Oberhausen

This research focuses on patients with very low, low, or intermediate risk myelodysplastic syndromes (MDS) who have anemia but do not require blood transfusions and have not previously been treated with erythropoiesis-stimulating agents (ESA). The study aims to evaluate the effectiveness of luspatercept in these patients, including those with or without ring sideroblasts, in improving their anemia condition. This is a Phase II, open-label, single-arm study designed to explore the potential benefits of luspatercept in this specific group of MDS patients. All patients enrolled in the study will receive luspatercept injections at a dose of 1.75 mg/kg administered under the skin every three weeks on day 1 of each 21-day cycle. This treatment will continue for 24 weeks initially. Patients who show a positive erythroid response at week 25 will continue to receive the same dose of luspatercept until their response is lost or for a maximum expected duration of 18 months. Participants will be closely monitored through scheduled assessments including blood tests and bone marrow sampling during screening and treatment. The primary outcome measured is the erythroid response at the end of cycle 8, where each cycle lasts 21 days. Safety and treatment effectiveness will be evaluated throughout the study period, with ongoing follow-up to track patient outcomes and any adverse effects. The total participation duration varies depending on individual response to treatment.

Researchers are evaluating the safety and effectiveness of Cemiplimab as a first-line treatment in patients with locally advanced basal cell carcinoma (BCC) who have not previously been treated with hedgehog inhibitors. This open-label, single-arm, multicenter phase II trial aims to measure the objective response rate (ORR) at six months and monitor safety and tolerability throughout the study. The study focuses on patients with advanced BCC not suitable for surgery or radiotherapy or who have refused these options. Participants will receive Cemiplimab at a dose of 350 mg intravenously on day 1 of every 21-day cycle for up to 12 months, with a maximum of 17 cycles. Treatment will continue until disease progression, unacceptable toxicity, or completion of the 12-month treatment period. Tumor samples will be collected during the study to support research into biomarkers that may predict treatment response. During the study, patients will undergo treatment response assessments every 12 weeks, with follow-up continuing for up to 12 months after the last Cemiplimab dose or until death. Researchers will collect tumor tissue for translational research and evaluate safety and treatment effects over this period. The total participation duration could extend beyond one year depending on treatment and follow-up timelines.

The International Collaborative Gaucher Group (ICGG) Gaucher Disease Registry is an ongoing global observational program tracking routine clinical outcomes in patients diagnosed with Gaucher disease. It includes patients regardless of their treatment status and aims to improve understanding of the variability, progression, and natural history of Gaucher disease. The Registry also seeks to support the medical community by developing monitoring recommendations, characterizing the patient population, and evaluating long-term treatment effectiveness of imiglucerase and eliglustat. The Registry involves no experimental treatments; patients receive clinical assessments and care as directed by their treating physicians. Additionally, there is a Gaucher Pregnancy Sub-registry that monitors pregnancy outcomes, complications, and infant growth up to 36 months postpartum for women with Gaucher disease. This Sub-registry collects medical and obstetric history and pregnancy data for participants who consent, without altering their standard care. Participants provide data through routine clinical visits, and researchers collect medical information to better understand patient outcomes and optimize care. The Registry tracks outcomes over long periods, including up to 42 years, to support ongoing care improvements. Women in the Pregnancy Sub-registry have additional data collected on pregnancy and infant growth, contributing to comprehensive monitoring of Gaucher disease impacts during and after pregnancy.

Researchers are evaluating the effectiveness and safety of a subcutaneous cladribine treatment for patients with hairy cell leukemia who need therapy. This study focuses on patients who are either untreated or have only been pretreated with alpha-interferon. It aims to assess remission rates four months after treatment and whether a second cycle benefits those with a non-optimal response, defined by residual disease or partial remission. Participants receive cladribine 0.14 mg/kg as a subcutaneous bolus injection once daily for five consecutive days. If the response after the first cycle is not optimal, patients may receive a second cycle of the drug to improve their remission status. This treatment approach is being studied in a Phase 2/3 clinical trial setting. During the study, remission status is evaluated four months after the first treatment cycle. Patients undergo assessments to detect residual disease in bone marrow and peripheral blood. Researchers monitor the rate of complete remissions and any treatment-related toxicities. The overall health, laboratory tests, and disease markers are regularly checked throughout the study period to ensure safety and to measure the treatment's impact.