Astellas Pharma (United States)

Clinical outcome assessments in Pompe disease: a pragmatic review of their validity, concept coverage, and value in clinical practice and clinical research

Abstract Background Pompe disease is a rare, autosomal recessive, neuromuscular disorder caused by a deficiency in the acid alpha-glucosidase enzyme leading to the build-up of glycogen within lysosomes, which can result in severe cardiac, pulmonary, and skeletal disorders. The ag...

How low do you need to go? Association between various prostate‐specific antigen response measures and clinical outcomes in metastatic castration‐sensitive prostate cancer in the Veterans Health Administration data

BACKGROUND: Multiple phase 3 metastatic castration-sensitive prostate cancer (mCSPC) studies consistently show a prostate-specific antigen (PSA) nadir of <0.2 ng/mL to be the preferred threshold for PSA response. However, in real-world practice, physicians often use other metrics...

BE-BOIN: A Dose Optimization Design Accommodating Backfill and Late-Onset Toxicity

The US Food and Drug Administration (FDA) launched Project Optimus and issued guidance to reform dose-finding and selection trials, shifting the paradigm from identifying the maximum tolerated dose (MTD) to determining the optimal biological dose (OBD), which optimizes the risk a...