Glasgow

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.

Researchers are investigating how the medicine Filgotinib works to relieve pain in patients with moderate to severe active rheumatoid arthritis RA. This study focuses on understanding two main factors influencing pain relief brain pathways linked to central nervous system sensitization seen in fibromyalgia, and the inflammation surrounding joints and related immune signals. The study aims to clarify why Filgotinib may provide faster pain relief compared to other treatments by examining these mechanisms. Participants prescribed Filgotinib will be observed using advanced 7 Tesla MRI brain imaging to measure changes in brain connectivity and glutamate levels associated with pain processing. The study will assess Filgotinibs impact on both central nervous system pain pathways and peripheral inflammation over periods of up to 12 weeks. This observational test-retest design does not involve altering prescribed treatments but focuses on detailed imaging and biological assessments. During the study, participants will undergo brain MRIs and evaluations of joint inflammation through ultrasound and blood tests measuring cytokines and chemokines. They will also complete questionnaires on pain, fatigue, anxiety, sleep, and cognitive function. Researchers will monitor changes in these measures at intervals up to 12 weeks to understand how Filgotinib affects pain and related symptoms. Total participation time aligns with the 12-week observation period.

Researchers are studying new treatment options for people with high-risk non-muscle invasive bladder cancer HR NMIBC, including cases with carcinoma in situ CIS. HR NMIBC affects the lining of the bladder but has not spread to muscle or beyond. The study aims to learn if adding intismeran autogene V940 to the standard Bacillus Calmette-Guerin BCG immunotherapy can improve outcomes by helping the immune system attack the cancer more effectively. Participants are divided into groups receiving different treatments. One group Cohort A receives both intismeran autogene via intramuscular injection every 3 weeks for 9 doses and BCG instillations weekly in specific weeks over about 75 weeks. Another group receives only BCG following the same weekly schedule. A third group Cohort B receives intismeran autogene alone every 3 weeks for 9 doses. The study evaluates these treatments over several years. During the study, participants will have regular treatments and follow-up visits where researchers will monitor cancer progression, recurrence, and survival for up to approximately 5 years. Assessments include event-free survival, recurrence-free survival, overall survival, response rates, time to cystectomy, and safety outcomes such as adverse events and treatment discontinuation. The study is randomized and open-label, with detailed long-term monitoring planned.

Researchers are studying pulmonary arterial hypertension PAH, a condition where lung blood vessels become thick and narrow, causing high blood pressure in the lungs and making it hard for the heart to work. PAH can cause difficulty breathing and limit activity. While standard treatments help symptoms, they do not stop the disease from worsening. This research focuses on sotatercept, a targeted therapy aimed at specific proteins involved in PAH, to learn about its long-term safety and tolerability when added to usual PAH treatments. Participants in this long-term follow-up study, who previously took part in certain sotatercept trials, may continue receiving sotatercept by subcutaneous injection every three weeks. Those coming from blinded studies start at 0.3 mgkg with possible increases up to 0.7 mgkg, while those from unblinded studies continue their current dose with possible titration to 0.7 mgkg. The study monitors participants over an extended period to assess continued effects alongside their usual PAH therapy. During the study, participants will have regular assessments including monitoring for adverse events, blood tests for blood components and chemistry, body weight, blood pressure, and ECG readings. Researchers will also evaluate exercise capacity, heart function markers, and risk scores related to PAH. The study aims to follow participants for up to approximately 7 to 8 years to understand long-term safety, treatment tolerability, and health changes while using sotatercept with standard PAH care.

Researchers are evaluating the effects of enicepatide, a dual GLP-1GIP receptor agonist, at multiple doses compared with placebo for weight management in adults with obesity or overweight who do not have Type 2 diabetes. This Phase III, randomized, double-blind study aims to assess both the efficacy and safety of once-weekly enicepatide in this population, addressing weight-related comorbidities such as prediabetes, hypertension, and cardiovascular conditions. Participants will be randomly assigned to receive either placebo or one of three enicepatide dosing regimens, administered once weekly via an integrated drug-device combination product. The treatment phase lasts through 72 weeks, during which changes in body weight and other health measures are monitored. The study includes multiple assessments to track body weight percentage change, waist circumference, fasting glucose and insulin levels, lipid profiles, blood pressure, and quality of life measures. Throughout the study, participants will undergo regular evaluations including physical examinations, laboratory tests, and questionnaires related to physical functioning and urinary incontinence. Researchers will monitor adverse events, patient-reported health questionnaires, and biomarkers at baseline and weekly intervals through week 72. This long-term follow-up allows for a comprehensive assessment of treatment effects and safety in participants managing obesity or overweight without Type 2 diabetes.

