La Laguna

Researchers are conducting a master protocol trial designed to efficiently study several different drugs for children and young adults with various types of cancer. This approach allows multiple clinical trials under one common research plan, with each trial focusing on specific cancers like desmoplastic small round cell tumor, synovial sarcoma, and Ewings sarcoma. New drug studies may be added over time as new treatments emerge. Participation depends on how long the treatment benefits last. The study evaluates combinations of drugs given in cycles, including intravenous and oral medicines such as ramucirumab, cyclophosphamide, vinorelbine, gemcitabine, docetaxel, abemaciclib, irinotecan, and temozolomide. Different treatment groups receive specific drug combinations tailored to the cancer type, with treatment cycles lasting 21 or 28 days depending on the regimen. Each group is randomized to receive either an experimental drug combination or an active comparator without any blinding. Participants will be involved from screening through treatment and monitored regularly. Researchers will assess how many participants are assigned to each specific intervention plan within the first four weeks and track treatment effects. Participants must have measurable or evaluable disease, adequate organ function, and meet performance status criteria. Female participants of childbearing potential undergo pregnancy testing and must use contraception during and after treatment. Safety, adherence, and side effects are carefully monitored throughout the study, which is expected to continue until 2027.

This research aims to compare two different weekly doses of Alpha1-Proteinase Inhibitor given by injection under the skin with the standard doses given through a vein in adults with Alpha-1 Antitrypsin Deficiency. The trial focuses on understanding how the body processes the medicine and evaluates how safe and tolerable the different weekly doses are. Both study doctors and participants know which treatment is being given during this open-label study. Participants will be randomly assigned to one of two treatment groups. One group receives 8 weeks of intravenous treatment with 60 mgkgweek Liquid Alpha1-PI followed by 8 weeks of subcutaneous treatment with 90 mgkgweek Alpha-1 15%. The other group receives 8 weeks of intravenous treatment with 120 mgkgweek Liquid Alpha1-PI followed by 8 weeks of subcutaneous treatment with 180 mgkgweek Alpha-1 15%. The study uses a parallel design to evaluate these dosing regimens. During the study, participants will undergo assessments to measure the steady-state exposure of the medicine over weekly dosing intervals and trough levels at specified weeks. Safety and tolerability will be monitored closely throughout. The study lasts about 16 weeks of active treatment, with visits scheduled to collect blood samples and monitor health status. The main outcome is the measurement of the medicines activity over time to understand how different doses are processed by the body.

Researchers are studying the impact and burden of three skin conditions moderate or severe alopecia areata, non-segmental vitiligo, and moderate to severe hidradenitis suppurativa. The study includes adolescents and adults and aims to understand how these conditions affect quality of life and daily functioning in a large global population. This is an observational study where participants with each condition will have a single visit for data collection following routine clinical practice. No experimental treatments are given instead, the study gathers information during this one visit to assess disease characteristics and impact. During the visit, participants will complete questionnaires and clinical assessments specific to their condition. These include tools measuring symptom impact, hair loss severity, skin depigmentation, and quality of life related to each disease. This helps researchers better understand the real-world burden of these conditions. Participation involves only this one visit, with no long-term follow-up or additional procedures.

This research aims to evaluate the effectiveness and safety of zanidatamab combined with a physicians choice of chemotherapy compared to trastuzumab combined with chemotherapy in treating adults with metastatic HER2-positive breast cancer who have either progressed on or cannot tolerate previous trastuzumab deruxtecan T-DXd treatment. Zanidatamab has shown promising results against various HER2-positive advanced tumors, including metastatic breast cancer, and may serve as a potential treatment option for these patients. The study also investigates patient-reported tolerability and physical functioning, as well as the pharmacokinetics and immune response to zanidatamab with chemotherapy. Participants will be randomly assigned to receive either zanidatamab or trastuzumab, each given by intravenous infusion alongside one of several chemotherapy options chosen by the physician eribulin, vinorelbine, gemcitabine, or capecitabine the latter is taken orally. Treatment will be administered according to the assigned group, and the study is open-label and multicenter, designed to compare these two treatment combinations in this patient population. During the study, participants will undergo regular assessments to monitor disease progression using imaging criteria RECIST version 1.1, evaluate survival, treatment response, and duration of response. Safety and side effects will be tracked through adverse event reporting and patient questionnaires on symptoms and physical function. Blood samples will be collected to study drug levels and immune reactions. Participants will be followed until disease progression, death, or for up to approximately 44 months for key outcomes, with overall survival monitored for up to about 80 months.

Researchers are evaluating elacestrant compared to standard endocrine therapies in adults with node-positive, Estrogen Receptor-positive ER, HER2-negative early breast cancer who are at high risk of cancer returning. The study focuses on those who have had prior endocrine therapy and aims to measure how well elacestrant may prevent invasive breast cancer recurrence over five years. Participants are randomly assigned to receive either 345 mg of elacestrant daily for five years or continue their prior standard endocrine therapy, which may include an aromatase inhibitor anastrozole, letrozole, or exemestane or tamoxifen. The trial is open-label, meaning both participants and researchers know which treatment is given. During the study, participants will have regular assessments to monitor cancer recurrence, survival, side effects, and quality of life. Evaluations include questionnaires on health status and physical functioning at baseline, six months, and annually for up to five years. Safety is tracked through adverse event reporting up to five years plus 28 days. The total participation duration can last up to five years with ongoing monitoring and data collection.

