Philadelphia

Researchers are evaluating an early behavioral intervention program for toddlers aged 16 to 48 months who are waiting to receive a formal autism diagnosis. The study aims to address delays in autism diagnosis and intervention by offering caregivers access to a self-directed online training resource during the waitlist period. This approach seeks to empower parents to support their childrens development early, potentially reducing the harms associated with waiting for diagnosis and intervention services. Participants will be randomly assigned to either receive access to the Online Parent Training in Early Behavioral Intervention OPT-In-Early program or be placed on a waitlist without intervention for six months. The OPT-In-Early program includes 14 modules 6 mandatory and 8 optional with text and video materials teaching caregivers strategies to improve language, social, and adaptive skills and reduce disruptive behaviors. Parents can also schedule brief support calls with a clinician for guidance during the intervention period. After six months, waitlist participants will gain access to the program, but outcomes will not be measured for them. Throughout the study, caregivers complete assessments including individualized goal setting with video recordings to capture their childs current skills and progress. Standardized measures of adaptive behavior, parental stress, and autism-related challenges are collected at baseline and after six months. Researchers will monitor program engagement using website usage data and support call frequency. The primary outcome is change in goal attainment as measured by Goal Attainment Scaling, with additional measures assessing adaptive functioning and parental well-being. The total participation time is six months, during which families receive ongoing support and data collection.

Researchers are evaluating the combination of CGT9486 and sunitinib compared to sunitinib alone in patients with locally advanced, unresectable, or metastatic Gastrointestinal Stromal Tumors GIST. This Phase 3, open-label international trial involves multiple parts, including dose confirmation, drug interaction assessments, and efficacy comparisons. The study also includes substudies focusing on drug-drug interaction potential and first-line treatment in patients with specific genetic mutations KIT exon 9. Approximately 482 patients will participate across these parts.

Researchers are evaluating whether 18F-fluciclovine Axumin PET imaging can help doctors distinguish between true tumor growth and other changes in children with high-grade gliomas, including diffuse midline glioma. This distinction is important because true tumor progression may require a change in treatment, while post-treatment changes usually do not. Conventional MRI scans cannot reliably make this differentiation, so this study aims to see if 18F-fluciclovine PET imaging can serve as a useful diagnostic tool. Participants will receive a single intravenous injection of 18F-fluciclovine before undergoing a combined PET-MRI scan. This imaging process is designed to assess tumor status by detecting physiological changes. The study focuses on children and young adults aged 1 to under 21 years with measurable high-grade gliomas or diffuse midline gliomas. The study is an early phase 1 trial, and treatment involves only this one-time imaging procedure. During the study, researchers will analyze the imaging results and compare them to histopathology findings within four weeks and evaluate safety over six months. Participants will be monitored for any side effects related to the imaging agent. The study will assess the usefulness and safety of 18F-fluciclovine PET-MRI in guiding treatment decisions for pediatric high-grade glioma. Participation lasts through these assessments, with imaging and follow-up evaluations scheduled accordingly.

Researchers are studying a new imaging tracer called 18FFTHA to visualize the intestinal and central lymphatic system in adults. The project spans about two years, starting with development and testing of the tracer on healthy volunteers, followed by investigation in patients suspected of having obstruction in the thoracic duct TD, a key lymphatic vessel. This early phase 1 study aims to better understand lymphatic system function and obstruction using this novel imaging technique. Participants will receive an oral dose of the 18FFTHA tracer in capsule form, followed by several whole-body PETCT scans over approximately four hours with breaks between scans. Initially, up to 10 healthy adults will undergo this single tracer administration and imaging session. Subsequently, about 15 patients with symptoms like chronic abdominal pain and edema suspected to be due to TD obstruction will undergo the same procedure. Some patients who undergo clinical surgical bypass for their condition will be invited back for a follow-up PETCT scan after surgery. During the study, participants will fast overnight before imaging and consume an Ensure drink after tracer administration. Researchers will monitor tracer transit time and radiation dosimetry over the following weeks. Safety assessments include pregnancy testing for women of childbearing potential and evaluation of ability to tolerate imaging. The total study participation period is about two years, with imaging visits scheduled on study days and follow-up imaging for surgical patients as applicable.

Researchers are evaluating the safety, tolerability, and early effects of 4D-710, an investigational gene therapy, in adults with cystic fibrosis CF lung disease who cannot use or tolerate existing CFTR modulator therapies. A sub-study also includes adults with advanced CF lung disease or frequent lung flare-ups who are currently on CFTR modulator therapy. This Phase 12 open-label trial aims to find appropriate dosing and assess potential benefits for these patient groups. Participants receive a single inhaled dose of 4D-710, which is a gene therapy designed to deliver a corrected version of the CFTR gene to lung cells. The study includes a dose exploration phase for those ineligible for modulator therapy, a dose expansion phase at selected doses, and a sub-study for participants on modulator therapy receiving various doses. Each participant undergoes one administration of the therapy during the trial. Throughout the study, participants are monitored for adverse events over a 60-month period. Evaluations include lung function tests, oxygen saturation measurements, and tracking of pulmonary exacerbations. Participants maintain their existing treatments if applicable, and researchers assess safety and early signs of effectiveness. The total study duration extends up to approximately nine years, including long-term observation after dosing.

