Morristown

Researchers are evaluating treatments for germinal center B-cell-like diffuse large B-cell lymphoma GCB DLBCL, a fast-growing blood cancer affecting immature B-cells. The study compares two treatment combinations to see if more people respond to zilovertamab vedotin MK-2140 plus R-CHP versus polatuzumab vedotin plus R-CHP. This Phase 2 trial aims to assess the effectiveness and safety of these regimens in participants with newly diagnosed GCB DLBCL. Participants receive either zilovertamab vedotin along with rituximab, cyclophosphamide, doxorubicin, and prednisone R-CHP, or polatuzumab vedotin combined with R-CHP. Treatments are given by intravenous infusion on Day 1 of each 3-week cycle for up to 6 cycles, approximately 4 months, with prednisone or prednisolone taken orally for 5 days of each cycle. For participants with high-risk DLBCL, up to 2 additional cycles of rituximab or biosimilar are given. During the study, participants are monitored for response to treatment using Lugano Response Criteria, with follow-up lasting up to about 31 months for the primary outcome. Secondary outcomes include progression-free survival, overall survival, event-free survival, duration of complete response, adverse events, and quality of life assessments. Safety and health status are regularly checked through exams, lab tests, and questionnaires over several years, with total study participation extending up to 7 years.

Primary immune thrombocytopenia ITP is a condition where the immune system mistakenly destroys platelets, leading to a lower number of platelets and increased risk of bruising or bleeding. This Phase 3 study evaluates the long-term safety, tolerability, and effectiveness of mezagitamab in adults with chronic primary ITP. The study also investigates how the body processes mezagitamab over an extended period. Participants who completed previous mezagitamab studies TAK-079-3002 or TAK-079-1004 will be invited to join this continuation trial. Eligible participants may receive mezagitamab injections on demand, with treatment courses repeated as needed based on specific criteria and the investigators clinical judgment. The treatment is administered subcutaneously. During the study, participants will visit the clinic several times for assessments. Researchers will monitor safety by tracking treatment-emergent adverse events, and evaluate effectiveness through platelet response and remission rates. Measurements of drug levels and antibodies will also be taken. The study may last up to approximately 108 weeks, allowing detailed long-term follow-up.

Researchers are studying the long-term safety of vedolizumab given as a subcutaneous injection to children and teenagers aged 2 to 17 years with moderate to severe active ulcerative colitis UC or Crohns disease CD. The study aims to understand medical problems that may arise from extended use of vedolizumab SC, as well as its impact on hospital visits due to bowel inflammation and on the quality of life for these young participants. This Phase 3b extension study follows participants from an earlier study VedolizumabSC-3003 who have responded well or not to treatment. Participants who responded well to vedolizumab SC in the parent study will continue treatment in this extension study, receiving the same dose and frequency. They will be randomly assigned to receive vedolizumab 108 mg either via a prefilled syringe with an autoinjector pen or with a needle safety device. Dosage is every two weeks for participants weighing at least 30 kg and every four weeks for those weighing between 10 and under 30 kg. Those who did not respond well or recently used corticosteroids will not receive vedolizumab in this study but will be observed in an observational cohort. Throughout the study, participants will visit their study clinic multiple times over up to two years. Researchers will monitor adverse events and serious adverse events up to 18 weeks after the last dose, as well as special safety events in the observational group. They will also assess time to major inflammatory bowel disease-related events and changes in quality of life using the IMPACT-III questionnaire at regular intervals. Participants will have a safety follow-up visit after treatment ends, and those in the observational group will be followed for about two years after their last dose in the parent study.

This research aims to provide continued access to niraparib and to further assess its long-term safety in participants with ovarian or breast neoplasms who are currently receiving niraparib treatment within previous GlaxoSmithKlineTESARO-sponsored studies. It focuses on participants who have completed earlier studies where the primary objectives were met and are judged by their doctors to still benefit from niraparib. The study is a phase 2, open-label extension trial designed to monitor treatment continuation and safety over time. Participants will receive niraparib once daily by mouth, following the same dose and schedule as in their previous parent study. Treatment is organized in 90-day cycles and will continue until disease progression, unacceptable side effects, new anticancer therapy unrelated to the parent study, withdrawal, or other reasons for discontinuation occur. The dosing regimen mirrors what was assigned in the prior study, ensuring consistency for each participant. During the study, participants will have regular evaluations to monitor safety and health status for up to five years. These assessments include tracking adverse events, serious side effects, and specific safety concerns, as well as monitoring physical exams, vital signs, blood tests, and medication use. Participants must comply with scheduled visits and treatments while using effective contraception if of childbearing potential. The study duration and follow-up are designed to gather detailed long-term safety information on niraparib treatment.

Researchers are evaluating whether retatrutide and tirzepatide can prevent major adverse liver outcomes in adults with metabolic dysfunction-associated steatotic liver disease MASLD who are at high risk based on non-invasive tests. This Phase 3 randomized controlled trial aims to assess these treatments compared to placebo in about 4,500 adults over approximately 224 weeks. The study is sponsored by Eli Lilly and Company and focuses on liver disease progression and related health measures. Participants will be randomly assigned to receive retatrutide, tirzepatide, or placebo, all administered by subcutaneous injection. The trial includes two placebo groups corresponding to each experimental drug. After completing the main study, eligible participants may join a 2-year extension where all will receive either retatrutide or tirzepatide regardless of their initial assignment. During the study, participants may attend around 25 to 30 clinic visits for health monitoring, study procedures, and assessments of liver function and disease status. Researchers will measure the time to major adverse liver outcomes, changes in liver fibrosis scores, liver stiffness, liver fat content, liver enzyme levels, body weight, and cardiovascular events. Monitoring will continue from baseline through study completion, with detailed evaluations at multiple timepoints including week 104.

