Stanford

Researchers are investigating the drug bezuclastinib in an open-label, two-part Phase 2 study for patients with Advanced Systemic Mastocytosis (AdvSM), including 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, and how the drug behaves in the body for these serious conditions. Bezuclastinib is given orally as tablets taken continuously in 28-day cycles. The study has two parts: Part I focuses on identifying safe and tolerable doses of bezuclastinib over 18 months, while Part II evaluates its effectiveness by measuring the objective response rate and confirming the relationship between drug exposure and response during another 18-month period. Participants will undergo assessments including clinical evaluations, laboratory tests, and monitoring of their disease status to determine treatment effects and safety. Researchers will track the drug's impact on the disease and patient health throughout the study, which involves continuous treatment and follow-up over the specified time frames.

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 studying the safety and effectiveness of 67Cu-SAR-bisPSMA in men with metastatic castrate resistant prostate cancer that expresses PSMA. This Phase I/IIa trial focuses on participants whose cancer has progressed despite androgen deprivation therapy and prior treatments with androgen receptor pathway inhibitors. The study aims to understand how the drug distributes in the body, determine safe dosing levels, and evaluate treatment responses over several years. Participants receive two drugs: 64Cu-SAR-bisPSMA for imaging and dosimetry, and 67Cu-SAR-bisPSMA for treatment. The study includes a dose escalation phase to find the maximum tolerated or feasible dose of a single 67Cu-SAR-bisPSMA dose over 8 weeks, followed by a recommended dosing phase with two doses over 14 weeks. Imaging with PET/CT scans using 64Cu-SAR-bisPSMA helps model dosimetry for the therapeutic 67Cu-SAR-bisPSMA. The treatment is given under close monitoring to assess safety and response. During the study, participants undergo PET/CT scans, blood tests, ECGs, and vital sign monitoring at various times up to 5 years to track drug distribution, treatment effects on prostate specific antigen levels, radiographic cancer response, and any side effects. Safety is closely followed through laboratory results and adverse event reporting. Participants are monitored for long-term tolerability and treatment outcomes, with study involvement lasting up to five years.

Researchers are evaluating the use of a PET isotope called Fluorine-18 (18F) bound to Choline to detect parathyroid adenomas in patients whose standard 99mTc Sestamibi SPECT/CT scans are negative or unclear. This prospective, single-center, single-arm study focuses on participants suspected of having parathyroid adenoma, a condition characterized by high serum calcium and abnormal parathyroid hormone levels. The study is conducted in phases 2 and 3 to assess this imaging method's ability to identify lesions more effectively. Participants will receive an intravenous injection of 18F Fluorocholine at a dose of 5 mCi ± 20%. The study involves only this investigational imaging approach without a comparison group. The goal is to observe how many lesions are detected within one hour using the 18F Fluorocholine PET/CT scan after the injection. During the study, participants will be monitored for lesion detection using PET/CT imaging. Safety and liver and kidney function will be evaluated through laboratory tests, including bilirubin, ALT, AST, and creatinine levels. Performance status will be assessed, and ECG monitoring will be done to check QT interval. The main outcome is the number of lesions detected by PET/CT within one hour after tracer administration, and participants must provide written consent to join the study.

Researchers are evaluating a 12-week early intervention program designed to improve social communication skills in children with developmental disorders, including Autism Spectrum Disorder, neurogenetic disorders, and intellectual disability. This study focuses on preschool-aged children who experience social communication deficits and aims to assess the effectiveness of intensive treatment in improving these skills. The intervention involves 12 hours per week of behavioral therapy delivered either in a center-based preschool environment or at home. This intensive program spans 12 weeks, with treatment sessions tailored to support social communication development in young children with developmental challenges. Participants will be assessed before and after the 12-week program using tools such as the Parent Rated Social Responsiveness Scale (SRS-2) to measure changes in social responsiveness. The study involves thorough testing procedures to obtain valid scores, with ongoing monitoring to evaluate treatment effects over the course of the program.

