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Saturday / June 22.
HomeminewsPhase 2b Trial for Stargardt Treatment

Phase 2b Trial for Stargardt Treatment

The first patient has been enrolled in the phase 2b randomised, double-masked, sham-controlled clinical trial assessing the efficacy and safety of Zimura (avacincaptad pegol), complement C5 inhibitor, in patients with autosomal recessive Stargardt disease (STGD1).

STGD1 is an inherited orphan retinal disease causing vision loss during childhood or adolescence and there are currently no FDA or EMA approved treatment options available.

Ophthotech expects to enrol 120 patients in its trial, which will take place in 30 sites, making it one of the largest interventional clinical trials for STGD1 to date.

Recent scientific literature emphasises the role of complement in Stargardt disease, supporting our strategy for the development of Zimura in this devastating orphan retinal condition,” said Dr. Kourous A. Rezaei, Senior Vice President and Chief Medical Officer of Ophthotech. “Our work with the highly-distinguished organisation, Foundation Fighting Blindness, provided us access to publicly available data from ProgStar, the largest natural history study in autosomal recessive Stargardt disease to date, playing an integral role in the design of our clinical trial.”

The company plans to use an anatomic endpoint, the mean rate of change in the area of ellipsoid zone defect as measured by en face spectral domain optical coherence tomography (SD-OCT), as the primary endpoint, which will be assessed at 18 months. Initial top-line data is expected to be available in 2020.