Sankt Peterburg

Researchers are evaluating the pharmacokinetics, safety, and immune response of two treatments, RPH-030 and Vectibix®, in patients with metastatic colorectal cancer (mCRC) who have wild-type RAS genes. This phase I, multicenter, double-blind, randomized study aims to demonstrate that these treatments have equivalent pharmacokinetic properties when given as first-line therapy in combination with the chemotherapy regimen FOLFIRI. The study also includes a pilot evaluation of the efficacy of these treatments. Participants will be randomly assigned to receive either RPH-030 or Vectibix® intravenously at a dose of 6 mg/kg every two weeks alongside FOLFIRI chemotherapy. Treatment will continue for up to two years or until disease progression, unacceptable toxicity, or withdrawal of consent. The study is divided into several periods: a screening period lasting up to 27 days (extendable to 42 days if biopsy is needed), a 6-month main treatment period, a continued therapy period up to one year, a treatment extension period for responders lasting up to two years, and a follow-up period after treatment ends. During the study, patients will undergo regular tumor assessments approximately every 6 to 8 weeks depending on the study phase. Hospitalizations of at least 24 hours will occur at certain visits for drug administration. Researchers will monitor drug levels in the blood at multiple time points to understand treatment pharmacokinetics. Follow-up will include imaging tests, survival data collection, and safety monitoring until one year after treatment or until patient withdrawal or death. The goal is to assess treatment safety, immune response, effectiveness, and patient well-being throughout the study timeline.

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.

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 studying metastatic cancers that express the fibroblast growth factor receptor 1 (FGFR1), focusing on a new targeted treatment using a monoclonal antibody called OM-RCA-01. This Phase 1b/2 clinical trial aims to evaluate how well OM-RCA-01 works and its safety in patients with various types of metastatic tumors such as renal cell carcinoma, non-small cell lung cancer, head and neck cancer, breast cancer, and prostate cancer. The study will help determine the appropriate dosage and observe any medical issues during treatment, while also assessing if the tumor growth slows. All participants will receive the OM-RCA-01 antibody intravenously every two weeks. The antibody works by blocking the activation of FGFR1, which is involved in tumor development. Patients will continue to receive the treatment as long as the disease does not progress and the drug is tolerated. The study uses a basket design enrolling patients based on FGFR1 expression regardless of tumor type, and will include up to 58 patients divided into five tumor-specific groups. During the study, participants will undergo various assessments including tumor evaluations according to RECIST 1.1 criteria, laboratory tests to monitor organ function, and biomarker analysis from tumor tissue samples. Safety and efficacy will be observed over six months for Phase 1b and twelve months for Phase 2. Researchers will monitor for treatment side effects, tumor response, and patient health throughout the trial to gather comprehensive information on the study drug's impact.

Researchers are evaluating the safety and effectiveness of Radotinib in patients with chronic phase Philadelphia chromosome-positive chronic myeloid leukemia (CP-CML) who have not responded well or cannot tolerate previous treatments with tyrosine kinase inhibitors (TKIs) including Imatinib. This Phase 3, multinational, multicenter, open-label study aims to enroll 173 participants to better understand Radotinib's impact on this condition. Participants will receive Radotinib capsules at a dose of 400 mg twice daily in a single treatment arm. Radotinib is provided as hard capsules containing 100 mg or 200 mg doses of the drug. Treatment will be administered continuously, and the study includes monitoring for safety and efficacy throughout the course. The study does not include a comparator group but follows participants closely for response to therapy. During the study, participants will be regularly evaluated to monitor their response, including measuring the major cytogenetic response at 6 months. Assessments will include laboratory tests to check organ function and disease status, as well as safety monitoring for side effects. The study requires participants to attend scheduled visits and comply with study procedures, including pregnancy testing for women of childbearing potential and contraception use. The overall participation duration and follow-up details are based on the study protocol.

