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 adults with indolent systemic mastocytosis ISM whose symptoms are not well controlled by SDT alone. This randomized, double-blind, placebo-controlled Phase 23 study includes multiple parts to assess different doses and durations of elenestinib treatment, including an open-label extension for participants finishing earlier phases. The study also enrolls participants who have previously received an approved selective KIT inhibitor and includes pharmacokinetic groups. Participants receive oral elenestinib or placebo once daily alongside SDT, which is personalized based on individual symptom management needs. Part 1 focuses on short-term treatment lasting up to 12 weeks, while Part 2 extends treatment to approximately 48 weeks. Part 3 and other parts allow treatment for up to about 5 years. The study monitors participants through these phases to evaluate how elenestinib affects symptoms, disease markers, and safety over time. During the study, participants undergo regular assessments of symptoms using the ISM-Symptom Assessment Form ISM-SAF, laboratory tests including serum tryptase and KIT D816V allele levels, bone marrow evaluations, and quality of life measures. Researchers track adverse events and changes in disease-related factors at various points up to 5 years. This thorough monitoring helps measure treatment effects and safety over both short and long-term periods, with total participation lasting several years depending on the study part.

Researchers are studying patients with soft tissue sarcoma of the extremity, trunk, or retroperitoneum to evaluate a shorter, five-day course of preoperative radiation therapy. This approach is being compared to the standard preoperative radiation given over 25 days to assess safety and effectiveness. The study is a Phase 2 trial sponsored by Stanford University focusing on treatment for this type of cancer. Participants will receive external beam radiotherapy delivering a total dose of 30 Gy divided into five equal daily fractions of 6 Gy each over 5 to 10 business days before surgery. This abbreviated radiation schedule aims to reduce the treatment duration while still targeting the tumor and any areas where microscopic disease might exist. During the study, participants will be monitored for post-operative complications up to 120 days after surgery. Researchers will also evaluate the rates of local cancer recurrence at 2 and 5 years following surgical removal. The study includes assessments of health status, and participants must attend scheduled visits for treatment and follow-up as part of their involvement, which continues until at least the primary outcome measurement is completed.

Researchers are studying metastatic castration-resistant prostate cancer mCRPC to find new treatment options. This trial evaluates if the study medicine ifinatamab deruxtecan I-DXd or MK-2400 helps people live longer overall and experience slower cancer growth or spread compared to chemotherapy. The study is a Phase 3 trial comparing I-DXd with standard chemotherapy for mCRPC patients. Participants are randomly assigned to receive either I-DXd at 12 mgkg every 3 weeks through intravenous infusion or docetaxel chemotherapy at 75 mgm2 every 3 weeks combined with daily prednisone pills. Treatment continues until the disease progresses, unacceptable side effects occur, or treatment is stopped for other reasons. Premedication is given before each dose of I-DXd to help prevent nausea and vomiting. During the study, participants will have regular visits for treatment and monitoring. Researchers will assess overall survival and radiographic progression-free survival for up to about 36 months. Additional measures include response rates, time to pain progression, PSA progression, and adverse events. The study tracks safety, treatment effects, and quality of life over a long follow-up period to better understand the potential benefits and risks of I-DXd compared to chemotherapy.

Researchers are evaluating letermovir in children and adolescents under 18 years old who weigh less than 40 kilograms and have received a kidney transplant. The study aims to understand how letermovir behaves in the body over time and to assess its safety and how well participants tolerate it. This phase 1, open-label study focuses on cytomegalovirus CMV prevention in this specific pediatric transplant population. Participants who are between 4 and 52 weeks post-kidney transplant will receive letermovir either orally as tablets or pellets, or through a gastrostomy or nasogastric tube as pellets, for 7 consecutive days. The dosing is adjusted based on weight bands. This is a single-group study without a placebo or comparator arm. During the study, participants will be closely monitored through blood samples to measure drug levels and CMV DNA, including the primary outcome of the drug concentration area under the curve over 24 hours on Day 7. Safety will be assessed by recording any adverse events up to 21 days and study discontinuations due to adverse events up to 7 days. The study is expected to last until 2028 and includes assessments of kidney function stability and medication absorption tolerability.

Researchers are evaluating the safety, tolerability, and therapeutic effects of BNT113 combined with pembrolizumab compared to pembrolizumab alone as a first-line treatment for patients with unresectable recurrent or metastatic head and neck squamous cell carcinoma HNSCC positive for human papilloma virus 16 HPV16 and expressing the protein PD-L1 with a combined positive score of 1 or higher. This is an open-label, multi-site, Phase IIIII clinical trial consisting of two parts an initial safety run-in phase and a randomized phase. In the safety run-in phase Part A, patients receive BNT113 in combination with pembrolizumab to confirm safety and tolerability at selected dose levels. The randomized phase Part B compares BNT113 combined with pembrolizumab against pembrolizumab monotherapy. Treatments are given by intravenous injection or infusion and continue for up to 24 months. An optional pre-screening phase allows tumor samples to be tested for HPV16 DNA and PD-L1 expression before the main trial screening. Participants will be closely monitored throughout the study. Assessments include safety evaluations, tumor response, and survival outcomes such as overall survival and progression-free survival. Tumor tissue samples must be provided for testing. Researchers will measure treatment-emergent adverse events, response rates, duration of response, and disease control. The study may last up to 48 months, with ongoing safety and efficacy monitoring during and after treatment.

