English
 
User Manual Privacy Policy Disclaimer Contact us
  Advanced SearchBrowse

Item

ITEM ACTIONSEXPORT
  Design and analysis of three-arm trials with negative binomially distributed endpoints.

Mütze, T., Munk, A., & Friede, T. (2016). Design and analysis of three-arm trials with negative binomially distributed endpoints. Statistics in Medicine, 35(4), 505-521. doi:10.1002/sim.6738.

Item is

Basic

show hide
Item Permalink: http://hdl.handle.net/11858/00-001M-0000-0029-AFE7-6 Version Permalink: http://hdl.handle.net/11858/00-001M-0000-0029-AFEB-D
Genre: Journal Article

Files

show Files
hide Files
:
2248933.pdf (Publisher version), 846KB
 
File Permalink:
-
Name:
2248933.pdf
Description:
-
Visibility:
Restricted (Max Planck Institute for Biophysical Chemistry (Karl Friedrich Bonhoeffer Institute), Göttingen; )
MIME-Type / Checksum:
application/pdf
Technical Metadata:
Copyright Date:
-
Copyright Info:
-
License:
-
:
2248933_Suppl.pdf (Supplementary material), 327KB
Name:
2248933_Suppl.pdf
Description:
-
Visibility:
Public
MIME-Type / Checksum:
application/pdf / [MD5]
Technical Metadata:
Copyright Date:
-
Copyright Info:
-
License:
-

Locators

show
hide
Description:
-

Creators

show
hide
 Creators:
Mütze, T., Author
Munk, A.1, Author              
Friede, T., Author
Affiliations:
1Research Group of Statistical Inverse-Problems in Biophysics, MPI for biophysical chemistry, Max Planck Society, ou_1113580              

Content

show
hide
Free keywords: negative binomial distribution; non-inferiority; restricted maximum likelihood; superiority; three-arm trial
 Abstract: A three-arm clinical trial design with an experimental treatment, an active control, and a placebo control, commonly referred to as the gold standard design, enables testing of non-inferiority or superiority of the experimental treatment compared with the active control. In this paper, we propose methods for designing and analyzing three-arm trials with negative binomially distributed endpoints. In particular, we develop a Wald-type test with a restricted maximum-likelihood variance estimator for testing non-inferiority or superiority. For this test, sample size and power formulas as well as optimal sample size allocations will be derived. The performance of the proposed test will be assessed in an extensive simulation study with regard to type I error rate, power, sample size, and sample size allocation. For the purpose of comparison, Wald-type statistics with a sample variance estimator and an unrestricted maximum-likelihood estimator are included in the simulation study. We found that the proposed Wald-type test with a restricted variance estimator performed well across the considered scenarios and is therefore recommended for application in clinical trials. The methods proposed are motivated and illustrated by a recent clinical trial in multiple sclerosis. The R package Three Armed Trials, which implements the methods discussed in this paper, is available on CRAN.

Details

show
hide
Language(s): eng - English
 Dates: 2015-09-212016-02-20
 Publication Status: Published in print
 Pages: -
 Publishing info: -
 Table of Contents: -
 Rev. Method: Peer
 Identifiers: DOI: 10.1002/sim.6738
 Degree: -

Event

show

Legal Case

show

Project information

show

Source 1

show
hide
Title: Statistics in Medicine
Source Genre: Journal
 Creator(s):
Affiliations:
Publ. Info: -
Pages: - Volume / Issue: 35 (4) Sequence Number: - Start / End Page: 505 - 521 Identifier: -