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Assessing the Efficacy and Safety of an 11β-Hydroxysteroid Dehydrogenase Type 1 Inhibitor (AZD4017) in the Idiopathic Intracranial Hypertension Drug Trial, IIH:DT: Clinical Methods and Design for a Phase II Randomized Controlled Trial

Markey, Keira Annie; Ottridge, Ryan; Mitchell, James L; Rick, Caroline; Woolley, Rebecca; Ives, Natalie; Nightingale, Peter; Sinclair, Alexandra J

Assessing the Efficacy and Safety of an 11β-Hydroxysteroid Dehydrogenase Type 1 Inhibitor (AZD4017) in the Idiopathic Intracranial Hypertension Drug Trial, IIH:DT: Clinical Methods and Design for a Phase II Randomized Controlled Trial Thumbnail


Authors

Keira Annie Markey

Ryan Ottridge

James L Mitchell

Rebecca Woolley

Natalie Ives

Peter Nightingale

Alexandra J Sinclair



Abstract

Background: Idiopathic intracranial hypertension (IIH) is a condition with few effective management options. So far, there have been no randomized controlled trials evaluating new treatments in IIH.

Objectives: The purpose of this paper is to outline the trial design for the Idiopathic Intracranial Hypertension Drug Trial (IIH:DT), assessing an innovative medical treatment in IIH and the rationale for the chosen trial methodology.

Methods: IIH:DT is a phase II double-blind randomized placebo-controlled trial recruiting 30 female participants with active IIH (intracranial pressure >25cm H2 O and papilledema). Participants are randomized in a 1:1 ratio to 12 weeks of either AZD4017, an 11β-hydroxysteroid dehydrogenase type 1 inhibitor, or a matching placebo. They receive either 400 mg of AZD4017 or placebo twice daily. Participants are followed up at Weeks 1, 2, 3, 4, 6, 8, 10, 12, and 16 postrandomization. The primary outcome is to examine the effect of AZD4017 on intracranial pressure, measured by lumbar puncture, over 12 weeks. Secondary outcome measures include IIH symptoms, visual function, papilledema, headache measures, safety, and tolerability. Cerebrospinal fluid, serum, plasma, urine, and adipose tissue are also taken for exploratory outcomes.

Results: All participants were recruited between April 2014 and August 2016.

Conclusions: IIH:DT is the first phase II double-blind randomized placebo-controlled trial assessing the efficacy and safety of the novel pharmacological intervention, AZD4017, for the treatment of IIH.

Trial Registration: Clinicaltrials.gov NCT02017444; https://clinicaltrials.gov/ct2/show/NCT02017444 (Archived by WebCite at http://www.webcitation.org/6tVHesN6s)

Citation

Markey, K. A., Ottridge, R., Mitchell, J. L., Rick, C., Woolley, R., Ives, N., …Sinclair, A. J. (2017). Assessing the Efficacy and Safety of an 11β-Hydroxysteroid Dehydrogenase Type 1 Inhibitor (AZD4017) in the Idiopathic Intracranial Hypertension Drug Trial, IIH:DT: Clinical Methods and Design for a Phase II Randomized Controlled Trial. JMIR Research Protocols, 6(9), 1-10. https://doi.org/10.2196/resprot.7806

Journal Article Type Article
Acceptance Date Sep 18, 2017
Online Publication Date Sep 18, 2017
Publication Date Sep 18, 2017
Deposit Date Jun 27, 2019
Publicly Available Date Jul 2, 2019
Journal JMIR Research Protocols
Electronic ISSN 1929-0748
Publisher JMIR Publications
Peer Reviewed Peer Reviewed
Volume 6
Issue 9
Article Number e181
Pages 1-10
DOI https://doi.org/10.2196/resprot.7806
Keywords 11beta-HSD1; randomised controlled trial; clinical protocol; idiopathic intracranial hypertension; clinical trials, Phase II
Public URL https://nottingham-repository.worktribe.com/output/2235677
Publisher URL https://www.researchprotocols.org/2017/9/e181/
Contract Date Jul 2, 2019

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