Ampio Pharmaceuticals has informed the Transplant and Ophthalmology Division of the FDA of its intent to reduce the patient sample size in the OptimEyes study for the treatment of diabetic macular edema (oral treatment with Optina). This trial was intended to enroll 450 patients and was powered at 95%. The present enrollment of over 355 patients provides an adequate power of 88%, which the company believes is more than sufficient for statistical evaluation.
"There have been anecdotal reports from several principal investigators that Optina may be providing benefit to patients," said Dr. Vaughan Clift, chief medical officer. "The decision to reduce the sample size of the trial took into consideration requests from these site investigators, asking to extend the open label enrollment portion (where all patients receive active drug dose) of the trial beyond the 12 weeks allowed for in the protocol.
“These clinicians believed that their patients were improving while on the active drug and subsequently regressed quickly once they completed the open label phase. The enrollment in the open label extension phase was much higher than expected. Since DME is a very serious and progressive condition that often leads to blindness, the company believes it is in the patient's best interest to analyze the data as soon as possible to confirm any observed benefits. By reducing the patient sample size, we can expect data by Q4."
Conducted in 22 U.S. Sites, this study was designed to explore the potential benefits of administering oral Optina to patients diagnosed with diabetic macular edema. The primary end point was Best Corrected Visual Acuity, (BCVA) and a secondary endpoint of Ocular Coherence Tomography (OCT). Once the patients completed the 12 week masked placebo controlled trial, there was a four week wash out period, then patients could volunteer for an open label extension and receive only the active dose of Optina.