Morristown

Researchers are looking for ways to treat germinal center B-cell-like diffuse large B-cell lymphoma (GCB DLBCL). DLBCL is a fast-growing blood cancer that affects B-cells. GCB is a type of DLBCL that affects young B-cells that are still maturing. The goal of this study is to learn if more people who receive zilovertamab vedotin (MK-2140) and R-CHP have the cancer respond (go away) than those who receive polatuzumab vedotin and R-CHP.

This research focuses on participants with ovarian and breast cancers who have previously been treated with niraparib in GlaxoSmithKline/TESARO-sponsored studies. The aim is to provide continued access to niraparib and to better understand its long-term safety over an extended period. This global extension study involves adult participants who have met the primary objectives in prior clinical trials and are still benefiting from niraparib treatment. Participants will continue to take niraparib orally in the form of tablets or capsules once daily. This open-label, multicenter study allows participants who are judged by their investigators to benefit from ongoing treatment to receive niraparib beyond their initial studies. The treatment focuses on maintaining the therapy rather than comparing it to another drug or placebo. During the study, participants will be regularly monitored for safety through assessments such as adverse events, changes in performance status, blood tests, vital signs, and physical examinations. Researchers will also track the use of other medications. The study collects data for up to five years, ensuring comprehensive long-term safety information while participants adhere to scheduled visits and treatment plans.

Researchers are evaluating whether the drugs retatrutide and tirzepatide can prevent major adverse liver outcomes (MALO) in adults with metabolic dysfunction-associated steatotic liver disease (MASLD) who are at high risk. This Phase 3 trial enrolls about 4,500 adults with MASLD identified by non-invasive tests indicating an increased likelihood of developing serious liver problems. The study aims to understand how these treatments might affect liver health over time compared to a placebo. Participants will be randomly assigned to receive either retatrutide, tirzepatide, or a placebo, all given by subcutaneous injection. The study will last approximately 224 weeks, during which participants may attend 25 to 30 clinic visits for monitoring and assessment. After the main study, eligible participants can join an optional 2-year extension where all will receive either retatrutide or tirzepatide regardless of their original group. Throughout the trial, participants’ liver function and disease progression will be closely monitored through various health assessments. Researchers will track the time to the first major adverse liver event as the main outcome. Safety and health status will be evaluated regularly during clinic visits, ensuring thorough observation over the long study period.

Researchers are evaluating a phase 1/2 open-label study to investigate the safety, pharmacokinetics, pharmacodynamics, and clinical effects of an oral drug called Enzomenib (DSP-5336) in patients with acute leukemia, including relapsed or refractory acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), ambiguous lineage acute leukemia, and in certain sites, high-risk myelodysplastic syndromes (MDS) or relapsed multiple myeloma (MM). The study also examines Enzomenib combined with standard AML treatments such as venetoclax plus azacitidine and the intensive chemotherapy 7+3 regimen in patients newly diagnosed with AML who have specific genetic mutations (MLL rearrangement or NPM1 mutation). Participants receive oral Enzomenib either alone or combined with other drugs: venetoclax and azacitidine for a nonintensive treatment group, gilteritinib for a certain relapsed AML group, or intensive chemotherapy with cytarabine and daunorubicin (7+3) for newly diagnosed AML patients. The study includes dose escalation and expansion phases to determine recommended doses for phase 2. Treatment schedules and doses are adjusted based on response and safety, with some patients enrolled in specialized cohorts according to their genetic markers. Throughout the study, participants undergo regular assessments including clinical exams, laboratory tests, bone marrow samples for genetic analysis, and monitoring for adverse events. Researchers measure safety outcomes such as adverse and serious adverse events, determine optimal dosing for phase 2, and evaluate treatment effectiveness by tracking complete response rates. Safety is monitored up to 30 days after the last dose, with dose recommendations made within four months of treatment start and response assessed around six months. The total participation time varies based on individual treatment and study phase.

Researchers are evaluating the safety, tolerability, and effectiveness of LP352 in reducing seizures among children and adults diagnosed with Dravet Syndrome (DS). This phase 3, double-blind, randomized, placebo-controlled study aims to compare LP352 with a placebo to better understand its impact on seizure frequency in this population. The study involves participants aged 2 to 65 years and addresses the challenges patients with DS face due to various seizure types and treatment responses. Participants will receive either LP352 or a matching placebo, administered orally or through a feeding tube (G-tube or PEG tube). The study includes several phases: an initial screening period, followed by a titration period to adjust doses, then a maintenance period where treatment continues, and finally a taper period to gradually reduce treatment before a follow-up phase. The entire study duration is approximately 24 months. During the study, participants will be monitored for changes in the frequency of countable motor seizures compared to their baseline seizure activity over up to 15 weeks. They will be required to complete diaries throughout the study to track seizures and treatment adherence. Safety and tolerability will also be assessed throughout all study phases. The researchers will collect data on seizure counts and monitor participants' health to evaluate LP352's effects comprehensively.

