Clinical Trial: Anagrelide Retard vs. Placebo: Efficacy and Safety in "At-risk" Patients With Essential Thrombocythaemia

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: A Phase III, Randomized, Multicenter, Subject and Sponsor-blinded, Placebo Controlled Study to Compare the Efficacy and Safety of "Anagrelide Retard" Versus Placebo in "at Risk" Su

Brief Summary:

This is a multicenter, phase III, randomized, subject and sponsor-blinded, placebo-controlled study to determine the treatment effect of "Anagrelide retard" in subjects with Essential Thrombocythaemia (ET) at "defined risk" (definition of risk criteria: see Inclusion Criteria Section 5.1) The study is planned as a 2-stage procedure according to Bauer and Köhne: After recruitment of 140 subjects an interim analysis with re-assessment of sample size is planned in an adaptive manner.

As the confirmatory analysis will be based on a time-to-event evaluation (i.e. time to 1st clinically significant ET related event), there is no stipulated observation time identically applying for all subjects. Yet, with an interim analysis being performed after having recruited 140 subjects - which is expected to be reached after 1 year - the estimated observation time for a subject in stage I will also be about 1 year. (Details are explained in the section "Statistical Considerations").

Subjects will be randomized in a 1:1 ratio to one of the following two arms:

Group A: Anagrelide retard Group B: Placebo

An a priori stratification is planned for the JAK-2 mutational status. For exploratory purposes a post hoc stratification is used for obtaining covariate adjusted results, for the following other potentially predictive factors: sex, age, Factor V Leiden, and BMI.

Dosing will be started with 1 tablet per day for week 1 and will be titrated up according to response (platelet reduction) to 2 tablets in week 2. Dosing may be further increased or decreased according to platelet response in week 3 and 4. However, the maximum dose is 4 tablets (=8mg) per day. After we

Detailed Summary:
Sponsor: AOP Orphan Pharmaceuticals AG

Current Primary Outcome: Time to 1st clinically significant ET related event [ Time Frame: beginning 2012 ]

Original Primary Outcome:

Current Secondary Outcome: Efficacy and safety [ Time Frame: end 2013 ]

Original Secondary Outcome:

Information By: AOP Orphan Pharmaceuticals AG

Dates:
Date Received: October 28, 2010
Date Started: December 2010
Date Completion:
Last Updated: May 25, 2016
Last Verified: May 2016