Researchers are evaluating the effectiveness of two PTSD treatments, Cognitive Processing Therapy CPT and STAIR Narrative Therapy SNT, among adults who identify as part of the LGBTQIA community. The study aims to find out which treatment better reduces PTSD symptoms, improves quality of life, and lessens depression. It also explores how stress from stigma, discrimination, and drug or alcohol use may affect treatment outcomes, and whether these treatments work differently for various groups within the LGBTQIA population. Participants will be randomly assigned to receive either CPT, which involves about 12 sessions focusing on changing beliefs and processing emotions related to trauma, or SNT, a longer therapy with about 16 sessions including coping skills for emotional regulation and a trauma-focused narrative component. Treatments are delivered either in person or via videoconferencing. The study compares these two therapies to understand which is more effective for LGBTQIA adults with PTSD. During the study, participants will complete assessments at the start and at 3, 6, and 12 months to measure changes in PTSD symptoms, depression, quality of life, and satisfaction with therapy. Researchers will also track treatment completion rates. The total participation time covers these assessments and treatment sessions, with monitoring to understand how well participants respond and adhere to the therapies.

Researchers are evaluating BGB-16673, an oral drug, in adults with various types of B-cell malignancies such as marginal zone lymphoma, follicular lymphoma, mantle cell lymphoma, chronic lymphocytic leukemia, Waldenstrm macroglobulinemia, diffuse large B-cell lymphoma, and Richters transformation. This study includes Phase 1 dose finding and safety expansion, followed by Phase 2 expansion cohorts to determine recommended doses and further assess safety and efficacy. The study is divided into several parts, starting with Phase 1 dose escalation to find safe dosage levels, including monotherapy dose escalation and safety expansion in selected doses. Phase 2 involves expansion cohorts where participants receive the recommended doses identified in Phase 1 for further safety and efficacy evaluation. Some cohorts include participants who have not received prior BTK inhibitors, and Japanese participants are also enrolled to assess safety. Treatments are orally administered. Participants will undergo regular assessments including monitoring for adverse events, disease response, and drug concentration levels in the blood at various time points. Researchers will measure outcomes such as overall response rate and progression-free survival over approximately three years. Safety and tolerability will be closely tracked, and quality of life questionnaires will be completed at scheduled intervals. Participation may last several years, including follow-up periods to monitor long-term effects.

Researchers are evaluating the safety and potential benefits of the Eclipse XL1 System in both children and adults who have short bowel syndrome, a condition where part of the small intestine is missing or has been removed. This single-group, open-label clinical study includes participants aged from 3 months to 65 years old and is conducted across multiple sites in the United States to better understand how this device might help with intestinal growth. The study involves placing the Eclipse XL1 device inside the intestine during either a standalone surgery or an already planned procedure. Participants may receive up to two devices simultaneously or undergo multiple procedures if needed. The device is secured inside the intestine and marked with metal clips to monitor its position. Researchers will track how the device affects intestinal lengthening and nutritional status over time, and the device will be observed through imaging until it is removed or naturally passes out. Participants will be monitored while the device is in place and followed up at 3 and 6 months after the device is removed or passes naturally. During the study, their nutrition, weight, and stool will be assessed, and radiographic exams will confirm device placement and safety. Depending on their health, subjects may stay in the hospital or be discharged while participating. The total study duration allows for careful evaluation of device-related adverse events and improvements in intestinal health.

Researchers are studying MEN2312, a lysine acetyltransferase 6 KAT6 inhibitor, in adults with advanced breast cancer that is not curable. This first-in-human, phase 1 study evaluates MEN2312 alone and in combination with elacestrant to understand its safety and determine the best dose. Participants have specific genetic alterations in their tumors and have received prior endocrine therapy and cyclin-dependent kinase 4 and 6 inhibitor treatment. Participants will be randomly assigned to receive either MEN2312 by itself or MEN2312 combined with elacestrant, both given as oral tablets. The study follows a sequential design and aims to identify dose-limiting toxicities and recommend the phase 2 dose over several months of treatment. This includes monitoring drug levels in the body and how the body processes the medications. During the study, participants will have regular assessments to monitor side effects, tumor response, and overall health. These include evaluating the number of dose-limiting toxicities within the first 28 days and tracking response rates, progression-free survival, and overall survival for up to nine months after treatment ends. Researchers will also measure drug concentration and excretion to better understand the treatment effects and safety over time.

This research aims to evaluate the safety and gene expression of delandistrogene moxeparvovec, a gene transfer therapy, in males with Duchenne Muscular Dystrophy DMD. The study focuses on non-ambulatory participants in Cohort 8, while enrollment for earlier cohorts has been completed. The study is open-label and conducted by Sarepta Therapeutics, Inc., with a maximum participant duration of 156 weeks. Participants will receive a single intravenous infusion of delandistrogene moxeparvovec on Day 1. The study measures dystrophin protein expression at 12 weeks post-infusion, as well as safety outcomes including acute liver injury and other adverse events up to 72 weeks for Cohort 8. Additional assessments monitor vector shedding, antibody levels, treatment-emergent adverse events, and steroid use for up to 156 weeks. During the study, participants undergo various assessments including laboratory tests, biomarker evaluations, and motor function testing. Researchers will collect samples such as urine, saliva, and stool to track vector shedding and measure immune responses. Safety monitoring includes tracking liver-related events and infections. The total study participation may last up to about 3 years, allowing for long-term follow-up of treatment effects and safety.