Belfast

Researchers are evaluating the safety, tolerability, and therapeutic effects of a combination treatment using BNT113 and pembrolizumab compared to pembrolizumab alone for patients with unresectable recurrent or metastatic head and neck squamous cell carcinoma (HNSCC) that is positive for human papillomavirus 16 (HPV16+) and expresses the PD-L1 protein with a combined positive score of 1 or higher. This Phase II/III trial includes patients whose cancer cannot be treated with local therapies and who have not received prior systemic anticancer therapy for their current disease condition. The trial consists of two parts. Part A is a non-randomized Safety Run-In Phase to confirm the safety and tolerability of BNT113 combined with pembrolizumab at the selected dose. Part B is a randomized phase that compares BNT113 plus pembrolizumab against pembrolizumab alone as first-line treatment. Patients in Part A continue their treatment without randomization. Treatments are given by intravenous injection or infusion, and patients may receive either combination therapy or monotherapy for up to 24 months. There is also an optional pre-screening phase to test tumor samples for HPV16 DNA and PD-L1 expression before entering the main trial. Participants undergo regular assessments including tumor measurements based on RECIST 1.1 criteria confirmed by independent review. Researchers monitor treatment-emergent adverse events for up to 27 months in Part A and evaluate overall survival and progression-free survival for up to 48 months in Part B. Tumor tissue samples are collected before treatment to confirm eligibility. The study involves ongoing safety monitoring and efficacy evaluations throughout the treatment and follow-up periods.

Neuroblastoma is a common and serious childhood cancer that often leads to death, especially in high-risk cases where the cancer is harder to treat. This trial focuses on children aged one year and older with relapsed neuroblastoma, which means the cancer has come back or is resistant after initial treatment. The study aims to find better treatment combinations to improve survival, evaluate their safety and effectiveness, and learn more about the biology of relapsed neuroblastoma through biomarker research. This is a phase I/II international trial designed to potentially impact clinical practice and advance targeted therapies. Participants will be randomly assigned to one of two main treatment groups receiving combinations of drugs including dinutuximab beta, irinotecan, temozolomide, and bevacizumab, administered every three weeks for up to 12 cycles. A third treatment group with a more experimental combination is also available for a smaller number of patients to confirm dosing and safety before possibly expanding. The trial includes detailed drug regimens and allows for adjustments based on safety and effectiveness findings. During the study, participants will undergo various assessments such as imaging scans, blood tests, and bone marrow evaluations to monitor disease status and treatment effects. Researchers will track progression-free survival and observe any dose-limiting toxicities over up to five years after randomization. Biological samples will be collected to support research on neuroblastoma. Patient quality of life and safety will be closely monitored throughout the treatment and follow-up periods, which together may last up to eight years from enrollment.

Researchers are investigating the safety and tolerability of ARGX-119 in adults diagnosed with DOK7-Congenital Myasthenic Syndromes (CMS). This Phase 1b study also evaluates how ARGX-119 is processed in the body, how the immune system responds to it, and its potential effects on patients' symptoms and physical function. Participants who qualify after screening will be randomly assigned in a 4:1 ratio to receive either intravenous ARGX-119 or a placebo during a double-blinded treatment period. Following this, they will enter a follow-up period. After completing the follow-up, participants can join an open-label active-treatment phase where they receive ARGX-119. The total study duration is about 38 months. Throughout the study, researchers will monitor adverse events up to week 42 and track changes in participants' walking distance over 72 weeks. Safety, immune response, and how the drug is processed will be regularly assessed. Participants will undergo various evaluations to measure how they feel and function during the study and its follow-up phases.

Researchers are studying the effects of ensifentrine inhalation suspension compared to placebo in adults with non-cystic fibrosis bronchiectasis (NCFBE). The goal is to evaluate how ensifentrine affects the rate of lung flare-ups, symptoms, and quality of life in this condition. This Phase II trial is randomized, double-blind, and placebo-controlled to ensure unbiased assessment of ensifentrine's safety and effectiveness alongside standard care. Participants will be randomly assigned to receive either 3 mg of nebulized ensifentrine suspension or a placebo, both administered twice daily using a standard jet nebulizer. The treatment period will last at least 24 weeks and may continue up to 52 weeks, with neither the participants nor the study staff knowing which treatment is given. This setup helps compare ensifentrine's effects directly against placebo over an extended period. During the study, participants will attend regular visits for lung function tests, symptom assessments, and quality of life evaluations. Researchers will monitor lung flare-ups defined by the need for antibiotics or antivirals over approximately 52 weeks. Safety will be closely observed, and participants' ability to use the nebulizer and provide sputum samples will be assessed. This thorough monitoring aims to gather detailed data on ensifentrine's impact on NCFBE symptoms and flare-up frequency.

The COVID-19 pandemic has highlighted the urgent need to support the mental health of healthcare staff, especially those assessed by Occupational Health services. This research evaluates an online compassion-based intervention designed to improve psychological wellbeing by fostering self-compassion. The study compares this intervention with usual care, aiming to reduce burnout, stress, and improve mental health symptoms in healthcare professionals. Participants will be randomly assigned to one of two groups: the Compassionate Mind Training (CMT) intervention group or the Treatment as Usual (TAU) group. The CMT group will access a four-week online program led by a CMT expert, including weekly 30-minute videos, audio exercises, and reading materials focused on mindfulness and compassionate practices. The TAU group will receive standard care through six weekly counselling sessions provided by Staffcare via the Employee Assistance Programme. Participants will complete questionnaires online at four time points: before treatment, mid-treatment, after treatment, and four weeks after treatment ends. These assessments measure professional quality of life, depression, anxiety, stress, burnout, self-compassion, and treatment credibility. Data will be securely collected and stored, and support will be available throughout the intervention to address any distress. Total participation will last approximately five to six weeks including follow-up.

