Phase 3 NT-501 Trial

CNTF-Secreting Cells for Treatment of MacTel

Neurotech Pharmaceuticals, with support from the Lowy Medical Research Institute, is testing the safety and efficacy of ciliary neurotrophic factor (CNTF) in patients with macular telangiectasia type 2 in a Phase 3 clinical trial.  This is a multi-center trial, with clinical sites in the United States, Europe, and Australia.

Ciliary neurotrophic factor is a therapeutic macromolecule that has been tested in numerous preclinical applications for its ability to decrease photoreceptor degeneration in disease models of the eye. Among the many growth factors, cytokines, and neurotrophic factors that have been tested for their ability to minimize photoreceptor loss, CNTF is among the most effective. However, CNTF degrades relatively quickly when injected into the eye. Therefore, it requires a special delivery device.

Neurotech developed an encapsulated cell technology implant (NT-501) to address this problem. The device is surgically implanted into the vitreous, which is the clear jelly-like substance that fills the area between the retina and the lens. The NT-501 device continuously delivers CNTF into the vitreous cavity of the eye. It results in controlled, long-term delivery of CNTF. From the vitreous, CNTF can diffuse to the retina, which is the part of the eye affected by MacTel.

Based on promising results from the Phase 1 and Phase 2 trials, LMRI and Neurotech are conducting a Phase 3 clinical trial. Participants enrolled in the Phase 3 trial will be followed for two years with a total of nine visits. This is a Treatment/Sham (placebo) study.

The Phase 1 and Phase 2 participants continue to be followed in a long-term extension study.

Enrollment in the Phase 3 trial is now closed.