Tirzepatide in Idiopathic Intracranial Hypertension Trial

Summary

This will be a randomized, double-blind, parallel, placebo-controlled trial of 60 participants. The primary analysis will be a statistical comparison of the estimates of the mean difference in the 12-month change in intracranial pressure (ICP) between participants assigned to the tirzepatide and Placebo arms in 1:1 randomization using the intention-to-treat (ITT) population. Hypothesis testing will be performed using analysis of covariance (ANCOVA), with the treatment indicator as the independent variable and the 12-month change in ICP as the dependent variable. Models will include baseline ICP as a covariate. This primary endpoint will use a significance level of 0.05 to declare significance.

Timeline

Start

2026-08-25

Primary completion

2027-08

Completion

2028-08

Publications

• Background Spitze A, Lam P, Al-Zubidi N, Yalamanchili S, Lee AG. Controversies: Optic nerve sheath fenestration versus shunt placement for the treatment of idiopathic intracranial hypertension. Indian J Ophthalmol. 2014 Oct;62(10):1015-21. doi: 10.4103/0301-4738.146012.
• Background Chen J, Wall M. Epidemiology and risk factors for idiopathic intracranial hypertension. Int Ophthalmol Clin. 2014 Winter;54(1):1-11. doi: 10.1097/IIO.0b013e3182aabf11. No abstract available.
• Background Mollan SP, Ali F, Hassan-Smith G, Botfield H, Friedman DI, Sinclair AJ. Evolving evidence in adult idiopathic intracranial hypertension: pathophysiology and management. J Neurol Neurosurg Psychiatry. 2016 Sep;87(9):982-92. doi: 10.1136/jnnp-2015-311302. Epub 2016 Feb 17.
• Background Miah L, Strafford H, Fonferko-Shadrach B, Hollinghurst J, Sawhney IMS, Hadjikoutis S, Rees MI, Powell R, Lacey A, Pickrell WO. Incidence, Prevalence, and Health Care Outcomes in Idiopathic Intracranial Hypertension: A Population Study. Neurology. 2021 Feb 22;96(8):e1251-e1261. doi: 10.1212/WNL.0000000000011463.
• Background Mollan SP, Aguiar M, Evison F, Frew E, Sinclair AJ. The expanding burden of idiopathic intracranial hypertension. Eye (Lond). 2019 Mar;33(3):478-485. doi: 10.1038/s41433-018-0238-5. Epub 2018 Oct 24.

Drugs

Indications