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Clinical Trial News

Subthreshold Electrical Stimulation Shows Promise in LHON Treatment

  • A prospective study reveals that subthreshold electrical stimulation (SES) can lead to statistically significant improvements in visual acuity in LHON patients.
  • The study demonstrated that younger patients at disease onset may experience more pronounced benefits from SES treatment, irrespective of the time between disease onset and SES initiation.
  • Visual field analysis showed significant improvements in summed actual sensitivity in half of the patients, suggesting potential for broader visual function enhancement.
  • While critical flicker frequency did not show significant changes, some patients exhibited notable increases, indicating variable responses to SES.

FDA Accepts NDA for Oral Semaglutide 25 mg for Weight Management and Cardiovascular Risk Reduction

  • The FDA has accepted the New Drug Application for oral semaglutide 25 mg, positioning it as a potential daily treatment option for both weight management and cardiovascular risk reduction.
  • If approved, oral semaglutide 25 mg would represent a significant advancement in GLP-1 receptor agonist therapy, offering patients a non-injectable alternative for obesity treatment.
  • The acceptance comes amid growing clinical interest in GLP-1 therapies, with this higher dose formulation potentially addressing the increasing demand for effective weight management solutions.

Global Clinical Trial Landscape Adapts to Post-Pandemic Research Challenges

• Clinical trial operations worldwide have undergone significant transformations in response to lessons learned from the COVID-19 pandemic, emphasizing remote monitoring and decentralized approaches.
• The evolution of CAR-T cell therapies, including HCAR-19 and PBCAR-19B, represents a new frontier in personalized medicine with promising developments in clinical research.
• Regulatory bodies and research institutions are implementing enhanced protocols for trial conduct, focusing on patient safety and data integrity in the modern clinical research environment.

NIAID's LMIV Spearheads Development of Novel Malaria Vaccines Targeting Transmission

  • The Laboratory of Malaria Immunology and Vaccinology (LMIV) is leading efforts in developing malaria vaccines, focusing on reducing severe disease and eliminating malaria in low-transmission areas.
  • LMIV's research spans basic discovery to product development, facilitating rapid translation of ideas into clinical trials, with a focus on understanding human immunity and responses to infection.
  • The lab's leading transmission-blocking vaccine candidate, Pfs230D1, is currently in a phase 2 clinical trial in Mali, with plans for further trials across West Africa.
  • Malaria remains a significant global health burden, with over 200 million cases and 400,000 deaths annually, emphasizing the need for effective vaccines to complement existing control measures.

Former GSK CEO Sir Andrew Witty Appointed to Lead WHO's Global COVID-19 Vaccine Initiative

• Sir Andrew Witty, former GlaxoSmithKline CEO, takes temporary leave from UnitedHealth's Optum to spearhead WHO's COVID-19 vaccine development efforts starting next week.
• Over 70 COVID-19 vaccine candidates are in development globally, with three frontrunners from CanSino Bio, Moderna, and Inovio already advancing to clinical testing phases.
• The appointment comes at a critical time as WHO faces funding challenges, with the Gates Foundation pledging $150 million following US funding withdrawal.

Gene Therapy Shows Promise in Treating Severe β-Thalassemia

A recent phase 3 trial has demonstrated the high efficacy of betibeglogene autotemcel, a gene therapy, in potentially curing severe transfusion-dependent β-thalassemia. Additionally, new research underscores the long-term safety of lentiviral gene therapies in primates, with no evidence of somatic mutations or malignancy.

PRISYM ID Launches Validated SaaS Platform to Accelerate COVID-19 Clinical Trial Labeling

  • PRISYM ID has introduced a pre-validated, cloud-based version of PRISYM 360 SaaS labeling platform specifically designed for COVID-19 clinical trials, offering rapid deployment with minimal IT requirements.
  • The platform features comprehensive capabilities including role-based security, multi-language support, and GS1-compliant barcode functionality, ensuring regulatory compliance and risk reduction in clinical trial processes.
  • This solution addresses critical labeling challenges in accelerated clinical trials, providing pharmaceutical companies with a validated, ready-to-use system that streamlines the trial supply chain.

First In Vivo CRISPR Therapy Dosed in Clinical Trial for Congenital Blindness

  • Editas Medicine and Allergan administered the first in vivo CRISPR-based therapy to an adult with congenital blindness, marking a significant milestone in gene editing.
  • The therapy, EDIT-101 (AGN-151587), aims to correct a point mutation in the CEP290 gene responsible for Leber congenital amaurosis type 10 (LCA10).
  • EDIT-101 uses an AAV5 vector with guide RNAs and Cas9 enzyme to target and remove the IVS26 mutation, which causes functional loss of the CEP290 protein.
  • Unlike Luxturna, which introduces a correct copy of the affected gene, EDIT-101 directly edits the genome to correct the mutation, representing a novel approach to treating LCA.

CDK4/6 Inhibitors Plus Endocrine Therapy Improves Survival in HR+/HER2- Advanced Breast Cancer

  • A meta-analysis of 9 RCTs with 5043 women showed that CDK4/6 inhibitors combined with endocrine therapy significantly improved progression-free survival in HR+/HER2- advanced breast cancer.
  • The combination therapy also demonstrated a significant overall survival benefit compared to endocrine therapy alone, regardless of treatment line or menopausal status.
  • The study found a higher risk of grade 3/4 adverse events in the combination therapy group compared to the endocrine therapy group.
  • These findings support the use of CDK4/6 inhibitors with endocrine therapy as a superior therapeutic strategy for HR+/HER2- advanced breast cancer.

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