Dallas

Researchers are evaluating 4D-710, an investigational gene therapy, in adults with cystic fibrosis (CF) lung disease who cannot use or tolerate CFTR modulator therapy. This Phase 1/2, multicenter, open-label trial also includes a sub-study assessing 4D-710 in adults with advanced CF lung disease or frequent lung flare-ups despite using CFTR modulators. The study aims to assess the safety, tolerability, and early effectiveness of this gene therapy in these populations. The trial involves a single dose of 4D-710, which is a gene therapy using a specialized virus to deliver a modified CFTR gene. Participants receive this treatment once, and those in the sub-study must be on a stable CFTR modulator regimen for at least 60 days before screening and continue it during a 24-month observation period. The study monitors participants with CF lung disease ranging from moderate to advanced stages. During the study, participants undergo regular evaluations including lung function tests, oxygen level checks, and monitoring for adverse effects. Researchers track the occurrence and severity of any side effects over a 60-month period. The study also includes assessments of lung health, medication adherence, and clinical status to understand the therapy's impact and safety over time.

Researchers are conducting a multi-center, open-label, single-arm Phase 1/2 study to evaluate the safety, radiation dosimetry, and early diagnostic performance of [18F]FPyQCP in detecting several cancers including colorectal cancer, gastric cancer, pancreatic ductal adenocarcinoma, invasive lobular breast cancer, and epithelial ovarian cancer. The study aims to better understand how this imaging agent behaves in the body and how well it identifies cancer in specific areas like the peritoneum. Participants will receive the investigational drug [18F]FPyQCP, which is used in positron emission tomography (PET)/computed tomography (CT) imaging. The study includes two cohorts: Cohort A focuses on patients with stage I-III or oligometastatic stage IV disease, while Cohort B requires recent conventional imaging including contrast-enhanced CT scans. Imaging with [18F]FPyQCP will be performed, and some participants may have scheduled biopsies or surgical resections within 42 days following imaging. Throughout the study, participants will undergo monitoring to assess radiation exposure up to 360 minutes post-injection and diagnostic performance up to 42 days post-injection. They will be evaluated through scans, laboratory tests including pregnancy testing for women of childbearing potential, and clinical assessments. Safety and image quality will be closely observed to gather reliable data on the use of [18F]FPyQCP in these cancer types.

Researchers are conducting an open-label, multi-center, non-randomized pivotal Phase 3 study to evaluate the effectiveness and safety of PET imaging using [18F]PI-2620 for detecting tau protein buildup in people with Alzheimer's disease and control subjects. The study compares PET imaging results during life with brain tissue analysis obtained after death through autopsy, aiming to improve diagnosis of tau-related brain changes. Participants will receive an intravenous injection of the radioligand [18F]PI-2620 at a dose of 185 MBq 20%. The PET imaging will be performed to visualize tau deposits in the brain. This study focuses on assessing the diagnostic accuracy of this imaging method by comparing it to post-mortem histopathology findings. Throughout the study, participants will undergo PET scans and assessments to determine the presence and extent of tau pathology. The primary outcome measure is the ability of visual assessment of [18F]PI-2620 PET images to correctly distinguish tau neurofibrillary pathology associated with Alzheimer's disease, confirmed at autopsy within about one year. Safety and tolerability during imaging procedures will also be monitored, with a total participation period depending on the timing of brain autopsy after death.

Researchers are exploring the use of a special imaging technique called [18F]PT2385 PET/CT to study patients with renal cell carcinoma (RCC), a type of kidney cancer. This open-label, non-therapeutic Phase 1 trial aims to link the levels of a protein called hypoxia-inducible factor-2alpha (HIF2b1) seen on PET/CT scans with levels found in tumor tissue samples using immunohistochemistry. The study includes three groups: patients scheduled for kidney surgery, patients with metastatic clear cell RCC, and patients with Von Hippel-Lindau (VHL) syndrome who have RCC or related tumors. Participants will receive an intravenous injection of the investigational drug [18F]PT2385 followed by PET/CT or PET/MRI scans to observe how the tracer moves and accumulates in tumors. The first group will have scans before surgery with tissue samples taken during surgery. The second group will have scans followed by mandatory biopsies of metastatic tumors. The third group will have scans, and biopsies are encouraged but not required. Some participants may have repeat scans if needed. Standard or experimental treatments for RCC will continue as decided by their doctors. Throughout the study, participants will undergo imaging sessions lasting from 25 to 60 minutes, with additional whole-body scans at later times to track tracer distribution. Researchers will collect biopsy samples when applicable and analyze them for HIF2b1 levels. They will also monitor the correlation between PET scan results and tissue findings over up to five years. Safety and effectiveness of the imaging process will be observed, with overall participation lasting as long as necessary for follow-up and additional scans.

This research aims to evaluate the effects of litifilimab (BIIB059), a monoclonal antibody, in adults with active subacute or chronic cutaneous lupus erythematosus (CLE), with or without systemic lupus erythematosus (SLE). Participants have active skin symptoms of CLE that have not improved with antimalarial therapy or had difficulties continuing that treatment. The study focuses on reducing skin disease activity using several scores including CLA-IGA-R and CLASI, while also assessing safety, immune response, and quality of life. Participants will be randomly assigned to receive either litifilimab or a placebo injection under the skin every four weeks during a 24-week double-blind period where neither participants nor researchers know which treatment is given. After this, all participants will receive litifilimab injections every four weeks for an additional 28 weeks. Those who complete the treatment may join a long-term extension study or enter a follow-up safety period lasting up to 24 weeks. Total participation may last up to 80 weeks. Throughout the study, researchers will monitor skin disease activity using the CLA-IGA-R erythema score and the CLASI-A activity score to see how many participants improve. They will also assess safety, tolerability, immune system effects, and participants' quality of life using questionnaires. These evaluations occur regularly during both treatment periods and follow-up to understand the impact of litifilimab on CLE symptoms and overall health.

