Combining proof-of-concept with dose-finding: utilization of adaptive designs in migraine clinical trials.
There is an obvious need to improve clinical trial designs with respect to efficiency, duration and the number of patients recruited. Adaptive (flexible) designs may be valuable in this respect. We simulated the properties of a two-stage adaptive proof-of-concept and dose-finding trial design in adult migraine patients with moderate to severe headache, with or without aura. We also assessed the usefulness of a combined Bayesian and frequentist approach in the estimation of the probability of success of subsequent Phase III studies. Applying such an innovative approach would result in a reduction of the required sample size by 30 patients and no prolongation of the trial duration. The probability of success in Phase III is > 81%. An innovative adaptive design can facilitate testing of investigational migraine medications by reducing patient numbers and improving predictivity of success in Phase III.
Boehringer Ingelheim Pharma GmbH & Co. KG, Ingelheim, Germany.
A
A
Sagkriotis
J
J
Scholpp
D002986:Clinical Trials as Topic / Q000379:methods*; D003627:Data Interpretation, Statistical; D004305:Dose-Response Relationship, Drug; D019317:Evidence-Based Medicine / Q000379:methods*; D006801:Humans; D008881:Migraine Disorders / Q000175:diagnosis / Q000188:drug therapy* / Q000453:epidemiology*; D010865:Pilot Projects; D012107:Research Design; D018401:Sample Size