor disease activity and treatment safety up to Week 48. Researchers will track responses based on disease activity indices and monitor for any adverse effects. The study includes careful screening and follow-up evaluations to understand the long-term effects of adding DZP to usual care in people with moderately to severely active SLE.

Researchers are evaluating whether different doses of the medicine called BI 3000202 can help adults with moderate to severe systemic lupus erythematosus (SLE). This phase II study is designed to find the best dose of BI 3000202 for people living with this condition. Participants must have a confirmed diagnosis of SLE with specific disease activity and antibody markers. Participants are randomly divided into five groups. Four groups receive varying doses of BI 3000202, while one group receives a placebo that looks like the real medicine but contains no active drug. All participants continue their usual SLE treatments during the study. The tablets are taken daily for one year. During the study, participants visit the study site regularly for health checkups and to monitor any side effects. Researchers measure the treatment's effectiveness by the achievement of a Systemic Lupus Erythematosus Responder Index (SRI)-4 response at week 32. The total participation time is a bit longer than one year, during which safety and health are closely observed and compared between groups.