Researchers are evaluating nemtabrutinib compared with investigators choice of ibrutinib or acalabrutinib in adults with untreated chronic lymphocytic leukemia CLL or small lymphocytic lymphoma SLL. The study aims to assess whether nemtabrutinib is not worse than these comparators in terms of objective response rate and whether it can provide longer progression-free survival. This is a Phase 3 randomized clinical trial sponsored by Merck Sharp & Dohme LLC. Participants will receive either nemtabrutinib, ibrutinib, or acalabrutinib orally at specified doses until their disease progresses, unacceptable side effects occur, or other discontinuation criteria are met. The trial uses a parallel-group design where participants are randomly assigned to one of the treatment groups, and no masking is involved. Both treatment arms continue until progression or intolerance. During the study, participants will be monitored regularly up to about 33 months for response rate and up to about 104 months for progression-free survival and overall survival. Assessments include clinical evaluations, safety monitoring for adverse events, and duration of response measurements. The study tracks treatment tolerability, discontinuations due to adverse events, and overall outcomes to better understand the therapies effects in this patient population.

Researchers are evaluating the addition of Tersolisib LY4064809STX-478 to other anti-cancer drugs as a first treatment for adults with advanced hormone receptor-positive HRhuman epidermal growth factor receptor 2-negative HER2- breast cancer that has a PIK3CA mutation. This Phase 3 randomized, double-blind, placebo-controlled trial aims to understand the efficacy and safety of this combination compared to placebo, focusing on improving outcomes for patients with this specific genetic change. Participants receive LY4064809 orally in one of two doses combined with a CDK46 inhibitor such as Ribociclib, Palbociclib, or Abemaciclib and endocrine therapy ET administered orally or via intramuscular injection. The comparison group receives a placebo combined with the same CDK46 inhibitor and ET. The study includes two parts Part 1 explores dose optimization, and Part 2 evaluates the treatment combinations effectiveness and safety as a first-line therapy. During the study, participants will have regular assessments to monitor cancer response, progression, and safety over an estimated period of up to 5 years or more. Researchers will measure outcomes such as overall response rate, progression-free survival, duration of response, overall survival, and quality of life. Treatment continues as long as the cancer benefits without intolerable side effects. Safety monitoring, laboratory tests, and quality of life questionnaires are part of the participant involvement throughout the trial.

Researchers are evaluating whether combining tucatinib with trastuzumab and mFOLFOX6 works better than standard treatments for people with HER2 positive colorectal cancer that has spread or cannot be removed by surgery. This Phase 3 study also aims to learn about the side effects that may occur when taking this combination of drugs. Participants have metastatic or unresectable colorectal cancer and are randomly assigned to different treatment groups. Participants are randomly placed in one of two study groups. One group receives tucatinib taken orally twice daily along with trastuzumab given intravenously every 3 weeks and mFOLFOX6 chemotherapy every 2 weeks. The other group receives standard care, which may be mFOLFOX6 alone or combined with bevacizumab or cetuximab, both given intravenously on different schedules. Tissue samples and biopsies are collected before treatment to confirm HER2 positivity and other markers. During the study, participants will have regular evaluations including imaging scans to measure cancer progression, blood tests, and assessments of side effects and quality of life. Progression-free survival is the primary outcome measured for up to about 3 years, with other outcomes like overall survival and response rate also tracked. Safety monitoring continues for about one year after the last treatment. The study lasts several years, with ongoing follow-up to understand long-term effects and benefits.

Researchers are conducting a multicenter, randomized, double-blind, placebo-controlled Phase 3 study to evaluate the effects of teplizumab compared with placebo in children and young adults aged 1 to 25 years with recently diagnosed Stage 3 type 1 diabetes. The study aims to measure changes in blood sugar control and independence from mealtime insulin over 52 weeks while participants continue standard insulin therapy. Participants will be randomly assigned to receive either increasing doses of teplizumab or a matching placebo, both given by intravenous IV infusion. The treatment period lasts 52 weeks, during which participants receive the study drug or placebo by IV infusion. The total study duration for each participant is approximately 84 weeks 19 months, including treatment and follow-up. Throughout the study, participants will undergo regular assessments including blood tests measuring glycated hemoglobin HbA1c, C-peptide levels from meal tolerance tests, and continuous glucose monitoring to track blood sugar levels. Researchers will also monitor insulin use, adverse events, drug concentrations in the blood, and antibody responses. Safety and efficacy are evaluated up to 78 weeks, with multiple visits scheduled across the study period.

Researchers are evaluating how the study medicine PF-06823859 dazukibart works in adults with idiopathic inflammatory myopathies, specifically dermatomyositis DM and polymyositis PM. These conditions cause muscle inflammation leading to weakness and may include a skin rash in DM. The study aims to assess the safety and effects of dazukibart compared to a placebo in people receiving stable doses of corticosteroids or immunosuppressants. Participants will receive either the study medicine or a placebo through an intravenous infusion lasting about one hour. These infusions occur every four weeks from Day 1 through Week 48 at the study site. The study is randomized and double-blind, meaning neither participants nor researchers know who gets the medicine or placebo during the trial. Participants will be involved for about 13 months, attending 15 visits at the study site. During these visits, muscle strength, skin condition, physical function, fatigue, itch, and corticosteroid use are assessed. The main measurement is the Total Improvement Score at 24 or 52 weeks depending on location. Safety and other symptom measures will be monitored throughout the study period.