Mineola

Researchers are investigating sacituzumab tirumotecan (MK-2870) alone or combined with other treatments to treat certain gastrointestinal cancers. These include colorectal cancer that cannot be removed by surgery or has spread, advanced pancreatic ductal adenocarcinoma, and biliary tract cancer. The study aims to understand the safety and tolerability of sacituzumab tirumotecan and measure how many participants respond to the treatment by having their cancer shrink or disappear. Participants may receive sacituzumab tirumotecan by intravenous infusion alone or with other anticancer drugs such as fluorouracil (5-FU), leucovorin or levoleucovorin, cisplatin, and pembrolizumab. Rescue medications like diphenhydramine, H2 receptor antagonists, acetaminophen, dexamethasone, and a steroid mouthwash are given to prevent infusion reactions and oral side effects. Supportive care treatments for side effects, including antidiarrheal and antiemetic agents, are allowed throughout the study. During the study, researchers monitor participants for dose-limiting toxicities within about 4 weeks and track adverse events, treatment discontinuations, and tumor response over up to approximately 63 months. Assessments include safety evaluations and measuring cancer response using standardized criteria. This long-term follow-up helps evaluate both the effectiveness and safety of the treatments being studied.

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.

Researchers are evaluating the safety and effectiveness of EscharEx (EX-03 5% formulation), a gel made from a sterile lyophilized powder containing proteolytic enzymes, compared to a placebo gel without enzymes. The study focuses on the treatment of Venous Leg Ulcers (VLU), which are wounds on the legs caused by poor venous blood flow. This Phase 3 trial will involve at least 216 adults with VLUs that range in size from 2 to 25 square centimeters and have lasted between 4 weeks and 12 months. Participants will be randomly assigned to receive either EscharEx or the placebo in a double-blinded setup, meaning neither patients nor researchers know which treatment is given. The study lasts up to 29 weeks and includes several stages: a screening period with two visits one week apart; a daily treatment phase with up to eight daily applications over two weeks to remove necrotic tissue; a weekly wound management period lasting up to 12 weeks with up to 13 visits, plus up to two weeks for confirming wound closure; and finally, a 12-week monthly follow-up with three visits to monitor wound closure durability. Throughout the study, researchers will visually assess the removal of dead tissue after each treatment application and monitor the time it takes for the wound to fully close. Patients will undergo standardized wound care and regular clinical evaluations, including measurements of the wound area. Safety and wound healing progress will be carefully tracked during all visits, ensuring comprehensive monitoring of treatment effects and wound status over the entire study duration.

Researchers are conducting a first-in-human phase 1 study to evaluate MEN2312, a lysine acetyltransferase 6 (KAT6) inhibitor, in adults with advanced breast cancer. This study focuses on participants whose cancer has recurred locally or metastasized and who have limited treatment options after prior therapies. The trial aims to assess the safety and appropriate dosing of MEN2312, alone or combined with elacestrant, an oral drug also being studied. Participants will receive MEN2312 tablets orally, either as a single treatment or alongside elacestrant tablets. The study allows participants who have undergone up to six prior systemic therapies for advanced disease, including chemotherapy or antibody drug conjugates. The study involves careful selection of participants based on genetic alterations in their tumor tissue related to PIK3CA, AKT1, or PTEN genes. Throughout the trial, researchers will monitor participants for dose-limiting toxicity over the first 28 days and determine the recommended phase 2 dose by six months. Safety assessments, treatment response, and side effects will be tracked closely. Participation requires ongoing evaluations to measure how the participant's cancer responds and to ensure safety while receiving the study treatments.

Researchers are evaluating ASTX030, a combination of azacitidine and cedazuridine, as a treatment for myeloid neoplasms including myelodysplastic syndromes (MDS), chronic myelomonocytic leukemia (CMML), and acute myeloid leukemia (AML). This multi-phase study includes Phase 1 through Phase 3 monotherapy arms and Phase 1 and Phase 2 combination therapy arms with venetoclax. The study aims to assess pharmacokinetics, safety, efficacy, and drug interactions over an approximate duration of 8 years. The study treatments involve oral administration of ASTX030 and azacitidine, with some arms including subcutaneous azacitidine for comparison. Phase 1 monotherapy includes dose escalation and expansion stages, while Phase 2 and Phase 3 monotherapy arms are randomized crossover studies comparing oral ASTX030 to subcutaneous azacitidine. The combination therapy arms explore ASTX030 combined with venetoclax in participants with treatment-nafve AML, either in an open-label randomized exploratory setting or a single-arm study. Participants undergo evaluations including pharmacokinetic measurements such as total cycle area under the curve (AUC) for drug exposure, assessment of treatment-emergent adverse events, and investigator-assessed complete response rates. Monitoring occurs at multiple timepoints up to 36 months in some study arms. Safety, efficacy, and drug interaction assessments are integral throughout the study, with follow-up periods extending up to 8 years.

