Ufa

Researchers are evaluating the safety, pharmacokinetics, pharmacodynamics, and effectiveness of an investigational drug called GNR-055 in patients with Mucopolysaccharidosis Type II MPS II, also known as Hunter syndrome. This condition is a genetic disorder caused by a deficiency of the enzyme iduronate-2-sulfatase ID2S, leading to harmful buildup of certain substances in cells that affects growth, organs, and the nervous system. The study is a phase 23, multicenter, open-label trial involving different age groups to better understand how GNR-055 works and its safety profile. GNR-055 is a modified enzyme replacement therapy designed to cross the blood-brain barrier, potentially preventing neurological damage and improving quality of life for patients with MPS II. Participants receive weekly intravenous infusions of GNR-055 at doses ranging from 1.0 to 3.0 mgkg, depending on their study group. The study includes multiple cohorts, with adult and pediatric patients receiving specific dosing regimens over the trial period. During the study, participants will undergo various assessments including monitoring of adverse events, urine and serum levels of glycosaminoglycans GAG, cerebrospinal fluid analysis, joint motion measurements, MRI scans of liver, spleen, and brain, heart and lung function tests, neurocognitive evaluations, and biomarker analysis. These evaluations occur at baseline and multiple follow-up visits up to week 56. The study aims to gather detailed data on the drugs impact on disease symptoms, safety, and biological markers to inform future treatment options.

Researchers are evaluating the safety and preliminary efficacy of OM-RCA-01, a monoclonal antibody targeting fibroblast growth factor receptor 1 FGFR1, in patients with metastatic solid tumors that express FGFR1. This Phase 1b2, multicenter, open-label study uses a basket trial design enrolling patients regardless of tumor type, focusing on cancers such as renal cell carcinoma, non-small cell lung cancer, head and neck cancer, breast cancer, and prostate cancer. The study aims to understand the medical issues participants may experience, determine appropriate dosing for future studies, and assess whether tumor growth slows with treatment. All participants will receive OM-RCA-01 through an intravenous infusion every two weeks. Treatment will continue as long as the disease remains controlled and the drug is well tolerated. The study includes five tumor-specific groups and plans to enroll 58 patients. The drug is given at dose levels of 50 mg or 100 mg, and therapy will proceed until disease progression or unacceptable toxicity occurs. Participants will be monitored through regular assessments including imaging to measure tumor lesions, laboratory tests for organ function, and questionnaires to evaluate quality of life. Safety will be closely observed by tracking adverse events, serious side effects, and the presence of anti-drug antibodies. The study will follow patients for up to 12 months to evaluate outcomes such as progression-free survival, overall survival, and duration of response.

Researchers are studying the use of sarilumab, a drug given by injection, in children and adolescents aged 1 to 17 years who have systemic juvenile idiopathic arthritis sJIA. The study aims to understand how the drug behaves in the body, its effects, and its long-term safety for treating this condition. This trial is a phase 2, open-label study sponsored by Sanofi, designed to find the best dose and treatment schedule for young patients with sJIA. Participants will receive sarilumab injections under the skin at doses that increase during the study based on body weight. The treatment includes a 12-week core phase where patients receive the drug, followed by a 144-week extension phase for continued treatment. After completing treatment, a 6-week follow-up period will monitor patients. The study includes careful dose adjustments and long-term observation to assess the drugs impact. Throughout the study, participants will undergo various assessments including blood tests to measure drug levels, evaluations of disease activity using scales like the Investigator Global Assessment and ParentPatient Global Assessment, and tracking of symptoms and medication use. Safety will be monitored continuously by recording any side effects or local reactions to injections. The total participation time is about 166 weeks, during which the researchers will collect data on how well sarilumab works and how safe it is for children and adolescents with sJIA.

Researchers are evaluating the safety and effectiveness of a new bevacizumab drug made by Mabscale, LLC, compared to Avastin, both combined with paclitaxel and carboplatin, for treating adults with advanced or inoperable non-squamous non-small cell lung cancer NSCLC. This phase III trial aims to show that the new bevacizumab works as well and is as safe as Avastin. The study also looks at how the drug is processed in the body. Participants are randomly assigned to one of two groups. One group receives the Mabscale bevacizumab with paclitaxel and carboplatin, and the other gets Avastin with the same chemotherapy drugs. Treatment cycles last about three weeks, with up to six cycles initially. After that, eligible patients continue with bevacizumab alone every three weeks. The study is double-blind, so neither patients nor researchers know who receives which bevacizumab. During the study, patients will undergo regular assessments including tumor measurements and blood tests. Researchers measure the tumor response at 18 weeks and track progression-free and overall survival at 18 and 42 weeks. The duration of response is also evaluated up to 48 weeks. Safety and side effects are closely monitored throughout the trial, which started in 2023 and will continue until 2027. Participants are followed closely to assess the treatments impact and safety.

Researchers are evaluating the safety and effectiveness of BCD-248 combined with daratumumab compared to a combination of daratumumab, pomalidomide, and dexamethasone for treating adults with relapsed or refractory multiple myeloma. This study focuses on participants aged 18 and older who have measurable multiple myeloma and have received previous treatments including a proteasome inhibitor and lenalidomide, specifically those who are refractory to lenalidomide or have had disease progression after prior therapies. Participants will receive either BCD-248 administered under the skin with daratumumab given intravenously, or the combination of daratumumab intravenously with pomalidomide and dexamethasone taken orally. The study is randomized without masking, comparing these two treatment groups in parallel. The intervention period includes monitoring for up to 36 months to assess disease progression and response, with additional long-term evaluations lasting up to 5 years. During the trial, participants will undergo assessments including measuring minimal residual disease using flow cytometry at 12 months, and monitoring progression-free survival per established criteria. Other evaluations include response rates, survival, immune markers, and adverse events over several years. Safety and treatment effects will be closely followed with regular clinical visits and laboratory tests throughout the study duration, which extends until October 2032.

