Homiel

Researchers are evaluating the efficacy, safety, pharmacokinetics, and immune response to BCD-236 combined with chemotherapy in women with relapsed or metastatic triple negative breast cancer (TNBC). This Phase 2 study focuses on patients who have received at least one prior systemic therapy and whose cancer has progressed or relapsed. The study aims to better understand how this combination treatment works in later lines of therapy for this aggressive breast cancer subtype. Participants will receive BCD-236 as an intravenous infusion along with chemotherapy, which will be chosen at the investigator's discretion. The study compares this combination treatment's effects and monitors participants over time. The primary outcome measured is the overall response rate at 24 weeks after starting treatment, assessing how well tumors respond to the therapy. Throughout the study, participants will undergo tumor assessments using RECIST 1.1 criteria to measure treatment response. Eligibility requires confirmation of AXL expression in tumor cells from fresh or archival tumor samples. Patients will be monitored for safety and disease progression, with evaluations including physical exams and performance status assessments. The study includes women aged 18 to 74 years with adequate health to participate and a life expectancy of at least four months.

Researchers are evaluating the real-life effectiveness, safety, and usage patterns of Octapharma's factor VIII (FVIII) concentrates Nuwiq, Octanate, and Wilate in patients with severe haemophilia A who have either never been treated or have had minimal previous treatment. This study focuses on previously untreated patients (PUPs), often young children, and minimally treated patients (MTPs), to better understand treatment outcomes and inhibitor development in routine clinical practice. Because haemophilia A is rare and treatment practices vary, especially for PUPs, collecting real-world data is important to improve treatment guidelines and benefit-risk assessments. The study observes patients prescribed Octapharma's FVIII concentrates without changing their treatment, capturing how these products are used in everyday healthcare settings. There are no experimental interventions as this is a non-interventional study. The study includes patients starting or continuing treatment with these FVIII concentrates, monitoring their usage, dosing, and frequency as determined by their healthcare providers. Participants are monitored for effectiveness by tracking the annual rate of breakthrough bleeding episodes and safety by recording adverse drug reactions over 100 exposure days. Data on inhibitor development is also collected. Patients of all ages and genders with severe haemophilia A are eligible if they meet treatment history criteria. Informed consent is obtained before data collection. The study gathers information from routine clinical visits and treatment records without additional procedures or interventions.

Researchers are evaluating the safety, immune response, and effectiveness of ANB-002, a gene therapy candidate, in male patients with Hemophilia B. This multicenter, open-label study uses a dose-escalation design combining elements of phase I and II to find the best dose. The goal is to understand how the treatment affects blood clotting factor IX (FIX) activity and to monitor for any adverse reactions over five years. The study involves four cohorts receiving single intravenous infusions of ANB-002 at increasing doses. Cohort 1 starts with dose 1, followed by cohorts 2 and 3 with higher doses if no dose-limiting toxicities occur. An independent committee reviews safety data before advancing doses or enrolling more participants. Cohort 4 includes patients with anti-AAV5 antibodies or hepatitis B history and receives the highest dose, dose 3. Participants are followed for five years, with close monitoring for changes in FIX activity and any side effects throughout this period. The study includes assessments to determine the best therapeutic dose and safety profile. Data from the first three cohorts guide decisions for further enrollment and dosing. This long-term follow-up helps evaluate the treatment's lasting impact and safety in patients with Hemophilia B.