Multivariate Survival Analysis of a Comprehensive Clinical Trial of Rapamycin in Amyotrophic Lateral Sclerosis Explores Prognostic Factors and Survival Patterns in C9orf72 Carriers

De Nardi A, Paris A, Lauria M, Martinelli I, Zucchi E, Simonini C, Mandrioli J, Marchetti L · 2026 · European Journal of Neurology · Atlas ID DEN2026B

What this study shows

Secondary, observational survival analysis of the RAP-ALS rapamycin trial (n=63) finds a protective association between rapamycin treatment and survival in C9orf72 mutation carriers (log-rank p=0.026), consistent with a comparison against an independent C9orf72+ cohort (n=40; RMST p=0.04); confounding by indication is not excluded, and the authors flag this as exploratory given the small treated-carrier subgroup (n=6).

Abstract

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Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease marked by considerable variability in survival times. This retrospective study aims to evaluate the prognostic value of a broad range of variables by conducting a comprehensive survival analysis on longitudinal data from RAP-ALS, a clinical trial investigating the effects of rapamycin in ALS patients (n = 63). Covariates were classified as risk or protective factors according to their hazard ratios. Regularized Cox regression was utilized to select the best-performing multivariate models in cross-validation.

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At a glance

Evidence type	H Human study Marked H because it is direct evidence from a human clinical trial or human cohort; the code names the kind of study, not its quality -- a small, well-run trial is still H.
Study type	3 - Human Observational
Model system	Human (ALS patients, RAP-ALS trial, n=63)
Journal	European Journal of Neurology
Year	2026
Peer reviewed	Yes
Record last updated	2026-09-23
Source	DOI 10.1111/ene.70756 · PMID 42760861

Cite this paper

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De Nardi, A., Paris, A., Lauria, M., Martinelli, I., Zucchi, E., Simonini, C., Mandrioli, J., & Marchetti, L. (2026). Multivariate Survival Analysis of a Comprehensive Clinical Trial of Rapamycin in Amyotrophic Lateral Sclerosis Explores Prognostic Factors and Survival Patterns in C9orf72 Carriers. European Journal of Neurology. https://doi.org/10.1111/ene.70756

@article{DEN2026B,
  author       = {De Nardi, A. and Paris, A. and Lauria, M. and Martinelli, I. and Zucchi, E. and Simonini, C. and Mandrioli, J. and Marchetti, L.},
  title        = {{Multivariate Survival Analysis of a Comprehensive Clinical Trial of Rapamycin in Amyotrophic Lateral Sclerosis Explores Prognostic Factors and Survival Patterns in C9orf72 Carriers}},
  journal      = {European Journal of Neurology},
  year         = {2026},
  doi          = {10.1111/ene.70756},
  note         = {PMID: 42760861},
}

Cite this Atlas record

The record is the Atlas's own work — the evidence label, the extracted findings and the links. It is cited as part of the dataset, not as the paper.

Barton, O. (2026). Oliver's mTOR Atlas (record DEN2026B) [Data set]. https://mtor-atlas.org/study/DEN2026B/ · Dataset DOI 10.5281/zenodo.22059963

@misc{atlas_DEN2026B,
  author       = {Barton, Oliver},
  title        = {{Oliver's mTOR Atlas}, record DEN2026B},
  howpublished = {Data set},
  year         = {2026},
  url          = {https://mtor-atlas.org/study/DEN2026B/},
  doi          = {10.5281/zenodo.22059963}
}