Acetazolamide Trial in Normal Pressure Hydrocephalus

Summary

A pharmacological treatment to patients with normal pressure hydrocephalus (NPH) is missing. The aim is to investigate if acetazolamide given to patients with NPH improves gait function and study the pathophysiological mechanisms leading to reduced symptoms. Patients will be randomized to acetazolamide or placebo and duration of treatment will be from diagnosis to the day of shunt surgery. Target dose is 500 mg/day. Study design is a double-blind randomized controlled trial and the plan is to include 42-50 patients. The study is investigator-initiated without financial sponsorship from the industry.

Timeline

Start

2022-02-17

Primary completion

2024-11-18

Completion

2026-04-21

Publications

• Background Aimard G, Vighetto A, Gabet JY, Bret P, Henry E. [Acetazolamide: an alternative to shunting in normal pressure hydrocephalus? Preliminary results]. Rev Neurol (Paris). 1990;146(6-7):437-9. French.
• Background Adams RD, Fisher CM, Hakim S, Ojemann RG, Sweet WH. SYMPTOMATIC OCCULT HYDROCEPHALUS WITH "NORMAL" CEREBROSPINAL-FLUID PRESSURE. A TREATABLE SYNDROME. N Engl J Med. 1965 Jul 15;273:117-26. doi: 10.1056/NEJM196507152730301. No abstract available.
• Background Garcia-Gasco P, Salame Gamarra F, Tenllado Doblas P, Chazarra Talens C. [Complete resolution of chronic hydrocephalus of adult with acetazolamide]. Med Clin (Barc). 2005 Apr 9;124(13):516-7. doi: 10.1157/13073568. No abstract available. Spanish.
• Background Alperin N, Oliu CJ, Bagci AM, Lee SH, Kovanlikaya I, Adams D, Katzen H, Ivkovic M, Heier L, Relkin N. Low-dose acetazolamide reverses periventricular white matter hyperintensities in iNPH. Neurology. 2014 Apr 15;82(15):1347-51. doi: 10.1212/WNL.0000000000000313. Epub 2014 Mar 14.
• Background Relkin N, Marmarou A, Klinge P, Bergsneider M, Black PM. Diagnosing idiopathic normal-pressure hydrocephalus. Neurosurgery. 2005 Sep;57(3 Suppl):S4-16; discussion ii-v. doi: 10.1227/01.neu.0000168185.29659.c5.
• Background Hellstrom P, Klinge P, Tans J, Wikkelso C. A new scale for assessment of severity and outcome in iNPH. Acta Neurol Scand. 2012 Oct;126(4):229-37. doi: 10.1111/j.1600-0404.2012.01677.x. Epub 2012 May 16.
• Virhammar J, Fasth O, Ekblom M, Ekman O, Holmstrand N, Fallmar D, Nyholm D. Safety, tolerability, and efficacy of acetazolamide in idiopathic normal pressure hydrocephalus (DRAIN) in Sweden: a randomised, double-blind, placebo-controlled, phase 2 trial. Lancet Neurol. 2026 Jun;25(6):550-559. doi: 10.1016/S1474-4422(26)00126-2.

Drugs

Indications