Woluwe Saint Lambert

Researchers are comparing INCA033989 with the best available therapy for adults who have essential thrombocythemia ET with a CALR mutation and have previously received cytoreductive treatment. The study aims to evaluate the effects of these treatments on this specific patient group. It is a Phase 3 clinical trial sponsored by Incyte Corporation to assess treatment responses and safety. Participants will be randomly assigned to receive either INCA033989 administered intravenously or the best available therapy chosen by their doctor. The treatments are given according to the study protocol. The study focuses on treatment outcomes over a period of weeks, including response durability and symptom changes, with assessments at specified timepoints. During the study, participants will have regular visits to monitor their clinical and hematologic responses, symptoms, and any side effects. Researchers will collect data on mutation levels, symptom questionnaires, and fatigue assessments up to 48 weeks. Safety monitoring will continue for 60 days following the last dose. The total duration of participation may extend up to several months as outlined by the trial schedule.

This trial is for adults with recurrent or metastatic head and neck squamous cell carcinoma HNSCC that has not been treated with systemic therapy before. The study compares the anti-tumor effects of amivantamab combined with pembrolizumab and carboplatin against pembrolizumab with 5-fluorouracil and platinum therapy carboplatin or cisplatin. Participants have HNSCC in the oral cavity, oropharynx, hypopharynx, or larynx but not nasopharynx or unknown primary tumor sites, and HPV status is considered for oropharynx cases. Participants are randomly assigned to one of two groups. One group receives pembrolizumab, amivantamab, and carboplatin, while the other receives pembrolizumab, 5-fluorouracil given as a 4-day infusion, and carboplatin or cisplatin. Treatments are given according to standard protocols for these drugs. The study is open-label and conducted across multiple centers. During the trial, participants will be monitored for overall survival and tumor response using established criteria up to about 3 years and 7 months. Additional assessments include progression-free survival, duration of response, quality of life questionnaires specific to head and neck cancer, and safety evaluations through adverse event and lab test monitoring. Blood samples will check amivantamab levels and antibodies. Participation involves regular visits for treatment and assessments throughout the study period.

Researchers are studying the effectiveness and safety of lebrikizumab in people aged 12 and older who have chronic rhinosinusitis with nasal polyps and are treated with intranasal corticosteroids. This Phase 3 trial compares different dosing schedules of lebrikizumab with a placebo to find out how well it reduces symptoms such as nasal congestion and polyp size over about 18 months. Participants receive lebrikizumab or placebo as subcutaneous injections while continuing their regular intranasal corticosteroid therapy. Adolescents aged 12 to under 18 weighing at least 40 kg will receive open-label lebrikizumab every 2 or 4 weeks. The study includes two experimental lebrikizumab groups with different dosing intervals and a placebo group, all alongside background intranasal corticosteroids. During the study, participants will have regular assessments including symptom severity scores, nasal polyp size measured by endoscopy, sinus imaging, lung function tests, and questionnaires about nasal symptoms and quality of life. Researchers will monitor changes from baseline to week 24 primarily for nasal congestion and polyp scores. Safety and long-term effects will also be observed throughout the study duration of about 18 months.

Researchers are evaluating the efficacy and safety of talquetamab, a drug given by injection, in adults with relapsed or refractory multiple myeloma who have received at least three prior treatments. This Phase 2 study focuses on participants who may or may not have been exposed to T cell redirection therapies. The goal is to understand how well talquetamab works and its safety profile in this patient group. Participants are divided into five cohorts based on their previous treatments and exposure to T cell redirection therapies. They will receive talquetamab by subcutaneous injection at doses selected from earlier parts of the study. Treatment continues until disease progression, and participants will move into open-label and long-term extension phases to keep receiving the study drug as approved by the sponsor. During the trial, participants will have regular assessments including laboratory tests, safety evaluations, and quality of life questionnaires. Researchers will measure outcomes such as overall response rate, duration of response, progression-free survival, and safety events for up to nearly three years. Participants health and treatment effects will be closely monitored throughout this period.

Researchers are studying pediatric participants aged 2 to under 18 years who have chronic graft-versus-host disease cGVHD that is moderate to severe and requires systemic immune suppression. The study compares the drug axatilimab to the best available therapy BAT after participants have already received at least two prior systemic treatments, including corticosteroids and ruxolitinib. This phase 2 trial aims to evaluate the response to these treatments in children and teens with this condition. Participants will be randomly assigned to receive either axatilimab at a protocol-defined dose or the best available therapy chosen by the investigator. The BAT options include specific drugs such as cyclosporine, tacrolimus, extracorporeal photopheresis, mycophenolate mofetil, mTOR inhibitors, rituximab, imatinib, methotrexate, or ibrutinib, excluding experimental agents. Both treatment groups will be monitored over time to assess responses and side effects. During the study, participants will be evaluated for objective response at 6 months as the primary outcome, with additional assessments up to 5 years including drug pharmacokinetics, overall response at 12 months, duration of response, organ-specific responses, and quality of life changes. Safety will be monitored by tracking treatment-emergent adverse events. The total study duration allows for long-term follow-up to better understand the effects and potential benefits of the treatments.