Researchers are evaluating a combination treatment using BNT326 and BNT327 in adults with advanced or metastatic non-small cell lung cancer (NSCLC), including those with relapsed, progressive, or treatment-nafve disease. This multi-site, open-label study includes dose-finding and dose-expansion phases to investigate the safety, tolerability, and preliminary effectiveness of this combination therapy. The study targets patients whose tumors are advanced, metastatic, or recurrent with no curative treatment options available and includes participants with different genomic alterations. The study is divided into several parts: Part 1 is a dose escalation phase to find safe dose levels of BNT326 with BNT327; Part 2a expands the dose to further evaluate safety and initial efficacy; Part 2b focuses on dose optimization and understanding the contributions of each component. Participants receive intravenous infusions of BNT326 and BNT327, with some cohorts possibly receiving additional treatments such as pembrolizumab or standard chemotherapy. Treatment continues until disease progression, unacceptable side effects, withdrawal, or a maximum of 24 months. Dose levels for certain cohorts are determined based on earlier phase data, and some parts include randomization to different treatment groups. Participants undergo a screening period before starting treatment, followed by treatment, safety follow-up, efficacy follow-up, and long-term survival monitoring, totaling about 36 months. Researchers assess dose-limiting toxicities within the first 21 days of treatment and monitor adverse events, treatment interruptions, and objective response rates up to 36 months. Tumor measurements, safety labs, imaging, and patient health status are regularly evaluated. The study tracks tolerability and efficacy while ensuring participant safety throughout treatment and follow-up.

Researchers are evaluating the safety and potential benefits of the Eclipse XL1 System in both adult and pediatric patients with short bowel syndrome, a condition characterized by having 50% or less of the expected bowel length. This open-label, single-arm study will enroll participants aged 3 months to 65 years across up to 10 sites in the United States, aiming to recruit over 24 to 36 months. The study focuses on how this device may help with distraction enterogenesis, a process to stimulate intestinal growth or function. The Eclipse XL1 device is placed into the intestine during a standalone surgical procedure or one already planned for the patient. Surgeons select the proper device size based on intestinal diameter and secure it inside the intestine with sutures and metal clips for radiographic monitoring. Participants may receive up to two devices at once or undergo multiple procedures as needed. The device's position and effects will be regularly checked using imaging until it is removed or naturally passes through the intestine. Participants will be monitored while the device is in place and followed up at 3 and 6 months after device removal or passage. The study will track nutrition, weight, and stool assessments to evaluate safety and probable benefit. Some patients might stay in the hospital during the study depending on their health, with ongoing assessments including radiographic exams and clinical reviews over several months. The main outcome measured is the occurrence of device and procedure-related adverse events within 4 to 6 months.

Researchers are studying BAY 3713372, a new drug being developed to treat solid tumors with a specific genetic change called MTAP deletion. The drug works by blocking a protein called PRMT5, which may kill cancer cells with this deletion while sparing normal cells. This first-in-human study aims to understand the safety, how the body processes the drug, and its effectiveness in people with these MTAP-deleted solid tumors. Participants will receive BAY 3713372 orally every day. The study starts with a dose escalation phase, where different groups get increasing doses to find a safe and effective dose. After this, a dose expansion phase will include more participants receiving the drug alone or with other treatments. Participants can continue treatment as long as they benefit and do not experience severe problems. During the study, participants will visit the study site multiple times before and during treatment, and follow-up visits after treatment ends. Doctors will monitor health through blood and urine tests, heart checks with electrocardiograms, and imaging scans like CT or MRI to track cancer changes. Tumor samples may also be taken. Safety and treatment response will be closely assessed, including adverse events and how the drug behaves in the body. Participants will be contacted every three months for up to two years after treatment to check their health.

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 assess the long-term safety of a gene-modified regulatory T cell (Treg) therapeutic in people who have previously received this treatment. It focuses on identifying any delayed adverse events, including their type, severity, and potential link to the gene-modified Treg therapy. The study is observational and involves individuals treated in earlier clinical studies, with conditions including Rheumatoid Arthritis and Hidradenitis Suppurativa. Participants will undergo long-term safety monitoring for up to 15 years following their initial gene-modified Treg treatment. During scheduled visits, they will complete health questionnaires, have routine physical exams, and provide biospecimens such as blood and tissue samples. These procedures help monitor for any delayed adverse effects connected to the prior cell therapy. Throughout the study, researchers will review adverse events, medical history, and medication use to evaluate safety over time. The main outcome measure is the incidence of delayed adverse events possibly related to the gene-modified Treg therapeutic. Participants must comply with all study procedures during this extended follow-up period, ensuring thorough monitoring and safety evaluation.