Clinical Trial News

Investigators explore alternative interventions like intubation, inferior turbinate fracture, or balloon dilation for CNLDO in older children, contrasting with age-based single procedure approach. A one-stage approach categorizes patients by obstruction type, requiring different interventions for simple vs. complex CNLDO. Clinical trial results show no significant change in success rate for simple CNLDO with added interventions in 12-24 months olds, but higher success with intubation in 24-36 months olds, suggesting treatment should consider patient age, surgical skills, instruments, and parental preferences.

Callum, diagnosed with acute myeloid leukaemia at age three, underwent intensive chemotherapy and participated in a Cancer Research UK-funded clinical trial. Now in remission, he enjoyed a magical trip to Lapland with his family, highlighting the importance of research in improving children's cancer survival rates.

Bristol Myers Squibb presents long-term follow-up results from the EXPLORER-LTE cohort of the MAVA-LTE trial, showing sustained efficacy and safety of CAMZYOS in treating NYHA class II-III symptomatic obstructive hypertrophic cardiomyopathy (oHCM) over 3.5 years, with consistent improvements in echocardiographic measures and biomarkers, and most patients reaching NYHA class I.

Human respiratory syncytial virus (hRSV) is a major cause of acute lower respiratory infections globally, affecting newborns, young children, older adults, and immune-compromised individuals. It presents with a range of clinical manifestations, from mild to severe lower respiratory tract diseases like bronchiolitis or pneumonia. Approximately 90% of children under 2 years are infected with hRSV, and the virus can lead to repeated infections, posing a serious health threat. The global incidence of hRSV increased after the COVID-19 pandemic due to relaxed public health measures. Safe and effective hRSV vaccines are highly required, especially in low- and middle-income countries. hRSV is an enveloped, negative-sense RNA virus encoding 11 proteins, with the F and G proteins being the main targets of neutralizing antibodies. Vaccine development has historically faced challenges, including safety concerns and insufficient efficacy, but recent advancements focus on using PreF as the antigen to induce potent neutralizing antibodies and prevent non-neutralizing antibody production and enhanced respiratory disease responses. Over 20 hRSV vaccine candidates are currently under clinical development, with several in phase III trials and three already approved for marketing.

The article discusses the prevalence, pathogenesis, and current treatments for cervicogenic headache (CEH), emphasizing the need for safer, more effective therapies. It explores the potential of meridian sinew tuina, a traditional Chinese manipulative therapy, in treating CEH, supported by clinical evidence. The article also introduces an innovative ultrasound localization technique for guiding meridian sinew tuina therapy and targeted injections into the greater and third occipital nerves, aiming to alleviate CEH symptoms. A randomized controlled trial is proposed to assess the effectiveness of this combined approach in improving pain scores, headache intensity, and cervical spine mobility in CEH patients.

Tolebrutinib met primary endpoint in HERCULES phase 3 study for non-relapsing secondary progressive multiple sclerosis, showing reduction in disability progression. GEMINI 1 and 2 studies in relapsing MS did not meet primary endpoint but showed delay in disability worsening. Results to be discussed with regulatory authorities and presented at ECTRIMS.

Arrowhead Pharmaceuticals announces positive Phase 3 PALISADE study results for plozasiran in patients with familial chylomicronemia syndrome (FCS), showing significant reductions in triglycerides and risk of acute pancreatitis, with plans to file a New Drug Application with the FDA by year-end 2024.

Innovent Biologics announces mazdutide clinical study results to be presented at EASD 60th Annual Meeting in Madrid, including Phase 3 DREAMS-2 study in type 2 diabetes. Mazdutide, a GLP-1R/GCGR dual agonist, shows superior blood sugar-lowering and weight loss efficacy. Innovent has two NDAs accepted by China's NMPA for review and five ongoing Phase 3 studies.

From April 2019 to August 2023, 15 patients with various cancers and confirmed germline or somatic POL-MUTs were enrolled. Median age was 50 years, with most diagnosed at stage IV and having undergone multiple prior treatments. Most (66.7%) had POLE mutation. Response to toripalimab was evaluated in 14 patients, showing an objective response rate of 21.4% and disease control rate of 57.1%. Three patients had complete or partial responses, and two completed two-year protocol treatment. Treatment-related adverse events were reported in 60% of patients, with one death related to treatment. POLE/POLD1 mutation status influenced treatment response, with POLE-EDMs showing better response rates than POLE-non-EDMs. Patients with POLE/POLD1 and PBRM1 co-mutations exhibited higher TMB and immunotherapy response signature scores, suggesting a favorable response to immunotherapy.

Dr. Deborah Belfort presented CATHEDRAL-HF trial findings at ESC, showing discontinuation of HF treatment while maintaining carvedilol could be safe in HF with improved EF. Key opinion leaders highlighted suboptimal medication doses in chronic HF patients, and HF is a lifelong condition with various acute causes.