BACKGROUND AND PURPOSE: Preclinical and retrospective clinical data indicate 
that glyburide, a selective inhibitor of sulfonylurea receptor 1-transient 
receptor potential melastatin 4, is effective in preventing edema and improving 
outcome after focal ischemia. We assessed the feasibility of recruiting and 
treating patients with severe stroke while obtaining preliminary information on 
the safety and tolerability of RP-1127 (glyburide for injection).
METHODS: We studied 10 patients with acute ischemic stroke, with baseline 
diffusion-weighted imaging lesion volumes of 82 to 210 cm3, whether treated with 
intravenous recombinant tissue-type plasminogen activator, age 18 to 80 years, 
and time to RP-1127≤10 hours.
RESULTS: Recruitment was completed within 10 months. The mean age was 50.5 
years, and baseline diffusion-weighted image lesion volume was 102±23 cm3. There 
were no serious adverse events related to drug and no symptomatic hypoglycemia. 
The increase in ipsilateral hemisphere volume was 50±33 cm3. The proportion of 
90-day modified Rankin Scale≤4 was 90% (40% modified Rankin Scale, ≤3).
CONCLUSIONS: RP-1127 at a dose of 3 mg/d was well tolerated and did not require 
any dose reductions. A clinical trial of RP-1127 is feasible.
CLINICAL TRIAL REGISTRATION: URL: http://www.clinicaltrials.gov. Unique 
identifier: NCT01268683.