Bristol

Researchers are evaluating how well adults with new-onset generalized myasthenia gravis gMG respond to treatment with efgartigimod PH20 SC. This study focuses on adults who have had generalized disease signs or symptoms for less than one year and aims to assess clinical outcomes during a phase 4, open-label, single-group trial. Participants will receive subcutaneous injections of efgartigimod PH20 SC during a treatment period lasting 51 weeks. The study follows each participant for approximately 58 weeks to monitor their response to the treatment and any changes in their condition over time. During the study, participants will be regularly evaluated using measures like the proportion who achieve minimal symptom expression MSE within the first 16 weeks, changes in MG-ADL and MG-QOL-15r scores over time, and use of systemic corticosteroids as add-on therapy. Researchers will also monitor the incidence of adverse events throughout the study period to assess safety and tolerability.

Researchers are conducting a study to develop a tissue repository for collecting and storing blood, serum, tissue, urine, and tumor samples from children and adults diagnosed with common childhood cancers, such as leukemia and central nervous system tumors. The study aims to improve understanding of cancer biology and support research into new therapies through comprehensive molecular, genomic, proteomic, and metabolomic profiling. This includes patients with tumors, pre-malignant disorders, suspected cancer susceptibility syndromes, and their biological relatives, as well as healthy volunteers undergoing routine care. The study collects extra biological samples, such as blood, serum, tissue, urine, or tumors, during medically necessary procedures or approved research studies without additional risk. Samples may be obtained from patients at any age with malignancies or pre-malignancies, their relatives, and healthy volunteers during surgeries or routine visits. Collected specimens are used for advanced analyses including DNA, RNA, protein extraction, immune profiling, and growth factor investigations. The repository supports future research by storing these samples and overseeing their distribution to investigators. Participants provide consent to allow use of their samples for genomic and molecular research. Sample collection is done alongside standard medical care, minimizing risk and avoiding extra procedures solely for research purposes. The study measures include ongoing tissue analysis and may involve interviews with parents or caregivers about knowledge and attitudes related to genetic testing for pediatric cancer. Participation duration varies as samples are collected during routine care visits or approved studies, with an expected enrollment of 100-150 patients annually.

Researchers are evaluating the effectiveness, safety, tolerability, and how the body processes two different doses of the drug EXPD-101 in adults with Chronic Obstructive Pulmonary Disease COPD. This phase 2 study compares these two doses against a placebo to better understand treatment options for COPD over a year. Participants will be randomly assigned to one of three groups one receiving EXPD-101FXS7553 Dose 1, another receiving Dose 2, or a third receiving a placebo. Each participant will take their assigned oral tablet once daily for 52 weeks. The study will closely monitor these treatments throughout this year-long period. During the study, participants will undergo regular evaluations including physical exams, vital sign checks, electrocardiograms ECGs, and laboratory tests. Researchers will measure the number of moderate and severe COPD flare-ups, changes in lung function, quality of life, and any side effects or abnormalities. Blood samples will be collected at multiple time points to study the drugs presence and metabolism. The total participation time is approximately one year, with continuous safety and health monitoring throughout.