Researchers are conducting a prospective observational sub-study involving participants with pediatric-onset hypophosphatasia (HPP), which includes perinatal/infantile or juvenile onset forms. The study will follow participants of any age for at least 5 years at sites in the United States and potentially one or two other countries. The focus is on describing the potential risk of immune-mediated loss of the pharmacological effect of asfotase alfa treatment in these individuals. All participants will receive asfotase alfa by subcutaneous injection according to standard care. For children under 2 years old, clinic visits are recommended approximately every 3 months until age 2, then every 6 months thereafter. Participants are expected to be followed for up to 5 years as possible. Treatment may be newly started, resumed, or ongoing at enrollment based on the physician's decision. During the study, participants will have their alkaline phosphatase (ALP) activity and ALPL gene mutation status documented if not already done. The study will monitor for immune-mediated loss of treatment effectiveness and serious immune-related adverse events over up to 5 years. Participants or their caregivers will provide informed consent and complete questionnaires in their local language. The study excludes those currently enrolled in an Alexion-sponsored interventional clinical study but allows previous trial participants who have completed their involvement to join this sub-study.

Researchers are evaluating the safety, tolerability, and effect size of STIMULAN VG combined with debridement and systemic antibiotics compared to standard care involving debridement and systemic antibiotics alone for treating osteomyelitis associated with stage IV pressure ulcers. This is a phase II, open-label, multi-center, randomized controlled trial involving patients diagnosed with stage IV pressure ulcers. Participants are randomly assigned in a 1:1 ratio to either receive STIMULAN VG inserted into the ulcer cavity before flap or primary closure surgery along with systemic antibiotics or to receive the standard care which includes ulcer bursectomy, debridement, flap or primary closure, and peri-operative antibiotics. The trial focuses on comparing these two treatment methods for their safety and effectiveness in managing the condition. During the study, patients will be closely monitored and evaluated, with the main outcome being individual patient success assessed at an 8-week follow-up visit. The study includes assessments such as imaging to confirm osteomyelitis, treatment adherence, and safety monitoring. Participants must be adults and able to comply with all study procedures, including scheduled visits and treatment plans.

This research aims to evaluate the safety and effectiveness of BMS-986504, a selective PRMT5 inhibitor, when combined with Nab-paclitaxel and Gemcitabine, compared to a placebo combined with Nab-paclitaxel and Gemcitabine. The study focuses on participants with untreated metastatic Pancreatic Ductal Adenocarcinoma (PDAC) who have a specific genetic alteration called homozygous MTAP deletion. This is a randomized Phase 2/3 trial designed to explore treatment options for this patient population. Participants will be assigned to receive either BMS-986504 at specified doses on certain days along with Nab-paclitaxel and Gemcitabine, or a placebo with the same chemotherapy drugs. The treatments are given according to protocol schedules. Some participants may have received up to one cycle of Nab-paclitaxel and Gemcitabine before starting the study treatment, provided they did not experience disease progression or intolerable side effects. The initial cycle must be completed before randomization. During the study, researchers will monitor participants for progression-free survival and overall survival for up to three years after the last participant is randomized. Assessments include measuring tumor response using established criteria (RECIST v1.1). Participants will undergo evaluations to track safety, treatment effects, and disease status throughout the trial period.

Researchers are evaluating the safety of atezolizumab alone or combined with bevacizumab as first-line treatment in adults with unresectable, locally advanced, or metastatic hepatocellular carcinoma (HCC) who have Child-Pugh B7 or B8 liver cirrhosis. This Phase II, open-label study involves participants who have not received prior systemic therapy for their condition and aims to understand treatment safety in this specific population. Participants will be assigned to one of two groups: one receiving atezolizumab plus bevacizumab, and the other receiving atezolizumab alone. Atezolizumab is given as an intravenous infusion at a dose of 1200 mg on the first day of each 21-day cycle. Bevacizumab is administered by intravenous infusion at 15 mg/kg on the same schedule. The study is conducted across multiple centers and includes multiple cohorts to evaluate these treatments separately. During the study, researchers will monitor participants for adverse events from the start until up to approximately 36 months. Assessments include measuring disease status with imaging and clinical evaluations, checking blood tests for liver and kidney function, and monitoring overall health and safety. Participants will be followed closely throughout the treatment and observation periods to gather safety data and evaluate treatment tolerability.

Researchers are evaluating the safety, how the body processes the drug (pharmacokinetics), and the activity of divarasib alone or combined with other anti-cancer treatments in adults with advanced or metastatic non-small cell lung cancer (NSCLC) who have not received previous treatment. This study focuses on patients with a specific KRAS G12C mutation and includes both Phase Ib and II stages to understand the drug effects better. Participants will receive divarasib orally once daily on days 1 to 21 during each 21-day treatment cycle. Depending on the treatment group, participants may also receive pembrolizumab, an intravenous infusion given every three weeks, and chemotherapy drugs such as carboplatin or cisplatin administered intravenously every three weeks for four cycles. Pemetrexed, another chemotherapy drug, may be given intravenously every three weeks as well. These treatments are designed to be studied either alone or in combination. Throughout the study, participants will be monitored for any adverse events from the start of treatment until 60 days after the last dose or until they begin another anti-cancer therapy, up to about five years. Researchers will conduct evaluations including tumor measurements, safety assessments, and pharmacokinetic sampling. The study requires participants to have measurable disease and good performance status. Close monitoring will ensure participant safety and collect data to understand the drug’s activity and side effects over time.