Researchers are evaluating the use of XYOSTED as a testosterone replacement therapy in adolescent males aged 12 to under 18 years with primary or secondary hypogonadism, a condition where the body produces little or no testosterone. This Phase 34 open-label study aims to assess how well XYOSTED supports the continuation or start of puberty, along with its safety and the testosterone levels it maintains. Participants will receive XYOSTED injections at doses tailored to their weight and targeted pubertal stage. Dose adjustments will be made based on testosterone levels measured at specific intervals after dosing, with evaluations approximately every three months to reach the desired hormone levels. After completing the 52-week initial study period, participants may enter a 24-month extension to further monitor long-term safety and treatment effects. Throughout the study and extension, participants will undergo clinical examinations including pubertal staging, blood tests for testosterone and other labs, bone density scans, body composition assessments, and X-rays to monitor bone age. Researchers will track changes in puberty progression, bone health, body measurements, and hormone levels. Participants will attend regular clinic visits every six months during the extension phase to continue safety and pharmacokinetic evaluations.

Researchers are evaluating the long-term safety and tolerability of LB-102 in adults with stable schizophrenia who have had inadequate responses, side effects, or issues with their current antipsychotic medications, or who have completed prior LB-102 studies. This Phase 3, open-label, multicenter trial focuses on patients aged 18 to 65 years with stable disease and aims to provide extended monitoring of this treatment. Participants will receive LB-102 with flexible dosing ranging from 50 mg to 100 mg. This single-group study involves administering the drug openly over 52 weeks to assess how well patients tolerate it and to monitor safety during this period. Throughout the study, participants will undergo evaluations including monitoring adverse events and treatment-emergent events. Effectiveness will be assessed using the Positive and Negative Syndrome Scale PANSS. The study lasts up to 52 weeks, during which safety and tolerability are carefully observed and recorded.

This research aims to evaluate the uptake of the imaging agent 18FF-GLN in patients with known or suspected primary or metastatic breast cancer who have at least one lesion 1.0 cm or larger. Up to 40 patients will participate, including at least 10 with estrogen-receptor-expressing breast cancer and 10 with triple-negative breast cancer. Participants may be new to treatment or have received up to three weeks of therapy at the time of the imaging scan. This is an observational study where imaging results do not guide treatment decisions, which remain based on clinical judgment. Participants will undergo approximately 60 minutes of dynamic PET scanning after injection of 18FF-GLN, followed by up to two static scans from skull base to mid-thigh. A baseline FDG-PETCT scan will also be performed, either as part of routine care or research. Some participants undergoing systemic therapy may have a second 18FF-GLN PETCT and possibly a second FDG-PETCT to explore changes in uptake related to treatment. Additional low-dose CT scans may be done if motion occurs during imaging. Throughout the study, metabolism data and image quality will be collected, along with preliminary information on 18FF-GLN uptake in breast cancer. Uptake will be compared to tumor markers of glutamine metabolism when tissue is available. Safety of 18FF-GLN will be monitored in all participants. The primary outcome is to assess the kinetics and biodistribution of 18FF-GLN, with secondary outcomes including safety, associations with tumor markers, metabolism, and changes after therapy, over a three-year period.

Researchers are evaluating the use of a new imaging method called 18Ffluoroestradiol FES PETCT to study estrogen receptor activity in women with recurrent, metastatic, or non-operated uterine cancer. This phase II study aims to understand how estrogen receptors are present in the cancer, which could help guide future research and treatments. The study involves women who are at least 18 years old and receiving care at the University of Pennsylvania. Participants will undergo up to two PETCT scans using the investigational FES tracer. The first scan is required and happens before starting a new therapy. Some patients may have a second optional scan if their disease progresses, to compare changes in estrogen receptor activity. The choice of therapy will remain with the treating doctor and will not be influenced by the scan results. Optional biopsies and blood samples may also be collected to study tumor cells and hormone levels. During the study, participants will have scans from the skull base to mid-thigh about 60 minutes after receiving the FES injection. Researchers will review standard imaging and clinical exams to evaluate cancer sites. Optional blood draws will analyze circulating tumor cells and hormone levels. The main outcome measured is the rate of FES positivity in estrogen receptor-positive uterine cancer over one year. Safety, progression, and changes in imaging and pathology will also be monitored. Participation may last up to several years as outcomes and progression are followed.

Researchers are evaluating a radioactive tracer called 18Ffluoropropyl-trimethoprim 18FF-TMP, an imaging drug injected to detect active bacterial infections in the body using Positron Emission TomographyComputed Tomography PETCT. This Phase 1 study aims to understand how this tracer distributes and moves through the body in human patients with known or suspected bacterial infections. The tracer is based on trimethoprim, a widely used antibiotic, and is studied here for its imaging potential rather than treatment. Participants will join one of two study groups the Biodistribution cohort or the Dynamic cohort. The Biodistribution group, including up to 5 patients, will undergo several PETCT scans from head to mid-thigh or feet over about 4 hours to observe tracer distribution. The Dynamic group, with up to 15 patients, will have approximately 60 minutes of dynamic scanning followed by up to two static scans after injection of 18FF-TMP. These scans help researchers analyze tracer uptake and movement in the body over time. During the study, participants will have PETCT imaging sessions to monitor the tracers uptake in infected and non-infected tissues. Researchers will measure the tracers biodistribution and kinetics, including changes after therapy. Safety monitoring includes assessments for any inability to tolerate imaging and pregnancy testing for women of childbearing potential. The study duration for outcome measures is approximately 3 years, with no mention of long-term treatment or follow-up beyond imaging evaluations.