Belfast

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 conducting an observational study to better understand fucosidosis, a rare disease, its symptoms, and how it changes over time. The study aims to gather detailed information about the diseases natural history and progression, including patients who have or have not undergone stem cell transplant. There is currently no approved treatment for fucosidosis, so this study focuses on observing the condition without introducing new medications. The study is divided into two parts Part A involves collecting past medical data from up to 57 participants, and Part B involves following up with up to 31 participants prospectively, many of whom may also be in Part A. Participants will continue to receive their usual medical care throughout the study, with no additional treatments or interventions provided by the study team. Participants will provide medical history and health information that will be reviewed over time. The main focus is to track disease progression in individuals untreated by investigational products for up to four years. This includes gathering retrospective data and ongoing prospective observations to aid future research and potential treatment development. The study is expected to run until January 2031.

Relapsed neuroblastoma is a common and serious childhood cancer that is difficult to treat, especially in high-risk cases where the disease returns after initial treatment or does not respond to standard therapies. This trial aims to improve survival for children aged 1 year and older with relapsed neuroblastoma by testing new combinations of treatments. It is an international, open-label, randomized phase III study designed to develop and evaluate these new therapies while also studying the biology of the disease using biomarker samples. Participants will be randomly assigned to one of two main treatment groups or enrolled in smaller dose confirmation groups testing new drug combinations. The current main treatments include combinations of dinutuximab beta, irinotecan, temozolomide, and bevacizumab given in 12 cycles every three weeks. Additional experimental groups may receive different combinations of these drugs. The trial includes a translational sub-study collecting tumor, blood, and bone marrow samples to better understand neuroblastoma and improve targeted therapies. During the study, patients will receive their assigned treatments over multiple cycles and be closely monitored for safety, treatment response, and quality of life. Researchers will perform various assessments including imaging scans, laboratory tests, and questionnaires to measure progression-free survival, side effects, and overall clinical benefit. Follow-up will continue up to five years after treatment to assess long-term outcomes and collect further data on patient health and disease progression.

Researchers are evaluating the effects of ensifentrine inhalation suspension compared to a placebo in adults with non-cystic fibrosis bronchiectasis NCFBE. This study aims to understand how ensifentrine, added to standard care, impacts lung flare-ups, symptoms, and quality of life. It is a randomized, double-blind, placebo-controlled Phase II trial designed to assess both efficacy and safety over up to 52 weeks. Participants will be randomly assigned to receive either 3 mg of nebulized ensifentrine or a placebo, both delivered twice daily using a standard jet nebulizer. The treatment period may last up to 52 weeks, although the study may end earlier once the last participant completes 24 weeks. Neither participants nor study staff will know which treatment is administered during the study. During the study, participants will be monitored regularly, including assessments of lung function, respiratory symptoms, quality of life, and tracking of any pulmonary exacerbations. Measurements will be taken at baseline and at various time points up to 52 weeks. Safety will be evaluated through adverse event tracking. The primary outcome focuses on the rate of pulmonary exacerbations per year, with additional assessments of symptom changes and lung function.

This research aims to pilot and evaluate an online compassion-based program designed to support the mental health of healthcare staff accessing psychological assessment within an Occupational Health Service. It compares this program with the usual treatment offered, which is Staffcare counselling. The study focuses on reducing burnout and stress by fostering self-compassion through Compassion Focused Therapy CFT and Compassionate Mind Training CMT. Participants are randomly assigned to one of two groups the Compassionate Mind Training CMT intervention or Treatment As Usual TAU. The CMT group receives a four-week online program led by a CMT expert, including weekly 30-minute videos, audio exercises, and reading materials to complete at their own pace. The TAU group receives standard care through Staffcare counselling, which involves six weekly counselling sessions via the Employee Assistance Programme. Participants complete online questionnaires at four points before treatment, mid-treatment, after treatment, and one month later. These assessments measure mental health, burnout, self-compassion, fear of self-compassion, and treatment credibility. Data is collected securely online. The study will monitor participant wellbeing and provide support as needed. Participation lasts through the intervention and follow-up periods, with confidentiality and anonymity maintained throughout.