Researchers are evaluating Enzomenib DSP-5336, an oral drug, in patients with various types of acute leukemia, including relapsed or refractory acute myeloid leukemia AML, acute lymphocytic leukemia ALL, and acute leukemia of ambiguous lineage. The study also includes patients with high-risk myelodysplastic syndromes MDS and relapsed multiple myeloma MM in selected sites. This phase 12 trial aims to assess the safety, pharmacokinetics, pharmacodynamics, and clinical activity of DSP-5336 alone or combined with standard AML treatments, particularly in patients with specific genetic mutations like MLL rearrangement or NPM1 mutation. The study involves dose escalation and dose expansion of DSP-5336 administered orally. Participants may receive DSP-5336 alone or combined with standard AML regimens such as venetoclax plus azacitidine or intensive chemotherapy with cytarabine and daunorubicin 73. Different study arms include patients with or without certain medications like CYP3A4 inhibitor azoles, and those with specific genetic profiles. The trial evaluates recommended phase 2 doses for various patient groups and combination treatments. Participants will undergo assessments including monitoring for adverse events within 30 days after the last dose and evaluation of clinical responses approximately six months after treatment begins. Researchers will collect blood and bone marrow samples for genomic analysis and track drug levels in the body. Safety labs, ECGs, physical exams, and patient questionnaires will be performed throughout the study. The trial includes long-term follow-up of overall survival up to two years after treatment ends, with visits and tests scheduled to monitor health and treatment effects.

Researchers are evaluating the drug LP352 in a phase 3, randomized, double-blind, placebo-controlled trial to study its effects on seizures in children and adults with Dravet Syndrome DS. This serious condition involves various seizure types with onset between 1 and 20 months of age. The study aims to test the efficacy, safety, and tolerability of LP352 compared to placebo over a total duration of about 24 months. Participants will be randomly assigned to receive either LP352 or a matching placebo. LP352 or placebo will be given orally or through a feeding tube. The study includes three main phases a Screening phase, a Titration period where doses are gradually increased to the highest tolerated level, and a Maintenance period to assess ongoing treatment effects. Afterward, participants will undergo a Taper period to reduce dosing and a Follow-Up phase for observation. During the study, participants will be monitored for seizure frequency changes, safety, and tolerability. Researchers will track countable motor seizures and measure percent change compared to baseline over up to 15 weeks. Participants or caregivers will complete seizure diaries, and stable antiseizure medication use is required. Safety evaluations will continue up to 21 weeks, with study visits scheduled throughout these phases. Total participation lasts approximately two years.

Researchers are evaluating the long-term safety of lonapegsomatropin in children with growth hormone deficiency who are already being treated with this medication. This study is observational and aims to gather real-world safety data over time after the drug has been authorized for use. The focus is on monitoring potential risks such as the development of tumors and type 2 diabetes over a period of five years. Participants receive lonapegsomatropin, administered once weekly by subcutaneous injection as part of their usual care. The study does not involve additional interventions but observes patients who are already treated with this therapy. The observational period extends for at least five years to collect safety data and compare it with historical information from previous studies. During the study, researchers will monitor for the occurrence of benign and malignant tumors, type 2 diabetes, and other adverse events affecting the kidneys, liver, immune system, and nervous system. They will also track medication errors and measure the response of Insulin-like Growth Factor-1 IGF-1 to treatment. Participants are followed up regularly to collect this information, with the study lasting until March 2033.

Researchers are studying real-world patient characteristics, treatment patterns, and both short- and long-term outcomes in people with symptomatic obstructive hypertrophic cardiomyopathy HCM across the United States and Europe. The study focuses on patients receiving mavacamten, other treatments for obstructive HCM, or no treatment due to intolerance or prior treatment failure. The U.S. portion evaluates the safety of mavacamten in this setting, while the European part assesses both its effectiveness and safety. Participants receive treatments as part of standard care, either mavacamten or other medications such as beta-blockers, non-dihydropyridine calcium channel blockers, or disopyramide. Treatments are prescribed by physicians according to routine clinical management. The study observes outcomes over time without altering prescribed care. During the study, participants are monitored for changes in heart function, symptoms, and adverse events through clinical assessments including echocardiography and patient-reported questionnaires. Researchers evaluate heart failure events, heart function measures like left ventricular outflow tract gradient and ejection fraction, arrhythmias, hospitalizations, mortality, and quality of life scores. Data is collected at baseline and followed for up to five years to understand real-world treatment effects and safety.

This research aims to advance treatments for people with cystic fibrosis CF who do not take cystic fibrosis transmembrane conductance regulator CFTR modulators. It focuses on those genetically ineligible or not using these modulators, a group with different health challenges compared to most people with CF. The study seeks to collect detailed health data and specimens to support new therapy development and improve clinical trial designs for this underserved population. The study is observational and will follow participants over time, collecting research-quality CF outcome data aligned with clinical trial endpoints important for developing new therapies. Sub-studies will gather specialized measures to help evaluate safety and effectiveness of future treatments. Researchers also aim to understand research participation and engagement in this community. Participants will provide health information and samples during scheduled visits over at least 12 months. Researchers will monitor lung function using measures like ppFEV1, CFQ-R-RD scores, lung clearance index LCI, and mucociliary clearance MCC indices. The study will help characterize this CF group and support the development of innovative trials. It will also assess research involvement and provide data for comparison with new therapies.