Exploring concurrent validity of the CLN2 Clinical Rating Scale: Comparison to PedsQL using cerliponase alfa clinical trial data.

Nicola SpecchioPaul GissenEmily de Los ReyesAndrew OlayeCharlotte CampTristan CurteisAnnabel GriffithsThomas ButtJessica Cohen-PfefferPeter SlasorZlatko SisicMohit JainAngela Schulz

Published in: PloS one (2024)

NCT01907087, NCT02485899.

Keyphrases

clinical trial
electronic health record
big data
phase ii
open label
study protocol
locally advanced
double blind
replacement therapy
randomized controlled trial
machine learning
radiation therapy
phase iii
data analysis
clinical evaluation