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
locally advanced
replacement therapy
study protocol
randomized controlled trial
artificial intelligence
rectal cancer