Yekaterinburg

This research aims to evaluate the safety, how the body processes, and the effects of an investigational drug called GNR-055 in patients with Mucopolysaccharidosis Type II (MPS II), also known as Hunter syndrome. MPS II is a genetic condition caused by a deficiency of a specific enzyme, leading to harmful buildup in the body that affects growth, bones, joints, respiratory and cardiovascular systems, and may involve the nervous system causing cognitive and motor impairments. This phase 2/3, multicenter, open-label study includes patients of different age groups to better understand the drug's impact. Participants will receive weekly intravenous infusions of GNR-055 in doses ranging from 1.0 to 3.0 mg/kg. The drug is a modified enzyme designed to reach the brain to potentially prevent neurological damage. The study includes several cohorts receiving different dosages, with treatment ongoing up to 56 weeks. This approach allows researchers to monitor how the drug behaves in the body and how it affects disease markers over time. Participants will be monitored through clinical assessments, including measurements of urine glycosaminoglycan (GAG) levels at multiple points up to week 52 and tracking adverse events throughout the 56-week study period. Safety evaluations, pharmacokinetics, pharmacodynamics, and efficacy will be assessed regularly. The study aims to provide detailed information about the treatment's safety and potential benefits to improve quality of life for patients with MPS II.

Researchers are evaluating the effectiveness and safety of two drugs, BCD-264 and Darzalex, as single treatments for people with relapsed and refractory multiple myeloma. This study focuses on patients who have previously been treated with proteasome inhibitors and immunomodulatory drugs but whose disease progressed after prior therapy. The goal is to compare how well each drug works and how safe they are for these patients. Participants receive either BCD-264 or Darzalex through intravenous infusion at a dose of 16 mg/kg. Both treatments are given as monotherapy, meaning each drug is used alone without combining with other therapies. The study is designed as a double-blind, randomized clinical trial, which means neither the participants nor the researchers know who receives which drug during the trial. During the study, researchers monitor participants for up to 24 weeks to measure the overall response rate using the International Myeloma Working Group criteria. Participants will have regular assessments to track their disease status and treatment safety. Safety and efficacy data are collected throughout the study to evaluate and compare the two treatments' profiles in this patient population.

This research investigates treatment patterns and the evaluation of homologous recombination repair mutations (HRRm) in circulating tumor DNA (ctDNA) among patients with aggressive high-volume metastatic hormone-sensitive prostate cancer (mHSPC) in the Russian Federation. The study focuses on patients with high-aggressive disease characterized by Gleason scores 8-10 and high-volume disease as defined by specific criteria for bone and visceral metastases. Approximately 400 male patients aged 18 years and older with known tumor HRRm status will participate to better understand demographic and clinical characteristics and treatment approaches in routine practice. The study does not introduce new treatments but observes and collects data as patients receive standard care. Two study visits will occur: the first at baseline to gather medical history, demographic data, and treatment information from diagnosis to enrollment, including routine blood samples for ctDNA and HRRm testing. The second visit will happen at disease progression or after about 12 months to collect follow-up data on progression to metastatic castration-resistant prostate cancer (mCRPC) and subsequent treatments. Blood samples will be analyzed centrally. Participants will have their medical records reviewed and may be interviewed to complete missing information. Data will be entered into electronic records by the study physician. Outcome measures include the proportion of patients receiving various treatments (such as androgen deprivation therapy, chemotherapy, radiation, surgery, and specific inhibitors), duration of therapies, time to progression, mutation presence in ctDNA, testosterone levels, and sites of disease progression over 36 months. Follow-up may be completed by phone if in-person visits are not possible, with the total study duration lasting about 38 months or until data from 400 patients are collected.

Researchers are evaluating the physical impact of multiple sclerosis (MS) from the participant's perspective while providing continued access to the drug ocrelizumab. This Phase 3 extension study focuses on assessing the safety and tolerability of ocrelizumab, a treatment for MS, at approved doses. The study includes participants who were already receiving ocrelizumab in previous Genentech and/or F. Hoffmann-La Roche Ltd sponsored studies and do not have local access to this treatment through other means. Participants will receive ocrelizumab either by intravenous (IV) infusion at 600 milligrams or by subcutaneous (SC) injection at 920 milligrams, following the dosing schedule from their previous parent study. Treatment will begin no earlier than five months after their last dose in the parent study. This open-label, multicenter extension provides ongoing access to ocrelizumab for up to five years. Throughout the study, participants will be monitored for changes in their physical functioning using the Patient-Reported Outcome Measure Information System/Quality of Life in Neurological Disorders - Physical Function Measure for Multiple Sclerosis (PROMISnq PFMS-15a). Researchers will also track the number of participants receiving ocrelizumab during the study and assess safety and tolerability over the long term. Monitoring includes regular evaluations to ensure participant well-being during the extended treatment period.

