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 children's 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 child's 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 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.

This research aims to evaluate the safety, tolerability, and early effectiveness of 4D-710, an investigational gene therapy, in adults with cystic fibrosis (CF) lung disease who cannot use or tolerate CFTR modulator therapy. It also includes a sub-study to assess this gene therapy in adults with advanced CF lung disease or frequent lung flare-ups while on current modulator treatments. The study is a Phase 1/2, open-label, multicenter trial focused on this population. Participants receive a single inhaled dose of 4D-710, a gene therapy using an adeno-associated virus to deliver a modified CFTR gene. The trial has different groups: those who cannot use modulator therapy will get varying doses to find the best dose for further study, while those on modulator therapy receive selected doses in the sub-study. The treatment is given once, and doses are explored and expanded to identify recommended levels. During the trial, participants are monitored for up to 60 months to track any side effects and overall safety. The main measure is the number and severity of adverse events. Participants will have lung function tests, oxygen level checks, and other health assessments throughout the observation period. The study will gather detailed safety and tolerability data to guide future research on this gene therapy in CF lung disease.

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 3/4 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 studying the combination of [177Lu]Lu-NeoB with ribociclib and fulvestrant in adults with advanced breast cancer that is estrogen receptor positive, HER2 negative, and gastrin releasing peptide receptor positive. This trial focuses on participants who have experienced early relapse after endocrine therapy or whose disease has progressed on endocrine therapy combined with a CDK4/6 inhibitor. The goal is to find the recommended dose of [177Lu]Lu-NeoB when used with these other treatments. The trial consists of a dose escalation phase testing four planned doses of [177Lu]Lu-NeoB (100, 150, 200, and 250 millicurie) in small groups, followed by a backfill phase to gather additional safety and preliminary effectiveness data at an established dose. Participants receive [177Lu]Lu-NeoB once every 28-day cycle for six cycles, along with daily ribociclib for the first 21 days of each cycle and fulvestrant on specific days starting from cycle 1 day 1. Pre- and perimenopausal women and men also get goserelin each cycle. Imaging with [68Ga]Ga-NeoB is done during screening, possibly at cycle 2 day 15, and after treatment to assess disease. Participants attend clinic visits every 28 days for treatment, safety checks, and dosimetry assessments, with additional visits early in cycles 1, 2, 3, and 5. Tumor evaluations occur every 8 weeks up to 18 months, then every 12 weeks until 36 months, and further as needed. Safety follow-up lasts 8 weeks after treatment ends, followed by long-term monitoring every 12 to 24 weeks until 5 years from enrollment or until withdrawal, death, or loss to follow-up. Researchers measure dose-limiting toxicities, adverse events, dose changes, drug levels in blood, tumor responses, and survival outcomes.

This research aims to evaluate the uptake of the imaging agent [18F]F-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 [18F]F-GLN, followed by up to two static scans from skull base to mid-thigh. A baseline FDG-PET/CT scan will also be performed, either as part of routine care or research. Some participants undergoing systemic therapy may have a second [18F]F-GLN PET/CT and possibly a second FDG-PET/CT 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 [18F]F-GLN uptake in breast cancer. Uptake will be compared to tumor markers of glutamine metabolism when tissue is available. Safety of [18F]F-GLN will be monitored in all participants. The primary outcome is to assess the kinetics and biodistribution of [18F]F-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 [18F]fluoroestradiol (FES) PET/CT 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 PET/CT 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 [18F]fluoropropyl-trimethoprim ([18F]F-TMP), an imaging drug injected to detect active bacterial infections in the body using Positron Emission Tomography/Computed Tomography (PET/CT). 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 PET/CT 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 [18F]F-TMP. These scans help researchers analyze tracer uptake and movement in the body over time. During the study, participants will have PET/CT imaging sessions to monitor the tracer's uptake in infected and non-infected tissues. Researchers will measure the tracer's 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.

Researchers are evaluating the effects of delgocitinib cream 20 mg/g applied twice daily compared to a non-active cream (vehicle) in adults aged 18 years and older with mild to severe palmoplantar pustulosis (PPP). This skin condition involves pustules on the palms and soles and can persist for more than three months. The study aims to determine if delgocitinib improves the severity and symptoms of PPP over 16 weeks. Participants will be randomly assigned to one of two groups: one applying delgocitinib cream twice a day and the other applying a matching vehicle cream twice a day, both for 16 weeks. The study is double-blind, meaning neither participants nor researchers know which treatment is given. The total study duration for each participant is about 18 weeks, including approximately 9 visits. During the study, participants will attend regular visits where their skin condition will be evaluated using several measures, including the Palmoplantar Pustulosis Area and Severity Index (PPPASI) and Physician Global Assessment (PGA). Researchers will also assess symptoms like pustule counts, itch, pain, quality of life, and work productivity. Safety and any side effects will be monitored throughout the study period.

Researchers are evaluating litifilimab (BIIB059), a monoclonal antibody, in adults with active cutaneous lupus erythematosus (CLE). This includes those with subacute or chronic CLE, with or without systemic lupus erythematosus (SLE), who have not responded well or tolerated antimalarial treatments. The study aims to assess how litifilimab affects skin disease activity using scoring tools such as CLA-IGA-R and CLASI, as well as its safety and impact on quality of life. The study has two parts: Part A and Part B. After screening, participants are randomly assigned to receive either litifilimab or a placebo injection under the skin every four weeks for 24 weeks in a double-blind setup. After this, all participants receive litifilimab for another 28 weeks. Those who finish may join a long-term extension study or enter a safety follow-up lasting up to 24 weeks. Treatment involves regular injections and monitoring during these periods. Participants will undergo assessments of skin symptoms, immune responses, and quality of life using questionnaires. Researchers will measure outcomes like the percentage of participants achieving low skin redness scores and significant reductions in skin disease activity. Safety monitoring continues through the study and follow-up, with total participation lasting up to 80 weeks.