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Researchers are evaluating 64Cu-LNTH-1363S, a radiolabeled FAP inhibitor, in patients with sarcomas or gastrointestinal tract cancers. This multicenter, open-label Phase 1/2a study aims to assess the safety and tolerability of the agent, establish dosimetry, and identify the optimal imaging dose and timing. The study also compares the imaging biodistribution of 64Cu-LNTH-1363S with FAP expression measured by immunohistochemistry in tumor tissues. The study is divided into two parts. Part 1 involves six patients with metastatic sarcomas receiving a single dose of approximately 8 ± 1 mCi of 64Cu-LNTH-1363S, followed by serial PET/CT scans at multiple timepoints on the intervention day to determine biodistribution and optimal imaging parameters. Part 2 includes twenty patients with non-metastatic, operable sarcomas or gastrointestinal cancers planned for surgery within 60 days. These patients undergo imaging with the agent, and tissue samples collected during surgery are analyzed for FAP expression. Both parts monitor cardiac safety through ECG assessments. Participants will go through screening up to 14 days before intervention, receive the imaging dose on one day, and have safety follow-ups for 1 to 7 days depending on the study part. Assessments include serial PET/CT imaging at specified timepoints post-dose, tumor tissue sampling for immunohistochemistry, and cardiac monitoring for changes in heart rate and QT intervals. Part 2 extends up to 10 to 11 weeks to cover the surgical tissue collection period. The study measures biodistribution, dose optimization, correlation with FAP expression, and cardiac safety throughout the participation period.

Researchers are investigating the targets of disease-reactive T cells in patients with various autoimmune diseases, including inflammatory bowel diseases, celiac disease, ankylosing spondylitis, multiple sclerosis, scleroderma, systemic sclerosis with lung involvement, and others. The study focuses on identifying the natural peptide targets recognized by T cell receptors (TCRs) in inflamed tissues, which may help develop new treatments that specifically target these immune responses. This approach uses high-throughput technology developed by TScan to discover these targets from active disease tissues. Participants will provide tissue samples during clinically indicated procedures or research biopsies, along with companion blood samples collected around the same time. The study involves isolating T cells from diseased tissues and matched blood or normal tissues, then identifying T cell clones expanded in the affected organs. These clones' TCR targets will be determined using TScan's genome-wide technology. No specific drug treatments are administered by the study; instead, it collects biospecimens for analysis. During the study, researchers will collect and analyze tissue and blood samples to identify disease-associated TCRs and their peptide targets over a 3-year period. Participants must be willing and able to consent and undergo the procedures. The study tracks T cell targets as the primary outcome, aiming to discover new therapeutic targets. Safety considerations include excluding those with conditions that increase biopsy risks. Total participation duration depends on the timing of tissue collection and subsequent analyses.

Researchers are evaluating a new treatment called ifinatamab deruxtecan (I-DXd) for men with metastatic castration-resistant prostate cancer (mCRPC). This study compares I-DXd to chemotherapy to see if it helps people live longer overall and live longer without their cancer worsening. It is a Phase 3, open-label trial focused on patients who have progressed on prior therapies and have evidence of metastatic disease. Participants receive either I-DXd through an intravenous infusion every 3 weeks or docetaxel chemotherapy administered every 3 weeks. Prednisone tablets are also given daily as part of the treatment plan. Before each I-DXd dose, premedication is provided to help prevent nausea and vomiting using a combination of drugs such as corticosteroids and anti-nausea medicines. Treatment continues until disease progression, unacceptable side effects, or other reasons to stop. During the study, researchers monitor overall survival and how long patients live without their cancer progressing, for up to about 36 months. Participants undergo tumor tissue collection, scans, and assessments to track disease status and side effects. Safety is closely watched throughout treatment. The study includes men aged 18 and older with confirmed prostate cancer and metastatic disease who have previously received certain hormone therapies but no prior taxane chemotherapy for mCRPC.

Researchers are evaluating the efficacy, safety, pharmacokinetics, and pharmacodynamics of a new treatment called NXT007 compared to emicizumab prophylaxis in people aged 12 years and older with severe or moderate congenital hemophilia A without factor VIII inhibitors, as well as those with hemophilia A of any severity who have factor VIII inhibitors. This phase III clinical trial aims to better understand how well these treatments work and how safe they are for managing hemophilia A. Participants will receive either NXT007 or emicizumab as prophylactic treatments. NXT007 is given by subcutaneous injection using a combined drug-device product, while emicizumab is administered subcutaneously using a vial and syringe. The study compares these two methods of treatment over a main study treatment period starting from the second month until at least seven months after the first dose. During the study, researchers will monitor how often treated bleeding episodes occur, measured as the Annualized Bleed Rate (ABR). Participants' medical histories, including previous treatments and bleeding episodes, will be carefully documented. Safety and treatment effects will be closely observed throughout the study to evaluate the overall impact of both treatments on the participants' condition.

