Idiopathic intracranial hypertension (IIH), also called pseudotumor cerebri, is a disorder of
elevated intracranial pressure of unknown cause [Corbett, et al., 1982; Wall, et al., 1991].
Its incidence is 22.5 new cases each year per 100,000 overweight women of childbearing age,
and is rising [Garrett, et al., 2004] in parallel with the obesity epidemic. It affects about
100,000 Americans. Most patients suffer debilitating headaches. Because of pressure on the
optic nerve (papilledema), 86% have some degree of permanent visual loss and 10% develop
severe visual loss [Wall, et al., 1991]. Interventions to prevent loss of sight, all with
unproven efficacy, include diet, diuretics such as acetazolamide, repeated spinal taps, optic
nerve sheath fenestration surgery, and cerebrospinal fluid (CSF) shunting procedures. The
purported goal of these therapies is to lower intracranial pressure; however, it is unclear
which treatments work and by what mechanism. None of these strategies has been verified by
properly designed clinical trials. Thus, there is confusion, uncertainty, and weak scientific
rationales to guide treatment decisions. This trial will study subjects who have mild visual
loss from IIH to (1) establish convincing, evidence-based treatment strategies for IIH to
restore and protect vision, (2) follow subjects up to 4 years to observe the long-term
treatment outcomes and (3) determine the cause of IIH. To meet those aims, the trial will be
divided into a 12-month intervention phase and a 3-year observational phase. Subjects are not
required to complete the observational phase of the study, but will be asked to do so and
consented for the observational phase of the study at the conclusion of the intervention
phase (12 months).
Phase:
Phase 2/Phase 3
Details
Lead Sponsor:
St. Luke's-Roosevelt Hospital Center
Collaborators:
National Eye Institute (NEI) University of California, Davis University of Iowa University of Rochester