Bochum

Researchers are conducting a phase II, multicenter, open-label trial to investigate the combination of Fruquintinib and Tislelizumab in patients with microsatellite stable (MSS) or proficient mismatch repair (pMMR) metastatic colorectal cancer who do not have active liver metastases. The study aims to evaluate the effectiveness of this combination in comparison to a control treatment for this specific group of patients. Participants will be randomly assigned to one of two treatment groups. The experimental group will receive oral Fruquintinib (5 mg daily for 21 days in each 28-day cycle) along with intravenous Tislelizumab (400 mg every 42 days). The control group will be treated with oral Trifluridine/tipiracil (35 mg/m2 twice daily on days 1-5 and 8-12 of each 28-day cycle) plus intravenous Bevacizumab (5 mg/kg every 14 days). Treatment continues until disease progression, unacceptable side effects, patient choice, or a maximum of 15 months. During the study, patients will undergo regular assessments including imaging to monitor disease status and safety evaluations. Follow-up will continue for up to 18 months after the last patient enrolls or until death, withdrawal, or loss to follow-up. The main outcome measure is the efficacy of Fruquintinib combined with Tislelizumab in this patient population over a 54-month period.

Researchers are evaluating whether tirzepatide can improve ovarian dysfunction in premenopausal women with polycystic ovary syndrome (PCOS) who are overweight or have obesity. This phase IV clinical trial compares tirzepatide to a placebo to see if tirzepatide is better at improving menstrual irregularity and ovulation frequency in this group. Tirzepatide is already approved for diabetes and obesity, but its effects on ovarian function in PCOS are not yet known. Participants will be randomly assigned to receive either tirzepatide or placebo through weekly subcutaneous injections using prefilled pen injectors. Treatment lasts 72 weeks, starting with a 20-week dose escalation period to find the maximum tolerated dose, followed by a 52-week maintenance phase at that dose. If side effects occur, lower doses may be used. After treatment ends, a 4-week safety follow-up will be conducted, with an additional one-year long-term follow-up. During the study, participants will have a screening visit before starting treatment. Researchers will monitor menstrual cycles, ovulation frequency, and signs of ovarian dysfunction throughout the 72-week treatment. Safety assessments and questionnaires will be completed, and adherence to lifestyle, dietary, and exercise advice will be tracked. The main outcomes are improvement in menstrual irregularity at 72 weeks and ovulation frequency within 24 weeks after dose escalation. The study is conducted at five sites in Germany.

Researchers are studying oligodendrogliomas, a type of brain tumor identified by specific genetic changes such as IDH1 or IDH2 mutations and 1p/19q co-deletion. These tumors are classified as CNS WHO grade 2 or 3, and the study aims to determine the best treatment approach to improve survival while preserving brain function and quality of life. This phase 3 trial compares current standard treatments because existing data is unclear about the best timing and combination of chemotherapy and radiotherapy for these tumors. The study compares two treatment approaches for adults with newly diagnosed grade 2 or 3 oligodendrogliomas. One group receives an initial chemotherapy regimen of lomustine and temozolomide (CETEG) with delayed radiotherapy plus PCV chemotherapy at progression. The other group receives standard radiotherapy followed by PCV chemotherapy. Radiotherapy doses vary based on tumor grade. The study plans to assess if delaying radiotherapy and adjusting chemotherapy can maintain survival while reducing side effects. Participants will undergo regular monitoring including MRI scans every three months, neurological assessments, quality of life questionnaires, and annual cognitive testing. Researchers will measure qualified overall survival, defined as survival without decline in function, cognition, or quality of life. The study will last up to 10 years, with close tracking of side effects, tumor response, and patients' well-being to determine the best balance of treatment effectiveness and quality of life.

