Woluwe Saint Lambert

Researchers are evaluating the effectiveness, safety, and tolerability of two different doses of remibrutinib compared to a placebo in adults and adolescents with moderate to severe hidradenitis suppurativa (HS). This phase 3 study aims to determine how well remibrutinib works in treating this chronic skin condition characterized by painful abscesses and inflammatory nodules. The study lasts a total of 76 weeks and includes several phases: up to 4 weeks for screening, followed by a 16-week double-blind treatment period where participants receive either remibrutinib Dose A, Dose B, or a matching placebo. After this, there is a 52-week treatment period where all participants receive remibrutinib (Dose A or Dose B). Finally, a 4-week safety follow-up period occurs without treatment. Participants who stop treatment early are encouraged to stay in the study and complete the safety follow-up. During the study, participants will be regularly assessed for clinical response to treatment, focusing on the proportion achieving a 50% improvement in HS symptoms by week 16. Researchers will monitor safety and tolerability throughout the study, including during the follow-up period. Various evaluations such as physical exams and clinical assessments will be conducted to measure treatment effects and ensure participant safety over the entire 76-week duration.

Researchers are evaluating the anti-tumor activity of amivantamab combined with pembrolizumab and carboplatin compared to pembrolizumab, 5-fluorouracil (5-FU), and platinum therapy (carboplatin or cisplatin) in participants with recurrent or metastatic head and neck squamous cell carcinoma (HNSCC). This trial focuses on participants who have not received prior systemic treatment in the recurrent/metastatic setting. HNSCC is a type of cancer affecting the outer tissue layer of the mouth and throat and other head and neck regions. Participants will receive either amivantamab added to pembrolizumab and carboplatin or the standard care regimen of pembrolizumab, 5-FU, and a platinum agent (carboplatin or cisplatin). 5-FU will be given as an infusion over a 4-day period. The study is a phase 3, randomized, open-label, multicenter trial comparing these treatment combinations. During the study, researchers will monitor overall survival and the objective response rate using standard tumor evaluation criteria for up to about 3 years and 7 months. Participants will undergo assessments to measure disease response, including imaging and other evaluations, to track how well the treatments work. Safety and side effects will also be monitored throughout the trial period.

Researchers are evaluating the safety and effectiveness of lebrikizumab in people aged 12 years and older who have chronic rhinosinusitis with nasal polyps and are being treated with intranasal corticosteroids. This Phase 3 study is designed to better understand how lebrikizumab works alongside standard nasal spray treatments over a period of about 18 months. Participants will receive either lebrikizumab or a placebo by subcutaneous injection, while continuing their regular intranasal corticosteroid spray treatment. The study is randomized, double-blind, and placebo-controlled, meaning neither participants nor researchers know who receives the active drug or placebo. The study measures changes from baseline in nasal congestion severity and nasal polyp size using participant reports and endoscopic scoring at the start and after 24 weeks. During the study, participants will undergo evaluations including nasal examinations and symptom assessments at specified times. Researchers will monitor nasal polyp scores and nasal congestion severity to assess treatment impact. Safety and side effects will also be closely observed throughout the study. The total duration of participation is approximately 18 months, allowing careful tracking of treatment outcomes and safety over time.

Researchers are evaluating the safety and effectiveness of talquetamab, a humanized bispecific antibody, in adults with relapsed or refractory multiple myeloma. This study focuses on participants who have measurable disease and seeks to determine the recommended Phase 2 dose(s) of talquetamab. It is an open-label, dose escalation trial designed to treat hematological malignancies, specifically multiple myeloma. Participants will receive talquetamab administered subcutaneously (under the skin) and continue treatment until their disease progresses. The study includes multiple cohorts based on disease measurability assessed by either central or local laboratory testing. The investigational drug is given repeatedly under careful monitoring as part of this Phase 2 study. Throughout the study, participants will undergo regular assessments to monitor response to treatment, with the primary outcome being the overall response rate measured for up to nearly three years. Other evaluations include performance status checks, pregnancy testing for women of childbearing potential, and close observation for any side effects. Safety and treatment effectiveness will be followed until disease progression or other study endpoints.

Researchers are evaluating Axatilimab compared to the best available therapy in children and adolescents aged 2 to under 18 years who have chronic graft-versus-host disease (cGVHD) that is moderate to severe and has not responded to at least two prior systemic treatments. This Phase 2 study focuses on pediatric participants who need ongoing immune suppression due to persistent cGVHD after prior therapies including corticosteroids and ruxolitinib. The goal is to assess how well Axatilimab works compared to other approved treatments in this difficult-to-treat group. Participants will be randomly assigned to receive either Axatilimab at the specified dose or the best available therapy (BAT) chosen by the investigator. The BAT options include drugs such as cyclosporine, tacrolimus, extracorporeal photopheresis (ECP), mycophenolate mofetil (MMF), mTOR inhibitors, rituximab, imatinib, methotrexate, or ibrutinib. Systemic corticosteroids may be continued at a stable dose, and topical or inhaled steroids are allowed. The study excludes certain treatments like experimental agents not approved for any use. Treatment is carefully monitored throughout the study period. Participants will have regular assessments to measure their response to treatment, with the primary outcome being the objective response at 6 months. The study includes detailed monitoring of safety and liver and kidney function. Participants must have a history of allogeneic hematopoietic cell transplantation (allo-HCT) but cannot have had more than one prior allo-HCT. The total participation duration includes screening, treatment, and follow-up visits to evaluate the effects and safety of the study drugs in managing chronic graft-versus-host disease in this pediatric population.