Researchers are investigating the safety and recommended dose of new drugs called DNA damage response inhibitors (DDRis) combined with radiotherapy for patients with stage IIB to IIIB non-small cell lung cancer (NSCLC). This Phase IB study aims to improve radiotherapy effectiveness by blocking cancer cells' ability to repair DNA damage, potentially leading to better treatment outcomes. After radiotherapy and DDRi treatment, some patients will receive up to 12 months of durvalumab immunotherapy to align with standard care practices. Participants will receive radiotherapy delivered in 30 daily fractions of 2 Gy each, for a total dose of 60 Gy, Monday through Friday. Three out of every four patients will also take a single DDRi drug orally alongside radiotherapy. This open-label trial means both patients and doctors know which treatment is given. Selected study arms include consolidation treatment with durvalumab through intravenous infusion for up to 12 months following radiotherapy. The study uses a special model to carefully evaluate dose limiting toxicities up to 13.5 months after starting radiotherapy to guide dosing decisions. Throughout the study, participants will be closely monitored for side effects and cancer response. Assessments include blood tests to track treatment progress and identify which patients may benefit most. Researchers will measure dose limiting toxicities as the primary outcome. Participants must be followed for at least 13.5 months after radiotherapy start to evaluate safety. The study involves a rigorous schedule of clinical and laboratory evaluations to ensure patient safety and gather detailed information about treatment effects.

Researchers are conducting a global study to understand the impact of moderate to severe alopecia areata (AA), non-segmental vitiligo (NSV), and hidradenitis suppurativa (HS) on adolescents and adults. This study aims to assess the burden these conditions place on patients' quality of life and daily functioning in a large real-world population. The study involves participants diagnosed by a physician with one of the three conditions: AA, NSV, or HS. There are no interventional treatments or medications being tested in this study, as it is observational in nature. Data collection focuses on patient-reported outcomes and measures that evaluate disease severity and its effects. Participants will complete various questionnaires and assessments related to their condition, such as the Alopecia Areata Symptom Impact Scale (AASIS) for AA, the Severity of Alopecia Tool (SALT) for scalp hair loss in AA, the Facial Vitiligo Area Scoring Index (F-VASI) and Vitiligo Quality of Life Score (VitiQoL) for vitiligo, and the Dermatology Life Quality Index (DLQI) and International Hidradenitis Suppurativa Severity Scoring System (IHS4) for HS. These tools help researchers understand how symptoms affect quality of life and disease severity. The study collects information up to the day of the study visit.

This research focuses on men with prostate cancer who have previously participated in an enzalutamide clinical study sponsored by Astellas or Medivation. It aims to gather long-term safety information from participants who continue to benefit from enzalutamide treatment. This is a Phase 2 open-label extension study designed to monitor ongoing treatment effects after the initial study has completed its primary analysis or evaluation period. Participants will continue their previous treatment regimens, which may include enzalutamide taken orally once daily. Some may also receive abiraterone acetate with prednisone or leuprolide acetate depending on their prior study enrollment. Dose adjustments are allowed with medical monitor approval. The first visit of this study should occur within seven days of the last visit of the prior study unless treatment is temporarily paused. Participants are asked to return to their study site every 24 weeks for safety reviews, including adverse event monitoring and medication checks. At visits every 12 weeks, participants return unused study drugs and receive new supplies if needed. Safety data, including all adverse events and serious adverse events, are collected from consent until study completion, which may last up to 96 months. The study follows local standard care guidelines and includes a post-marketing phase in South Korea.

Researchers are observing the use of the drug elafibranor in people with Primary Biliary Cholangitis (PBC), a rare and progressive liver disease where bile ducts are damaged. This damage can lead to scarring of the liver (cirrhosis) and is often linked with symptoms like itching and fatigue. If PBC worsens, it may require a liver transplant or could be fatal without one. The study aims to understand how effective, safe, and tolerable elafibranor is for those being treated in everyday clinical settings. Participants in this study will be those who have been diagnosed with PBC and are either starting or already receiving treatment with commercial elafibranor at a dose of 80 mg per day. The study is non-interventional, meaning it observes participants as they receive their usual care without altering treatment. The total study duration for each participant is approximately 60 months, or 5 years. During the study, researchers will gather information about participants’ responses to treatment, safety, and tolerability over time. Outcome measurements include the percentage of participants who respond to treatment after 6 months. Participants and their caregivers may complete questionnaires, and researchers will monitor treatment effects and side effects throughout the study period to better understand how elafibranor works in real-world use.

This research aims to evaluate the safety and effectiveness of iza-bren, a bi-specific antibody-drug conjugate targeting EGFR and HER3 with a topoisomerase inhibitor, compared to the treatment of physician's choice (paclitaxel, nab-paclitaxel, carboplatin plus gemcitabine, or capecitabine). The study focuses on patients with previously untreated, locally advanced, recurrent inoperable, or metastatic triple-negative breast cancer (TNBC) or estrogen receptor (ER)-low, HER2-negative breast cancer who are not eligible for anti-PD(L)1 or endocrine therapies. The trial is conducted in two phases, phase 2 and phase 3, to thoroughly assess these treatments.