University of Sussex
Browse

File(s) not publicly available

Use of clinical staging in amyotrophic lateral sclerosis for phase 3 clinical trials

journal contribution
posted on 2023-06-08, 20:55 authored by Rubika Balendra, Ashley Jones, Naheed Jivraj, I Nick Steen, Carolyn A Young, Pamela J Shaw, Martin R Turner, Nigel LeighNigel Leigh, Ammar Al-Chalabi, UK-MND LiCALS Study Group, Mito Target ALS Study Group
OBJECTIVES The use of clinical staging in the fatal neurodegenerative disease amyotrophic lateral sclerosis would have value in optimising future therapeutic trials. We aimed to use previous clinical trial data to determine the length of time patients spend in each of four proposed stages, its range and transition patterns to subsequent stages. METHODS Using databases from two multicentre clinical trials, patients were retrospectively staged through the trial course. At each stage we assessed whether patients then progressed to an earlier, consecutive or later stage or death. Duration spent in each stage before progression to a later stage was calculated. RESULTS There were 725 patients. No patients moved to an earlier stage. More patients at stages 1, 2 and 3 progressed to the consecutive stage rather than skipping a stage. 59.3% of patients at Stage 1 progressed to Stage 2, 54.0% of patients at Stage 2 progressed to Stage 3, 42.3% of patients at Stage 3 progressed to Stage 4 and 47.0% of Stage 4 patients progressed to death. Transition times between stages had a median duration of 3 to 7 months for stages 2 to 4. DISCUSSION We have shown using trial data that transition times between stages are short. Use of stage duration as an endpoint might allow a shorter trial duration. We have shown face validity in this system as most patients progress through consecutive stages, and none revert to earlier stages. Furthermore, we have shown the system is reliable across populations and therefore has content validity.

History

Publication status

  • Published

Journal

Journal of Neurology, Neurosurgery and Psychiatry

ISSN

0022-3050

Publisher

BMJ Publishing Group

Issue

1

Volume

86

Page range

45-49

Department affiliated with

  • BSMS Neuroscience Publications

Full text available

  • No

Peer reviewed?

  • Yes

Legacy Posted Date

2015-06-01

Usage metrics

    University of Sussex (Publications)

    Categories

    No categories selected

    Exports

    RefWorks
    BibTeX
    Ref. manager
    Endnote
    DataCite
    NLM
    DC