Stanford

Researchers are investigating CGT9486, also known as bezuclastinib, in an open-label Phase 2 study for patients with Advanced Systemic Mastocytosis AdvSM. This includes those diagnosed with Aggressive Systemic Mastocytosis ASM, Systemic Mastocytosis with an Associated Hematologic Neoplasm SM-AHN, and Mast Cell Leukemia MCL. The study aims to evaluate the safety, effectiveness, pharmacokinetics, and pharmacodynamics of bezuclastinib in this patient population. Participants will receive bezuclastinib tablets orally, taken continuously in 28-day cycles. The study is divided into two parts Part I focuses on identifying effective and tolerable dosing exposures over 18 months, while Part II evaluates the drugs efficacy by measuring objective response rates and confirming the exposure-response relationship, also over 18 months. Additional assessments include effects on mutation allele burden, serum tryptase levels, histopathologic changes, spleen and liver volume, and safety monitoring. During the study, participants will undergo various clinical evaluations, including laboratory tests, imaging to monitor organ size changes, and assessments of disease response and progression. Researchers will track adverse events and pharmacokinetic profiles throughout the 18 months. The study involves continuous monitoring of participants to understand the treatments impact on survival and disease progression over this period.

Researchers are evaluating the imaging agent 64Cu-LNTH-1363S in patients with sarcomas or gastrointestinal tract GIT cancers to assess its safety, determine the best imaging dose and timing, and compare the imaging results with fibroblast activation protein FAP expression in tumor samples. This Phase 12a open-label study is divided into two parts and aims to better understand how this radiolabeled agent behaves in the body and how well it highlights tumors that express FAP. In Part 1, six patients with metastatic sarcomas will receive a fixed dose of 64Cu-LNTH-1363S to evaluate its distribution, radiation dose, and optimal imaging window during a one-day intervention, followed by a safety follow-up. In Part 2, approximately 20 patients with non-metastatic, operable sarcomas or GIT cancers scheduled for surgery will receive the optimal dose determined in Part 1 to study the correlation between imaging results and tissue FAP expression. Both parts include detailed cardiac monitoring to assess any changes in heart activity related to the agent. Participants will undergo screening before receiving the imaging agent, followed by serial PETCT scans at multiple timepoints on the intervention day to measure biodistribution and image quality. Tissue samples collected during surgery will be analyzed to compare with imaging findings. Safety and tolerability will be monitored through follow-up visits, ECGs, and phone contact. The total study duration varies from about three weeks for Part 1 to up to 11 weeks for Part 2, including surgery and post-surgery sample collection.

Researchers are evaluating the safety and effectiveness of two drugs, 64Cu-SAR-bisPSMA and 67Cu-SAR-bisPSMA, in men with prostate cancer that has spread and no longer responds to hormone-lowering treatments. This study focuses on cancers that express a protein called PSMA and aims to find safe and effective dosing while monitoring how the drugs behave in the body. Participants will go through different study phases. In the initial dosimetry phase, a single dose of 200 MBq of 64Cu-SAR-bisPSMA is given. Later phases involve receiving multiple doses of 64Cu-SAR-bisPSMA or 67Cu-SAR-bisPSMA, with doses and number of administrations varying by phase and participant group. The dose escalation phase tests increasing doses of 67Cu-SAR-bisPSMA, followed by a cohort expansion phase with up to six doses of the recommended 67Cu-SAR-bisPSMA dose. During the study, participants will undergo PETCT scans to track drug distribution and dosimetry within 48 hours of dosing. Researchers will measure prostate-specific antigen PSA levels and radiographic responses over up to five years to evaluate treatment effects. Safety will be monitored through assessments of vital signs, ECGs, laboratory tests, and recording any adverse events. The total study duration can extend up to five years for long-term follow-up.

Researchers are evaluating the use of a PET isotope called Fluorine-18 18F attached to Choline in people suspected of having parathyroid adenoma who have negative or unclear results from the standard 99mTc Sestamibi SPECTCT scan. The study focuses on detecting parathyroid adenomas using this new imaging technique in a single-center, single-arm trial. Participants receive an intravenous injection of 18F Fluorocholine about 5 mCi with a 20% margin, followed 45 to 60 minutes later by a low dose CT scan from the skull base to mid-thighs. This is immediately followed by a static PET emission scan over the same area, aiming to locate parathyroid adenomas more effectively than the standard scan. During the study, participants undergo the PETCT imaging process once, and researchers measure the number of lesions detected within an hour after the scan. Safety is monitored through blood tests and heart monitoring prior to the scan. The total participation time is brief and focused on imaging and related assessments.

Researchers are studying a 12-week early intervention program designed for preschool children with developmental disorders, including Autism Spectrum Disorder, neurogenetic disorders, or intellectual disability. The goal is to evaluate how this program helps improve social communication skills in these children by comparing changes in social responsiveness and related behaviors before and after treatment. The intervention involves 12 hours per week over 12 weeks, delivered either in an intensive center-based preschool setting or at home. This behavioral program focuses on structured activities aimed at addressing social communication deficits in young children with developmental disorders. Participants will be assessed at the start and end of the 12-week program using parent-rated questionnaires measuring social responsiveness, repetitive behaviors, social dimensions, adaptive behaviors, sensory profiles, and self-efficacy. The study tracks changes in these areas to understand the programs effects, with continued monitoring until the studys completion in 2034. Children and their families will engage regularly in the intervention and assessments during this period.

