Sankt Polten

Researchers are evaluating the safety and effectiveness of three different doses of MORF-057 in adults with moderately to severely active Crohn's disease (CD). This Phase 2 study is randomized, double-blind, placebo-controlled, and conducted at multiple centers. It aims to compare MORF-057 to placebo to see how well it works in reducing disease activity and symptoms in this patient population. Participants will first go through a 14-week induction period where they receive one of three doses of MORF-057 or a matching placebo, all given orally. After this, all participants will enter a 38-week maintenance phase where they receive open-label MORF-057. Those who complete these 52 weeks of treatment may continue in a 52-week long-term extension to further monitor treatment effects and safety. Throughout the study, participants will have evaluations to assess their response to treatment using endoscopic scoring at Week 14. Researchers will monitor safety, symptom changes, and disease activity over the full treatment and extension periods. Study visits will include assessments, questionnaires, and clinical monitoring to track participants' health and treatment adherence over time.

Researchers are studying patients with symptomatic obstructive hypertrophic cardiomyopathy (HCM) in the United States and Europe to understand their characteristics, treatment patterns, and outcomes over time. The study focuses on individuals who are receiving mavacamten, other treatments for obstructive HCM, or no treatment due to intolerance or failure of prior therapies. The research includes a United States sub-study to evaluate mavacamten's safety and a European sub-study to assess both its effectiveness and safety in real-world settings. Participants may receive mavacamten according to its product label or other symptomatic therapies such as beta-blockers, non-dihydropyridine calcium channel blockers, or disopyramide based on standard care. The study includes those starting mavacamten, currently on other treatments, or untreated due to intolerance or failure of prior therapy. Treatment is observed during routine clinical care without altering prescribed therapy. Data collection occurs over several years to monitor long-term outcomes. During the study, participants will be regularly assessed for heart function and symptoms, including measuring the left ventricular outflow tract gradient and monitoring the incidence of new or worsening heart failure up to five years. Researchers will gather information on patient health, treatment safety, and heart function changes through echocardiography and symptom evaluations. The study allows for long-term observation to better understand real-world treatment effects and outcomes in obstructive HCM patients.

Researchers are evaluating the effectiveness of anitocabtagene autoleucel compared to standard of care therapy in adults with relapsed or refractory multiple myeloma who have previously received one to three treatments, including an anti-CD38 monoclonal antibody and an immunomodulatory drug. The study is a Phase 3, randomized, open-label trial aiming to assess how well anitocabtagene autoleucel works versus existing therapies in this patient group. Participants will receive either a single infusion of anitocabtagene autoleucel, which is a CAR+ transduced autologous T cell therapy, or one of several standard of care treatments. The standard treatments include combinations involving drugs such as pomalidomide, bortezomib, dexamethasone, daratumumab, carfilzomib, cyclophosphamide, and fludarabine, administered either orally or intravenously/subcutaneously. After the treatment period, those receiving anitocabtagene autoleucel will enter a follow-up phase and then transition to a long-term follow-up study lasting up to 15 years. During the study, participants will be monitored for progression-free survival for up to four years and for minimal residual disease complete response rate at nine months. Researchers will assess disease progression, treatment safety, and other health markers. Follow-up includes regular evaluations to track the participant's response and overall health status, with continued long-term monitoring planned for those treated with anitocabtagene autoleucel.

This research aims to evaluate the effectiveness and safety of two different dose schedules of pegozafermin compared to a placebo in adults with metabolic dysfunction-associated steatohepatitis (MASH) who have liver fibrosis at stage F2 or F3. This phase 3 study focuses on improving liver fibrosis and steatohepatitis in this patient group, which involves chronic liver disease associated with metabolic dysfunction. Participants will receive either pegozafermin or a placebo through subcutaneous injections. The study compares two doses of pegozafermin to assess their impact on liver fibrosis and steatohepatitis. The treatment period lasts up to 52 weeks, with outcomes measured at this time point. During the study, participants will be monitored for improvements in liver fibrosis and resolution of steatohepatitis without worsening fibrosis by week 52. Researchers will also track the time until any disease progression occurs, up to 5 years. Throughout the trial, safety and efficacy will be carefully assessed through clinical evaluations and laboratory tests to ensure participant well-being.

Researchers are evaluating the anti-tumor activity of amivantamab combined with pembrolizumab and carboplatin compared to pembrolizumab, 5-fluorouracil (5-FU), and platinum therapy (carboplatin or cisplatin) in participants with recurrent or metastatic head and neck squamous cell carcinoma (HNSCC). This trial focuses on participants who have not received prior systemic treatment in the recurrent/metastatic setting. HNSCC is a type of cancer affecting the outer tissue layer of the mouth and throat and other head and neck regions. Participants will receive either amivantamab added to pembrolizumab and carboplatin or the standard care regimen of pembrolizumab, 5-FU, and a platinum agent (carboplatin or cisplatin). 5-FU will be given as an infusion over a 4-day period. The study is a phase 3, randomized, open-label, multicenter trial comparing these treatment combinations. During the study, researchers will monitor overall survival and the objective response rate using standard tumor evaluation criteria for up to about 3 years and 7 months. Participants will undergo assessments to measure disease response, including imaging and other evaluations, to track how well the treatments work. Safety and side effects will also be monitored throughout the trial period.

