Oberhausen

Researchers are evaluating a medicine called nerandomilast for adults with systemic sclerosis, a condition affecting the skin and internal organs. The study focuses on people with limited or diffuse cutaneous systemic sclerosis who have had symptoms for less than 7 years. The goal is to see if nerandomilast helps improve symptoms and slows disease progression while assessing how well the medicine is tolerated. Participants are randomly assigned to one of two groups one takes nerandomilast tablets, and the other takes placebo tablets that look identical but do not contain medicine. Tablets are taken twice daily. The study lasts from 1 to about 4 years, with regular visits to the study site and phone check-ins. During visits, participants have blood tests and assessments of skin thickening, lung function, and organ health to monitor changes and treatment effects. Throughout the study, doctors monitor participants overall health and any side effects. They regularly assess safety and tolerability of the treatment. The main outcome measured is the time to disease progression or death over up to 4 years. Other outcomes include changes in skin scores, lung capacity, physical function, and disease impact questionnaires. Participants health is closely followed during the study period.

This research aims to evaluate the effectiveness of luspatercept in adults with very low, low, or intermediate risk myelodysplastic syndromes MDS who have anemia but do not require blood transfusions and have not been treated with erythropoiesis-stimulating agents ESA. It focuses on patients who are transfusion-independent and have symptomatic anemia, studying the response to treatment in this specific group. All participants receive luspatercept administered as a subcutaneous injection at a dose of 1.75 mg per kg of body weight every 21 days. This treatment continues for 24 weeks initially, and for those who respond by week 25, therapy may extend for up to 1.5 years or until loss of response. This is a single-arm, open-label phase II study without a placebo group. Participants will have assessments at regular intervals, including blood tests and bone marrow sampling, to monitor blood cell counts and treatment response. The main outcome is erythroid response measured after eight treatment cycles each cycle is 21 days. Secondary outcomes include the duration of response, time to response, neutrophil and platelet responses, safety monitoring for adverse effects, and quality of life evaluations using validated questionnaires. The study lasts up to 48 months, including long-term follow-up.

Researchers are evaluating the effectiveness and safety of Cemiplimab as a first-line treatment for patients with locally advanced basal cell carcinoma BCC who have not previously received hedgehog inhibitor therapy. This phase II, open-label, single-arm trial aims to measure the objective response rate after six months of treatment in this patient group. Patients with advanced BCC who cannot undergo surgery or radiotherapy are included to explore new treatment options. Participants will receive Cemiplimab intravenously at a dose of 350 mg on day 1 of every 21-day cycle for up to 12 months or a maximum of 17 cycles. Treatment may stop earlier if intolerable side effects or disease progression occurs. Tumor samples will be collected for research to identify potential biomarkers that may relate to the drugs effectiveness. The study also includes follow-up visits every 12 weeks to monitor response and safety until death or for up to 12 months after the last dose. Throughout the study, patients will have regular assessments including tumor response evaluations every 12 weeks during treatment and follow-up. Researchers will monitor safety by tracking adverse events and laboratory parameters. The main outcome measured is the objective response rate at six months, with additional measures such as progression-free survival and overall survival tracked for up to 42 months. Participants are followed closely to understand both the benefits and risks of Cemiplimab in this setting.

Researchers are tracking the natural history and clinical outcomes of patients with Gaucher disease through the ICGG Gaucher Registry, an international, multi-center observational program. This registry does not involve any experimental treatments, but collects information to better understand the variability, progression, and identification of Gaucher disease, aiming to improve patient care and therapeutic guidance. It also evaluates the long-term use of treatments like imiglucerase and eliglustat. The study includes two groups patients with Gaucher disease who receive routine clinical assessments and standard care as determined by their physicians, and a Pregnancy Sub-registry for women with Gaucher disease who are pregnant or have been pregnant. The Pregnancy Sub-registry collects information on pregnancy outcomes, complications, and infant growth up to 36 months postpartum, regardless of whether the women receive disease-specific therapy. Participants undergo clinical assessments and receive care according to their treating physicians decisions. Data collected includes medical history, pregnancy and birth details for the sub-registry, and patient outcomes over time. The primary goals are to provide recommendations for monitoring Gaucher disease patients, report outcomes to optimize care, and track pregnancy and infant growth outcomes. This ongoing registry allows long-term follow-up without experimental interventions.

Researchers are evaluating the effectiveness and safety of a subcutaneous treatment using one cycle of cladribine for patients with hairy cell leukemia who need treatment. This study focuses on patients who are either untreated or have been previously treated only with alpha-interferon. The goal is to determine the rate of complete remission and to assess whether a second cycle of treatment benefits those with a non-optimal response, defined as detectable residual disease or partial remission four months after the initial treatment. Participants receive cladribine at a dose of 0.14 mgkg body weight administered subcutaneously as a bolus injection once daily for five consecutive days. After four months, remission status is evaluated. Patients showing non-optimal response may be considered for a second treatment cycle. This phase 23 trial is designed to optimize therapy for hairy cell leukemia by closely monitoring treatment response and toxicity. During the study, participants undergo evaluations including disease status assessment through bone marrow and blood tests, with a focus on remission rates four months post-treatment. Researchers track complete remission rates and monitor for residual disease. Safety is also monitored by assessing potential toxicities. Participants general health status and other laboratory tests are reviewed to ensure eligibility and ongoing suitability for treatment throughout the study period, which spans several months with follow-up assessments.