Researchers are evaluating the efficacy, safety, and tolerability of zorevunersen in patients with Dravet syndrome, a condition marked by reduced levels of the Nav1.1 protein due to mutations in the SCN1A gene. Zorevunersen is an investigational antisense oligonucleotide designed to increase the expression of the sodium channel Nav1.1 protein by boosting the production of its messenger RNA. This Phase 3, multicenter, randomized, double-blind, sham-controlled study aims to assess the potential of zorevunersen for disease modification by measuring changes in major motor seizure frequency and other health outcomes. The study has two treatment periods. In Treatment Period 1, participants assigned to zorevunersen receive the drug by intrathecal administration on Day 1, Day 57, Day 169, and Day 281 with doses of 70 mg initially and then 45 mg later. The sham group undergoes a procedure mimicking drug administration without receiving the drug. In Treatment Period 2, those initially on zorevunersen receive 45 mg doses on Day 393, Day 477, and Day 589. Participants initially in the sham group are then given zorevunersen doses of 70 mg on Day 393 and Day 477, and 45 mg on Day 589. Participants will be closely monitored throughout the study with a primary focus on seizure changes measured at Week 28. Secondary assessments include behavior, cognition, clinical status, and quality of life. The study includes an initial 8-week baseline period and a 6-week observation period to confirm seizure frequency and stability of other treatments. Patients may continue to an open-label extension study to receive zorevunersen if eligible. The study involves children aged 2 to under 18 years and tracks safety and tolerability alongside efficacy outcomes.

Researchers are evaluating the safety, tolerability, pharmacokinetics, and pharmacodynamic effects of a drug called S230815 in children aged 2 to 12 years who have Developmental Epileptic Encephalopathy caused by specific genetic changes in the KCNT1 gene. This is a Phase Ib/II, first-in-human, multicenter, open-label study focusing on this rare and severe form of epilepsy. The study includes two main parts. Part 1 involves giving participants increasing doses of S230815 through injection to determine the best dose. After completing Part 1, participants may enter Part 2, a long-term extension where they continue receiving their assigned dose of S230815 for up to 72 weeks. Participants will be closely monitored throughout the study for side effects and drug levels. Researchers will collect safety information, including any adverse events, for up to 116 weeks. Genetic testing confirms eligibility, and participants will undergo various assessments during the screening and treatment periods to evaluate the drug's effects and safety over time.

Researchers are evaluating the safety and tolerability of SPK-8011QQ, a gene therapy, in adult males with severe or moderately severe hemophilia A. This Phase 2b, single-arm, open-label, multicenter study focuses on men with low levels of factor VIII activity who have a history of treatment with factor VIII products. The study aims to understand how well participants tolerate the therapy and monitor for any adverse effects over an extended period. Participants will receive SPK-8011QQ intravenously. The study does not include a comparison group and follows a single treatment plan. The treatment is administered once, and participants are then monitored closely for safety and any adverse reactions related to the gene therapy. During the study, participants will undergo regular assessments for up to approximately five years. These include monitoring for any adverse events, serious adverse events, and treatment-related side effects, using standardized grading scales. Laboratory tests will be performed to check for abnormal values, and researchers will track overall health and any impacts of the therapy throughout the study period.

The trial investigates the use of volrustomig in participants with unresected locally advanced head and neck squamous cell carcinoma (LA-HNSCC) who have not shown disease progression after receiving definitive concurrent chemoradiotherapy (cCRT). The study aims to evaluate the efficacy and safety of volrustomig compared to observation in this patient population. Participants have tumors that express PD-L1 and the study is conducted as a Phase III, randomized, open-label, multi-center global trial. Participants are assigned to receive either volrustomig as sequential therapy following cCRT or to an observation group. The treatment period involves monitoring participants who have completed definitive cCRT but remain unresected and have no evidence of metastatic disease. The study focuses on participants with Stage III, IVA, or IVB LA-HNSCC according to AJCC criteria, who have not undergone tumor resection before cCRT and have not been treated with radiotherapy alone. During the study, participants are regularly evaluated for progression-free survival, with follow-up lasting up to approximately 8 years to assess long-term outcomes. Researchers will monitor safety and disease progression closely. The overall participation duration includes screening, treatment or observation, and extended follow-up to capture both efficacy and safety data over time.

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 conducting a master protocol study called CAMPFIRE to efficiently carry out multiple clinical trials testing new drugs in children and young adults with cancer. This master protocol allows for adding new studies as new cancer drugs become available, focusing on the treatment of measurable or evaluable tumors in participants aged 1 to 39 years. The goal is to evaluate various drugs under a unified research plan to improve treatment options for young cancer patients. The study involves several investigational drugs administered either intravenously or orally, including Ramucirumab, Cyclophosphamide, Vinorelbine, Gemcitabine, Docetaxel, Abemaciclib, Irinotecan, and Temozolomide. Each drug is tested under specific clinical trials within the master protocol, with treatment schedules and dosing tailored to each drug. Participants receive these treatments following standard clinical procedures, with adjustments based on individual study protocols and treatment responses. Participants will be closely monitored throughout the trial, with assessments including performance status evaluations, laboratory tests to check organ and blood function, and pregnancy testing for females of childbearing potential. Researchers will track how many participants receive each treatment during the first four weeks and observe the duration of treatment benefits. Safety evaluations, adherence to contraceptive measures, and recovery from prior therapies are also part of the study monitoring. Participation duration and additional assessments depend on the specific trial and treatment plan assigned.