Troisdorf

Researchers are evaluating the real-world effectiveness, safety, and patient compliance of ribociclib combined with an aromatase inhibitor for adjuvant treatment in patients with hormone receptor-positive, HER2-negative early breast cancer at high risk of recurrence. This observational study also compares ribociclib treatment with abemaciclib plus endocrine therapy and endocrine therapy alone, aiming to better understand treatment decisions and clinical adoption in routine practice. The study is conducted across breast centers and gynecological practices to represent typical healthcare settings. Participants receive treatment based on their physicians clinical judgment without randomization or intervention assignment. The study collects data from patients treated with ribociclib plus aromatase inhibitor with or without luteinizing hormone-releasing hormone LHRH, abemaciclib plus endocrine therapy with or without LHRH, or endocrine therapy alone with or without LHRH. Baseline data and follow-up information on adverse events, quality of life, treatment adherence, and socio-economic factors are gathered over time. During the study, participants undergo assessments including evaluation of invasive disease-free survival up to 36 months, quality of life questionnaires, medication adherence reports, and monitoring of adverse events and treatment changes. Data on reasons for treatment decisions, patient perceptions, and healthcare provider involvement are also collected. The study duration extends up to 39 months to provide a comprehensive overview of treatment impact and patient experience in real-world clinical settings.

This research aims to observe the real-world use and effects of pegcetacoplan in adults diagnosed with Paroxysmal Nocturnal Hemoglobinuria PNH. As a new treatment with a unique mechanism of action, pegcetacoplans effectiveness and safety in routine medical practice are being studied to provide valuable information for patients, healthcare providers, and payers. The study will also gather data on blood transfusions and healthcare resource use before and after starting pegcetacoplan. Patients who have started pegcetacoplan treatment within the past 12 months or are prescribed the drug at enrollment will be included. Data collection includes retrospective information from up to 12 months before treatment start and prospective monitoring for approximately 36 months, with the total data period extending up to about 48 months. After stopping pegcetacoplan, patients remain in the study for 8 weeks to record any adverse events. Patients continue regular clinic visits, where data from each visit will be gathered. Participants will have data collected on various health measures such as hemoglobin levels, blood markers, transfusion needs, and patient and physician treatment satisfaction at regular intervals up to 36 months. Safety and adverse events will be monitored throughout. The main outcome measured is the change in hemoglobin level from treatment start to 6 months. This long-term observational study allows for comprehensive tracking of pegcetacoplans use and effects over time in usual care settings.

Researchers are evaluating the study drug Corabotase IPN10200 for treating Cervical Dystonia CD in adults. This condition causes abnormal movements and symptoms in the head and neck, including pain, stiffness, and headaches, possibly related to brain and genetic factors. The study compares Corabotase to a placebo to assess its safety and effectiveness, aiming for a longer-lasting treatment than current botulinum toxin injections. This is a Phase II, randomized, double-blind, placebo-controlled trial. Participants will be randomly assigned to receive one of several doses of Corabotase Dose A, Dose B, or possibly Dose C or placebo. The study drug is given by intramuscular injection into different muscles in the head, neck, and shoulders in a single treatment cycle on Day 1 of the treatment period. The study includes a screening period of up to 4 weeks followed by a 36-week treatment period with 10 in-clinic visits and one remote phone visit 12 visits for those receiving Dose C. During the study, participants will undergo physical and neurological exams, blood and urine sample collections, and clinical assessments. They will complete questionnaires to track symptoms and treatment effects. Researchers will measure changes in the Toronto Western Spasmodic Torticollis Rating Scale TWSTRS scores and other pain and disability scales at multiple time points up to week 36. Safety, antibody formation, and adverse events will also be closely monitored. Total participation lasts about 40 weeks.

Researchers are evaluating trastuzumab deruxtecan T-DXd as a treatment for adult patients with advanced HER2-positive gastric or gastroesophageal junction GEJ adenocarcinoma who have previously received a trastuzumab-based regimen. This study aims to assess the real-world effectiveness of T-DXd, patient characteristics, treatment patterns, and safety in this population. It also includes data collection on patients receiving conventional therapies for exploratory comparison. This is a non-interventional observational study where patients receive T-DXd or conventional therapies as part of routine clinical care according to approved guidelines SmPC. No investigational drugs are given. The study tracks patients starting T-DXd as a second-line or later treatment and collects data on other therapies such as chemotherapy and immunotherapy used in clinical practice. Participants will be followed for up to approximately 2 years from baseline to monitor outcomes including time to next treatment, changes in treatment, physician-reported safety events, use of prophylactic treatments, and quality of life using validated questionnaires. Data on physician visits, treatment discontinuation, and safety events will also be collected to understand treatment tolerability and patient experience in a real-world setting.

Researchers are evaluating the safety and effectiveness of different doses of Corabotase also called IPN10200 for treating adults with upper limb spasticity. This study aims to understand how Corabotase affects the body and to determine which doses provide the best balance of safety and benefit. The trial involves adult participants aged 18 to 70 years who have spastic hemiparesis following stroke or traumatic brain injury and meet other specific health criteria. The study includes several groups receiving different doses of Corabotase, Dysport, or placebo as single injections into various upper limb muscles. Participants are assigned randomly to groups, with some in dose-escalation cohorts and others in fixed-dose groups. The injections are given once, and doses are carefully adjusted to assess safety and response. The trial uses a double-blind design, meaning neither participants nor researchers know who receives which treatment. Participants will be monitored over nine months with regular assessments including physical exams, vital signs, lab tests, and evaluations of muscle spasticity using the Modified Ashworth Scale. Researchers will track any side effects, antibody development, and changes in muscle tone and disability. Patient and physician impressions of treatment effects and pain levels will also be recorded. This long-term follow-up helps researchers understand how the treatments work and their safety over time.