Researchers are evaluating the safety, how the body processes, and effects on body weight of the investigational drug CRB-913 in participants with obesity. This Phase 1b study includes two parts: Part 1 focuses on healthy adults to measure drug levels in the blood after a single dose, while Part 2 involves obese participants to assess safety and weight effects using different doses compared to placebo. Part 2 is blinded so that participants, doctors, and the sponsor do not know who receives the drug or placebo. In Part 1, healthy adults receive a single dose of CRB-913 tablets to study how much of the drug enters the bloodstream and how long it stays. In Part 2, obese participants take one of three doses of CRB-913 or placebo once daily for 12 weeks. After treatment ends, participants are monitored for 28 days. The study includes a randomized, double-blind, placebo-controlled design for Part 2. Participants will attend study visits for drug administration, safety assessments, and blood tests to measure drug levels and effects on body weight. Researchers will monitor for side effects and adverse events from the first dose through 28 days after final dosing. The total participation time includes the 12-week treatment period plus the 28-day follow-up phase to evaluate safety and drug behavior in the body.

Researchers are evaluating the safety and effectiveness of tenapanor in adults with Chronic Idiopathic Constipation (CIC) in this 26-week phase 3 study. The study is randomized, double-blind, and placebo-controlled, involving multiple centers. It aims to compare three doses of tenapanor (5 mg, 25 mg, and 50 mg taken twice daily) against a placebo, with a focus on improving spontaneous bowel movements. Participants will first undergo a 2-week screening where their eligibility is assessed through medical history, physical exams, lab tests, ECG, and self-reported constipation symptoms using an electronic diary (eDiary). Eligible patients will then be randomly assigned to receive one of the three doses of tenapanor or placebo twice daily for 26 weeks. During this treatment period, patients will continue daily and weekly symptom reporting via the eDiary and attend regular safety visits at weeks 2, 4, 8, 12, 16, 20, and 26. After completing the 26-week treatment, patients enter a 4-week treatment-free safety follow-up period to monitor any adverse events. A final visit occurs at the end of this follow-up to assess safety. The main outcome measured is the durable complete spontaneous bowel movements response over 12 weeks. Overall, the study involves careful monitoring of symptoms, safety, and treatment effects over approximately 32 weeks.

Researchers are evaluating new treatment options for adults with locally advanced or metastatic colorectal cancer that cannot be removed by surgery and has a specific KRAS G12C gene mutation. This study compares the safety and effectiveness of adding calderasib and cetuximab, both targeted therapies, to a standard chemotherapy regimen called mFOLFOX6. The goal is to see if this combination can help patients live longer without their cancer growing or spreading compared to current treatments that may include mFOLFOX6 with or without bevacizumab. The study has two parts. It involves treatment with calderasib taken as an oral tablet, cetuximab given according to standard procedures, and mFOLFOX6 chemotherapy combining oxaliplatin, leucovorin/levofolinate calcium, and 5-fluorouracil. Some participants may receive bevacizumab or a bevacizumab biosimilar as part of the comparison. The treatments are given following approved dosing schedules. This design allows researchers to assess the safety and tolerability of these drug combinations in treating this type of colorectal cancer with the KRAS G12C mutation. Participants will be monitored for side effects, treatment tolerability, and cancer progression over a period that may last up to about 44 months. Researchers will track outcomes such as how many participants experience dose-limiting toxicities or adverse events, how many stop treatment due to side effects, and progression-free survival time. Assessments include health evaluations, laboratory tests, and imaging to observe cancer status. This long-term follow-up aims to understand both safety and effectiveness of the treatment combinations.

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 evaluating the safety and preliminary effectiveness of T3011, a treatment given directly into tumors, alone and combined with pembrolizumab, an intravenous medication, in adults with advanced or metastatic solid tumors. This Phase 1/2a open-label study focuses on several types of cancers including melanoma, head and neck squamous cell carcinoma, sarcoma, cutaneous squamous cell carcinoma, and non-small cell lung cancer. The study aims to find the best dose of T3011 and understand its safety when used alone or with pembrolizumab. The study begins with a Phase 1 dose escalation to test increasing doses of T3011 using a 3+3 design. After determining the recommended dose, Phase 2a Part 1 will evaluate T3011 alone in different cancer types across multiple arms, while Phase 2a Part 2 will assess the combination of T3011 with pembrolizumab in participants with metastatic non-small cell lung cancer. T3011 is given as an intratumoral injection up to 4mL every two weeks, and pembrolizumab is given intravenously every three weeks when combined. A rollover arm allows participants whose disease progresses on T3011 alone to receive the combination treatment. Participants will be closely monitored for safety and treatment effects for up to two years from the first dose of T3011. Assessments include tumor measurements, biopsies, laboratory tests, and evaluation of adverse effects. The study records the tolerability of escalating doses and the combination treatment, tracking disease progression and response. Participants must attend scheduled visits for injections, monitoring, and evaluations throughout the study period.