Researchers are evaluating the safety and potential benefits of the investigational drug BNT329 for people with advanced solid tumors that express the tumor marker CA19-9. This Phase I/IIa study aims to find the best dose of BNT329 by tracking side effects and their severity. It also explores how well the drug works by measuring participants' tumor responses and how long the tumor stays controlled. Additionally, the study examines how BNT329 moves through and affects the body. The trial has up to four parts (A, B, C, and D). Parts A, B, and C focus on increasing doses to assess safety and tolerability in participants with various advanced cancers expressing CA19-9, including pancreatic, bile duct, bladder, colorectal, gastroesophageal junction, endometrial, and ovarian cancers. Part B tests a more frequent dosing schedule, and Part C tests pre-treatment with a CA19-9-targeting monoclonal antibody before BNT329. Part D evaluates safety and early signs of effectiveness in pancreatic cancer patients who have had prior treatments. Participants receive BNT329 through intravenous infusion. Parts A, B, and C are non-randomized, while Part D randomizes participants to two different dose levels. Participants go through screening, treatment lasting up to two years, end-of-treatment visits, two safety follow-ups, and survival monitoring until death, withdrawal, or study end. Researchers monitor side effects, serious adverse events, dose changes due to side effects, and tumor response rates over time, with follow-up periods lasting up to 36 months. Safety assessments continue up to 60 days after the last dose, and long-term survival is tracked throughout the study.

Researchers are evaluating the safety and effectiveness of EscharEx (EX-03 5% formulation), a gel made from a sterile lyophilized powder containing proteolytic enzymes, compared to a placebo gel without enzymes. The study focuses on the treatment of Venous Leg Ulcers (VLU), which are wounds on the legs caused by poor venous blood flow. This Phase 3 trial will involve at least 216 adults with VLUs that range in size from 2 to 25 square centimeters and have lasted between 4 weeks and 12 months. Participants will be randomly assigned to receive either EscharEx or the placebo in a double-blinded setup, meaning neither patients nor researchers know which treatment is given. The study lasts up to 29 weeks and includes several stages: a screening period with two visits one week apart; a daily treatment phase with up to eight daily applications over two weeks to remove necrotic tissue; a weekly wound management period lasting up to 12 weeks with up to 13 visits, plus up to two weeks for confirming wound closure; and finally, a 12-week monthly follow-up with three visits to monitor wound closure durability. Throughout the study, researchers will visually assess the removal of dead tissue after each treatment application and monitor the time it takes for the wound to fully close. Patients will undergo standardized wound care and regular clinical evaluations, including measurements of the wound area. Safety and wound healing progress will be carefully tracked during all visits, ensuring comprehensive monitoring of treatment effects and wound status over the entire study duration.

Researchers are conducting a global, multicenter, prospective observational registry to study patients with Pompe disease, including those with late-onset Pompe disease (LOPD) and infantile-onset Pompe disease (IOPD). The study includes both patients who are untreated and those receiving approved Pompe disease therapies. The main goals are to assess the long-term safety and real-world effectiveness of these treatments, understand their impact on quality of life and patient-reported outcomes, and describe the natural history of untreated Pompe disease. Participants may be treated with various therapies including enzyme replacement therapies such as cipaglucosidase alfa delivered by intravenous infusion, alglucosidase alfa or avalglucosidase alfa once approved locally, and miglustat co-administered with ATB200. Patients not receiving any medical therapy for Pompe disease are also included. The study gathers data from both treated and untreated patients as they are managed in routine clinical practice. Throughout the study, participant data will be collected to monitor the frequency of adverse events and serious adverse events over a period of five years. Researchers will also evaluate treatment effectiveness, quality of life, and patient-reported outcomes during this time. This observational approach allows for long-term safety monitoring and understanding of Pompe disease progression in a real-world setting.