Researchers are conducting a national, multicenter, prospective study in the Russian Federation to collect real-world data on patients with aggressive, advanced endometrial cancer (stages III-IV). The study aims to understand the prevalence of molecular markers such as POLE mutations, dMMR/pMMR, p53 abnormalities, HER2, and PD-L1, as well as to observe first-line postoperative treatment approaches in these patients. Approximately 500 female patients with newly diagnosed aggressive subtypes of advanced endometrial cancer will be enrolled across about 30 sites. The study involves two visits aligned with routine clinical practice. At the first visit, demographic and clinical information will be collected from medical records or patient interviews, along with biopsy or archival tumor samples for molecular testing using immunohistochemistry and genetic sequencing methods. The second visit occurs six months after baseline or at disease progression, whichever is earlier, to gather follow-up data on treatments and disease status. No additional procedures beyond standard care are applied. Participants' data will be securely entered into electronic case report forms by study physicians. Researchers will monitor the rates of molecular markers such as POLE mutation positivity, mismatch repair status, p53 abnormalities, PD-L1 expression, and HER2 expression over 24 months. The overall study duration, from first patient enrollment to final data analysis, is expected to be about 27 months or until all data from 500 patients are collected, including follow-up information.

This research aims to evaluate the long-term safety and tolerability of pelacarsen (TQJ230) in adults with established cardiovascular disease and elevated Lipoprotein(a) who have completed the parent trial CTQJ230A12301. The study is an open-label extension following the phase 3 parent study, providing participants continued access to pelacarsen after the initial trial. Participants will receive pelacarsen 80 mg by subcutaneous injection once a month during this open-label extension. The study is single-arm and multicenter, focusing on continued treatment with pelacarsen for up to 36 months after completion of the parent study. Throughout the study, participants will be monitored regularly to assess safety and tolerability, with particular attention to adverse events occurring up to 36 months. Researchers will collect data on health status throughout this period to understand the long-term effects of pelacarsen in this patient population.

Researchers are evaluating the effectiveness and safety of BCD-248 as a treatment for patients with relapsed or refractory multiple myeloma. This open-label Phase 2 study focuses on individuals who have previously received at least two lines of therapy, including specific treatments like proteasome inhibitors, immunomodulatory drugs, and anti-CD38 therapy. Participants must have measurable disease and documented progression according to established criteria. The study treatment involves administering BCD-248 subcutaneously. Patients eligible for the trial will receive this investigational drug during the study period. There are no comparator groups mentioned, and the treatment is given as a single intervention. This trial does not mention additional phases or extension periods. Participants will be monitored for their overall response rate to treatment up to 24 weeks, based on criteria set by the International Myeloma Working Group. Assessments include disease evaluations and safety monitoring. The study involves careful screening to ensure participants meet specific health and prior treatment requirements, with follow-up to track treatment outcomes and adverse events throughout the study duration.

Researchers are evaluating the immunogenicity, reactogenicity, and safety of the drug GNG-DE compared to a reference drug for preventing meningococcal infections caused by serogroups A, C, W, and Y. This Phase 3 study includes participants aged 3 to 55 years and aims to assess how well GNG-DE stimulates an immune response and its safety profile versus the reference vaccine. Participants are divided into two groups: one group of 40 participants will receive a 0.5 mL dose of GNG-DE administered intramuscularly into the deltoid muscle, while the other group of 40 will receive a 0.5 mL dose of the Menactra® vaccine via the same method. Both treatments are given as single doses during the study. During the study, participants will be monitored for immune response by measuring primary immunogenicity parameters around Day 29. Safety and reactogenicity will also be assessed. Participants must attend scheduled visits, complete observation diaries, and comply with study requirements. The study includes screening tests such as SARS-CoV-2 antigen and pregnancy tests when applicable, and participants will be observed for any adverse reactions to the vaccines.

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.

This research aims to understand the clinical and demographic characteristics of adult patients living with Neurofibromatosis type 1 (NF1) in Russia. It is an open-label, single-arm, non-interventional, multi-center cohort study focused on evaluating clinical outcomes and patient-reported experiences in routine care settings. The study includes adults diagnosed with NF1 who have plexiform neurofibromas (PN) confirmed by clinical or imaging methods and who experience symptoms related to PN. The study does not involve any investigational treatments or interventions but observes patients during their routine care. It enrolls adults aged 18 years or older with newly diagnosed PN or established PN who have not been treated with MEK inhibitors for PN. Diagnosis confirmation includes clinical assessment, ultrasound imaging, MRI, or biopsy. Patients with certain cancers requiring chemotherapy or radiation, or those who have recently used MEK inhibitors, are excluded. Participants will undergo a variety of assessments at the start of the study, including measurement of age, body metrics, educational background, NF1 complications, and symptoms related to PN. Researchers will review medical histories, hospitalizations, disability status, and prior examinations. The study collects data on PN volume and duration of symptoms and diagnosis. Follow-up visits and further evaluations will be conducted as per routine care. The study monitors changes in disability and overall health status during participation.