Freiburg Im Breisgau

Researchers are investigating the drug bezuclastinib in an open-label, two-part Phase 2 study for patients with Advanced Systemic Mastocytosis (AdvSM), including Aggressive Systemic Mastocytosis (ASM), Systemic Mastocytosis with an Associated Hematologic Neoplasm (SM-AHN), and Mast Cell Leukemia (MCL). The study aims to evaluate the safety, effectiveness, and how the drug behaves in the body for these serious conditions. Bezuclastinib is given orally as tablets taken continuously in 28-day cycles. The study has two parts: Part I focuses on identifying safe and tolerable doses of bezuclastinib over 18 months, while Part II evaluates its effectiveness by measuring the objective response rate and confirming the relationship between drug exposure and response during another 18-month period. Participants will undergo assessments including clinical evaluations, laboratory tests, and monitoring of their disease status to determine treatment effects and safety. Researchers will track the drug's impact on the disease and patient health throughout the study, which involves continuous treatment and follow-up over the specified time frames.

Researchers are evaluating the safety, tolerability, and levels of the study drug SYX-5219 in healthy volunteers and people with moderate to severe atopic dermatitis (AD). This multi-part, Phase 1, first-in-human study includes participants aged 18 to 65 years. The study aims to understand how SYX-5219 behaves in the body and to assess its safety in different dosing scenarios, including single and multiple doses as well as food effects. The study is divided into three parts. Part 1 involves single ascending doses (SAD) and a food effect evaluation in up to 48 healthy volunteers, who receive oral capsules of SYX-5219 or placebo. Part 2 tests multiple ascending doses (MAD) in up to 24 healthy volunteers with multiple oral doses given over a treatment period. Part 3 enrolls up to 45 participants with confirmed active AD to receive SYX-5219 or placebo daily for up to 42 days. This part is conducted at multiple global sites. Participants will undergo safety and exploratory efficacy assessments during treatment and follow-up periods. Researchers will monitor adverse events from the date of consent through various time points depending on the study part, including up to 10 days after dosing in Part 1 and up to 56 days in Part 3. Assessments include laboratory tests, vital signs, ECGs, and clinical evaluations to gather information on safety, tolerability, and drug levels in blood and urine throughout the study duration.

Researchers are evaluating whether an investigational drug called OHB-607 can prevent Bronchopulmonary Dysplasia (BPD), a common chronic lung disease, in extremely premature infants. The study compares infants receiving OHB-607 alongside standard neonatal care to those receiving standard care alone to reduce the burden of this lung condition. This is a Phase 2b, multicenter, randomized, open-label study focused on safety and clinical efficacy. Participants will receive an intravenous infusion of OHB-607 from birth until reaching a postmenstrual age (PMA) of 29 weeks and 6 days. The study includes two arms: one group receives the investigational drug plus standard care, while the other group receives only standard neonatal care. The treatment period ends at 29 weeks plus 6 days PMA, after which infants are monitored. Throughout the study, researchers will track the incidence of severe BPD or death up to 36 weeks PMA, whichever occurs first. Assessments will include clinical evaluations and monitoring for safety and any side effects. The study also involves long-term follow-up to observe the infants' health outcomes beyond the treatment period. Participation involves consent from parents and collection of birth and medical history information.

Researchers are evaluating a combination treatment using BNT326 and BNT327 in adults with advanced or metastatic non-small cell lung cancer (NSCLC), including those with relapsed, progressive, or treatment-nafve disease. This multi-site, open-label study includes dose-finding and dose-expansion phases to investigate the safety, tolerability, and preliminary effectiveness of this combination therapy. The study targets patients whose tumors are advanced, metastatic, or recurrent with no curative treatment options available and includes participants with different genomic alterations. The study is divided into several parts: Part 1 is a dose escalation phase to find safe dose levels of BNT326 with BNT327; Part 2a expands the dose to further evaluate safety and initial efficacy; Part 2b focuses on dose optimization and understanding the contributions of each component. Participants receive intravenous infusions of BNT326 and BNT327, with some cohorts possibly receiving additional treatments such as pembrolizumab or standard chemotherapy. Treatment continues until disease progression, unacceptable side effects, withdrawal, or a maximum of 24 months. Dose levels for certain cohorts are determined based on earlier phase data, and some parts include randomization to different treatment groups. Participants undergo a screening period before starting treatment, followed by treatment, safety follow-up, efficacy follow-up, and long-term survival monitoring, totaling about 36 months. Researchers assess dose-limiting toxicities within the first 21 days of treatment and monitor adverse events, treatment interruptions, and objective response rates up to 36 months. Tumor measurements, safety labs, imaging, and patient health status are regularly evaluated. The study tracks tolerability and efficacy while ensuring participant safety throughout treatment and follow-up.

