Methodological insights from the EPISTOP trial to designing clinical trials in rare diseases—A secondary analysis of a randomized clinical trial
The statistical re-analysis of the raw trial data therefore supports the published results and confirms that there is no additional bias introduced by randomization, thereby increasing the value of the results. However, this highlights that this aspect needs to be considered in future trials, especially in rare diseases, to avoid additional biases in an already small sample size where it may be difficult to reach significance.
Authors
- Sergiusz Jóźwiak (ORCID: https://orcid.org/0000-0003-3350-6326)
- Rima Nabbout (ORCID: https://orcid.org/0000-0001-5877-4074)
- Mathieu Kuchenbuch (ORCID: https://orcid.org/0000-0002-5944-2204)
- N Heussen (ORCID: https://orcid.org/0000-0002-6134-7206)
- R.-D Hilgers (ORCID: https://orcid.org/0000-0002-5945-1119)
- Katarzyna Kotulska (ORCID: https://orcid.org/0000-0002-5015-250X)
- Stephanie Wied (ORCID: https://orcid.org/0000-0003-0616-6610)
- Maya Dirani (ORCID: https://orcid.org/0000-0002-7175-0867)
Institutions
- Sigmund Freud Privatuniversität Wien (AT)
- Children's Memorial Health Institute (PL)
- Institut des Maladies Génétiques Imagine (FR)
- RWTH Aachen University (DE)
Publication Details
- Journal
- PLoS ONE
- Published
- 2024-12-03
- DOI
- https://doi.org/10.1371/journal.pone.0312936
- Primary Topic
- Genomics and Rare Diseases
- Type
- article
- Field-Weighted Citation Impact
- 0.00
Funders
- Horizon 2020