Palo Alto

Researchers are evaluating the safety and effectiveness of elenestinib (BLU-263) combined with symptom-directed therapy (SDT) compared to placebo plus SDT in people with indolent systemic mastocytosis (ISM) whose symptoms are not well controlled by SDT alone. This Phase 2/3 randomized, double-blind, placebo-controlled study includes participants with ISM and smoldering systemic mastocytosis, and also involves groups for pharmacokinetic studies and participants who previously received a selective KIT inhibitor. The study is divided into multiple parts. Parts 1 and 2 enroll participants with ISM who will receive either elenestinib oral tablets or placebo alongside their symptom-directed therapy. Participants from Part 2 may continue into Part 3, which is an open-label extension where all receive elenestinib. Part K enrolls participants with ISM who have prior experience with selective KIT inhibitors. The study tracks treatment effects and safety over time. Participants will be monitored for up to 5 years, with assessments including the number of treatment-emergent adverse events, changes in symptom scores measured by the ISM-Symptom in Assessment Form, and overall safety monitoring. Evaluations occur at baseline, 13 weeks, 49 weeks, and throughout the long-term follow-up. The study also includes detailed tracking of symptom control and adverse events to evaluate the impact of treatment on participants' health and quality of life.

Researchers are evaluating the NSite device, an investigational 3D body surface modeling tool, in adolescents with idiopathic scoliosis aged 10 to 18 years. The study aims to validate whether the device produces consistent results when used by different users in a clinical setting. This will help support the device's regulatory submission by confirming its reproducibility across multiple operators. Participants will be scanned using the NSite application by three separate users, who may include research coordinators, physicians, or authorized staff. Each user will scan the patient's back while the patient assumes a forward bending position. Users will follow a tutorial before scanning to ensure proper use. The scans will be analyzed to determine if different users generate similar predicted probabilities of a major spinal curve (Cobb angle) of 20 degrees or more. During the study, participants will undergo at least two scans by different users, with poor quality scans excluded from analysis. The researchers will compare the device outputs across users to assess congruence. The primary outcome is the reproducibility of the NSite device outputs, measured by the percentage of participants with overlapping confidence intervals in predicted curve magnitude. The study plans to enroll 13 patients and complete the scanning process within about one month.

Researchers are evaluating a shortened course of preoperative radiation therapy for patients with soft tissue sarcoma located in the extremity, trunk, or retroperitoneum. This study aims to assess the safety and effectiveness of delivering radiation over five days, compared to the standard treatment which spans 25 days. The study is a Phase 2 clinical trial focused on improving treatment schedules for this type of cancer. Participants will receive external beam radiation therapy targeted to the tumor and surrounding areas that may contain microscopic disease. The total radiation dose will be 30 Gy, divided into five equal doses of 6 Gy each, administered over a period of 5 to 10 business days. This abbreviated radiation course is given before surgery as part of the treatment plan. Throughout the study, researchers will monitor participants for complications occurring within 120 days after surgery. Patients will be assessed for treatment safety and response, with follow-up visits to evaluate outcomes. The study involves consenting adults aged 18 or older who meet specific health criteria and agree to participate in the research.

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 investigating ways to prevent cytomegalovirus (CMV) infection in children and adolescents who have received a kidney transplant and weigh less than 40 kilograms. This Phase 1 study aims to understand how the drug letermovir behaves in the body over time and to evaluate its safety and tolerability in this young population. Participants receive letermovir orally, either as tablets or pellets, or through a gastrostomy or nasogastric tube if pellets are used. The treatment is given once daily for seven consecutive days. This study is open-label and single-arm, meaning all participants receive the same treatment, and the study monitors them closely throughout this period. During the study, participants will have blood samples collected before the first dose and at several points up to 24 hours after dosing on Day 7 to measure how the drug is processed by the body. Researchers will also monitor kidney function stability, CMV DNA levels, and any side effects to assess safety. The study focuses on children and adolescents younger than 18 years and weighing between 2.5 and less than 40 kilograms, with a total participation time covering at least seven days of treatment and associated assessments.

This research aims to evaluate the effectiveness and safety of leriglitazone in adult male patients diagnosed with cerebral adrenoleukodystrophy (cALD), a progressive neurological condition. The study is a Phase 3 clinical trial focusing on males aged 18 years and older who have specific brain lesions related to cALD. It excludes patients who are candidates for or willing to undergo hematopoietic stem cell transplantation (HSCT). Participants will be randomly assigned to receive either leriglitazone, given once daily at a dose of 15 mg/ml with an initial volume of 10 ml, or a matching placebo that looks and tastes the same but contains no active drug. The treatment period includes planned assessments at 18, 27, and 36 months, with the primary measure being the time until death or becoming bedridden requiring permanent ventilatory support. Throughout the study, participants will be monitored regularly to assess neurological function and overall health. Researchers will collect data on brain lesion progression, functional disabilities, and cognitive status to evaluate treatment impact and safety. The total duration of treatment and follow-up spans up to 36 months, with interim analyses at 18 and 27 months to evaluate ongoing results.

