Miami

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 the use of 18F-Fluciclovine positron emission tomography PET as a biomarker to measure the response or progression of brain metastasis in participants treated with stereotactic radiosurgery SRS. This pilot imaging study focuses on adults diagnosed with brain metastases who are planned to receive SRS treatment. The study is sponsored by Baptist Health South Florida and is a Phase 1 clinical trial. Participants will undergo an 18F-fluciclovine PET scan at the time of their SRS planning magnetic resonance imaging MRI. They will then receive a single dose of SRS as part of their standard care. A second 18F-fluciclovine PET scan will be performed approximately 8 weeks after the SRS treatment, with a window of plus or minus 2 weeks. During PET scans, participants receive a 5-mCi dose of 18F-fluciclovine through intravenous injection, with imaging data collected up to 25 minutes post-injection. Throughout the study, participants will be monitored using PET imaging to evaluate changes in standardized uptake values SUV over 8 weeks, which helps assess tumor response. Tumor control will also be evaluated at 12 months. The study involves standard imaging, including MRI and PET scans, and tracks participant safety and treatment progress. Total participation timelines include initial scans at SRS planning and follow-up imaging about 8 weeks later, with tumor control assessed after one year.

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 PET imaging with the radioligand 18FPI-2620 to detect tau protein deposits in people with Alzheimers disease and healthy controls. This open-label, multi-center, non-randomized Phase 3 study aims to compare PET imaging results during life with brain tissue analysis after death to better understand tau pathology in Alzheimers. The study is sponsored by Lantheus Biosciences Ltd. and focuses on diagnostic accuracy and safety of this imaging technique. Participants receive an intravenous injection of 18FPI-2620 at a dose of 185 MBq 20%. The study involves a PET scan procedure that participants must tolerate, including lying still in the scanner. There are no randomized groups or placebo controls as this is an open-label study. The research compares the PET imaging findings with post-mortem brain autopsy results to evaluate the ability of this imaging to detect tau deposits accurately. During the study, participants undergo PET imaging and are monitored for their ability to tolerate the scan. Brain donation consent is required for post-mortem histopathological comparison. Researchers assess the diagnostic performance of the PET scan in correctly identifying tau-related pathology and Alzheimers disease changes. The primary outcome focuses on the accuracy of visual assessment of PET images compared to autopsy findings, with follow-up continuing until study completion and an average of one year after death.

Researchers are evaluating the safety and effectiveness of tenapanor in adults with Chronic Idiopathic Constipation CIC. This study is a 26-week, multi-center, randomized, double-blind, placebo-controlled trial followed by a 4-week treatment-free safety follow-up period. It aims to compare three different doses of tenapanor with a placebo taken twice daily to assess their impact on constipation symptoms. The study includes a 2-week screening period to confirm eligibility, followed by a 26-week randomized treatment period where patients receive either 5 mg, 25 mg, or 50 mg of tenapanor twice daily, or a matching placebo. Patients record their constipation symptoms daily in an electronic diary. After the treatment period, there is a 4-week safety follow-up without treatment to monitor any adverse effects. Participants will have regular visits every 2 to 6 weeks for safety checks including medical assessments, vital signs, ECG, and lab tests. Their symptom diaries will be reviewed throughout the study. The main outcome measured is the durable complete spontaneous bowel movements response at 12 weeks. Secondary outcomes include changes in bowel movement frequency, stool consistency, and straining. The total study duration is approximately 32 weeks including all phases.

Researchers are conducting a Phase 3, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of rilzabrutinib in adults with active Immunoglobulin G4-related disease IgG4-RD. The study aims to measure the time to the first adjudicated disease flare and assess other important outcomes such as flare-free rates, disease activity control, glucocorticoid use, and safety parameters including adverse events, laboratory tests, and electrocardiograms ECG. Participants will be assigned to one of two groups one receiving rilzabrutinib tablets and the other receiving placebo tablets, both administered orally. The treatment period lasts 52 weeks in a double-blind manner, preceded by a 4 to 6 week screening period. After treatment, there is a 2-week follow-up, with an optional open-label extension lasting up to 108 weeks. The study includes a total of 16 visits during the main period and up to 9 additional visits during the optional extension. During their participation, adults diagnosed with IgG4-RD will undergo repeated imaging procedures such as CT, MRI, PET, or ultrasound to assess disease status. Researchers will monitor disease flares, remission status, glucocorticoid dosage, clinical activity scores, laboratory values, vital signs, and ECG results. Safety monitoring continues up to week 160 to capture treatment-emergent adverse events. Overall, participation lasts up to 60 weeks, with possible extension for those continuing in the optional phase.

