Morgantown

Researchers are investigating the safety and tolerability of delivering gemcitabine directly to the pancreas using the ACT-IOP-003 implantable iontophoresis chemotherapy delivery device in patients with nonmetastatic, locally advanced, nonresectable pancreatic adenocarcinoma. This Phase 1b study aims to assess whether this method is safe, how much gemcitabine is present in the blood before and after treatment, the tumor's response to the treatment, and whether side effects are reduced compared to traditional intravenous delivery. Participants will have the ACT-IOP-003 device surgically implanted on the pancreas and receive gemcitabine either once or twice weekly. The study includes a 4-week screening period, 8 weeks of treatment, and 12 weeks of follow-up, totaling 24 weeks of participation. Gemcitabine is delivered directly to the pancreas rather than through the bloodstream, potentially reducing systemic side effects. During the study, participants will provide blood, urine, and stool samples to monitor safety and measure gemcitabine levels. They will also undergo CT scans at least three times to evaluate tumor response. Safety, tolerability, and maximum tolerated delivered dose are assessed by tracking adverse events from screening through week 20. The study monitors participants closely throughout all phases to gather comprehensive data on treatment effects and safety.

Researchers are evaluating the efficacy and safety of rilvegostomig compared to pembrolizumab as first-line treatments for patients with metastatic non-small cell lung cancer (mNSCLC) whose tumors have high PD-L1 expression. This Phase III, randomized, double-blind, and global study focuses on participants with stage IV mNSCLC who do not have certain genetic mutations or rearrangements and are eligible for systemic therapy. Participants receive either rilvegostomig or pembrolizumab intravenously on Day 1 of each 21-day cycle. The study compares these two biological treatments given as monotherapy. Both groups will be monitored over time to assess treatment impact and safety. Throughout the study, participants undergo evaluations including tumor measurements by CT or MRI, performance status assessments, and organ function tests. Researchers will measure overall survival and progression-free survival for up to approximately five years. Tumor samples are collected before treatment for central testing, and participants’ health and treatment responses are closely followed during the trial period.

This research aims to collect data on women with hypoparathyroidism who have been exposed to palopegteriparatide during pregnancy or breastfeeding. The study evaluates potential risks related to pregnancy and maternal health, as well as any adverse effects on the developing fetus, newborn, and infant. The purpose is to understand these outcomes through a global pregnancy registry. Participants include women aged 15 to 50 years who have received at least one dose of YORVIPATH within 15 days before conception or during pregnancy. The drug is prescribed as part of normal clinical practice, and exposure timing is calculated based on the medication's half-life. The study collects both prospective and retrospective data from these participants. During the study, researchers monitor various outcomes over approximately 21 months, including the number of fetuses, pregnancy results, congenital malformations, adverse events, hospitalizations, growth and developmental milestones, signs of calcium imbalance, infant developmental deficiencies, failure to thrive, neonatal and infant mortality, and maternal complications. Participants provide consent and medical information, with adolescent participants also involving parent or guardian consent. The study carefully tracks maternal and infant health throughout this period.

This research aims to collect long-term safety and effectiveness data for participants treated with ibrutinib, a medicine used for various blood cancers and conditions including Chronic Lymphocytic Leukemia, Small Lymphocytic Lymphoma, Mantle Cell Lymphoma, Follicular Lymphoma, Diffuse Large B-cell Lymphoma, Waldenstrom Macroglobulinemia, and Chronic Graft Versus Host Disease. It also provides ongoing access to ibrutinib for participants who have completed previous ibrutinib studies, continue treatment, and benefit from it. This is an open-label Phase 3b study without formal hypothesis testing. Participants will continue their current ibrutinib dosing regimen from the prior study, taken orally once daily as capsules in doses of 560 mg, 420 mg, 280 mg, or 140 mg, around the same time each day. Treatment continues until the investigator decides the participant no longer benefits due to disease progression or side effects, the participant withdraws, alternative ibrutinib access becomes available, or the study ends. Participants not able to access ibrutinib elsewhere can keep receiving the single-agent ibrutinib until all transition or stop treatment, or until the study is stopped. During the study, safety is monitored throughout and summarized, and effectiveness may be analyzed together with previous study data. The main outcome measured is the number of participants experiencing any adverse events within 30 days after the last dose or until starting another cancer treatment. Participants will undergo assessments including pregnancy testing and investigator evaluations to ensure ongoing benefit and safety. The study duration depends on when participants stop treatment or transition to other access.

Researchers are investigating the Mechanical Tissue Resuscitation (MTR) therapy system in a multi-center, early feasibility study to evaluate its use for removing excess fluid in patients who have had part of their skull removed, exposing the dura or brain, and require fluid drainage. The study includes adult men and women aged 22 to 65 who have undergone craniotomy, craniectomy, or cranioplasty procedures, and it aims to assess safety and effectiveness within 30 days. Participants will receive MTR therapy, which is designed for short-term use of up to seven days to externally drain excess fluid from the surgical site. The study is divided into phases to enroll patients based on their condition: phase 1 includes elective procedure patients; phase 2 involves patients with mild-to-moderate traumatic brain injury (TBI) with a Glasgow Coma Scale (GCS) score of 9 or above and two reactive pupils; and phase 3 includes patients with severe TBI with a GCS of 7 or 8 and two reactive pupils. Treatment involves placement of the MTR device in patients who require a Jackson Pratt drain or equivalent. During the study, participants will be closely monitored during the treatment period of up to seven days and followed up approximately one month after treatment to evaluate safety and effectiveness. Assessments include clinical evaluations related to fluid drainage and patient outcomes. The total participation involves initial treatment and a follow-up evaluation about 30 days later to observe results and any potential risks associated with the therapy.