Researchers are evaluating various approved injectable and oral disease-modifying treatments (DMTs) in patients with relapsing multiple sclerosis (RMS) in Germany. This observational, non-interventional, multicenter, open-label study collects primary data prospectively over up to four years, alongside retrospective data. The study captures medical history, disease duration, laboratory values, disability scores (EDSS), MRI results, and relapse information to provide real-world insights into treatment use and outcomes. Patients receiving routine medical treatment with any approved injectable or selected oral DMTs—including ofatumumab, glatiramer acetate, interferon 21, teriflunomide, dimethyl fumarate, and diroximel fumarate—are enrolled without treatment allocation by the study. Two cohorts are observed: one treated primarily with injectable DMTs and another with injectable or oral DMTs. The core study period lasts about two years, with an optional extension providing an additional two years of observation, totaling up to four years. Follow-up visits and monitoring happen at the investigator's discretion and may include telemedicine. During the study, participants provide data through questionnaires and electronic case report forms. Routine clinical care procedures, such as diagnostic tests and monitoring, continue as usual. Researchers measure the proportion of patients continuing their baseline treatment at 24 months and collect ongoing clinical and imaging data. The study emphasizes real-world treatment patterns, safety, and disease activity over the extended follow-up period.

Researchers are evaluating the clinical utility of serum neurofilament light (sNfL) as a prognostic marker for disease activity in patients with relapsing multiple sclerosis (MS). This prospective, multicenter, observational, non-interventional study in Germany aims to understand how sNfL values can influence patient management and treatment decisions. The study focuses on patients treated with category 1 disease-modifying therapies (DMTs) who have incorporated sNfL testing into their care. Participants will either continue their current category 1 DMT, which includes therapies such as dimethylfumarate, glatiramer acetate, interferon beta, and teriflunomide, or switch to ofatumumab based on their physician’s clinical judgment. There is no treatment allocation by the study itself. Data collection will cover up to 24 months, and the frequency of visits and assessments will follow routine clinical practice without a fixed protocol. During the study, baseline and follow-up data will be gathered according to standard care recommendations, including clinical evaluations and sNfL measurements. Researchers will monitor the proportion of patients with high sNfL levels over time to assess disease activity. The observational period is flexible and guided by the treating physician, with no additional diagnostic or monitoring procedures beyond standard care. Participants will be followed for up to two years to better understand how sNfL influences treatment management in relapsing MS.

Researchers are evaluating the real-world effectiveness, safety, and tolerability of ribociclib combined with an aromatase inhibitor, with or without luteinizing hormone-releasing hormone (LHRH) therapy, for adjuvant treatment in patients with hormone receptor-positive, HER2-negative early breast cancer at high risk of recurrence. The study also compares data from patients treated with abemaciclib plus endocrine therapy with or without LHRH, and those receiving endocrine monotherapy with or without LHRH. This observational study aims to understand treatment decisions and clinical use of ribociclib after its approval, collecting socio-economic data, quality of life, and patient compliance information. Participants receive treatment based on their physician's clinical judgment without study-assigned interventions. The treatments observed include ribociclib with an aromatase inhibitor LHRH, abemaciclib with endocrine therapy LHRH, or endocrine monotherapy LHRH. The study is conducted in various breast cancer centers and gynecological practices in Germany and Austria to represent local healthcare settings. Participants undergo assessments to monitor treatment effectiveness, safety, quality of life, and adherence to therapy over time. Data collected include clinical outcomes, adverse events, socio-economic status, and patient-reported compliance. The primary outcome measured is invasive disease-free survival over 36 months. This information will help inform clinical decision-making and improve outcomes for patients with early breast cancer in routine practice.

Researchers are assessing the effectiveness of avapritinib (BLU-285) in managing indolent systemic mastocytosis (ISM) among patients in Germany. This non-interventional study aims to fill gaps in understanding the natural history and treatment outcomes of ISM, particularly in participants whose symptoms are not adequately controlled with current symptomatic treatments. The study observes participants in real-world clinical settings without altering their standard care. Avapritinib is given as an oral tablet, and treatment decisions are made by healthcare providers as part of routine care for those with moderate to severe ISM symptoms. Participants start avapritinib treatment at the provider's discretion, following approved prescribing guidelines. No other interventions are assigned by the study, emphasizing observation of usual care in a real-world environment. Participants are followed for up to 24 months, during which researchers monitor changes in quality of life related to mastocytosis using the Mastocytosis Control-quality of Life (MC-QoL) Questionnaire, assessed at the start and at 6 months. The study also collects data on symptom control and safety, focusing on participants who have not previously used avapritinib. This long-term observation helps understand treatment effectiveness and patient experiences over time.