Researchers are investigating a combination therapy of BNT326 and pumitamig also called BNT327 or PM8002 in adults with advanced or metastatic non-small cell lung cancer NSCLC who may have relapsed, progressive, or treatment-nafve disease. This multi-site, open-label study aims to find the best dose levels for this combination, assess how well participants tolerate the therapy, including side effects, and evaluate its ability to shrink tumors in this population. The study has three parts Part 1 focuses on finding safe dose levels for the combination Part 2a expands the dose evaluation to assess preliminary effectiveness and safety Part 2b is a randomized phase to optimize doses and understand the contribution of each drug component. Participants will receive intravenous infusions of BNT326 and pumitamig or pumitamig alone in some arms. Treatment continues until disease progression, unacceptable side effects, withdrawal, study end, or up to 24 months. Dose levels for later parts are chosen based on earlier safety and efficacy data. Participants will go through screening, treatment, safety follow-up, efficacy follow-up, and long-term survival follow-up phases, with total involvement expected to last about 36 months unless treatment benefit continues. Assessments include monitoring for dose-limiting toxicities, adverse events, tumor response, progression-free survival, overall survival, and pharmacokinetics of the drugs. Safety evaluations continue up to 90 days after treatment ends, and antibody responses to the drugs are also measured for up to one year post-treatment.

Researchers are evaluating the safety and effects of a single oral dose of HB-2121 in adults suspected of having celiac disease. This phase 1 clinical trial aims to understand any side effects after taking HB-2121 and how the drug interacts with the small intestine. The study focuses on adult participants undergoing standard diagnostic procedures for suspected celiac disease. Participants will receive a one-time oral dose of 250 mg HB-2121 four hours before their scheduled esophagogastroduodenoscopy EGD. The study includes a total of six visits four in-person clinic visits for health checkups, lab tests, and monitoring, as well as two remote visits involving safety lab assessments. Participants will also complete a daily questionnaire for seven days to report symptoms and health status. Throughout the study, researchers will monitor participants for 30 days post-dose to observe the frequency and severity of any adverse events. They will assess how the drug affects the small intestine by measuring specific markers such as fluorescence intensity and tissue characteristics from biopsy samples taken during the EGD. Safety labs, physical exams, and symptom questionnaires will be used to track participant health and drug effects during the trial period.

This research aims to gather long-term safety and effectiveness information for people treated with ibrutinib, a medicine taken by mouth that blocks a specific enzyme called brutons tyrosine kinase. The study focuses on participants who previously took part in ibrutinib studies that have finished and are still receiving ibrutinib treatment, continuing to benefit from it. It is an open-label study, meaning both participants and researchers know the treatment being given. Participants will continue taking ibrutinib capsules daily at the dose they were given in their prior study until the doctor decides the treatment is no longer helpful due to disease progression or side effects, the participant chooses to stop, other treatment options become available, or the study ends. Safety will be monitored throughout, and effectiveness data may be combined with previous study results. No formal testing of hypotheses is planned in this extension. During the study, participants will be regularly monitored for safety and disease status. The main outcome is the number of participants experiencing side effects within 30 days after the last ibrutinib dose or before starting another cancer therapy. Participants may continue treatment until alternative access to ibrutinib is arranged or the study ends, which is planned for December 2029. Researchers will collect ongoing data to understand the long-term effects of ibrutinib treatment.

This long-term follow-up observational study is designed to assess the safety of a gene-modified regulatory T cell Treg therapeutic in individuals who have previously received this treatment. It focuses on evaluating the type, severity, and possible causes of delayed adverse events over an extended period. The study enrolls adults aged 18 to 71 who have been treated in earlier clinical trials involving the gene-modified Treg therapy. Participants will undergo safety monitoring procedures including completing health questionnaires, routine physical exams, and reviews of medical history and medication use. Biospecimens such as blood and tissue samples will be collected at scheduled visits to detect any delayed adverse events related to the prior cell therapy. The study is planned to last up to 15 years following the initial dose received in parent treatment protocols. Throughout the study, participants will attend visits according to a set schedule for assessments and monitoring. Researchers will measure the incidence of delayed adverse events possibly linked to the gene-modified Treg therapeutic and track persistence of the therapy, replication competent lentivirus incidence, and mortality. The studys long duration allows careful observation of safety outcomes related to the treatment over time.

This study focuses on participants who have previously been treated with ciltacabtagene autoleucel cilta-cel, an autologous CAR-T therapy targeting B-cell maturation antigen BCMA used in multiple myeloma. The purpose is to collect long-term follow-up data to understand delayed adverse events and the long-term safety profile of cilta-cel over a period of up to 15 years after the last dose. Participants were originally treated in company-sponsored clinical trials evaluating cilta-cel. No treatment is administered during this follow-up study. Participants will be observed in two phases the first phase covers the initial 5 years after their last cilta-cel dose, and the second phase covers years 6 through 15 post-treatment. The study includes yearly safety evaluations involving review of adverse events, laboratory tests, and physical examinations including neurological assessments. Participants will be followed up at least once per year for up to 15 years to monitor for new or worsening medical conditions such as malignancies, neurological or autoimmune disorders, hematologic disorders, infections, and serious adverse events. The research team will also assess laboratory markers related to the CAR-T therapy, such as lentivirus presence and CAR transgene levels. This extended monitoring aims to provide a comprehensive safety profile of cilta-cel over the long term.