Zaragoza

Researchers are evaluating the combination of CGT9486 and sunitinib compared to sunitinib alone in patients with locally advanced, unresectable, or metastatic Gastrointestinal Stromal Tumors GIST. This Phase 3, open-label international trial involves multiple parts, including dose confirmation, drug interaction assessments, and efficacy comparisons. The study also includes substudies focusing on drug-drug interaction potential and first-line treatment in patients with specific genetic mutations KIT exon 9. Approximately 482 patients will participate across these parts.

Preterm birth, defined as birth before 37 weeks of gestation, occurs in about 8 percent of pregnancies in Canada and is linked to many health challenges, especially when it happens before 29 weeks. At this early stage, infants often face breathing difficulties and may require resuscitation. This trial compares resuscitation using either low 30% or high 60% oxygen levels to determine which approach results in better survival and neurodevelopmental outcomes at around 24 months of age. The study uses a cluster randomized crossover design where hospitals alternate between starting resuscitation with 30% or 60% oxygen for groups of 30 infants. Resuscitation includes standard care steps like lung expansion and ventilation support as needed. Oxygen is initially given at the assigned concentration for the first 5 minutes, then adjusted based on the infants oxygen saturation levels and heart rate over the next 5 minutes to maintain target saturation ranges. This approach aims to balance risks of too much or too little oxygen. Participants are infants born between 23 and 28 weeks gestation who receive full resuscitation at participating centers. During the study, infants have oxygen saturation monitored continuously, and adjustments to oxygen concentration are made carefully. Researchers will evaluate survival and major neurodevelopmental outcomes at 24 months corrected age, along with several safety and health measures during the neonatal intensive care stay. The trial is expected to provide important evidence to guide oxygen use during resuscitation of extremely preterm infants.

Researchers are evaluating the safety, tolerability, and therapeutic effects of BNT113 combined with pembrolizumab compared to pembrolizumab alone as a first-line treatment for patients with unresectable recurrent or metastatic head and neck squamous cell carcinoma HNSCC positive for human papilloma virus 16 HPV16 and expressing the protein PD-L1 with a combined positive score of 1 or higher. This is an open-label, multi-site, Phase IIIII clinical trial consisting of two parts an initial safety run-in phase and a randomized phase. In the safety run-in phase Part A, patients receive BNT113 in combination with pembrolizumab to confirm safety and tolerability at selected dose levels. The randomized phase Part B compares BNT113 combined with pembrolizumab against pembrolizumab monotherapy. Treatments are given by intravenous injection or infusion and continue for up to 24 months. An optional pre-screening phase allows tumor samples to be tested for HPV16 DNA and PD-L1 expression before the main trial screening. Participants will be closely monitored throughout the study. Assessments include safety evaluations, tumor response, and survival outcomes such as overall survival and progression-free survival. Tumor tissue samples must be provided for testing. Researchers will measure treatment-emergent adverse events, response rates, duration of response, and disease control. The study may last up to 48 months, with ongoing safety and efficacy monitoring during and after treatment.

Researchers are evaluating LY3884961, a genetic therapy delivered as a single intravenous infusion, in adults with peripheral symptoms of Gaucher Disease. This Phase 12, open-label, multicenter study aims to assess the safety and tolerability of different dose levels of LY3884961. The study includes dose-finding cohorts followed by an expansion cohort to better understand the therapys effects in this patient group. Participants will receive one dose of LY3884961 and will be monitored closely for 18 months to evaluate safety, tolerability, immune response, biomarkers, and treatment effects. The study may include up to three dose levels tested in small groups of patients, with an additional group enrolled afterward. The total study duration for each patient is about five years, including a screening period of up to 60 days. During the trial, participants will have regular evaluations including laboratory tests, imaging, and clinical assessments to track treatment-emergent adverse events and changes in spleen volume, platelet count, and relevant biomarkers. Researchers will also monitor the use and discontinuation of enzyme replacement or substrate reduction therapies. Long-term safety and immune response will be followed for up to 42 months after the initial 18-month period.

Researchers are evaluating the safety and effects of marstacimab, a study medicine, for potential treatment of hemophilia in boys and male children aged 1 to 17 years. The trial focuses on those with severe Hemophilia A or moderately severe to severe Hemophilia B, including participants with or without inhibitors. The study aims to compare participants experiences with marstacimab to their historical bleeding episodes without the medicine. All participants will receive weekly subcutaneous injections of marstacimab. The first dose is administered at the study site by staff, and subsequent doses can be given at home or by study staff during the 12-month treatment period. Enrollment opens sequentially by age groups, starting with adolescents 12-17 years, followed by children 6-11 years, and finally children 1-5 years. The study includes about 1 month of screening, 12 months of treatment, and 1 month of follow-up. Participants will visit the study site at least 10 times during the study, with the option for two visits to occur at home if allowed. They will also receive six phone calls approximately every two months. Researchers will monitor bleeding rates, adverse events, injection site reactions, and immune responses. The overall participation lasts about 14 months, including screening, treatment, and follow-up periods.

