El Draguillo

Researchers are evaluating the safety, tolerability, how the body processes, and effectiveness of TERN-701, a selective allosteric inhibitor targeting BCR-ABL1, in adults with chronic phase chronic myeloid leukemia (CP-CML) who have been previously treated. The study is divided into two parts: Part 1 focuses on dose escalation to find safe dosage levels, and Part 2 involves randomized dose expansion to further assess the chosen doses and includes a mutation cohort for participants with certain resistance mutations. Participants in both parts will take TERN-701 orally once daily in 28-day cycles. Part 1 involves sequential dose escalation cohorts, while Part 2 evaluates two recommended dose levels selected from Part 1. The mutation cohort (Part 2m) will assess a specific 500 mg dose in participants with particular resistance mutations. Scheduled visits occur frequently during the first treatment cycle and then regularly throughout the study to monitor treatment effects. During the study, participants will have regular visits for evaluations including safety checks and laboratory tests. Researchers will measure dose-limiting toxicities, adverse events, hematologic response, molecular response, and changes in BCR-ABL1 transcript levels up to three years. The trial plans to enroll about 180 participants, with up to 80 in Part 1, about 80 in Part 2, and around 20 in the mutation cohort. All participants will receive the active treatment throughout the study duration.

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.

Progressive Familial Intrahepatic Cholestasis (PFIC) is a group of inherited liver conditions that affect bile flow and can cause serious liver problems. This research aims to observe genetic changes related to PFIC in adults with unexplained recurrent or chronic cholestasis in Spain, where little is known about the frequency of these genetic variations. Understanding these changes may help doctors diagnose PFIC more clearly and develop personalized treatment plans. The study will take place across several centers in Spain over 10 months. Each adult participant will attend a single-day visit during which their health information will be collected. A blood sample will be taken to perform routine blood tests and analyze genetic variations in PFIC-related genes. During the visit, researchers will collect medical history and health data and perform genetic testing on the blood sample. The main outcome measured is the percentage of participants who have at least one variant in PFIC-related genes at the time of enrollment. This study helps to better characterize the genetic causes of unexplained cholestasis and may support future personalized care.

Researchers are evaluating ivonescimab as a first-line treatment option for patients with metastatic non-small cell lung cancer (NSCLC) whose tumors show high PD-L1 expression. This phase 3 randomized, double-blinded study compares ivonescimab with pembrolizumab to assess overall survival and progression-free survival in this patient group. Participants will receive either ivonescimab or pembrolizumab as intravenous injections. The study is designed to monitor these treatments over time to determine which may provide better outcomes in controlling metastatic NSCLC in patients with high PD-L1 levels. The study includes patients with measurable non-brain lesions and no prior systemic treatment for metastatic NSCLC. During the trial, patients will be closely followed for up to approximately 36 months to measure overall survival and progression-free survival. Researchers will assess the safety and effectiveness of the treatments through regular evaluations, including monitoring for disease progression and survival status. This long-term follow-up ensures comprehensive understanding of treatment impact over time.

Gynecological cancers such as ovarian, uterine, and cervical cancers are common among women and often treated with radiotherapy and chemotherapy. While these treatments have improved survival, many women suffer from chronic pelvic pain caused by these therapies, which can be hard to manage and severely impact quality of life. Researchers are investigating how ozone therapy, a promising complementary treatment, may relieve this pain by studying its effects on gene expression and biological aging markers called epigenetic clocks. This prospective study observes two groups of adult women with gynecological tumors treated by radiotherapy and chemotherapy: those with chronic pelvic pain receiving compassionate ozone therapy and those without pelvic pain. There is no experimental intervention as patients continue their usual care. The study aims to compare gene expression and biological age differences between these groups and to measure changes after ozone therapy over 16 weeks. Participants will be evaluated at the start and after 16 weeks of ozone treatment, with assessments including gene expression profiles and epigenetic clock analysis. Researchers will also explore relationships between these molecular changes and pain severity, toxicity grade, quality of life, and biochemical markers of inflammation and oxidative stress. The study will run until August 2027 to gather comprehensive data on ozone therapy’s molecular impact in this patient population.

