Samara

This research aims to collect long-term safety and effectiveness data for participants treated with ibrutinib, a medicine used for various blood cancers and conditions including Chronic Lymphocytic Leukemia, Small Lymphocytic Lymphoma, Mantle Cell Lymphoma, Follicular Lymphoma, Diffuse Large B-cell Lymphoma, Waldenstrom Macroglobulinemia, and Chronic Graft Versus Host Disease. It also provides ongoing access to ibrutinib for participants who have completed previous ibrutinib studies, continue treatment, and benefit from it. This is an open-label Phase 3b study without formal hypothesis testing. Participants will continue their current ibrutinib dosing regimen from the prior study, taken orally once daily as capsules in doses of 560 mg, 420 mg, 280 mg, or 140 mg, around the same time each day. Treatment continues until the investigator decides the participant no longer benefits due to disease progression or side effects, the participant withdraws, alternative ibrutinib access becomes available, or the study ends. Participants not able to access ibrutinib elsewhere can keep receiving the single-agent ibrutinib until all transition or stop treatment, or until the study is stopped. During the study, safety is monitored throughout and summarized, and effectiveness may be analyzed together with previous study data. The main outcome measured is the number of participants experiencing any adverse events within 30 days after the last dose or until starting another cancer treatment. Participants will undergo assessments including pregnancy testing and investigator evaluations to ensure ongoing benefit and safety. The study duration depends on when participants stop treatment or transition to other access.

Researchers are evaluating the safety and effectiveness of a new biosimilar drug called bevacizumab (made by Mabscale, LLC) compared to the existing drug Avastin4 in treating patients with advanced non-squamous non-small cell lung cancer (NSCLC) that cannot be removed by surgery or has recurred or spread. This is a phase III randomized, double-blind trial designed to show that the new bevacizumab works as well and is as safe as Avastin4. The study also includes assessments of how the body processes the drug (pharmacokinetics). Participants will receive treatment with bevacizumab at 15 mg/kg or Avastin4, combined with chemotherapy drugs paclitaxel (175 mg/m2) and carboplatin (AUC 6). This combination is given as the first-line therapy for advanced NSCLC. The study is conducted across multiple centers and participants are randomly assigned to one of the two treatment groups without knowing which they receive. Throughout the study, participants will be monitored for their response to treatment, specifically measuring the Objective Response Rate at 18 weeks after starting therapy. Researchers will also assess safety and side effects. Various tests including tumor measurements, blood tests, and other evaluations will be done to ensure participants meet criteria and to track treatment effects. The total duration includes screening, treatment, and follow-up visits to monitor health and outcomes.

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.

Researchers are evaluating etelcalcetide in children aged 28 days to under 18 years who have secondary hyperparathyroidism (SHPT) and chronic kidney disease (CKD) while on hemodialysis. SHPT is a serious condition that develops early in CKD and worsens as kidney function declines, leading to bone problems, growth issues, and higher cardiovascular risks in children on dialysis. Current treatments like vitamin D sterols can sometimes worsen complications, so this phase 3 trial aims to assess etelcalcetide's safety and effectiveness in this pediatric population, building on its approval and use in adults with SHPT on hemodialysis. The study compares etelcalcetide treatment to standard care, which may include vitamin D sterols, calcium supplements, and phosphate binders. Etelcalcetide is given as a drug to control intact parathyroid hormone (iPTH), calcium, and phosphorus levels. The trial is randomized, open-label, and controlled, with multiple dosing to evaluate efficacy, safety, and related pharmacokinetics and pharmacodynamics. Participants continue on their hemodialysis regimen with stable dialysate calcium levels throughout the trial. Participants will have screenings and assessments including laboratory tests for iPTH, calcium, and phosphorus levels, as well as ECG monitoring and other safety evaluations. The primary outcomes measure the percentage of participants achieving at least a 30% reduction in mean iPTH from baseline during weeks 20 to 27. The study monitors participants closely for adverse effects and treatment response over the trial period to gather critical pediatric data on etelcalcetide's use in this vulnerable group.

