Evaluating the efficacy and safety of GKT137831 in adults with type 1 diabetes and persistently elevated urinary albumin excretion: a statistical analysis plan.
Albumins
/ analysis
Albuminuria
/ etiology
Australia
Diabetes Mellitus, Type 1
/ drug therapy
Double-Blind Method
Humans
Models, Statistical
Multicenter Studies as Topic
New Zealand
Pyrazolones
/ administration & dosage
Pyridones
/ administration & dosage
Randomized Controlled Trials as Topic
Time Factors
Treatment Outcome
Albuminuria
GKT137831
Randomised controlled trial
Statistical analysis plan
Type 1 diabetes
Journal
Trials
ISSN: 1745-6215
Titre abrégé: Trials
Pays: England
ID NLM: 101263253
Informations de publication
Date de publication:
03 Jun 2020
03 Jun 2020
Historique:
received:
14
01
2020
accepted:
11
05
2020
entrez:
5
6
2020
pubmed:
5
6
2020
medline:
16
2
2021
Statut:
epublish
Résumé
The investigational medicinal product GKT137831 is a selective inhibitor of NOX 1 and 4 isoforms of the nicotinamide adenine dinucleotide phosphate (NADPH) oxidase family of enzymes, which has the potential to ameliorate diabetic kidney disease. An investigator-initiated, double-blind, randomised, placebo-controlled, multicentre phase 2 clinical trial started recruitment in December 2017, with the aim of evaluating the efficacy and safety of GKT13783, in adults with type 1 diabetes mellitus and persistently elevated urinary albumin excretion over a period of 48 weeks. The trial is currently recruiting in Australia and New Zealand, with recruitment expected to end on 30 June 2020. The primary outcome measure of the trial is the urinary albumin excretion level measured at 48 weeks of treatment. This statistical analysis plan presents an update to the published trial protocol and provides a comprehensive description of the statistical methods that will be used for the analysis of the data from this trial. In doing so, we follow the "Guidelines for the content of statistical analysis plans in clinical trials" to support transparency and reproducibility of the trial findings. With the use of this prior statistical analysis plan, we aim to minimise bias in the reporting of the findings of this trial, which evaluates the investigational medicinal product GKT137831. The results of the trial are expected to be published in 2022. ANZCTR registry: ACTRN12617001187336. Registered on 14 July 2017. Universal Trial Number: U1111-1187-2609; Protocol number: T1DGKT137831; Genkyotex trial number: GSN000241.
Sections du résumé
BACKGROUND
BACKGROUND
The investigational medicinal product GKT137831 is a selective inhibitor of NOX 1 and 4 isoforms of the nicotinamide adenine dinucleotide phosphate (NADPH) oxidase family of enzymes, which has the potential to ameliorate diabetic kidney disease. An investigator-initiated, double-blind, randomised, placebo-controlled, multicentre phase 2 clinical trial started recruitment in December 2017, with the aim of evaluating the efficacy and safety of GKT13783, in adults with type 1 diabetes mellitus and persistently elevated urinary albumin excretion over a period of 48 weeks.
METHODS/DESIGN
METHODS
The trial is currently recruiting in Australia and New Zealand, with recruitment expected to end on 30 June 2020. The primary outcome measure of the trial is the urinary albumin excretion level measured at 48 weeks of treatment. This statistical analysis plan presents an update to the published trial protocol and provides a comprehensive description of the statistical methods that will be used for the analysis of the data from this trial. In doing so, we follow the "Guidelines for the content of statistical analysis plans in clinical trials" to support transparency and reproducibility of the trial findings.
DISCUSSION
CONCLUSIONS
With the use of this prior statistical analysis plan, we aim to minimise bias in the reporting of the findings of this trial, which evaluates the investigational medicinal product GKT137831. The results of the trial are expected to be published in 2022.
TRIAL REGISTRATION
BACKGROUND
ANZCTR registry: ACTRN12617001187336. Registered on 14 July 2017. Universal Trial Number: U1111-1187-2609; Protocol number: T1DGKT137831; Genkyotex trial number: GSN000241.
Identifiants
pubmed: 32493401
doi: 10.1186/s13063-020-04404-0
pii: 10.1186/s13063-020-04404-0
pmc: PMC7268311
doi:
Substances chimiques
Albumins
0
Pyrazolones
0
Pyridones
0
setanaxib
45II35329V
Types de publication
Clinical Trial Protocol
Letter
Langues
eng
Sous-ensembles de citation
IM
Pagination
459Références
Int J Epidemiol. 2019 Aug 1;48(4):1294-1304
pubmed: 30879056
Diabetes Care. 2003 Apr;26(4):1258-64
pubmed: 12663607
BMC Med Res Methodol. 2001;1:2
pubmed: 11336663
Stat Methods Med Res. 2007 Jun;16(3):219-42
pubmed: 17621469
PLoS One. 2018 Feb 5;13(2):e0191629
pubmed: 29401521
Stat Med. 2010 Dec 10;29(28):2920-31
pubmed: 20842622
Stat Sci. 2010 Feb 1;25(1):1-21
pubmed: 20871802
Emerg Themes Epidemiol. 2017 Aug 23;14:8
pubmed: 28852415
BMJ. 1998 Aug 8;317(7155):362-3
pubmed: 9694748
Contemp Clin Trials. 2020 Mar;90:105892
pubmed: 31740428
Anal Chem. 2012 Dec 18;84(24):10768-76
pubmed: 23150939
J Clin Epidemiol. 2014 Mar;67(3):335-42
pubmed: 24291505
Int J Methods Psychiatr Res. 2011 Mar;20(1):40-9
pubmed: 21499542
JAMA. 2017 Dec 19;318(23):2337-2343
pubmed: 29260229
Biostatistics. 2016 Jan;17(1):16-28
pubmed: 26286812