Chemotherapy-induced peripheral neuropathy (CIPN) is a common and disabling side effect of chemotherapy that can reduce treatment effectiveness and lower quality of life. Effective treatments for CIPN are limited, but emerging evidence suggests rectal ozone therapy may help, and the gut microbiome might play a role in CIPN development and response to treatment. This study is an observational analysis linked to the randomized clinical trial OzoParQT (NCT06706544) that investigates how gut microbiome profiles relate to CIPN severity and treatment outcomes. Participants will receive standard care plus rectal insufflation therapy over 16 weeks with 40 sessions. One group will receive ozone therapy, with O3/O2 concentrations increasing from 10 to 30 µg/mL, while the control group will receive placebo treatment with oxygen only. The study compares gut microbiome profiles and symptom changes between these groups and examines their relationship to quality of life, anxiety, depression, and biochemical markers of oxidative stress and inflammation. Participants will be adults with any tumor type experiencing CIPN-related numbness or tingling for at least 3 months with a certain severity level. Researchers will assess changes from baseline to week 16 in gut microbiome, patient-reported symptoms, neuropathy severity, toxicity grades, quality of life, anxiety and depression scores, and biochemical markers. The study lasts 16 weeks concurrent with ozone therapy, contributing to a larger project planned for 60 months.

Researchers are evaluating the safety and effectiveness of the Myval Transcatheter Heart Valve (THV) Series compared to Contemporary Valves (Sapien THV Series and Evolut THV Series) in patients with severe symptomatic native aortic valve stenosis. This prospective, randomized, multinational, and open-label trial involves 768 participants, evenly divided between the two treatment groups, using a transfemoral approach for valve replacement. Participants will receive either the Myval THV Series or one of the Contemporary Valves, with device sizes ranging from 20 mm to 29 mm for Myval and Sapien series, and 23 mm to 34 mm for the Evolut series. The study includes non-randomized registries for patients requiring extra-large Myval valve sizes and for initial lead-in cases performed under guidance before randomization. Throughout the study, participants will be monitored for safety and effectiveness outcomes, including the Primary Combined Safety and Effectiveness Endpoint defined by the Valve Academic Research Consortium-3 within 30 days post-procedure. Researchers will assess valve function and patient health to compare the performance of the devices, with follow-up evaluations conducted to ensure thorough safety monitoring.

Lysosomal Acid Lipase (LAL) Deficiency is a rare inherited disorder that causes a severe decrease in the enzyme responsible for breaking down certain fats in the body. This condition can present in infants as a rapidly progressing and often fatal disease called Wolman Disease, or in older children and adults as Cholesteryl Ester Storage Disease (CESD). It leads to serious liver and spleen problems, abnormal fat levels, and increased risk of heart disease. The overall prevalence and natural progression of LAL Deficiency are not well understood. This research is a global, observational registry designed to collect detailed, long-term data from patients with LAL Deficiency. The registry gathers information to improve understanding of the disease and support better care. It also helps monitor the effects of treatments such as sebelipase alfa. Both doctors and patients participate voluntarily in this study, which aims to build a comprehensive knowledge base across many countries and healthcare settings. Participants provide ongoing health information that helps researchers track disease progression, treatment outcomes, and clinical effects over time. The study collects data on symptoms, liver and spleen changes, blood tests, and other health measures. This continuous monitoring supports safety assessments and advances knowledge of LAL Deficiency, guiding future care and treatment strategies for patients.

Researchers are evaluating whether taking the Ovosicare4 Fertility food supplement can increase the chance of becoming pregnant in women aged 32 to 38 years who have been trying to conceive for at least six months. This observational study focuses on fertility and aims to understand the supplement's impact on spontaneous pregnancy rates within this age group. Participants will take the Ovosicare4 Fertility supplement, which contains a combination of MYO/DCI in a 3.6:1 ratio, antioxidants, vitamins, and minerals. The study does not involve extra tests or visits beyond routine clinical care. Each participant will be followed for up to 18 months, or until pregnancy occurs, whichever happens first. During the study, participants will attend a total of three visits. The researchers will monitor pregnancy occurrence over a maximum of nine months after starting supplementation to assess the rate of spontaneous pregnancy. No additional procedures are required beyond usual clinical practice, making participation convenient and integrated into regular healthcare visits.

Researchers are investigating whether the presence of isolated tumor cells in the first lymph nodes near the tumor can provide important information about the future outcomes of patients with low-risk endometrial (uterine) cancer. This study compares a group of patients with isolated tumor cells in their sentinel lymph nodes to a historical group without these cells, focusing on the likelihood of cancer recurrence. The study is observational and involves patients diagnosed with specific grades and stages of endometrial cancer without extensive lymphovascular space invasion. Participants in this study have undergone complete surgical staging, including pelvic sentinel lymph node biopsy and detailed tissue analysis, with no additional treatment given after surgery. The study observes patients with isolated tumor cells defined as very small groups of tumor cells in the lymph nodes, without larger metastases. No new treatments are administered during the study, as it is non-interventional. Throughout the study, patients provide tissue samples and allow researchers to review their medical records. The main outcome measured is recurrence-free survival, tracked for up to five years. This long-term follow-up helps assess whether isolated tumor cells in sentinel lymph nodes affect the chance of cancer returning. The study focuses on careful monitoring without additional treatment to better understand disease outcomes in this patient group.