Data about the treatment of a neurodegenerative disease
Table of Contents
Description
These data are simulated data, but they closely resemble that of a double-blind, randomized, placebo-controlled, parallel arm, trial sponsored by H. Lundbeck A/S. The aim of the trial was to evaluate the efficacy and safety of a drug to treat a certain neurodegenerative disease. Patients are randomized 1:1 to an active and a placebo arm. The primary endpoint is the change from baseline to Week 12 on a quantitative outcome. This outcome is measured at baseline and at 6 and 12 weeks after. The outcome of interest is a score between 0 and 20, where lower scores indicate a worse disease status. There are a few missing data for the values of the score measured at 6 and 12 weeks (patients did not show up at the follow-up visit). For confidentiality reasons, the data from the original study and further details about the study could not be shared.
Variables
| Variable | Explanation (Unit) |
|---|---|
| id | Patient ID |
| trt | Treatment arm, standard of case (SoC) or experimental (Exp) |
| baseline | Baseline score |
| week6 | Change in score from baseline to week 6 |
| week12 | Change in score from baseline to week 12 |
Load data into R
load(url("http://paulblanche.com/files/Neuro.rda")) head(Neuro)
References
Baayen, Blanche, Jennison, Ozenne. Design and analysis of group sequential trials for repeated measurements when pipeline data occurs: a tutorial (2025+).
Source
Supplementary material of the publication cited above. Data are those of the 'decision' analysis.