Clinical Trial News
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.
AI Revolutionizes Cancer Detection: Breakthroughs in Liquid Biopsies, Imaging, and Pathology
• Advanced machine learning algorithms enable liquid biopsies for early lung cancer detection, potentially increasing annual lives saved from 600 to 12,000 in the United States through blood-based screening.
• AI-powered imaging systems match radiologists' accuracy in breast cancer detection, demonstrating potential to reduce second reader workload by 88% in UK screening processes.
• Deep neural networks in pathology achieve expert-level accuracy in tumor pattern classification, processing slides in under one minute while maintaining diagnostic quality comparable to practicing pathologists.
Cardiol Therapeutics' Cannabidiol Formulation Shows Cardioprotective Effects in Heart Failure Model
• Study results demonstrate that Cardiol's pharmaceutical cannabidiol formulation significantly reduced cardiac hypertrophy and produced dose-dependent reductions in inflammation markers and fibrosis in a heart failure model.
• The findings confirm the anti-inflammatory and anti-fibrotic properties of the company's cannabidiol formulation, supporting its potential as a cardioprotective therapy for heart conditions.
• Cardiol Therapeutics is advancing plans for an international Phase 2 trial of its CardiolRx formulation in acute myocarditis, a leading cause of sudden cardiac death in young adults.
Long-term Benefits of Dose-dense Adjuvant Chemotherapy in Early Breast Cancer Patients
A study with a median follow-up of 15.8 years suggests that dose-dense (DD) chemotherapy may offer long-term benefits for high-risk early breast cancer patients, particularly those with hormone receptor-negative and HER2-positive tumors. The MIG-1 trial compared the efficacy and safety of DD versus standard-interval FEC chemotherapy, showing a trend towards improved outcomes with the DD approach.
Mycophenolate Mofetil Shows Promise in Phase II Osteosarcoma Trial
• A Phase II clinical trial is evaluating mycophenolate mofetil's efficacy and safety in patients with high-grade locally advanced or metastatic osteosarcoma.
• The trial aims to determine progression-free survival at 16 weeks as the primary endpoint, with secondary endpoints including overall survival and safety parameters.
• Mycophenolate mofetil, an immunosuppressant, has shown anticancer activity against osteosarcoma in preclinical studies, warranting further investigation in humans.
• The study involves multiple centers in Thailand and includes adolescent and adult patients who have progressed after standard chemotherapy or are not candidates for it.
Five Key Strategies for Enhancing Patient-Centric Clinical Trials Amid Low Recruitment Rates
• Clinical trials face significant challenges with less than 5% eligible participant enrollment and 30% dropout rates globally, highlighting the urgent need for patient-centric approaches.
• Patient-reported outcomes (PROs) are gaining prominence, with 27% of studies on ClinicalTrials.gov incorporating patient perspective measurements to better assess treatment impact.
• Modern clinical trials are embracing remote reporting technologies, rapid expense reimbursement, and lay result summaries to reduce participant burden and improve retention rates.
China Approves Clinical Trials for First COVID-19 Vaccine
• China has approved clinical trials for its first vaccine against COVID-19, developed by a research group led by academician Chen Wei.
• Pre-trial testing of the vaccine indicated it was both effective and safe, according to China Central Television.
• Chen Wei and her team arrived in Wuhan on January 26 to investigate the coronavirus and develop methods to combat the epidemic.
• The vaccine's approval marks a significant step in China's efforts to combat the COVID-19 pandemic, with clinical trials now underway.
Free Genetic Testing Initiative Accelerates Drug Development for Inherited Retinal Diseases
• The Foundation Fighting Blindness's My Retina Tracker program offers free genetic testing and counseling for inherited retinal disease patients in the US, having enrolled over 6,000 participants since 2017.
• ProQR Therapeutics becomes the first industry partner of the program, gaining access to crucial genotype/phenotype data to advance development of RNA-based therapies for specific genetic mutations.
• The initiative aims to test 20,000 patients in coming years, helping overcome key challenges in rare disease drug development including patient identification and clinical trial optimization.