This research involves both pediatric and adult patients with various blood-related cancers and other disorders affecting the blood and immune system. It focuses on using unlicensed cryopreserved cord blood units (CBUs) for transplantation, aiming to study how well these unlicensed CBUs support recovery after transplant. The study also looks at important outcomes such as infection transmission, infusion reactions, survival rates, and graft-versus-host disease. Participants will receive transplants using these unlicensed cord blood units as part of a multicenter access and distribution protocol. The study is conducted at multiple U.S. transplant centers under the care of transplant physicians. The transplantation process involves administering these CBUs to patients with hematologic malignancies and other relevant conditions. Patients will be monitored for neutrophil recovery at 60 and 100 days post-transplant to assess engraftment success. Researchers will also evaluate infection rates, serious infusion reactions, survival one year after transplant, and incidences of acute and chronic graft-versus-host disease. Platelet recovery will be tracked as well. The study involves regular assessments to follow patients’ health and transplant outcomes over time.

Researchers are evaluating the efficacy and safety of benralizumab, given as a subcutaneous injection, in children and adolescents aged 6 to under 18 years who have severe eosinophilic asthma. These patients have a history of asthma exacerbations and uncontrolled symptoms despite treatment with high-dose inhaled corticosteroids plus at least one other controller medication. This Phase III study aims to compare benralizumab to placebo in reducing the time to the first asthma exacerbation. The study includes a screening period lasting from 4 to 12 weeks to confirm eligibility. After screening, patients are randomly assigned in a 1:1 ratio to receive either benralizumab or placebo via subcutaneous injections during a double-blind treatment period lasting a minimum of 16 weeks. This period continues until the patient experiences an asthma exacerbation or a set number of events occur. Patients who exacerbate can enter an open-label extension where all receive benralizumab for at least 48 weeks. An end-of-treatment visit occurs 8 weeks after the last dose in the extension phase. Participants will be monitored through visits and assessments including confirmation of severe eosinophilic asthma, asthma control questionnaires, and symptom diaries. Researchers will measure the time to first asthma exacerbation as the primary outcome. Medication adherence is tracked during screening, and safety is monitored throughout both the double-blind and extension periods. Total participation may span over a year, considering screening, treatment, extension, and follow-up visits.

Researchers are evaluating the efficacy and safety of HBS-301 in treating symptoms of idiopathic hypersomnia (IH), including excessive daytime sleepiness, sleep inertia, and fatigue. This Phase 3, multicenter, randomized, double-blind, placebo-controlled study focuses on adults aged 18 years and older diagnosed with IH. The study aims to compare HBS-301 to a placebo in relieving these symptoms and to assess its overall safety profile. Participants will be randomly assigned to receive either HBS-301 tablets or matching placebo tablets during an 8-week double-blind treatment period. Before this, there is a screening and baseline period lasting up to 28 days. Following the double-blind phase, participants have the option to join a one-year open-label extension period where they may receive HBS-301. After completing treatment, a 30-day safety follow-up will monitor participants for any adverse effects. Throughout the study, participants will undergo various assessments including sleep studies and symptom evaluations to measure the effectiveness of HBS-301. Researchers will track changes in idiopathic hypersomnia symptoms from baseline through the end of the double-blind period. Safety will also be closely monitored during treatment and follow-up periods to ensure participant well-being over the course of the study, which may last over a year for those in the extension phase.

Researchers are evaluating the effectiveness of pemigatinib in adults with advanced or metastatic pancreatic cancer that has spread to nearby tissues, lymph nodes, or distant body parts, and that have specific genetic changes in the FGFR gene. The study focuses on patients whose cancer has FGFR2 gene fusions or other FGFR alterations, aiming to see if pemigatinib can block these abnormal gene functions to stop tumor growth and possibly improve quality of life. This is a phase II trial conducted nationwide using a fully decentralized telemedicine approach to reach participants. Participants receive pemigatinib as an oral medication once daily for 14 days within each 21-day cycle. Treatment continues unless the disease progresses or unacceptable side effects occur. Alongside the drug treatment, patients undergo various imaging tests including CT scans, MRI, optical coherence tomography (OCT), and when needed, whole body bone scans and dilated eye exams (ophthalmoscopy). After finishing treatment, patients are followed up at 30 days and then every four months for one year to monitor their condition. Throughout the study, patients provide blood samples and undergo scans to evaluate treatment response and detect resistance mutations. Researchers track the overall response rate for up to 24 months and assess safety and tolerability. Patients must comply with scheduled visits, tests, and oral medication intake. The total study participation includes treatment cycles and a follow-up period lasting up to approximately 16 months after treatment completion.

Researchers are evaluating AZD0780, an oral PCSK9 inhibitor, in a phase 3, randomized, placebo-controlled study to see if it can reduce the risk of major adverse cardiovascular events (MACE-PLUS) in adults with established atherosclerotic cardiovascular disease (ASCVD) or those at high risk for a first ASCVD event. The study compares AZD0780 to a placebo and monitors participants from randomization until the primary analysis censoring date, followed by a final study closure visit. Participants will be randomly assigned to receive either oral AZD0780 or an oral placebo once daily. The treatment period lasts until the primary analysis censoring date, after which a study closure visit will occur. The study is event-driven and designed to assess the time to the first major cardiovascular event during treatment. During the study, participants will be closely monitored with various assessments to evaluate cardiovascular outcomes and safety over approximately 54 months. Researchers will track the time to first event of any component of MACE-PLUS and collect data to assess the effect of AZD0780 compared to placebo. The study includes regular visits and evaluations to ensure participant safety and adherence to treatment.