Researchers are investigating ETX101, a gene therapy delivered via an adeno-associated viral vector, in infants and children diagnosed with SCN1A-positive Dravet syndrome. The study includes multiple age groups ranging from 6 months to under 18 years and aims to assess the safety and effects of ETX101. It is designed as a Phase 12, multicenter trial with different parts evaluating dose levels and treatment impact using a randomized, controlled approach in later stages. The study is divided into parts Part 1A uses an open-label, dose-escalation design to test up to four doses of ETX101 in participants aged 6 to under 36 months. Part 1B is open-label and tests a single dose in participants aged 48 months to under 18 years. Part 2 is a double-blind, randomized, sham delayed-treatment control study involving participants aged 6 to under 48 months, where participants receive either ETX101 or a sham procedure with delayed treatment. This part includes two cohorts and lasts up to 52 weeks. Participants will be closely monitored through the study with assessments of seizure frequency, cognitive and adaptive behavior changes using tools like the Bayley-4 and Vineland-3 scales, and clinical global impressions over 52 weeks. Safety and effectiveness are evaluated by comparing seizure counts before and after dosing. The study includes various cognitive and behavioral outcome measures and follows participants from dosing up to one year for treatment effects and safety observations.

Researchers are evaluating the safety, effectiveness, optimal dose, and behavior of an investigational drug called BNT326, alone or combined with other immunotherapy agents, in adults with advanced solid tumors. This study includes patients with tumors that have either spread metastatic, returned after treatment, or progressed despite previous therapies, across various cancer types such as melanoma, lung cancer, breast cancer, gastric cancer, colorectal cancer, and cervical cancer. Participants are divided into two parts Part 1 tests BNT326 alone in different tumor-specific groups, some with dose randomization to find optimal dosing. Part 2 evaluates BNT326 alone or combined with another investigational drug called pumitamig in several cancer types, with some groups receiving randomized doses and others non-randomized treatments. Treatments are given via intravenous infusion, with some oral medications combined in Part 1. The study includes dose escalation and randomization phases, and treatment can continue for up to 24 months or until disease progression or other reasons. During the study, participants undergo screening, treatment, safety follow-up, efficacy follow-up, and long-term survival monitoring phases. Researchers assess adverse events, treatment responses, disease progression, and drug behavior in the body using clinical evaluations and laboratory tests. Follow-up assessments occur up to approximately 38 months for Part 1 and 48 months for Part 2, with continued treatment possible for those benefiting from the therapy. The study aims to gather comprehensive data on safety, dosing, and effectiveness in this patient population.

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 the efficacy and safety of trontinemab in people with early symptomatic Alzheimers disease, ranging from mild cognitive impairment to mild dementia due to Alzheimers. This Phase III trial aims to understand how trontinemab affects cognitive decline and disease progression in this population. Participants are randomly assigned to receive either intravenous trontinemab or a placebo in a parallel-group design. Treatment is administered by IV infusion, and the effects are compared over a period of 72 weeks. The study includes comprehensive safety and efficacy assessments throughout this period. During the 72 weeks of the study, participants will undergo various evaluations including cognitive tests such as the Clinical Dementia Rating-Sum of Boxes CDR-SB, Alzheimers Disease Assessment Scales, brain imaging with PET and MRI scans, and biomarker measurements in cerebrospinal fluid and blood. Safety monitoring includes tracking adverse events, infusion reactions, and antibody development. The study requires participants to have a study partner and to complete all study procedures over this time.

Researchers are evaluating zorevunersen, an investigational antisense oligonucleotide drug, in children with Dravet syndrome, a rare and severe form of epilepsy. This Phase 3, global, multicenter, randomized, double-blind, sham-controlled study aims to assess the efficacy, safety, and tolerability of zorevunersen by measuring changes in major motor seizure frequency and other important aspects such as behavior, cognition, clinical status, and quality of life. Participants will be randomly assigned to receive either zorevunersen or a sham procedure during Treatment Period 1, which lasts about 52 weeks. Zorevunersen is given by intrathecal injection at specific doses and intervals throughout this period. After Treatment Period 1, all eligible patients enter Treatment Period 2, where everyone receives zorevunersen for additional dosing over several months. Patients who complete the study may have the chance to join an open-label extension to continue receiving the drug. During the study, participants will undergo regular assessments including seizure monitoring, behavioral and cognitive evaluations, and health-related quality of life measurements. The primary outcome is the change in major motor seizure frequency at Week 28, with secondary outcomes assessed at Week 52. Safety and tolerability are also closely monitored. Overall participation lasts through both treatment periods and possible extension, with detailed follow-up to evaluate the drugs potential for disease modification.