A Profile-Based Stratified Randomization and Its Application to a Double-Blind, Placebo-Controlled Clinical Trial

• Activity Number: 435
• Type: Contributed
• Date/Time: Tuesday, August 11, 2015 : 2:00 PM to 3:50 PM
• Sponsor: Biopharmaceutical Section
• Author(s): Andrew Francis Magyar* and Jihao Zhou and Brenda Jenkins and Cornelia Haag-Molkenteller
• Companies: Allergan, Inc. and Allergan, Inc. and Allergan, Inc. and Allergan, Inc.
• Keywords: propensity score ; stratified randomization ; urinary incontinance ; neurogenic detrusor overactivity ; multiple sclerosis

Abstract

Stratified randomization is a common technique used in clinical trials to balance important baseline characteristics between treatment and control groups. However, as the number of stratification factors increases, the number of strata will become too large to handle in practice. A double-blind, placebo-controlled study (NCT01600716) was designed to investigate a compound for the treatment of urinary incontinence due to neurogenic detrusor overactivity in patients with multiple sclerosis, in which there are 4 stratification factors: age (< 55/>=55 years), baseline urinary incontinence episodes (< =9/>9), MS duration (< 12/>=12 years) and concurrent anticholinergic treatment use (Yes/No). To determine the strata for randomization, we proposed a profile-based stratification method, which used each subject's baseline profile determined using a propensity score. The parameters in the propensity score function was estimated based on the MS patients' data pooled from two similar previous Phase 3 clinical trials.