A Phase IV Placebo-controlled Single Center Trial for Tirzepatide in Idiopathic Intracranial Hypertension Trial (TIIHT)
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.
• Signs and symptoms of increased intracranial pressure:
‣ headaches, tinnitus, visual obscurations, papilledema;
⁃ absence of localizing findings on neurological examination (except for VI nerve palsy);
⁃ no secondary causes identified on imaging, e.g., hydrocephalus, space- occupying lesion;
⁃ elevated lumbar puncture (LP) opening pressure (OP) ≥25 cm H2O in lateral decubitus position with legs extended (\>= (≥ 20cm H2O if one of the following is present: pulse synchronous tinnitus, abducens palsy, Frisen grade II papilledema, transverse venous sinus stenosis, partially empty sella or enlarged optic nerve sheath on magnetic resonance imaging (MRI);
⁃ the patient is awake and alert.
• BMI ≥30 kg/m2
• Age 18-60 years of age
• Unilateral or bilateral papilledema
• Able to provide informed consent
• Women of child-bearing age must use birth control (non-oral contraceptive method or add a barrier method of contraception).