Researchers are evaluating how well elacestrant works compared to standard endocrine therapy in adults with node-positive, Estrogen Receptor-positive (ER+), Human Epidermal Growth Factor-2 negative (HER2-) early breast cancer who are at high risk of the cancer returning. This is a Phase 3 global, multicenter, randomized, open-label study focusing on participants who have had early invasive breast cancer removed and meet specific receptor and risk criteria. The study aims to understand which treatment better prevents invasive breast cancer over up to five years. Participants will receive either elacestrant or one of several standard endocrine therapies, including anastrozole, letrozole, exemestane, or tamoxifen, all given as oral tablets. Treatments will be administered according to the study plan, with careful monitoring throughout the trial. The study includes adults who have already received between 24 and 60 months of prior endocrine therapy, with or without certain inhibitors, and who have completed or stopped these treatments as required. During the study, participants will be monitored for invasive breast cancer-free survival for up to five years. Researchers will perform regular assessments to track treatment effects, side effects, and cancer recurrence. The study also includes safety monitoring and may involve additional tests or evaluations as needed to ensure participant well-being throughout the trial.

Multiple myeloma is a cancer affecting plasma cells in the bone marrow. Researchers are evaluating how well Immune Globulin Infusion (human), 10% (IGI, 10%) can help prevent infections in adults with multiple myeloma receiving B-cell maturation antigen (BCMA) x CD3-directed bispecific antibody therapy. This phase 3 study aims to compare primary infection prevention using IGI, 10% versus secondary infection prevention in this patient group. Participants will be randomly assigned to one of two groups: the primary infection prevention group will receive IGI, 10% infusions for 12 months, while the secondary infection prevention group will receive IGI, 10% only if they develop a serious infection during the 12-month study period. The IGI, 10% is given intravenously. The study includes a screening period of up to 8 weeks, followed by treatment and monitoring. During the study, participants will attend 15 clinic visits if on a 4-week dosing schedule or 19 visits if on a 3-week dosing schedule, with total participation lasting up to 14 months. Researchers will monitor the time to first serious infection over 12 months. Participants will undergo evaluations to assess infection status and treatment safety throughout the study.

This research aims to evaluate the safety and effectiveness of iza-bren, a bi-specific antibody-drug conjugate targeting EGFR and HER3 with a topoisomerase inhibitor, compared to the treatment of physician's choice (paclitaxel, nab-paclitaxel, carboplatin plus gemcitabine, or capecitabine). The study focuses on patients with previously untreated, locally advanced, recurrent inoperable, or metastatic triple-negative breast cancer (TNBC) or estrogen receptor (ER)-low, HER2-negative breast cancer who are not eligible for anti-PD(L)1 or endocrine therapies. The trial is conducted in two phases, phase 2 and phase 3, to thoroughly assess these treatments.

This research aims to evaluate the effectiveness of trastuzumab deruxtecan (T-DXd) in adult patients with advanced HER2-positive gastric or gastroesophageal junction (GEJ) adenocarcinoma who have previously been treated with a trastuzumab-based regimen. The study also collects patient demographic and clinical information, treatment patterns, and safety data including serious adverse events and drug reactions. It includes a disease registry for patients receiving conventional therapies in a real-world European setting. The study is non-interventional, meaning no investigational drugs are administered beyond standard care. Patients receiving T-DXd will be treated according to the Summary of Product Characteristics (SmPC), and data on conventional therapies such as platinum-fluoropyrimidine chemotherapy, nivolumab, ramucirumab-paclitaxel, taxane, irinotecan, and pembrolizumab will also be gathered. Treatment choices and administration follow the physician's decision and routine clinical practice. Participants will be monitored from baseline to about two years to assess the time to next treatment. Researchers will collect clinical data, treatment details, tolerability, and patient surveys to understand outcomes and safety. The study involves regular follow-up and data collection to track treatment effectiveness and patient experience in real-world settings.

Researchers are evaluating the overall survival of males with chemotherapy-naefve metastatic castration-resistant prostate cancer (mCRPC). This phase 3 randomized study compares treatment with xaluritamig plus abiraterone to the investigator's choice of docetaxel, cabazitaxel, or abiraterone. Participants must have confirmed prostate adenocarcinoma with metastatic lesions and evidence of disease progression despite prior androgen receptor pathway inhibitor therapy. The study includes two treatment groups: one receiving intravenous xaluritamig combined with oral abiraterone acetate, and the other receiving the investigator's choice of chemotherapy drugs docetaxel or cabazitaxel administered intravenously, or abiraterone taken orally. Participants intended for cabazitaxel must have had up to six cycles of prior docetaxel in the metastatic hormone-sensitive setting. Treatments are administered per protocol during the randomized phase. Participants will be monitored for overall survival for up to approximately 51 months. Eligibility requires adequate organ function and good performance status. The study excludes those with prior chemotherapy in the mCRPC setting, certain prior therapies, unresolved toxicities, or CNS metastases. Researchers will assess survival outcomes along with safety and treatment tolerability throughout the study duration.