Chronic heart failure (CHF) is a condition where the heart struggles to pump or relax properly, causing symptoms such as shortness of breath, weakness, palpitations, fatigue, and swelling from fluid buildup. This condition often results from arterial hypertension or coronary artery disease, and many patients have a preserved left ventricular ejection fraction (LVEF). Acute decompensation of CHF involves a sudden worsening of symptoms that requires urgent medical care and hospitalization. This trial is a phase 4 study investigating the use of acetazolamide, a mild diuretic, to treat decompensated CHF by reducing fluid congestion in patients. Participants will receive acetazolamide orally, which works by inhibiting an enzyme in the kidneys to increase the excretion of sodium, potassium, and bicarbonate ions, affecting urine acidity. The study compares this treatment alongside standard diuretic therapy during acute decompensation requiring intravenous diuretics. The goal is to evaluate how acetazolamide impacts congestion symptoms compared to usual care, with monitoring starting from the hospital stage of treatment. During the study, patients will be closely monitored for a decrease in heart failure decompensation over three days. Assessments include clinical evaluations of edema and other congestion signs, laboratory tests, and monitoring for any side effects. Researchers will track improvements in symptoms and overall patient stability to understand acetazolamide's role in managing acute CHF worsening. The total study duration and follow-up details are aligned with the acute treatment phase and safety monitoring.

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 safety, effectiveness, and tolerability of sodium zirconium cyclosilicate (SZC) for treating high potassium levels (hyperkalaemia) in children under 18 years old. This Phase 3, open-label international study will enroll about 140 children across roughly 46 sites in Europe, North America, and other locations. Enrollment begins with children aged 6 to under 12 years and 12 to under 18 years, with plans to include younger age groups based on data review. The study aims to assess whether SZC can help children achieve and maintain normal potassium levels safely. Treatment involves three phases: a Correction Phase (CP) where all participants receive SZC three times daily for up to 3 days to reach normal potassium levels; a 28-day Maintenance Phase (MP) where SZC is taken once daily with dose adjustments to keep potassium normal; and an optional Long-Term Maintenance Phase (LTMP) for continued treatment with monthly visits. Younger children receive doses based on body weight equivalent to adult dosing, with dose levels adjusted after safety reviews by an independent committee. During the study, participants will have blood tests, ECGs, and other assessments to monitor potassium levels, electrolyte balance, and safety. Researchers will evaluate the ability to achieve normokalaemia during the CP and maintain it during the MP and LTMP. The study lasts about 28 weeks, including safety follow-up, and will also assess SZC's acceptability and palatability for children.

Researchers are evaluating ANB-002, a gene therapy delivered by a single infusion, for its effectiveness, safety, and how it works in adult men with hemophilia B who have low FIX activity (2% or less) and no inhibitors. The study aims to show that ANB-002 is not less effective than the standard preventive treatment using coagulation factor IX (FIX). This is a phase 3, open-label, single-arm study focusing on this specific condition. Participants will first enter a lead-in period lasting at least six months, where they will receive standard FIX preventive treatment without any intervention from the study drug. After this period, they will receive one dose of ANB-002 at the start of the main study phase. This main phase will last 18 months following the infusion. After the main phase, participants will enter a follow-up period where they will be observed and evaluated for up to five years after receiving ANB-002. Throughout the study, researchers will track and compare the participants' annualized bleeding rates before and after receiving ANB-002. This includes monitoring during the lead-in period and from 12 to 18 months after treatment. Participants will also undergo safety checks and pharmacodynamic assessments during the main and follow-up periods to evaluate how the treatment affects their condition over time and to ensure ongoing safety.

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.

Researchers are conducting an observational multicenter cross-sectional study to better understand the characteristics of adults with uncontrolled severe asthma in Russia who are not receiving biological therapy. The study aims to collect detailed information on the epidemiology, clinical features, treatment patterns, and demographics of these patients across different regions of the Russian Federation, which vary widely in population composition and environmental factors. The study will help fill the gap in data about severe asthma in Russia, especially in patients treated according to standard care but excluding biologics. The study plans to include 5,000 adult patients from about 50 outpatient centers across 50 regions of Russia. It will collect routine clinical data without altering standard medical care or introducing any new diagnostic or therapeutic procedures. The study design includes one visit per patient to gather demographic, clinical, and treatment information, focusing on patients with uncontrolled severe asthma receiving standard treatments like inhaled corticosteroids with other medications but not biological agents. Participants will provide data through medical records and assessments such as the Asthma Control Questionnaire. Researchers will analyze patterns of drug use, clinical characteristics including comorbidities, blood counts, immunoglobulin levels, and lifestyle factors. The study will characterize patients' demographics, treatment trends, and asthma control status from June 2024 to June 2027. Safety monitoring is observational, with no intervention beyond routine care, and the total participation involves a single study visit.