Researchers are evaluating the safety, tolerability, and therapeutic effects of a combination treatment using BNT113 and pembrolizumab compared to pembrolizumab alone for patients with unresectable recurrent or metastatic head and neck squamous cell carcinoma (HNSCC) that is positive for human papillomavirus 16 (HPV16+) and expresses the PD-L1 protein with a combined positive score of 1 or higher. This Phase II/III trial includes patients whose cancer cannot be treated with local therapies and who have not received prior systemic anticancer therapy for their current disease condition. The trial consists of two parts. Part A is a non-randomized Safety Run-In Phase to confirm the safety and tolerability of BNT113 combined with pembrolizumab at the selected dose. Part B is a randomized phase that compares BNT113 plus pembrolizumab against pembrolizumab alone as first-line treatment. Patients in Part A continue their treatment without randomization. Treatments are given by intravenous injection or infusion, and patients may receive either combination therapy or monotherapy for up to 24 months. There is also an optional pre-screening phase to test tumor samples for HPV16 DNA and PD-L1 expression before entering the main trial. Participants undergo regular assessments including tumor measurements based on RECIST 1.1 criteria confirmed by independent review. Researchers monitor treatment-emergent adverse events for up to 27 months in Part A and evaluate overall survival and progression-free survival for up to 48 months in Part B. Tumor tissue samples are collected before treatment to confirm eligibility. The study involves ongoing safety monitoring and efficacy evaluations throughout the treatment and follow-up periods.

Researchers are evaluating two treatments, Cognitive Processing Therapy and STAIR Narrative Therapy, to see how well they reduce posttraumatic stress disorder (PTSD) symptoms in adults who identify as lesbian, gay, bisexual, transgender, queer/questioning, intersex, asexual/aromantic, or other sexual and gender minorities (LGBTQIA+). This study also aims to understand if these treatments can improve quality of life and reduce depression, how stress from stigma and discrimination or drug and alcohol use might affect treatment results, and whether patients are satisfied with and complete these treatments. Additionally, the study looks at differences in treatment effects among various groups within the LGBTQIA+ community, including different genders, living areas, and racial or ethnic backgrounds. Participants will receive one of the two PTSD treatments being studied. Cognitive Processing Therapy focuses on teaching skills to change beliefs and process emotions related to trauma. STAIR Narrative Therapy teaches coping skills such as emotional regulation and interpersonal relationships, along with a trauma-focused narrative component. Treatments are delivered in person in San Francisco or through videoconferencing for those who have access to a suitable device. Throughout the study, participants will complete assessments at the start and after treatment to measure changes in PTSD symptoms using the PTSD Checklist for DSM-5. Researchers will also evaluate depression symptoms, quality of life, treatment satisfaction, and completion rates. The total duration and frequency of assessments are planned at baseline, 3, 6, and 12 months. The study requires participants to be able to comply with procedures, speak English or Spanish, and live in California, with safety monitored throughout.

Researchers are investigating BGB-16673, a targeted protein degrader aimed at treating various B-cell cancers including marginal zone lymphoma, follicular lymphoma, mantle cell lymphoma, chronic lymphocytic leukemia, Waldenström macroglobulinemia, and diffuse large B-cell lymphoma. The study includes both Phase 1 and Phase 2 parts to determine safe and effective dosing and to evaluate the drug's response in patients. The trial is conducted under the new company name BeOne Medicines, previously known as BeiGene. The treatment involves oral administration of BGB-16673. Phase 1 focuses on dose escalation and safety expansion to identify the maximum tolerated dose and recommended dose for expansion over approximately 28 days to 3 years. Phase 2 includes expansion cohorts to assess overall response rates over about 3 years. Participants may have prior treatments including Bruton tyrosine kinase inhibitors and other anticancer therapies depending on their cancer type and study phase. Participants will be monitored closely with assessments of adverse events from the first dose until 30 days after the last dose or before starting new therapy, whichever comes first, for up to 47 weeks. The study measures tolerability, dosing recommendations, and treatment response. Eligibility assessments include performance status and measurable disease, with safety and response evaluations continuing through both phases for up to three years.

Researchers are evaluating the safety of aerosolized RSP-1502 in people with cystic fibrosis who have chronic lung infections caused by Pseudomonas aeruginosa. This phase 1b/2a study compares different doses of RSP-1502 to an active control, aiming to find the maximum tolerated dose (MTD) and assess safety outcomes. Participants must meet specific lung function and infection criteria to join the study. The study involves administering RSP-1502 or an active control solution by inhalation using a nebulizer for 14 days. RSP-1502 contains tobramycin and CaEDTA in a sterile solution, while the active control is a tobramycin inhalation solution. After dose escalation to identify the MTD, a dose expansion phase compares the MTD of RSP-1502 to the active control for another 14 days. Participants will then be followed for 14 days after treatment ends. Participants will have their lung function tested with spirometry and undergo electrocardiograms on specific days during treatment. Researchers will monitor for any treatment-related adverse events and serious adverse events throughout the 28-day treatment and follow-up period. They will also track pulmonary exacerbations and other safety measures. The total participation includes dosing and a 14-day follow-up after treatment completion.