Researchers are evaluating the retention rates of two treatments, Upadacitinib (UPA) and tumor necrosis factor inhibitors (TNFi), in adults with moderate to severe active rheumatoid arthritis (RA). The study is observational, conducted in Germany, and aims to compare how long patients stay on each treatment under real-world conditions according to local labels and standard care. About 678 participants will be enrolled across approximately 80 sites in Germany. Participants will have been prescribed UPA or TNFi independently of the study, following approved labels and local regulations. The study will observe participants receiving either UPA or TNFi therapy over a period of up to 24 months. Participants will be followed for up to 24 months to assess treatment retention. Researchers will monitor how long participants remain on their prescribed treatment and collect related clinical data. The total study duration, including recruitment and follow-up, is expected to last about 48 months.

The trial investigates the use of volrustomig in participants with unresected locally advanced head and neck squamous cell carcinoma (LA-HNSCC) who have not shown disease progression after receiving definitive concurrent chemoradiotherapy (cCRT). The study aims to evaluate the efficacy and safety of volrustomig compared to observation in this patient population. Participants have tumors that express PD-L1 and the study is conducted as a Phase III, randomized, open-label, multi-center global trial. Participants are assigned to receive either volrustomig as sequential therapy following cCRT or to an observation group. The treatment period involves monitoring participants who have completed definitive cCRT but remain unresected and have no evidence of metastatic disease. The study focuses on participants with Stage III, IVA, or IVB LA-HNSCC according to AJCC criteria, who have not undergone tumor resection before cCRT and have not been treated with radiotherapy alone. During the study, participants are regularly evaluated for progression-free survival, with follow-up lasting up to approximately 8 years to assess long-term outcomes. Researchers will monitor safety and disease progression closely. The overall participation duration includes screening, treatment or observation, and extended follow-up to capture both efficacy and safety data over time.

Researchers are conducting a master protocol study called CAMPFIRE to efficiently carry out multiple clinical trials testing new drugs in children and young adults with cancer. This master protocol allows for adding new studies as new cancer drugs become available, focusing on the treatment of measurable or evaluable tumors in participants aged 1 to 39 years. The goal is to evaluate various drugs under a unified research plan to improve treatment options for young cancer patients. The study involves several investigational drugs administered either intravenously or orally, including Ramucirumab, Cyclophosphamide, Vinorelbine, Gemcitabine, Docetaxel, Abemaciclib, Irinotecan, and Temozolomide. Each drug is tested under specific clinical trials within the master protocol, with treatment schedules and dosing tailored to each drug. Participants receive these treatments following standard clinical procedures, with adjustments based on individual study protocols and treatment responses. Participants will be closely monitored throughout the trial, with assessments including performance status evaluations, laboratory tests to check organ and blood function, and pregnancy testing for females of childbearing potential. Researchers will track how many participants receive each treatment during the first four weeks and observe the duration of treatment benefits. Safety evaluations, adherence to contraceptive measures, and recovery from prior therapies are also part of the study monitoring. Participation duration and additional assessments depend on the specific trial and treatment plan assigned.

Researchers are evaluating whether the drugs retatrutide and tirzepatide can prevent major adverse liver outcomes (MALO) in adults with metabolic dysfunction-associated steatotic liver disease (MASLD) who are at high risk. This Phase 3 trial enrolls about 4,500 adults with MASLD identified by non-invasive tests indicating an increased likelihood of developing serious liver problems. The study aims to understand how these treatments might affect liver health over time compared to a placebo. Participants will be randomly assigned to receive either retatrutide, tirzepatide, or a placebo, all given by subcutaneous injection. The study will last approximately 224 weeks, during which participants may attend 25 to 30 clinic visits for monitoring and assessment. After the main study, eligible participants can join an optional 2-year extension where all will receive either retatrutide or tirzepatide regardless of their original group. Throughout the trial, participants’ liver function and disease progression will be closely monitored through various health assessments. Researchers will track the time to the first major adverse liver event as the main outcome. Safety and health status will be evaluated regularly during clinic visits, ensuring thorough observation over the long study period.

Researchers are evaluating ASTX030, a combination of azacitidine and cedazuridine, as a treatment for myeloid neoplasms including myelodysplastic syndromes (MDS), chronic myelomonocytic leukemia (CMML), and acute myeloid leukemia (AML). This multi-phase study includes Phase 1 through Phase 3 monotherapy arms and Phase 1 and Phase 2 combination therapy arms with venetoclax. The study aims to assess pharmacokinetics, safety, efficacy, and drug interactions over an approximate duration of 8 years. The study treatments involve oral administration of ASTX030 and azacitidine, with some arms including subcutaneous azacitidine for comparison. Phase 1 monotherapy includes dose escalation and expansion stages, while Phase 2 and Phase 3 monotherapy arms are randomized crossover studies comparing oral ASTX030 to subcutaneous azacitidine. The combination therapy arms explore ASTX030 combined with venetoclax in participants with treatment-nafve AML, either in an open-label randomized exploratory setting or a single-arm study. Participants undergo evaluations including pharmacokinetic measurements such as total cycle area under the curve (AUC) for drug exposure, assessment of treatment-emergent adverse events, and investigator-assessed complete response rates. Monitoring occurs at multiple timepoints up to 36 months in some study arms. Safety, efficacy, and drug interaction assessments are integral throughout the study, with follow-up periods extending up to 8 years.

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.