Researchers are evaluating new DNA damage repair inhibitors DDRis combined with radiotherapy for patients with stage IIB to IIIB non-small cell lung cancer NSCLC in this Phase IB study. The purpose is to find the recommended dose and safety of DDRis given with a fixed dose of curative radiotherapy, followed by up to 12 months of consolidation durvalumab immunotherapy in some groups. The study uses a specialized method to monitor dose-limiting toxicities over 13.5 months to guide safe dosing decisions. Participants receive radiotherapy administered in 30 daily fractions of 2Gy each, totaling 60Gy Monday to Friday. Some groups also receive oral DDRi drugs such as Olaparib, AZD1390, Ceralasertib, or AZD5305 alongside radiotherapy. Selected groups receive up to 12 months of durvalumab immunotherapy after radiotherapy. No placebo is used both patients and doctors know the assigned treatments. One study arm was discontinued. During the study, patients are closely followed to monitor side effects, cancer response, and treatment compliance. Blood samples are collected to track progress and explore which patients may benefit most. Researchers assess dose-limiting toxicities up to 13.5 months after radiotherapy start and evaluate safety, response, disease control, survival, and quality of life up to two years post-treatment. Participation lasts through treatment and follow-up periods for comprehensive monitoring.

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.

Researchers are evaluating Mim8, a new medicine designed to help people with haemophilia A, including those with or without inhibitors. Mim8 aims to prevent bleeding episodes by replacing the function of the missing clotting factor VIII. This long-term study will last up to 5.5 years, ending either when Mim8 is approved in the participants country or by June 2028, whichever comes first. The study includes participants who have been involved in earlier related studies or are new infants with severe haemophilia A. Participants will receive Mim8 as a preventive treatment through subcutaneous injections. Depending on their entry point, participants may use an enhanced cartridge or a DV3407 pen-injector device for administering Mim8. The treatment is given regularly over the study period, with participants potentially receiving up to 262 injections. In the event of bleeding, additional haemostatic medications may be used as agreed with the study doctor. Female participants who are pregnant, breastfeeding, or planning pregnancy during the study are not eligible. During the study, participants will be monitored for any side effects, including injection site reactions and the development of antibodies against Mim8. Researchers will also track bleeding episodes, Mim8 blood levels, and device handling for some participants. Participants and their representatives will complete diaries and questionnaires about their treatment and health. Safety will be carefully followed throughout the study, which may last several years depending on individual enrollment and study progress.

Researchers are evaluating the safety and effects of a new medicine called NNC0487-0111 in people who have Heart Failure with preserved Ejection Fraction HFpEF or Heart Failure with mildly reduced Ejection Fraction HFmrEF and excess body weight. This phase 3 clinical trial aims to find out if NNC0487-0111 is safe and effective for treating these conditions compared to a placebo. Participants have HFpEF or HFmrEF and a body mass index of 30 or above. The study is sponsored by Novo Nordisk AS and uses a randomized, quadruple-masked design. Participants will receive either NNC0487-0111 or a matching placebo by injection under the skin once a week. The NNC0487-0111 is given in increasing doses over time. The study is parallel in design, meaning participants are randomly assigned to one of the two groups and receive that treatment throughout the trial. This treatment period extends for up to about 165 weeks. The study evaluates the time to certain heart failure events, hospitalizations, cardiovascular deaths, and other major cardiovascular events. During the study, participants will be monitored regularly to assess heart failure outcomes and kidney function, as well as quality of life using questionnaires like the Kansas City Cardiomyopathy Questionnaire. Safety and effectiveness are assessed through hospital visits, heart failure event tracking, and blood tests including kidney function and blood sugar levels. The total participation spans over three years, with ongoing evaluations to measure the time to heart failure events and cardiovascular outcomes. Participants receive close medical monitoring throughout the study period.

Researchers are observing the use of the drug elafibranor in people with Primary Biliary Cholangitis PBC, a rare progressive liver disease that damages bile ducts and can lead to liver scarring. The study aims to collect real-world information on how effective, safe, and tolerable elafibranor is for participants receiving this treatment. The total participation lasts about 5 years for each person. Participants in the study are those who have been diagnosed with PBC and are either starting or currently receiving treatment with the commercialized drug elafibranor. The study does not intervene with treatment but monitors participants as they use the drug in routine care. Data is collected during regular physician follow-up visits over the 60-month period. During the study, researchers will assess response to treatment at 6 months and continue monitoring various health measures such as liver function tests, symptom scales for itching and fatigue, quality of life questionnaires, and liver stiffness. They will also track adverse events, treatment satisfaction, and adherence throughout the study. Follow-up is based on routine medical visits, with no extra visits required specifically for the study.