Seamless phase II/III/IIIb clinical trial designs with different endpoints for different phases

Hui Quan; Xiaodong Luo; Tianyue Zhou; Peng Liang Zhao

doi:10.1080/03610926.2019.1618871

Seamless phase II/III/IIIb clinical trial designs with different endpoints for different phases

Hui Quan
, Xiaodong Luo
, Tianyue Zhou
, Peng Liang Zhao

Research output: Contribution to journal › Article › peer-review

3 Scopus citations

Abstract

In a seamless phase II/III/IIIb trial, K (K ≥ 2) doses versus placebo control are evaluated at phase II. Based on phase II results, one dose will be selected for phases III and IIIb. Pre-specified additional numbers of patients will be enrolled into the selected dose and placebo control during phases III and IIIb. Results of the phase III endpoint may be submitted for an early New Drug Application. Final analyses will be conducted for ultimate claims of treatment effects for the selected dose on the phase III and IIIb endpoints. Multiplicity adjustment is performed for the overall type I error rate control.

Original language	English
Pages (from-to)	5436-5454
Number of pages	19
Journal	Communications in Statistics - Theory and Methods
Volume	49
Issue number	22
DOIs	https://doi.org/10.1080/03610926.2019.1618871
State	Published - 16 Nov 2020
Externally published	Yes

Keywords

Multiplicity adjustment
combination test
conditional power
estimation of treatment effect

Access to Document

10.1080/03610926.2019.1618871

Cite this

@article{3333cdec8e014e49a5c65933b837fdd2,

title = "Seamless phase II/III/IIIb clinical trial designs with different endpoints for different phases",

abstract = "In a seamless phase II/III/IIIb trial, K (K ≥ 2) doses versus placebo control are evaluated at phase II. Based on phase II results, one dose will be selected for phases III and IIIb. Pre-specified additional numbers of patients will be enrolled into the selected dose and placebo control during phases III and IIIb. Results of the phase III endpoint may be submitted for an early New Drug Application. Final analyses will be conducted for ultimate claims of treatment effects for the selected dose on the phase III and IIIb endpoints. Multiplicity adjustment is performed for the overall type I error rate control.",

keywords = "Multiplicity adjustment, combination test, conditional power, estimation of treatment effect",

author = "Hui Quan and Xiaodong Luo and Tianyue Zhou and Zhao, \{Peng Liang\}",

note = "Publisher Copyright: {\textcopyright} 2019 Taylor \& Francis Group, LLC.",

year = "2020",

month = nov,

day = "16",

doi = "10.1080/03610926.2019.1618871",

language = "English",

volume = "49",

pages = "5436--5454",

journal = "Communications in Statistics - Theory and Methods",

issn = "0361-0926",

publisher = "Taylor and Francis Ltd.",

number = "22",

}

TY - JOUR

T1 - Seamless phase II/III/IIIb clinical trial designs with different endpoints for different phases

AU - Quan, Hui

AU - Luo, Xiaodong

AU - Zhou, Tianyue

AU - Zhao, Peng Liang

PY - 2020/11/16

Y1 - 2020/11/16

N2 - In a seamless phase II/III/IIIb trial, K (K ≥ 2) doses versus placebo control are evaluated at phase II. Based on phase II results, one dose will be selected for phases III and IIIb. Pre-specified additional numbers of patients will be enrolled into the selected dose and placebo control during phases III and IIIb. Results of the phase III endpoint may be submitted for an early New Drug Application. Final analyses will be conducted for ultimate claims of treatment effects for the selected dose on the phase III and IIIb endpoints. Multiplicity adjustment is performed for the overall type I error rate control.

AB - In a seamless phase II/III/IIIb trial, K (K ≥ 2) doses versus placebo control are evaluated at phase II. Based on phase II results, one dose will be selected for phases III and IIIb. Pre-specified additional numbers of patients will be enrolled into the selected dose and placebo control during phases III and IIIb. Results of the phase III endpoint may be submitted for an early New Drug Application. Final analyses will be conducted for ultimate claims of treatment effects for the selected dose on the phase III and IIIb endpoints. Multiplicity adjustment is performed for the overall type I error rate control.

KW - Multiplicity adjustment

KW - combination test

KW - conditional power

KW - estimation of treatment effect

UR - https://www.scopus.com/pages/publications/85066629005

U2 - 10.1080/03610926.2019.1618871

DO - 10.1080/03610926.2019.1618871

M3 - Article

AN - SCOPUS:85066629005

SN - 0361-0926

VL - 49

SP - 5436

EP - 5454

JO - Communications in Statistics - Theory and Methods

JF - Communications in Statistics - Theory and Methods

IS - 22

ER -

Seamless phase II/III/IIIb clinical trial designs with different endpoints for different phases

Abstract

Keywords

Access to Document

Fingerprint

Cite this