Researchers are evaluating the effectiveness of delgocitinib cream applied twice daily compared to a cream vehicle for treating adults with mild to severe lichen sclerosus LS, a skin condition affecting mostly females in the anogenital area. The study is conducted in two parts Part 1 enrolls female participants to determine the best dose, and Part 2 includes both female and male participants to further assess the chosen dose and safety in males. Participants receive different dosing regimens of delgocitinib cream or cream vehicle applied twice daily during a 12-week initial treatment period, followed by a 40-week continuation treatment period. Different groups receive varying doses or sequences of delgocitinib cream and cream vehicle to compare effects. A substudy evaluates male participants separately to assess safety and efficacy. During the trial lasting between 55 and 60 weeks, participants undergo regular assessments including clinical evaluations of LS severity using IGA-LS scores, pain and itch numerical rating scales, and quality of life indexes. Safety is monitored through adverse event tracking and laboratory tests. The main outcome measured is the number of participants achieving a specific improvement in LS severity at Week 12, with additional evaluations continuing through Week 52.

Researchers are evaluating the safety and efficacy of the study drug LY4065967 for treating diabetic peripheral neuropathic pain DPNP. This trial is part of a larger chronic pain master protocol designed to accelerate the development of new treatments for chronic pain conditions. The study focuses on adults with DPNP related to type 1 or type 2 diabetes. Participants will be randomly assigned to receive either LY4065967 or a placebo, both taken orally. The study is double-blinded, meaning neither participants nor researchers know who receives the active drug or placebo. The treatment period lasts eight weeks, during which participants take the assigned study drug daily. Throughout the trial, participants will report their pain intensity and other symptoms at the start and after eight weeks using various scales, including the Numeric Rating Scale and Brief Pain Inventory. Researchers will also monitor sleep quality, emotional functioning, and the use of rescue medication. Safety and tolerability will be assessed, and the study concludes in July 2027.

Researchers are evaluating the progress of chronic venous leg ulcers in adults using a non-bordered foam dressing. This prospective, open, multi-center, interventional study aims to track wound healing over a 6-week period or until the wound heals. The study focuses on venous leg ulcers that produce moderate to large amounts of fluid and includes 20 participants across centers in the US and Canada. All participants will use the ALLEVYN Non-Adhesive foam dressing as the primary absorbent dressing. This device combines a breathable top film, a highly absorbent foam core, and a non-adhesive wound contact layer designed for comfort and optimal moist wound healing. The dressing will be worn continuously and changed every week during the treatment period. Participants will attend seven visits a baseline visit followed by weekly visits up to six weeks. At each visit, wound progress will be assessed by measuring wound size and evaluating wound condition. Researchers will also monitor pain, comfort, quality of life, dressing performance, and safety throughout the study. The main outcome measured is wound progress from baseline to last follow-up, with additional assessments including wound area reduction, tissue changes, exudate characteristics, skin condition, and adverse events.

Researchers are evaluating the progress of chronic wounds, specifically venous leg ulcers VLU and diabetic foot ulcers DFU, using an absorbent dressing called Mepilex Up. This study is a prospective, open, multi-center, interventional trial designed to observe wound healing over a period of up to six weeks. The goal is to track changes in wound area and condition while participants receive treatment according to local standards of care. Participants will use the Mepilex Up dressing, which is a soft, silicone-based absorbent foam dressing designed to manage wound exudate and protect the skin. The dressing will be applied as the primary dressing and changed weekly for up to six weeks or until the wound heals. The study includes seven visits a baseline visit followed by weekly visits to monitor wound healing and dressing performance. During each visit, researchers will assess wound progress through measurements and evaluations of wound area, volume, tissue quality, exudate, and skin condition. Participants will report on pain, comfort, and quality of life related to wound care. Safety will be monitored at all visits. The total treatment period is a maximum of 44 days or until healing occurs, with detailed tracking of dressing wear time, wound changes, and any adverse events.