Researchers are evaluating the effectiveness and safety of ruxolitinib cream in children aged 6 to under 12 years with nonsegmental vitiligo, a condition causing skin depigmentation. This phase 3 study focuses on children who have vitiligo affecting specific body areas, including the face and other parts, with certain minimum involvement percentages required for enrollment. Participants will be randomly assigned to receive either ruxolitinib cream or a matching vehicle cream, both applied topically as a thin film twice daily to affected areas. The study is double-blinded, meaning neither the participants nor the researchers know who receives which cream. Treatment will continue with regular assessments to monitor progress and safety. During the study, children will have their vitiligo area measured using the Facial Vitiligo Area Scoring Index (F-VASI) to assess improvement, with the main goal being at least a 75% improvement by week 24. Participants must stop all other vitiligo treatments during the study and will be closely monitored for safety and adherence through scheduled visits and evaluations. The total body vitiligo area must be 10% or less for participation.

Researchers are evaluating the safety, tolerability, and how the body processes (pharmacokinetics) a drug called maribavir in children and teenagers with cytomegalovirus (CMV) infection after receiving a hematopoietic stem cell transplant (HSCT) or a solid organ transplant (SOT). The study aims to find the best dose of maribavir using either a 200 mg tablet or a powder for oral suspension in this young population. This is a Phase 3, open-label, single-arm study focusing on antiviral activity and safety. Participants will receive maribavir treatment for 8 weeks. There is no comparison group as all participants will be treated with maribavir. The drug is given orally either as tablets or as a powder suspension. After the treatment period, participants will have a 12-week follow-up during which they will visit their doctor for monitoring and evaluation. During the study, participants will undergo blood tests to measure various pharmacokinetic parameters of maribavir, including maximum and minimum drug levels in the plasma and how long the drug stays in the body. Researchers will also monitor for any side effects or serious adverse events from the start of treatment until 20 weeks later. The total participation involves treatment and follow-up visits over approximately 20 weeks to fully assess safety, drug behavior, and antiviral effects.

Researchers are investigating a new treatment called BAY 3389934 for people who have sepsis-induced coagulopathy, a condition where an infection causes excessive blood clotting that can damage blood vessels and organs. This Phase 1 study aims to understand how safe BAY 3389934 is, determine the right dose, and observe its effects on patients being treated for sepsis-induced coagulopathy in an intensive care unit (ICU). Participants will receive BAY 3389934 as an intravenous infusion. They will be divided into two groups: the first group receives the lowest starting dose, and based on safety and tolerance, the dose may be adjusted. If no serious side effects occur, the second group will receive a higher dose. Each participant will be involved in the study for about 28 days. During the study, doctors will monitor participants closely by taking blood and urine samples, performing physical exams, checking vital signs like body temperature and heart rate, and examining heart health with electrocardiograms (ECG). Researchers will track any medical problems that arise during and after treatment, called adverse events, to evaluate safety. The main outcomes measured include the number and severity of treatment-emergent adverse events from the first dose up to 97 hours after stopping the treatment.

Researchers are evaluating ivosidenib in adults with previously treated, locally advanced, or metastatic cholangiocarcinoma (CCA) to confirm its safety, effectiveness, and impact on quality of life. This Phase 3b open-label study includes patients who have documented IDH1 gene mutations and have tried at least one prior systemic therapy for CCA. The study aims to consolidate data on ivosidenib use in this patient group, focusing on adverse events, quality of life, and treatment outcomes. All participants will receive ivosidenib tablets orally once daily at a dose of 500 mg for 28-day cycles. Treatment continues as long as clinical benefit is observed and consent is maintained. The study includes a screening visit, a study visit on day 1 of each cycle, a withdrawal visit within 42 days after stopping treatment, and follow-up visits every 6 months for up to 18 months after treatment ends. Additional study visits will be added for each completed cycle. Participants will undergo various assessments at study visits, including electrocardiograms (ECG), physical exams, tumor assessments according to local practice, and blood and urine tests. Researchers will monitor safety by tracking adverse events, serious adverse events, ECG changes, lab abnormalities, vital signs, and performance status through the treatment and follow-up periods. If ivosidenib becomes available as a prescription outside the study, patients will be withdrawn from treatment but followed for overall survival data.

This research is focused on patients diagnosed with Atypical Hemolytic-Uremic Syndrome (aHUS). It aims to gather post-marketing safety data for patients treated with the drugs eculizumab or ravulizumab, as well as to monitor the progression of the disease in all participating patients regardless of treatment status. The study is observational and non-interventional, conducted across multiple centers and countries. No specific treatments or interventions are administered as part of this study; rather, it observes patients who have already been treated or untreated with eculizumab or ravulizumab. The study collects data from these patients over time to understand safety outcomes and disease progression. Participants will be followed to record the proportion experiencing certain events over a period of 10 years and the timing of first and subsequent occurrences of these events over 5 years. The study involves gathering clinical information and safety data through routine monitoring. Patients or their guardians provide informed consent and may be asked for assent if applicable. The study tracks long-term outcomes and safety in aHUS patients.