This research focuses on adults with advanced non-small cell lung cancer NSCLC who have been prescribed cemiplimab-based treatments as part of their standard care. The study aims to better understand how cemiplimab regimens are used, their benefits, and safety in real-world clinical practice across Europe. Participants receive cemiplimab either alone or combined with platinum-based chemotherapy. Data is collected during routine clinical visits about every three months while on treatment and then about every six months for up to two years after stopping cemiplimab. The study follows patients from starting cemiplimab until death, loss to follow-up, withdrawal, or the study end, spanning up to 72 months. During the study, participants complete questionnaires and undergo regular clinical assessments at their usual care visits. Researchers monitor overall survival, response rates, duration and timing of responses, progression-free survival, and time to next treatment. Safety is assessed by tracking treatment-related adverse events, immune-related effects, and infusion reactions throughout the study period.

Researchers are studying metastatic colorectal carcinoma mCRC patients who have a specific BRAFV600E mutation, which is linked to poorer outcomes compared to those without it. This mutation leads to shorter survival times after initial treatments, prompting the need for new therapy combinations. This research aims to observe how the drugs encorafenib and cetuximab work together in real-world settings for patients who have already received prior systemic therapies. This non-interventional, prospective, longitudinal study focuses on patients treated with encorafenib plus cetuximab following prior systemic therapy. The study collects data on treatment effectiveness, safety, and quality of life among a broader patient population in Germany, Austria, and Switzerland. Patients may have started treatment up to three months before joining the study or plan to start soon, and the study observes their outcomes without influencing treatment decisions. Participants will be monitored through data collection on their disease and treatment profiles, including patient and physician assessments, adverse events, and treatment details. The main outcome measured is overall survival at 12 months after starting treatment. Additional information gathered includes treatment duration, dose intensity, interruptions, and patient-reported quality of life using questionnaires. Safety and tolerability are also evaluated throughout treatment, with follow-up averaging nine months.

Researchers are evaluating the effect of adding capivasertib to fulvestrant compared with fulvestrant alone as neoadjuvant treatment for women with primary high-risk lobular breast cancer that is hormone receptor positive and HER2 negative. This phase II, open-label, randomized study focuses on measuring the complete cell cycle arrest CCCA by assessing Ki67 levels at baseline, week 2, and week 10. The study aims to identify patients who may benefit from combined treatment and potentially avoid chemotherapy, considering the unique characteristics of invasive lobular breast cancer. Participants are randomly assigned to one of two treatment groups. One group receives capivasertib orally twice daily for 4 days followed by a 3-day break for 2 weeks, then continues this capivasertib schedule combined with fulvestrant injections every 28 days for an additional 8 weeks, totaling four doses of fulvestrant. The other group receives fulvestrant injections alone on the same schedule for 10 weeks. Treatment continues until surgery, disease progression, unacceptable side effects, or patient withdrawal. During the study, all patients will undergo core biopsies before and after treatment to measure Ki67 levels. Researchers will assess safety, pathological response, breast conservation rates, and survival outcomes. Patients will be monitored throughout treatment and follow-up, which may last up to two years to assess invasive disease-free survival and overall survival. Additional therapies such as surgery, chemotherapy, or radiotherapy will be given as needed outside the trial according to standard care.

Researchers are investigating ribociclib combined with standard endocrine therapy for women with advanced hormone receptor-positive HR and HER2-negative breast cancer receiving first-line treatment. This phase IV, open-label study aims to understand how ribociclib works over time and identify patterns of resistance. The study also focuses on survival rates at 12 months and includes a comprehensive program to discover and validate biomarkers related to treatment outcomes. Participants will receive ribociclib once daily for 21 days followed by 7 days off, in 28-day cycles, alongside standard endocrine therapy prescribed by their doctors. The treatment follows the approved guidelines Summary of Product Characteristics for ribociclib. This trial plans to enroll 1000 female patients across 75 sites in Germany and involves extensive biomarker sampling from blood, tissue, and immune cells before, during, and after treatment or disease progression. During the study, participants will have regular assessments including survival monitoring at various time points up to 36 months, quality of life evaluations, and safety checks for treatment-related side effects. Samples for biomarker research will be collected to help understand treatment response. Follow-up includes collecting data on progression-free and overall survival, quality of life, and adverse events, with total study involvement lasting up to 36 months.

Researchers are evaluating whether patients with hormone-receptor positive, HER2-negative early breast cancer HRHER2- eBC who have intermediate to high risk of recurrence may benefit from an initial 5-year treatment with elacestrant, with or without a CDK 46 inhibitor, followed by standard endocrine therapy ET for an additional 0 to 2.5 years. This is compared to the current standard of care ET lasting between 5 and 7.5 years, with or without CDK 46 inhibitors. The study is designed for patients identified by genomic tests like Oncotype DX and response to preoperative endocrine therapy, aiming to improve long-term survival by targeting resistance mechanisms such as ESR1 mutations. Participants are randomly assigned to one of two groups one receiving elacestrant orally for 5 years, possibly combined with ribociclib a CDK 46 inhibitor for 3 years if indicated, and the other receiving standard endocrine treatments for 5 to 10 years with optional CDK 46 inhibitors as decided by their doctor. The treatments are evaluated in a parallel study design. The trial builds on previous findings in metastatic breast cancer and early breast cancer studies exploring endocrine therapy responses and genomic risk assessments. Throughout the study, participants undergo regular clinical assessments, including tumor pathology reviews, laboratory tests, and electrocardiograms to monitor safety and treatment effects. Researchers measure outcomes such as 5-year invasive disease-free survival and other survival metrics. Participants must comply with contraception requirements and be followed for treatment adherence and safety until study completion, which is planned for up to 2033.