Researchers are conducting a global, multicenter, randomized, double-blind, placebo-controlled phase III study to evaluate the effectiveness of APG-2575 (Lisaftoclax) combined with azacitidine compared to placebo with azacitidine. The study focuses on patients with newly diagnosed acute myeloid leukemia (AML) who are not eligible for standard induction chemotherapy. This research aims to provide new treatment options for elderly patients or those with specific health conditions preventing standard treatment. Participants will be randomly assigned to one of two groups: one receiving APG-2575 (Lisaftoclax) orally once daily every 28 days plus azacitidine given by injection either under the skin or into a vein daily on days 1 through 7 of each 28-day cycle, and the other receiving a placebo orally on the same schedule plus azacitidine injections. The study treatment cycles continue with close monitoring to assess treatment effects and safety. Throughout the study, participants will undergo various assessments including survival monitoring for up to five years as the primary outcome. Researchers will also track treatment adherence, side effects, and overall health status through scheduled examinations and follow-up visits. Participants are expected to complete all study procedures, including laboratory tests and physical evaluations, during the study period to help determine the treatments' impact on survival and disease progression.

Researchers are investigating the long-term safety and tolerability of open-label iptacopan in adults with primary IgA nephropathy who have previously completed specific clinical trials (CLNP023X2203 or CLNP023A2301). This extension study is designed to allow participants continued access to iptacopan until certain conditions are met, such as reaching three years from the last patient first visit, loss of treatment benefit, negative benefit-risk profile, initiation of dialysis or kidney transplant, or commercial availability of the drug. The study will also assess the drug's effects on disease progression every six months. Participants who completed the prior trials and meet inclusion criteria may receive oral iptacopan capsules at a dose of 200 mg twice daily. The study is open-label and non-randomized and will continue treatment under this regimen until one of the study-defined stopping points is reached. Supportive care with ACE inhibitors or ARBs is maintained as per clinical guidelines, and vaccination against certain infections is required before enrollment. During the study, participants will be monitored for safety, including serious adverse events, adverse events of special interest, vital sign abnormalities, ECG changes, and laboratory test abnormalities from the first day of treatment until seven days after the last dose. Efficacy assessments occur every six months to evaluate clinical effects on disease progression. The study aims to collect long-term safety and tolerability data while providing ongoing treatment access until the drug becomes commercially available or other stopping criteria apply.

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.

This research focuses on men with prostate cancer who have previously participated in an enzalutamide clinical study sponsored by Astellas or Medivation. It aims to gather long-term safety information from participants who continue to benefit from enzalutamide treatment. This is a Phase 2 open-label extension study designed to monitor ongoing treatment effects after the initial study has completed its primary analysis or evaluation period. Participants will continue their previous treatment regimens, which may include enzalutamide taken orally once daily. Some may also receive abiraterone acetate with prednisone or leuprolide acetate depending on their prior study enrollment. Dose adjustments are allowed with medical monitor approval. The first visit of this study should occur within seven days of the last visit of the prior study unless treatment is temporarily paused. Participants are asked to return to their study site every 24 weeks for safety reviews, including adverse event monitoring and medication checks. At visits every 12 weeks, participants return unused study drugs and receive new supplies if needed. Safety data, including all adverse events and serious adverse events, are collected from consent until study completion, which may last up to 96 months. The study follows local standard care guidelines and includes a post-marketing phase in South Korea.

This research aims to provide ongoing access to treatments for participants with multiple myeloma or smoldering multiple myeloma who are benefiting from treatment in certain Janssen studies that include daratumumab. It allows all participants from daratumumab studies and those in daratumumab-containing arms of related studies, which have reached clinical cutoff for final analysis, to continue treatment. The study also collects long-term safety data from these participants. The treatments being evaluated include daratumumab, which is given either intravenously or subcutaneously, carfilzomib administered intravenously, dexamethasone given orally or intravenously, and oral medications lenalidomide and pomalidomide. Participants will continue to receive these treatments as part of this long-term extension study following their previous study treatment. During the study, participants will be monitored for safety, including tracking serious adverse events, adverse events of special interest, pregnancies, and abnormal pregnancies over a period of 3 years and 7 months. Assessments include pregnancy testing for women of childbearing potential and adherence to lifestyle restrictions. Participants must provide informed consent and will be followed closely to evaluate the long-term effects and safety of their treatment.