Ampio Pharmaceuticals has dosed the first patient in a multicentre clinical trial of its Optina drug for the treatment of diabetic macular edema (DME).
Optina is an oral drug based on a low-dose of the weak androgen, low molecular weight, very lipophilic steroid, danazol.
Ampio Pharmaceuticals chairman and CEO Michael Macaluso said; "Based on the encouraging results from the Canadian study, we are very excited about Optina’s potential to allow diabetic patients to avoid direct injections into the eye, a requirement of all other treatment regimens."
The trial, which will enrol 450 patients, will evaluate the safety and efficacy of oral Optina compared with placebo given over a period of 12 weeks in adult patients with DME.
Patients will be randomised to receive treatment with either one of two oral doses of Optina (0.5mg per BMI and 1.0mg per BMI per day), or placebo.
The company said after completion of four weeks of initial treatment, an interim analysis will be conducted to determine the best dose of Optina.
Following the 12 week active treatment period, there will be a further four-week washout period to determine the regression of treatment effect, according to the company.
Improvement in visual acuity (VA) compared to placebo is the primary endpoint of the study, while measurements of changes in VA and central macular thickness (CMT) in treated patients compared to placebo, as well as safety and tolerability of the two Optina doses,are secondary endpoints.
After completion of the treatment and washout, patients will be assessed for vision regression, and a 12 week open-label extension study will be carried out to demonstrate the duration of effect of the optimal dose.