Researchers are evaluating the effect of Xeomin injections compared to placebo injections for preventing chronic migraine. This Phase 3, randomized, double-blind, placebo-controlled trial includes an extension period and aims to measure changes in the number of monthly migraine days. Participants have chronic migraine and meet specific criteria related to headache frequency and migraine history. Participants receive Xeomin or placebo injections into muscles of the head and neck at pericranial and cervical points. The study includes two Xeomin dose groups and a placebo group during the controlled period, with all groups receiving Xeomin in the extension phase. Four treatments are given approximately 12 weeks apart over a total study duration of 52 to 55 weeks. Participants take part in 14 visits over the study period, with the first, last, and four treatment visits conducted in person and the remaining eight visits by phone or video call. Researchers collect headache and migraine data from diaries and assess changes in monthly migraine days as the primary outcome. Safety is monitored by tracking treatment-related adverse events throughout the trial.

Researchers are evaluating the use of Xeomin injections to prevent episodic migraine. This Phase 3 clinical trial compares Xeomin to placebo injections in the muscles of the head and neck to measure changes in the number of monthly migraine days. Participants have episodic migraine with or without aura, and the study aims to assess the efficacy and safety of different Xeomin doses over time. Participants receive a series of four Xeomin or placebo injections spaced about 12 weeks apart. The study includes two experimental groups receiving different Xeomin doses and a placebo group, followed by an extension period where some participants receive Xeomin. Injections are given at specific points around the head and neck. The trial lasts approximately 52 to 55 weeks, starting with a 4 to 5 week screening period. Participants attend about 14 visits, including the first and last visits and four treatment visits conducted on-site, with other visits done remotely by phone or video call. Researchers monitor changes in monthly migraine days, headache days, and medication use, as well as any treatment-related side effects throughout the study.

Researchers are evaluating the gene therapy FLT201 in adults with Gaucher disease Type 1 who have been on stable enzyme replacement therapy ERT or substrate reduction therapy SRT for at least two years. This Phase 3, non-randomized, multicenter study aims to confirm the efficacy and safety of FLT201 after participants stop their current treatments. FLT201 is an advanced therapy designed to address this rare genetic condition by delivering a gene using a recombinant adeno-associated virus vector. Participants receive a single intravenous infusion of FLT201 during the study. This gene therapy uses a replication-incompetent viral vector to introduce the therapeutic gene. The study includes only one treatment group and does not involve placebo or comparison arms. The primary treatment period focuses on evaluating FLT201s impact over one year after stopping prior therapies. Throughout the study, participants undergo regular assessments to monitor their health and treatment effects. Researchers evaluate the therapys efficacy by measuring clinical outcomes at one year. Safety is closely observed to identify any adverse effects. The total study duration extends to 2032, allowing long-term follow-up of participants responses and overall well-being.

Researchers are evaluating the efficacy and safety of rilvegostomig compared to pembrolizumab monotherapy as the first-line treatment for patients with metastatic non-small cell lung cancer mNSCLC whose tumors express high levels of PD-L1. This Phase III, randomized, double-blind, multicenter global study focuses on patients with mNSCLC without certain genetic mutations who are suitable for this treatment approach. Participants are randomly assigned to receive either rilvegostomig or pembrolizumab intravenously on Day 1 of each 21-day cycle. The study compares these two drugs over repeated treatment cycles as first-line therapy. Both treatments are biological agents given by infusion, and the study is designed to monitor their effects over up to approximately five years. During the trial, participants will undergo regular assessments including physical exams, imaging scans such as CT or MRI to measure tumor lesions, and laboratory tests to evaluate organ function. Researchers will closely monitor overall survival, progression-free survival, treatment response, duration of response, and patient-reported outcomes on physical functioning and quality of life. Safety and immunogenicity of rilvegostomig will also be evaluated. Participants are followed and assessed for up to five years to gather comprehensive data on treatment effects and long-term outcomes.

Researchers are evaluating the long-term safety and tolerability of JNJ-81201887, given as an intravitreal injection a shot into the eye, in participants with Geographic Atrophy GA secondary to Age-related Macular Degeneration AMD. This study is a long-term extension of parent clinical trials where participants had previously received either low or high doses of JNJ-81201887 or a sham procedure. The goal is to monitor participants over an extended period to understand any lasting treatment effects or side effects. Participants entering this long-term extension study will not receive additional doses of the study drug or any new intervention as part of this trial. They previously participated in parent studies where they were treated with either low dose or high dose JNJ-81201887 or sham procedure. Some participants who were in the sham group of the parent study may receive open-label treatment outside this study before entering this extension. This study focuses solely on follow-up without new treatment administration. Throughout the study, participants will undergo regular assessments to monitor ocular and systemic safety. These include tracking treatment-emergent adverse events, clinical laboratory tests, retinal imaging, and eye examinations over up to five years. This extended monitoring aims to evaluate the long-term safety profile of the previous treatments. Participants will be followed with periodic visits and evaluations, with the total study duration extending until 2030.