Study to Understand Fatal Genetic Diseases in Children Incorporates Digital Participant Education Program
A clinical trial was launched last week to help researchers understand how two rare and fatal genetic diseases develop in children and provide insight into treatment for them in the future. The study, run by Azafaros, will follow patients in the UK, US, France, Italy, Germany and Brazil for four years.
The PROspective Neurological Disease TrajectOry study (PRONTO) is a Natural History Study that follows children who have been diagnosed with GM1 or GM2 gangliosidoses (also known as Tay-Sachs and Sandhoff disease), a class of rare inherited disorders that are fatal in childhood and which currently have no approved treatments.
Read more about this new clinical trial here.
Unifying Industry to Better Understand GCP Guidance
May 7th 2025In this episode of the Applied Clinical Trials Podcast, David Nickerson, head of clinical quality management at EMD Serono; and Arlene Lee, director of product management, data quality & risk management solutions at Medidata, discuss the newest ICH E6(R3) GCP guidelines as well as how TransCelerate and ACRO have partnered to help stakeholders better acclimate to these guidelines.
Arcus’ Quemliclustat Earns Orphan Drug Designation as Phase III Pancreatic Cancer Trial Advances
July 11th 2025The FDA has granted orphan status to Arcus Biosciences’ CD73 inhibitor quemliclustat for metastatic pancreatic cancer, as the global PRISM-1 Phase III trial nears full enrollment following promising survival data from ARC-8.
QWINT-1 Trial: Once-Weekly Efsitora Matches Daily Glargine in Type 2 Diabetes Management
July 10th 2025Results from the Phase III QWINT-1 trial show that Eli Lilly’s once-weekly insulin efsitora is noninferior to once-daily glargine in reducing HbA1c among insulin-naïve adults with type 2 diabetes, offering a simplified fixed-dose regimen with fewer hypoglycemic events and less treatment burden.