Researchers are studying the use of ruxolitinib cream in children aged 6 to under 12 years who have nonsegmental vitiligo. The study aims to evaluate how well and how safely the cream works for treating this condition, which causes loss of skin color. This is a Phase 3 clinical trial focused on treatment effects in this pediatric population. Participants are assigned to one of three groups receiving either ruxolitinib cream at 0.75% or 1.5%, or a matching vehicle placebo cream. Each cream is applied topically as a thin film twice daily to the affected skin areas as defined in the study protocol. The treatment period lasts up to 24 weeks, during which changes in skin pigmentation are monitored. Throughout the study, participants undergo regular assessments including evaluations of vitiligo improvement on the face and body using the Vitiligo Area Scoring Index and other scales. Safety is monitored by tracking adverse events and blood levels of the drug at multiple timepoints up to 52 weeks. Quality of life and psychosocial effects are also measured. The total study duration includes treatment and follow-up visits to assess long-term outcomes and safety.

Researchers are studying the safety, tolerability, and how the body processes maribavir in children and teenagers who have cytomegalovirus CMV infection after receiving a hematopoietic stem cell transplant HSCT or a solid organ transplant SOT. This phase 3 trial aims to find the best dose of maribavir using either a 200 mg tablet or a powder for oral suspension formulation. The study focuses on these young patients who have documented CMV infection and evaluates maribavirs antiviral activity along with its pharmacokinetics. Participants receive maribavir for up to 8 weeks with doses adjusted based on age and body weight. For ages 12 to less than 18 years, dosing ranges from 100 mg to 400 mg twice daily depending on weight. Those aged 6 to less than 12 years follow a similar dosing scheme, and children younger than 6 years may receive doses from 50 mg once or twice daily up to 400 mg twice daily. The medication is taken orally as tablets or powder for oral suspension during the treatment period. During the study, participants will have multiple blood tests to measure maribavir levels at various time points, and adverse events will be monitored up to 20 weeks. There is a 12-week follow-up period after treatment ends, during which participants will visit their doctor to assess continued viral control and safety. Researchers will also evaluate the clearance of CMV viremia, recurrence rates, and resistance development, along with participant feedback on medication palatability.

Researchers are investigating a new drug called BAY 3389934 to find a better way to treat people with sepsis-induced coagulopathy, a serious condition where an active immune response causes uncontrolled blood clotting, damaging blood vessels and organs. This early-phase study aims to learn about the safety, appropriate dose, and effects of BAY 3389934 in patients receiving treatment for this condition in intensive care units. The research is focused on monitoring medical problems, called adverse events, that occur during and after treatment. Participants will receive BAY 3389934 as a continuous intravenous infusion for up to 96 hours. They will be divided into two groups the first group will receive a low starting dose, and their response will be closely monitored to adjust the dose if needed. If there are no serious side effects, the second group will receive a higher dose. This dose escalation approach helps determine the best dose for future studies. During the approximately 28-day study, participants will have blood and urine samples taken, physical exams, vital signs checked, and heart health monitored with electrocardiograms. Researchers will track the number and severity of any treatment-emergent adverse events within about four days after starting the infusion. They will also measure blood clotting times over the first six days to assess effects on coagulation. The study is designed to carefully observe safety and drug effects while patients receive intensive care.

Researchers are investigating a new treatment approach for children with chronic kidney disease CKD and proteinuria, conditions that affect kidney function and cause protein leakage into the urine. The study focuses on whether adding a drug called finerenone to existing treatments with angiotensin-converting enzyme inhibitors ACEI or angiotensin receptor blockers ARB can better control kidney problems related to overactivity of a system that regulates blood pressure and fluid balance. This Phase 3 study aims to see if finerenone can reduce protein levels in the urine more effectively than a placebo. Participants in this trial will receive either finerenone or a placebo alongside their usual ACEI or ARB medication. The study treatment lasts about 180 days, with doses adjusted for age and body weight. Before starting treatment, children must pass screening visits to confirm eligibility. During treatment, participants will attend at least seven visits where various health checks, blood and urine tests, heart exams, and questionnaires about medication experience and side effects will be performed. Throughout the study, researchers will monitor kidney function, electrolyte levels, and how the body processes finerenone. They will also track any medical problems participants experience. After completing treatment, participants will have a follow-up visit about 30 days later to assess their health. The main measure of success is the change in the urinary protein-to-creatinine ratio from before treatment to about six months later, helping to understand the treatments impact on proteinuria.

This research aims to collect long-term safety and disease progression data on patients diagnosed with atypical hemolytic-uremic syndrome aHUS, including those treated or untreated with the drugs eculizumab or ravulizumab. The study is observational and involves multiple centers and countries, focusing on real-world information after these treatments have been marketed. Participants include patients of any age diagnosed with aHUS, regardless of whether they have identified complement genetic variants or antibodies. The study gathers data without administering new treatments, monitoring patients who may or may not have received eculizumab or ravulizumab. The registry collects information over extended periods to understand safety events and disease course. During the study, researchers track safety-related events over 10 years and the timing of these events within 5 years. Data collection involves reviewing patient health status and disease progression without altering their usual care. The study relies on informed consent and may include minors with appropriate assent. Participation duration varies, with continuous observation to gather comprehensive post-marketing safety data.