Researchers are studying aggressive high-volume metastatic hormone-sensitive prostate cancer mHSPC in male patients in the Russian Federation. This observational study focuses on treatment patterns and the evaluation of homologous recombination repair mutations HRRm in circulating tumor DNA ctDNA. The study aims to better understand demographic and clinical characteristics along with how patients with different HRR gene statuses are treated. The study enrolls about 400 male patients with high-aggressive Gleason 8-10 and high-volume mHSPC who have known tumor HRRm status from routine tumor samples. No additional procedures beyond routine clinical care are performed. Data are collected during two visits a baseline visit where medical history and treatment approaches since diagnosis are recorded, and a final visit at disease progression or after about 12 months to gather follow-up treatment and progression information. Blood samples for ctDNA and HRRm testing are collected from routine blood draws and analyzed centrally. Participants contribute data through medical record reviews and, if needed, patient interviews. Researchers collect information on treatments received, disease progression, lab tests, and mutation presence. The study evaluates various outcomes including treatment proportions, therapy durations, progression times, and mutation rates over 36 months. Data are entered into electronic forms, and follow-up may occur by phone if in-person visits are not possible. The total study duration is about 38 months or until all data are collected.

Researchers are conducting a national, multi-center, prospective study to collect real-world data on patients in the Russian Federation with advanced and aggressive endometrial cancer, specifically stage III-IV disease. The study focuses on molecular profiling markers such as POLE mutations, dMMRpMMR, p53 abnormalities, HER2 expression, and PD-L1 status, alongside demographic and clinical characteristics. It aims to understand first-line postoperative treatment approaches in these patients. The study involves no additional procedures beyond routine clinical practice. Patients provide archival tumor tissue samples obtained from biopsy or post-operative formalin-fixed paraffin-embedded FFPE blocks for molecular testing. Testing uses immunohistochemistry IHC for certain markers and next-generation sequencing NGS or polymerase chain reaction PCR for POLE mutations. Data will be collected at two visits a baseline visit to gather clinical and demographic information and tissue testing results, and a final visit 6 months later or at disease progression to collect follow-up treatment and progression data. Participants data and outcomes will be recorded in an electronic case report form. The study measures include rates of positive molecular markers and various clinical and treatment-related outcomes over 24 to 33 months. Approximately 500 patients will be enrolled across about 30 sites. The overall study duration is about 27 months or until data collection for all patients is complete.

Researchers are evaluating the efficacy and safety of BCD-248 as a treatment for patients with relapsed or refractory multiple myeloma, a condition where the disease has returned or is resistant to previous therapies. This open-label phase 2 clinical study aims to assess the overall response rate and other important outcomes in this patient population. The study is sponsored by Biocad and focuses on patients who have received multiple prior treatments including proteasome inhibitors, immunomodulatory drugs, and anti-CD38 therapy. Participants will receive BCD-248 administered subcutaneously. The study monitors various outcomes including overall response rate up to 24 weeks and progression-free survival up to 104 weeks, among others, over a total duration extending to about 3.7 years for some measures. Pharmacokinetic parameters like Cmax, Cmin, AUC0-t, and Ctrough will be tracked shortly after administration and up to 6 months. Additional assessments include measurement of soluble BCMA levels and the presence of binding and neutralizing antibodies. During the study, participants will undergo regular evaluations including disease assessments based on International Myeloma Working Group criteria, safety monitoring for adverse events, and laboratory tests. Long-term follow-up will include evaluation of response duration, time to response and progression, and overall survival. The study started in December 2024 and is expected to continue until July 2028, providing extended observation for participant outcomes and safety.

Researchers are evaluating the safety, effectiveness, and pharmacodynamics of a single dose of ANB-002, a gene therapy, in adult men with hemophilia B who have low factor IX FIX activity and no FIX inhibitor. The study aims to show that ANB-002 is not less effective than standard preventive treatment using FIX concentrates. This is an open-label, phase 3 trial sponsored by Biocad. Participants first undergo a non-interventional lead-in period lasting at least six months during which they receive standard FIX preventive treatment. After this, they enter the main interventional period where they receive a single infusion of ANB-002. This main period lasts 18 months, followed by a follow-up period of up to five years after the ANB-002 infusion. During the study, researchers will monitor participants bleeding rates before and after treatment, FIX activity levels, and FIX concentrate use. They will also assess the proportion of subjects not needing FIX prevention, and track various bleeding events including spontaneous bleedings and hemarthrosis. Safety and treatment effects will be evaluated over the long-term follow-up, with study participation lasting up to five years after treatment.

Researchers are evaluating the effectiveness, safety, how the body processes, and immune response to BCD-236 combined with chemotherapy in women with relapsed or metastatic triple negative breast cancer TNBC who have received previous treatments. This is a Phase 2 study that compares BCD-236 plus chemotherapy versus chemotherapy alone in patients whose cancer has returned or progressed after earlier therapies. Participants will be randomly assigned to one of two groups one receiving BCD-236 by intravenous infusion plus chemotherapy chosen by the investigator, and the other receiving chemotherapy alone. Treatment continues until the cancer worsens, side effects become intolerable, or the study ends. During the study, participants will undergo regular assessments including tumor measurements and health evaluations to monitor response and side effects. The main outcome measured is the overall response rate at 24 weeks, with secondary outcomes including progression-free survival, overall survival, disease control rate, time to response, and duration of response assessed at various times up to 102 weeks. Participants will be monitored closely throughout their treatment and follow-up periods.