This research aims to better understand the long-term safety risks of lonapegsomatropin in children with growth hormone deficiency who are treated with this medication in real-world settings. The study focuses on patients aged 1 to 18 years and is conducted in Europe and the USA. It is designed as a prospective, non-interventional, long-term safety study following the drug's approval for use. Participants continue their regular treatment with lonapegsomatropin without any additional interventions from the study. The study does not involve changes to their therapy but observes patients who are already receiving lonapegsomatropin. Eligible participants are pediatric patients clinically managed with this medication, and the study follows them over time to monitor safety outcomes. Throughout the study, researchers will track the occurrence of benign, malignant, and unspecified tumors as well as the development of type 2 diabetes mellitus over a period of five years. Participants provide informed consent and agree to follow-up requirements, which include regular monitoring and data collection to assess these outcomes. The study aims to provide important safety information about lonapegsomatropin in its post-marketing use.

Researchers are studying patients with symptomatic obstructive hypertrophic cardiomyopathy (HCM) in the United States and Europe to understand their characteristics, treatment patterns, and outcomes over time. The study focuses on individuals who are receiving mavacamten, other treatments for obstructive HCM, or no treatment due to intolerance or failure of prior therapies. The research includes a United States sub-study to evaluate mavacamten's safety and a European sub-study to assess both its effectiveness and safety in real-world settings. Participants may receive mavacamten according to its product label or other symptomatic therapies such as beta-blockers, non-dihydropyridine calcium channel blockers, or disopyramide based on standard care. The study includes those starting mavacamten, currently on other treatments, or untreated due to intolerance or failure of prior therapy. Treatment is observed during routine clinical care without altering prescribed therapy. Data collection occurs over several years to monitor long-term outcomes. During the study, participants will be regularly assessed for heart function and symptoms, including measuring the left ventricular outflow tract gradient and monitoring the incidence of new or worsening heart failure up to five years. Researchers will gather information on patient health, treatment safety, and heart function changes through echocardiography and symptom evaluations. The study allows for long-term observation to better understand real-world treatment effects and outcomes in obstructive HCM patients.

This research focuses on people with cystic fibrosis (CF) who do not take cystic fibrosis transmembrane conductance regulator (CFTR) modulators. It aims to collect health data and specimens to support CF research, help design clinical trials, and assist in developing new treatments specifically for those who cannot use CFTR modulators. The study also seeks to engage this community in research and provide information about participating in CF studies. Participants will be part of a prospective, observational study that collects monitored research data over time. The study will gather core CF outcome data aligned with clinical trial endpoints needed for therapies targeting people without CFTR modulators. Sub-studies will collect specialized measurements to inform the safety and effectiveness of new treatments. There is no investigational drug or treatment administered in this study. During the study, participants will follow a visit schedule to provide health information and biological samples. Researchers will monitor their clinical status and collect data to characterize this CF population. The main outcome measured is the change in percent predicted forced expiratory volume in one second (ppFEV1) at 12 months. The study also assesses research participation and engagement. The total study period includes visits over 12 months, with data collected to support future CF research and therapy development.

Researchers are evaluating PRL-02 depot, a potential injectable treatment for men with advanced prostate cancer whose cancer has returned after previous treatments or did not respond well. This phase 1 study aims to assess the safety, tolerability, and appropriate dosing of PRL-02 depot when given alone or with another medicine called enzalutamide. The study includes men with different types of metastatic prostate cancer, including castration-resistant and castration-sensitive forms, some of whom have previously taken specific hormone therapies. The study is conducted in two parts. In the first part, small groups of men receive increasing doses of PRL-02 depot along with other medicines like dexamethasone, prednisone, or enzalutamide depending on the group. In the second part, men who have taken hormone therapy abiraterone acetate or one other hormone therapy participate. All men receive PRL-02 depot injections into a muscle every 12 weeks and take daily oral medications as per their group assignment. Participants will visit the clinic regularly for health checks, scans, and laboratory tests to monitor safety and effectiveness. Researchers will track side effects, laboratory and heart monitoring results, performance status, and testosterone levels over up to four years. Men whose cancer does not worsen after the study will continue with periodic health assessments and scans. The total participation time varies based on individual response and study progression.

Researchers are studying KB707, a genetically modified herpes simplex type 1 virus designed to trigger an immune response against tumors. This Phase 1/2 open-label trial is focused on adults with locally advanced or metastatic solid tumors, including advanced melanoma, especially those who have progressed on or cannot tolerate standard treatments. The study also explores the safety, tolerability, preliminary effectiveness, and immune effects of KB707 when combined with immunotherapy drugs for melanoma. Participants receive KB707 through injections directly into tumors approximately every three weeks during the dose escalation and initial expansion phases, which are now closed to new participants. In later phases, those with advanced melanoma receive biweekly KB707 injections combined either with Opdualag every four weeks or Keytruda every six weeks. Treatment continues until disease progression, unacceptable side effects, worsening symptoms, maximum response, patient choice, investigator decision, or study termination. During the study, participants undergo evaluations including assessments of adverse and serious adverse events for up to 36 months. Researchers monitor safety, tolerability, immune response, and preliminary efficacy through clinical exams, imaging, and laboratory tests. The goal is to understand how KB707 alone and with immunotherapy acts in treating advanced solid tumors and melanoma over the treatment and follow-up periods.